Premium
Langerhans cell histiocytosis: therapeutic strategy and outcome in a 30‐year nationwide cohort of 1478 patients under 18 years of age
Author(s) -
Rigaud Charlotte,
Barkaoui Mohamed A.,
Thomas Caroline,
Bertrand Yves,
Lambilliotte Anne,
Miron Jean,
Aladjidi Nathalie,
Plat Geneviève,
Jeziorski Eric,
Galambrun Claire,
Mansuy Ludovic,
Lutz Patrick,
Deville Anne,
ArmariAlla Corinne,
Reguerre Yves,
Fraitag Sylvie,
Coulomb Aurore,
Gandemer Virginie,
Leboulanger Nicolas,
Moshous Despina,
HoangXuan Khe,
Tazi Abdellatif,
Heritier Sébastien,
Emile JeanFrançois,
Donadieu Jean
Publication year - 2016
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.14140
Subject(s) - medicine , cladribine , cytarabine , cohort , langerhans cell histiocytosis , cohort study , pediatrics , histiocytosis , confidence interval , surgery , disease , chemotherapy
Summary The French national cohort of children with Langerhans cell histiocytosis ( LCH ) has included 1478 patients since it was established in 1983. LCH therapeutic strategies substantially changed in 1998, so we have divided the cohort into two 15‐year periods. Starting in 1998, therapy duration increased from 6 to 12 months, repeated induction therapy was performed in cases showing a poor response to the first induction with vinblastine and steroids, and refractory disease in a risk organ ( RO +) was treated with cladribine and cytarabine. A total of 483 (33%) patients were enrolled before 1998, and 995 (67%) after 1998. Five‐year survival was 96·6% (95% confidence interval: 95·4–97·5%) overall, improving from 92% pre‐1998 to 99% post‐1998 ( P < 0·001 adjusted to disease extent). This change was supported by an increase in 5‐year survival from 60% to 92% in the RO + group. Survival was particularly associated with cladribine and cytarabine among refractory RO + patients. Disease reactivation was slightly less frequent after 1998, due to better enrolment of single‐system patients, extended therapy duration, and more efficient second‐line therapy. The crude rates of endocrine and neurological sequelae (the most frequent sequelae) appeared to improve over time, but this difference was not observed when the analysis was stratified by disease extent.