4 Hematology Headlines You Missed in April 2026

April 2026 brought major developments in hematology, particularly in sickle cell disease (SCD), with advances spanning phase 3 therapeutics, gene-editing strategies, pediatric thrombosis prevention, and global disparities research.

From promising efficacy data for etavopivat and renizgamglogene autogedtemcel (reni-cel) to new anticoagulant prophylaxis guidance for pediatric venous thromboembolism (VTE), the month’s headlines highlighted both innovation and persistent unmet needs across hematologic care.

Phase 3 HIBISCUS: Etavopivat Cuts VOC Events, Improves Hemoglobin in SCD Novo Nordisk announced positive topline results from the phase 3 HIBISCUS trial evaluating etavopivat in sickle cell disease. Added to standard of care, etavopivat achieved both co-primary endpoints, including a 27% reduction in vaso-occlusive crisis (VOC) events and an approximately 4-month delay to first VOC compared with placebo. Additionally, 48.7% of treated patients achieved a hemoglobin increase of >1 g/dL after 24 weeks versus 7.2% in the placebo arm, reinforcing the therapy’s potential disease-modifying benefit.

12 New Recommendations for Anticoagulant Prophylaxis in Pediatric Patients at Risk for VTE New evidence-based guidance from the American Society of Hematology (ASH) and the International Society on Thrombosis and Haemostasis (ISTH) addressed a longstanding gap in pediatric thrombosis management. Published April 8 in Blood Advances, the recommendations provide clinicians with 12 new consensus-based strategies for anticoagulant prophylaxis in pediatric patients at elevated risk for venous thromboembolism, aiming to support more standardized and evidence-driven care in this vulnerable population.

Increased Sickle Cell Pain Burden in Sub-Saharan Africa Despite Younger Patients Findings from the multinational SWAY survey highlighted significant disparities in sickle cell disease burden across global regions. Despite being younger on average, patients in sub-Saharan Africa experienced more frequent severe vaso-occlusive crises than patients in low-, middle-, and high-income countries. The results also underscored limited access to pain management and supportive therapies in these regions, emphasizing ongoing inequities in SCD care worldwide.

One-Time Gene-Editing Therapy Gives Patients “Freedom” From Pain Crisis An investigational gene-editing cell therapy, renizgamglogene autogedtemcel (reni-cel), continued to generate optimism in sickle cell disease treatment. Data presented in April showed that 27 of 28 treated patients reported no severe vaso-occlusive events following infusion, suggesting the one-time therapy may substantially reduce disease burden and dependence on recurrent medical interventions. Researchers described the therapy as offering some patients newfound “freedom” from pain crises and chronic disease management.