Chronic graft-versus-host disease severity should be measured by patient-reported outcomes

Key takeaways:

• Patients with sclerotic-type or combination disease had worst outcomes than epidermal-type disease.
• Clinical worsening of quality of life was associated with a 9.1% increase in nonrelapse mortality risk.

Chronic graft-versus-host disease was associated with clinically meaningful quality of life impairment and symptom burden, making patient-reported outcomes an important prognostic marker, according to a study.

Chronic graft-versus-host disease is a systemic disorder where a graph’s cells attack the host’s cells after a transplant procedure. While other organs can be affected, up to 80% of patients with this chronic condition report skin involvement, characterized as either epidermal or sclerotic, and approximately half do not respond to therapy.

“There is a pressing need to identify patients who will maximally benefit from treatment addressing this debilitating condition,” Emily Baumrin, MD, MSCE, assistant professor of dermatology at Perelman School of Medicine at the University of Pennsylvania, and colleagues wrote. “This multicenter prospective cohort study aims to juxtapose longitudinal [patient-reported outcomes (PROs)] in patients with epidermal and sclerotic disease and assess whether PROs independently pinpoint individuals with elevated mortality risk.”

The study included 436 patients (median age at transplant, 51 years; 59.9% men) with cutaneous chronic graft-versus-host disease, of which 229 had epidermal-type disease, 131 had sclerotic-type disease and 76 had combination disease.

Patient-reported symptom burden was evaluated using the Lee Symptom Scale (LSS), with higher scores equaling worse outcomes, and quality of life was assessed using the Functional Assessment of Cancer Therapy-Bone Marrow Transplantation (FACT-BMT) instrument, with lower scores signifying worse outcomes.

According to measurements attained through FACT-BMT, patients with sclerotic chronic graft-versus-host disease had worse quality of life than those with epidermal disease, scoring 6.1 points (95% CI, 11.7-0.4) lower. According to the LSS, patients with combination disease also had worse outcomes, reporting a higher burden of symptoms by 9 points (95% CI, 4.2-13.8) compared with those with epidermal disease.

It was also found that patients with a greater impairment in PROs at diagnosis of cutaneous chronic graft-versus-host disease had increased rates of mortality. Results showed that for every clinically meaningful difference — defined as a 7-point worsening — in FACT-BMT score at diagnosis there was a 9.1% (95% CI, 2%-16.7%) nonrelapse mortality increase. For every clinically meaningful difference in PROs — or 11-point worsening — there was an increased risk of nonrelapse mortality of 16.4% (95% CI, 5.4%-28.5%).

“PROs are critical measures of disease severity in cutaneous chronic graft-versus-host disease and could potentially serve as a clinical prognostic marker for mortality,” the authors concluded. “Future studies should focus on psychometric validation of PROs so that they can be incorporated as a prognostic and response marker in clinical practice and clinical trials.”