1. ¹Department of Pediatric Hematology-Oncology, King Faisal Specialist Hospital and Research Center (KFSHRC), Riyadh, Saudi Arabia
2. ²Department of Pathology and Laboratory Medicine, KFSHRC, Riyadh, Saudi Arabia
3. ³Department of Pediatrics, King Hussein Cancer Center, Amman, Jordan

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

Received 21 June 2004; Accepted 21 October 2004; Published online 17 January 2005.

Abstract

In all, 22 patients with confirmed Fanconi anemia (FA) underwent stem cell transplantation (SCT) from HLA-matched, related donors at KFSHRC. Median age at SCT was 7.6 years (range, 2.5–14.6 years). Conditioning regimen consisted of cyclophosphamide (CY) 15 mg/kg/day intravenously (i.v.) for 4 consecutive days, in addition to equine antithymocyte globulins (ATG) given i.v. at 40 mg/kg/day for four doses pre-SCT. No radiation therapy was given. For graft-versus-host disease prophylaxis, we used cyclosporin at the standard doses; ATG was added at 20 mg/kg/dose i.v. on days 2, 4, 6, 8, 10, and 12 post-SCT (total of six doses). All patients engrafted and are alive and transfusion independent with a median follow-up time of 20.2 months (range, 3.3–59 months). One patient however developed a decrease in her WBC and platelet count. Her work-up revealed slightly hypocellular bone marrow, and a series of chimerism studies over 1 year confirmed that she has stable mixed chimerism; she remains transfusion independent. We conclude that low-dose CY without radiation therapy can be used satisfactorily in the conditioning of patients with FA undergoing related SCT.