Long-term follow-up of children with risk organ-negative Langerhans cell histiocytosis after 2-chlorodeoxyadenosine treatment.
2-chlorodeoxyadenosine
Langerhans cell histiocytosis
children
cladribine
long-term follow-up
Journal
British journal of haematology
ISSN: 1365-2141
Titre abrégé: Br J Haematol
Pays: England
ID NLM: 0372544
Informations de publication
Date de publication:
12 2020
12 2020
Historique:
received:
10
03
2020
accepted:
13
06
2020
pubmed:
24
7
2020
medline:
24
3
2021
entrez:
24
7
2020
Statut:
ppublish
Résumé
The nucleoside analogue, 2-chlorodeoxyadenosine (2CDA), was reported to be an active treatment for childhood Langerhans cell histiocytosis (LCH) without risk organ (RO-) involvement. However, we lack data on long-term effects of 2CDA treatment, including the disease reactivation rate, permanent sequelae and long-term tolerance. This study included 44 children from the French LCH registry, treated for a RO- LCH with 2CDA monotherapy (median number of six courses). The median age at the beginning of 2CDA was 3·6 years (range, 0·3-19·7 years) and the median follow-up after was 5·4 years (range, 0·6-15·1 years). Objective response to 2CDA was observed in 25 patients (56·8%), while six patients (13·6%) had stable disease and 13 patients (29·5%) exhibited progressive disease. Among patients without progression, only two experienced disease reactivation after 2CDA discontinuation. The five-year cumulative incidence of disease progression or reactivation after 2CDA therapy initiation was 34·3%. The lymphopenia reported in all cases [72% below absolute lymphocyte count (ALC) of 0·5 G/l], was addressed with appropriate prophylactic measures. Other toxicities above grade 2 were uncommon, and no second malignant neoplasm or neuropathy was reported. The five-year overall survival was 97·7%. In conclusion, we could confirm that 2CDA monotherapy was a beneficial long-term therapy for treating patients with RO- LCH. Appropriate management of induced immune deficiency is mandatory.
Substances chimiques
Cladribine
47M74X9YT5
Types de publication
Clinical Trial
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
825-834Subventions
Organisme : Société Française de lutte contre les Cancers de l'Enfant et de l'Adolescent
Organisme : Fédération Enfants et Santé; the Association Recherche et Maladie Hématologiques de l'Enfant
Organisme : Association Les 111 des Arts de Paris
Organisme : Association la Petite Maison dans la Prairie
Organisme : Association Histiocytose France
Organisme : InVS
Organisme : INSERM
Organisme : Roche
Organisme : Gardrat family
Organisme : Centre de Reference des Histiocytoses
Informations de copyright
© 2020 British Society for Haematology and John Wiley & Sons Ltd.
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