Efficacy Study of Azithromycin-based Therapy for Bronchiolitis Obliterans
- Conditions
- Graft vs Host DiseaseBronchiolitis Obliterans
- Interventions
- Registration Number
- NCT01327625
- Lead Sponsor
- Asan Medical Center
- Brief Summary
\[Study Objectives\]
* To evaluate the efficacy of azithromycin, N-acetylcystein, and inhaled corticosteroid combination therapy in patients with bronchiolitis obliterans as a complication of allogeneic hematopoietic cell transplantation in terms of response rate at 6 months after treatment initiation based on the improvement of FEV1.
- Detailed Description
* Bronchiolitis obliterans (BO) is a graft-versus-host disease of respiratory organs.
* Prognosis of BO is very poor, and the overall outcome of patients who are involved in BO is very dismal.
* The mechanism of BO has been known to be associated with immune / non-immune response.
* Corticosteroid and immunosuppressants are recommended as a best current treatment options for BO, which have been not satisfactory.
* Many treatment options have been tried to improve the outcome of BO.
* Azithromycin, as an immune modulating agent, has been tried for the treatment of BO, and has been reported to show hopeful results.
* N-acetylcystein, as an antioxidative agent, has been tried for BO.
* Inhaled corticosteroid may help to improve airway inflammation and decrease the amount of systemic corticosteroid.
* These 3 drugs are widely used for other respiratory disease, have been proven to be safe, and have shown some efficacy for BO in various depth of evidence.
* In these rationale, we'd like to try the 3-drug combination for BO, to assess the efficacy and safety of these drug combination.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 6
- Patients who previously received allogeneic hematopoietic cell transplantation due to hematologic malignancy, bone marrow failure syndrome, and other compatible disease.
- Patients who are diagnosed as bronchiolitis obliterans (BO) according to the NIH diagnostic guideline which is suggested as below.
- Patients should be 15 years of age or older, but younger than 75 years.
- Patients should have estimated life expectancy of more than 3 months.
- Patients must have adequate hepatic function (bilirubin less than 3.0 ㎎/㎗, AST and ALT less than three times the upper normal limit).
- Patients must have adequate renal function (creatinine less than 2.0 ㎎/㎗).
- Presence of significant active infection
- Presence of uncontrolled bleeding
- Any coexisting major illness or organ failure
- Patients with a psychiatric disorder or mental deficiency severe as to make compliance with the treatment unlike, and making informed consent impossible.
- Nursing women, pregnant women, women of childbearing potential who do not want adequate contraception
- Patients with a diagnosis of prior malignancy unless disease-free for at least 5 years following therapy with curative intent (except curatively treated nonmelanoma skin cancer, in situ carcinoma, or cervical intraepithelial neoplasia)
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Azithromycin azithromycin + N-acetylcystein + inhaled corticosteroid Patient who are diagnosed as bronchiolitis obliterans according to the WHO criteria
- Primary Outcome Measures
Name Time Method Response rate based on the improvement of FEV1 6 months Response rate at 6 months after treatment initiation based on the improvement of FEV1
- Secondary Outcome Measures
Name Time Method Clinical benefit rate based on the degree of change in FEV1 6 months Clinical benefit rate at 6 months after treatment initiation based on the degree of change in FEV1
change in FEV1 compared with pretreatment level 6 months after treatment initiation Change in FEV1 at 6 months after treatment initiation compared with pretreatment level
Reduction rate in immunosuppressive agent / systemic corticosteroid 6 months after treatment initiation Reduction rate in immunosuppressive agent / systemic corticosteroid at 6 months after treatment initiation
Discontinuation rate in immunosuppressive agent / systemic corticosteroid 6 months after treatment initiation Discontinuation rate in immunosuppressive agent / systemic corticosteroid at 6 months after treatment initiation
Change in dose-intensity of immunosuppressive agent / systemic corticosteroid compared with pretreatment dose-intensity 6 months after treatment initiation Change in dose-intensity of immunosuppressive agent / systemic corticosteroid at 6 month after treatment initiation compared with pretreatment dose-intensity
event-free survival 1 year overall survival 1year
Trial Locations
- Locations (1)
Asan Medical Center
🇰🇷Seoul, Korea, Republic of
Asan Medical Center🇰🇷Seoul, Korea, Republic of