Your SlideShare is downloading. ×
aplastic anemia
aplastic anemia
aplastic anemia
aplastic anemia
aplastic anemia
aplastic anemia
aplastic anemia
aplastic anemia
aplastic anemia
aplastic anemia
aplastic anemia
aplastic anemia
aplastic anemia
aplastic anemia
aplastic anemia
Upcoming SlideShare
Loading in...5
×

Thanks for flagging this SlideShare!

Oops! An error has occurred.

×
Saving this for later? Get the SlideShare app to save on your phone or tablet. Read anywhere, anytime – even offline.
Text the download link to your phone
Standard text messaging rates apply

aplastic anemia

570

Published on

Novel therapies for aplastic anemia …

Novel therapies for aplastic anemia
Richard Champlin, MD

Published in: Health & Medicine
0 Comments
0 Likes
Statistics
Notes
  • Be the first to comment

  • Be the first to like this

No Downloads
Views
Total Views
570
On Slideshare
0
From Embeds
0
Number of Embeds
3
Actions
Shares
0
Downloads
9
Comments
0
Likes
0
Embeds 0
No embeds

Report content
Flagged as inappropriate Flag as inappropriate
Flag as inappropriate

Select your reason for flagging this presentation as inappropriate.

Cancel
No notes for slide

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

×