HemAware is conducting a series of interviews with recipients of the NHF-Shire Clinical Fellowship. Funded through the generous support of Shire, the program strives to increase the number of skilled clinicians committed to providing comprehensive care for people with bleeding disorders. It provides hands-on clinical training for physicians at hemophilia treatment centers (HTCs) across the country.
This interview was conducted with Leslie J. Raffini, MD, medical director of the Comprehensive Hemostasis and Thrombosis Center at The Children’s Hospital of Philadelphia (CHOP). She is also associate professor at the University of Pennsylvania (Penn) in Philadelphia. Her mentor was Catherine Manno, MD, former director of the HTC at CHOP. The fellowship was funded from 2003–2005.
Why did you decide to study medicine?
As an undergraduate student at the University of Wisconsin-Madison, Raffini toyed with the idea of becoming a basic scientist. After a pediatric hematologist-oncologist gave a lecture and slideshow of his patients in a virology course she was taking, Raffini’s future was determined. “I made a connection about how neat it would be to be able to enjoy the science part, but also be involved in clinical care,” she says.
What initially attracted you to hematology?
Raffini’s interest in oncology segued into hematology during her hematology-oncology fellowship. But it wasn’t until she began a master’s in clinical epidemiology program at Penn that she got the guidance she needed. Manno, then director of the HTC at CHOP, helped Raffini identify research projects to pursue. “Then she asked me if I was interested in starting a pediatric thrombosis program here at CHOP,” Raffini says. “That’s really how things got going.”
How did the training and mentorship you received as an NHF-Shire Clinical Fellow affect your decision to pursue clinical care and/or research in bleeding disorders?
“It allowed me to have another two years of funded and protected time to develop real clinical expertise in hemostasis and thrombosis,” Raffini says. During that time, she initiated several research projects and got the thrombosis program off the ground. “We were seeing more and more patients with clotting problems at CHOP, but didn’t have a consistent approach or program.” Raffini drafted recommendations for patient care and collected data. “We were able to use that to publish several articles and as preliminary data for NIH grant funding,” she says.
Are you still engaged in the clinical aspects of patient care or bleeding disorders research? What aspect of care are you most interested in?
In 2008, Raffini was appointed director of the CHOP HTC. Since then, she says, she’s had one foot firmly planted in each world. “I spend about 50% of my time doing clinical research in both bleeding and clotting disorders, and 50% of my time doing patient care.”
Her research interests run the gamut, from new bleeding disorders drugs, to preventing catheter-associated thrombosis in children with chronic conditions. “I’m very interested and excited about the new long-acting factor products for patients with hemophilia,” Raffini says. “I’m equally as interested in the new blood thinners for children—the new oral anticoagulants.” CHOP is participating in clinical trials for both.
Did your NHF-Shire Clinical Fellowship assist in advancing your own position at your institution? Or did it serve as a building block to further your career in coagulation?
“It served as a building block, allowing me to gain the expertise,” Raffini says. She was one of the first two fellows to receive the fellowship nearly a decade ago. “It was a great opportunity,” she says.
Where do you think bleeding disorders research and clinical care may be headed in the immediate future? Within the next decade?
Raffini endorses the recent advancements made using gene therapy techniques to treat hemophilia B, factor IX deficiency. She hopes these will lead to such approaches to treat hemophilia A, factor VIII deficiency. She will be the clinical principal investigator for an upcoming study at CHOP on gene therapy for hemophilia B patients. The study was developed by Katherine A. High, MD.
“In the next decade, there is going to be a lot of progress with gene therapy,” says Raffini. She also predicts improved care for some difficult-to-treat people. “I think we’ll start to understand how to reduce the risk of inhibitors in our patients.”
When you are not working, how do you relax or “escape” from your work?
Raffini enjoys traveling with her husband and 9-year-old daughter. She stays active by training for triathlons. “Most days, I’m either swimming, biking or running,” she says. This summer, she’s registered for an Olympic triathlon in Philadelphia. But she draws the line when describing her level of involvement. “‘Compete’ might be a strong word. I participate,” she says with a laugh.
How to apply for the NHF-Shire Clinical Fellowship.