title: Feasibility of reduced-intensity conditioning followed by unrelated cord blood transplantation for primary hemophagocytic lymphohistiocytosis: a nationwide retrospective analysis in Japan.
abstract: A nationwide retrospective analysis was performed on patients who received allogeneic hematopoietic stem cell transplantation for primary or familial hemophagocytic lymphohistiocytosis (HLH) in Japan. The present analysis investigated whether reduced-intensity conditioning (RIC) followed by cord blood transplantation (CBT) (RIC-CBT) is feasible, compared to the outcomes of myeloablative conditioning and bone marrow transplantation. Based on the JSHCT data, 53 patients were analyzed. The overall survival rate (OS) was 65.4 ± 6.6 %. RIC-CBT (n = 13) was not inferior to other methods. Patients with a performance status of PS 4 (ECOG scale) with HLH-associated severe organ dysfunction during the initiation of conditioning had extremely poor outcomes. The OS rate in the RIC-CBT patients, excluding those with a performance status 4, was 80.0 ± 12.6 %. RIC may reduce treatment-related mortality; in addition, patients with engraftment failure, which is the main adverse event following RIC-CBT, were successfully rescued with secondary CBT. Unrelated cord blood may represent an alternative source if a patient has no related donor. As a RIC regimen for CBT, 140 mg/m(2) melphalan with fludarabine and anti-lymphocyte globulin or anti-thymocyte globulin may be feasible, but further dosage optimization should be performed in controlled clinical trials.
authors.affiliation: Department of Hematology/Oncology, Osaka Medical Center and Research Institute for Maternal and Child Health, 840 Murodo, Izumi, Osaka 594-1101, Japan. asawada@mch.pref.osaka.jp
authors.forename: Akihisa
authors.initials: A
authors.lastname: Sawada
authors.forename: Shouichi
authors.initials: S
authors.lastname: Ohga
authors.forename: Eiichi
authors.initials: E
authors.lastname: Ishii
authors.forename: Masami
authors.initials: M
authors.lastname: Inoue
authors.forename: Keiko
authors.initials: K
authors.lastname: Okada
authors.forename: Jiro
authors.initials: J
authors.lastname: Inagaki
authors.forename: Hiroaki
authors.initials: H
authors.lastname: Goto
authors.forename: Nobuhiro
authors.initials: N
authors.lastname: Suzuki
authors.forename: Kazutoshi
authors.initials: K
authors.lastname: Koike
authors.forename: Yoshiko
authors.initials: Y
authors.lastname: Atsuta
authors.forename: Ritsuro
authors.initials: R
authors.lastname: Suzuki
authors.forename: Hiromasa
authors.initials: H
authors.lastname: Yabe
authors.forename: Keisei
authors.initials: K
authors.lastname: Kawa
authors.forename: Koji
authors.initials: K
authors.lastname: Kato
authors.forename: Koji
authors.initials: K
authors.lastname: Yasutomo
mesh_terms: D000293:Adolescent; D064591:Allografts; D000961:Antilymphocyte Serum / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage; D007223:Infant; D007564:Japan / Q000453:epidemiology; D051359:Lymphohistiocytosis, Hemophagocytic / Q000401:mortality* / Q000628:therapy*; D008297:Male; D008558:Melphalan / Q000008:administration & dosage; D019653:Myeloablative Agonists / Q000008:administration & dosage; D015996:Survival Rate; D019172:Transplantation Conditioning; D061349:Unrelated Donors; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D019653:Myeloablative Agonists; D014740:Vidarabine; C024352:fludarabine; D008558:Melphalan