Plastic Bronchitis
Conditions
Keywords
plastic bronchitis, sirolimus
Brief summary
There is no confirmed drug therapy for plastic bronchitis. The study wish to test the effectiveness and safety of sirolimus (rapamycin) in patients with lymphatic plastic bronchitis.
Detailed description
Plastic bronchitis is a rare respiratory disorder characterized by formation of gelatinous or rigid cast of bronchial tree. Confirmed drug therapy for plastic bronchitis is lacking. Recently, part of plastic bronchitis has been found to have dysfunction of lymphatic circulation in the lung. Pulmonary lymphatic perfusion syndrome (PLPS) is regarded as a major mechanism of lymphatic plastic bronchitis. Sirolimus (rapamycin) has not been studied in plastic bronchitis, however, it has been shown effective in several lymphatic disorders, such as lymphangioleiomyomatosis, generalized lymphatic anomaly (GLA) including lymphangiomatosis, etc. We wish to test the effectiveness of sirolimus in lymphatic plastic bronchitis.
Interventions
DRUGSirolimus
Patients will receive sirolimus for 6 months.
Sponsors
North-China Pharmaceutical Company, China
Peking Union Medical College Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Ten lymphatic bronchitis patients are planned to be recruited for the study. Sirolimus will be administered for 6 months.
Eligibility
Sex/Gender
ALL
Age
13 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* plastic bronchitis * pulmonary lymphatic perfusion syndrome demonstrated on 68Ga-NEB positron emission tomography (PET)
Exclusion criteria
* Pregnancy and breastfeeding * Severe cardiovascular, hepatic and renal dysfunction * allergy to sirolimus or 68Ga-NEB
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| changes of pulmonary lymphatic perfusion distribution | 6 months | quantitatively measured with 68Ga-NEB positron emission tomography (PET) at baseline and the end of study |
| changes of coughing score | 6 months | measured by coughing VAS score (0-10) at baseline, 3 months and the end of study |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| changes of six minutes walking distance | 6 months | measured by six minutes walking test at baseline, 3 months and the end of study |
| change of health-related quality of life | 6 months | measure by St George Respiratory Questionnaire at baseline, 3 months and the end of study |
| Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 | 6 months | Common Terminology Criteria for Adverse Events (CTCAE) was used to collect adverse effects at baseline and through study completion |
| changes of breathlessness score | 6 months | measured by Borg scale (0-10) at baseline, 3 months and the end of study |
| changes of pulmonary function (FEV1, FVC) | 6 months | measured by spirometry at baseline, 3 months and the end of study |
Countries
China
Contacts
Primary ContactKai-Feng Xu
Outcome results
None listed