Department of Medicine
Faculty Profiles by Division

Department of Medicine

Faculty Profiles

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photo Margaret V. Ragni, MD, MPH

Hematology/Oncology

Professor of Medicine and Clinical and Translational Research, Division Hematology/Oncology

Director, Hemophilia Center of Western PA

Email: ragni@pitt.edu

Phone: 412-209-7288

Contact
Office: Hemophilia Center Western PA
3636 Blvd of the Allies
Pittsburgh, PA 15213
 
Phone: 412-209-7288
Fax: 412-209-7281
E-mail: ragni@pitt.edu
Administrative Assistant:
Devera Hoblak
Address: Hemophilia Center WPA
3636 Blvd of the Allies
Pittsburgh, PA 15213
Email: hoblakd@pitt.edu
Phone: 412-209-7288
Fax: 412-209-7281
Education and Training
Education
BS, Chatham College, Pittsburgh, PA, 1971
MD, University Pittsburgh School of Medicine, 1975
MPH, Graduate School of Public Health, University of Pittsburgh, 1993
Training
Medical Internship/ Residency, University of Pittsburgh Medical Center, 1978
Fellowship, Hematology, University of Pittsburgh School of Medicine, 1981
Coagulation Hematology, Central Blood Bank, Pittsburgh, PA, 1982
Research Interest
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.
Clinical Interest
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.
Educational Interest
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.
Publications
For my complete bibliography, Click Here.
Selected Publications:
Ragni, M.V. Hemophilia as a blueprint for gene therapy. Science. 2021; 374(6563).: 40-41.
George, L.A., Monahan, P.E., Eyster, M.E., Sullivan, S.K., Ragni, M. V. Multiyear stable factor VIII expression after AAV gene transfer for hemophilia A. New England Journal of Medicine. 2021; 385: 1961-1973.
Sadler, B., Minard, C.G., Haller, G., Gurnett, C.A., O'Brien, S.H., Wheeler, A., Jain, S., Sharma, M., Aziz, B., Kulkarni, R., Mullins, E., Ragni, M.V., Sidonio, R., Dietrich, J.E., Kouides, P.A., Di Paola, J., Srivaths, L. Whole exome analysis of adolescents with low VWF identifies novel underlying genetic factors. Blood. 2021; 137: 3277-3283.
Pasi, K.J., Lissitchkov, T., Mamonov, V., Mant, T., Timofeeva, M., Bagot, C., Chowdary, P., Pencho, G., Gercheva-Kyuchokova, L., Madigan, K., Nguyen, H., Yu, Q., Mei, B., Benson, C., Ragni, M.V. Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran - Results of the phase inhibitor cohort. Journal of Thrombosis and Haemostasis. 2021; 19: 1436-1446.
Reipert, B.M., Gandadharan, B., Berg, V., Schweiger, H., Scheiflinger, F., Bowen, J., Blatny, J., Fijnvandraat, K., Gruppo, R., Klintman, J., Male, C., McGuinn, C., Meeks, S.L., Radulescu, V.C., Ragni, M.V., Recht, M., Santagostino, E., Shapiro, A.D., Staber, J.M., Yaish, H.M. The prospective hemophilia inhibitor PUP study (HIPS) reveals distinct signatures prior to FVIII inhibitor development. Blood Advances. 2020; 4: 5785-5796.
Bertolet MH, Brooks MM, Ragni MV. The design for an adaptive trials platform trial to prevent and eradicate inhibitors in patients with hemophilia. Blood Advances. 2020; 4: 5433-5444.
Konkle, B.A., Shapiro, A.D., Quon, D.V., Staber, J.M., Suzuki, T., Kulkarni, R., Ragni, M.V., Chhabra, E., Poloskey, S., Rice, K., Katragadda, S., Rudin, D., Freebies, J., Benson, C. BIVV))1 fusion protein as factor VIII replacement therapy for hemophilia A. New England Journal of Medicine. 2020; 383: 1018-1027.
Machin, N., Ragni, M.V. Recombinant vs plasma-derived von Willebrand factor to prevent postpartum hemorrhage in von Willebrand disease. Blood Advances. 2020; 4: 3234-3238.
Seaman, C.D., Ragni, M.V. The effects of age on von Willebrand factor and bleeding symptoms in von Willebrand disease. Thrombosis Haemostasis. 2020; 120: 1159-1165.
Ragni, M.V., Berntorp, E., Carcao, M., Escuriola, C., Nedzinskas, A., Ouzel, M.C., Preza, E.D., Selvaggi, A., van den Berg, H.M., Srivastava, A. Inhibitors to Clotting Factor: WFH Guidelines. Haemophilia. 2020; 26(Suppl 6): 95-107.
Sponsored Research/Activities
Title: Hemophilia Inhibitor Prevention and Eradication (INHIBIT) Trials Platform
Role: Principal Investigator
Funding Agency: Hemophilia Center of Western PA / Health Research and Services Administration
Grant Number: CONTRACT xxx416932xxx
Start Year: 2020
End Year: 2021
Title: Dose-finding study of SPK-8016 gene therapy in patients with hemophilia A to support future evaluations in individuals with FVIII
Role: Principal Investigator
Funding Agency: Spark Therapeutics
Start Year: 2019
End Year: 2022
Title: A Multicenter Study of Eradication of Hepatitis C Virus (HCV) Infection in People with Hemophilia and Bleeding Disorders (ATHN 6: HCV Eradication Study)
Role: Principal Investigator
Funding Agency: American Thrombosis and Hemostasis Network
Start Year: 2019
End Year: 2021
Title: A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII at a dose of 4E13 vg/kg in Hemophilia A Patients (270-302)
Role: Principal Investigator
