Treatment With Octreotide in Patients With Lymphangioleiomyomatosis
Condition: Lymphangioleiomyomatosis; Lymphangiomyomas; Pleural Effusions; Ascites
MeSH terms: Lymphangioleiomyomatosis; Lymphangiomyoma; Pleural Effusion; Ascites
Study type: Interventional
Phase: Phase 2

Summary: 
      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 effusions and
      lymphangioleiomyomas. Treatment of many of these symptoms has been ineffective. Previous
      studies with somatostatin and octreotide in other clinical settings have shown reduction in
      chylous effusions. This study assesses the effectiveness of octreotide in symptomatic
      patients with LAM, lymphangioleiomyomas and/or chylous effusions/ascites, peripheral
      lymphedema and chyluria.
    

Detailed description: 
      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.
    

Intervention name(s): Octreotide
Intervention type(s): Drug
Intervention description(s): Treatment with octreotide starts at a dose of 50 micrograms(ug) twice a day which is increased to 100 ug twice a day after two weeks and to 200 ug twice a day two weeks later. After two months, if there is no response the dose shall be increased to 400 ug twice a day.
Intervention MeSH terms: Octreotide
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Chylous Ascites; Chylous Pleural Effusion; Inhibitory Effects; Lymphangioleiomyoma; Somatostatin; Lymphangioleiomyomatosis (LAM)