Sirolimus Can Increase the Disappearance Rate of Cardiac Rhabdomyomas Associated with Tuberous Sclerosis: A Prospective Cohort and Self-Controlled Case Series Study.

Abstract

OBJECTIVES:

To investigate the efficacy and safety of sirolimus in the treatment of cardiac rhabdomyomas associated with tuberous sclerosis complex and the specific benefits in different subgroups.

STUDY DESIGN:

The study was a prospective cohort and self-controlled case series study. Based on the prevalence of cardiac rhabdomyoma at different ages, we estimated the natural tumor disappearance rate. The subgroup analysis was done by Cox regression. Self-controlled case series method was used to assess the magnitude and duration of the drug effect. Adverse events were described.

RESULTS:

A total of 217 patients were included in the cohort study. Tumor disappearance rate was higher in younger age groups (hazard ratio = 0.99, P = .027) and female patients (hazard ratio = 2.08, P = .015). The age-adjusted incidence ratio showed that the disappearance of rhabdomyomas between 3 and 6 months was more related to sirolimus. Adverse events were observed 60 times in 42 of 217 children, mainly stomatitis.

CONCLUSIONS:

Sirolimus can increase the disappearance rate of cardiac rhabdomyoma in the tuberous sclerosis complex population. Efficacy varies by sex and age: female and younger patients have higher tumor disappearance rate. Sirolimus is well-tolerated.

• Authors: Xiao-Qiao Chen, Yang-Yang Wang, Meng-Na Zhang, Qian Lu, Ling-Yu Pang, Li-Ying Liu, Yu-Fen Li, Li-Ping Zou
• Journal: The Journal of pediatrics (J Pediatr) Vol. 233 Pg. 150-155.e4 (06 2021) ISSN: 1097-6833 [Electronic] United States
• PMID: 33631166 (Publication Type: Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)
• Copyright: Copyright © 2021 Elsevier Inc. All rights reserved.

Chemical References

• Antibiotics, Antineoplastic
• Sirolimus

Topics

• Age Factors
• Antibiotics, Antineoplastic (therapeutic use)
• Child, Preschool
• Cohort Studies
• Female
• Heart Neoplasms (drug therapy, etiology)
• Humans
• Infant
• Male
• Rhabdomyoma (drug therapy, etiology)
• Sex Factors
• Sirolimus (therapeutic use)
• Tuberous Sclerosis (complications)