Stopping sirolimus in cardiac rhabdomyoma: is 75% regression the right finish line?

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Cardiac rhabdomyoma regrowth occurs in approximately one-third of cases following mTOR inhibitor withdrawal across both prenatal and postnatal settings, indicating that discontinuation criteria should extend beyond a simple 75% regression threshold. Novelty: ClaimNovelty.CONFIRMATORY. Consensus alignment: ConsensusAlignment.NEUTRAL.

Meera Jindal; Murtaza Kamal

doi:10.1017/s1047951126125074

November 6, 2026Cardiology in the Young

Stopping sirolimus in cardiac rhabdomyoma: is 75% regression the right finish line?

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Key Points

Evaluates whether discontinuing sirolimus therapy after achieving a 75% tumour regression threshold is optimal for infants with cardiac rhabdomyoma.
Letter to the editor analyzing clinical data from a published series of 11 infants receiving sirolimus for cardiac rhabdomyoma.
Evaluated tumour regression thresholds (>75% reduction), recurrence post-discontinuation, and the potential impact of TSC1/TSC2 genotype.
Stopping sirolimus after tumour size fell by >75% resulted in a 27% recurrence rate, with all 3 children responding again upon reinstitution.
Adverse effects in the evaluated series were limited to mild hypertriglyceridaemia in 2 infants, contrasting with reported rates of up to 60% in broader neonatal literature.

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Implication

Highlights tumour regrowth following sirolimus cessation in infants with cardiac rhabdomyoma, suggesting genetic and biological factors should guide treatment duration.

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Jindal et al. (2026) studied this question.

synapsesocial.com/papers/6aef1ace30498f842f31bf8c https://doi.org/10.1017/s1047951126125074

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Stopping sirolimus in cardiac rhabdomyoma: is 75% regression the right finish line?

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Cite This Study

Stopping sirolimus in cardiac rhabdomyoma: is 75% regression the right finish line?

Key Points

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Implication

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