Interdisciplinary Care Model for Transition and Re-Transition after Gene Therapy in Sickle Cell Disease

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Introduction Hematopoietic cell transplantation (HCT)-based gene therapies (GT) have successfully demonstrated significant reduction in vasocclusive crises (VOCs) from sickle cell disease (SCD), leading to U.S. licensing of 2 gene therapy products in 2023 for patients ≥12 years of age with SCD. For patients, GT is characterized by significant unfamiliar adverse effects, the potential for persistent freedom from VOCs, and unknown long term outcomes. For both transplant and referring centers, GT also presents an unfamiliar logistical process compared to allogeneic HCT. These factors could potentially lead to deficiencies in medical care as well as high anxiety for patients and families. Objectives To develop a collaborative transition of care plan for patients and families prior to and long after completion of SCD gene therapy. Methods Upper Midwest Sickle Cell Curative Therapies Consortium is a collaborative model including multiple stakeholders between 7 centers across the upper Midwest United States, a regional community-based organization (CBO) and National Marrow Donor Program (NMDP). We developed a interdisciplinary care model in collaboration with community stakeholders. Results As part of comprehensive evaluation prior to GT, our shared collaborative checklist includes medical history, treatment plan, individualized pain plan and adherence, transfusion planning (simple or exchange; setting of target hemoglobin and hemoglobin S% targets), and involvement of apheresis, fertility preservation, child life, and social work. Pain, psychology, and integrative medicine specialists address ongoing concerns and help plan for future transition back to the primary site of SCD care (“re-transition”) after GT. Neuropsychological evaluations establish baseline emotional coping and cognitive function in the context of chronic fatigue and pain. When appropriate and available, warm handoffs are encouraged between centers within each of these specialties. CBOs offer available resources for education and ongoing support. In the post-GT transition back to primary SCD care and community, ongoing psychology, integrative medicine, pain medicine, peer mentoring, and support groups are critical. Using this interdisciplinary approach across GT clinical trials, we successfully weaned opioids in 83% of patients (5/6) by day 60 post-GT infusion and successfully re-transitioned these patients to primary SCD care. Ongoing follow up by sickle cell and transplant specialists, CBO, and other supportive services for both patients and their families continues into post-GT survivorship. Conclusion As new therapies emerge for transformative SCD care, development of a model for early engagement of an interdisciplinary team is critical for empowering sickle cell warriors and for their successful transition back into the community.

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