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Sirolimus Can Increase the Disappearance Rate of Cardiac Rhabdomyomas Associated with Tuberous Sclerosis: A Prospective Cohort and Self-Controlled Case Series Study.
Chen, Xiao-Qiao; Wang, Yang-Yang; Zhang, Meng-Na; Lu, Qian; Pang, Ling-Yu; Liu, Li-Ying; Li, Yu-Fen; Zou, Li-Ping.
Afiliación
  • Chen XQ; Department of Pediatrics, First Medical Center, Chinese PLA General Hospital, Beijing, China; Department of Pediatrics, The 904th Hospital of Joint Logistic Support Force, Wuxi, China.
  • Wang YY; Department of Pediatrics, First Medical Center, Chinese PLA General Hospital, Beijing, China.
  • Zhang MN; Department of Pediatrics, First Medical Center, Chinese PLA General Hospital, Beijing, China.
  • Lu Q; Department of Pediatrics, First Medical Center, Chinese PLA General Hospital, Beijing, China.
  • Pang LY; Department of Pediatrics, First Medical Center, Chinese PLA General Hospital, Beijing, China.
  • Liu LY; Department of Pediatrics, First Medical Center, Chinese PLA General Hospital, Beijing, China; Department of Neurology, Beijing Jingdu Children's Hospital, Beijing, China.
  • Li YF; Department of Pediatrics, Shandong Linyi People's Hospital, Linyi, China.
  • Zou LP; Department of Pediatrics, First Medical Center, Chinese PLA General Hospital, Beijing, China; Center of Epilepsy, Beijing Institute for Brain Disorders, Beijing, China. Electronic address: zouliping21@hotmail.com.
J Pediatr ; 233: 150-155.e4, 2021 06.
Article en En | MEDLINE | ID: mdl-33631166
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.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Rabdomioma / Esclerosis Tuberosa / Sirolimus / Neoplasias Cardíacas / Antibióticos Antineoplásicos Tipo de estudio: Clinical_trials / Etiology_studies / Incidence_studies / Observational_studies / Risk_factors_studies Límite: Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: J Pediatr Año: 2021 Tipo del documento: Article País de afiliación: China Pais de publicación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Rabdomioma / Esclerosis Tuberosa / Sirolimus / Neoplasias Cardíacas / Antibióticos Antineoplásicos Tipo de estudio: Clinical_trials / Etiology_studies / Incidence_studies / Observational_studies / Risk_factors_studies Límite: Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: J Pediatr Año: 2021 Tipo del documento: Article País de afiliación: China Pais de publicación: Estados Unidos