Immunoablative therapy with autologous hematopoietic stem cell transplantation in the treatment of poor risk multiple sclerosis

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1 Immunoablative therapy with autologous hematopoietic stem cell transplantation in the treatment of poor risk multiple sclerosis T Kozák, P Lhotáková Department of Clinical Haematology, 3r d School of Medicine, Charles University in Prague, Czech Republic.

2 Multiple sclerosis (MS) Autoimmune inflammatory disease of central nervous system CD+4 lymphocytes cross blood/brain barrier perivascular infiltrates Myelin destruction clinical course Main disability: lower extremities motoric disability

3 MS: classification RR: relapsing remmiting MS PR: progressive relapsing MS SP: secondary progressive MS PP: primary progressive MS (Malignant MS) EDSS (Kurtzke scale): 0 (no disability) 10 (death from MS) EDSS 1-4.5: fully ambulatory, EDSS 7.0: wheel chair

4 MS: treatment Pulses of methylprednisolone Beta interferon Copaxone (glatiramer acetate) Second line: natalizumab Secondary progressive MS: mitoxantrone? ASCT? Emerging therapies

5 Axonal loss RR-MS SP-MS CNS reserves time Courtesy of E. Havrdová

6 Rationale for high dose immunoablation in MS MS is T-cell mediated autoimmune disease CD4+ (CD8+) cells may undergo a new ontogeny New T cell repertoire, Treg release after autotx Positive experiments in animal models Concomitant AID to a malignancy improved after SCT High dose chemotherapy with AutoHSCT support is safe procedure

7 First clinical evidence of auto PBPC transplantation efficacy in MS Fassas et al. Peripheral blood stem cell transplantation in the treatment of progressive MS. (Bone Marrow Transpl. 1997, 20: ) Burt et al. T cell- depleted autologous hematopoietic stem cell transplantation for MSreport on the first 3 patients (Bone Marrow Transpl. 1998, 21: )

8 Clinical trials with MS overview ( ) Diversity in inclusion criteria However: majority SPMS Diversity in conditioning regimen Data from randomized trials lacking High intensity conditioning (TBI + ) Intermediate intensity (BEAM ± ATG) Non-myeloablative (Cy 200mg/kg ± ATG)

9 Clinical trials with MS overview - outcome Metaanalysis on 161 patients (8 series) 77% SPMS, EDSS: ( ) at inclusion 5 series with intermediate intensity conditioning (BEAM or Carmustine) 3 series with high intensity conditioning (TBI) Reston JT, et al., Mult Scler J 2010;17:

10 Clinical trials with MS overview - outcome Progression free survival (PFS) Intermediate intensity conditioning: 79.4% ( %) in 39months (6-72) High intensity conditioning: 44.6% ( %) in 24months (6-60) Reston JT, et al., Mult Scler J 2010;17:

11 Clinical trials with MS overview - toxicity 259 patients from 13 series analyzed Treatment related death: 2.7% Leading cause of death: pneumonia Most frequent AEs: fever, engraftment syndrome, enteritis, transient neurological worsening Non treatment related deaths: 2.3%, most of them related to MS progression Reston JT, et al., Mult Scler J 2010;17:

12 MS EBMT registry data Observational study (12 years experience) patients with MS Follow up 31 months (0,5 121) Conditioning regimen: TBI 16 BEAM: 168 Other:101 Farge D et al. Haematologica 2010;95:

13 MS EBMT registry data Transplant related death: 13 (2%, CI 0-4) (TRM to year 2000: 7%) Progression free in 3 years: 55% Progression free in 5 years: 43% Overall survival: 93% in 3 years Farge D et al. Haematologica 2010;95:

14 Results in late x early phases of MS Czech trial (10-year experience) 26 pts included, median follow up: 66 months No TRM PFS all pts 3 years: 70,8%, in 6 years: 29.2% PFS in RRMS (11 pts): 84.4% (median fup: 19m) PFS in SPMS (15 pts): 60.0% (median fup: 96m) p= Krasulova E. et al. Mult Scler 2010;16:685-93

15 PFS in early MS x SPMS Krasulova E. et al. Mult Scler 2010;16:685-93

16 To purge or not to purge (in vitro)?

17 Schedule of the treatment Definite MS SP, RR, EDSS 5,0-8,5, 1.0 in last year Cy 2.5-4g/m 2 + G-CSF PBSC harvest enough CD34+ cells + resources: in vitro purging HD BEAM + APBSCT HD BEAM + APBSCT + ATG excluded Kozak T, et al., Bone Marow Transplant 2008

18 Progression free survival: grafts purged in vivo vs. in vitro p = 0,175 Kozak T, et al., Bone Marow Transplant 2008

19 CONCLUSIONS Immunoablative therapy with autohsc support in MS is feasible procedure, significant number of patients may at least stabilize their otherwise intractable disease The risk of the procedure can be minimized by detailed individual risk assessment and TRM in experienced centers is < 2% AutoHSCT is more effective in eraly phase of MS In vitro purging of the graft has no significant role in the management Randomized trials are needed

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