* CHAPTER 6. Choice of the donor according to HLA typing and stem cell source. Eliane Gluckman

Size: px
Start display at page:

Download "* CHAPTER 6. Choice of the donor according to HLA typing and stem cell source. Eliane Gluckman"

Transcription

1 * CHAPTER 6 Choice of the donor according to HLA typing and stem cell source Eliane Gluckman

2 CHAPTER 6 Choice of the donor according to HLA typing and stem cell source 1. Introduction Allogeneic haematopoietic stem cell transplantation (allohsct) is widely used to treat patients with malignant and nonmalignant haematological disorders. Initially, the principal source of HSCs was bone marrow (BM) from an HLAidentical sibling for transplantation in children and young adults. Subsequently, the choice of donors and the sources of HSCs have enlarged, extending transplant indications to more patients, especially adults. Today, transplant physicians must choose among stem cell sources between bone marrow, granulocyte colonystimulating factor (G CSF)mobilised peripheral blood stem cells (PBSC), or umbilical cord blood (UCB). The donor can be an HLA identical sibling, an matched unrelated donor (MUD), a haploidentical family peripheral blood stem cell (PBSC) or bone marrow donor or, an HLAmismatched unrelated UCB donor. 2. Use of different cell sources for HSCT in Europe Baldomero et al. performed an EBMT survey of HSCT activity in 2009 and analysed the trends over the previous 5 years (1, 2). They collected reports of 31,322 HSCT from 624 centres from 43 countries of which 28,033 were first transplants (41% allogeneic and 59% autologous). The main indications were leukaemias (31%; 92% allogeneic), lymphomas (58%; 12% allogeneic), solid tumours (5%; 6% allogeneic) and nonmalignant disorders (6%; 88% allogeneic). For allogeneic HSCT there were more unrelated than HLAidentical sibling donors (51% vs 43%). The proportion of peripheral blood as stem cell source was 99% for autologous and 71% for allogeneic HSCT. Unrelated cord blood was used in 756 cases (7%). For further details, see the EBMT activity survey at Figure 1 shows World Marrow Donor Association (WMDA) data on the use of different stem cell sources worldwide. 3. Choice of the stem cell source Traditionally, haematopoietic stem cells were harvested from the posterior iliac crests under general anaesthesia. More recently, mobilised peripheral blood stem cells (PBSC) have been increasingly used in both auto and allohsct. Mobilisation of haematopoietic stem cells to the peripheral blood in sufficient numbers can be achieved by the classical administration of growth factors such as GCSF (allohsct) and/or myelosuppressive chemotherapy (autohsct). Unmanipulated cord blood (CB) cells collected and cryopreserved at birth have been used both in related and unrelated HLAmatched and mismatched allogeneic transplants in children, and more recently in adults. Comparison of the yield of various cell sources is given in Table 1. In all cases, safety of the donor is the major concern meaning that the precollection workup should be particularly meticulous, and should be performed by HAEMATOPOIETIC STEM CELL TRANSPLANTATION 91

3 Figure 1: Number of BM, PBSC and CBU donations according to continents Number of BM and PBSC donations 6,000 5,000 4,000 3,000 2,000 1, Year Africa Asia Australia Europe North America South America Number of CBUs provided 1,600 1,400 1,200 1, Year Africa Asia Australia Europe North America South America WMDA data a trained experienced doctor. Informed consent must be fully and deeply explained, and all the risks must be discussed. In some countries, this medical examination is performed by a different medical team (e.g. anaesthetists). All possible medical contraindications must be looked for and no risk should be taken however great is the need of the patient. In some countries a special procedure is applied for child donors. It is a requirement to establish a central reporting system to collect adverse events and longterm followup of the donors (3, 4). 92 THE EBMT HANDBOOK 2012 EDITION

4 CHAPTER 6 Choice of the donor according to HLA typing and stem cell source Table 1 : Number of cells according to stem cell source Volume collected Med CD34 content Med CD3 content Target cell dose Bone marrow ml/kg 2 3 x 10 6 /kg* 25 x 10 6 /kg >2 x 10 8 TNC/kg Peripheral blood ml 8 x 10 6 /kg 250 x 10 6 /kg 5 10 x 10 6 CD34 + /kg Umbilical cord blood ml 0.2 x 10 6 /kg 2.5 x 10 6 /kg >3 x 10 7 TNC/kg *per kg recipient body weight 3.1 HLA identical sibling bone marrow transplant When available, this is the preferred source of HSCs; typically bone marrow cells are harvested under general anaesthesia in the posterior iliac crest. There are no contraindications related to age; collections have been performed at all ages from a few months to elderly adults. As long as general anaesthesia can be given. The yield is better in young children and adults and decreases with age. The volume of collection must be adjusted to the donor and recipient weight but should not exceed ml/kg donor body weight. Collection teams should not aspirate a volume more than 5 ml for each aspiration to avoid dilution of the bone marrow with blood. After collection, the bone marrow is filtered and volume reduced and is then infused iv directly to the patient. 3.2 Peripheral blood stem cell transplant GCSF mobilised PBSC have been recently used more frequently for HLA identical sibling transplants. The indications for using PBSC rather than BM are not really standardised, but many factors are involved: availability of operating room, lack of personnel to perform bone marrow aspiration, contraindication to general anaesthesia and choice of the donor. Most teams avoid collecting PBSCs in young children because of possible side effects of GCSF or problems of venous access. According to NMDP, 76% of adult unrelated donors donated GCSF mobilised PBSC. Severe but rare adverse effects have been observed including splenic rupture and sickle cell crisis. There are no definitive conclusions regarding the longterm effects of GCSF treatment on healthy donors (4). More recently plerixafor, a novel small molecule antagonist, which reversibly inhibits the interaction of SDF1 and CXCR4, has been used in poor mobilisers with or without GCSF. It has not been tested in healthy donors (5). Recently biosimilars of GCSF appeared in the market. In view of the absence of longterm studies their use is not recommended in healthy normal donors (6). 3.3 Haploidentical related HSCT donors The number of related haploidentical donors is increasing, but there are very few large comparative or randomised studies (7, 8). HAEMATOPOIETIC STEM CELL TRANSPLANTATION 93

5 Historically, the first haploidentical transplants pioneered by the Perugia group utilised GCSF mobilised Tcell depleted CD34 + cells. More recently, interesting preliminary data have been reported with new protocols using unmanipulated bone marrow cells or marrow harvested after in vivo GCSF, together with high dose immunosuppression or cyclophosphamide (9 12). 3.4 Matched unrelated HSCT donors The number of matched unrelated donor transplants has been increasing worldwide, with more than 18 million adult donors registered in Bone Marrow Donor Worldwide (BMDW; This collection of HLA phenotypes from bone marrow donor registries and cord blood banks give the opportunity to transplant physicians from around the world to perform preliminary searches through the internet for a preliminary view of the availability of potential suitable donors (Figure 1 and Figure 2). The annual report of the WMDA (www.wmda.org) is a product of the Donor Registries Figure 2: Number of bone marrow and cord blood units worldwide 300 Umbilical cord blood units, n= 450,000 Thousands Millions Adult marrow donors, n= 14,000,000 From WMDA and BMDW 94 THE EBMT HANDBOOK 2012 EDITION

6 CHAPTER 6 Choice of the donor according to HLA typing and stem cell source Working Group whose mission is to develop guidelines for donor recruitment and maintenance of donor confidentiality, track the efficiency of donor searches and develop consensus standards for the logistics of transporting stem cell products across international borders (13, 14). The standards by which stem cell products are collected and transported result from the collaborative efforts of several organisations represented within the Worldwide Network for Blood and Marrow Transplantation (www.wbmt.org). Most often the donors are chosen on the basis of high resolution allelic typing for HLAA, B, C, DRB1, DQB1, DPB1. The best choice is a 9/10 or 10/10 donor. In the case of several potential donors, it is better to recruit a donor who is male, young, ABO matched and CMV negative if the recipient is negative or CMV positive if the recipient is positive. The choice between bone marrow and PBSC depends on donor choice, centre preference and indication for HSCT. It is known that using PBSC gives more GvHD which is favourable in malignant diseases where increasing GvHD decreases the risk of relapse while in nonmalignant diseases, especially in aplastic anaemia, it decreases survival (15). 3.5 Cord blood Cord blood bank organisation Since the first human cord blood transplant, performed in 1988, cord blood banks have been established worldwide for collection and cryopreservation of cord blood for allogeneic haematopoietic stem cell transplant (16). Today, a global network of cord blood banks and transplant centres has been established and provides a common inventory. Several studies have shown that the number of cells is the most important factor for engraftment, while some degree of HLA mismatch is acceptable (16, 17). The absence of ethical concern and the unlimited supply of cells explains the increasing interest in using cord blood for developing regenerative medicine. Since the first UCBT, more than 20,000 CBT have been reported worldwide and more than 600,000 cord blood units have been stored in more than 100 cord blood banks (www.bmdw.org, (18). The main practical advantages of using cord blood as an alternative source of stem cells are the relative ease of procurement, the absence of risk for mothers and donors, the reduced likelihood of transmitting infections, particularly CMV, and the ability to store fully tested and HLA typed potential transplants in the frozen state, available for immediate use. Eurocord was established in 1995; its principal objectives were to collect outcomes data provided by cord blood banks and transplant centres. Eurocord has collected, from 1988 to October 2010, 6736 UCBT from Europe and transplant centres in other countries. Five hundred and ninetysix transplants have been reported using HAEMATOPOIETIC STEM CELL TRANSPLANTATION 95

