Patient-reported outcomes (PROs) for chronic graft-versus-host-disease (cGVHD) are an important part of clinical practice and are becoming almost a required component of clinical trials of new cGVHD therapies. In addition to helping the medical team understand their patients’ quality of life (QOL), there may also be a role for PRO end points to be the basis of future US Food and Drug Administration (FDA) drug approvals.

An assessment of PROs in cGVHD is critical because, at the end of the day, the goal is to make our patients feel better and live longer. Understanding which therapies impact patients the most and make them better is absolutely crucial. Based on the severity of a patient’s cGVHD, we can try to extrapolate their risk of morbidity and mortality. The severity of cGVHD also very strongly correlates with patient-reported QOL.

PRO scoring scales for cGVHD include the Lee Chronic GVHD Symptom Scale, which has 7 subscales devoted to skin, eye, mouth, nutrition, energy, psychological status, and lung function. There are also the Patient-Reported Outcomes Measurement Information System (PROMIS) Global-10 and PROMIS-29 scoring scales. Patients can fill these out while they are in the waiting room before their appointments, and each questionnaire can often be completed in 5 to 10 minutes.

I think that current clinical trials in cGVHD almost have to include PROs. FDA drug approval based purely on PROs as a primary end point is a new concept and is overdue. We have not yet seen a drug for the cGVHD space use that as the sole path to FDA approval, but I think that it certainly is coming.

Work is also being done to integrate PROs into the identification of potential biomarkers that could predict patient response to treatment. The longitudinal prospective PQRST trial is following patients who are getting first-, second-, or third-line systemic therapy for cGVHD. This study is trying to understand the relationship between some biomarkers and clinical outcomes, as well as whether there is a subset of patients in whom a biomarker would suggest that one drug is better than another. In this study, PROs are being collected using PROMIS Global-10, PROMIS-29, and the Lee Chronic GVHD Symptom Scale. The holy grail in cGVHD is getting the right drug to the right patient, and that is what I think this study is really going to do, because it is about understanding which drugs work for which patients.