Familial hemophagocytic lymphohistiocytosis type 3 diagnosed at school age: a case report

J Pediatr Hematol Oncol. 2014 Mar;36(2):e128-30. doi: 10.1097/MPH.0b013e318292bc7c.

Abstract

Familial hemophagocytic lymphohistiocytosis (HLH) is a rare, life-threatening condition characterized by immune hyperactivation and clinical signs of extreme inflammation. We describe a 7-year-old male who presented with fever resistant to antibiotic therapy, pancytopenia, splenomegaly, hypertriglyceridemia, and hyperferritinemia. Bone marrow aspirate showed hemophagocytosis. Epstein-Barr virus genome was positive in blood. Functional screening showed reduced capacity of cytotoxic degranulation. Mutation analysis of the FHL-related genes revealed compound heterozygous for UNC13D mutations: c. 753+1G>T, and the novel c.544C>T (p.P182S). Patients with a clinical presentation of HLH, even if older than typically seen, should be screened for familial HLH by mutation analysis.

Publication types

  • Case Reports

MeSH terms

  • Age of Onset
  • Child
  • DNA Mutational Analysis
  • Humans
  • Lymphohistiocytosis, Hemophagocytic / diagnosis*
  • Lymphohistiocytosis, Hemophagocytic / epidemiology*
  • Lymphohistiocytosis, Hemophagocytic / genetics
  • Male
  • Membrane Proteins / genetics

Substances

  • Membrane Proteins
  • UNC13D protein, human

Supplementary concepts

  • Hemophagocytic lymphohistiocytosis, familial, 3