Stem Cell Transplantation In Patients with Fanconi Anemia

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1 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

2 Improvements in Unrelated Donor Transplant Outcomes for Non-Malignant Disorders 100 Probability, % (n=2201) (n=767) (n=846) Years 2 3 * Includes severe aplastic anemia, inherited erythrocyte abnormalities, platelet disorders, histiocytic disorders, inherited metabolism disorders, osteopetrosis, and immune deficiencies 2

3 Improvements in Transplant Outcome for Non-Malignant Disorders Better supportive care Improved HLA matching of unrelated donors Improved understanding of etiology and basic pathophysiology of these disorders Better understanding of gaps in previous transplant strategies for these disorders

4 The Best example.. HCT for Fanconi Anemia

5 Transplantation for FA Only potentially curative option Indications for Transplantation - Marrow failure requiring transfusions - Myelodysplastic syndrome - Leukemia

6 HCT for FA Historical Perspective Gluckman and colleagues (Paris): 1970s and 1980s Transplanted patients with FA using Cyclophosphamide 50 mg/kg of 4 days, based on prior experience in leukemia and SAA High morbidity and mortality Four out of 5 patients died, 1 alive Gluckman E. et al. Br J Haematol. 1980

7 HCT for FA Historical Perspective First clinical evidence - special sensitivity of FA cells to alkylating chemotherapy agents The same group also confirmed the increased radiation-sensitivity in these patients New standard: - low-dose cyclophosphamide (20 mg/kg) - with 5 Gy thoraco-abdominal irradiation Gluckman E et al. Br J Haematol. 1983, Gluckman E. Stem Cells. 1993

8 HCT for FA - Progressive Refinement of conditioning Reduced doses of DNA damaging agents Addition of antithymocyte globulin (ATG) Addition of Fludarabine No Total body irradiation (TBI) in matched sibling donor setting Improved outcomes of Unrelated donor (URD) transplants (75-80% survival) Ayas M et al., Bone Marrow Transplantation, 2008; Wagner JE et al. Blood 2007

9 Unrelated Donor Transplant Outcomes: Cincinnati Last Cincinnati protocol for unrelated donor transplants used fludarabine, cyclophosphamide, ATG and radiation (single dose 450cGY) T-cell depletion 32 patients treated 25 alive and well Overall survival (OS) 78%

10 HCT for Fanconi Anemia Latest Efforts Brazil Group - CY/Flu/ATG German Group - Campath/Bu/Flu/CY Chao MM/Ebell W et al. Ann Hematol US (Multi-Institutional study) - Pharmcokinetically adjusted Bu + CY/Flu/ATG Unrelated donor transplant without TBI?

11 Multi-institutional Study of Chemotherapyonly Preparative Regimen for Alternative Donor Hematopoietic Cell Transplantation for Patients with FA

12 Chemotherapy-only Preparative Regimen DONOR G-CSF 10 mcg/kg/dose Daily SC PBSC Collection PATIENT BU PK studies BU BU Flu Flu Flu Flu CY CY CY CY CD34+ selection/t cell depletion CD34+ selection / T-cell depleted PBSCT ATG ATG ATG ATG Start CSA G-CSF Steroids

13 Chemotherapy-only Preparative Regimen for Alternative Donor HCT for Patients with FA Planned dose de-escalation for Bu - First 25 patients: mg/kg/dose IV q 12hrs x 4 - Next 20 patients: mg/kg/dose IV q 12hrs x 4 < 10 kg: 0.6 mg/kg/dose 10 kg but 4 years old: 0.8 mg/kg/dose > 4 years: 0.6 mg/kg/dose Bu pharmacokinetic results (Pk) were utilized to adjust the next 3 doses when required

14 Chemotherapy-only Preparative Regimen for Alternative Donor HCT for Patients with FA Cyclophosphamide (CY) 10mg/kg/day x 4 Fludarabine (Flu) 35mg/m2/day x 4 ATG 2.5mg/kg/day x 4

15 Chemotherapy-only Preparative Regimen for Alternative Donor HCT for Patients with FA Target Cell dose: - CD34+ cell dose of > 5.0 x 10 6 cells/kg recipient weight (min 2.5 x 10 6 cells/kg) - Maximum CD3+ cell dose of <5.0 x 10 4 cells/kg of recipient weight

16 Chemotherapy-only Preparative Regimen for Alternative Donor HCT for Patients with FA Transplant Centers Number of Transplants Total patients transplanted 45 Cincinnati 29 Memorial Sloan Kettering 10 Boston 3 Seattle 2 Wisconsin 1