7 related donors (majority HLA identical sibling donors), mainly for children with malignant and nonmalignant disorders and 6140 have been performed in the unrelated transplant setting for children (n=3287) and adults (n=2770). In order to promote education and information, Eurocord has launched a new European program entitled: an Online CME program in cord blood technology and transplantation for providing a learning tool on the scientific, technical, clinical, regulatory aspects of cord blood, easily accessible at a time and language convenient for users (www.eurocorded.org). Based on this international cooperation Eurocord has published crucial reports which have been the basis of the rapid development of cord blood transplant. Netcord (www.netcord.org) was created in 1998 to establish good practices in umbilical cord blood storage, facilitate donor search, improve the quality of the grafts, standardise excellence criteria on an international scale and importantly establish procedures for bank accreditation. The inventory of Netcord, the cooperative network of large experienced UCB banks, currently has more than 300,000 cryopreserved UCB units ready for clinical use for unrelated recipients and more than 8,624 grafts shipped. In the USA, the National Marrow Donor Program (NMDP) has established a similar cord blood bank network. Collaborations between Netcord Eurocord and NMDP have been established with the goal to provide the most appropriate and high quality cord blood unit for a specific patient. A summary of cord blood banks activity is available at (18). National regulatory agencies and transplant centres are aware of the need of international standards whose major objectives are to promote quality throughout all phases of cord blood banking with the goal to achieve consistent production of high quality placental and umbilical cord blood units for transplantation. All these aspects are detailed in the last version of the NetcordFACT Standards (www.factwebsite.org). As the number of cord blood units is increasing, it appeared that it was necessary to improve the quality of the units for costefficient management of the banks. The optimal number of cord blood units is not really known but should approach 9 per 10,000 inhabitants. Most banks prefer to bank only the largest units of more than 70 ml in order to obtain at least 3 x 10 7 nucleated cells/kg recipient bodyweight Criteria for cord blood donor choice In several studies including Eurocord, neutrophil and platelet recovery were associated with the degree of HLA mismatch, the number of total nucleated cells (TNC) collected and infused and the use of GCSF after transplant. Coexistence of HLA class I and II disparities and high CD34 + cell dose in the graft were associated with only with more severe GvHD grade IIIIV. Disease relapse was higher in matched transplants, showing a graft versus leukaemia effect (17 20). 96 THE EBMT HANDBOOK 2012 EDITION

8 CHAPTER 6 Choice of the donor according to HLA typing and stem cell source General considerations: Current HLA definition in cord blood banks is based on HLA serological typing for A and B and allelic typing for HLADRB1. Avoid cord blood units with 3 or 4 HLA disparities. At selection, diagnosis and presence of patient HLA antibodies against the HLA antigens of the cord blood unit should be considered. HLA compatibility appears to be more important for patients with nonmalignant disorders than for those with malignant disorders. If the criterion for the minimum number of cells for a single UCB transplantation is not achieved, a double cord blood transplantation should be considered. CFUGM and viability are generally not used for donor selection. Cell dose requirements must increase with the number of HLA mismatches. Cord blood units with 6/6 or 5/6 HLA match. HLAA or HLAB mismatches are preferable to DRB1 mismatches. In malignant disorders: Nucleated cell dose: at freezing, the cell dose should be >2.5 to 3.0 x 10 7 /kg after thawing, the cell dose should be >2.0 to 2.5 x 10 7 /kg. CD34 + cell dose: at freezing or after thawing, the dose should be approximately 1.2 to 1.7 x 10 5 /kg. Based on Eurocord unpublished and preliminary data, it seems likely that HLA DRB1 mismatch may lead to high graftversusleukaemia (GvL) effect in patients transplanted in nonremission (but there is also a greater risk of GvHD). In nonmalignant disorders: Higher total and CD34 + cell dose is requested, and HLA match is preferable. Cord blood units with 4/6 HLA match. HLAA or HLAB mismatches are better than HLADRB1 mismatches. In malignant disorders: Nucleated cell dose: at freezing >3.5 x 10 7 /kg after thawing >3.0 x 10 7 /kg. CD34 + cell dose: at freezing or after thawing, approximately >1.7 x 10 5 /kg HLADRB1 mismatch may lead to a high GvL effect in advanced phase of the diseases. In nonmalignant disorders: Nucleated cell dose: at freezing, minimum cell dose 4 to 5 x 10 7 /kg HAEMATOPOIETIC STEM CELL TRANSPLANTATION 97

9 after thawing, minimum 3.5 x 10 7 /kg. CD34 + cell dose: no available data, but should be higher than 2 to 2.5 x 10 5 /kg. CB units with 3/6 HLA match. Should be avoided, but in extremely severe cases may be considered for patients with malignant disorders; a high nucleated cell dose should be given. Not recommended for patients with nonmalignant disorders. Other considerations: If several cord blood units are available that fit the above criteria, the following should be taken into consideration: Cord blood bank accreditation status and location ABO compatibility Allele HLA typing of HLAA and B Other HLA factors such as HLAC, high resolution HLA typing, NIMA; KIR are under study and cannot be used at this stage for donor choice. Double cord blood transplants can be recommended if the cell dose is insufficient with a single CBU. The total dose of both combined units should be at least 3 x 10 7 TNC/kg. HLA matching between the 2 units and the recipient must be as matched as possible but no firm recommendation can be given. Note that double cord transplant gives a good engraftment and survival with more GvHD and less relapse than single cord blood transplant. 4. Comparison between stem cell sources and algorithm for donor search 4.1 Bone marrow compared to PBSC An EBMTinitiated prospective randomised trial that involved 350 patients with standardrisk leukaemia compared HLA identical sibling BM to PBSC and showed that the incidence of grades IIIV acute GvHD was significantly higher in patients who underwent PBSCT. There was a higher incidence of chronic GvHD after PBSCT (21). On the other hand, there were no significant differences in overall survival or leukaemiafree survival between the BMT and PBSCT groups at the 3year followup (5). A large retrospective International Bone Marrow Transplant Registry (IBMTR) and EBMT registry analysis involving 824 patients revealed the following data: the incidence of chronic GvHD at 1 year was significantly higher in patients after PBSCT, and treatmentrelated mortality rates and leukaemiafree survival rates were in favour of PBSCT in patients in advanced stages of leukaemia. A later retrospective EBMT 98 THE EBMT HANDBOOK 2012 EDITION

10 CHAPTER 6 Choice of the donor according to HLA typing and stem cell source registry analysis of 3465 adult patients with AML and ALL again revealed a higher incidence of chronic GvHD after PBSCT. However, rates of acute GvHD, leukaemiafree survival and overall survival were similar after BMT and PBSCT (5). In aplastic anaemia, 3 studies with HLA identical siblings or matched unrelated donors have shown that PBSC gave more GvHD and decreased survival (22, 23). Despite these results the number of PBSCT is increasing steadily. There are many possible reasons, including preference of the centres, since the logistics of organising PBSC collection are easier than organising bone marrow collection with general anesthaesia. One main reason advocated by the centres is donor safety, but a recent report on over 50,000 donations for PBSC in Europe documented a significantly higher rate of severe adverse events with PBSC collection, per 10,000 donations for PBSC compared with 4.32 per 10,000 donations for BM. Fatalities were also higher, though not significantly, in PBSC versus BM donors (4 versus 1 fatality) (5). 4.2 HLA identical siblings versus matched unrelated transplants To determine whether the risks of allogeneic transplantation are different when the donor is a fully matched unrelated donor based on 10/10 HLA alleles compared to an HLAidentical sibling, Woolfrey et al. performed a retrospective analysis of 1448 patients with highrisk or advanced haematologic malignancies given T replete grafts after myeloablative conditioning. No statistically differences were found between the two groups in survival, diseasefree survival and nonrelapse mortality for patients with highrisk disease or those given bone marrow as a graft source. However, for patients with intermediaterisk disease receiving peripheral blood grafts, they observed higher nonrelapse mortality and lower overall survival in the 10/10 MUD compared to matched sibling donors (24). A prospective French study showed no statistically significant difference between MUD and HLA matched sibling donors. The Essen group performed the same study in 101 patients who were given either a 10/10 MUD or a HLA identical sibling transplant and did not find any difference in survival. In both studies MUD transplant gave more GvHD. These studies show that use of a HLA 10/10 MUD is a good option when an HLA identical sibling donor is unavailable. A higher degree of HLA mismatches, 9 or 8/10, is often used but there are no strong data to suggest what mismatches are acceptable. 4.3 Cord blood versus unrelated bone marrow transplant In children, with malignant diseases, two studies have compared the outcome of unrelated UCBT and BMT (25, 26). Eurocord published a study comparing the outcome of matched unrelated BMT (HLA 6 out of 6), either unmanipulated or T depleted, with that of mismatched UCBT. Results showed that after UCBT, engraftment was delayed, GvHD was reduced similarly to Tcell depleted BMT and there was no HAEMATOPOIETIC STEM CELL TRANSPLANTATION 99

