Retrospective French nationwide survey of childhood aggressive vascular anomalies of bone, 1988-2009

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• 作者：Sébastien Héritier (1)
  Martine Le Merrer (2)
  Francis Jaubert (3)
  Michèle Bigorre (4)
  Marion Gillibert-Yvert (5)
  Benoit de Courtivron (5)
  Makram Ziade (4)
  Yves Bertrand (6)
  Christian Carrie (7)
  Pascal Chastagner (8)
  Cécile Bost-Bru (9)
  Jean-Claude Léonard (10)
  Marie Ouache (11)
  Liliane Boccon-Gibod (12)
  Pierre Mary (13)
  Jacques de Blic (14)
  Isabelle Pin (9)
  Daniel Wendling (15)
  Yann Revillon (16)
  Véronique Houdoin (14)
  Véronique Forin (17)
  Hubert Ducou Lepointe (18)
  Jane Languepin (19)
  Jeanne Wagnon (20)
  Ralph Epaud (21)
  Brigitte Fauroux (21)
  Jean Donadieu (1)
  
• 刊名：Orphanet Journal of Rare Diseases
• 出版年：2010
• 出版时间：December 2010
• 年：2010
• 卷：5
• 期：1
• 全文大小：2688KB
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• 作者单位：Sébastien Héritier (1)
  Martine Le Merrer (2)
  Francis Jaubert (3)
  Michèle Bigorre (4)
  Marion Gillibert-Yvert (5)
  Benoit de Courtivron (5)
  Makram Ziade (4)
  Yves Bertrand (6)
  Christian Carrie (7)
  Pascal Chastagner (8)
  Cécile Bost-Bru (9)
  Jean-Claude Léonard (10)
  Marie Ouache (11)
  Liliane Boccon-Gibod (12)
  Pierre Mary (13)
  Jacques de Blic (14)
  Isabelle Pin (9)
  Daniel Wendling (15)
  Yann Revillon (16)
  Véronique Houdoin (14)
  Véronique Forin (17)
  Hubert Ducou Lepointe (18)
  Jane Languepin (19)
  Jeanne Wagnon (20)
  Ralph Epaud (21)
  Brigitte Fauroux (21)
  Jean Donadieu (1)
  
  1. Service d'hématologie oncologie pédiatrique, Centre de référence des histiocytoses, AP-HP H?pital Armand Trousseau, Paris, France
  2. Service de génétique, Centre de référence des maladies osseuses, AP-HP H?pital Necker, Paris, France
  3. Service d'anatomie et cytologie pathologique, AP-HP H?pital Necker, Paris, France
  4. Service de chirurgie plastique pédiatrique, H?pital Lapeyronie, Montpellier, France
  5. Service d'orthopédie, H?pital Clocheville, CHU de Tours, France
  6. Institut d'hématologie et d'oncologie Pédiatrique, Lyon, France
  7. Département de radiothérapie, Centre Léon Bérard, Lyon, France
  8. Service de Médecine Infantile, H?pitaux de Brabois, Vandoeuvre les Nancy, France
  9. Pédiatrie, P?le Couple enfant, CHU de Grenoble, France
  10. Institut Calot, Berck sur Mer, France
  11. Service d'hématologie, AP-HP H?pital Robert Debré, Paris, France
  12. Service d'anatomie et cytologie pathologique, AP-HP H?pital Armand Trousseau, Paris, France
  13. Service d'orthopédie, AP-HP H?pital Armand Trousseau, Paris, France
  14. Service de pneumologie, AP-HP H?pital Necker, Paris, France
  15. Service de rhumatologie, CHU de Besan?on, France
  16. Service de chirurgie viscérale, AP-HP H?pital Necker, Paris, France
  17. Service de rééducation, Centre de référence des maladies osseuses, AP-HP H?pital Armand Trousseau, Paris, France
  18. Service de radiologie, AP-HP H?pital Armand Trousseau, Paris, France
  19. Service de pédiatrie générale, CHU de Limoges, France
  20. Service de pédiatrie générale, CH de Morlaix, France
  21. Service de pneumologie, Centre de référence des maladies pulmonaires rares, AP-HP H?pital Armand Trousseau, Paris, France
  

文摘

Objective To document the epidemiological, clinical, histological and radiological characteristics of aggressive vascular abnormalities of bone in children. Study design 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. Conclusion 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.