Quality of life in children with complex vascular anomalies treated with sirolimus: A quasi-experimental study

Introduction. Complex vascular anomalies have a severe impact on quality of life. Sirolimus is used offlabel, but local evidence regarding its impact on health-related quality of life (HRQoL) is limited. Objective. To evaluate the effect of sirolimus treatment in patients with complex vascular anomalies. Population and methods. A single-group, quasi-experimental before-and-after study without a control group, conducted at a pediatric hospital. Patients aged 4 to 21 years with complex vascular malformations were included. HRQoL was measured using the PedsQL 4.0 questionnaire before starting sirolimus and at 6 months. Clinical characteristics, pain intensity and duration, and adverse events were recorded, and multivariate analyses were performed. Results. A total of 39 patients were included (56.4% were female). At baseline, low HRQoL scores were observed. After 6 months of treatment, total scores increased from 64.6 (29.34-94.5) to 79.9 (55.43-98.91) in self-reports and from 65.2 (27.17-90.62) to 77.2 (46.73-93.47) in caregiver reports (p <0.005). Chronic pain, present in 21 patients (53.8%), was the main factor associated with a worse baseline HRQoL (-14.4 points; 95% CI -24.4 to -4.3; p = 0.0065) and decreased in 95% of patients. Adverse events, observed in 41%, were mild and did not require treatment discontinuation. Conclusion. Treatment with sirolimus was associated with an improvement in HRQoL and pain in pediatric patients with complex vascular anomalies.