title: Outcomes of hematopoietic cell transplantation in adult patients with acquired aplastic anemia using intermediate-dose alemtuzumab-based conditioning.
abstract: Graft-versus-host disease (GVHD) has no therapeutic benefit after hematopoietic cell transplantation (HCT) for patients with acquired aplastic anemia (AA), and its prevention is highly desirable. We designed a conditioning regimen using an intermediate dose of alemtuzumab (50 to 60 mg) and describe our institutional experience of 41 patients who underwent HCT for AA. The median age at HCT was 37 years (range, 17 to 59). The conditioning regimen was high-dose cyclophosphamide (n = 9) or fludarabine based (n = 32). Additional GVHD prophylaxis was with cyclosporine. With a median follow-up of 3.6 years, overall survival at 3 years was 85%. Survival in patients <40 years and ≥40 years was 96% and 67%, respectively (P = .04). Graft failure occurred in 4 (10%) patients; 2 primary and 2 secondary. The cumulative incidences of acute (grades 1 to 2) and chronic GVHD were 27% and 15%, respectively. No patients developed grade 3 to 4 acute GVHD or severe chronic GVHD. The following viral complications were frequent: cytomegalovirus reactivation (79%), herpes simplex (18%), varicella zoster (25%), and BK virus hemorrhagic cystitis (8%). The majority of patients had no significant long-term health issues. This intermediate-dose alemtuzumab-based conditioning regimen results in excellent survival with a favorable impact on GVHD and long-term health outcomes, but close monitoring for viral complications is important.
authors.affiliation: Allogeneic Blood and Marrow Transplant Program, Princess Margaret Cancer Center, University of Toronto, Toronto, Canada.
authors.forename: Nada
authors.initials: N
authors.lastname: Hamad
authors.affiliation: Department of Biostatistics, Princess Margaret Cancer Center, Toronto, Canada.
authors.forename: Ryan
authors.initials: R
authors.lastname: Del Bel
authors.affiliation: Allogeneic Blood and Marrow Transplant Program, Princess Margaret Cancer Center, University of Toronto, Toronto, Canada.
authors.forename: Hans A
authors.initials: HA
authors.lastname: Messner
authors.affiliation: Allogeneic Blood and Marrow Transplant Program, Princess Margaret Cancer Center, University of Toronto, Toronto, Canada.
authors.forename: Dennis
authors.initials: D
authors.lastname: Kim
authors.affiliation: Allogeneic Blood and Marrow Transplant Program, Princess Margaret Cancer Center, University of Toronto, Toronto, Canada.
authors.forename: John
authors.initials: J
authors.lastname: Kuruvilla
authors.affiliation: Allogeneic Blood and Marrow Transplant Program, Princess Margaret Cancer Center, University of Toronto, Toronto, Canada.
authors.forename: Jeffrey H
authors.initials: JH
authors.lastname: Lipton
authors.affiliation: Allogeneic Blood and Marrow Transplant Program, Princess Margaret Cancer Center, University of Toronto, Toronto, Canada.
authors.forename: Jieun
authors.initials: J
authors.lastname: Uhm
authors.affiliation: Allogeneic Blood and Marrow Transplant Program, Princess Margaret Cancer Center, University of Toronto, Toronto, Canada.
authors.forename: Matthew
authors.initials: M
authors.lastname: Seftel
authors.affiliation: Allogeneic Blood and Marrow Transplant Program, Princess Margaret Cancer Center, University of Toronto, Toronto, Canada.
authors.forename: Naheed
authors.initials: N
authors.lastname: Alam
authors.affiliation: Department of Biostatistics, Princess Margaret Cancer Center, Toronto, Canada.
authors.forename: Wei
authors.initials: W
authors.lastname: Xu
authors.affiliation: Allogeneic Blood and Marrow Transplant Program, Princess Margaret Cancer Center, University of Toronto, Toronto, Canada. Electronic address: vikas.gupta@uhn.ca.
authors.forename: Vikas
authors.initials: V
authors.lastname: Gupta
mesh_terms: D000293:Adolescent; D000328:Adult; D000074323:Alemtuzumab; D000741:Anemia, Aplastic / Q000188:drug therapy* / Q000401:mortality / Q000628:therapy*; D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use*; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D008297:Male; D008875:Middle Aged; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000379:methods*; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D061067:Antibodies, Monoclonal, Humanized; D000970:Antineoplastic Agents; D000074323:Alemtuzumab
keywords: Alemtuzumab; Allogeneic transplantation; Aplastic anemia; Graft-versus-host disease; Survival