Transplant Trial Watch

A randomized controlled trial of liposomal cyclosporine A for inhalation in the prevention of bronchiolitis obliterans syndrome following lung transplantation.

Neurohr, C., et al.

American Journal of Transplantation 2021 [record in progress].


Aims
This study aimed to examine the effect of adding liposomal cyclosporine A for inhalation (LCsA-i) to standard of care systemic immunosuppression for preventing bronchiolitis obliterans syndrome (BOS) in lung transplant recipients.

Interventions
Participants were randomised to either the LCsA-i group or the placebo group.

Participants
130 lung transplant recipients (≥18 years).

Outcomes
The primary outcome was BOS-free survival. Secondary efficacy outcomes included incidence of BOS, acute cellular rejection, pulmonary function, overall survival, and 6-minute walk test distance (6-MWD). Safety outcomes were evidence of infections and treatment-emergent adverse events.

Follow-up
The mean follow-up duration was 13.3 months in the L-CsA-i group and 13.4 months in the placebo grou

CET Conclusions
This is a well-written report of a good quality study in lung transplantation. The study was adequately randomised, blinded and would have been adequately powered based on prior calculations. Unfortunately, the study was stopped early due to the sponsor terminating support after an estimated 3.5 years of extended duration would be required. 130 patients were included, out of a planned cohort of 180. There was no significant difference in Bronchiolitis Obliterans Syndrome (BOS), however there was a trend towards benefit of the study drug in the per protocol analysis. The scale of the potential benefit, and the fact that the study failed to reach its target sample size, suggest that further trials are needed, and indeed two separate phase 3 trials are ongoing.

Jadad score
3

Data analysis
Per protocol analysis

Allocation concealment
No

Trial registration
ClinicalTrials.gov - NCT01334892; Eudra CT No: 2008-003800-73

Funding source
Industry funded