11 difference in relapse or in leukaemiafree survival. Eapen et al. for the CIBMTR and the New York Cord Blood Bank (NYCBB) compared the outcomes of 503 children with acute leukaemia given an unrelated mismatched UCBT with 282 unrelated BM transplant recipients. HLA allelemismatched BM recipients had more acute and chronic GvHD without decreasing leukaemiafree survival (LFS). Importantly, they found that even using an allelematched BM donor, LFS was not statistically different from one or 2 HLA disparate UCBT and that an HLAmatched UCBT recipient had better outcomes compared to HLA allelematched BM recipients. However, an increased transplant related mortality was observed in children transplanted with a low CB cell dose (<3 x 10 7 /kg) and a 1 HLA disparate CB graft or in children given a 2 HLA disparate UCBT, independently of the cell dose infused. Interestingly, use of 2 HLA mismatched UCBT was associated with a lower incidence of relapse (27). The same studies were performed in adults with malignancies. Eurocord compared adults with acute leukaemia receiving either a matched unrelated bone marrow transplant (HLA 6 out of 6) or a mismatched cord blood transplant. Results showed that, despite a delay of engraftment, UCBT gave a similar leukaemiafree survival to BMT. In the same issue of the journal, CIBMTR and NYCBB showed that, in adults with malignancies, UCBT gave the same LFS survival as 1 antigen mismatched unrelated bone marrow transplant (UBMT) (25, 26). At the same time, a Japanese study showed that CBT gave better results than matched unrelated bone marrow transplants. In a metaanalysis, combining these published studies, 161 children and 316 adults undergoing UCBT, along with 316 children and 996 adults undergoing UBMT were analysed. Tcell depleted UBMT was excluded where data were available, and only fully matched UBMT was used in the analysis. Pooled comparisons of studies of UCBT and UBMT in children found that the incidence of chronic GvHD was lower with UCBT, but the incidence of grade III IV acute GvHD did not differ. There was no difference in 2year overall survival in children when studies were pooled. For adults, transplantationrelated mortality (TRM) and LFS were not statistically different. Recently, Eurocord and CIBMTR performed a study comparing the outcome of unrelated HLAmatched or 12 antigen mismatched bone marrow (n=364) or GCSF mobilised peripheral blood (n=728) with that of mismatched cord blood transplant (n=148) in adults with acute leukaemia. In multivariate analysis, TRM was higher after UCBT, but relapse rate and GvHD were lower, resulting in the same LFS compared to the other sources of stem cells (Figure 3) (28, 29). The results of these comparative studies and the metaanalysis (30), gathered together, showed that UCBT is feasible in adults when a cord blood unit contains a high number of cells and should be considered an option as an allogeneic stem cell source for patients lacking a HLAmatched bone marrow donor; despite increased HLA disparity, UCB 100 THE EBMT HANDBOOK 2012 EDITION

12 CHAPTER 6 Choice of the donor according to HLA typing and stem cell source Figure 3: Leukaemiafree survival according to stem cell source Adjusted probability, % Leukaemiafree survival BM matched, 41% PBPC matched, 39% 70 UCB, 33% PBPC mismatched, 34% 30 BM mismatched, 34% Months Reproduced with permission from (29) from unrelated donors offers sufficiently promising results compared with matched UBMT in adults with haematological malignancies leading to the conclusion, as in children, that the donor search process for BM and UCB from unrelated donors should be started simultaneously, especially in patients with acute leukaemia, where the time factor is crucial (30, 31). Further improvement has been obtained by the use of double cord blood transplant and of reduced intensity conditioning regimens (31). These papers were the hallmark of the worldwide development of cord blood transplant as they clearly demonstrated that cord blood transplant could be used in adults as well as in children, and furthermore showed that unrelated mismatched cord blood transplant gave the same results as a HLA matched unrelated bone marrow transplant. 4.4 Related haploidentical transplants compared to other sources of hematopoietic stem cells There are very few publications on the longterm results of haploidentical related transplants. Although the technology has changed over the years, large randomised studies are needed. Retrospective comparative studies show that early results are comparable to other sources of cells but longterm results are not available. Leung et al. evaluated 190 children with very highrisk leukaemia who underwent allogeneic haematopoietic cell transplantation in two sequential treatment eras to determine HAEMATOPOIETIC STEM CELL TRANSPLANTATION 101

13 whether those treated with contemporary protocols had a lower risk of relapse or toxic death and whether nonhla identical transplantations yielded poorer outcomes (32). For the recent cohorts, 5year overall survival rates were 65% for the 37 patients with ALL and 74% for patients with AML. These rates compared favourably with those of earlier cohorts (28% and 34% respectively). The recent improvement was observed regardless of the cell source (5yr OS for matched sibling transplants 70 versus 24%, MUD 61 versus 37% and haploidentical 88 versus 19%). This improvement was attributable to a reduction in infections, in regimenrelated toxicity and in leukaemiarelated death. Survival probability was dependent on leukaemia status and minimal residual disease. The authors conclude that with the improvement of results over time, bone marrow transplantation should be considered for all children with very highrisk leukaemia regardless of matched donor availability. 5. Algorithm of donor choice and conclusion (see Figure 4) Results of allogeneic HSCT have been markedly improving over time, due to a better expertise of the centres, better supportive care and better prevention and treatment of infections. Figure 4: Algorithm of donor search Patient and family HLA typing HLA identical sibling: HSCT No HLA identical sibling: Allele typing for A, B, C, DRB1, DQB1, DPB1 Search for an unrelated donor in bone marrow registries and cord blood banks HLA 9/10 or 10/10 matched unrelated donor: HSCT Unrelated cord blood 3 x 10 7 TNC/kg and 1 2 HLA MM: HSCT Related haploidentical: HSCT If transplant urgent: prefer cord blood or related haploidentical transplant If not enough cells in a single cord blood unit: consider double cord Consider other factors: indication of the transplant, ABO, CMV, donor sex Expertise of the centres is very important for donor selection for HLA mismatched transplants 102 THE EBMT HANDBOOK 2012 EDITION

14 CHAPTER 6 Choice of the donor according to HLA typing and stem cell source Indications have increased because of the diminution of transplantrelated mortality due to reduced intensity conditioning adapted to disease status and comorbidities. Choice of donor source has extended the possibility of offering HSCT to almost all patients who need a transplant. The major recent advances are due to the use of HLA mismatched transplant using either cord blood or haploidentical family donors. Choice of donor source is dependent on the indication for HSCT, its urgency, the age of the patient, and the expertise and resources of the centre. HLA mismatched HSCT (haplomismatched or cord blood) should be performed in experienced centres. Centres who perform these transplants must report the outcomes to international registries in order to evaluate results. Transplant centres, transplant coordinators and the cell therapy laboratory should work very closely with bone marrow registries and cord blood banks in order to find the best possible donor source for each patient. References 1. Gratwohl H, Baldomero H, Aljurf M et al. Hematopoietic stem cell transplantation. A global perspective. JAMA 2010; 303: Baldomero H, Gratwohl M, Gratwohl A et al. The EBMT activity survey 2009: Trends over the past 5 years. Bone Marrow Transplant 2011; 46: Halter J, Kodera Y, Ispizua AU et al. Severe events in donors after allogeneic hematopoietic donation. Haematologica 2009; 94: Shaw AU, Ball L, Beksac M et al. Donor safety: The role of the WMDA in ensuring the safety of volunteer unrelated donors: Clinical and ethical considerations. Bone Marrow Transplant 2010; 45: Korbling M, Freireich EJ. 25 years of peripheral blood stem cell transplantation. Blood 2011; 117: Shaw BE, Confer DL, Hwang WY et al. Concerns about the use of biosimilar granulocyte colonystimulating factors for the mobilization of stem cells in normal donors: Position of the World Marrow Donor Association. Haematologica 2011; 96: Ballen KK, Spitzer TR. The great debate: Haploidentical or cord blood transplant. Bone Marrow Transplant 2011; 46: Barrett J, Gluckman E, Handgretinger R, Madrigal A. Pointcounterpoint haploidentical family donors versus cord blood transplantation. Biol Blood Marrow Transplant 2011; 17 (Suppl 1): S Tuve A, Gayoso J, Scheid C et al. Haploidentical bone marrow transplantation with post grafting cyclophosphamide multicenter experience with an alternative salvage strategy. Leukemia 2011; 25: XJ Huang, YJ Chang. Unmanipulated HLAmismatched/haploidentical blood and marrow hematopoietic stem cell transplantation. Biol Blood Marrow Transplant 2011; 17: HAEMATOPOIETIC STEM CELL TRANSPLANTATION 103

