Hemophilia Treatment Center Marks 55 Years
This year marks the 55th anniversary of Cincinnati Children’s Comprehensive Hemophilia Treatment Center. Ralph Gruppo, MD, retired director of the Hemophilia Program, wrote an account of the center’s history to commemorate the occasion. The following are excerpts from his recollections.
The story of the Cincinnati Children’s Comprehensive Hemophilia Treatment Center is remarkable because of the changes that have occurred that now make the lives of persons with bleeding disorders better than ever before. It is also remarkable because of the heart-breaking tragedy that occurred along the way with the development of blood-borne infections, including hepatitis virus and HIV, but which ultimately led to improved treatments through medical research. It is the story of numerous patients who unselfishly participated in research trials with the goal to improve hemophilia care, and who faced the future with unbridled faith and courage. And lastly, it is the story of the many patients, families, and medical caregivers, who have worked, laughed, and cried together while they have taken this journey together. I am proud to be part of this story.
A Grim Prognosis
Patients with hemophilia are deficient in either clotting factor VIII or IX. Classically, they experience multiple muscle and joint bleeds, leading to chronic muscle or joint damage. Prior to the 1960s, many children with severe hemophilia became physically disabled as early as 3-5 years of age. Mainly, whole blood or plasma was used to help stop bleeding. These products were not very efficient in raising the level of the deficient coagulation factor in the blood to control bleeding. They also required administration in the emergency room or hospital. Patients frequently required prolonged hospitalizations for bleeding episodes. Prior to 1960 the life expectancy for those with severe hemophilia was less than 20 years. During this time there were very few medical centers in the US that specialized in the care of hemophilia patients. Healthcare providers focused more on the disease than on comprehensive care.
The impact of hemophilia on the whole family was enormous. At that time, patients would experience severe bleeds weekly or even more frequently. To treat bleeding episodes, families had to call the hematologist, travel to the emergency room where they would meet the hematology fellow, who would infuse the patient with cryo or plasma, often for several consecutive days. Many times, bleeding episodes required a hospitalization. Because the process was so disruptive to a family’s life, patients would often wait to see if the bleed resolved on its own. It was hard for families to plan events, such as baptisms, weddings, or vacations. School absences were often prolonged. Bleeds always seemed to occur during the worst possible times, in the middle of the night or during family events.
Click on the timeline below to read about important developments in the history of Cincinnati Children's HTC:
1966 - Cincinnati Children's Hemophilia Treatment Center (HTC) Is Established
Cincinnati Children's was the first to pilot an HTC in the state of Ohio, under the direction of Alvin Mauer, MD.
1974 – HTC Gets New Director
Ralph Gruppo, MD, took over as director of the HTC with support from Bea Lampkin, MD, director of Hematology/Oncology.
1970s – Home Infusion Treatment Program Reduces ED Visits and Hospitalizations
Freeze-dried concentrates made home infusion more practical but introduced risks of hepatitis.
Mid 1970s – The First Annual Children’s Hemophilia Summer Camp Begins in Cincinnati
Offered free of charge, the camp experience helped kids with hemophilia feel like they weren't so different after all.
1981 – HIV is discovered in hemophilia patients
The emergence of AIDS devastated the hemophilia population and isolated them further from the community.
1985 - The first HIV test becomes available
Discovery of the virus that caused HIV helped make the blood supply safe and led to increasingly effective treatment of the disease.
1990s – Development of Recombinant Clotting Factors
New DNA technology led to safer therapies and less frequent infusions.
1990s – Gene Therapy Trials Begin
Preliminary results in human trials of gene therapy for hemophilia have yielded encouraging results, but it gets complicated.
1972-2018 – Brief History of the Special Research Coagulation Laboratory
Collaboration with UC Health and Cincinnati Children's Division of Human Genetics resulted in the development of a panel of genetic tests for a rare clotting disorder.
Our hemostasis and thrombosis program, including the HTC, and the Hemostasis and Thrombosis Laboratory at Cincinnati Children’s would not have continued to expand and thrive without the contributions of several exceptionally talented and dedicated pediatric hematologists. I have great confidence in these individuals to carry our program into the future. I owe immense gratitude to Dr. Cristina Tarango, currently associate professor at Cincinnati Children’s. Dr. Tarango assumed directorship of our hemophilia program prior to my retirement in 2018. Under her leadership, our program has continued to excel clinically, as well as taking a leadership role within the national network of HTCs.
I am also particularly indebted to Dr. Eric Mullins, associate professor at Cincinnati Children’s and director of research for our hemophilia program. He developed the clinic for young women with bleeding disorders in collaboration with the Division of Adolescent Medicine. He has assumed a leadership role in our HTC’s participation in the development of therapeutic products for bleeding disorders, as well as participating in the gene therapy clinical trials.
Our Hemostasis and Thrombosis Laboratory has also continued to expand and thrive under the distinguished joint leadership of Dr. Lori Luchtman-Jones and Dr. Joseph Palumbo, co-directors of the laboratory. Dr. Luchtman-Jones is professor of pediatrics at Cincinnati Children’s and has had a particular clinical interest in thrombotic disorders and stroke in children. She, along with Dr. Palumbo, has taken a leadership role in the supervision and expansion of the hemostasis laboratory.
In summary, the last 55 years have seen enormous progress in the care of persons with hemophilia and other bleeding and thrombotic disorders. Until cures are available, this story will continue to evolve. Our journey over these last 55 years has taught us many things. We have learned the importance of medical research if treatment is to progress. So too, we have learned to appreciate the optimism and self-sacrifice of the numerous patients and families who have volunteered to participate in clinical research studies, without whom no progress is possible. We have learned the value of multi-disciplinary comprehensive care for children with hemophilia and other rare diseases of blood coagulation and that true comprehensive care must focus on the whole person, as well as the family. Treatment at a comprehensive HTC has proven to be associated with improved medical outcomes in persons with bleeding disorders. The national network of HTCs has demonstrated the value of collaboration with other centers and provides a rich resource for research opportunities in hemophilia and other rare coagulation disorders. Lastly, we learned that patients, families, and medical caregivers must support each other and work together to continue to lobby for affordable, accessible, quality care for everyone with a coagulation disorder. We can overcome any obstacle or setback, no matter how serious, when we continue this journey together.
Ralph Gruppo, MD June, 2021
Ralph Gruppo, MD, (r) catches up with Erik Mullins, MD, director of research for the Hemophilia program.
Ralph Gruppo, MD, presents Bea Lampkin, MD, with the Jefferson Award in this undated photo.
Showing off their quality improvement work for the National Hemophilia Foundation in collaboration with the Emergency Department in 2019.
The HTC team celebrates World Hemophilia Day 2019.