“You Can Always Pay Forward” ~ Woody Hayes
American Society of Hematology
ASH is the world's largest professional society concerned with the causes and treatments of blood disorders.
By Kyle Davis, MD, Pediatric Hematologist Oncologist, Indiana Hemophilia & Thrombosis Center
"Flash. Blood fills the thin tubing of a 25-gauge needle before injecting factor VIII, and so begins a day in the life of a pediatric resident with hemophilia A. As long as I can remember, seeing my blood has represented a small achievement for having successfully aimed a needle into one of my veins.”
Rereading the above words of my fellowship application, I reflect on the many new treatments for patients with bleeding disorders. Being an optimist, I reframed my chronic condition into a career as a pediatric hematologist and now serve on the research leadership team at the Indiana Hemophilia & Thrombosis Center in Indianapolis.?
From my perspective, there are three broad generations of people with hemophilia living today: those who were alive in the early 1980s, who were directly impacted by HIV/AIDS; those born in the 1990s prior to the implementation of prophylactic hemophilia treatment; and those born in the late 2010s, who have access to the subcutaneous non-factor therapy emicizumab. Patients with hemophilia unfortunately helped the world learn that HIV/AIDS could be transmitted through blood and factor transfusions. Treatment for their hemophilia would ultimately end their lives early. Nearly all of my mentors in the hemophilia community died of HIV/AIDS, hepatitis C, or bleeding complications due to lack of safe treatment.??
As a rambunctious kid born in 1988 with a condition that should have limited my activities, my parents found ways to channel my boundless energy into safer sports. Despite that, I was often on crutches at school, wore a never-ending series of bruises on my forehead, and received many factor infusions in our local ER. By the mid-’90s, research showed that prophylactic factor therapy could limit bleeding risk and protect young joints from chronic damage. This damage would otherwise lead to immobility as early as age 10 for patients without access to factor. Families like mine learned to stick their hemophilia nurses’ veins and then brought those skills home to treat their children with every-other-day IV infusions. By age 15, I felt guilty that my dad was waking up at 5:30 a.m. to give me my factor before school, so I learned to give myself my own IVs.??
In 2011, the first brave patient with hemophilia received a gene therapy treatment that used their liver cells to produce missing factor. A decade later, the technology has improved such that the first gene therapy was approved for patients with hemophilia.? 2017, another revolution occurred in hemophilia with the introduction of emicizumab. This incredible drug, a modified antibody that mimics factor VIII, spares the veins of young patients, provides long-acting coverage from bleeding, and can be used in patients who have inhibitors. Patients can be started on emicizumab within their first year of life, and many of these young patients have not experienced bleeding after starting the medication.? This would have been unthinkable when I was born.??
Thirty-five years ago, people like me were at risk of dying of tainted blood products. Now, in the 2020s, young patients may not have had a single painful, debilitating bleed, and they are expected to live full, successful lives.??
While hemophilia has shaped my life, my unique personal experience allows me to better relate to patients with similar conditions. Thankfully, due to the ready availability of safe treatment, I have absolutely no limitations in my work as a pediatric hematologist. I am driven to take the great care I received and pay it forward to the next generation of children with bleeding disorders.