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Artigo Acesso aberto Produção Nacional Revisado por pares
BIBTEX RIS

Low‐dose cyclophosphamide conditioning for haematopoietic cell transplantation from HLA‐matched related donors in patients with Fanconi anaemia

2005; Wiley; Volume: 130; Issue: 1 Linguagem: Inglês

10.1111/j.1365-2141.2005.05549.x

ISSN

1365-2141

Autores

J. Zanis‐Neto, Mary E.D. Flowers, Carlos Roberto de Medeiros, Marco A. Bitencourt, Carmem Bonfim, D.C. Setubal, Vaneuza Araújo Moreira Funke, Jan‐Stephan Sanders, H. Joachim Deeg, Hans‐Peter Kiem, Paul J. Martin, Wendy M. Leisenring, Rainer Storb, Ricardo Pasqüini,

Tópico(s)

Acute Lymphoblastic Leukemia research

Resumo

Summary Allogeneic haematopoietic cell transplantation (HCT) is effective therapy for Fanconi anaemia (FA). FA patients do not tolerate conditioning with 200 mg/kg of cyclophosphamide (Cy), typically used in aplastic anaemia. We previously published results of studies in which Cy doses were gradually reduced from 200 to 100 mg/kg. Here we update results of the initial studies and report data on 30 new patients conditioned with Cy either at 80 mg/kg ( n = 7) or at 60 mg/kg ( n = 23), given over 4 days before HCT from human leucocyte antigen‐matched related donors. Methotrexate and cyclosporine were given for graft‐ versus ‐host disease (GVHD) prophylaxis. All seven patients given Cy at 80 mg/kg and 21 of 23 given Cy at 60 mg/kg had sustained engraftment, while two patients, both with clonal cytogenetics abnormalities, experienced graft failure. Grades 2–3 acute GVHD rates were 57% and 14% for patients given the higher and lower Cy doses, respectively ( P = 0·001). Four patients given Cy at 80 mg/kg and 22 given Cy at 60 mg/kg were alive at a median of 47 (44–58) months and 16 (3–52) months, respectively. Cy at 60 mg/kg has acceptable toxicities, low rates of GVHD, and is sufficient for engraftment of related grafts in most FA patients.

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