title: Increased serum baseline tryptase levels and extensive skin involvement are predictors for the severity of mast cell activation episodes in children with mastocytosis.
abstract: BACKGROUND
  Despite the good prognosis of pediatric mastocytosis, some patients suffer from severe mast cell (MC) mediator-associated symptoms. The aim of this study was to identify predictors for severe MC mediator release symptoms in children with mastocytosis in the skin (MIS).
  
  
  METHODS
  Serum baseline total tryptase (sbT) levels in 111 children with MIS - 80 maculopapular cutaneous mastocytosis/plaque mastocytosis, 22 nodular mastocytosis, and nine diffuse cutaneous mastocytosis - were investigated as a predictive biomarker for the occurrence of MC mediator-related signs and symptoms within the first 18 months after disease onset.
  
  
  RESULTS
  Twelve children (11%) who showed extensive cutaneous disease involving >90% of body surface area (BSA) suffered from severe symptoms requiring hospitalization, with (n = 5) or without (n = 6) management in the intensive care unit (ICU) owing to life-threatening complications. The median sbT was significantly (P < 0.001) higher in patients with extensive cutaneous disease vs those with <90% of BSA involved (45.5 vs 5.2 μg/l, respectively), as well as in children with grade 4 (severe mastocytosis-related symptoms requiring emergency therapy and hospitalization) vs those with grade <4 (46.2 vs 5.2 μg/l, respectively). Receiver operating characteristics curve analyses showed that the optimal cutoff s for sbT to predict the need for daily antimediator therapy, hospitalization, and the management in an ICU were 6.6, 15.5, and 30.8 μg/l, respectively (sensitivity and specificity of 77% and 79%, 100% and 95%, and 100% and 96%, respectively).
  
  
  CONCLUSIONS
  Increased sbT in association with extensive cutaneous involvement identifies patients at risk for severe MC activation events in pediatric mastocytosis.
authors.affiliation: Instituto de Estudios de Mastocitosis de Castilla La Mancha, Hospital Virgen del Valle, Toledo, Spain.
authors.forename: I
authors.initials: I
authors.lastname: Alvarez-Twose
authors.forename: S
authors.initials: S
authors.lastname: Vañó-Galván
authors.forename: L
authors.initials: L
authors.lastname: Sánchez-Muñoz
authors.forename: J M
authors.initials: JM
authors.lastname: Morgado
authors.forename: A
authors.initials: A
authors.lastname: Matito
authors.forename: A
authors.initials: A
authors.lastname: Torrelo
authors.forename: P
authors.initials: P
authors.lastname: Jaén
authors.forename: L B
authors.initials: LB
authors.lastname: Schwartz
authors.forename: A
authors.initials: A
authors.lastname: Orfao
authors.forename: L
authors.initials: L
authors.lastname: Escribano
mesh_terms: D019540:Area Under Curve; D015415:Biomarkers / Q000097:blood; D015550:Cell Degranulation; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D008407:Mast Cells / Q000378:metabolism / Q000473:pathology*; D034701:Mastocytosis, Cutaneous / Q000097:blood / Q000201:enzymology* / Q000473:pathology*; D012372:ROC Curve; D012680:Sensitivity and Specificity; D053802:Tryptases / Q000097:blood*
chemical_list: D015415:Biomarkers; D053802:Tryptases