How can measurement of QoL and use of PROs be improved in cGvHD?

During the GvHD Hub Steering Committee Meeting on May 12, 2025, key opinion leaders met to discuss how to improve measurement of quality of life (QoL) and use of patient-reported outcomes (PROs) in cGvHD. The discussion was preceded by a presentation by Mohamad Mohty, and the discussion featured Nico Gagelmann, Bipin Savani, Ali Bazarbachi, Corey Cutler, and Andrew Harris. 

Mohty began by explaining the necessity of measuring QoL in patients with graft-versus-host disease (GvHD) and the current use of PROs in trials and clinical practice.

Key learnings

Presentation

Background¹

Both acute and chronic GvHD can have a profound impact on QoL, causing significant morbidity and mortality. In particular, cGvHD can persist for years resulting in a reduced functional status, affecting employment and daily living, and imposing a significant symptom burden. Use of PROs in clinical practice can be helpful in monitoring disease progression more effectively and improving long-term QoL.

Current use of PROs

There are barriers to consistent integration of PROs in clinical practice, including:²

• A lack of reference values for some instruments, making interpretation difficult
• Inconsistency of PRO use in clinical trials
• Potential increase in clinician workload

Despite efforts to assess the validity of various PROs (Figure 1) in the GvHD patient population, a consensus is lacking on the best PRO to use across different GvHD types and severities, giving a further challenge in more widespread and harmonious use of PROs in everyday practice.² The Lee Symptom Scale (LSS) is currently utilized in trials and clinical practice to enable patients to report the impact of each symptom. It uses a 30-item scale assessing 7 subscales (Figure 1) over a 30-day period.³

A modified version of the LSS, which assesses symptoms over 7 days, was utilized in a real-world study of belumosudil, to assess QoL in patients with fibrotic manifestations of cGvHD.⁴ In this study, fluctuations observed in the proportion of patients achieving a clinically meaningful reduction in mLSS emphasizes the value of longitudinal mLSS assessment for monitoring patient outcomes in cGvHD (Figure 2).⁴

Discussion 

• The LSS tool has not been validated for use in the pediatric population; therefore, in these patients, pediatric scales such as PROMIS might be more appropriate. Furthermore, the slightly modified and abridged version of the LSS has the potential for utilization in telehealth programs for adult and pediatric GvHD clinics.
• It can be difficult to implement QoL assessment in clinical practice due to time and financial constraints. The use of technology, such as computer tablets to collect data from patients, could be a solution in the future.
• Challenges remain in interpreting collected data in clinical practice, as this can vary from trials where QoL tools are more commonly used. In addition, patients may change their report of QoL at different time points during their disease, as fluctuations are common, and this can be difficult to capture.
• Despite these challenges, QoL is gaining momentum through various forums, and HCPs should advocate to integrate QoL and PROs as part of the drug assessments.

This educational resource is independently supported by Sanofi. All content was developed by SES in collaboration with an expert steering committee; funders were allowed no influence on the content of this resource.