Sirolimus for the treatment of complicated vascular anomalies in children

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‐threaten...

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Published in:Pediatric blood & cancer 2011-12, Vol.57 (6), p.1018-1024
Main Authors: Hammill, Adrienne M., Wentzel, MarySue, Gupta, Anita, Nelson, Stephen, Lucky, Anne, Elluru, Ravi, Dasgupta, Roshni, Azizkhan, Richard G., Adams, Denise M.
Format: Article
Language:English
Subjects:
Adolescent
Child
Child, Preschool
Compassionate Use Trials
Female
Humans
Immunosuppressive Agents - adverse effects
Immunosuppressive Agents - therapeutic use
Infant
kaposiform hemangioendothelioma
Kasabach-Merritt phenomenon
lymphatic malformation
Male
rapamycin
Retrospective Studies
sirolimus
Sirolimus - adverse effects
Sirolimus - therapeutic use
Treatment Outcome
vascular anomalies
vascular malformations
Vascular Malformations - complications
Vascular Malformations - drug therapy
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Summary: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. © 2011 Wiley‐Liss, Inc.
ISSN:1545-5009
1545-5017
DOI:10.1002/pbc.23124