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December 8, 2025BloodOpen Access

Implementing patient-centered PROMs in sickle cell disease care: A european consensus approach

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Authors

RCRaffaella ColombattiMMMariane de MontalembertEBEduard J. van Beers

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Overview

Consensus panel highlights patient-reported outcomes measures to assess treatment efficacy in sickle cell disease, indicating improved clinical endpoints and health-related quality of life.

Key Points

  • This research aims to develop and implement patient-reported outcomes measures (PROMs) for sickle cell disease (SCD) to enhance clinical endpoint evaluation.
  • Formulation of PROMs based on a patient-centered approach focusing on health-related quality of life in SCD.
  • Utilization of the Delphi method with a multinational expert panel to validate content and select relevant items.
  • Inclusion of diverse cohorts: adults, pediatric patients, and caregivers, across multiple countries.
  • Implementation of PROMs is expected to provide improved assessment of treatment efficacy and clinical endpoints in SCD.
  • The focus on fatigue and physical function aims to enhance the understanding of disease impact on health-related quality of life.
  • The final PROMs forms will be accessible in several languages and incorporated into routine clinical practice by 2025.

Cite This Study

Colombatti et al. (2025) studied this question.

synapsesocial.com/papers/69362f7f4fa91c937236e547https://doi.org/10.1182/blood-2025-2666
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  4. 4Sickle cell disease in europe: A cross-border real-world data analysis from the radeep registry2025
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