New Hemophilia Drugs Let Kids Surf, Kickbox, Live Fully

Two brothers who once bruised so easily they were covered in black and blue marks now surf, ride dirt bikes, and practice jujitsu near their home in Kauai, Hawaii. Their transformation shows how new hemophilia treatments are giving families hope that seemed impossible just a decade ago.

Kapono, 18, and Duke, 13, were diagnosed with hemophilia soon after birth. Their mother Sara Pavao feared the blood-clotting disorder would leave them disfigured or severely limit their lives. As young children, they bruised constantly from simple bumps and everyday activities.

Hemophilia affects roughly 30,000 Americans who lack crucial proteins needed for blood to clot properly. Without treatment, many children end up in wheelchairs from repeated bleeding into their joints. The genetic condition mostly affects males because it's transmitted on the X chromosome.

For decades, treatment meant replacing the missing clotting factor through frequent intravenous infusions every few days. Patients had to carefully manage peaks and troughs in protection. About a third of people with severe hemophilia developed antibodies that made the medicines stop working entirely.

The new class of medicines called nonfactor therapies works differently. They help blood clot without replacing the missing factor at all. Some require only a shot every few weeks instead of constant infusions, and they provide steady protection around the clock.

The Bright Side

Clinical trials show these drugs reduce bleeding events by up to 96 percent compared to no preventive treatment. Doctors at Children's Hospital of Philadelphia and Children's Hospital of Los Angeles call it one of the biggest success stories in medicine. The treatments are highly effective even in people with severe forms of the disease.

Gene therapy is adding another option for some adults. A single treatment allows certain patients to produce their own clotting factor, though results vary and doctors are still learning how long the benefits last.

The difference shows up in everyday life. The Pavao family used to travel with a whole suitcase full of medicines. Now their drugs fit in a small box. Both boys can participate in sports that would have been unthinkable for hemophilia patients just decades ago.

Leslie Raffini, who treats children with hemophilia, emphasizes the medicines don't eliminate bleeding risk entirely. But they've transformed what's possible for families living with this condition.

Sara Pavao says her sons are living the full life she always wanted for them. They're proof that medical breakthroughs can turn fear into freedom.