A New Standard of Care: UCSF's Leadership in the Fight Against Sickle Cell Disease
For thousands of children and adults living with sickle cell disease, every day brings the possibility of intense pain, organ damage, and life-threatening complications. At UCSF, we’re rewriting that story.
Leading this work is the UCSF Sickle Cell Center of Excellence and its co-director, Marsha Treadwell, PhD, a professor of pediatrics in UCSF’s Division of Pediatric Hematology. Through her leadership roles, she links state, regional, and national efforts to create networks of care and set new standards for patient-centered treatment.
The Courage of Sickle Cell "Warriors"
SCD was a disease of childhood until the 1970s. Since then, the average life expectancy has improved from 10 years to 50 years and many live much longer, but individuals with SCD still have their lives disrupted by severe pain episodes, and ongoing sickling of the red blood cells can permanently damage their bodies. These realities are why many in the community ask to be called “warriors,” a term that reflects their courage and strength.
“Because SCD is a rare disease in the United States, progress must be made in collaboration with other sickle cell specialists, advocates, and researchers,” says Treadwell. “And it takes targeted outreach to healthcare providers to ensure they’re knowledgeable about care guidelines and preventative measures.”
Weaving a System of Care
UCSF Benioff Children’s Hospital Oakland has an over 50-year legacy of transforming sickle cell care and research, led by pioneers like Elliott Vichinsky, MD, and Bert Lubin, MD. Carrying that tradition forward with a vision for system-wide change, Treadwell creates powerful synergies between federal and state programs.
Treadwell is a co-Principal Investigator for the federally funded Pacific Sickle Cell Regional Collaborative, a 13-state network that supports provider training and tests new models for delivering care.
Each participating state was required to create a state-level action plan, which she helped lead in California. “We brought together warriors, clinicians, and community partners in a year-long process to identify key strategies for improving the health of Californians with SCD,” Treadwell explains.
That process culminated in the strategic blueprint for the Networking California for Sickle Cell Care (NCSCC) initiative. This project has built a cohesive network of specialized SCD centers and community organizations across California. It expands the healthcare workforce’s knowledge of SCD and makes high-quality care more accessible, with the goal of improving the health of adults with SCD and reducing emergency room visits.
Leading a New Standard of Care
At the request of the NCSCC, the UCSF Sickle Cell Center of Excellence created the Standard of Care Guidelines for SCD. The comprehensive guidelines compile evidence-based recommendations from national and international sources and incorporate holistic practices derived from UCSF’s decades of experience.
“Clinicians appreciate the wealth of information provided in one document, from health maintenance to screening and curative therapies,” says Treadwell. “I am particularly proud that the Guidelines include algorithms for screening and management of depression, anxiety, and neurocognitive challenges — a largely unmet need for the population.”
Treadwell’s work to ensure access to SCD-informed care extends to the national level; she was on the inaugural Executive Committee for the National Alliance of Sickle Cell Centers and remains on its board. The alliance promotes the nationwide adoption of SCD care standards, and other UCSF sickle cell experts are leading consensus-building efforts focused on improving treatment of specific complications.
A Blueprint for the Future
Central to Treadwell’s work is the input of community advisors: patients and family members with lived experience. She emphasizes that their participation is essential to build trust and design programs that are truly relevant. “We want to co-create health interventions that are not only effective, but also respectful and sustainable,” she says.
Nowhere is this more critical than in helping families navigate the new, potentially curative gene therapies recently approved by the FDA. To that end, Treadwell was part of a 50-person team of scientists, physicians, and warriors recruited by the National Human Genome Research Institute for its Democratizing Education for Sickle Cell Disease Gene Therapy Project, which created accessible materials to help families make informed decisions about these new treatments.
This UCSF-led approach of connecting federal policy, state funding, academic research, and community wisdom offers an effective framework for the future and other rare, complex conditions. By building an equitable system of care from the ground up, this work provides hope for patients that they’re finally being heard.
Learn more about how we’re transforming SCD outcomes and quality of life on the UCSF Sickle Cell Center of Excellence website.