National Academy of Medicine Elects Alexis Thompson, MD, MPH

By Kate Knab

For Alexis Thompson, MD, MPH, Chief of the Division of Hematology and Elias Schwartz, MD, Endowed Chair in Hematology at Children’s Hospital of Philadelphia, the question driving her research passions has always been: “How can we combine scientific discovery and high quality medical care to impact the largest number of people?”

In answer to this, Dr. Thompson has dedicated her career to advocating for bringing cutting-edge treatments and technology to sickle cell disease (SCD) patients locally and globally to help improve their quality of life. She brings hematological expertise as a member of CHOP’s Sickle Cell and Red Cell Disorders Curative Therapy Center (CuRED), which is one of a select group of treatment centers offering gene therapies to treat SCD. Her clinical research on the lentiviral vector therapy lovotibeglogene autotemcel for severe sickle cell disease also led to the approvals of the first two gene therapies for SCD.

The National Academy of Medicine (NAM) elected Dr. Thompson in recognition of her achievements in the field.

“The National Academy of Medicine is a prestigious organization that often provides a compelling lens on so many areas of policy, medicine, and science,” Dr. Thompson said. “It’s an extraordinary honor to be elected.”

Navigating a Global Impact

As a trainee of the late Kwaku Ohene-Frempong, MD, Dr. Thompson took to heart Dr. Ohene Frempong’s mission to deliver quality care on a global scale. He was the first investigator to establish a newborn screening program for SCD in his native country of Ghana, which is still in operation today. Dr. Thompson continues these efforts by collaborating with colleagues in Western Africa where SCD is a prominent health challenge.

Her goal is to provide a level of care to patients in Western Africa that children might receive in West Philadelphia. Screening babies, diagnosing them early, and getting them into clinics that have access to treatments could be life-changing for some SCD patients who otherwise may not live past 5-years-old. She strives to share her knowledge of the tools that scientists know already work and provide educational opportunities for families to ensure children all over the world will benefit from advances in SCD research.

“We can have a global impact by moving discovery science forward, just as we can be sure that there are children who are receiving the best possible basic care,” Dr. Thompson said.

Bringing Community to SCD Research

While she was president of the American Society for Hematology (ASH), Dr. Thompson saw an opportunity to advocate for a learning community to benefit SCD research. Originally spearheaded by the Institute of Medicine, the precursor to NAM, learning communities are research collaborations driven by patient and family questions related to a specific disease. The goal is to balance the need to pursue new knowledge and advance science with the responsibility to ensure the largest number of people benefit from this research. Funding from the US Department of Health and Human Services allowed Dr. Thompson to establish the first national learning community for SCD.

Researchers selected topics to improve both short- and long-term health outcomes that resonated with patients, including efficient pain management and accessibility of disease-modifying therapies. Because patient voices and concerns are at the core of learning communities, potential research topics grow and change around what families find the most important to learn.

Investigators have been studying how to improve the transition from pediatric to adult care for SCD patients by taking a systems perspective and analyzing it alongside patient and provider-level interventions. Although the program launched prior to Dr. Thompson’s arrival in Philadelphia, CHOP is now one of the lead sites for the SCD learning communities. 

“It’s gratifying to find new ways to improve patient care through a different lens beyond the usual clinical trials or bench discoveries,” Dr. Thompson said. “I hope we will continue to have the patient’s voice in mind as we seek to improve their outcomes and transform care.”

Driving Discovery Through Big Data

Dr. Thompson was also fundamental to the development of the ASH Research Collaborative Data Hub, which has grown into the largest single-source SCD data repository. The Data Hub integrates electronic health record data and contains information from more than 25,000 patients. Researchers and clinicians can view broad datasets collected from x-rays and lab studies, alongside patient-level observations to dig deeper into improving the health and wellness of people with SCD. 

“Harnessing big data to drive improvements in care is the future,” Dr. Thompson said. “There’s so much work to be done, and there continue to be additional opportunities to make treatments safer and more effective. It is rewarding to be in an environment like CHOP that has always been at the forefront of these kinds of initiatives.” 

Under Dr. Thompson’s guidance, CHOP became a flagship site for the Data Hub, contributing more than 1,000 SCD patient samples. This is the first year that investigators will be able to report out scientific abstracts using SCD data collected since its launch in 2018.

During the ASH Annual Meeting in December, Dr. Thompson and colleagues will give an oral presentation on the information collected in the Data Hub on patients across the US, and a second oral abstract presentation on a CHOP-led study that resulted in the creation of innovative personalized pain management plans for CHOP patients with SCD inspired by the SCD Learning Community. 

“Dr. Thompson is one of CHOP’s most esteemed and visionary clinicians and investigators, whose passion and leadership continue to drive transformative, life-saving solutions for children,” said Joseph St. Geme, MD, Physician-in-Chief and Chair of the Department of Pediatrics at CHOP. “Her unmatched expertise and forward-thinking have elevated CHOP, not just as a provider of exceptional pediatric care, but also as a leader in solving the most complex pediatric health challenges.”