Clinical predictors of response in cGvHD: Results from the PQRST trial

Results from the prospective, observational Predicting the Quality of Response to Specific Treatments (PQRST) trial (NCT04431479), evaluating clinical predictors of treatment response in 181 patients with moderate-to-severe chronic graft-versus-host disease (cGvHD) requiring first-, second-, or third-line therapy, were published in the American Journal of Hematology by Hamilton et al. Clinical endpoints included best and 6-month response rates, failure-free survival (FFS), and clinical predictors of these responses.

Key data: The best responses rates were 72% by National Institutes of Health (NIH) criteria, 76% by clinician assessment, and 71% by patient-reported assessment. Response rates declined to ~40% at 6 months. FFS was 66% at 6 months and 41% at 18 months, and did not vary based on agent or line of therapy (LoT). Patient-reported fatigue measured by patient-reported outcomes measurement information system-29 (PROMIS-29; odds ratio [OR] 0.6; 95% confidence interval [CI], 0.5–0.9; p = 0.01) was the only clinical predictor of NIH response. A lower PROMIS physical health score was the only predictor of shorter FFS (OR, 0.6; 95% CI, 0.4–0.9; p = 0.02).

Key learning: Despite initial meaningful response rates, responses to contemporary cGvHD therapies are not durable across all agents and lines of therapy. Patient-reported fatigue and physical health measures represent the strongest clinical predictors of response and FFS, highlighting the need for integrating patient-reported outcomes into cGvHD management.