Endocrine involvement in pediatric-onset Langerhans' cell histiocytosis: a population-based study.
OBJECTIVE
To document the frequency and outcome of endocrine involvement in pediatric-onset Langerhans' cell histiocytosis (LCH), and the association with other types of organ involvement.


METHODS
This retrospective nationwide multicenter study involved 589 patients with pediatric-onset LCH, 148 of whom had endocrine dysfunction. Median follow-up was 11.6 years.


RESULTS
Pituitary dysfunction was present in 145 patients, and 141 had diabetes insipidus (DI). The estimated 10-year risks of pituitary involvement were 24.2% +/- 1.8%. GH deficiency occurred in 61 patients. Median age at onset was 2.8 years for LCH, 3.9 years for DI, and 7.7 years for GH deficiency. The risk of cranial involvement; ear, nose, and throat involvement; pneumothorax; and cholangitis was significantly higher in patients with endocrinopathy. The chronology of episodes did not support a causal link between pituitary involvement and involvement of other organs. Systemic treatment of LCH did not prevent pituitary involvement. The most severe complication was a neurodegenerative syndrome, which affected 4.3% and 10.8% of patients, respectively, 5 and 15 years after initial diagnosis, and appeared to be linked to pituitary involvement.


CONCLUSIONS
Patients who develop endocrine LCH disorders are at a high risk of neurodegenerative LCH and require long-term follow-up.
Service d'Hémato-Oncologie Pédiatrique, Hôpital Trousseau and Orphanet, Hôpital Broussais, Paris, France. michel.polak@nck.ap-hop-paris.fr
Jean
J
Donadieu
Maria-Alejandra
MA
Rolon
Caroline
C
Thomas
Laurence
L
Brugieres
Dominique
D
Plantaz
Jean François
JF
Emile
Didier
D
Frappaz
Michel
M
David
Raja
R
Brauner
Thierry
T
Genereau
Dominique
D
Debray
Sylvie
S
Cabrol
Marie-Anne
MA
Barthez
Khé
K
Hoang-Xuan
Michel
M
Polak
D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D004703:Endocrine System / Q000503:physiopathology*; D004700:Endocrine System Diseases / Q000503:physiopathology*; D006646:Histiocytosis, Langerhans-Cell / Q000503:physiopathology*; D006801:Humans; D007223:Infant; D015999:Multivariate Analysis; D012189:Retrospective Studies