15 11. Leung W, Campana D, Yang J et al. High success rate of hematopoietic cell transplantation regardless of donor source in children with veryhighrisk leukemia. Blood 2011; 118: Lee KH, Lee JH, Lee JH et al. Reduced intensity conditioning therapy with busulfan, fludarabine and antithymocyte globulin for HLAhaploidentical hematopoietic cell transplantation in acute leukemia and myelodysplastic syndrome. Blood 2011; 118: Petersdorf EW. The world Marrow Donor Association: 20 years of international collaboration for the support of unrelated donor and cord blood hematopoietic cell transplantation. Bone Marrow Transplant 2010; 45: Foeken L, Green A, Hurley CK, Marry E, Wiegand T, Oudshoorn M, on behalf of the Donor Registries Working group of the World Marrow Donor Association (WMDA). Monitoring the international use of unrelated donors for transplantation: The WMDA annual reports. Bone Marrow Transplant 2010; 45: Eapen M, Le Rademacher J, Antin JH et al. Effect of stem cell source on outcomes after adult unrelated donor transplantation in severe aplastic anemia. Blood 2011; 118: Gluckman E, Ruggeri A, Volt F et al. Milestones in umbilical cord blood transplantation. Br J Haematol 2011; 154: Gluckman E, Rocha V, Arcese W et al. Factors associated with outcomes of unrelated cord blood transplant: Guidelines for donor choice. Exp Hematol 2004; 32: Welte K, Foeken L, Gluckman E, Navarrete C. International exchange of cord blood units: The registry aspects. Bone Marrow Transplant 45: , Barker JN, Byam C, Scaradavou A. How we search: A guide to the selection and acquisition of unrelated cord blood grafts. Blood 2011; 117: Barker JN, Scaradavou A, Stevens CE. Combined effect of total nucleated cell dose and HLA match on transplantation outcome in 1061 cord blood recipients with hematologic malignancies. Blood 2010; 115: Schrenzenmeier H, Passweg J, Marsh J et al. Worse outcome and more chronic GVHD with peripheral blood progenitor cells than bone marrow in HLAmatched sibling donor transplants for young patients with severe acquired aplastic anemia. Blood 2007; 110: Chu R, Brazauskas R, Kan F et al. Comparison of outcomes after transplantation of G CSF stimulated bone marrow grafts versus bone marrow or peripheral blood grafts from HLA identical siblings donors for patients with severe aplastic anemia. Biol Blood Marrow Transplant 2011; 17: Bacigalupo A. Back to the OR? Blood 118: Woolfrey A, Lee SJ, Gooley TA et al. HLAallele matched unrelated donors compared to HLA matched sibling donors: Role of cell source and disease risk category. Biol Blood Marrow Transplant 2010; 16: Rocha V, Labopin M, Sanz G et al. Transplants of umbilicalcord blood or bone marrow from unrelated donors in adults with acute leukemia. N Engl J Med 2004; 351: THE EBMT HANDBOOK 2012 EDITION

16 CHAPTER 6 Choice of the donor according to HLA typing and stem cell source 26. Laughlin MJ, Eapen M, Rubinstein P et al. Outcomes after transplantation of cord blood or bone marrow from unrelated donors in adults with leukemia. N Engl J Med 2004; 351: Eapen M, Rubinstein P, Zhang MJ et al. Outcomes of transplantation of unrelated donor umbilical cord blood and bone marrow in children with acute leukaemia: A comparison study. Lancet 2007; 369: Eapen M, Rocha V, Sanz G et al. on behalf of the center for International Blood and Marrow Transplant Research, the Acute Leukemia Working Party and Eurocord (the European Group for Blood and Marrow Transplantation) and the National Cord Blood program of the New York Blood Center. Effect of graft source on unrelated donor haematopoietic stemcell transplantation in adults with acute leukemia: A retrospective analysis. Lancet Oncol 2010; 11: Eapen M, Rocha V, Sanz G et al. Effect of graft source on unrelated donor haematopoietic stemcell transplantation in adults with adult leukaemia: A retrospective analysis. Lancet Oncol 2010; 11: Hwang WY, Samuel M, Tan D et al. A meta analysis of unrelated donor cord blood transplantation versus unrelated donor bone marrow transplantation in adult and pediatric patients. Biol Blood Marrow Transplant 2007; 13: Brunstein CG, Fuchs EJ, Carter SL et al. Alternative donor transplantation after reduced intensity conditioning: Results of parallel phase 2 trials using partially HLAmismatched related bone marrow or unrelated double umbilical cord blood grafts. Blood 2011; 118: Leung W, Campana D, Yang J et al. High success rate of hematopoietic cell transplantation regardless of donor source in children with very high risk leukemia. Blood 2011; 118: Multiple Choice Questionnaire To find the correct answer, go to 1. In recent years use of peripheral blood progenitor cells has surpassed bone marrow for allogeneic haematopoietic stem cell transplantation in Europe: a) True b) False Peripheral blood progenitor cell collections are associated with which of the following: a) Bone pain HAEMATOPOIETIC STEM CELL TRANSPLANTATION 105

17 b) Splenic rupture c) Haematoma d) All of the above The following characteristics are considered when selecting a cord blood unit for transplantation: a) Allelelevel HLA typing at class I and II b) Cell viability c) Total nucleated cell dose per kilogram of patient body weight d) b + c A 35 year old gentleman with acute lymphoblastic leukemia experiences an ontherapy bone marrow relapse. He achieves a second complete remission after reinduction chemotherapy. He does not have a matched sibling and you decide the best treatment option for this gentleman is an unrelated donor transplantation. He has an adult unrelated donor who is HLAmatched at A, B, C, DRB1 and DP1. He also has a cord blood unit with adequate total nucleated cell dose/kg and mismatched at 1locus (5/6 HLAmatched). The waiting period to procure the adult donor graft is 3 months. Which of the donor sources would you choose for this patient? a) Matched unrelated adult donor b) Mismatched cord blood unit Compared to bone marrow, peripheral blood stem cell transplant gives: a) Better engraftment b) Less GvHD c) More relapse d) Better survival THE EBMT HANDBOOK 2012 EDITION

18 NOTES HAEMATOPOIETIC STEM CELL TRANSPLANTATION 107

Pr Eliane Gluckman, MD, FRCP, Disclosure of Interest: Nothing to Disclose

Pr Eliane Gluckman, MD, FRCP, Disclosure of Interest: Nothing to Disclose Pr Eliane Gluckman, MD, FRCP, Hospital Saint Louis, University Paris- Diderot, France Should Haplo-identical transplantation be preferred to cord blood in patients without a matched donor? Disclosure of

More information

UMBILICAL CORD BLOOD TRANSPLANTATION: KFSH EXPERIENCE

UMBILICAL CORD BLOOD TRANSPLANTATION: KFSH EXPERIENCE UMBILICAL CORD BLOOD TRANSPLANTATION: KFSH EXPERIENCE HIND AL HUMAIDAN, MD,FRCPA Director, Blood Bank (Donor & Transfusion Services) and Stem Cell Cord Blood Bank Consultant Hematopathologist INTRODUCTION

More information

The donor search: the best donor or cord blood unit

The donor search: the best donor or cord blood unit The donor search: the best donor or cord blood unit Dr Bronwen Shaw Consultant in haematopoietic cell transplantation Royal Marsden Hospital /Anthony Nolan Overview Where do we find donors/units for transplantation

More information

Cord Blood Transplant. E. Gluckman Eurocord ESH-EBMT training course Vienna 2014

Cord Blood Transplant. E. Gluckman Eurocord ESH-EBMT training course Vienna 2014 Cord Blood Transplant E. Gluckman Eurocord ESH-EBMT training course Vienna 2014 Background Since 1988, umbilical cord blood (CB) has been successfully used to treat children and adults needing stem cell

More information

Selecting an appropriately matched donor for hematopoietic

Selecting an appropriately matched donor for hematopoietic Transplant Outcomes in Acute Leukemia (I) Mary Eapen a and John E. Wagner b Umbilical cord blood (UCB) has gradually emerged over the last decade as an alternative source of hematopoietic cells for transplantation

More information

Bone Marrow, Peripheral Blood Stem Cells or Umbilical Cord Blood transplantation? Federica Giannotti, MD Eurocord-Hôpital Saint Louis, Paris

Bone Marrow, Peripheral Blood Stem Cells or Umbilical Cord Blood transplantation? Federica Giannotti, MD Eurocord-Hôpital Saint Louis, Paris Bone Marrow, Peripheral Blood Stem Cells or Umbilical Cord Blood transplantation? Federica Giannotti, MD Eurocord-Hôpital Saint Louis, Paris Background Hematopoietic stem cell transplantation (HSCT) is

More information

Beyond Cell Dose: Selection of the Optimal Umbilical Cord Blood Unit. Karen Ballen, MD Massachusetts General Hospital June, 2012

Beyond Cell Dose: Selection of the Optimal Umbilical Cord Blood Unit. Karen Ballen, MD Massachusetts General Hospital June, 2012 Beyond Cell Dose: Selection of the Optimal Umbilical Cord Blood Unit Karen Ballen, MD Massachusetts General Hospital June, 2012 OUTLINE Cell Dose HLA Typing HLA C and KIR HLA Antibodies ABO and Racial/Ethnic

More information

Pros and Cons of Stem Cell Sources and their availability in Africa. Dr Jaimendra Singh Inkosi Albert Luthuli Central Hospital Durban, South Africa

Pros and Cons of Stem Cell Sources and their availability in Africa. Dr Jaimendra Singh Inkosi Albert Luthuli Central Hospital Durban, South Africa Pros and Cons of Stem Cell Sources and their availability in Africa Dr Jaimendra Singh Inkosi Albert Luthuli Central Hospital Durban, South Africa Introduction The ability to perform a haematopoietic stem

