Care in Context: From Individual Decisions to System-Level Solutions in Pediatric Hematology
"I can give all the recommendations I want for iron deficiency," says Madhav Vissa, MD, a pediatrician in the UCSF Division of Pediatric Hematology. "But a child's developmental stage, family dynamics, and life situation all shape what's actually possible."
He’s describing a familiar challenge: treatments that appear straightforward on paper but are more complex in practice. Kid-friendly iron formulations often aren’t available by prescription, leaving families to pay out of pocket or rely on options children may refuse to take.
Barriers that make treatment plans difficult to sustain can also prevent patients with chronic illnesses from accessing the specialty care they need. Population estimates suggest several hundred young people with sickle cell disease in Northern California may not be receiving consistent follow-up. How can they be identified and reconnected to care?
Tracking Who's in Care — and Who Isn't
At UCSF, Vissa is establishing the infrastructure to answer that question.
Working with a data engineer and an Epic analyst, he helped create a patient registry of everyone with sickle cell disease who has sought care at UCSF – home to one of the largest and longest-running sickle cell programs in the country. Built within the electronic medical record, the registry creates a unified and actionable view of this patient population, supporting both large-scale research and proactive clinical follow-up.
This work also introduced a new medical record template for individualized pain plans, with updates captured in a searchable, easy-to-reference format. Instead of searching long chart notes for care plan updates, the pediatric hematology team can now instantly see when patients need their plans reviewed.
The longer-term vision extends beyond UCSF. By connecting registry infrastructure with community hospitals across the Bay Area, Vissa aims to identify patients who present to emergency departments without ongoing specialty care – and link them back to consistent care.
Making Complex Care Work in Practice
Building these systems can change the course of a child’s health. When patients do remain connected to care, the clinical relationship can deepen into a strong partnership. In thalassemia care, patients return monthly for transfusions, creating consistent touch points that build familiarity.
“The more you understand a patient’s day-to-day reality, the better your recommendations become,” Vissa says. “When families see those work, it builds trust and makes it easier to adjust care together over time.”
That kind of connection shapes how families navigate treatment decisions and how children stay involved with care as they transition to adulthood. It’s also what the registry work is designed to support: ensuring that more patients, including those not yet connected to UCSF, can access and remain in specialty care.
More Options, Greater Need for Connection
Treatment options for sickle cell disease and thalassemia are rapidly expanding. Even a decade ago, children with these diseases were left without options besides lifelong disease management. Today, multiple gene therapies and disease-modifying therapies – including landmark UCSF-led trials – have transformed this landscape. Vissa includes clinical trial eligibility assessments in every patient note, ensuring each family has access to the full range of options.
More options don’t simplify care. Clinicians must help patients and families weigh the transformative potential of these therapies against their associated challenges.
“There’s a lot we can do right now to make patients’ lives easier, but only if they remain connected to care,” Vissa says. “Otherwise, advances won’t reach all the patients who could benefit.”
Learn more about the UCSF Division of Pediatric Hematology and its approach to improving the lives of children with sickle cell disease, thalassemia, and other blood disorders.