Feasibility of Patient-Reported Outcomes for Acute Graft-Versus-Host-Disease

Betty K. Hamilton, MD, Cleveland Clinic, Cleveland, Ohio, discusses the findings of a pilot trial analyzing patient-reported outcomes for the management of patients with acute graft-versus-host-disease (GVHD). 

Dr Hamilton et al found that though there were challenges in collecting patient-reported outcomes, the PROMIS-10 and PRO-CTCAE measures used did capture several symptoms and quality of life domains of acute GVHD. 

Transcript:

Hi, my name is Betty Hamilton. I'm an adult transplant physician from the Cleveland Clinic, and I'm associate professor of medicine. I'm going to talk a little bit about our pilot trial of patient-reported outcomes for acute graft-versus-host-disease.

Acute graft-versus-host-disease, of course, is a major cause of morbidity/mortality after allogeneic bone marrow transplant. We sought to evaluate the feasibility of obtaining patient-reported outcome measures in patients who have developed acute graft-versus-host-disease to measure their symptom burden and quality of life.

We conducted this pilot study of adult patients undergoing their first bone marrow transplant and used questions from several validated patient-reported outcome measures, including the FACT-BMT, the PROMIS-10 and the PRO-CTCAE. We obtained these measures at pre-transplant, and at days 14, 50, and 100 after transplant. And the goal was to administer these electronically.

We enrolled patients from 2018 to 2020 and were able to have a final study enrollment of 66 patients. The median age was 63 years. There was a low response rate in terms of patients' ability to fill out all the surveys. Only about 47% of the expected surveys were completed. And this just was due to the expected challenges of patients who could be sick and have a lot of symptoms, and have difficulty with answering some of the questions and using the electronic platform. 

But we did find that patients did report several physical and mental-emotional symptoms, especially in those patients with graft-versus-host-disease, and this was captured by all of the PRO measures, including the PRO-CTCAE. Almost all patients complained of fatigue and decreased appetite, as well as things like problems tasting, or difficulty with tasting, loose stools and pain, as well as some skin symptoms, as well as depression. And these symptoms were most common among patients with clinically significant acute graft-versus-host-disease.

In this study, we did identify many challenges with collecting patient reported outcomes. We found that it did require a dedicated person and a large team to be able to adequately collect these measures. And patients had difficulty with some of the electronic nature of the surveys, the fact that they were in some cases acutely ill, and again, that we needed more support in terms of collecting these data.

However, we also found that these measures were able to capture many symptoms and quality of life domains of acute graft-versus-host-disease, and underscores the need [to be] able to capture symptoms and quality of life directly from the patient in an acute illness like this.

Source:

Patel SS, Hong S, Rybicki L, et al. A pilot trial of patient-reported outcomes for acute graft-versus-host-disease. Transplant Cell Ther. Published online March 31, 2023. doi:https://doi.org/10.1016/j.jtct.2023.03.030