Stem cell transplantation for patients with Fanconi anemia with low-dose cyclophosphamide and antithymocyte globulins without the use of radiation therapy.
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.
Ayas
M
M
Department of Pediatric Hematology-Oncology, King Faisal Specialist Hospital and Research Center (KFSHRC), Riyadh, Saudi Arabia. mouhab@kfshrc.edu.sa
Al-Jefri
A
A
Al-Mahr
M
M
Rifai
S
S
Al-Seraihi
A
A
Tbakhi
A
A
Mustafa
M
M
Khairy
A
A
Moussa
E
E
Iqbal
A
A
Shalaby
L
L
El-Solh
H
H
D000293:Adolescent; D000961:Antilymphocyte Serum / Q000008:administration & dosage*; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage*; D005199:Fanconi Anemia / Q000150:complications / Q000628:therapy*; D005260:Female; D005500:Follow-Up Studies; D006085:Graft Survival; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D006801:Humans; D007239:Infections / Q000188:drug therapy; D008297:Male; D015996:Survival Rate; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous
D000961:Antilymphocyte Serum; D003520:Cyclophosphamide