Late Breaking Abstract - Nintedanib for Lymphangioleiomyomatosis: an open-label phase II study

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Introduction: Lymphangioleiomyomatosis (LAM) is an ultra-rare disease, affecting women in childbearing age. In the MILES trial, sirolimus, a mammalian target of rapamycin inhibitor, has been proven to be an effective treatment. Drug toxicity and development of resistance are potential limitations related to therapy with sirolimus. Nintedanib is a multi- kinase inhibitor, including PDGF receptor, present and active in human and murine LAM lesions. We investigated the efficacy and safety of nintedanib in LAM patients. Methods: LAM patients with progressive pulmonary function decline over the last year, despite treatment with sirolimus or side effects to sirolimus were enrolled and treated open label with nintedanib for 12 months, followed by a period of additional 12 months of follow-up, off treatment. The primary endpoint was improvement of Forced Expiratory Volume in the first second (FEV1) over the treatment period. Results: 30 LAM patients (age 50±11 years, 2 TSC) entered the study: 8 withdrew (4 for progression and 4 for minor side effects), 22 completed the first year of study and 19 the second observational year. FEV1 (∆FEV1 0.001 L, p=0.07), Forced Volume Capacity (FVC) (∆FVC 0.03 L, p=0.49), Diffusion Lung Carbon monoxide (DLCO) (∆DLCO 0.03 mL/min/mmHg, p=0.92) and 6-minute walking distance (6MWD) (∆6MWD 15.7 m, p=0.394) were all stable after one year of treatment. During the off-treatment year, a slight statistically significant decline in FEV1 was observed (∆FEV1 -0.08 L, p=0.04), while the other parameters remained stable. No severe adverse events were observed. Conclusions: Nintedanib in LAM patients stabilizes progression of the disease and was generally well tolerated. IIS supported by Boehringer Ingelheim

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