Defective UNC13D gene-associated familial hemophagocytic lymphohistiocytosis triggered by visceral leishmaniasis: a diagnostic challenge

J Pediatr Hematol Oncol. 2014 Jan;36(1):e42-5. doi: 10.1097/MPH.0b013e31829b7f22.

Abstract

Background: Visceral leishmaniasis (VL) triggered genetic hemophagocytic lymphohistiocytosis (HLH) is clinically challenging.

Observations: One-year-old VL-HLH patient improved after liposomal-amphotericin-B therapy, but subsequently deteriorated, although bone marrow amastigotes disappeared. Symptoms resolved after 8 weeks of HLH-2004 therapy but recurred upon cessation. Homozygous UNC13D gene 627delT mutation was identified however stem cell donor was unavailable. The patient died at age 4 years after central nervous system attacks and HLH recurrences.

Conclusions: VL in HLH patients does not exclude a genetic etiology and requires structured clinical management. VL should be excluded in all HLH patients in endemic regions before immunochemotherapy, which is recommended for VL-HLH patients unresponsive to VL treatment and/or reactivated.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Diagnosis, Differential
  • Female
  • Humans
  • Infant
  • Leishmaniasis, Visceral / complications*
  • Leishmaniasis, Visceral / diagnosis*
  • Lymphohistiocytosis, Hemophagocytic / genetics*
  • Lymphohistiocytosis, Hemophagocytic / parasitology*
  • Membrane Proteins / genetics*

Substances

  • Membrane Proteins
  • UNC13D protein, human