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EHA 2026 | New EHA guidelines for patient-reported outcome assessment in MPNs
Esther Oliva, MD, London North West University Healthcare NHS Trust, London, UK, discusses the new European Hematology Association (EHA) Guidelines for patient-reported outcome (PRO) assessment in myeloproliferative neoplasm (MPN) clinical trials, highlighting the importance of capturing the patient’s experience of living with the disease, beyond objective markers. Dr Oliva explains that the guidelines provide practical international recommendations for the use of patient-reported outcome measures in trials, emphasizing the need for a tailored approach to selecting measures, standardized administration, and transparent reporting. This interview took place at the 31st Congress of the European Hematology Association (EHA) in Stockholm, Sweden.
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Transcript
Well, in myeloproliferative neoplasms, we often follow objective markers really carefully, blood counts, spleen size, thrombotic risk, molecular markers, and treatment response, which are essential, but they do not fully capture how the patient is living with the disease. So we know that patients with MPNs can experience fatigue, pruritus, night sweats, bone pain, abdominal symptoms, cognitive problems, sexual difficulties, anxiety, social or work limitations...
Well, in myeloproliferative neoplasms, we often follow objective markers really carefully, blood counts, spleen size, thrombotic risk, molecular markers, and treatment response, which are essential, but they do not fully capture how the patient is living with the disease. So we know that patients with MPNs can experience fatigue, pruritus, night sweats, bone pain, abdominal symptoms, cognitive problems, sexual difficulties, anxiety, social or work limitations. Some of these symptoms are chronic, fluctuating, and invisible during a standard consultation. So without structured patient-reported outcome assessment, we may underestimate the true burden of disease and the effects of treatment, both positive and negative. So our work addresses an important gap in myeloproliferative neoplasm clinical trials. Most studies include patient-reported outcomes, but the way they are selected, measured, and analyzed and reported remains inconsistent, making it difficult to compare results and fully understand the patient perspective.
So using a COSMIN systematic review of the available evidence, we developed practical international recommendations for the use of patient-reported outcome measures in myeloproliferative neoplasm trials, clinical trials. Our guidance recommends selecting PRO measures according to the specific trial objective and disease subtype rather than using a one-size-fits-all approach. For example, the MPN-SAF version 4 currently has the strongest evidence for symptom assessment in myelofibrosis, while broader quality of life instruments such as MPN-PRO or the EORTC-QLQ-C30 should complement disease-specific measures when appropriate. The guidelines emphasize standardized administration, minimizing missing data, pre-specified statistical analyses, transparent reporting, following international standards. Ultimately, our goal is to improve the quality and consistency of PRO data so that clinical trials better capture outcomes that truly matter to patients and generate evidence that is meaningful for regulators, clinicians, and most importantly, for patients.
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