TY - JOUR
T1 - Additive Prognostic Impact of Gastrointestinal Involvement in Severe Multisystem Langerhans Cell Histiocytosis
AU - Minkov, Milen
AU - Pötschger, Ulrike
AU - Thacker, Nirav
AU - Astigarraga, Itziar
AU - Braier, Jorge
AU - Donadieu, Jean
AU - Henter, Jan-Inge
AU - Lehrnbecher, Thomas
AU - Rodriguez-Galindo, Carlos
AU - Sieni, Elena
AU - Nanduri, Vasanta
AU - van den Bos, Cor
AU - Abla, Oussama
AU - LCH Study Group of the Histiocyte Society
AU - Aricò, M.
AU - Gadner, H.
AU - Grois, N.
AU - Janka-Schaub, G.
AU - Ladisch, S.
AU - McClain, K.
AU - Weitzman, S.
AU - Windebank, K.
N1 - Funding Information: Financial support was provided for the management of the original clinical trials of the Histiocyte Society by the Histiocytosis Association and the Children's Cancer Research Institute (Vienna, Austria), both nonprofit organizations. The authors declare no conflicts of interest. Publisher Copyright: © 2021 The Authors Copyright: Copyright 2021 Elsevier B.V., All rights reserved.
PY - 2021/10
Y1 - 2021/10
N2 - Objective: To evaluate the prognostic impact of gastrointestinal involvement on the survival of children with Langerhans cell histiocytosis (GI-LCH) registered with the international clinical trials of the Histiocyte Society. Study design: This was a retrospective analysis of 2414 pediatric patients registered onto the consecutive trials DAL-HX 83, DAL-HX 90, LCH-I, LCH-II, and LCH-III. Results: Among the 1289 patients with single-system LCH, there was no single case confined to the GI tract; 114 of 1125 (10%) patients with multisystem LCH (MS-LCH) had GI-LCH at initial presentation. GI-LCH was significantly more common in children aged <2 years at diagnosis (13% vs 6% in those aged >2 years; P < .001) and in those with risk organ involvement (15% vs 6% in those without risk organ involvement; P < .001). The 5-year overall survival (OS) in patients without risk organ involvement was excellent irrespective of GI disease (98% vs 97% in patients with GI-LCH; P = .789). In patients with risk organ involvement, the 5-year OS was 51% in 70 patients with GI-LCH vs 72% in 394 patients without GI-LCH (P < .001). Conclusions: GI-LCH has an additive unfavorable prognostic impact in children with MS-LCH and risk organ involvement. The emerding need for more intensive or alternative treatments mandates prospective evaluation.
AB - Objective: To evaluate the prognostic impact of gastrointestinal involvement on the survival of children with Langerhans cell histiocytosis (GI-LCH) registered with the international clinical trials of the Histiocyte Society. Study design: This was a retrospective analysis of 2414 pediatric patients registered onto the consecutive trials DAL-HX 83, DAL-HX 90, LCH-I, LCH-II, and LCH-III. Results: Among the 1289 patients with single-system LCH, there was no single case confined to the GI tract; 114 of 1125 (10%) patients with multisystem LCH (MS-LCH) had GI-LCH at initial presentation. GI-LCH was significantly more common in children aged <2 years at diagnosis (13% vs 6% in those aged >2 years; P < .001) and in those with risk organ involvement (15% vs 6% in those without risk organ involvement; P < .001). The 5-year overall survival (OS) in patients without risk organ involvement was excellent irrespective of GI disease (98% vs 97% in patients with GI-LCH; P = .789). In patients with risk organ involvement, the 5-year OS was 51% in 70 patients with GI-LCH vs 72% in 394 patients without GI-LCH (P < .001). Conclusions: GI-LCH has an additive unfavorable prognostic impact in children with MS-LCH and risk organ involvement. The emerding need for more intensive or alternative treatments mandates prospective evaluation.
KW - Langerhans cell histiocytosis
KW - children
KW - digestive tract
KW - gastrointestinal tract
KW - prognosis
UR - http://www.scopus.com/inward/record.url?scp=85109094974&partnerID=8YFLogxK
U2 - https://doi.org/10.1016/j.jpeds.2021.06.016
DO - https://doi.org/10.1016/j.jpeds.2021.06.016
M3 - Article
C2 - 34146548
SN - 0022-3476
VL - 237
SP - 65-70.e3
JO - Journal of pediatrics
JF - Journal of pediatrics
ER -