From the Research
The recommended dose of sirolimus for treating lymphangioleiomyomatosis (LAM) is 1-2 mg daily, with a target trough level of 5-9.99 ng/mL, as this range has been associated with improved lung function and reduced symptoms in patients with LAM 1. To initiate treatment:
- Start with 2 mg orally once daily
- Check sirolimus trough levels after 10-14 days
- Adjust dose to maintain trough levels between 5-9.99 ng/mL
- Monitor levels every 2-3 months once stable, or more frequently if dose changes or drug interactions occur Additional considerations:
- Take sirolimus consistently with or without food
- Avoid grapefruit juice, which can increase sirolimus levels
- Be aware of potential side effects, including mouth ulcers, diarrhea, and increased risk of infections
- Regular monitoring of lipid levels and renal function is recommended Sirolimus works in LAM by inhibiting the mTOR pathway, which is overactive in LAM cells, helping to slow the abnormal growth and proliferation of smooth muscle-like cells in the lungs, lymphatics, and kidneys, thereby stabilizing or improving lung function and reducing complications associated with LAM 1, 2. Key benefits of sirolimus therapy in LAM include:
- Improved lung function, as measured by forced expiratory volume in 1 second (FEV1) and forced vital capacity (FVC) 1, 2
- Reduced symptoms, such as dyspnea and chylothorax 1, 2
- Improved quality of life, as measured by the St. George's Respiratory Questionnaire (SGRQ) 1
- Reduced size of angiomyolipomas and chylous effusions 3, 4 It is essential to note that the optimal dose and trough level of sirolimus may vary depending on individual patient factors, such as renal function and concomitant medications 5, 2. Therefore, close monitoring and adjustment of the dose as needed are crucial to ensure the safe and effective use of sirolimus in patients with LAM.