According to results from a phase 3 trial published in Biology of Blood and Marrow Transplantation, brincidofovir did not reduce cytomegalovirus (CMV) infection, a common complication of allogeneic hematopoietic cell transplantation (HCT), when compared with placebo. Additionally, brincidofovir was associated with gastrointestinal toxicity.

The randomized, double-blind, placebo-controlled trial included 452 CMV-seropositive patients aged 18 years or older who had undergone HCT in the United States, Canada, and Belgium between August 2013 and June 2015. The patients received either the placebo or one 100 mg brincidofovir tablet twice weekly (alternating 3- and 4-day intervals) through week 24 post-HCT. The primary endpoint was the proportion of patients with clinically significant CMV infection.

Patients who developed clinically significant CMV infection discontinued brincidofovir treatment and were given anti-CMV treatment instead. In total, 76.6% of brincidofovir-treated patients and 81.9% of placebo-treated patients completed the study.

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The proportion of patients who developed clinically significant CMV infection or were assumed to have clinically significant CMV on account of missing data was not significantly different among patients treated with brincidofovir compared with patients treated with placebo (51.2% vs 52.3%; OR 0.95 [95% CI: 0.6-1.4]; P =.805). However, brincidofovir was associated with lower levels of CMV viremia compared with the placebo (25.7% vs 41.6%, P <.001).

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Brincidofovir recipients experienced more serious adverse events than placebo recipients (57.1% vs 37.6%), the most common of which were acute graft-vs-host disease (32.3% vs 6.0%) and diarrhea (6.9% vs 2.7%). At 24 weeks post HCT, mortality was 15.5% and 10.1% for brincidofovir and placebo recipients, respectively (HR 1.6, 95% CI: 0.9-2.8; log-rank P =.117).

Due to the gastrointestinal adverse events experienced by the patients, the authors concluded that an intravenous formulation of brincidofovir “could provide a more effective and safer prevention and treatment of CMV and the multiple double-stranded DNA viruses that frequently affect allogeneic HCT recipients.”

Disclosure: Multiple authors declare affiliations with industry. Please refer to the original study for a full list of disclosures.


1. Marty FM, Winston DJ, Chemaly RF, et al. A randomized, double-blind, placebo-controlled phase 3 trial of oral brincidofovir for cytomegalovirus prophylaxis in allogeneic hematopoietic-cell transplantation [published online October 4, 2018]. Biol Blood Marrow Transplant. doi: 10.1016/j.bbmt.2018.09.038