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

Int J Hematol. 2013 Aug;98(2):223-30. doi: 10.1007/s12185-013-1391-z. Epub 2013 Jul 11.

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.

Publication types

  • Clinical Trial
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Allografts
  • Antilymphocyte Serum / administration & dosage
  • Child
  • Child, Preschool
  • Cord Blood Stem Cell Transplantation
  • Disease-Free Survival
  • Female
  • Humans
  • Immunosuppressive Agents / administration & dosage
  • Infant
  • Japan / epidemiology
  • Lymphohistiocytosis, Hemophagocytic / mortality*
  • Lymphohistiocytosis, Hemophagocytic / therapy*
  • Male
  • Melphalan / administration & dosage
  • Myeloablative Agonists / administration & dosage
  • Survival Rate
  • Transplantation Conditioning*
  • Unrelated Donors*
  • Vidarabine / administration & dosage
  • Vidarabine / analogs & derivatives

Substances

  • Antilymphocyte Serum
  • Immunosuppressive Agents
  • Myeloablative Agonists
  • Vidarabine
  • fludarabine
  • Melphalan