Transcranial Doppler Screening of Children with Sickle Cell Disease for a Large, Multinational Interventional Study: Experience from the Phase 3 HOPE-Kids 2 Trial Investigating the Effect of Voxelotor Treatment on Transcranial Doppler Flow Velocity

• Published in: Blood 2023-11, Vol.142 (Supplement 1), p.3871-3871
• Main Authors: Bello-Manga, Halima, Nduba, Videlis, Abdullahi, Shehu Umar, Githanga, Jessie, Ogutu, Bernhards, El-Beshlawy, Amal M., Hassab, Hoda MA, Badr, Mohamed, Tantawy, Azza, Brown, Biobele J, Paintsil, Vivian, Akinsete, Adeseye Michael, Segbefia, Catherine I., Okpala, Iheanyi, Wali, Yasser, Essa, Mohammed, Farthing, Jason, Doss, Jennifer, Dixon, Sandy, Brown, Clark, Kirkham, Fenella
• Format: Article
• Language: English
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood-2023-188677

Introduction: Transcranial Doppler (TCD) ultrasound is an effective screening tool for identifying children with sickle cell disease (SCD) who are at high risk of stroke. Implementing TCD screening, along with appropriate therapies, for the purpose of primary stroke prevention in resource-constrained settings remains challenging. HOPE-KIDS 2 (NCT04218084) is an ongoing, phase 3, multicenter, double-blind, placebo-controlled trial of voxelotor in children with SCD and elevated arterial cerebral blood flow (measured by time-averaged maximum of the mean velocity [TAMMV]). Our aim is to describe the findings from successful implementation of a standardized TCD screening protocol that was conducted in a multinational setting. Methods: The purpose of HOPE-KIDS 2 is to evaluate the effect of voxelotor treatment on reducing the risk of stroke in participants aged 2 to