BACKGROUND: To potentially reduce late effects of malignancy, chronic graft-versus-host disease (GVHD), endocrinopathy, and infertility in patients with Fanconi anemia (FA) undergoing HLA-matched related donor hematopoietic cell transplantation (HCT), we developed a regimen using fludarabine (FLU), cyclophosphamide (CY), and anti-thymocyte globulin (ATG) followed by infusion of T-cell depleted (TCD) bone marrow (BM) or unmanipulated umbilical cord blood (UCB). GVHD prophylaxis consisted of cyclosporine and short course methylprednisolone. PROCEDURE: Between April 2000 and June 2003, 11 patients (10 aplastic anemia (AA), 1 myelodysplastic syndrome (MDS)) underwent HCT using this regimen. Stem cell sources were BM and UCB in eight and three patients, respectively. RESULTS: All patients demonstrated primary engraftment. Median days to neutrophil and platelet engraftment were 11 days (range 9-21) and 38 days (range 19-381), respectively. No patient developed GVHD after primary HCT. The patient with MDS relapsed with AML and a maternal donor recipient experienced secondary graft failure. For the nine FA patients with AA who underwent HLA-identical sibling donor HCT, the Kaplan-Meier estimates of overall survival and event-free survival (EFS) at 2 years are 100% and 82%, respectively, at a median follow-up of 2.9 years (range 1.9-4.8). CONCLUSIONS: In summary, a FLU-based, non-irradiation approach is effective for FA patients with AA undergoing HLA-identical sibling donor HCT.
BACKGROUND: To potentially reduce late effects of malignancy, chronic graft-versus-host disease (GVHD), endocrinopathy, and infertility in patients with Fanconi anemia (FA) undergoing HLA-matched related donor hematopoietic cell transplantation (HCT), we developed a regimen using fludarabine (FLU), cyclophosphamide (CY), and anti-thymocyte globulin (ATG) followed by infusion of T-cell depleted (TCD) bone marrow (BM) or unmanipulated umbilical cord blood (UCB). GVHD prophylaxis consisted of cyclosporine and short course methylprednisolone. PROCEDURE: Between April 2000 and June 2003, 11 patients (10 aplastic anemia (AA), 1 myelodysplastic syndrome (MDS)) underwent HCT using this regimen. Stem cell sources were BM and UCB in eight and three patients, respectively. RESULTS: All patients demonstrated primary engraftment. Median days to neutrophil and platelet engraftment were 11 days (range 9-21) and 38 days (range 19-381), respectively. No patient developed GVHD after primary HCT. The patient with MDS relapsed with AML and a maternal donor recipient experienced secondary graft failure. For the nine FA patients with AA who underwent HLA-identical sibling donor HCT, the Kaplan-Meier estimates of overall survival and event-free survival (EFS) at 2 years are 100% and 82%, respectively, at a median follow-up of 2.9 years (range 1.9-4.8). CONCLUSIONS: In summary, a FLU-based, non-irradiation approach is effective for FA patients with AA undergoing HLA-identical sibling donor HCT.
Authors: Lisa J McReynolds; Youjin Wang; Ashley S Thompson; Bari J Ballew; Jung Kim; Blanche P Alter; Belynda Hicks; Bin Zhu; Kristine Jones; Stephen R Spellman; Tao Wang; Stephanie J Lee; Sharon A Savage; Shahinaz M Gadalla Journal: Biol Blood Marrow Transplant Date: 2020-01-23 Impact factor: 5.742
Authors: Ricardo Pasquini; Jeanette Carreras; Marcelo C Pasquini; Bruce M Camitta; Anders L Fasth; Gregory A Hale; Richard E Harris; Judith C Marsh; Anibal J Robinson; Mei-Jie Zhang; Mary Eapen; John E Wagner Journal: Biol Blood Marrow Transplant Date: 2008-10 Impact factor: 5.742
Authors: Christen L Ebens; Todd E DeFor; Rebecca Tryon; John E Wagner; Margaret L MacMillan Journal: Biol Blood Marrow Transplant Date: 2017-12-02 Impact factor: 5.742
Authors: Mouhab Ayas; Wael Saber; Stella M Davies; Richard E Harris; Gregory A Hale; Gerard Socie; Jennifer LeRademacher; Monica Thakar; H Joachim J Deeg; Amal Al-Seraihy; Minoo Battiwalla; Bruce M Camitta; Richard Olsson; Rajinder S Bajwa; Carmem M Bonfim; Ricardo Pasquini; Margaret L Macmillan; Biju George; Edward A Copelan; Baldeep Wirk; Abdullah Al Jefri; Anders L Fasth; Eva C Guinan; Biljana N Horn; Victor A Lewis; Shimon Slavin; Polina Stepensky; Marc Bierings; Robert Peter Gale Journal: J Clin Oncol Date: 2013-04-01 Impact factor: 44.544
Authors: M S Thakar; P Kurre; R Storb; M Kletzel; H Frangoul; M A Pulsipher; W Leisenring; M E D Flowers; B M Sandmaier; A Woolfrey; H-P Kiem Journal: Bone Marrow Transplant Date: 2010-06-28 Impact factor: 5.483
Authors: Navneet S Majhail; Jaya M Mothukuri; Margaret L Macmillan; Michael R Verneris; Paul J Orchard; John E Wagner; Daniel J Weisdorf Journal: Pediatr Blood Cancer Date: 2010-01 Impact factor: 3.167