Contributions
Abstract: EP1187
Type: E-Poster Presentation
Session title: Quality of life, palliative care, ethics and health economics
Background
The treatment landscape for the management of multiple myeloma (MM) has substantially evolved over the past decade. Inclusion of humanistic outcomes reported by patients (PROs) to supplement the clinical outcomes is becoming increasingly important in MM clinical trials to ensure the intended benefits based on patients’ perspective. For patients with MM, PRO measurement is essential to understand the impact of treatment on their physical and psychosocial functional behaviour and evaluate the benefit-risk balance of specific treatments. However, it is unclear which PRO instruments are utilized in multiple myeloma trials, how often they are used and whether results are consistently reported.
Aims
The aim of this project was to explore the use and frequency of PRO instruments in randomized controlled trials (RCTs) for MM and to assess the consistency of their reporting.
Methods
Under the auspices of the European Hematology Association (EHA), development of guidelines for the use of PROs in adult patients with hematological malignancies was conceptualised. MEDLINE and CENTRAL were systematically searched for RCTs investigating multiple myeloma between 2015 and 2020. Study design, disease and treatment characteristics, primary outcome, PRO instrument(s) as well as the methods of their reporting were extracted using a pre-defined template. To assess the consistency of PRO reporting, study registries were compared with publications, study protocol and Health Technology Assessment (HTA) reports, where available.
Results
Overall, 10,707 records were found. Following screening for RCTs, 283 ongoing, completed or published studies were included for review. 118 studies planned the use of PROs. The most frequently used PRO instrument (92 studies) was the EORTC QLQ-30 measuring health-related quality-of-life (HRQoL) in cancer patients. The disease-specific questionnaire EORTC-MY20 was also frequently used (50 studies). Likewise, the HRQoL instrument EQ-5D was used in 50 studies. Overall, 38 different PRO instruments were reported. In 39 studies for which a study protocol was found, only 19 reported PRO instruments consistently with the trial registry for the study. In addition, in 30 studies for which a publication reported PRO results (58 overall), the information on PRO instruments differed between publication and the trial registry. Further, information on PRO in HTA reports was available for 26 studies of which 18 reports were consistent with the trial registries.
Conclusion
The findings indicate that despite the general agreement on the importance of measurement of PROs in multiple myeloma RCTs, they remain under-utilized and under-reported. While most studies identified in this review comply with the recommendations for incorporating PROs into adult oncology trials, they are often inconsistently reported. Thus, guidelines for the appropriate use of PROs are needed to ensure standardisation in selection and reporting of PROs.
Keyword(s): Multiple myeloma, Quality control, Quality of life
Abstract: EP1187
Type: E-Poster Presentation
Session title: Quality of life, palliative care, ethics and health economics
Background
The treatment landscape for the management of multiple myeloma (MM) has substantially evolved over the past decade. Inclusion of humanistic outcomes reported by patients (PROs) to supplement the clinical outcomes is becoming increasingly important in MM clinical trials to ensure the intended benefits based on patients’ perspective. For patients with MM, PRO measurement is essential to understand the impact of treatment on their physical and psychosocial functional behaviour and evaluate the benefit-risk balance of specific treatments. However, it is unclear which PRO instruments are utilized in multiple myeloma trials, how often they are used and whether results are consistently reported.
Aims
The aim of this project was to explore the use and frequency of PRO instruments in randomized controlled trials (RCTs) for MM and to assess the consistency of their reporting.
Methods
Under the auspices of the European Hematology Association (EHA), development of guidelines for the use of PROs in adult patients with hematological malignancies was conceptualised. MEDLINE and CENTRAL were systematically searched for RCTs investigating multiple myeloma between 2015 and 2020. Study design, disease and treatment characteristics, primary outcome, PRO instrument(s) as well as the methods of their reporting were extracted using a pre-defined template. To assess the consistency of PRO reporting, study registries were compared with publications, study protocol and Health Technology Assessment (HTA) reports, where available.
Results
Overall, 10,707 records were found. Following screening for RCTs, 283 ongoing, completed or published studies were included for review. 118 studies planned the use of PROs. The most frequently used PRO instrument (92 studies) was the EORTC QLQ-30 measuring health-related quality-of-life (HRQoL) in cancer patients. The disease-specific questionnaire EORTC-MY20 was also frequently used (50 studies). Likewise, the HRQoL instrument EQ-5D was used in 50 studies. Overall, 38 different PRO instruments were reported. In 39 studies for which a study protocol was found, only 19 reported PRO instruments consistently with the trial registry for the study. In addition, in 30 studies for which a publication reported PRO results (58 overall), the information on PRO instruments differed between publication and the trial registry. Further, information on PRO in HTA reports was available for 26 studies of which 18 reports were consistent with the trial registries.
Conclusion
The findings indicate that despite the general agreement on the importance of measurement of PROs in multiple myeloma RCTs, they remain under-utilized and under-reported. While most studies identified in this review comply with the recommendations for incorporating PROs into adult oncology trials, they are often inconsistently reported. Thus, guidelines for the appropriate use of PROs are needed to ensure standardisation in selection and reporting of PROs.
Keyword(s): Multiple myeloma, Quality control, Quality of life