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Facilitators: J.M. D'Armiento, MD, PhD, S.Y. El-Chemaly, MD, MPH, D.F. Dilling, MD, L.R. Young, MD
Session Info: Thematic Poster Session, [A35] I'VE GOT A FEELING WE'RE GOING TO SOLVE THIS ONE: INSIGHTS
INTO LYMPHANGIOLEIOMYOMATOSIS
Day/Date: Sunday, May 17, 2015
Session Time: 9:30 AM - 4:15 PM
Poster Viewing: 11:30-1:15
Room: Area G, Hall A-B (Upper Level)
Location: Colorado Convention Center
[Poster Board # P758] Eighteen Months Sirolimus Medication Benefits not Only Patients with Moderate to Severe LAM but also Those with Mild Disease, [Publication Number: A1393]
K. Nakata, MD, PhD1, Y. Inoue, MD, PhD2, K. Seyama, MD, PhD3, R. Tazawa, MD, PhD1, T. Tamada, MD, PhD4, M.
Suzuki, MD PhD5, M. Mishima, MD, PhD6, A. Mikami, MD7
1Niigata/JP, 2Osaka/JP, 38421/JP, 48574/JP, 58648/JP, 68567/JP, 71193/JP
MLSTS group in Japan
Multicenter international lymphangioleiomyomatosis efficacy of Sirolimus (MILES) trial conducted during 2006 to 2010
in US, Japan, and Canada revealed that sirolimus stabilized lung function, reduced symptoms, and improved QOL. In
this trial, 28 Japanese patients with LAM were enrolled and 24 completed the two-year trial course. PMDA (Japanese
FDA) demanded us to perform an open labeled single arm trial on more than 50 Japanese patients for confirmation of
safety, because only 13 Japanese patients had been assigned to Sirolimus group in MILES trial. The new trial,
multicenter lymphangioleiomyomatosis Sirolimus trial for safety (MLSTS) had been conducted during September 2012
to March 2015, in which 55 patients has been treated with 2 mg daily for 18 months as a single arm open-labeled
multicenter clinical trial. Distinct from MILES trial, patients with mild disease (present FEV1>70%, n=24) were not
excluded from enrollment, and thus, the new trial might assess the efficacy of early intervention for longer treatment
period. At 18 months treatment, the mean increase in FEV1 was 75 ml compared with the baseline data (n=44). Of
those 24 patients with %FEV1 less than 70%, the increase was 61ml, whereas it rather decreased by 6 months and
then increased by 18 months with 93ml in 20 patients with %FEV1 more than 70%. Using Friedman test, it was found
that FEV1 was not significantly smaller than the baseline level at 3, 6, 9,12, and 18 months in both patients with
%FEV1 >70% (mild) and <70% (moderate to severe). Similarly, the mean increase in FVC for 18 months treatment
was 38ml for all 44 patients tested, and the increases were 52 and 27 ml for patients with mild and moderate/severe
disease, respectively. There was no significant reduction in FVC during 18 month in both patient groups. One thousand
two hundred eighty six adverse events were reported in 18 months. Of those, 27 were severe adverse events with 3
cases of mild sirolimus pneumonitis that resolved within one month after cessation of sirolimus medication. There was
no difference in the frequency of adverse events between two groups. Thus, MLSTS data suggested that early
intervention by long-term sirolimus medication might inhibit the progression of LAM.
