Treatment With Octreotide in Patients With Lymphangioleiomyomatosis
Lymphangioleiomyomatosis (LAM), a disease primarily of women of child-bearing age, is
characterized by cystic lung disease and abdominal tumors (e.g., angiomyolipomas). Within
the LAM patient population is a subset of patients who develop chylous ascites, chylous
pleural effusions, chyluria, peripheral lymphedema, and/or lymphangioleiomyomas.
Lymphangioleiomyomas are believed to result from a proliferation of abnormal smooth muscle
cells within the lymphatic system, which appears to obstruct fluid outflow, leading to fluid
accumulation and an increase in size. The lymphangioleiomyomas may occur anywhere along the
axial lymphatic chain. In patients with LAM, they occur most frequently in the thorax,
abdomen and pelvis and may give rise to a myriad of symptoms (e.g., paresthesias,
palpitations, peripheral edema). In some patients, treatment of many of these symptoms,
i.e., elevation of lower extremities, paracentesis, thoracentesis, diuretics, and/or
surgery, has been ineffective. Previous studies with somatostatin and octreotide in other
clinical settings (e.g., traumatic damage to the lymphatics) have shown a successful
reduction in chylous effusions, chyluria, ascites, and peripheral lymphedema, when other
therapies were less effective. This study will assess the effectiveness of octreotide in
symptomatic patients with LAM, lymphangioleiomyomas and/or chylous effusions/ascites,
peripheral lymphedema and chyluria. The dose of octreotide starts at 50 micrograms (ug) by
the subcutaneous route twice a day. After two weeks the dose will be increased to 200 ug per
day and two weeks later to 400 ug/day. Maximal dose is 400 ug twice a day.
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Number of Participants With a Reduction in Total Tumor Volume of at Least 20%.
Octreotide treatment will be considered successful if the patient receiving treatment for six months shows a reduction in total tumor mass/ fluid collection or reaccumulation of at least 20%.
United States: Federal Government
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