title: Prognostic Factors and Long-Term Outcome in 52 Turkish Children With Hemophagocytic Lymphohistiocytosis.
abstract: OBJECTIVE
  Hemophagocytic lymphohistiocytosis is a syndrome of pathologic immune activation that shares similar clinical and laboratory phenotypes with severe sepsis. Recent studies led to better recognition of hemophagocytic lymphohistiocytosis by clinicians, but no consensus exists on the criteria for high-risk patients.
  
  
  METHODS
  We retrospectively reviewed the medical records of patients diagnosed with hemophagocytic lymphohistiocytosis to analyze the risk factors associated with poor outcome.
  
  
  METHODS
  Pediatric intensive care and hematology units of three tertiary hospitals in Turkey.
  
  
  METHODS
  Fifty-two children with hemophagocytic lymphohistiocytosis.
  
  
  METHODS
  None.
  
  
  RESULTS
  There were a total of 52 children meeting the diagnostic criteria of Histiocytic Society. Of them, 28 (54%) had a primary hemophagocytic lymphohistiocytosis. Mutation studies were performed in 18 of 28 patients (65%). Fourteen of them had PRF1, STX11, STXBP2, and UNC13D mutations, and four had Rab27a and LYST mutations. The remaining 24 patients (46%) were defined as having secondary hemophagocytic lymphohistiocytosis. Twenty-one of them had infection-associated hemophagocytic lymphohistiocytosis, and three had lysinuric protein intolerance. The mortality rate was significantly higher in primary hemophagocytic lymphohistiocytosis (64%) than in secondary hemophagocytic lymphohistiocytosis (16%) (p < 0.05). There were no significant differences for survival rate between hemophagocytic lymphohistiocytosis 94 (44%) and hemophagocytic lymphohistiocytosis 2004 (64%) protocols (p > 0.05). Age below 2 years, hyperferritinemia, thrombocytopenia, high disseminated intravascular coagulation score at diagnosis, and no clinical response at 2 weeks of treatment were independent prognostic factors for poor prognosis.
  
  
  CONCLUSIONS
  Our data suggest that disseminated intravascular coagulation score greater than or equal to 5 can be used in the definition of high-risk patients. Early recognition of poor risk factors has important prognostic and therapeutic implications.
authors.affiliation: 1Pediatric Hematology Unit, Department of Pediatrics, Medical School of Gazi University, Ankara, Turkey. 2Department of Pediatrics, Medical School of Gaziantep University, Gaziantep, Turkey. 3Department of Pediatrics, Medical School of Kirikkale University, Kirikkale, Turkey.
authors.forename: Zühre
authors.initials: Z
authors.lastname: Kaya
authors.forename: Ali
authors.initials: A
authors.lastname: Bay
authors.forename: Meryem
authors.initials: M
authors.lastname: Albayrak
authors.forename: Ulker
authors.initials: U
authors.lastname: Kocak
authors.forename: Idil
authors.initials: I
authors.lastname: Yenicesu
authors.forename: Turkiz
authors.initials: T
authors.lastname: Gursel
mesh_terms: D002648:Child; D002675:Child, Preschool; D004211:Disseminated Intravascular Coagulation / Q000209:etiology; D005260:Female; D006801:Humans; D007223:Infant; D051359:Lymphohistiocytosis, Hemophagocytic / Q000209:etiology* / Q000382:microbiology / Q000628:therapy*; D008297:Male; D008565:Membrane Proteins / Q000235:genetics; D051938:Munc18 Proteins / Q000235:genetics; D054353:Perforin / Q000235:genetics; D011379:Prognosis; D050765:Qa-SNARE Proteins / Q000235:genetics; D012189:Retrospective Studies; D018570:Risk Assessment; D015996:Survival Rate; D013997:Time Factors; D016896:Treatment Outcome; D014421:Turkey
chemical_list: D008565:Membrane Proteins; D051938:Munc18 Proteins; C510468:PRF1 protein, human; D050765:Qa-SNARE Proteins; C541732:STX11 protein, human; C497940:STXBP2 protein, human; C507969:UNC13D protein, human; D054353:Perforin