title: Lack of bone lesions at diagnosis is associated with inferior outcome in multisystem langerhans cell histiocytosis of childhood.
abstract: Skeletal involvement is generally, but not universally, characteristic of Langerhans cell histiocytosis (LCH). We investigated whether the presence of bone lesions at diagnosis is a prognostic factor for survival in LCH. Nine hundred and thirty-eight children with multisystem (MS) LCH, both high (386 RO+) and low (RO-) risk, were evaluated for bone lesions at diagnosis. Risk organ (RO+) involvement was defined as: haematopoietic system (haemoglobin <100 g/l, and/or white blood cell count <4·0 × 10(9) /l and/or platelet count <100 × 10(9) /l), spleen (>2 cm below the costal margin), liver (>3 cm and/or hypoproteinaemia, hypoalbuminaemia, hyperbilirubinaemia, and/or increased aspartate transaminase/alanine transaminase). Given the general view that prognosis in LCH worsens with increasing extent of disease, the surprising finding was that in MS+RO+ LCH the probability of survival with bone involvement 74 ± 3% (n = 230, 56 events) was reduced to 62 ± 4% (n = 156, 55 events) if this was absent (P = 0·007). An even greater difference was seen in the subgroup of patients with both liver and either haematopoiesis or spleen involvement: 61 ± 5% survival (n = 105; 52 events) if patients had bony lesions, versus 47 ± 5% (n = 111; 39 events) if they did not (P = 0·014). This difference was retained in multivariate analysis (P = 0·048). Although as yet unexplained, we conclude that bone involvement at diagnosis is a previously unrecognized favourable prognostic factor in MS+RO+ LCH.
authors.affiliation: Azienda Sanitaria Provinciale 7, Ragusa, Italy.
authors.forename: Maurizio
authors.initials: M
authors.lastname: Aricò
authors.forename: Itziar
authors.initials: I
authors.lastname: Astigarraga
authors.forename: Jorge
authors.initials: J
authors.lastname: Braier
authors.forename: Jean
authors.initials: J
authors.lastname: Donadieu
authors.forename: Helmut
authors.initials: H
authors.lastname: Gadner
authors.forename: Evgenia
authors.initials: E
authors.lastname: Glogova
authors.forename: Nicole
authors.initials: N
authors.lastname: Grois
authors.forename: Jan-Inge
authors.initials: JI
authors.lastname: Henter
authors.forename: Gritta
authors.initials: G
authors.lastname: Janka
authors.forename: Kenneth L
authors.initials: KL
authors.lastname: McClain
authors.forename: Stephan
authors.initials: S
authors.lastname: Ladisch
authors.forename: Ulrike
authors.initials: U
authors.lastname: Pötschger
authors.forename: Diego
authors.initials: D
authors.lastname: Rosso
authors.forename: Elfriede
authors.initials: E
authors.lastname: Thiem
authors.forename: Sheila
authors.initials: S
authors.lastname: Weitzman
authors.forename: Kevin
authors.initials: K
authors.lastname: Windebank
authors.forename: Milen
authors.initials: M
authors.lastname: Minkov
mesh_terms: D001842:Bone and Bones / Q000473:pathology*; D002648:Child; D002675:Child, Preschool; D006646:Histiocytosis, Langerhans-Cell / Q000401:mortality* / Q000473:pathology*; D006801:Humans; D007223:Infant; D011379:Prognosis; D016016:Proportional Hazards Models
keywords: bone lesion; langerhans cell histiocytosis; multisystem disease; risk organ