Sirolimus Treatment for Kidney Angiomyolipomas in Tuberous Sclerosis
Author Information
Author(s): Dabora Sandra L., Franz David Neal, Ashwal Stephen, Sagalowsky Arthur, DiMario Francis J. Jr, Miles Daniel, Cutler Drew, Krueger Darcy, Uppot Raul N., Rabenou Rahmin, Camposano Susana, Paolini Jan, Fennessy Fiona, Lee Nancy, Woodrum Chelsey, Manola Judith, Garber Judy, Thiele Elizabeth A.
Primary Institution: Brigham and Women's Hospital, Boston, Massachusetts, United States of America
Hypothesis
The study aims to evaluate the efficacy and tolerability of sirolimus for treating kidney angiomyolipomas in patients with tuberous sclerosis.
Conclusion
Sirolimus treatment for 52 weeks led to regression of kidney angiomyolipomas and may be a useful biomarker for monitoring tumor size.
Supporting Evidence
- 44.4% of participants showed a partial response to sirolimus treatment.
- The mean decrease in kidney tumor size was 29.9% after 52 weeks.
- Serum VEGF-D levels decreased significantly with treatment.
- Regression of brain tumors was observed in 7 out of 11 cases.
- Stable lung function was maintained in women with TSC/LAM.
- Drug-related toxicities were mostly mild to moderate.
- Responses tended to persist if treatment was continued after week 52.
- Future studies are needed to evaluate long-term benefits and risks.
Takeaway
This study tested a medicine called sirolimus to see if it could help shrink kidney tumors in people with a condition called tuberous sclerosis, and it worked for many of them.
Methodology
A phase 2 multicenter trial where participants received daily sirolimus and were monitored for tumor response and side effects over 52 weeks.
Potential Biases
Potential biases due to the single-arm design and lack of a control group.
Limitations
The study was open-label and single-arm, with a follow-up duration of only 2 years and no pediatric subjects included.
Participant Demographics
36 adults, average age 34 years, with 72% female and 28% male participants.
Statistical Information
P-Value
p=0.001
Confidence Interval
95% CI, 28 to 61
Statistical Significance
p<0.05
Digital Object Identifier (DOI)
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