Feasibility of reduced-intensity conditioning followed by unrelated cord blood transplantation for primary hemophagocytic lymphohistiocytosis: A nationwide retrospective analysis in Japan

Akihisa Sawada, Shouichi Ohga, Eiichi Ishii, Masami Inoue, Keiko Okada, Jiro Inagaki, Hiroaki Goto, Nobuhiro Suzuki, Kazutoshi Koike, Yoshiko Atsuta, Ritsuro Suzuki, Hiromasa Yabe, Keisei Kawa, Koji Kato, Koji Yasutomo

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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/m2 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.

Original languageEnglish
Pages (from-to)223-230
Number of pages8
JournalInternational journal of hematology
Issue number2
Publication statusPublished - Aug 1 2013


All Science Journal Classification (ASJC) codes

  • Hematology

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