Sickle Cell Treatment, Part I

Thursday, March 21, 2019

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 NOTES
BY 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)

Contact us: podcasts@mdedge.com
Twitter: @MDedgeHemOnc

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David Henry, MD
David Henry, MD, FACP, is a clinical professor of medicine at the University of Pennsylvania and vice chairman of the department of medicine at Pennsylvania Hospital in Philadelphia. He received his bachelor’s degree from Princeton University and his MD from the University of Pennsylvania, then completed his internship, residency, and fellowship at the Hospital of the University of Pennsylvania. After 2 years as an attending in the U.S. Air Force, he was drawn to practicing as a hem-onc because of the close patient contact and interaction, and his belief that, win or lose with each patient, one can always make a difference in their care and lives. Follow Dr. Henry on Twitter: @davidhenrymd. Dr. Henry reported being on the advisory board for Amgen, AMAG Pharmaceuticals, and Pharmacosmos. He reported institutional funding from the National Institutes of Health and FibroGen.



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