Vorinostat for graft-versus-host disease prevention in pediatric and young adult patients with hematologic malignancies.
Nicolas Parnell, Xiao Cao, Jan H Beumer et al.
✦ AI-curated · Sources linked
In 30 seconds
This prospective, single-arm, multicenter phase I/II trial evaluated vorinostat added to standard GVHD prophylaxis in patients aged 3–39 years undergoing allogeneic hematopoietic cell transplantation from matched or haploidentical donors. Among 43 enrolled patients, the recommended phase II dose was 60 mg/m² twice daily. By day +100, grade 2–4 acute GVHD occurred in 14% (95% CI, 5.6%–26%); grade 3–4 acute GVHD occurred in 4.7% (95% CI, 0.83%–14%). No dose-limiting toxicities or primary graft failures were reported. The findings support randomized evaluation, but do not establish comparative efficacy.
Key findings
- Day +100 grade 2–4 acute GVHD incidence was 14% (95% CI, 5.6%–26%); grade 3–4 incidence was 4.7% (95% CI, 0.83%–14%).
- The recommended phase II vorinostat dose was 60 mg/m² twice daily.
- No dose-limiting toxicities, unexpected vorinostat-related serious adverse events, or primary graft failures occurred.
- One-year overall survival was 88.4% (95% CI, 79.3%–98.5%), and GVHD-free/relapse-free survival was 55.8% (95% CI, 42.8%–72.8%).
Why it matters
Acute GVHD remains an important outcome after allogeneic transplantation. This study provides early prospective evidence on the feasibility and safety of adding vorinostat to prophylaxis regimens across matched and haploidentical donor settings in younger patients. The reported GVHD incidence and survival outcomes can inform the design of comparative studies, while the single-arm design means they cannot show that vorinostat improves outcomes over standard prophylaxis.
What to keep in mind
The trial was single-arm and enrolled 43 patients, so it cannot establish comparative efficacy; the reported outcomes are from an early-phase study.