Description
Title: Debating Curative Therapies for Sickle Cell Disease: Gene Therapy vs Allogeneic Transplant
Presented By: Misty Evans, DNP, APRN, CPNP-AC, BMTCN
Overview of the Course: Presented at the 2025 Spring Education Exchange (SEE) this course presents that Sickle cell disease (SCD) is an inherited blood disorder caused by variants of the β-globin (HBB) gene, affecting approximately 300,000 people worldwide each year. This condition leads to complications such as pain from vaso-occlusive crises (VOCs), anemia, and end-organ damage, significantly impacting life expectancy and quality of life. Allogeneic hematopoietic stem cell transplant (HSCT) offers a curative option, particularly from HLA-identical sibling donors, but less than 20% of patients have a compatible sibling donor. Alternative donor options, such as haploidentical biological parents or siblings, have increased donor availability but come with risks like graft-versus-host disease (GVHD). Gene therapies have shown promising results in clinical trials, though long-term data and accessibility due to high costs remain challenges. This session will review the latest data on allogeneic HSCT and gene therapies as curative treatments for children, adolescents, and young adults with SCD.
This Course will Explore:
- The genetic basis and clinical complications of sickle cell disease
- Curative potential and limitations of allogeneic hematopoietic stem cell transplant (HSCT)
- The role and risks of alternative donors, including haploidentical family members
- Emerging gene therapy approaches, including current evidence and ongoing challenges
- Recent data on curative treatment options for children, adolescents, and young adults with SCD
Upon successful completion of this course, nurses will earn 1 NCPD hours associated with this activity.
Association for Pediatric Hematology Oncology Nurses is accredited as a provider of nursing continuing professional development by the American Nurses Credentialing Center’s Commission on Accreditation. Provider approved by the California Board of Registered Nursing, Provider Number 14513.
Individual Learning Needs Assessment (ILNA) Coding
The program content has been reviewed by the Oncology Nursing Certification Corporation (ONCC) and is acceptable for recertification points. You may claim 1 NCPD in one of the following ILNA categories: Disease Related Biology, Care of the Pediatric Hem/Onc Patient, Supportive Care, Palliative Care and Symptom Mgt, Pediatric Oncologic and Hematologic Emergencies, Pediatric Hem/Onc Nursing Practice, Treatment, Foundations of Transplant, Transplant Process and Infusion, Cellular Collection, Prep Regimen and Infusion, Early Post Transplant Management and Education, Late Post Transplant Management and Education, Quality of Life, Professional Performance
Please reach out to the Education Team at education@aphon.org with your name, the email associated with your APHON account, and the above ILNA category you would like to have reflected on your certificate, and APHON can update your completion certificate to reflect your choice.
Any questions? Contact education@aphon.org
