Tuyển tập các báo cáo nghiên cứu về y học được đăng trên tạp chí y học 'Respiratory Research cung cấp cho các bạn kiến thức về ngành y đề tài:" Is sirolimus a therapeutic option for patients with progressive pulmonary lymphangioleiomyomatosis?. | Neurohr et al. Respiratory Research 2011 12 66 http content 12 1 66 RESPIRATORY RESEARCH RESEARCH Open Access Is sirolimus a therapeutic option for patients with progressive pulmonary lymphangioleiomyomatosis Claus NeurohO Anna L Hoffmann1 Patrick Huppmann Vivian A Herrera Franziska Ihle Stefan Leuschner Werner von Wulffen Tobias Meis Carlos Baezner Hanno Leuchte Rainer Baumgartner Gregor Zimmermann and Juergen Behr Abstract Background Lymphangioleiomyomatosis LAM is a rare lung disease characterised by progressive airflow obstruction. No effective medical treatment is available but therapy with sirolimus has shown some promise. The aim of this observational study was to evaluate sirolimus in progressive LAM. Methods Sirolimus trough level 5-10 ng ml was administered to ten female patients years with documented progression. Serial pulmonary function tests and six-minute-walk-distance 6-MWD assessments were performed. Results The mean loss of FEV1 was ml day before therapy and a significant mean gain of FEV1 of ml day was detected during treatment p . Mean FEV1 and FVC at baseline were l pred. and l pred. respectively. At three and six months during follow-up a significant increase of FEV1 and FVC was demonstrated 3 months AFEV1 220 82 ml p 6 months AFEV1 345 58 ml p 3 months AFVC 360 141 ml p 6 months AFVC 488 138 ml p . Sirolimus was discontinued in 3 patients because of serious recurrent lower respiratory tract infection or sirolimus-induced pneumonitis. No deaths and no pneumothoraces occurred during therapy. Conclusions Our data suggest that sirolimus might be considered as a therapeutic option in rapidly declining LAM patients. However sirolimus administration may be associated with severe respiratory adverse events requiring treatment cessation in some patients. Moreover discontinuation of sirolimus is mandatory prior to lung .
