Associate professor of Hematology Hematology-Oncology Clinic/Tartu University Tallinn, Harjumaa, Estonia
Introduction: Patient-reported outcome measures (PROM) are important in assessing significant and meaningful changes as a result of an intervention based on a patient’s own perspective. Multiple myeloma (MM) is characterized by a high burden of disease and treatment-related symptoms with considerable worsening of quality of life (QoL). Optimization of QoL of patients with MM is an important treatment goal and the incorporation of PROs into clinical trials has the potential of improving treatment outcomes.
Methods: Under the auspices of the European Hematology Association (EHA), development of guidelines for the use and reporting of PROs in clinical trials in adult patients with MM was conceptualized following the EHA core Guidelines Development Methodology. The Cosmin guideline for Systematic Reviews for PROMs was followed. The PROMs commonly used in MM patients within randomized clinical trials (RCT) between 2015 and 2020 and their psychometric properties were reviewed. Study design, disease and treatment characteristics, the primary outcome and the implemented PRO instrument(s) were extracted using a pre-defined template.
Results: Overall, 10,707 records were found in which 38 different PRO instruments were reported. Finally, 118 studies were selected as appropriate for review. The most frequently used instrument was the EORTC QLQ-30 used in 92 studies. The EORTC-MY20 MM and the EQ-5D were both used in 50 studies. Further, information on PRO in HTA reports was available for 26 studies, of which 18 reports were consistent with the trial registries. Out of the 38 instruments used, 6 had been validated for patients with MM, 6 for patients with haematological malignancies, (HM) and 10 for cancer patients in general.
Conclusions: The findings indicate that the measurement of PROs in RCTs for MM is underutilised, underreported, often inconsistent and not all PRO instruments identified have been validated for MM patients or patients with HM. Thus, guidelines for the appropriate use and reporting of PROs are needed in MM to ensure standardisation in the selection and reporting of PROs. The instrument of choice should possess robust measurement properties and meet the following requirements: validity, reliability, and sensitivity to change in MM, with a published minimal clinically identified differences. The choice of PROM(s) in clinical trials depends on the combination of the following factors: disease status; primary and secondary objectives; and in case of multinational trials, the availability of translations. The QoL measures that have been identified through systematic literature review as appropriate are: EQ-5D-3L, EORTC QLQ-C30, EORTC QLQ-MY20, FACT-MM (BMT), MDASI-MM, MyPOS and HM-PRO. Recommended symptom assessment PROMs are: NRS/VAS, FACIT-F, HAD and HM-PRO SS. The inclusion of PROMs should be evaluated with guidance. It is therefore hoped that clinicians, regulatory agencies, and the pharmaceutical industry find these Guidelines useful for clinical trials in MM.
