There have been very few new treatments developed for patients with sickle cell disease since Hydroxyurea was first introduced in the early 1980s. Essentially in isolation, Endari (L-glutamine), indicated to reduce acute complications of sickle cell disease, became available to patients approximately 2 years ago.
However, within the past year, several new pharmacologic agents have been introduced and gene therapy is gaining momentum as a viable option for the treatment of patients with sickle cell disease. We will specifically discuss Adakveo (crizanlizumab), a monoclonal antibody targeted towards P-selectin, and Oxbryta (voxelotor), a hemoglobin S polymerization inhibitor. We will also discuss the advent of gene therapy in sickle cell disease, in particular the use of gene editing, which uses the patient's own stem cells that have been harvested, genetically modified, and then transplanted back into the patient. Bedside nurses and providers are often the first line of family education and encouragement. This interactive session will allow for Q and A and discussion regarding how to introduce these treatment modalities into everyday disease management discussions with patients and their families.
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- Robin Pitts, C-FNP MN BSN CPHON®
- CNE Hours: