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PROs May Successfully Risk Stratify Patients With Cutaneous Chronic GVHD


Two existing, well-validated patient-reported outcome (PRO) measures may be useful as a prognostic marker for mortality and assist with treatment selection for patients with cutaneous chronic graft-vs-host disease (GVHD).

Patient-reported outcomes (PROs) may have a role in risk stratification and treatment selection for patients with skin chronic graft-vs-host disease (GVHD). The disease is independently associated with long-term PRO impairment, with the degree of impairment being a prognostic marker for mortality, according to a study published in JAMA Dermatology.1

Chronic GVHD is a complication affecting up to half of allogeneic hematopoietic cell transplants. As a chronic, multisystem disorder, chronic GVHD can have a significant negative impact on physical and psychological well-being. In the majority of patients affected by chronic GVHD, the skin is involved, and these skin changes can range from mild to severe, including disfiguring and permanently disabling.2

“Cutaneous chronic GVHD is refractory to therapy in approximately half of patients exhibiting refractoriness to therapy,” the authors wrote.1 “There is a pressing need to identify patients who will maximally benefit from treatment addressing this debilitating condition.”

Patient speaking with doctor | Image credit: DragonImages - adobe.stock.com

Using 2 existing, well-validated patient-reported outcomes measures can help providers make more precise clinical assessments of cutaneous chronic graft-vs-host disease.

Image credit: DragonImages - adobe.stock.com

This multicenter prospective cohort study included 436 patients with cutaneous chronic GVHD in the Chronic GVHD Consortium, a longitudinal cohort of patients with chronic GVHD treated with systemic immunosuppression from 9 US medical centers. The majority (59.9%) were male and the median (IQR) age at transplant was 51 (41.5-56.6) years.

Slightly more than half (52.5%) of patients included had epidermal-type chronic GVHD, 30.0% had sclerotic disease, and 17.4% had a combination.

The researchers used the Lee Symptom Scale (LSS) skin subscale to assess symptom burden and the Functional Assessment of Cancer Therapy-Bone Marrow Transplantation (FACT-BMT) instrument to measure quality of life (QOL). On the LSS, higher scores indicate worse outcomes, while lower scores on the FACT-BMT indicate worse outcomes:

  • At time of disease onset, patients with sclerotic disease had a median LSS score of 25 compared with 35 for combination disease and 20 for epidermal, indicating sclerotic and combination disease had significantly worse outcomes.
  • While patients with sclerotic disease had worse FACT-BMT scores at disease onset than those with epidermal disease (median, 104 vs 109), the difference was not statistically significant.
  • All patients being treated for cutaneous chronic GVHD showed improvement of PRO scores over time.
  • LSS and FACT-BMT scores remained significantly worse in sclerotic and combination disease compared with epidermal disease throughout the study period.

Overall, 188 patients (43.1%) died and 29.1% died without relapse. For the FACT-BMT, clinically significant worsening (every 7 points the score worsened) at the time of diagnosis increased the odds of death from any cause by 9.1% (95% CI, 3.2-15.2; P = .002) and increased the odds of nonrelapse mortality by 9.1% (95% CI, 2.0-16.7; P = .01). The patients with the highest tertile of QOL impairment had the clearest association between PROs and death.

For the LSS score, clinically significant worsening (every 11 points the score worsened) increased the odds of death from any cause by 10.0% (95% CI, 1.2-19.5; P = .02) and the odds of nonrelapse mortality by 16.4% (95% CI, 5.5-28.5; P = .003). While the patients with the highest tertile of skin symptoms burden had the highest odds of nonrelapse mortality, the association with overall survival was not statistically significant.

The researchers noted the small sample size as a limitation to the study findings and recommend a larger study. As an observational study, it may also be subject to unmeasured confounding. They also recommended additional studies with better representation.

“This study population was predominantly White, well educated, and middle or upper class,” the authors wrote. “QOL impairment and skin symptom burden may be differentially impaired in samples with greater racial, ethnic, and socioeconomic representation. For example, patients with skin of color may have higher QOL impairment from epidermal disease and dyspigmentation than from sclerosis.”

In an accompanying editorial,2 Sandra A. Mitchell, PhD, CRNP, and Edward W. Cowen, MD, MHSc, both from the National Institutes of Health, noted appreciation that 2 well-validated PRO measures were used instead of a newly proposed instrument. The findings suggest that PROs may make clinical assessments of cutaneous chronic GVHD more precise and could mitigate measurement errors of therapeutic responses to treatment.

“This is an important consideration since sclerotic skin cGVHD assessment remains a hurdle in clinical trials of new agents to establish evidence-based treatment options,” they wrote. However, there are challenges with interpreting PROs as trial end points and the multisystem organ involvement in cutaneous chronic GVHD must be considered when interpreting changes in PRO scores.


1. Baumrin E, Shin DB, Mitra N, Pidala J, et al. Patient-reported outcomes and mortality in cutaneous chronic graft-vs-host disease. JAMA Dermatol. Published online February 28, 2024. doi:10.1001/jamadermatol.2023.6277

2. Mitchell SA, Cowen EW. Improving outcomes in chronic graft-vs-host disease. JAMA Dermatol. Published online February 28, 2024. doi:10.1001/jamadermatol.2023.6276

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