17 Patient Demographics Characteristics Number Median age in years 8.2 (4.3-44) <10 years of age years of age 18 Gender - Males 20 - Females 25

18 Disease Status at Transplant and Follow-up Characteristics Number Severe Single Lineage Cytopenia 5 Severe Aplastic Anemia 29 Myelodysplastic Syndrome 11 - Low grade 7 - High grade 4 History of transfusions 37 Past use of androgens 18 Median follow-up in months 30.6 ( )

19 Donor Source and Cell Dose Characteristics Median (Range) Mismatched related (7/8, 6/8, 5/8 and 4/8 matched donors) Unrelated donors - 8/8 matched - 7/8 matched CD34+ cells/kg CD3 cells/kg x 10 6 ( ) 0.9 x 10 4 ( )

20 Engraftment Characteristic Number Total number of patients 45 Evaluable for engraftment * 44 Engrafted 43 Late graft failure * 1 Days to Neutrophil engraftment 9 (7-15) Days to Platelet engraftment 16 (11-230) * One patient not evaluable for engraftment due to early relapse * Mosaic

21 Transplant Complications/Toxicity Complication First Cohort Second Cohort Oral mucositis 15 9 Hyperbilirubinemia 9 3 Hypertension 8 3 Sinusoidal obstruction syndrome (SOS)* 1 0 Infections (number of patients) Bacterial Viral Fungal 2 3 *No SOS since lowering target Bu levels to Css of <350ng/ml

22 Graft vs Host Disease (GVHD) Complication Number GVHD Acute GVHD - Acute Gr I-II 4 - Acute Gr III-IV 0 Chronic GVHD - Chronic, limited 3 - Chronic, extensive 0

23 Overall Survival for the Group (n=45) 80 %

24 Overall Survival by Age 87 % 61 %

25 OS by Disease Status at the time of Transplant 91 % 63 %

26 OS by Busulfan Dosing Group 80 %

27 OS in Age<10 with SAA according to Bu dose 100 % 86 %

28 Conclusion Chemotherapy only preparative regimen leads to excellent OS and DFS in patients with FA; comparable to historical controls Lower dose Bu yields outcomes comparable to previous results, while avoiding short-term and potential long-term toxicity associated with radiation

29 Conclusion Longer follow-up is needed to assess late effects and secondary neoplasms Transplant strategies for older patients and those with MDS/AML need to be optimized further to improve their outcomes

30 What s next? Our new Risk adjusted protocol Bu dose is adjusted based on: - age - disease status at transplant Providing personalized therapy for patients with FA

31 Risk-Adjusted Chemotherapy-only Preparative Regimen for Alternative Donor HCT for Patients with FA

32 Patient Demographics Characteristics Number Total patients transplanted 7 Median (range) age in years 9 ( ) <10 years of age 4 10 years of age 3 Gender - Males 3 - Females 4

33 Disease Status at Transplant and Follow-up Characteristics Number Progressive marrow failure 5 Myelodysplastic Syndrome (low grade) 1 Acute Myeloid leukemia 1 History of transfusions 5 Past use of androgens 3 Median (range) follow-up in months 9.4 (1-12.9)

34 Donor Source and Cell Dose Characteristics Unrelated donors - 8/8 matched - 7/8 matched - 6/8 Median (Range) CD34+ cells/kg - Median (range) CD3 cells/kg 19.6 x 10 6 ( ) 1 x 10 4 ( )

35 Engraftment Characteristic Number Evaluable for engraftment 7 Engrafted 7 Days to Neutrophil engraftment 9 (8-10) Days to Platelet engraftment 16 (13-31)

36 Transplant Complications/Toxicity Complication Number of patients Oral mucositis 5 Hyperbilirubinemia 0 Hypertension 2 Sinusoidal obstruction syndrome (SOS)* 1 Infections (number of patients) - Bacterial 1 - Viral 6 - Fungal 0

37 Cause of Death Progressive Post Transplant Lymphoproliferative Disorder (PTLD)

38 Graft vs Host Disease (GVHD) Complication Number GVHD Acute GVHD 0 Chronic GVHD 0

39 Thank You

40 Cincinnati Fanconi Anemia Team Clinical Team Stella M Davies Richard Harris Parinda A Mehta Kasiani Myers Robin Mueller Erica Goodridge Kathleen Ball Michelle Harris Susan Rose Research Collaborators Suzanne Wells Qishen Pang Paul Andreasson Melinda Butsch Kovacic Ruhi Meetei Yi Zheng Hartmut Geiger Lindsey - Romick- Rosendale

41 Thank You Patients with FA and their Families

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