Allogeneic hematopoietic stem cell transplantation from HLA-matched related donors in patients with Fanconi aneamia-3 cases report

Objective To investigate the clinic effect of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for patients with Fanconi's anemia (FA).Method Three cases of FA were successfully subjected to matched sibling donor allo-HSCT,and a fludarabine (Flu)-based conditioning regimen was used.The regimen contained Flu,reduced-dose cyclophosphamide (40mg/kg) and antihuman thymocyte globulin.Result All 3 patients achieved engraftment with donor chimerism.One patient had secondary graft failure at the + 118th day,and a second allo-HSCT performed at + 182nd day engrafted with donor chimerism again.During a follow-up period of 21,20and 27 months,respectively,all 3 patients were transfusion-independent,and maintained full donor chimerism.None of the patients developed severe graft-versus-host disease,hepatic veno-occlusive disease or other serious complications.Conclusion Sibling donor allo-HSCT with this alternative conditioning regimen can be used to effectively treat FA patients who develop aplasia. Key words: Fanconi anemia;  Anemia, aplastic;  Cyclophosphamide;  Fludarabine;  Hematopoietic stem cell transplantation