More information

Umbilical Cord Blood Transplantation

Umbilical Cord Blood Transplantation Umbilical Cord Blood Transplantation V Rocha MD, PhD Hopital Saint Louis, Paris University 7 CIBMTR Milwaukee Umbilical Cord blood transplantation Background History Clinical results in children and adults

More information

Selection of the Optimal Umbilical Cord Blood Unit

Selection of the Optimal Umbilical Cord Blood Unit Karen Ballen, MD Selection of the Optimal Umbilical Cord Blood Unit Massachusetts General Hospital September, 2013 OUTLINE Cell Dose HLA Match Allele Level HLA C KIR Directional Mismatch NIMA HLA Antibodies

More information

Cord Blood Transplant Past and Future. E. Gluckman Eurocord ISCT Paris 24/04/2014

Cord Blood Transplant Past and Future. E. Gluckman Eurocord ISCT Paris 24/04/2014 Cord Blood Transplant Past and Future E. Gluckman Eurocord ISCT Paris 24/04/2014 Background Since 1988, umbilical cord blood (CB) has been successfully used to treat children and adults needing stem cell

More information

Navelstrengbloed tegen kanker

Navelstrengbloed tegen kanker Navelstrengbloed tegen kanker THERAPIEDAG 2008: "Zorgtrajecten in kanker" Zaterdag 27 september 2008 Gasthuisberg, Leuven. Hélène Schoemans, MD KUL, Stem Cell Institute Leuven Cord blood Collection and

More information

Fetal Maternal Immunity and Antileukemia Activity in Cord Blood Transplant. Recipients

Fetal Maternal Immunity and Antileukemia Activity in Cord Blood Transplant. Recipients Fetal Maternal Immunity and Antileukemia Activity in Cord Blood Transplant Recipients Filippo Milano, 1 J. Lee Nelson, 1, 2 Colleen Delaney 1,3 1 Clinical Research Division, Fred Hutchinson Cancer Research

More information

Bone Marrow, Peripheral Blood Stem Cells or Umbilical Cord Blood transplantation? E. Gluckman WBMT meeting Cape Town November 14-16, 2014

Bone Marrow, Peripheral Blood Stem Cells or Umbilical Cord Blood transplantation? E. Gluckman WBMT meeting Cape Town November 14-16, 2014 Bone Marrow, Peripheral Blood Stem Cells or Umbilical Cord Blood transplantation? E. Gluckman WBMT meeting Cape Town November 14-16, 2014 The ideal HSCs source Immediate availability Few HLA restrictions

More information

In contrast to the very high transplant-related

In contrast to the very high transplant-related Cord Blood: an Alternative Stem Cell Source or a New Standard? Juliet N. BARKER Memorial Sloan-Kettering Cancer Center, NY, ABD In contrast to the very high transplant-related mortality (TRM) associated

More information

EUROCORD. in 49 countries and 484 transplant centres* 264 EBMT 4847 (73%) cases 220 Non-EBMT 1797 (27%) cases

EUROCORD. in 49 countries and 484 transplant centres* 264 EBMT 4847 (73%) cases 220 Non-EBMT 1797 (27%) cases ! 21%! EUROCORD 6756 cord blood transplantations performed from 1988 to March 2010 in 49 countries and 484 transplant centres* 264 EBMT 4847 (73%) cases 220 Non-EBMT 1797 (27%) cases * missing center

More information

ANTHONY NOLAN SEARCH ALGORITHM FOR A BASIC CORD BLOOD UNIT SELECTION BY SERGIO QUEROL AND IRINA EVSEEVA FEBRUARY 2012

ANTHONY NOLAN SEARCH ALGORITHM FOR A BASIC CORD BLOOD UNIT SELECTION BY SERGIO QUEROL AND IRINA EVSEEVA FEBRUARY 2012 ANTHONY NOLAN SEARCH ALGORITHM FOR A BASIC CORD BLOOD UNIT SELECTION BY SERGIO QUEROL AND IRINA EVSEEVA FEBRUARY 2012 PAGE 1 OF 7 PRINCIPLES FOR A NEW ALGORITHM Once cord blood transplantation (CBT) is

More information

Myeloablative versus Reduced Intensity Conditioning Regimen Cord Blood Transplants

Myeloablative versus Reduced Intensity Conditioning Regimen Cord Blood Transplants Educational 2 Cord Blood Transplantation Myeloablative versus Reduced Intensity Conditioning Regimen Cord Blood Transplants William Arcese University of Rome Tor Vergata Rome Transplant Network 4th April

More information

Cord Blood: that other stem cell source. Donna Wall, MD Director, Manitoba Blood and Marrow Transplant Program

Cord Blood: that other stem cell source. Donna Wall, MD Director, Manitoba Blood and Marrow Transplant Program Cord Blood: that other stem cell source Donna Wall, MD Director, Manitoba Blood and Marrow Transplant Program CBMTG April 2012 The problem: In order to perform a BMT from one person to another one needs

More information

5. All cord blood banks should be subject to the same standards, regulations and accreditation requirements.

5. All cord blood banks should be subject to the same standards, regulations and accreditation requirements. WMDA Policy Statement for the Utility of Autologous or Family Cord Blood Unit Storage (This policy statement has been approved and adopted by the WMDA board on the 25 th of May 2006) The Cord Blood Registries

More information

4. All cord blood banks should be subject to the same standards, regulations and accreditation requirements.

4. All cord blood banks should be subject to the same standards, regulations and accreditation requirements. WMDA Policy Statement on the Utility of Autologous or Family Cord Blood Unit Storage The WMDA Board adopted this policy on 25 th of May 2006. Policy updated _April 2011 The Cord Blood Working Group and

More information

Hematopoietic Stem Cell Transplantation. Imad A. Tabbara, M.D. Professor of Medicine

Hematopoietic Stem Cell Transplantation. Imad A. Tabbara, M.D. Professor of Medicine Hematopoietic Stem Cell Transplantation Imad A. Tabbara, M.D. Professor of Medicine Hematopoietic Stem Cells Harvested from blood, bone marrow, umbilical cord blood Positive selection of CD34 (+) cells

More information

Placental/Umbilical Cord Blood as a Source of Stem Cells. Original Policy Date

Placental/Umbilical Cord Blood as a Source of Stem Cells. Original Policy Date MP 7.01.38 Placental/Umbilical Cord Blood as a Source of Stem Cells Medical Policy Section Surgery Issue 12:2013 Original Policy Date 12:2013 Last Review Status/Date Reviewed with literature search/12:2013

More information

Placental and Umbilical Cord Blood as a Source of Stem Cells

Placental and Umbilical Cord Blood as a Source of Stem Cells Placental and Umbilical Cord Blood as a Source of Stem Cells Policy Number: 7.01.50 Last Review: 12/2013 Origination: 12/2001 Next Review: 12/2014 Policy Blue Cross and Blue Shield of Kansas City (Blue

More information

The Value of Cord Blood Stem Cells. Mona Shafey, MD, FRCPC Medical Grand Rounds October 25 th, 2011

The Value of Cord Blood Stem Cells. Mona Shafey, MD, FRCPC Medical Grand Rounds October 25 th, 2011 The Value of Cord Blood Stem Cells Mona Shafey, MD, FRCPC Medical Grand Rounds October 25 th, 2011 Objectives To discuss umbilical cord blood as a stem cell source and the role of umbilical cord blood

More information

How to select a donor and product for allogeneic HCT

How to select a donor and product for allogeneic HCT How to select a donor and product for allogeneic HCT Dr Bronwen Shaw 10 February 2015 Overview Who (and where) are the donors What factors determine how we choose between them Acquisition Clinical Donor

More information

Double cord blood transplantation

Double cord blood transplantation DCTH - 2 2013-113-121 REVIEW Double cord blood transplantation R. Angarano, I. Donnini, B. Bartolozzi, A. Bosi Ematologia, Azienda Ospedaliera Universitaria Careggi, Università di Firenze, Italy SUMMARY

More information

UMBILICAL-CORD BLOOD TRANSPLANTATION FOR THE TREATMENT OF CANCER

UMBILICAL-CORD BLOOD TRANSPLANTATION FOR THE TREATMENT OF CANCER UMBILICAL-CORD BLOOD TRANSPLANTATION FOR THE TREATMENT OF CANCER Juliet N. Barker* and John E. Wagner Haematopoietic stem-cell transplantation is used to treat many haematological cancers, but is limited

More information

Cord Blood for Cellular Therapy: A Snapshot of this Evolving Market Landscape

Cord Blood for Cellular Therapy: A Snapshot of this Evolving Market Landscape GENReports: Market & Tech Analysis Cord Blood for Cellular Therapy: A Snapshot of this Evolving Market Landscape > Enal Razvi, Ph.D. Biotechnology Analyst, Managing Director SELECTBIO US enal@selectbio.us

More information

A Public Cord Blood Bank for South Africa? i

A Public Cord Blood Bank for South Africa? i No. 42/2007 A Public Cord Blood Bank for South Africa? i By Dr Robert Crookes MBChB (Wits), Dip. Internal Medicine (American Board of Internal Medicine, USA) Transfusion Medicine Consultant. South African

More information

Transplantation of cord blood stem cells from related or unrelated donors is considered investigational in all other situations.

