Stem cell transplantation with reduced-intensity conditioning for hemophagocytic lymphohistiocytosis

Blood. 2006 Feb 1;107(3):1233-6. doi: 10.1182/blood-2005-05-1819. Epub 2005 Oct 11.

Abstract

Allogeneic stem cell transplantation (SCT) is curative for hemophagocytic lymphohistiocytosis (HLH). However, patients frequently have significant morbidity before transplantation and there is high transplant-related mortality (TRM). Because first-degree HLH is caused by immune dysregulation, a reduced-intensity conditioned (RIC) regimen might be sufficient for cure while decreasing the TRM. Twelve patients with HLH underwent RIC SCT from a matched family/unrelated or haploidentical donor. Eleven were conditioned with fludarabine/melphalan with additional busulphan for haploidentical grafts. One received fludarabine and 2-Gy total body irradiation (TBI). All patients showed engraftment at a median of 14 days. Nine of 12 (75%) are alive and in complete remission (CR) a median of 30 months (range, 9-73 months) after SCT. Two patients died from pneumonitis and one from hepatic rupture. Four patients developed acute graft-versus-host disease (GVHD) and 3 have chronic GVHD. Three of 9 survivors have mixed chimerism but remain free of disease. In summary, RIC compares favorably to conventional SCT with long-term disease control in surviving patients despite a significant incidence of mixed chimerism.

MeSH terms

  • Adolescent
  • Child
  • Child, Preschool
  • Combined Modality Therapy / mortality
  • Disease-Free Survival
  • Female
  • Graft Survival
  • Graft vs Host Disease / etiology
  • Graft vs Host Disease / mortality
  • Humans
  • Infant
  • Liver Diseases / etiology
  • Liver Diseases / mortality
  • Lymphohistiocytosis, Hemophagocytic / complications
  • Lymphohistiocytosis, Hemophagocytic / mortality
  • Lymphohistiocytosis, Hemophagocytic / therapy*
  • Male
  • Myeloablative Agonists / administration & dosage*
  • Pneumonia / etiology
  • Pneumonia / mortality
  • Retrospective Studies
  • Rupture, Spontaneous / etiology
  • Rupture, Spontaneous / mortality
  • Stem Cell Transplantation*
  • Transplantation Chimera
  • Transplantation Conditioning*
  • Transplantation, Homologous
  • Whole-Body Irradiation*

Substances

  • Myeloablative Agonists