title: Allogeneic stem cell transplantation in Fanconi anemia patients presenting with myelodysplasia and/or clonal abnormality: update on the Saudi experience.
abstract: In the literature, there is an abundance of promising data on the outcome of allogeneic stem cell transplantation (SCT) in patients with Fanconi anemia (FA); however, the data on the outcome of FA patients who present with myelodysplasia and/or abnormal clone are sketchy as the entity itself is a rare one, although, it is believed that the presence of any of these factors confers a worse prognosis on the outcome of the transplant. This is an update of our experience in 11 such patients who underwent SCT at King Faisal Specialist Hospital and Research Center; 10 from the matched and related donors and 1 from a partially matched unrelated cord blood unit; the conditioning was with the same regimen consisting of cyclophosphamide (total of 20 mg/kg), anti-thymocyte globulin (total dose 160 mg/kg of the equine product or 52 mg/kg of the rabbit product) and total-body irradiation at 450 cGy. Ten patients remain currently alive, well and with no evidence of disease, with a median follow-up of almost 4 years.
authors.affiliation: Section of Pediatric Stem Cell Transplant, Department of Pediatric Hematology-Oncology, Riyadh, Saudi Arabia. mouhab@kfshrc.edu.sa
authors.forename: M
authors.initials: M
authors.lastname: Ayas
authors.forename: A
authors.initials: A
authors.lastname: Al-Jefri
authors.forename: A
authors.initials: A
authors.lastname: Al-Seraihi
authors.forename: M
authors.initials: M
authors.lastname: Al-Mahr
authors.forename: S
authors.initials: S
authors.lastname: Rifai
authors.forename: A
authors.initials: A
authors.lastname: Al-Ahmari
authors.forename: A
authors.initials: A
authors.lastname: Khairy
authors.forename: I
authors.initials: I
authors.lastname: El-Hassan
authors.forename: H
authors.initials: H
authors.lastname: El-Solh
mesh_terms: D000293:Adolescent; D002648:Child; D005199:Fanconi Anemia / Q000150:complications / Q000628:therapy*; D005260:Female; D005500:Follow-Up Studies; D006085:Graft Survival; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D009190:Myelodysplastic Syndromes / Q000150:complications; D012529:Saudi Arabia; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous