Bhavya Doshi, MD, a pediatric hematologist at the Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta and assistant professor at Emory University School of Medicine, doesn’t have to look far for inspiration. She finds it in the children and families she cares for every day. While she is fascinated by new scientific advances in treating hemophilia, it’s the relationships she builds with patients that drive her to keep pushing for novel therapies.

In addition to seeing patients in clinic, Dr. Doshi is a researcher, so she can clearly see the connection between the bench and the bedside. She is a physician scientist with expertise in basic and translational approaches to improving diagnostics and therapeutics for patients with bleeding and clotting disorders. She has special interest in creating therapies that work for more patients by focusing on overcoming the challenges faced by patients with hemophilia A who develop factor VIII (FVIII) inhibitors.

Revolution in Hemophilia Care

People with hemophilia are dealing with a complex genetic disorder that makes it hard for their blood to clot. It is considered a lifelong, chronic disease. There is no cure, but treatments are available. Dr. Doshi has seen incredible progress in the time she has been in practice.

“I spent years taking care of kids who didn’t have therapies, but now there is a revolution in hemophilia care,” she said. “There are treatments that can take people from severe to mild. It is so much better than when I started my first hemophilia project in 2010.”

While treatments have improved, Dr. Doshi knows there is still work to be done. Her research focuses on one of the biggest challenges in hemophilia care: why some patients develop antibodies that make treatments less effective. About 20–30% of children with severe hemophilia A develop these antibodies, called FVIII inhibitors, early in life, limiting therapies that can help them.

Dr. Doshi’s lab examines how the immune system responds to factor replacement therapy and gene therapy, looking for ways to predict who will develop inhibitors and how to prevent them. By identifying specific immune pathways and B-cell responses, her team hopes to design targeted treatments for these patients.

Inspired By Families Living with Hemophilia

For Dr. Doshi, this work is personal because of the relationships she’s built with patients and their families over the years. Dr. Doshi trained at Children’s and Emory, then practiced at Children’s Hospital of Philadelphia for nine years. When she came back to Children’s and Emory last year, she saw some familiar faces. “It was heartwarming to see patients I saw as a resident in clinic are still here,” she said. “Because hemophilia tends to run in families, we frequently get to know multiple family members. We build strong connections.”

Seeing these patients in clinic invigorates Dr. Doshi’s research efforts. She understands the burden that having limited therapies places on patients and clinicians and gets to know the children who could benefit from the diagnostics and therapies she and her colleagues are working to create. Getting to see and treat these patients firsthand is one of the reasons she chose to come back to Children’s and Emory.

“Because of the volume of patients we see here, we really have a pulse on what is happening with patients,” she said. “The team that cares for patients here is fantastic, with a shared common goal of making our patients’ lives better. We are all focused on how to improve the lives of people with hemophilia.”

Opening Doors to Future Therapies

As gene therapies and new treatments emerge, Dr. Doshi is working to make sure more children can benefit. “As we discover better treatments for kids with hemophilia, we could also unlock treatments for other genetic disorders we don’t have answers for yet,” she said. “There are more than 6,000 genetic disorders we know about, and 90% are diagnosed in kids.”

In other words, it is a time of tremendous opportunity—for researchers and for patients. It’s also a time for collaboration between researchers. Collaboration is another of the reasons Dr. Doshi came back to Children’s and Emory. “The concentration of expertise on campus is tremendous,” she said. “It’s really nice to be surrounded by so many researchers. We have these spiraling conversations that lead to new ideas.”

By bringing questions from the bedside into the lab—and discoveries from the lab back to patients—Dr. Doshi and her colleagues are helping create a future where children with hemophilia have more treatment choices and brighter possibilities.

Some of Dr. Doshi's most recent publications include: