Clinical differences in sirolimus treatment with low target levels between children and adults with vascular malformations - A nationwide trial.

ABSTRACT: The clinical presentation of patients with slow-flow vascular malformations is very heterogeneous. High clinical burden and subsequent reduced health-related quality of life is something they have in common. There is an unmet medical need for these patients for whom regular treatments like surgery and embolization are either insufficient or technically impossible. Sirolimus has been reported to be effective and overall well-tolerated in most patients. However, the main limitation of sirolimus is the reported high toxicity, especially when target levels of 10-15 ng/mL are being used. We report the results of a phase IIB single-arm open-label clinical trial consisting of 68 (67 in the challenge phase and 68 in the rechallenge phase) evaluable patients (children n = 33 and adults n = 35) demo

SUBMITTER: Harbers VEM 

PROVIDER: S-EPMC10176016 | biostudies-literature | 2023 May

REPOSITORIES: biostudies-literature