Phase II Study of Posttransplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis After HLA-Mismatched Unrelated Donor Reduced Intensity Transplantation: Results From the ACCESS Trial Expansion Cohort.
Brian C Shaffer, Monzr M Al Malki, Antonio Martin Jimenez Jimenez et al.
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In 30 seconds
This prospective, multicenter phase II study evaluated posttransplant cyclophosphamide (PTCy)-based graft-versus-host disease prophylaxis in adults receiving peripheral blood stem cell transplantation from HLA-mismatched unrelated donors after reduced-intensity or non-myeloablative conditioning. Combined results included 70 participants from the initial study and 123 from a planned expansion cohort. One-year survival was 79.6% (95% confidence interval: 73.2-84.7), similar across donor-matching groups and between study cohorts. Severe infection was common, with a one-year incidence of 39% (32%-46.9%).
Key findings
- One-year survival was 79.6% (95% confidence interval: 73.2-84.7), similar for HLA 7/8- and < 7/8-matched donor recipients.
- Survival was similar in the initial study (78.6%) and expansion cohort (80.3%), which involved more participating centers.
- One-year incidence of severe infection (Grade 3-5) was 39% (32%-46.9%).
- One-year non-relapse mortality and relapse estimates were 12.5% (8.3%-17.6%) and 17.3% (12.3%-23%), respectively.
Why it matters
These results add prospective multicenter evidence on PTCy-based prophylaxis for patients undergoing reduced-intensity or non-myeloablative transplantation from mismatched unrelated donors. Similar survival across donor-matching groups and study cohorts supports the potential applicability of this approach across centers, while the substantial incidence of severe infection highlights an important post-transplant outcome.
What to keep in mind
The abstract reports a phase II study without a comparator group, so it does not establish comparative effectiveness against other prophylaxis strategies.