New Precision Approaches to Pediatric Hemophilia Care at Children’s Hospital Los Angeles
According to a Newswise profile of Sibgha Zaheer, MD, an attending physician and investigator in CHLA's Cancer and Blood Disease Institute, we are now in what she describes as "an exciting time" for hemophilia care.
A pediatric hematologist at Children's Hospital Los Angeles is quietly reshaping how we approach treatment selection for children with hemophilia and other bleeding disorders — and her work could change the conversation at your next clinic visit.
Why the hemophilia toolkit finally looks different
Several newly approved therapies target the body's natural anticoagulants rather than simply replacing missing clotting factors, and they are delivered as regular subcutaneous injections — meaning families no longer need to anchor a child's life around an IV infusion schedule. For clinicians managing pediatric cases, this is the practical shift to know: the choice of agent can now be matched to the patient, not the other way around.
What "personalized" actually means at the bedside
Dr. Zaheer's group is building the laboratory evidence base that will let us tailor that choice with more confidence. She and her team are using patient plasma samples — including from children on emicizumab for hemophilia A — to model what happens to the coagulation cascade when a medication is held, switched, or layered with another agent such as fitusiran, measuring outcomes with thrombin generation assays. The clinical goal, as she frames it, is straightforward: pick a regimen based on how comfortable your child is with injections, how frequently they want to be dosed, and what level of bleed protection their daily life demands. She has also joined CHLA's Hematology-Adolescent Medicine Clinic, extending this individualized lens to teens presenting with heavy menstrual bleeding and other bleeding concerns.
What families and clinicians should watch next
Two adjacent developments make this a moment worth tracking closely. The U.S. Food and Drug Administration has approved Pharming's Joenja as the first treatment for children with activated PI3K delta syndrome (APDS) in the United States, expanding the menu of disease-specific options for rare pediatric immune disorders — though, as with any newly approved indication, long-term safety data will take time to mature. At the policy level, the Children's Hospital Association has flagged that proposed changes to federal grant regulations could disrupt pediatric research pipelines; a continuing resolution has temporarily paused the rule through December 11, 2026, but the underlying uncertainty around funding remains. For families already navigating a bleeding disorder diagnosis, the practical takeaway is this: ask your care team what injection-frequency options exist, what laboratory monitoring is in place for any medication switch, and whether your child's center is participating in the studies generating those answers — because the personalization Dr. Zaheer describes is built on exactly those conversations.