1. ¹Department of Pediatric Hematology-Oncology, King Faisal Specialist Hospital & Research Center (KFSHRC), Riyadh, Saudi Arabia
2. ²Department of Pathology, King Faisal Specialist Hospital & Research Center (KFSHRC), Riyadh, Saudi Arabia

Correspondence: Dr M Ayas, Department of Pediatric Hematology-Oncology, King Faisal Specialist Hospital and Research Center MBC 53, P.O. Box 3354, Riyadh 11211, Saudi Arabia. E-mail: mouhab@kfshrc.edu.sa

Received 24 June 2003; Accepted 26 August 2003; Published online 27 October 2003.

Abstract

Five patients with confirmed Fanconi's anemia (FA) and myelodysplasia and/or leukemia underwent stem cell transplantation (SCT) from related donors at KFSHRC. The median age at SCT was 12.6 year (range, 6.2–15 years). Conditioning regimen consisted of cyclophosphamide (CY) 5 mg/kg/day i.v. for 4 days, total body irradiation (TBI) 450 cGy in a single dose. Graft-versus-host disease (GVHD) prophylaxis was with cyclosporine and antithymocyte globulins (ATG). The median time to engraftment (defined as ANC0.5 10⁹/l) was 16 days (range, 12–26 days). The median time to a self-sustaining platelet count of 20 10⁹/l was 27 days (range, 12–40 days). All patients engrafted. Two patients developed acute GVHD; one of the gut (grade 3) and the other of the skin (grade 1), and one patient developed chronic GVHD of the liver. Four are alive and well with no evidence of the disease; one patient died of bacterial sepsis after controlling her GVHD and clearing her pulmonary aspergillosis and CMV infection. We conclude that the use of low-dose CY plus TBI in patients with FA and MDS/AML undergoing SCT is adequate; the regimen is well tolerated and may be curative for such patients.