Dramatic Response to Sirolimus in Lymphangioleiomyomatosis

Depatrment of Respirology, University of Toronto, Toronto General Hospital, Toronto, Ontario Correspondence: Dr Dmitry Rozenberg, Depatrment of Respirology, Toronto General Hospital, 9N 965 – 585 University Avenue, Toronto, Ontario M5G 2N2. Telephone 416-340-4591, fax 416-340-3109, e-mail dmitry.rozenberg@uhn.ca A 41-year-old woman presented with a one-year history of progressive dyspnea, early satiety and abdominal cramping. A clinical diagnosis of lymphangioleiomyomatosis (LAM) was made based on a confirmed right-sided chylous effusion (milky, exudative and triglyceride level of 52.6 mmol/L) and characteristic cystic changes on a highresolution computed tomography scan of the chest (Figure 1). A large left-sided retroperitoneal lymphangioleiomyoma supported the diagnosis (Figure 2), with no renal angiomyolipoma or clinical evidence of tuberous sclerosis. She was started on sirolimus therapy, which resolved her dyspnea and abdominal symptoms within six months. Her forced expiratory volume in 1 s (FEV1) (1.6 L) and FVC (2.1 L) had markedly improved during this time interval to 2.6 L and 4.2 L, respectively. Her chest x-ray demonstrated almost complete resolution of the right pleural effusion (Figure 3) and the lymphangioleiomyoma had also decreased in size (Figure 4). The patient remains clinically stable on sirolimus with few side effects and her FEV1 and FVC on therapy at nine months increased to 3.3 L (96%) and 4.8 L (118%), respectively. The present case is a dramatic example of a chylous pleural effusion and massive intra-abdominal lymphangioleiomyoma responding to sirolimus therapy. images in respiratory meDicine