Transplantation of cord blood stem cells from related or unrelated donors is considered investigational in all other situations. MEDICAL POLICY POLICY RELATED POLICIES POLICY GUIDELINES DESCRIPTION SCOPE BENEFIT APPLICATION RATIONALE REFERENCES CODING APPENDIX HISTORY Placental and Umbilical Cord Blood as a Source of Stem Cells

More information

Placental and Umbilical Cord Blood as a Source of Stem Cells

Placental and Umbilical Cord Blood as a Source of Stem Cells Placental and Umbilical Cord Blood as a Source of Stem Cells Policy Number: 7.01.50 Last Review: 12/2014 Origination: 12/2001 Next Review: 12/2015 Policy Blue Cross and Blue Shield of Kansas City (Blue

More information

Corporate Medical Policy Cord Blood as a Source of Stem Cells

Corporate Medical Policy Cord Blood as a Source of Stem Cells Corporate Medical Policy Cord Blood as a Source of Stem Cells File Name: Origination: Last CAP Review: Next CAP Review: Last Review cord_blood_as_a_source_of_stem_cells 2/2001 3/2015 3/2016 3/2015 Description

More information

POLICY PRODUCT VARIATIONS DESCRIPTION/BACKGROUND RATIONALE DEFINITIONS BENEFIT VARIATIONS DISCLAIMER CODING INFORMATION REFERENCES POLICY HISTORY

POLICY PRODUCT VARIATIONS DESCRIPTION/BACKGROUND RATIONALE DEFINITIONS BENEFIT VARIATIONS DISCLAIMER CODING INFORMATION REFERENCES POLICY HISTORY Original Issue Date (Created): 4/1/2011 Most Recent Review Date (Revised): 3/24/2015 Effective Date: 6/1/2015 POLICY PRODUCT VARIATIONS DESCRIPTION/BACKGROUND RATIONALE DEFINITIONS BENEFIT VARIATIONS DISCLAIMER

More information

Umbilical Cord Blood Stem Cells Current Status & Future Potential

Umbilical Cord Blood Stem Cells Current Status & Future Potential Umbilical Cord Blood Stem Cells Current Status & Future Potential Natasha Ali Assistant Professor Haematology Department of Pathology & Laboratory Medicine/Oncology The Aga Khan University Email: natasha.ali@aku.edu

More information

Graft Failure After HSCT

Graft Failure After HSCT Graft Failure After HSCT Vanderson Rocha, MD, PhD Professor of Haematology- Oxford University Bone Marrow Transplant Unit- Sirio Libanes Hospital- Sao Paulo Scientific Director of Eurocord-Paris Clinical

More information

Section: Transplant Last Reviewed Date: January 2015. Policy No: 45.16 Effective Date: April 1, 2015

Section: Transplant Last Reviewed Date: January 2015. Policy No: 45.16 Effective Date: April 1, 2015 Medical Policy Manual Topic: Placental and Umbilical Cord Blood as a Source of Stem Cells Date of Origin: December 2009 Section: Transplant Last Reviewed Date: January 2015 Policy No: 45.16 Effective Date:

More information

Name of Policy: Placental/Umbilical Cord Blood as a Source of Stem Cells

Name of Policy: Placental/Umbilical Cord Blood as a Source of Stem Cells Name of Policy: Placental/Umbilical Cord Blood as a Source of Stem Cells Policy #: 439 Latest Review Date: September 2014 Category: Medical Policy Grade: A Background/Definitions: As a general rule, benefits

More information

Sibling Donor Cord Blood Transplantation for Thalassemia Major: Experience of the Sibling Donor Cord Blood Program

Sibling Donor Cord Blood Transplantation for Thalassemia Major: Experience of the Sibling Donor Cord Blood Program Sibling Donor Cord Blood Transplantation for Thalassemia Major: Experience of the Sibling Donor Cord Blood Program MARK C. WALTERS, LYNN QUIROLO, ELIZABETH T. TRACHTENBERG, SANDIE EDWARDS, LISA HALE, JOANNA

More information

Rationale for cord blood banking from hematopoietic stem cell transplant to regenerative medicine. Milan November 2008

Rationale for cord blood banking from hematopoietic stem cell transplant to regenerative medicine. Milan November 2008 Rationale for cord blood banking from hematopoietic stem cell transplant to regenerative medicine Milan November 2008 Hematopoietic reconstitution in a patient with Fanconi's anemia by means of umbilical

More information

Placental and Umbilical Cord Blood as a Source of Stem Cells

Placental and Umbilical Cord Blood as a Source of Stem Cells Placental and Umbilical Cord Blood as a Source of Stem Cells Policy Number: 7.01.50 Last Review: 12/2015 Origination: 12/2001 Next Review: 12/2016 Policy Blue Cross and Blue Shield of Kansas City (Blue

More information

Stem Cell Transplantation in Severe Aplastic Anemia

Stem Cell Transplantation in Severe Aplastic Anemia Stem Cell Transplantation in Severe Aplastic Anemia Dr. D. Goodyear MD, FRCPC Division of Hematology and Hematological Malignancies, University of Calgary 1 of 11 Introduction Most cases of aplastic anemia

More information

Outcome of Unrelated HSCT in Patients Lacking HLA Matched Related Donors: Iranian Stem Cell Donor Program (ISCDP)

Outcome of Unrelated HSCT in Patients Lacking HLA Matched Related Donors: Iranian Stem Cell Donor Program (ISCDP) Outcome of Unrelated HSCT in Patients Lacking HLA Matched Related Donors: Iranian Stem Cell Donor Program (ISCDP) October 18, 2014 19th Congress of APBMT, Hangzhou, China AMIR ALI HAMIDIEH, MD Iranian

More information

Cord Blood Biology and Transplantation

Cord Blood Biology and Transplantation Cord Blood Biology and Transplantation Yossi Cohen MD MSc and Arnon Nagler MD Institute of Hematology, Department of Bone Marrow Transplantation and Cord Blood Bank, Sheba Medical Center, Tel Hashomer,

More information

Helping you find the one match.. Guide for Unrelated Stem Cell Transplant Patients OneMatch Stem Cell and Marrow Network BLOOD.

Helping you find the one match.. Guide for Unrelated Stem Cell Transplant Patients OneMatch Stem Cell and Marrow Network BLOOD. Helping you find the one match.. Guide for Unrelated Stem Cell Transplant Patients OneMatch Stem Cell and Marrow Network BLOOD.CA WWW This guide is intended for patients in need of an unrelated volunteer

More information

A Cure for Sickle Cell Anemia and Thalassemia

A Cure for Sickle Cell Anemia and Thalassemia IV Simpósio Internacional de Hemoglobinopatias A Cure for Sickle Cell Anemia and Thalassemia Bertram Lubin, MD and Mark Walters, MD 4 September 2007 Topics to be covered Cord blood: Importance and biology

More information

Corporate Medical Policy Cord Blood as a Source of Stem Cells

Corporate Medical Policy Cord Blood as a Source of Stem Cells Corporate Medical Policy Cord Blood as a Source of Stem Cells File Name: Origination: Last CAP Review: Next CAP Review: Last Review cord_blood_as_a_source_of_stem_cells 2/2001 3/2015 3/2016 3/2015 Description

More information

Update on Cord Blood Transplants

Update on Cord Blood Transplants Update on Cord Blood Transplants Vanderson Rocha, MD, PhD Scientific Director of Eurocord Chair of Cord Blood Subcommittee of EBMT Agence de Biomedecine and Saint Louis Hospital, Paris, France Hematopoietic

More information

One-Unit versus Two-Unit Cord-Blood Transplantation for Hematologic Cancers

One-Unit versus Two-Unit Cord-Blood Transplantation for Hematologic Cancers The new england journal of medicine Original Article One-Unit versus Two-Unit Cord-Blood Transplantation for Hematologic Cancers John E. Wagner, Jr., M.D., Mary Eapen, M.B., B.S., Shelly Carter, D.Sc.,

More information

Challenges of Hematopoietic Stem Cell Transplantation. Robert J. Soiffer, MD Dana Farber Cancer Institute

Challenges of Hematopoietic Stem Cell Transplantation. Robert J. Soiffer, MD Dana Farber Cancer Institute Challenges of Hematopoietic Stem Cell Transplantation Robert J. Soiffer, MD Dana Farber Cancer Institute Hematopoietic Stem Cell Transplantation Objectives Deliver sufficient chemo-radio therapy to destroy

More information

THE NEW ZEALAND MEDICAL JOURNAL

THE NEW ZEALAND MEDICAL JOURNAL THE NEW ZEALAND MEDICAL JOURNAL Vol 118 No 1208 ISSN 1175 8716 Private umbilical cord blood banking: a biological insurance of dubious future benefit! Michael Sullivan, Peter Browett, Nigel Patton In its

More information

Natasha Kekre, 1 Jennifer Philippe, 2 Ranjeeta Mallick, 3 Susan Smith, 2 and David Allan 1,2,4. 1. Introduction