Funding Agency: Biomarin Pharmaceuticals
Grant Number: RES
Start Year: 2018
End Year: 2023
Title: A Prospective Non-Interventional Study of Bleeding Episodes, Factor VIII Infusions, and Patient-Reported Outcomes in Individuals with Severe Hemophilia A (BMN 270-902)
Role: Principal Investigator
Funding Agency: Biomarin Pharmaceuticals
Grant Number: RES
Start Year: 2018
End Year: 2022
Title: SPK-8011-LTFU; A Multi-Center Evaluation of the Long-Term Safety and Efficacy of SPK-8011 (Adeno-Associated Viral Vector with B-Domain Deleted Human Factor VIII Gene) in Males with Hemophilia A
Role: Principal Investigator
Funding Agency: Spark Therapeutics
Grant Number: RES
Start Year: 2018
End Year: 2021
Title: Von Willebrand Disease Minimize Menorrhagia (VWDMin) Trial
Role: Principal Investigator
Funding Agency: National Heart, Lung, & Blood Institute
Grant Number: U01 HL133815
Start Year: 2017
End Year: 2022
Title: A Phase I/II, Open-Label, Adaptive, Dose -Ranging Study to Assess the Safety and Tolerability of SB-525 [AAV2/6 hFactor VIII Gene Therapy] in Adult Subjects with Severe Hemophilia A
Role: Principal Investigator
Funding Agency: Sangamo Therapeutics
Start Year: 2017
End Year: 2021
Title: Training Students in Biomedical Research Hematology
Role: Principal Investigator
Funding Agency: National Heart, Lung, & Blood Institute
Grant Number: HL074708
Start Year: 2017
End Year: 2021
Title: A Long-Term Follow-Up Study in Subjects with Severe Hemophilia B (Factor IX Deficiency) Who Received a Single-Stranded, Adeno-Associated Pseudotype 8 Viral Vector to Deliver the Gene for Human Factor IX (AAV8-hFIX19)
Role: Principal Investigator
Funding Agency: Spark Therapeutics
Grant Number: RES
Start Year: 2017
End Year: 2019
Title: Genotype and Phenotype Analysis of Adolescents with Heavy Menstrual Bleeding and Low Von Willerbrand Activity
Role: Principal Investigator
Funding Agency: Baylor College of Medicine
Grant Number: RES
Start Year: 2017
End Year: 2019
Title: Gene-transfer, Open-Label, Dose-Escalation Study of SPK-98011 [Adeno-Associated Viral Vector with B-Domain Deleted Human Factor VIII Gene] in Individuals with Hemophilia A
Role: Principal Investigator
Funding Agency: Spark Therapeutics
Grant Number: RES
Start Year: 2017
End Year: 2019
Title: Natural History Study of Factor IX Treatment and Complications (B-Natural)
Role: Principal Investigator
Funding Agency: Rho, Incorporated
Start Year: 2017
End Year: 2019
Title: Design of Less Immunogenic Factor VIII Proteins
Role: Principal Investigator
Funding Agency: Henry M. Jackson Foundation
Grant Number: R01 HL130448
Start Year: 2017
End Year: 2019
Title: Hepatitis C Virus (HCV) Outcomes after Treatment with DAA in Patients with Bleeding Disorders (ATH5: Outcomes Study)
Role: Principal Investigator
Funding Agency: American Thrombosis and Hemostasis Network
Grant Number: RES
Start Year: 2016
End Year: 2020
Title: A Phase I/II Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) rh10-Mediated Gene Transfer of Human Factor IX in Adults with Moderate/Severe to Severe Hemophilia B
Role: Principal Investigator
Funding Agency: PPD Development Corporation
Grant Number: DRUG
Start Year: 2016
End Year: 2019
Title: ALN-AT3SC-002 An Open Label Extension Study of Subcutaneously Administered ALN-AT3SC in Subjects with Moderate or Severe Hemophilia A or B who Have Completed a Previous Clinical Study with ALN-AT3SC
Role: Principal Investigator
Funding Agency: Anylan Pharmaceuticals
Start Year: 2016
End Year: 2018
Title: Comparative Effectiveness in the Diagnosis of VWD
Role: Principal Investigator
Funding Agency: Blood Center of Wisconsin/National Institute of Health
Grant Number: R01 HL112614
Start Year: 2014
End Year: 2018
Title: Hemophilia Inhibitor Response to Eloctate (HIRE Study)
Role: Co-Principal Investigator
Funding Agency: Blood Center of Wisconsin
Start Year: 2017
End Year: 2020
Title: DREAM AWARD: Longitudinal Ultrasound Evaluation of Joint Pathology in Severe Hemophilia (LONG-US)
Role: Co-Principal Investigator
Funding Agency: Blood Center of Wisconsin
Start Year: 2017
End Year: 2019
Notable Achievements
Fellow, American College of Physicians, 1985
Distinguished Alumna Award, Chatham College Alumna Association, 1989
Kenneth E. Schuit Award, Dean's Master Educator, University of Pittsburgh School of Medicine, 1998
Kenneth M. Brinkhous Award for Excellence in Clinical Research, National Hemophilia Foundation, 1998
G. David Roodman Excellence in Mentoring Award, University of Pittsburgh, Division of Hematology/Oncology, 2014
Murray Thelin Researcher of the Year Award, National Hemophilia Foundation, 2015
Women Leaders in Hematology, American Journal Hematology, 2016
Medical Student Research Mentoring Merit Award, University of Pittsburgh, 2016
Editorial Board, Associate Editor, Blood Advances, 2016
Maxwell Wintrobe Endowed Lectureship Internal Medicine, University Utah, 2017