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Novel therapies for aplastic anemia

Novel therapies for aplastic anemia
Richard Champlin, MD

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aplastic anemia Presentation Transcript

  • 1. Hematopoietic Transplantation for Aplastic Anemia Richard Champlin, MD
  • 2. Questions for HSCT for Aplastic Anemia • Preparative regimen and GVHD prevention – Cyclophosphamide +/- ATG – Cyclophosphamide, fludarabine + ATG or alemtuzumab – ? Role of TBI for MUD • Effect of Race • Cell Source – Bone marrow – PBPC • Alternative Donors – Cord blood – Haploidentical transplants
  • 3. Champlin et al Blood 2007
  • 4. HLA id siblings 1991-2002 : CY 200 mg/kg Survival Plot p<0.0001 days from BMT % Overall Survival 1,000 0,750 0,500 0,250 0,000 0,0 1500,0 3000,0 4500,0 6000,0 GG_BMT_F Survival C54 0 1 2 3 A B C D A= <20yy n=681 83% B= 21-30yy n=339 73% C= 31-40yy n=146 68% D=>40yy n=111 51%
  • 5. Preparative regimen (current version) Days -6 -5 -4 -3 -2 -1 0 F F F F C C Rest BMT A C C T T T T From Bacigalupo et al, BMT 2005. Used in 13 patients. A: Admission. F= Fludarabine 30 mg/m2; C = Cyclophosphamide 300 mg/m2; T= Thymoglobulin 3.75 mg/kg (MRD: three doses only). GVHD prophylaxis: tacrolimus - miniMTX
  • 6. Patient characteristics (2) Cell source BM PBSC UCB 17 (MRD n=6; MUD n=11) 3 (MRD n=1; MUD n=2) 1 Donor Matched related Matched unrelated 7 (33%) 14 (67%) Prior ATG therapy 17 (80%)
  • 7. Engraftment and chimerism • ANC 500: day 15 (8-30) • PLT 20K : day 15 (9-73) • Graft failure: 2/20 evaluable (10%). • No late graft failures. • Chimerism (best): --- ≥ 90% Donor 13 (65%) --- Mixed 6 (30%) --- NE 1 (5%)
  • 8. Graft-vs-host-disease (GVHD) • GVHD prophylaxis: CSA or tacrolimus + MTX • Acute GVHD (day 100): Y / N 4/20 ev pts (20%). • Chronic GVHD (at any time): Y / N 6/16 ev pts (37%)
  • 9. Patient outcome • Eight deaths (38%). Thirteen a/w. • Causes of death: ---- graft rejection n=1 ---- EBV PTLD n=2 ---- GVHD n=2 ---- infection n=2 ---- organ failure n=1 • Median f/up: 11 mo (0.5-112). • EBV reactivation: 9/20 pts (45%), including EBV-PTLD (n=2)
  • 10. Overall survival Overall survival (actuarial estimate) for the whole group (n=20: 61%) (curve A) and for the thirteen patients treated according to Bacigalupo et al (BMT 2005; 36, 947) (curve B; 82%).
  • 11. Alemtuzumab vs ATG for HSCT for Aplastic Anemia Marsh et al BMT 2014
  • 12. Alternative Donors • Considered in patients who fail immunosuppressive therapy with ATG and have no HLA matched donor – Prep regimen not well standardized • Cord Blood- Only small reports, initially high rate of TRM, graft failure; improved recent results – Chan 7 of 9 children alive – Yamamoto M-Flu TBI 4 cGy 10 of 12 adult patients alive with engraftment Blood 2011 • Haploidentical- improved results in general with haplos using post transplant cyclophosphamide, where results are similar to matched MUD, little data in AA – Xu et al BMT 2012 N=19 Bu Cy ATG 64% survival – Gao et al BMT 2014 N=26 Cy Flu ATG 84% survival