GENE THERAPY for sickle cell disease is widely supported by United States paediatric haematologists, yet real-world implementation remains inconsistent due to knowledge gaps, referral variability, and insurance barriers. These findings were presented as a research poster at the 52nd Annual Meeting of the EBMT.
Gene Therapy Adoption and Referral Patterns
The survey, conducted between July–September 2025, included 138 respondents providing care to paediatric patients with sickle cell disease. Most clinicians reported strong intent to refer eligible patients for gene therapy, with 86% indicating they were likely or very likely to do so. However, referral decisions varied significantly depending on clinical scenarios and provider specialisation. General haematologists were more likely to consider gene therapy for patients with silent cerebral infarcts compared with specialists (63% versus 39%; p=0.01), as well as for cerebral vasculopathy (59% versus 37%; p=0.04), despite reporting lower familiarity with gene therapy protocols.
Institutional Readiness and Knowledge Gaps
Institutional preparedness for gene therapy integration was uneven. While 77.5% of respondents had access to allogeneic bone marrow transplant services, only 68.8% reported capacity for gene therapy onboarding. Notably, just 18% routinely discussed specific gene therapy products during referrals. Among those who did, one product was strongly preferred by 94% of respondents, although many acknowledged limited comparative evidence to support this choice. Confidence in counselling patients also varied. Seventy percent of specialists focused on sickle cell disease felt comfortable discussing gene therapy, compared with 32% of general haematologists (p<0.001). Clinicians reported the greatest uncertainty regarding long-term risks such as toxicity, clonal haematopoiesis, and secondary malignancies, as well as differences in therapeutic mechanisms.
Insurance Barriers and Global Implications
Insurance approval emerged as the most influential factor in referral decisions, cited by 72% of respondents. This outweighed considerations such as disease severity, caregiver readiness, or institutional infrastructure. Free-text responses highlighted additional challenges including unclear eligibility criteria, inconsistent payer policies, and limited patient education resources.
These data suggest that enthusiasm for gene therapy is high, but translation into practice remains constrained. While European healthcare systems may mitigate some structural barriers, issues such as variable clinician knowledge, lack of standardised shared decision-making protocols, and the need for long-term safety data are likely to persist. Coordinated efforts in education, policy alignment, and clinical infrastructure will be essential to ensure equitable access and effective integration of gene therapy into patient care.
Reference
Karsenty C et al. Bridging readiness and reality: U.S. pediatric hematologists’ perspectives on gene therapy for sickle cell disease in the post-approval era. Abstract A282. EBMT 52nd Annual Meeting; 22-25 March 2026.
