Chronic graft-versus-host disease (cGVHD) significantly diminishes patient quality of life (QOL), even in those who would be considered to have mild or moderate disease based on severity scoring. The integration of patient-reported outcomes (PROs) into clinical practice may help us ensure that treatment success is measured not only by disease markers but also by meaningful improvements in patients’ lives.

It is well known and recognized that the development of cGVHD, especially in moderate or severe forms, is one of the biggest determinants of QOL, morbidity, and mortality for allogeneic transplant recipients. Typically, when we think about cGVHD, we worry about high-risk manifestations such as sclerotic or fibrotic disease, which commonly affect the skin or lungs and can shorten people’s life spans. What is interesting is that other disease manifestations such as oral and ocular cGVHD that are not high risk in the traditional sense can wear people down if they cannot eat, drink, or see comfortably. People with cGVHD can feel downtrodden and depressed.

We have National Institutes of Health (NIH) criteria that help us try to objectively evaluate disease severity, but the evaluations for several disease manifestations can largely depend on the patient’s experience. So, a lot of how we evaluate cGVHD is subjective, such as the Lee cGVHD Symptom Scale, which is the most well-established PRO assessment used in most clinical trials of cGVHD. Although cGVHD disease severity and patient QOL align many times, manifestations that seem subtle to us can have a negative impact on patients’ lives, even if our perspective as clinical providers is that their disease is not severe. For patients who have more mild GVHD, symptoms other than traditional disease manifestations that can really affect QOL are fatigue, reduced activity levels, and a decreased ability to go back to work or complete activities of daily life. I think everybody hopes that they will be able to do the things that they were doing before they got sick, but cGVHD can really limit their ability to achieve that.

While recent cGVHD clinical trials have reported overall response rates of higher than 70%, fewer than 5% of those patients typically achieve a complete response; most responses are partial. We recognize as clinical providers that some partial responses are clinically meaningful, while others are not. People are thinking about whether we can use PROs to help better differentiate more meaningful vs less meaningful partial responses. The question then becomes: If someone has a partial response with a really big improvement in symptom scores, does that carry more weight than if they have an improvement but their symptom scores are basically unchanged? There are a lot of challenges in determining this. We may need to refine the PROs to ask different types of questions and think about whether we should put more emphasis on single readings vs multiple readings that show consistent improvement over time. These are all questions that we are trying to figure out.