treat LAM disease with sirolimus
- related: lymphangioleiomyomatosis LAM
- tags: #literature #pulmonary
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Management of LAM includes supportive care, supplemental oxygen, bronchodilators, pulmonary rehabilitation, and sirolimus. Use of sirolimus has shown beneficial effects for the treatment of both LAM pulmonary disease and associated complications, including a reduction in renal AML size; stabilization to mild improvement of lung function and quality of life; and reduction in the volume, presence, and recurrence rates of chylous effusions in patients with LAM. Sirolimus should be started in patients with symptoms and/or abnormal lung function (FEV1 <70% predicted). Other indications for starting sirolimus include evidence of rapidly progressive disease, chylous effusions, or AMLs. Everolimus is an alternative in those who do not tolerate sirolimus. In addition, avoidance of pregnancy and estrogen-containing medications is an important part of LAM management. Birth control medications should be progesterone based (choice C is incorrect).
In the last decade, sirolimus (rapamycin), an mTOR inhibitor acting via suppression of mTOR signaling, has shown beneficial effects for the treatment of LAM and associated complications including a reduction in renal angiomyolipoma volume; stabilization to mild improvement of lung function; improvement in some measures of performance and quality of life; and reduction in the volume, presence, and recurrence rates of chylous effusions in patients with LAM. Sirolimus is the initial treatment for symptomatic chylothorax in LAM. Lower doses may be effective although the full effects of therapy may take 1 to 12 months.
The prognosis with LAM is variable, with median survival formerly reported to be 8 to 10 years, but now improved. In the National Heart, Lung, and Blood Institute longitudinal LAM registry, the estimated 5-, 10-, 15-, and 20-year transplant-free survival rates were 94%, 85%, 75%, and 64%, respectively. The cause of death is usually respiratory failure. Patients with LAM may eventually require lung transplantation because of progressive respiratory failure. The outcomes after lung transplantation in LAM appear to be comparable with or better than in patients receiving transplants for other lung diseases, although disease recurrence in the transplanted lungs with LAM is described.1