The Rare Disease Report Podcast: Sickle Cell Disease

Podcast

In this first episode, Kim Smith-Whitley, MD, discusses the history, presentations, and research status of sickle cell disease.

Quicks Facts about Sickle Cell Disease

  • Approximately 100,000 Americans are affected
  • Occurs in 1 out of every 365 Black or African-American births
  • Occurs in 1 out of every 16,300 Hispanic-American births
  • HbSS is the most severe form of sickle cell disease, called sickle cell anemia
  • Average life-span for males with HbSS is 42 years old
  • Average life-span for females with HbSS is 48 years old
  • Pain crises are the most common presentations of the disease

Quotes of Interest

"Historically, we used to wait for problems to happen before children with sickle cell disease were diagnosed."
"It's just striking to me that we're still doing supportive care for pain and acute chest syndrome."
"You don't have to have a sickle gene in order to have a child with sickle cell disease."
"There are so many therapies in the pipeline that will be able to benefit individuals with sickle cell disease."
"Every organ is involved in complications both short-term and long-term in individuals with sickle cell disease."

“When I think about sickle cell disease, I can’t help but think about sickle cell in a historic perspective,” said Kim Smith-Whitley, MD,  Executive Vice President-Elect of Global Blood Therapeutics, in the inaugural episode of the Rare Disease Report podcast.

The history of sickle cell disease is the history of healthcare disparity—as it primarily affects individuals of color and historically under-resourced populations.

Even more, the sickle cell story involves the push for increased universal screening, improved therapeutic options, and perhaps more pertinently, the need to develop effective ways to manage pain crises.

To understand sickle cell care in the United States is to appreciate the diagnostic and treatment milestones, while acknowledging the remaining—and urgent—gaps that need to be addressed by healthcare professionals and researchers.

Drawing from her clinical experiences as a pediatric hematologist, Smith-Whitley painted a detailed and comprehensive portrait of the reality of sickle cell care, the toll the disease can take on families, as well as the current and future state of research.

She emphasized the importance of trust between patients, their families, and their care team, which can ultimately prove foundational to improving patient quality of life.

HCPLive® and Rare Disease Report® are proud to present the first installment of a new podcast series that will provide a platform for experts and advocates to share their knowledge, tell their story, and augment the discussion surrounding diseases that have—for too long—fallen beneath the radar.

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