SCDF leads effort to use real-world patient data to transform SCD care
You Are a Marvel initiative aims for more personalized therapies, health equity
The Sickle Cell Disease Foundation (SCDF), in partnership with the Center for Inherited Blood Disorders (CIBD) and The Hills Tandem, is leading a real-world health data initiative that is progressing toward its goal of improving sickle cell disease (SCD) patients’ life quality and transforming their care.
The You Are a Marvel (YAAM) effort, created by and for people living with the blood disorder that affects an estimated 20 million people globally, emphasizes a holistic approach to patient-centered care and personalized medicine. It seeks to improve health outcomes in SCD by ensuring that patients’ voices and experiences are at the fore of research and innovation.
The initiative — a collaboration of patients, clinicians, investigators, community organizations, and other key stakeholders — involves the collection and initial analysis of patient-generated health data to promote patient empowerment and the development of personalized products and services.
At length, it seeks health equity for all members of underserved patient populations living with complex chronic conditions.
SCDF-led initiative aims to capture real-world experiences of SCD patients
“At its core, YAAM represents a shift in how we approach health care research and delivery,” Mary Brown, SCDF’s president and CEO, said in a press release from the organization.
“Inspired by the wisdom of cellist Pablo Casals, who recognized the innate marvel within each person, YAAM strives to honor each person touched by sickle cell disease as the marvel they truly are rather than just another data point,” Brown said. “In doing so, we aspire to transform the health care experience from one marked by commonality to one characterized by the uniqueness and dignity of each individual.”
The initiative was established in 2019 to collect and analyze patient-generated health data, allowing an accurate reflection of the real-world experiences of people living with SCD. Subsequently, it expanded its focus to identify prospective triggers of SCD pain crises in young adults. The idea is that if the occurrence of such crises can be predicted, their effects could be minimized or crises avoided altogether through timely therapeutic interventions.
These vaso-occlusive crises, or VOCs, occur when sickled red blood cells impede blood flow to the point that tissues become deprived of oxygen, sparking an inflammatory response as the body attempts a remedy. VOCs are the most common cause of SCD-related hospitalization and typically cause substantial pain. They can affect any part of the body, but often occur in the chest, back, or extremities.
YAAM already has reached significant milestones, completing two real-world data projects that include an SCDF-funded 15-patient quality improvement study and a 50-patient institutional review board-approved study sponsored by Pfizer, the SCDF reported. For each project, participants collected six weeks of real-world data covering biometrics, environmental influences, disease symptoms, and other relevant information.
Sickle cell patients had central role in design of You Are a Marvel effort
The larger Pfizer-sponsored study alone recorded 2,823 participant days and more than 73 million individual data points. Combined, the two datasets provide the basis of information required to discover pain crises triggers and other insights that can inform precision medicine approaches.
“We cannot overstate how important it is that this initiative was designed by and for individuals living with sickle cell disease who are highly motivated to collaborate on tools that can radically improve daily life as well as long-term health,” said Jennifer Fields, an SCD patient and the SCDF’s implementation strategist.
“As this initiative continues to unfold, we look forward to providing a new research model and software platform for clinical teams to learn more about their patients and ultimately improve quality of life and health outcomes beyond medications and treatments alone,” added Fields, who also is the founder and president of The Hills Tandem, a management consulting firm.
The SCDF is the nation’s oldest SCD community-based organization of its kind, while the CIBD provides healthcare services to people across southern California with inherited blood disorders.
For more information about the initiative, send an email to [email protected].
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