Margaret V. Ragni, MD, MPH

Dr. Ragni has actively pursued, initiated, collaborated, and participated in clinical translational research in congenital hemostasis and thrombosis disorders, serving as chair of clinical trials, prospective epidemiologic, observational, case-control studies, cost-effectiveness analyses, and investigator-initiated new drug trials in hemophilia and VWD. Her research studies were among the first multi-center NIH-funded investigator-initiated studies in hemophilia malignancy (NCI), hemophilia inhibitor formation (NHLBI), hemophilia HIV/HCV infection (NHLBI), hemophilia AIDS therapy (NIAID), and hemophilia adult prophylaxis (NHLBI). She co-chaired the NHLBI State of the Science (SOS) Inhibitor Prevention Trial Working Group (2018), and serves as PI of the Inhibitor Prevention and Eradication Platform Trial (HRSA). She also co-chaired the NHLBI SOS Hemophilia & VWD Subcommittee to design future trials, with three U34 trials funded NHLBI, and the VWDMin NHLBI U01 clinical trial to reduce bleeding in women with VWD, and the VWD-Woman trial prevent VWD postpartum bleeding (Takeda). She has collaborated on multi-center organ transplant HIV trials (NIAID), hemophilia gene therapy trials (NHLBI); VWD genotype-phenotype studies (NHLBI); novel therapeutics (fitusiran (siRNA AT) and hemlibra (emcizumab) and extended half-life protein trials (VIIa, VIII, IX) for hemophilia, and rhIL-11 and recombinant VWF for VWD. She has 9 active NIH grants, two of which she serves as PI; and local PI on 6 ATHN CDC grants.

As the Medical Director of the Hemophilia Center of Western Pennsylvania (HCWP), I lead a team that provides medical care to 500+ patients with congenital bleeding and clotting disorders and also direct the Center’s 340B HRSA (Health Resources Services Administration) clotting factor program. Our staff include a genetic counselor, physical therapist, psychosocial counselors, clinic and research nurses, administrative and support staff who provide comprehensive management of patients and their families affected by these rare diseases. Students, residents and fellows routinely rotate through our clinic, learning how gaps in care can be addressed by research, which is a critical component of this clinic. The overarching goal is to provide state-of-the-art care to affected individuals and their families, integrating research and quality initiatives to improve and optimize care and push science forward. We also provide national and international consultation to many patients and caregivers, and completed a 3-year program in a World Federation of Hemophilia-supported Twinning Program with a hemophilia center in Tanzania, to teach, share knowledge and resources, and hands on care. We also actively support and provide teaching at World Federation of Hemophilia (WFH) programs including for novel therapies, gene therapy, and global planning. HCWP has been supported by long-standing federal and state grants from the CDC/ATHN Hemophilia Treatment Center Region III and the Pennsylvania Department of Health.

Dr. Ragni served as 2021 Chair of NHF Research Summit/Infrastructure Committee; the 2018 Co-Chair NHLBI SoS Inhibitor Clinical Trials Working Group. She served as Co-Chair of the American Society of Hematology (ASH) Education Program in 2014; the Co-Chair of the 2014 ASH Annual Meeting Education Program. She lectures extensively, including for a symposia focusing on novel therapeutics in hemophilia and von Willebrand disease. Dr. Ragni has served as principal investigator of an NHLBI T35 training grant for medical students in hematology research, in its 15th year (75 students). She is a long-term member of the National Hemophilia Foundation Medical Advisory Committee (MASAC) which discusses and writes medical advisories for physicians and patient community on novel therapeutics and guidelines for bleeding disorders. She also serve on the Advisory Board of the Foundation for Women and Girls with Blood Disorders, which plans and gives talks, education, seminars, and webinars on women with bleeding disorders. Dr. Ragni actively mentors students, residents, and fellows in clinical and research projects in benign hematology. She has served as past Chair, Curriculum Committee, School of Medicine, directed the Medicine, Ethics, Society course in the Medical School, and currently on the Division's Curriculum Committee and serves as a Board member of the Hemophilia Research Society (HTRS), serving as Co-Chair of the 2021 Scientific Symposium. She currently mentors T32, K23, and CRTI trainees.