Episode 8:

John J. Strouse, MD, PhD, and Julie Kanter, MD, join guest host Ify Osunkwo, MD, of Levine Cancer Institute/Atrium Health in Charlotte, N.C., to discuss current therapies for sickle cell disease, as well as new treatments and cures under development.

Clinical Correlations:Ilana Yurkiewicz, MD, tackles informed consent and the capacity to choose in cancer care with a focus on the research setting. Dr. Yurkiewicz is a fellow in hematology and oncology at Stanford University and is a columnist for Hematology News. More from Dr. Yurkiewicz here.

 

 

SHOW NOTESBY EMILY BRYER, DO Resident in the department of internal medicine, University of Pennsylvania Health System.

Sickle cell disease is a multi-organ system vascular disease that affects both children and adults.

Pathophysiology in sickle cell disease includes increased expression of adhesion molecules resulting in vessel obstruction, activation of coagulation cascade and inflammatory cytokines, and a relative deficiency of nitric oxide that limits blood vessel dilatation. Universal newborn screening has translated into earlier diagnoses of sickle cell disease with improved outcomes.

Therapeutics for sickle cell disease are limited, with only two FDA approved medications:

1. Hydroxyurea (increases fetal hemoglobin) Decreases leukocyte counts, increases available nitric oxide.
2. Endari (L-glutamine) Helps to decrease inflammation in the blood cell and improve nitric oxide availability.
3. Blood transfusions are used conservatively.

Comprehensive Center (multidisciplinary team) remains the gold standard for sickle cell disease treatment, however many patients are limited by geographic location and/or by medical insurance.

 

EMBRACE (Education and Mentoring to Bring Access to CarE) Network:

• Collaboration of eight states in the southeastern United States to improve medical care of patients with sickle cell disease.
• Primary goals include increasing the use of hydroxyurea and improving outreach to providers using telemedicine.

New and emerging therapies for sickle cell disease:

• Selective inhibitors target the interaction between erythrocytes, leukocytes, and the endothelium inside of the blood vessel.
• Anticoagulants.
• Gene transplants and stem cell therapies to cure sickle cell disease and include both matched sibling donor transplant and half-matched donor transplant.

Resources: 1. Phase 3 study of L-glutamine (NCT01179217) 2. Sickle cell disease, a review (N Engl J Med 2017; 376:1561-73) 3. Gene therapy approaches to sickle cell disease (Blood 2016 127:839-48)

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