Retrospective French nationwide survey of childhood aggressive vascular anomalies of bone, 1988-2009.
OBJECTIVE
To document the epidemiological, clinical, histological and radiological characteristics of aggressive vascular abnormalities of bone in children.


METHODS
Correspondents of the French Society of Childhood Malignancies were asked to notify all cases of aggressive vascular abnormalities of bone diagnosed between January 1988 and September 2009.


RESULTS
21 cases were identified; 62% of the patients were boys. No familial cases were observed, and the disease appeared to be sporadic. Mean age at diagnosis was 8.0 years [0.8-16.9 years]. Median follow-up was 3 years [0.3-17 years]. The main presenting signs were bone fracture (n = 4) and respiratory distress (n = 7), but more indolent onset was observed in 8 cases. Lung involvement, with lymphangiectasies and pleural effusion, was the most frequent form of extraosseous involvement (10/21). Bisphosphonates, alpha interferon and radiotherapy were used as potentially curative treatments. High-dose radiotherapy appeared to be effective on pleural effusion but caused major late sequelae, whereas antiangiogenic drugs like alpha interferon and zoledrenate have had a limited impact on the course of pulmonary complications. The impact of bisphosphonates and alpha interferon on bone lesions was also difficult to assess, owing to insufficient follow-up in most cases, but it was occasionally positive. Six deaths were observed and the overall 10-year mortality rate was about 30%. The prognosis depended mainly on pulmonary and spinal complications.


CONCLUSIONS
Aggressive vascular abnormalities of bone are extremely rare in childhood but are lifethreatening. The impact of anti-angiogenic drugs on pulmonary complications seems to be limited, but they may improve bone lesions.
Héritier
Sébastien
S
Service d'hématologie oncologie pédiatrique, Centre de référence des histiocytoses, AP-HP Hôpital Armand Trousseau, Paris, France. heritier.sebastien@yahoo.fr
Le Merrer
Martine
M
Jaubert
Francis
F
Bigorre
Michèle
M
Gillibert-Yvert
Marion
M
de Courtivron
Benoit
B
Ziade
Makram
M
Bertrand
Yves
Y
Carrie
Christian
C
Chastagner
Pascal
P
Bost-Bru
Cécile
C
Léonard
Jean-Claude
JC
Ouache
Marie
M
Boccon-Gibod
Liliane
L
Mary
Pierre
P
de Blic
Jacques
J
Pin
Isabelle
I
Wendling
Daniel
D
Revillon
Yann
Y
Houdoin
Véronique
V
Forin
Véronique
V
Lepointe
Hubert Ducou
HD
Languepin
Jane
J
Wagnon
Jeanne
J
Epaud
Ralph
R
Fauroux
Brigitte
B
Donadieu
Jean
J
D001847:Bone Diseases / Q000175:diagnosis / Q000188:drug therapy / Q000453:epidemiology*; D002648:Child; D004164:Diphosphonates / Q000627:therapeutic use; D005260:Female; D005602:France / Q000453:epidemiology; D006801:Humans; D016898:Interferon-alpha / Q000627:therapeutic use; D053208:Kaplan-Meier Estimate; D008297:Male; D012189:Retrospective Studies; D054079:Vascular Malformations / Q000175:diagnosis / Q000188:drug therapy / Q000453:epidemiology*
D004164:Diphosphonates; D016898:Interferon-alpha