How should we be measuring GvHD QoL in clinical practice?

During the GvHD Hub Steering Committee Meeting on November 26, 2024, key opinion leaders met to discuss measuring quality of life (QoL) in patients with chronic graft-versus-host disease (cGvHD) in clinical practice. The discussion was chaired by Mohamad Mohty, and featured Steven Pavletic, Hildegard Greinix, Arnon Nagler, Florent Malard, and Attilio Oliveri.

The meeting began with a short presentation by Mohty, who explained why addressing QoL concerns is key for patients with cGvHD, how symptom burden can vary between patients, and the impact of comorbidities on QoL.

Presentation

Impact of cGvHD on QoL

Poor QoL and physical functioning can occur in cGvHD,¹ with ~1 in 3 patients with moderate-to-severe cGvHD experiencing clinically significant psychological distress.² Immunosuppression, prolonged steroid use, reduced mobility, and older age can all contribute to decreased QoL.³ Furthermore, cGvHD can impact multiple domains, including social, economic, and psychological, leading to restrictions in daily life;⁴ patients with cGvHD are less likely to be in work or school due to health, and those with moderate-to-severe disease are likely to take medication for pain, anxiety, or depression.⁵

How to assess QoL in cGvHD

Inclusion of patient-reported outcomes (PROs) in clinical trials is not yet consistent; ideally all trials would have PROs as endpoints.⁶​ The Lee Symptom Scale (LSS) is a 30-item scale that assesses seven subscales (Figure 1), and can be utilized in both​ trials and clinical practice to enable patients to report the impact of each symptom.⁷

Figure 1. The LSS subscales and improvement point scale*

*Data from Lee, et al.⁷

LSS, Lee Symptom Scale.

Discussion

• Formal QoL assessment is not usually performed in clinical practice, despite tools existing that are used in clinical trials, such as the LSS. QoL assessment also varies across countries and individual centers, and this can lead to suboptimal outcomes for patients.
• Patient advisory boards can be helpful in providing firsthand input to bridge gaps in QoL assessments, particularly for pediatric outcomes and specific manifestations, such as lung disease and skin sclerosis, but standardized tools and greater discipline in integrating QoL measures into daily clinical workflows are needed.
• The U.S. Food and Drug Administration (FDA) is encouraging the use of Common Terminology Criteria for Adverse Events (CTCAE) PROs in early-phase clinical trials to capture patients' perspectives on treatment impact. In addition, combining tools such as the National Institute of Health (NIH) criteria and SF-36 can provide efficient and standardized QoL measurement; however, limited time and resources can often result in an inaccurate assessment of QoL, and current methods, such as informal interviews or using tools including the LSS and SF-36 questionnaire, need standardization and broader implementation.
• Where physicians, nurses, and staff teams frequently see patients in clinical practice and build consistent, trusting relationships, personalized monitoring, treatment, and support can also contribute to an understanding of patients’ QoL. The quality of patient communication is highly dependent on both staff consistency and patient openness, and training is needed to recognize and report symptoms or concerns to improve outcomes.
• Strategies to improve assessing and addressing QoL could include:
  • Incorporating novel treatments into first-line and refractory settings with a focus on patient well-being and minimizing long-term immunosuppressive therapy.
  • Systematic multidisciplinary care and support groups to help patients manage the physical and social burdens of cGvHD.
  • Digital surveys and electronic health tools to streamline the process without adding to clinician workload.
  • Using QoL assessments to guide healthcare providers in determining when to reduce or discontinue therapy.
  • An increased focus on trials to evaluate new therapies and treatment combinations aiming to balance efficacy with minimized toxicity; the use of PROs and systematic QoL evaluations in trials could drive better integration of patient perspectives into therapeutic decisions.
• Whilst the future goal is preventing or curing cGvHD, the immediate focus remains on improving QoL and managing the disease effectively, and systematic multidisciplinary care and broader engagement with patients and caregivers are crucial for comprehensive care.

This independent educational activity was supported by Sanofi. All content was developed independently. The funder was allowed no influence on the content of this activity.