Background: The management of myeloproliferative neoplasms (MPNs) has substantially evolved over the past decade. Including patient-reported outcomes (PROs) to supplement clinical outcomes is becoming increasingly important in clinical trials of patients with MPNs, to ensure the intended benefits based on patients’ perspectives. 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 implemented in MPN trials, how often they are used and whether results are reported consistently.
Aims: This systematic review aimed to explore the use and frequency of PRO instruments in randomised controlled trials (RCTs) for MPNs and assess the consistency of their reporting.
Methods: Under the auspices of the European Hematology Association (EHA), the development of guidelines for the use of PROs in adult patients with haematological malignancies was conceptualised. MEDLINE and CENTRAL were systematically searched for RCTs investigating MPNs between January 1st, 2015 and August 20th, 2021. 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. In order to assess the consistency of PRO reporting, study registries were compared with publications and study protocol, where available.
Results: Overall, 2626 records were found. Following screening for RCTs, 287 ongoing, completed or published studies were included for review. Sixty-seven studies planned the use of PROs: 25 were completed, and 32 studies were ongoing (23 recruiting; 4 not yet recruiting; 7 active; 6 terminated but unpublished; 2 unknowns). For 12 studies, trial protocols were available. Thereof, 7 reported PROs inconsistently. In 2 trials, PROs were assessed as both primary and secondary outcomes. In most trials, they were reported as secondary outcomes (51 trials) and were a primary outcome in only 10 trials, while they were exploratory in 2 and not specified in 1. Twenty-six PRO measures (PROMs) were reported. The most frequently used (in 15 trials) generic PROMs were the EQ-5D (generic) and the EORTC QLQ-30 (disease-specific - created to assess health-related quality-of-life/ HRQoL in patients with solid tumours). The most frequently used (in more than 10 trials) MPN-specific PROMs were MPN-SAF TSS (or MPN-10) and MF-SAF (including v2.0 and v4.0). The version of MPN-SAF was not specified in 11 trials and that for MF-SAF in 2 trials. Noteworthy, the most frequently used MPN-specific instruments were created by the same authors, who also were involved in clinical trials.
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Summary/Conclusion: The findings indicate that there is increasing interest in the use of PROs as an outcome in MPNs with the availability of MPN-specific PROMs. The use of such instruments has been important for drug approval. Thus, guidelines for the appropriate use of PROs are needed to ensure standardisation in the selection and reporting of PROs.