BACKGROUND: We analyzed the outcome of 100 patients with acquired severe aplastic anemia undergoing an alternative donor transplant, after immune suppressive therapy had failed. DESIGN AND METHODS: As a conditioning regimen, patients received either a combination of fludarabine, cyclophosphamide, and antithymocyte globulin (n=52, median age 13 years) or this combination with the addition of low dose (2 Gy) total body irradiation (n=48, median age 27 years). RESULTS: With a median follow-up of 1665 and 765 days, the actuarial 5-year survival was 73% for the group that received fludarabine, cyclophosphamide, and antithymocyte globulin and 79% for the group given the conditioning regimen including total body irradiation. Acute graft-versus-host disease grade III-IV was seen in 18% and 7% of the groups, respectively. Graft failure was seen in 17 patients with an overall cumulative incidence of 17% in patients receiving conditioning with or without total body irradiation: 9 of these 17 patients survive in the long-term. The most significant predictor of survival was the interval between diagnosis and transplantation, with 5-year survival rates of 87% and 55% for patients grafted within 2 years of diagnosis and more than 2 years after diagnosis, respectively (P=0.0004). Major causes of death were graft failure (n=7), post-transplant-lymphoproliferative-disease (n=4) and graft-versus-host disease (n=4). CONCLUSIONS: This study confirms positive results of alternative donor transplants in patients with severe aplastic anemia, the best outcomes being achieved in patients grafted within 2 years of diagnosis. Prevention of rejection and Epstein-Barr virus reactivation may further improve these results.
BACKGROUND: We analyzed the outcome of 100 patients with acquired severe aplastic anemia undergoing an alternative donor transplant, after immune suppressive therapy had failed. DESIGN AND METHODS: As a conditioning regimen, patients received either a combination of fludarabine, cyclophosphamide, and antithymocyte globulin (n=52, median age 13 years) or this combination with the addition of low dose (2 Gy) total body irradiation (n=48, median age 27 years). RESULTS: With a median follow-up of 1665 and 765 days, the actuarial 5-year survival was 73% for the group that received fludarabine, cyclophosphamide, and antithymocyte globulin and 79% for the group given the conditioning regimen including total body irradiation. Acute graft-versus-host disease grade III-IV was seen in 18% and 7% of the groups, respectively. Graft failure was seen in 17 patients with an overall cumulative incidence of 17% in patients receiving conditioning with or without total body irradiation: 9 of these 17 patients survive in the long-term. The most significant predictor of survival was the interval between diagnosis and transplantation, with 5-year survival rates of 87% and 55% for patients grafted within 2 years of diagnosis and more than 2 years after diagnosis, respectively (P=0.0004). Major causes of death were graft failure (n=7), post-transplant-lymphoproliferative-disease (n=4) and graft-versus-host disease (n=4). CONCLUSIONS: This study confirms positive results of alternative donor transplants in patients with severe aplastic anemia, the best outcomes being achieved in patients grafted within 2 years of diagnosis. Prevention of rejection and Epstein-Barr virus reactivation may further improve these results.
Authors: A Bacigalupo; F Locatelli; E Lanino; J Marsh; G Socié; S Maury; A Prete; A Locasciulli; S Cesaro; J Passweg Journal: Bone Marrow Transplant Date: 2005-12 Impact factor: 5.483
Authors: R Storb; W Leisenring; C Anasetti; F R Appelbaum; C D Buckner; W I Bensinger; T Chauncey; R A Clift; H J Deeg; K C Doney; M E Flowers; J A Hansen; P J Martin; J E Sanders; K M Sullivan; R P Witherspoon Journal: Blood Date: 1997-05-15 Impact factor: 22.113
Authors: Ramaprasad Srinivasan; Yoshiyuki Takahashi; J Philip McCoy; Igor Espinoza-Delgado; Colleen Dorrance; Takehito Igarashi; Andreas Lundqvist; A John Barrett; Neal S Young; Nancy Geller; Richard W Childs Journal: Br J Haematol Date: 2006-05 Impact factor: 6.998
Authors: H J Deeg; I D Amylon; R E Harris; R Collins; P G Beatty; S Feig; N Ramsay; M Territo; S P Khan; D Pamphilon; J F Leis; S Burdach; C Anasetti; R Hackman; B Storer; B Mueller Journal: Biol Blood Marrow Transplant Date: 2001 Impact factor: 5.742
Authors: S R McCann; A Bacigalupo; E Gluckman; W Hinterberger; J Hows; P Ljungman; P Marin; C Nissen; E van't Veer Kerthof; A Raghavachar Journal: Bone Marrow Transplant Date: 1994-03 Impact factor: 5.483
Authors: Mark Lawler; Shaun R McCann; Judith C W Marsh; Per Ljungman; Jill Hows; Elisabeth Vandenberghe; Joan O'Riordan; Anna Locasciulli; Gérard Socié; Alan Kelly; Hubert Schrezenmeier; Pedro Marin; André Tichelli; Jakob R Passweg; Anne Dickenson; Jacqueline Ryan; Andrea Bacigalupo Journal: Br J Haematol Date: 2009-01-09 Impact factor: 6.998
Authors: J Y Pierga; G Socie; E Gluckman; A Devergie; M Henry-Amar; A Bridier; T Girinsky; J Nguyen; J M Cosset Journal: Radiother Oncol Date: 1994-01 Impact factor: 6.280
Authors: R Kumar; S Prem; M Mahapatra; T Seth; D R Chowdhary; P Mishra; L Pillai; A M V R Narendra; N K Mehra; R Saxena; V P Choudhry Journal: Bone Marrow Transplant Date: 2006-04 Impact factor: 5.483
Authors: J Aoki; S Seo; H Kanamori; M Tanaka; T Fukuda; M Onizuka; N Kobayashi; T Kondo; M Sawa; N Uchida; K Iwato; T Icihnohe; Y Atsuta; S Yano; A Takami Journal: Bone Marrow Transplant Date: 2015-12-07 Impact factor: 5.483
Authors: Michael A Pulsipher; Neal S Young; Jakub Tolar; Antonio M Risitano; H Joachim Deeg; Paolo Anderlini; Rodrigo Calado; Seiji Kojima; Mary Eapen; Richard Harris; Phillip Scheinberg; Sharon Savage; Jaroslaw P Maciejewski; Ramon V Tiu; Nancy DiFronzo; Mary M Horowitz; Joseph H Antin Journal: Biol Blood Marrow Transplant Date: 2010-10-27 Impact factor: 5.742