December 8, 2025BloodOpen Access

Sickle cell disease trials redefined: Toward unified clinical endpoints through international consensus

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Authors

MYMohamed A. YassinAin Shams University DJDipty Jain BABiree AndemariamSickle Cell Institute Chhattisgarh, Raipur

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Implication

International consensus defines key clinical endpoints in sickle cell disease trials, indicating improved measurement of treatment efficacy and outcomes.

Key Points

Sickle cell disease trials aim to standardize clinical endpoints to enhance treatment assessment consistency and efficacy.
Expert consensus achieved on defining vaso-occlusive episodes, pain frequency, and hemolysis as key clinical endpoints.
Consensus process employed modified Delphi methodology with 15 global hematology experts, validating key treatment measures.
This international agreement highlights the need for uniform clinical endpoints to guide future clinical trials and therapeutic strategies.

Cite This Study

Yassin et al. (2025) studied this question.

synapsesocial.com/papers/69362f7d4fa91c937236e48f https://doi.org/10.1182/blood-2025-1166

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Implication

International consensus defines key clinical endpoints in sickle cell disease trials, indicating improved measurement of treatment efficacy and outcomes.

Sickle cell disease trials redefined: Toward unified clinical endpoints through international consensus

Discussion

Member takes

Implication

Key Points

Cite This Study

Sickle cell disease trials redefined: Toward unified clinical endpoints through international consensus

Discussion

Member takes

Implication

Key Points

Cite This Study

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