2021
DOI: 10.1016/j.jpeds.2021.02.040
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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

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 designThe 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 magnitu… Show more

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Cited by 13 publications
(10 citation statements)
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“…8 Sirolimus, a mammalian target of rapamycin inhibitor, has been reported to be effective in treating cardiac rhabdomyomas associated with tuberous sclerosis and is well tolerated. 10 The present patient started taking sirolimus for graft-vs-host disease 10 weeks before the biopsy. During the biopsy, there was improvement in the vocal fold lesions, and a limited biopsy was performed.…”
Section: Discussionmentioning
confidence: 87%
See 1 more Smart Citation
“…8 Sirolimus, a mammalian target of rapamycin inhibitor, has been reported to be effective in treating cardiac rhabdomyomas associated with tuberous sclerosis and is well tolerated. 10 The present patient started taking sirolimus for graft-vs-host disease 10 weeks before the biopsy. During the biopsy, there was improvement in the vocal fold lesions, and a limited biopsy was performed.…”
Section: Discussionmentioning
confidence: 87%
“…Extralaryngeal extension requiring open hemilaryngectomy has also been reported . Sirolimus, a mammalian target of rapamycin inhibitor, has been reported to be effective in treating cardiac rhabdomyomas associated with tuberous sclerosis and is well tolerated . The present patient started taking sirolimus for graft-vs-host disease 10 weeks before the biopsy.…”
Section: Discussionmentioning
confidence: 99%
“…After a careful full-text review, 12 studies were eligible for the final data extraction and analysis. Finally, nine studies [ 22 , 23 , 24 , 25 , 29 , 30 , 31 , 32 , 33 ] were included in the analyses. The remaining three studies were excluded, including a prospective cohort study and two randomized controlled trials.…”
Section: Resultsmentioning
confidence: 99%
“…The characteristics of the subsumed studies are summarized in Table 1 . In total, the 9 studies consisted of 575 patients, and there were 7 prospective studies [ 22 , 23 , 24 , 29 , 30 , 31 , 32 ] and 2 randomized controlled trials [ 25 , 33 ]. Of these studies, four were related to tuberous sclerosis complex [ 25 , 31 , 32 , 35 ], two were related to lymphatic anomalies [ 31 , 32 ], two were related to vascular anomalies [ 22 , 25 ] and one was related to nephrotic syndrome [ 24 ].…”
Section: Resultsmentioning
confidence: 99%
“…Mutations in TSC 1 or TSC 2 genes cause overactivation of the mammalian target of rapamycin (mTOR), resulting in abnormal cell proliferation and cardiac rhabdomyoma formation. mTOR inhibitors such as sirolimus can treat cardiac rhabdomyoma associated with TSC (11). LVNC is a genetically heterogeneous disease caused by the cessation of normal myocardial compaction in the embryonic period.…”
mentioning
confidence: 99%