A Pilot Trial of Patient-Reported Outcomes for Acute Graft-Versus-Host-Disease

Researchers from the Cleveland Clinic have demonstrated the potential use of patient reported outcome (PRO) measures for acute graft-versus-host disease (GVHD) symptom burden and quality of life (QOL). Published in Transplantation and Cellular Therapy, the authors found that acute GVHD patients, on average, had lower QOL scores compared to patients with no (or mild) GVHD. While several challenges were encountered including an overall survey completion rate less than 50% and poor access to electronic surveys, this work validated the use of PRO measures for GVHD-associated QOL and symptom burden.  

Acute GVHD is a common complication of hematopoietic stem cell transplantation (HSCT) which can be life-threatening and is associated with a high mortality rate. PROs can be useful to monitor QOL and symptoms of acute GVHD, by providing real-time information from the patient's perspective. PROs can help track symptom severity and monitor response to treatment over time. The authors of this study sought to assess the feasibility of PRO measures in GVHD patients.

This study investigated 164 patients longitudinally, with 104 patients receiving an allogeneic HSCT (allo-HSCT). PROs to evaluate GVHD patients before HSCT, at days 14, 50, and 100 after HSCT were administered as electronic tests. Using questions from the Functional Assessment of Cancer Therapy–Bone Marrow Transplantation (FACT-BMT), Patient-Reported Outcomes Measurement Information System (PROMIS-10), and Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE), to assess QOL and symptom burden.

FACT-BMT and PROMIS-10 scores demonstrated an expected trajectory over time. There were significant differences in the area under the curve (AUC) for the mean of the top five symptoms (257 vs. 330, P < 0.036) in patients with acute GVHD compared to those without. This study demonstrates the challenges yet potential of using patient-reported outcomes in acute GVHD and further investigation into making PROs feasible in acute GVHD are needed.


Patel SS, Hong S, Rybicki L, et al. A Pilot Trial of Patient-Reported Outcomes for Acute Graft-Versus-Host-Disease [published online ahead of print, 2023 Mar 30]. Transplant Cell Ther. 2023;S2666-6367(23)01205-8. doi:10.1016/j.jtct.2023.03.030

Tags: AlloHCT, GVHD, Graft versus host disease, aGVHD, grafts, quality of life, graft, graft-versus-host disease, graft-versus-host

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