Extracorporeal Photopheresis for the Management of Progressive Bronchiolitis Obliterans Syndrome in Medicare-Eligible Recipients of Lung Allografts

• Status: Active, not recruiting
• Trial end date: 0000-00-00
• Target enrollment: 160
• Participant gender: Both

Summary

The purpose of this study is to determine if Extracorporeal Photopheresis (ECP) is effective in the treatment of progressive Bronchiolitis Obliterans Syndrome (BOS) in patients after lung transplantation. Lung transplantation has become the treatment of choice for selected patients with end-stage lung disease. However, long-term survival after transplantation remains disappointing. Chronic rejection (bronchiolitis obliterans syndrome [BOS]) has emerged as the leading obstacle to better long-term outcomes, and represents the leading cause of death beyond the first year after transplantation. BOS is diagnosed by the decline in the FEV1 measurement from a pulmonary function test. The management of BOS has been disappointing. In general, BOS is treated by intensifying the immunosuppressive regimen. Despite treatment, most patients continue to show progressive decline in lung function resulting in worsening functional status, quality of life, and ultimately graft failure and death.

Extracorporeal Photopheresis (ECP) has been used as a salvage treatment for progressive BOS with favorable clinical results in many cases showing patient improvement. On May 2, 2012, the Center for Medicare Services issued a decision memo statin the ECP is covered for Medicare beneficiaries for the treatment of BOS following lung allograft transplantation only wehn the procedure is provided under a clinical research study. It is highly unlikely that providers that currently order ECP for their BOS patients who have already failed optimized immunosuppressive drug therapy would be willing to randomize half of their patients to continue on their failed drug therapy.What is not well understood at this time, however, is whether certain coexisting disease states or patient-related demographic, functional, treatment-related or diagnostic variables might prove to have predictive value in identifying subsets of BOS patients that are likely, or unlikely, to experience reduced rate of decline or stabilization in FEV1 following ECP treatment. This is a Registry study to enroll 160 patients from multiple U.S. centers to (1) confirm that ECP significantly reduces the rate of FEV1 decline in BOS patients refractory to standard immunosuppressive drug therapy, and (2) capture and assess specified patient demographic, treatment-related, diagnostic, functional and co-morbidity- related variables that may predict outcomes after ECP therapy.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Washington University School of Medicine
• Collaborators: Centers for Medicare and Medicaid Services; Mallinckrodt
• Treatments: Methoxsalen
• Last Updated: 2016-11-22

Criteria

Inclusion Criteria:

• Adult age (at least 18 years old).

• Medicare-eligible status

• Lung transplant recipient (combined organ transplant recipients, e.g. heart- lung liver-lung recipients, are eligible).

• Progressive BOS (defined as ongoing decline in FEV1 despite at least one of the following treatments; azithromycin, high-dose steroid, anti-thymocyte globulin, total lymphoid irradiation, sirolimus, or everolimus).

• At minimum four FEV1 measurements obtained at intervals of one to six weeks apart, over the 6 months preceding initial ECP treatment (may include an FEV1 measurement on or just prior to the first ECP procedure date).

Exclusion Criteria:

• Participation in another clinical treatment trial with an investigational agent.

• Any condition that may interfere with the subject's ability to perform pulmonary function testing.

• Known allergy or hypersensitivity to pharmacologic agents used during ECP.

• Any condition that would significantly affect the participant's ability to adhere to the protocol, affect interpretation of the study results, or put the participant at unacceptable risk for study-related complications as judged by the referring clinician. This may include patients with specific acute contraindication to receiving ECP due to any acute condition such as new or evolving myocardial infarction or central nervous system disorder, hemodynamic instability or hypovolemia, acute bleeding, or respiratory distress.

• Inability to provide informed consent or to comply with study treatments or assessments (e.g. due to cognitive impairment or geographic distance).