Turacoz, a global scientific communications agency, supported an international Delphi consensus on clinical trial endpoints in sickle cell disease (SCD), from evidence synthesis to publication of the consensus manuscript in The Lancet Haematology.
SCD trials have long faced substantial variability in endpoint selection and the definitions of vaso-occlusive complications (historically described as “vaso-occlusive crisis”), severely limiting cross-trial comparability and the ability to demonstrate treatment efficacy. SCD, which includes sickle cell anemia, is a group of inherited red blood cell disorders marked by recurrent vaso-occlusive complications. Furthermore, recent high-profile clinical trials such as the HOPE trial of voxelotor demonstrated an increase of approximately 1.1 g/dL in hemoglobin levels; the annualized incidence of vaso-occlusive crises showed minimal reduction.1 Similarly, the crizanlizumab STAND trial demonstrated a significant reduction in annual rates of vaso-occlusive crisis, thus failing to achieve the endpoint.2 This highlights an urgent need for standardizing endpoints and trial design for SCD management.
The client partnered with Turacoz, a global scientific communications agency, for end-to-end strategic and publication support, encompassing evidence synthesis, expert meeting coordination, Delphi consensus coordination, comprehensive manuscript development, and journal submission support, including revisions in response to peer-review feedback. The project timeline was 14 months.
The project commenced with a comprehensive assessment of current practices in SCD clinical trials. The Turacoz team conducted an extensive evidence review and developed a detailed evidence deck summarizing recent SCD clinical trials, including study characteristics, endpoint definitions, eligibility criteria, and key results for each study. This evidence base supported the steering committee in defining the scope and objectives of the expert discussions.
An international panel of 15 experts from five continents, comprising the steering committee and additional experts, participated in the consensus process and voted on the proposed recommendations. Given the need to coordinate multiple experts across geographies and ensure timely completion of each stage, effective coordination and timeline management were critical to the success of the project. Turacoz facilitated this process by coordinating expert meetings, administering the online voting process, and systematically collating the responses.
Due to the clinical heterogeneity of SCD and the diverse backgrounds and healthcare settings represented by the experts, achieving agreement on standardized recommendations presented a significant challenge. Following the first round of voting, the steering committee carefully reviewed the areas of disagreement and refined the recommendations to incorporate relevant perspectives from the expert panel. The modified recommendations were subsequently subjected to another round of voting, after which consensus was achieved.
This initiative established the first international consensus recommendations for standardizing SCD clinical trial endpoints. This framework significantly enhances the design, interpretation, and reproducibility of future trials by guiding investigators, regulators, and the pharmaceutical industry in selecting clinically meaningful endpoints.
With Turacoz’s medical writing and strategic communication support, the consensus manuscript has been accepted for publication in The Lancet Haematology, providing a standardized framework to advance SCD research globally.
Variability in endpoint selection and in the definitions of vaso-occlusive complications limits cross-trial comparability and the ability to demonstrate treatment efficacy. Standardized endpoints and definitions support more consistent trial design, interpretation, and comparison across studies.
In the consensus, VOPC is reserved strictly for acute pain crises, whereas VOEps is the broader term that also covers non-pain vaso-occlusive complications.
The consensus recommended standardized trial inclusion criteria of at least 2 and no more than 12 VOEps per year.
An increase of ≥1 g/dL in hemoglobin was defined as clinically meaningful. The consensus also proposed distinct hemoglobin correction targets for acute versus chronic anemia.
Turacoz conducted the evidence review, developed an evidence deck, coordinated expert meetings, administered the online voting, collated responses, developed the manuscript, and supported journal submission and peer-review revisions, leading to the manuscript’s acceptance for publication in The Lancet Haematology.
Talk to the Turacoz team about publication writing, publication management, and submission support for consensus statements and journal manuscripts. Contact us: https://turacoz.com/contact-us/
Dr Sucharita Das
Senior Medical Writer
Medical Services, Turacoz Healthcare Solutions
About the author:
Dr. Sucharita is a Senior Medical Writer at Turacoz Healthcare Solutions with more than two years of experience in developing advisory board deliverables, drafting consensus manuscripts, and creating web-based encyclopedia content. She has hands-on experience supporting projects across the European and Middle Eastern markets, contributing to the development of high-quality, evidence-based scientific communications customized to regional healthcare needs.
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