BACKGROUND: Due to increased rates of secondary solid organ cancer in patients with severe aplastic anemia who received an irradiation-based conditioning regimen, we decided some years ago to use the combination of cyclophosphamide and antithymocyte globulin. We report the long-term follow up of patients who underwent hematopoietic stem cell transplantation from an HLA-matched sibling donor after this conditioning regimen. DESIGN AND METHODS: We analyzed 61 consecutive patients transplanted from June 1991 to February 2010, following conditioning with cyclophosphamide (200 mg/kg) and antithymocyte globulin (2.5 mg/kg/day × 5 days). RESULTS: Median age was 21 years (range 4-43); 41 of the 61 patients were adults. Median duration of the disease before hematopoietic stem cell transplantation was 93 days. All but 2 patients received bone marrow as the source of stem cells and all but 2 engrafted. Cumulative incidence of acute grade II-IV graft-versus-host disease was 23% (95%CI 13-34) and 18 developed chronic graft-versus-host disease (cumulative incidence 32% at 72 months, 95% CI 20-46). In multivariate analysis, a higher number of infused CD3 cells was associated with an increased risk of developing chronic graft-versus-host disease (P = 0.017). With a median follow up of 73 months (range 8-233), the estimated 6-year overall survival was 87% (95% CI 78-97). At 72 months, the cumulative incidence of avascular necrosis was 21% and 12 patients presented with endocrine dysfunction (cumulative incidence of 19%). Only one patient developed a secondary malignancy (Hodgkin's lymphoma) during follow up. CONCLUSIONS: Cyclophosphamide and antithymocyte globulin is an effective conditioning regimen for patients with severe aplastic anemia and is associated with low treatment-related mortality. Long-term complications include avascular necrosis and endocrine dysfunction.
BACKGROUND: Due to increased rates of secondary solid organ cancer in patients with severe aplastic anemia who received an irradiation-based conditioning regimen, we decided some years ago to use the combination of cyclophosphamide and antithymocyte globulin. We report the long-term follow up of patients who underwent hematopoietic stem cell transplantation from an HLA-matched sibling donor after this conditioning regimen. DESIGN AND METHODS: We analyzed 61 consecutive patients transplanted from June 1991 to February 2010, following conditioning with cyclophosphamide (200 mg/kg) and antithymocyte globulin (2.5 mg/kg/day × 5 days). RESULTS: Median age was 21 years (range 4-43); 41 of the 61 patients were adults. Median duration of the disease before hematopoietic stem cell transplantation was 93 days. All but 2 patients received bone marrow as the source of stem cells and all but 2 engrafted. Cumulative incidence of acute grade II-IV graft-versus-host disease was 23% (95%CI 13-34) and 18 developed chronic graft-versus-host disease (cumulative incidence 32% at 72 months, 95% CI 20-46). In multivariate analysis, a higher number of infused CD3 cells was associated with an increased risk of developing chronic graft-versus-host disease (P = 0.017). With a median follow up of 73 months (range 8-233), the estimated 6-year overall survival was 87% (95% CI 78-97). At 72 months, the cumulative incidence of avascular necrosis was 21% and 12 patients presented with endocrine dysfunction (cumulative incidence of 19%). Only one patient developed a secondary malignancy (Hodgkin's lymphoma) during follow up. CONCLUSIONS:Cyclophosphamide and antithymocyte globulin is an effective conditioning regimen for patients with severe aplastic anemia and is associated with low treatment-related mortality. Long-term complications include avascular necrosis and endocrine dysfunction.
Authors: J C W Marsh; S E Ball; P Darbyshire; E C Gordon-Smith; A J Keidan; A Martin; S R McCann; J Mercieca; D Oscier; A W W Roques; J A L Yin Journal: Br J Haematol Date: 2003-12 Impact factor: 6.998
Authors: A Bacigalupo; R Oneto; B Bruno; G Socié; J Passweg; A Locasciulli; M T Van Lint; A Tichelli; S McCann; J Marsh; P Ljungman; J Hows; P Marin; H Schrezenmeier Journal: Acta Haematol Date: 2000 Impact factor: 2.195
Authors: J Storek; A Joseph; G Espino; M A Dawson; D C Douek; K M Sullivan; M E Flowers; P Martin; G Mathioudakis; R A Nash; R Storb; F R Appelbaum; D G Maloney Journal: Blood Date: 2001-12-15 Impact factor: 22.113
Authors: R Storb; K G Blume; M R O'Donnell; T Chauncey; S J Forman; H J Deeg; W W Hu; F R Appelbaum; K Doney; M E Flowers; J Sanders; W Leisenring Journal: Biol Blood Marrow Transplant Date: 2001 Impact factor: 5.742
Authors: F Locatelli; B Bruno; M Zecca; M T Van-Lint; S McCann; W Arcese; S Dallorso; P Di Bartolomeo; F Fagioli; A Locasciulli; M Lawler; A Bacigalupo Journal: Blood Date: 2000-09-01 Impact factor: 22.113
Authors: Lindsay M Morton; Wael Saber; K Scott Baker; A John Barrett; Smita Bhatia; Eric A Engels; Shahinaz M Gadalla; David E Kleiner; Steven Pavletic; Linda J Burns Journal: Biol Blood Marrow Transplant Date: 2016-09-12 Impact factor: 5.742
Authors: Carlo Dufour; Marta Pillon; Jakob Passweg; Gerard Socié; Andrea Bacigalupo; Genny Franceschetto; Elisa Carraro; Rosi Oneto; Antonio Maria Risitano; Regis Peffault de Latour; André Tichelli; Alicia Rovo; Christina Peters; Britta Hoechsmann; Sujith Samarasinghe; Austin G Kulasekararaj; Hubert Schrezenmeier; Mahmoud Aljurf; Judith Marsh Journal: Haematologica Date: 2014-08-01 Impact factor: 9.941
Authors: David Buchbinder; Diane J Nugent; Ruta Brazauskas; Zhiwei Wang; Mahmoud D Aljurf; Mitchell S Cairo; Robert Chow; Christine Duncan; Lamis K Eldjerou; Vikas Gupta; Gregory A Hale; Joerg Halter; Brandon M Hayes-Lattin; Jack W Hsu; David A Jacobsohn; Rammurti T Kamble; Kimberly A Kasow; Hillard M Lazarus; Paulette Mehta; Kasiani C Myers; Susan K Parsons; Jakob R Passweg; Joseph Pidala; Vijay Reddy; Carmen M Sales-Bonfim; Bipin N Savani; Adriana Seber; Mohamed L Sorror; Amir Steinberg; William A Wood; Donna A Wall; Jacek H Winiarski; Lolie C Yu; Navneet S Majhail Journal: Biol Blood Marrow Transplant Date: 2012-08-01 Impact factor: 5.742
Authors: J C Marsh; R M Pearce; M B C Koh; Z Lim; A Pagliuca; G J Mufti; J Perry; J A Snowden; A J Vora; R T Wynn; N Russell; B Gibson; M Gilleece; D Milligan; P Veys; S Samarasinghe; M McMullin; K Kirkland; G Cook Journal: Bone Marrow Transplant Date: 2013-08-05 Impact factor: 5.483