Natasha Kekre, 1 Jennifer Philippe, 2 Ranjeeta Mallick, 3 Susan Smith, 2 and David Allan 1,2,4. 1. Introduction Hindawi Publishing Corporation Stem Cells International Volume 2013, Article ID 124834, 6 pages http://dx.doi.org/10.1155/2013/124834 Research Article Modelling Improvements in Cell Yield of Banked Umbilical

More information

Umbilical cord blood transplantation

Umbilical cord blood transplantation Review article http://dx.doi.org/10.3345/kjp.2012.55.7.219 Korean J Pediatr 2012;55(7):219-223 eissn 1738-1061 pissn 2092-7258 Umbilical cord blood transplantation Hong Hoe Koo, MD 1, Hyo Seop Ahn, MD

More information

The availability of haematopoietic stem or progenitor

The availability of haematopoietic stem or progenitor Update Article Umbilical Cord Blood Transplantation: Newer Trends MB Agarwal Abstract During last ten years, over 4000 umbilical cord blood transplantations have been performed worldwide. The interest

More information

Bone Marrow Transplantation and Peripheral Blood Stem Cell Transplantation: Questions and Answers. Key Points

Bone Marrow Transplantation and Peripheral Blood Stem Cell Transplantation: Questions and Answers. Key Points CANCER FACTS N a t i o n a l C a n c e r I n s t i t u t e N a t i o n a l I n s t i t u t e s o f H e a l t h D e p a r t m e n t o f H e a l t h a n d H u m a n S e r v i c e s Bone Marrow Transplantation

More information

CHAPTER 1 BACKGROUND AND CORD BLOOD BANK (CBB) ORGANIZATION

CHAPTER 1 BACKGROUND AND CORD BLOOD BANK (CBB) ORGANIZATION CHAPTER 1 BACKGROUND AND CORD BLOOD BANK (CBB) ORGANIZATION Chapter 1 BACKGROUND AND CORD BLOOD BANK (CBB) ORGANIZATION 1.1 OVERVIEW OF THE CORD BLOOD TRANSPLANTATION STUDY Bone marrow transplantation

More information

Blood cells are vital to the human body

Blood cells are vital to the human body Candide FONT-SALA Blood cells are vital to the human body Red cells transport oxygen From http://www.english-online.at/biology/blood/blood-supply-and-blood-diseases.htm Red cell life span 120 days 200

More information

Stem Cell Transplantation In Patients with Fanconi Anemia

Stem Cell Transplantation In Patients with Fanconi Anemia Stem Cell Transplantation In Patients with Fanconi Anemia FARF Annual Family Meeting 6/28/15 Casco, ME Parinda A. Mehta, M.D. Cincinnati Children s Hospital Medical Center Improvements in Unrelated Donor

More information

Blood-Forming Stem Cell Transplants

Blood-Forming Stem Cell Transplants Blood-Forming Stem Cell Transplants What are bone marrow and hematopoietic stem cells? Bone marrow is the soft, sponge-like material found inside bones. It contains immature cells known as hematopoietic

More information

Cord Blood Stem Cell Transplantation

Cord Blood Stem Cell Transplantation LEUKEMIA LYMPHOMA MYELOMA FACTS Cord Blood Stem Cell Transplantation No. 2 in a series providing the latest information on blood cancers Highlights Umbilical cord blood, like bone marrow and peripheral

More information

Stem cells from Cord Blood: Myths, reality and potential. Elisabeth Semple, PhD Scientific Director Cells for Life Cord Blood Institute

Stem cells from Cord Blood: Myths, reality and potential. Elisabeth Semple, PhD Scientific Director Cells for Life Cord Blood Institute Stem cells from Cord Blood: Myths, reality and potential Elisabeth Semple, PhD Scientific Director Cells for Life Cord Blood Institute Learning objectives Understand the current usage of stem cells from

More information

Preparation of cord blood for infusion: bedside thaw, dilute and wash, or somewhere in between

Preparation of cord blood for infusion: bedside thaw, dilute and wash, or somewhere in between Preparation of cord blood for infusion: bedside thaw, dilute and wash, or somewhere in between Donna Wall, MD Director, Manitoba Blood and Marrow Transplant Program ISCT 2012 Disclosures: none The problem:

More information

Part 2 Annexes. www.nhsbt.nhs.uk. The Future of Unrelated Donor Stem Cell Transplantation in the UK

Part 2 Annexes. www.nhsbt.nhs.uk. The Future of Unrelated Donor Stem Cell Transplantation in the UK The Future of Unrelated Donor Stem Cell Transplantation in the UK Part 2 Annexes A Report from the UK Stem Cell Strategic Forum July 2010 www.nhsbt.nhs.uk Table of Contents Abbreviations... 6 Glossary...

More information

Unrelated donor umbilical cord blood transplantation for the treatment of hematologic malignancies Craig Sauter and Juliet N.

Unrelated donor umbilical cord blood transplantation for the treatment of hematologic malignancies Craig Sauter and Juliet N. Unrelated donor umbilical cord blood transplantation for the treatment of hematologic malignancies Craig Sauter and Juliet N. Barker Adult Allogeneic Bone Marrow Transplantation Service, Memorial Sloan-Kettering

More information

Umbilical Cord Blood Transplantation for the Treatment of Hematologic Malignancies

Umbilical Cord Blood Transplantation for the Treatment of Hematologic Malignancies Umbilical cord blood transplantation is a useful treatment in several types of hematologic malignancies. Gene Elling. St. Lucia. Photograph. Umbilical Cord Blood Transplantation for the Treatment of Hematologic

More information

Donor Sources and Donor Selection for Hematopoietic Cell Transplant

Donor Sources and Donor Selection for Hematopoietic Cell Transplant Donor Sources and Donor Selection for Hematopoietic Cell Transplant 2 Ann E. Woolfrey and Vanderson Rocha Contents 2.1 Autologous Hematopoietic Stem Cells... 23 2.2 HLA-Identical Related Donors... 24 2.3

More information

UMBILICAL CORD BLOOD STATISTICS

UMBILICAL CORD BLOOD STATISTICS UMBILICAL CORD BLOOD STATISTICS INTRODUCTION Stem cells are the next frontier in medicine. Stem cells are thought to have great therapeutic and biotechnological potential. This will not only to replace

More information

P R E S S K I T 2013 TABLE OF CONTENTS. About the European Group for Blood and Marrow Transplantation

P R E S S K I T 2013 TABLE OF CONTENTS. About the European Group for Blood and Marrow Transplantation TABLE OF CONTENTS About the European Group for Blood and Marrow Transplantation Haematopoietic Stem Cell Transplantation (HSCT): Key facts & figures EBMT Data - A foundation for cutting-edge research Clinical

More information

A fact sheet UNRELATED BONE MARROW AND CORD BLOOD STEM CELL TRANSPLANTS

A fact sheet UNRELATED BONE MARROW AND CORD BLOOD STEM CELL TRANSPLANTS A fact sheet UNRELATED BONE MARROW AND CORD BLOOD STEM CELL TRANSPLANTS Each year, thousands of patients are diagnosed with diseases treatable by a blood stem cell transplant. These blood stem cells can

More information

CHAPTER 5 Sources and procurement of stem cells

CHAPTER 5 Sources and procurement of stem cells EBMT2008_1_21:EBMT2008 6-11-2008 9:10 Pagina 112 * CHAPTER 5 Sources and procurement of stem cells J. Larghero, J. Garcia, E. Gluckman EBMT2008_1_21:EBMT2008 6-11-2008 9:10 Pagina 113 CHAPTER 5 Stem cell

More information

The future of unrelated Stem Cell Transplant in the UK: DOH Working Party Findings. Prof. Tony Pagliuca

The future of unrelated Stem Cell Transplant in the UK: DOH Working Party Findings. Prof. Tony Pagliuca The future of unrelated Stem Cell Transplant in the UK: DOH Working Party Findings Prof. Tony Pagliuca UK STEM CELL STRATEGIC FORUM The future of unrelated donor SCT in the UK Antonio Pagliuca, Transplant

More information

The Danish Bone Marrow Donor Registry DBMDR

The Danish Bone Marrow Donor Registry DBMDR The DBMDR Vision To achieve and maintain a position as an internationally recognized hematopoietic stem cell donor registry with respect to high quality of donor data base and HLA typing, individualized,

More information

Umbilical Cord Blood (UCB) Transplantation: An Alternative to the Use of Unrelated Volunteer Donors?

Umbilical Cord Blood (UCB) Transplantation: An Alternative to the Use of Unrelated Volunteer Donors? Umbilical Cord Blood (UCB) Transplantation: An Alternative to the Use of Unrelated Volunteer Donors? Juliet N. Barker Memorial Sloan-Kettering Cancer Center, New York, NY Cryopreserved umbilical cord blood

More information

Disclosures. I have no disclosures.

