CINCINNATI, OH--(Marketwired - September 26, 2016) - The LAM Foundation hosted its annual LAMposium conference in conjunction with the 2016 International Rare Lung Diseases Research Consortium, the largest rare lung diseases conference of its kind. Both events were held concurrently in Northern Kentucky from September 22-25.
More than 450 scientists and patients representing some 20 countries were in attendance at the conference, which offered a forum to educate patients, jump start clinical trials and introduce the latest research on rare lung diseases.
LAM or Lymphangioleiomyomatosis is a systemic disease that affects about five women per one million. In patients with LAM, neoplastic smooth muscle-like cells arise from an unknown source, infiltrate the lung and result in cystic changes. Lung function declines at two to four times the normal rate, often punctuated by repeated lung collapses. Most patients are breathless with daily activities and require supplemental oxygen within 10 years of onset of symptoms.
The LAM Foundation, based in Cincinnati, is working on a national basis to enhance the care of women with LAM as well as fostering collaboration among scientists and clinicians to inspire innovative research efforts.
"By combining our LAMposium conference with the 2016 International Rare Lung Diseases Research Consortium, we were able to provide a direct means for our patients to contribute to research and inspire doctors and scientists to further their causes," said Susan Sherman, executive director of The LAM Foundation. "The conference allowed The LAM Foundation to continue its mission of fostering collaboration among scientists and clinicians while raising awareness and funds for LAM research."
During the conference, a $3.6 million grant from the National Institutes of Health was announced to fund a new five-year trial called MILED, the follow-up to the groundbreaking trial, MILES, which led to the approval of sirolimus to suppress the effects of LAM. The objective of MILED is to determine if sirolimus can be used much earlier and in lower doses to maintain normal or near normal lung function in patients with mild LAM.
On Sunday of the Conference, new Clinical Guidelines for LAM recently published in The American Thoracic Society (ATS) and the Japanese Respiratory Society (JRS), were introduced at the Clinic Director's Congress. The guidelines were published in the September issue of the ATS's American Journal of Respiratory and Critical Care Medicine.
The European Respiratory Society published comprehensive LAM guidelines in 2010. Over the last six years, however, rapid advances in LAM science have identified an effective treatment and a useful diagnostic biomarker. LAM experts within the ATS and JRS communities joined forces to develop recommendations for these new diagnostic and therapeutic tools.
Joel Moss, MD, PhD, deputy chief, cardiovascular and pulmonary branch, of the National Heart, Lung, and Blood Institute, and Francis X. McCormack, MD, director of the division of pulmonary, critical care and sleep medicine at the University of Cincinnati, chaired the guidelines committee. Input from LAM patients was obtained through a questionnaire distributed by The LAM Foundation.
The committee made five key recommendations based on evidence of varying quality:
- For patients with LAM with abnormal/declining lung function, we recommend treatment with sirolimus rather than observation. This is a strong recommendation based on moderate quality evidence.
- For selected patients with LAM with problematic chylous effusions, we suggest treatment with sirolimus before invasive management. This is a conditional recommendation based on very low-quality evidence.
- We suggest NOT using doxycycline as treatment for LAM. This is a conditional recommendation based on low-quality evidence.
- We suggest NOT using hormonal therapy as treatment for LAM. This is a conditional recommendation based on very low quality evidence.
- For patients whose computed tomography scan shows cystic abnormalities characteristic of LAM, but who have no other confirmatory clinical or extrapulmonary radiologic features of LAM, we recommend vascular endothelial growth factor D testing to establish the diagnosis of LAM before considering a diagnostic lung biopsy. This is a strong recommendation based on moderate-quality evidence.
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