Improvements of symptoms after sirolimus treatment in children with complex vascular malformations

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Abstract

Background and objectives
Management of complex vascular malformations is really challenging. They usually present at birth and have different complications, including bleeding, pain, cosmetic disfigurement, and functional impairment. This study was to done to determine symptomatic relief and quality-of-life improvement after sirolimus.
Patients and methods
The intervention phase included sirolimus administered orally on a continuous dosing schedule at a starting dose of 0.8 mg/m, and its level was maintained between 4 and 12 ng/ml. Dose was rounded to the nearest 1-mg tablet form. Assessment of response to sirolimus was done (usually 2 weeks after start of sirolimus) clinically by assessment of clinical improvement of the main complaints of the patients, for example, bleeding, pain, and cosmetic disfigurement. Improvement of patients’ laboratory investigations was assessed after the start of sirolimus, for example, elevation of hemoglobin and mean cell volume. The administration of Arabic translation of pediatric quality-of-life PedsQL 4.0 was done again 6 months after the start of sirolimus.
Results
A total of 18 patients were enrolled. The age of the patients during the start of sirolimus ranged from 5 months to 13 years, with a median age of 3 years. Bleeding stopped in eight (100%) patients, cosmetic disfigurement improved in 10 (76.9%) patients, and perception of pain improved in two (66.6%) patients. Moreover, quality of life of the study patients improved significantly, as the mean of the score of the patients before sirolimus was 65.18 and improved after sirolimus to be 78.
Conclusions
Symptomatic relief in patients after sirolimus administration is evident, with better quality of life.
The Egyptian Journal of Surgery
Volume 40, Issue 4
October 2021
Pages 1442-1448

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