Respiratory Tract (Lung and Bronchial) Diseases

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Lymphangioleiomyomatosis

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Lymphangiomyoma

Pleural Diseases

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Lymphoproliferative Disorders

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Somatostatin

Octreotide

Antineoplastic Agents, Hormonal

Patients with lymphangioleiomyomatosis and lymphatic tumors, ascites or pleural effusions who are symptomatic will receive subcutaneous injections of octreotide starting at a dose of 100 micrograms per day. Doses will be gradually increased to a maximum of 800 micrograms per day, two months after enrollment, if there is no response to lower doses.

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.

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.

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.

Treatment With Octreotide in Patients With Lymphangioleiomyomatosis

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%.

Octreotide treatment will be considered successful if the reported pain/symptom score is reduced by at least 2 levels at termination of treatment.

A simple visual numeric distress scale ranging from zero to 10 will be employed to rate the severity of individual symptoms. The best score is zero, which means absence of symptoms and the maximal is 10, meaning that the symptoms are very severe.

One or more abnormality of the following liver function tests:

1. Alkaline phosphatase above 116 i.u.
2. SGPT above 41 i.u.
3. SGOT from 34 i.u.
4. Total bilirubin above 1.0 mg/dl

National Heart, Lung, and Blood Institute (NHLBI)

Joel Moss, M.D./National Heart, Lung and Blood Institute

Patients with lymphangioleiomyomatosis and lymphatic tumors causing chylous effusions and abdominal pain

Age, Categorical

Age Continuous

Sex: Female, Male

United States

Region of Enrollment

Joel Moss

Number of Participants With a Reduction in Total Tumor Volume of at Least 20%.

Four patients with lymphangioleiomyomatosis and lymphangioleiomyomas and chylous effusions treated with octreotide injections by the subcutaneous route to determine whether the size of the tumors and effusions decrease with the therapy

Number of Participants With a Reduction of Pain/Symptoms as Measured by a Simple Numeric Symptom Distress Scale (NDS) to Rate the Severity of Individual Symptoms.

Number of Participants With Liver Function Abnormalities

Overall Study

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Treatment With Octreotide in Patients With Lymphangioleiomyomatosis	NCT00005906	Lymphangioleiom... Lymphangiomyoma... Pleural Effusio... Ascites	Octreotide	18 Years - 65 Years	National Institutes of Health Clinical Center (CC)
Treatment With Octreotide in Patients With Lymphangioleiomyomatosis	NCT00005906	Lymphangioleiom... Lymphangiomyoma... Pleural Effusio... Ascites	Octreotide	18 Years - 65 Years	National Institutes of Health Clinical Center (CC)

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