Langerhans cell histiocytosis: therapeutic strategy and outcome in a 30-year nationwide cohort of 1478 patients under 18 years of age.
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.
Registre français des histiocytoses, Service d'Hémato-oncologie Pédiatrique Hôpital Trousseau, AP-HP, Paris, France.|Service de Pédiatrie Institut Gustave Roussy, IGR, Villejuif.
Charlotte
C
Rigaud
Registre français des histiocytoses, Service d'Hémato-oncologie Pédiatrique Hôpital Trousseau, AP-HP, Paris, France.
Mohamed A
MA
Barkaoui
Service d'hémato-oncologie pédiatrique, Centre Hospitalo-Universitaire de Nantes, Nantes, France.
Caroline
C
Thomas
Institut d'Hématologie et d'Oncologie Pédiatrique, Lyon, France.
Yves
Y
Bertrand
Unité d'Hémato-oncologie Pédiatrique, Centre Hospitalo-Universitaire de Lille, Lille, France.
Anne
A
Lambilliotte
Registre français des histiocytoses, Service d'Hémato-oncologie Pédiatrique Hôpital Trousseau, AP-HP, Paris, France.
Jean
J
Miron
CIC 1401, Inserm CICP, Hopital Pellegrin-enfant, CEREVANCE, CHU de Bordeaux, Bordeaux, France.
Nathalie
N
Aladjidi
Service d'hémato-oncologie pédiatrique, Centre Hospitalo-Universitaire de Toulouse, Toulouse, France.
Geneviève
G
Plat
Service d'hémato et d'oncologie pédiatrique, Centre Hospitalo-Universitaire de Montpellier, Montpellier, France.
Eric
E
Jeziorski
Service d'Hématologie Oncologie pédiatrique, Centre Hospitalo-Universitaire de Marseille, Marseille, France.
Claire
C
Galambrun
Medecine Infantile 2, Centre Hospitalo-Universitaire de Nancy, Nancy, France.
Ludovic
L
Mansuy
Service d'hémato-oncologie pédiatrique, Centre Hospitalo-Universitaire de Strasbourg, Strasbourg, France.
Patrick
P
Lutz
Service d'hémato-oncologie pédiatrique, Centre Hospitalo-Universitaire de Nice, Nice, France.
Anne
A
Deville
Service d'hémato-oncologie pédiatrique, Centre Hospitalo-Universitaire de Grenoble, La Tronche, France.
Corinne
C
Armari-Alla
Service d'hémato-oncologie pédiatrique, Centre Hospitalo-Universitaire de la Réunion Hôpital Felix Guyon, Saint denis, La Réunion, France.
Yves
Y
Reguerre
Service d'Anatomopathologie, Hôpital Necker Enfants, AP-HP, Paris, France.
Sylvie
S
Fraitag
Service d'Anatomopathologie, Hopital Trousseau, Paris, France.
Aurore
A
Coulomb
Service d'Hématologie pédiatrique, Centre Hospitalo-Universitaire de Rennes, Rennes, France.
Virginie
V
Gandemer
Service d'Otorhinolaryngologie et Chirurgie Cervicofaciale, Hôpital Necker Enfants Malades, AP-HP, Paris, France.
Nicolas
N
Leboulanger
Unité d'Immuno-hématologie pédiatrique, Hôpital Necker Enfants Malades, AP-HP, Paris, France.
Despina
D
Moshous
Service de Neurologie, Groupe hospitalier Pitié Salpêtrière-Charles Foix, AP-HP, Sorbonne Universités - UPMC, Paris, France.
Khe
K
Hoang-Xuan
APHP Service de Pneumologie Centre de référence des histiocytoses Hôpital Saint Louis, Paris, France.
Abdellatif
A
Tazi
Registre français des histiocytoses, Service d'Hémato-oncologie Pédiatrique Hôpital Trousseau, AP-HP, Paris, France.
Sébastien
S
Heritier
Service de pathologie, Hôpital Ambroise Paré, UVSQ, Université Paris-Saclay, AP-HP, Boulogne, France.
Jean-François
JF
Emile
Registre français des histiocytoses, Service d'Hémato-oncologie Pédiatrique Hôpital Trousseau, AP-HP, Paris, France.
Jean
J
Donadieu
D000293:Adolescent; D000367:Age Factors; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D003131:Combined Modality Therapy; D005260:Female; D005500:Follow-Up Studies; D005602:France / Q000453:epidemiology; D006646:Histiocytosis, Langerhans-Cell / Q000175:diagnosis / Q000453:epidemiology* / Q000401:mortality / Q000628:therapy; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D011159:Population Surveillance; D059039:Standard of Care; D016019:Survival Analysis; D016896:Treatment Outcome
cohort study; historical comparisons; langerhans cell histiocytosis