Medical Director and Associate Investigator Lynn Malec, MD, MSc, is committed to providing the best care for patients at Versiti Comprehensive Center for Bleeding Disorders.
Just weeks after he was born, 11-year-old Zach Fechter was diagnosed with severe hemophilia A, a rare and incurable bleeding disorder. Shortly after his diagnosis, Zach and his family were referred to Versiti Comprehensive Center for Bleeding Disorders (CCBD), which provided them with medical and supportive care and taught Zach’s parents how to give him factor product infusions. These subcutaneous injections replace the missing factor protein in Zach’s blood that helps it to clot. And as he grew up, Zach learned how to self-infuse his factor product, helping him feel more confident and independent.
Zach is lucky: his factor infusions work and help promote blood clotting. But some patients with bleeding and clotting disorders who receive regular factor infusions develop antibodies to the medication, which prevents the treatment from working. CCBD hematologist and medical director Lynn Malec, MD, MSc, focuses some of her clinical research on hemophilia inhibitors and is closely following how some patients react to a new product called factor VIII-Fc fusion protein (rFVIIIFc). The idea is that patients with inhibitors are given high doses of rFVIIIFc with the hope that, over time, it will induce immune tolerance. “If we can better understand that, we can further approach immune tolerance,” she said.
To dive deeper into this area of research, Dr. Malec is partnering with Versiti Blood Research Institute Investigator Weiguo Cui, MD, PhD, an immunologist, and Associate Investigator Sid Rao, MD, PhD, an expert in genetics. Together, they hope to better understand why immune tolerance works to get rid of inhibitors—or why, in some patients, it doesn’t. “Versiti Blood Research Institute has critical mass—we have really bright, dedicated people,” she said. “Collaboration from a clinical and research interface is part of what we do.”
Dr. Malec’s research also extends to patients with blood disorders who experience bleeding in their index joints, or elbows, knees and ankles. Without proper treatment, these patients can develop arthritis and loss of range of motion. “We can have teenage patients who, if they don’t have good bleeding control, can experience loss of function,” Dr. Malec said. “In some cases, these young people need to consider joint replacement or joint surgery.” The trouble is, hardware wears out and young patients who opt for joint surgery or replacement will likely need it again later in life.
Dr. Malec is hoping to combat this by implementing point-of-care ultrasounds for patients with bleeding and clotting disorders. These ultrasounds will give hematologists a better look at patients’ joints over time and help avoid expensive tests and treatments like MRIs and radiation.
Young patients like Zach Fechter who are already being treated at the CCBD are perfectly positioned to benefit from this forward thinking. “We have a hemophilia population that is aging in a way that a generation or two ago didn’t age because people have had more access to therapies,” Dr. Malec said.
“With the help of the CCBD and the education we’ve received, Zach’s future is very bright,” Zach’s mom Tracie said.
Lynn Malec, MD, MSc, is the medical director for Versiti Comprehensive Center for Bleeding Disorders and an associate investigator at Versiti Blood Research Institute.