Abstract
BackgroundLung transplantation has been established as the definitive treatment option for patients with advanced lymphangioleiomyomatosis (LAM). However, the prognosis after registration and the circumstances of lung transplantation with sirolimus therapy have never been reported.MethodsIn this national survey, we analyzed data from 98 LAM patients registered for lung transplantation in the Japan Organ Transplantation Network.ResultsTransplantation was performed in 57 patients as of March 2014. Survival rate was 86.7% at 1 year, 82.5% at 3 years, 73.7% at 5 years, and 73.7% at 10 years. Of the 98 patients, 21 had an inactive status and received sirolimus more frequently than those with an active history (67% vs. 5%, p<0.001). Nine of twelve patients who remained inactive as of March 2014 initiated sirolimus before or while on a waiting list, and remained on sirolimus thereafter. Although the statistical analysis showed no statistically significant difference, the survival rate after registration tended to be better for lung transplant recipients than for those who awaited transplantation (p = 0.053).ConclusionsLung transplantation is a satisfactory therapeutic option for advanced LAM, but the circumstances for pre-transplantation LAM patients are likely to alter with the use of sirolimus.
Highlights
Lymphangioleiomyomatosis (LAM) is a rare neoplastic disease, characterized by the proliferation of abnormal smooth muscle-like cells (LAM cells), which lead to cystic destruction of the lungs, chylous effusions and the formation of lymphangioleiomyomas [1]
Of the 98 patients, 21 had an inactive status and received sirolimus more frequently than those with an active history (67% vs. 5%, p
Lung transplantation is a satisfactory therapeutic option for advanced LAM, but the circumstances for pre-transplantation LAM patients are likely to alter with the use of sirolimus
Summary
Lymphangioleiomyomatosis (LAM) is a rare neoplastic disease, characterized by the proliferation of abnormal smooth muscle-like cells (LAM cells), which lead to cystic destruction of the lungs, chylous effusions and the formation of lymphangioleiomyomas [1]. This disease is found primarily in women of childbearing age and can occur either as a sporadic disease (sporadic LAM) or as a pulmonary manifestation of tuberous sclerosis complex (TSC) (TSC-associated LAM) [2,3,4]. Lung transplantation has been established as the definitive treatment option for patients with advanced lymphangioleiomyomatosis (LAM). The prognosis after registration and the circumstances of lung transplantation with sirolimus therapy have never been reported
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