Disclosures. I have no disclosures. Not Your Own Marrow Jenni Krajewski, MD Clinical Assistant Professor, Rutgers New Jersey Medical School Attending Physician, Pediatric Blood and Marrow Transplantation The Institute for Pediatric Cancer

More information

Corporate Medical Policy

Corporate Medical Policy Corporate Medical Policy Hematopoietic Stem-Cell Transplantation for CLL and SLL File Name: Origination: Last CAP Review: Next CAP Review: Last Review: hematopoietic_stem-cell_transplantation_for_cll_and_sll

More information

Donor and stem cell source selection. 21 November 2013 E. Baudoux

Donor and stem cell source selection. 21 November 2013 E. Baudoux Donor and stem cell source selection 21 November 2013 E. Baudoux Table of contents Introduction HPC sources and donor types HLA and matching Unrelated donor searches Donor choice and eligibility Search

More information

Public Cord Blood Banking at the National Cord Blood Program (NCBP)

Public Cord Blood Banking at the National Cord Blood Program (NCBP) Public Cord Blood Banking at the National Cord Blood Program (NCBP) A. Scaradavou, MD Medical Director, National Cord Blood Program, New York Blood Center Associate Attending, Pediatric BMT Memorial Sloan-Kettering

More information

INFORMATION ON STEM CELLS/BONE MARROW AND REINFUSION/TRANSPLANTATION SUR703.002

INFORMATION ON STEM CELLS/BONE MARROW AND REINFUSION/TRANSPLANTATION SUR703.002 INFORMATION ON STEM CELLS/BONE MARROW AND REINFUSION/TRANSPLANTATION SUR703.002 COVERAGE: SPECIAL COMMENT ON POLICY REVIEW: Due to the complexity of the Peripheral and Bone Marrow Stem Cell Transplantation

More information

Hematopoietic Stem Cell Transplantation: Current Status and Future Directions RICHARD W. CHILDS M.D. NIH, BETHESDA MD

Hematopoietic Stem Cell Transplantation: Current Status and Future Directions RICHARD W. CHILDS M.D. NIH, BETHESDA MD Hematopoietic Stem Cell Transplantation: Current Status and Future Directions RICHARD W. CHILDS M.D. NIH, BETHESDA MD Stem cell transplantation Autologous Autologous stem cell collection Freeze Stem Cells

More information

Umbilical Cord Blood: An Alternative Allogeneic Stem Cell Source for Transplantation

Umbilical Cord Blood: An Alternative Allogeneic Stem Cell Source for Transplantation Umbilical Cord Blood: An Alternative Allogeneic Stem Cell Source for Transplantation Mary J. Laughlin, MD Associate Professor of Medicine and Pathology Dr. Donald and Ruth Weber Goodman Professor of Innovative

More information

In a number of genetic, hematologic, and oncologic

In a number of genetic, hematologic, and oncologic AMERICAN ACADEMY OF PEDIATRICS Cord Blood Banking for Potential Future Transplantation: Subject Review ABSTRACT. In recent years, umbilical cord blood, which contains a large number of hematopoietic stem

More information

HLA Match Likelihoods for Hematopoietic Stem-Cell Grafts in the U.S. Registry

HLA Match Likelihoods for Hematopoietic Stem-Cell Grafts in the U.S. Registry The new england journal of medicine special article HLA Likelihoods for Hematopoietic Stem-Cell Grafts in the U.S. Registry Loren Gragert, B.S., B.A., Mary Eapen, M.B., B.S., Eric Williams, Ph.D., John

More information

Clinical Policy Bulletin: Stem Cells for Hematopoietic Cell Transplant

Clinical Policy Bulletin: Stem Cells for Hematopoietic Cell Transplant Stem Cells for Hematopoietic Cell Transplant Page 1 of 8 Clinical Policy Bulletin: Stem Cells for Hematopoietic Cell Transplant Number: 0190 Policy Aetna considers compatibility testing of prospective

More information

UMBILICAL CORD BLOOD HARVESTING & STORAGE

UMBILICAL CORD BLOOD HARVESTING & STORAGE Protocol: TRP009 Effective Date: October 14, 2013 UMBILICAL CORD BLOOD HARVESTING & STORAGE Table of Contents Page COMMERCIAL, MEDICARE & MEDICAID COVERAGE RATIONALE... 1 BACKGROUND... 2 CLINICAL EVIDENCE...

More information

Reference: NHS England B04/P/a

Reference: NHS England B04/P/a Clinical Commissioning Policy: Haematopoietic Stem Cell Transplantation (HSCT) (All Ages): Revised Reference: NHS England B04/P/a 1 NHS England Clinical Commissioning Policy: Haematopoietic Stem Cell Transplantation

More information

New Zealand Bone Marrow Donor Registry

New Zealand Bone Marrow Donor Registry Title: Authorised by: SECTION 9.0 NZBMDR STANDARDS PROCESS FOR AN UNRELATED STEM CELL DONOR WORKUP & IDENTIFICATION OF CORD BLOOD UNITS Executive Officer Contributing Authors: Sally Gordon Dr Hilary Blacklock

More information

Artemisa. Hematopoietic stem-cell transplantation using umbilical-cord blood cells. medigraphic. pdf elaborado por medigraphic.

Artemisa. Hematopoietic stem-cell transplantation using umbilical-cord blood cells. medigraphic. pdf elaborado por medigraphic. medigraphic Artemisa en línea ARTÍCULO ESPECIAL Hematopoietic stem-cell transplantation using umbilical-cord blood cells Vanderson Rocha,* Federico Garnier,* Irina Ionescu,* Eliane Gluckman* * FRCP on

More information

THE INFLUENCE OF TISSUE (IN)COMPATIBILITY IN UMBILICAL CORD BLOOD TRANSPLANTATION

THE INFLUENCE OF TISSUE (IN)COMPATIBILITY IN UMBILICAL CORD BLOOD TRANSPLANTATION THE INFLUENCE OF TISSUE (IN)COMPATIBILITY IN UMBILICAL CORD BLOOD TRANSPLANTATION Matjaž Jeras Blood Transfusion Centre of Slovenia Tissue Typing Center Šlajmerjeva 6, 1000 Ljubljana, Slovenia matjaz.jeras@ztm.si

More information

Title: SECTION 7.0 NZBMDR STANDARDS PROCESS FOR DONOR IDENTIFICATION

Title: SECTION 7.0 NZBMDR STANDARDS PROCESS FOR DONOR IDENTIFICATION NZBMDRGuidelines-007.10 Date Effective: 01/05/2010 Page 1 Title: SECTION 7.0 NZBMDR STANDARDS PROCESS FOR DONOR IDENTIFICATION Authorised by: Executive Officer Contributing Authors: ABMDR Scientific Expert

More information

DEPARTMENT OF BONE MARROW AND STEM CELL TRANSPLANT

DEPARTMENT OF BONE MARROW AND STEM CELL TRANSPLANT www.narayanahealth.org DEPARTMENT OF BONE MARROW AND STEM CELL TRANSPLANT About Narayana Health City Narayana Health, one of India's largest and the world's most economical healthcare service providers

More information

SEARCHING FOR A BONE MARROW DONOR

SEARCHING FOR A BONE MARROW DONOR SEARCHING FOR A BONE MARROW DONOR Angela received a bone marrow transplant from an unrelated donor to treat her non-hodgkin s lymphoma. INFORMATION FOR PATIENTS AND THEIR FAMILIES For patients who need

More information

Review Article The Role of HLA in Cord Blood Transplantation

Review Article The Role of HLA in Cord Blood Transplantation Bone Marrow Research Volume 2012, Article ID 485160, 9 pages doi:10.1155/2012/485160 Review Article The Role of HLA in Cord Blood Transplantation Catherine Stavropoulos-Giokas, Amalia Dinou, and Andreas

More information

Narrator: Transplants using stem cells from the blood, bone marrow or umbilical cord blood

Narrator: Transplants using stem cells from the blood, bone marrow or umbilical cord blood [Track 2: What Is a Transplant?] Narrator: Transplants using stem cells from the blood, bone marrow or umbilical cord blood can be an effective treatment for people with blood cancers such as leukemia,

More information

Stem Cell Background Paper

Stem Cell Background Paper Stem Cell Background Paper Introduction...2 Stem Cell Basics...3 Stem Cell Process Flow...9 Comparison of Blood, Stem Cells, Tissues and Organs Processes...10 Responsibilities for the Blood, Stem Cells,

More information

Hematopoietic Stem Cell Transplantation: Evolving Strategies That Have Resulted in Improved Outcomes

Hematopoietic Stem Cell Transplantation: Evolving Strategies That Have Resulted in Improved Outcomes Hematopoietic Stem Cell Transplantation: Evolving Strategies That Have Resulted in Improved Outcomes Asad Bashey, MD, PhD Blood and Marrow Transplantation Program at Northside Hospital Atlanta, Georgia

More information

EBMT2008_1_21:EBMT2008 6-11-2008 9:22 Pagina 356 CHAPTER 20. HSCT for acute myeloid leukaemia in adults. R. Varaldo, F. Frassoni

EBMT2008_1_21:EBMT2008 6-11-2008 9:22 Pagina 356 CHAPTER 20. HSCT for acute myeloid leukaemia in adults. R. Varaldo, F. Frassoni EBMT2008_1_21:EBMT2008 6-11-2008 9:22 Pagina 356 * CHAPTER 20 HSCT for acute myeloid leukaemia in adults R. Varaldo, F. Frassoni EBMT2008_1_21:EBMT2008 6-11-2008 9:22 Pagina 357 CHAPTER 20 AML in adults

More information