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Chapter 9: Matched Sibling Donor HSCT 181 Table 2: HSCT for Fanconi Anemia from Matched Related Donors – Non-TBI-based Cytoreductive Regimens Author(s); Year Bonfim, Pasquini; Brazil 2007 Bitan, Slavin; Israel 2007 Yabe, M, Yabe, H; Japan 2008 Ebell; Germany; Personal Communication Wagner, MacMillan; Minnesota 2006 Gillio; New Jersey; Personal Communication N 43 5 5 2 3 11 Age 9 (5-29) 12 (9-31) 8 (6-17) 9.5 (4-22) Diagnosis AA AA 3 AA AA AA AA 10 AML 2 MDS 1 Cytoreduction CY 60 CY 10 Flu 180 CY 40 Flu 150 Bu 2 Flu 180 CY Flu CY 20 Flu 175 CSA/ MTX fATG 40 ATG/ Campath CSA Graft BM BM 3 Cord 1 PBSC 1 T-cell Depletion Graft Failure GvHD Acute Acute 17%; >Grade 2 Chronic Chronic 28% Mortality 6% 2 Rejection; 1 TRM GvHD Prophylaxis Disease- Free Survival rATG 5 CSA or CSA/ MTX BM fATG 60 OKT3 CSA BM PBSC ATG CSA/ Steroid ATG 150 CSA/ Steroid ATG BM BM 8 Cord 3 None None None None None Yes: 8 of 11 5/43 0 0 0 0 1/11 Acute 1; Chronic 1 None None Chronic 1 0% 0% 1/2 0% 2 of 11: 1 Leukemia; 1 GvHD 40/43 5/5 5/5 1/2 3/3 9/11 The combination of these cytoreductive regimens, followed by unmodified marrow grafts used by most centers, was associated with a risk of primary or secondary graft failure of 5-10%. Graft-versus-host disease prophylaxis included mostly cyclosporine (CSA), either alone or with steroids. A few centers used other agents, including methotrexate (MTX), ATG, OKT3 or Campath. With such regimens in the largest series, the risk of acute GvHD varied from as low as 8% (using CSA/ MTX) to as high as 55% (using CSA alone).
182 Fanconi Anemia: Guidelines for Diagnosis and Management One center, the University of Minnesota, used the CY/ Flu/ATG cytoreductive approach followed by T-cell depleted marrow grafts, resulting in minimal graft failure or GvHD. Physicians who transplant FA patients continue to test research protocols to find more effective transplantation methods. The reader is urged to contact the FA specialists listed in the Appendix for their most current protocols. The consensus of the physicians who participated in the development of these guidelines is as follows: if the local transplant center has performed fewer than five transplants for FA, strong consideration should be given for referral to a transplant center with greater experience in transplants for FA. FA patients often experience complications which are not routine in other transplants, such as a marked increased risk in organ toxicity (mucositis, GI toxicity, hemorrhagic cystitis), infections, graft failure, GvHD and the development of glucose intolerance requiring insulin therapy. Proceeding to Transplant Definitive diagnosis An FA patient being considered for a matched sibling donor BMT must first have a definitive diagnosis of FA (see Chapter 2). Patients with a proven diagnosis of FA who have a matched sibling donor are all potential candidates for transplant. The indication and timing of the transplant are sometimes controversial and depend on several factors including (1) the patient’s hematologic status; (2) the patient’s age and overall clinical condition; (3) the transplant center and experience, as well as the
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Chapter 4 Gastrointestinal, Hepatic
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Chapter 12 Novel Treatment Options
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Chapter 13 Head and Neck Squamous C
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Chapter 14 The Adult Fanconi Anemia
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