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

J Zanis‐Neto, MED Flowers… - British journal of …, 2005 - Wiley Online Library
J Zanis‐Neto, MED Flowers, CR Medeiros, MA Bitencourt, CM Bonfim, DC Setubal, V Funke
British journal of haematology, 2005Wiley Online Library
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 …
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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