title: Hematopoietic Stem Cell Transplant in Adolescent and Young Adults With Fanconi Anemia Is Feasible With Acceptable Toxicity, With Those Surviving 100 Days Posttransplant Having Excellent Outcomes.
abstract: Fanconi anemia is a congenital bone marrow failure syndrome that is associated with congenital anomalies and increased risk of cancer. Hematopoietic stem cell transplant is a potentially curative modality for bone marrow failure in Fanconi anemia patients. Here, we report our center's experience regarding adolescent and young adult patients with Fanconi anemia and hematopoietic stem cell transplant.
  
  
  
  We conducted a retrospective patient record analyses of patients who presented at our center from 1988 to 2014. We included patients greater than 14 years old with confirmed Fanconi anemia based on positive chromosome breakage study and who underwent hematopoietic stem cell transplant at our institution.
  
  
  
  Our study group comprised 12 patients with Fanconi anemia who underwent hematopoietic stem cell transplant at our institution. The median age was 20 years (range, 14-31 y) with a female predominance of 83%. Low-dose cyclophosphamide (20-80 mg/kg)-based conditioning regimens were used with different combinations that included fludarabine, antithymocyte globulin, or total body irradiation. All patients had HLA-matched sibling grafts. In all patients, stem cell source was the bone marrow. All patients showed engraftment. Four patients (33%) developed acute graft-versus-host disease. Three patients (25%) died early before day 100 after hematopoietic stem cell transplant due to infectious complications, with 1 patient having steroid refractory acute graft-versus-host disease. Overall survival was 75% at a median follow-up of 43 months. All patients who survived are well and remained transfusion independent without evidence of secondary malignancy.
  
  
  
  Our findings support the feasibility of reduced intensity conditioning allogeneic hematopoietic stem cell transplant in older and more heavily pretreated patients with Fanconi anemia, especially for those who are engrafted.
authors.forename: Ahmad
authors.initials: A
authors.lastname: Alhuraiji
authors.forename: Hazza
authors.initials: H
authors.lastname: Alzahrani
authors.forename: Fahad
authors.initials: F
authors.lastname: Al Mohareb
authors.forename: Naeem
authors.initials: N
authors.lastname: Chaudhri
authors.forename: Fahad
authors.initials: F
authors.lastname: Alsharif
authors.forename: Said
authors.initials: S
authors.lastname: Mohamed
authors.forename: Walid
authors.initials: W
authors.lastname: Rasheed
authors.forename: Ghuzayel
authors.initials: G
authors.lastname: Aldawsari
authors.forename: Syed Osman
authors.initials: SO
authors.lastname: Ahmed
authors.affiliation: From the Hematology/HSCT Section, Oncology Department, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia
authors.forename: Mahmoud
authors.initials: M
authors.lastname: Aljurf
mesh_terms: D000293:Adolescent; D000328:Adult; D016026:Bone Marrow Transplantation; D005199:Fanconi Anemia / Q000401:mortality / Q000628:therapy*; D005260:Female; D006087:Graft vs Host Reaction; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D011183:Postoperative Complications / Q000188:drug therapy* / Q000276:immunology / Q000401:mortality*; D012189:Retrospective Studies; D016019:Survival Analysis; D015996:Survival Rate; D013997:Time Factors; D019172:Transplantation Conditioning; D055815:Young Adult