Long-term follow-up of children with risk organ-negative Langerhans cell histiocytosis after 2-chlorodeoxyadenosine treatment.

Fiche publication

Date publication

juillet 2020

Journal

British journal of haematology

Auteurs

Membres identifiés du Cancéropôle Est :
Pr ENTZ-WERLE Natacha

Tous les auteurs :
Barkaoui MA, Queheille E, Aladjidi N, Plat G, Jeziorski E, Moshous D, Lambilliotte A, Kebaili K, Pacquement H, Leverger G, Mansuy L, Entz-Werlé N, Bodet D, Schneider P, Pagnier A, Lutun A, Gillibert-Yvert M, Millot F, Toutain F, Reguerre Y, Thomas C, Tazi A, Emile JF, Donadieu J, Héritier S

Lien Pubmed

https://www.ncbi.nlm.nih.gov/pubmed/32700439

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.