Sirolimus for the Treatment of Complicated Vascular Anomalies in Children

被引：464

作者：

Hammill, Adrienne M. ^{[1
,2
]}

Wentzel, MarySue ^{[2
]}

Gupta, Anita ^{[2
,3
]}

Nelson, Stephen ^{[4
]}

Lucky, Anne ^{[2
,5
]}

Elluru, Ravi ^{[2
,6
]}

Dasgupta, Roshni ^{[2
,7
]}

Azizkhan, Richard G. ^{[2
,7
]}

Adams, Denise M. ^{[1
,2
]}

机构：

[1] Univ Cincinnati, Coll Med, Cincinnati Childrens Hosp, Div Hematol Oncol,Med Ctr, Cincinnati, OH 45229 USA

[2] Cincinnati Childrens Hosp, Med Ctr, Hemangioma & Vasc Malformat Ctr, Cincinnati, OH USA

[3] Univ Cincinnati, Coll Med, Cincinnati Childrens Hosp, Div Pathol,Med Ctr, Cincinnati, OH 45229 USA

[4] Childrens Hosp & Clin Minnesota, Vasc Anomalies Ctr, Pediat Hematol Oncol Program, Minneapolis, MN USA

[5] Univ Cincinnati, Coll Med, Cincinnati Childrens Hosp, Div Dermatol,Med Ctr, Cincinnati, OH 45229 USA

[6] Univ Cincinnati, Coll Med, Cincinnati Childrens Hosp, Div Pediat Otolaryngol,Med Ctr, Cincinnati, OH 45229 USA

[7] Univ Cincinnati, Coll Med, Cincinnati Childrens Hosp, Div Pediat Surg,Med Ctr, Cincinnati, OH 45229 USA

来源：

PEDIATRIC BLOOD & CANCER | 2011年 / 57卷 / 06期

关键词：

vascular anomalies; vascular malformations; kaposiform hemangioendothelioma; Kasabach-Merritt phenomenon; lymphatic malformation; rapamycin; sirolimus; KASABACH-MERRITT PHENOMENON; MALFORMATIONS IN-VIVO; KAPOSIFORM HEMANGIOENDOTHELIOMA; ENDOTHELIAL-CELLS; MAMMALIAN TARGET; CALCINEURIN INHIBITOR; INTERFERON-ALPHA; SPASTIC DIPLEGIA; RAPAMYCIN; MTOR;

D O I：

10.1002/pbc.23124

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 [肿瘤学];

摘要：

Background. Vascular anomalies comprise a diverse group of diagnoses. While infantile hemangiomas are common, the majority of these conditions are quite rare and have not been widely studied. Some of these lesions, though benign, can impair vital structures, be deforming, or even become life-threatening. Vascular tumors such as kaposiform hemangioendotheliomas (KHE) and complicated vascular malformations have proven particularly difficult to treat. Procedure. Here we retrospectively evaluate a series of six patients with complicated, life-threatening vascular anomalies who were treated with the mTOR inhibitor sirolimus for compassionate use at two centers after failing multiple other therapies. Results. These patients showed significant improvement in clinical status with tolerable side effects. Conclusions. Sirolimus appears to be effective and safe in patients with life-threatening vascular anomalies and represents an important tool in treating these diseases. These findings are currently being further evaluated in a Phase II safety and efficacy trial. Pediatr Blood Cancer 2011; 57: 1018-1024. (C) 2011 Wiley-Liss, Inc.

引用

页码：1018 / 1024

页数：7

共 37 条

[1]

The role of the hematologist/oncologist in the care of patients with vascular anomalies [J].