Abstract
[Introduction]
Lymphangioleiomyomatosis (LAM) is a rare multisystem disease predominantly affecting premenopausal women. Previous reports showed that age, lung function, hormone therapy, pneumothorax, or pulmonary cystic changes was associated with prognosis. To clarify prognostic factors, we analyzed the clinical features and prognostic from our cohort.
[Subjects and Methods]
108 patients {Japanese female, sporadic LAM (n=93) and TSC-LAM (n=15), female} were consecutively registered in NHO Kinki-Chuo Chest Medical Center Cohort until 2013. The age (onset) was 36(30-45) {median(IQR)} years old. We retrospectively analyzed the clinical features and prognosis.
[Results and conclusion]
Angiomyolipomas and lymphangioleiomyomas were detected in 31 and 28 patients. 48 patients had pneumothoraces and 5 patients had chylous effusion. 27 patients were treated with Gn-RH analogues and 39 patients with sirolimus. Initial %FEV1.0 was 78(57-104) %, %DLco was 60(39-77) %. Serum VEGF-D level was 1786(781-3610) pg/ml. Eight patients died from respiratory failure (n=6), abdominal bleeding (n=1), and seizure (n=1), and 5 patients had lung transplantation. Estimated 10-year survival free from death and transplant from the registration (survival) was 73%. There was no significant difference of survival between sporadic LAM and TSC-LAM. Serum VEGF-D < 1000 pg/ml, %FEV1.0 ≥ 70%, and %DLco ≥ 60%, were associated with better prognosis (p<0.05, Log-rank). Menopause and hormone therapy were associated with poor survival.
The prognosis was relatively better than previous reports. Development of new drugs such as mTOR inhibitors are expected to make more improvement.
- © 2014 ERS
