Dr. Michael Callaghan: February 26, 2018


** Rare Disease Day is February 28 **


Dr. Michael Callaghan

Physician who treats hemophilia A with factor VIII inhibitors



Ken, a 50-year-old living with hemophilia A with factor VIII inhibitors


Hemophilia A is a rare genetic blood disorder in which a person’s blood does not clot properly, leading to uncontrolled and often spontaneous bleeding that causes chronic swelling, deformity, reduced mobility and long-term damage in the joints.1,2 Nearly one in three people with severe hemophilia A can develop inhibitors that prevent the standard treatment from working. This limits treatment options and can put them at greater risk for life-threatening bleeds and repeated bleeds.3

In November, the U.S. Food and Drug Administration (FDA) approved HEMLIBRA® (emicizumab-kxwh), a new preventative treatment option for hemophilia A with inhibitors that was shown to substantially reduce bleeds in adults and children. It’s the only treatment option that can be self-administered once-weekly by injection under the skin (subcutaneously). Current treatments may require frequent infusions up to three times a week.4

In recognition of Rare Disease Day (Feb. 28) and Bleeding Disorders Awareness Month (March), on February 26, hematologist Dr. Michael Callaghan will talk about why this new medicine is an important advancement and what it could mean for people who have hemophilia A with inhibitors. Dr. Callaghan will be joined by Ken – a 50-year-old who was diagnosed at age 5 and who received the medicine in a clinical study – who can share his inspirational story.

It’s important to remember that every patient is different, so they should speak with their doctor about treatment options and side effects. HEMLIBRA may cause serious side effects when used with aPCC (FEIBA®), including thrombotic microangiopathy (TMA) and blood clots (thrombotic events). Cases of thrombotic microangiopathy and thrombotic events were reported when on average a cumulative amount of >100 U/kg/24 hours of activated prothrombin complex concentrate (aPCC) was administered for 24 hours or more to patients receiving HEMLIBRA prophylaxis.4



Dr. Michael Callaghan, M.D., is a board-certified hematologist who specializes in pediatric hematology-oncology. He is affiliated with multiple hospitals in the Detroit area, including Children’s Hospital of Michigan and DMC Harper University Hospital. He is also an assistant professor of Pediatrics at Wayne State University.


Ken Martin, 50 years old, was diagnosed with hemophilia A when he was 2 years old, and a lifetime of serious and chronic bleeds have left him with severe joint pain and limited mobility. Three years after his diagnosis, Ken developed an inhibitor, which made managing the disorder even more challenging. Throughout his childhood, Ken was unable to be very active because of painful bleeds into his joints that required visits to the hospital. He enrolled in a clinical trial to help advance a new treatment so others “wouldn’t have to go through what he’s gone through.” Since taking Hemlibra, Ken has experienced very few bleeds and hasn’t had to keep his crutches or wheelchair nearby. Ken says having a treatment that works well “takes a lot of the worry out of walking out the door in the morning.”

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