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Department of Medicine

Department of Medicine

  Division of Hematology/Oncology

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photo Margaret V. Ragni, MD, MPH

Professor of Medicine, Division Hematology/Oncology

Director, Hemophilia Center of Western PA

Email: ragni@dom.pitt.edu

Phone: 412-209-7288

Contact
Office: Hemophilia Center WPA,3636 Blvd of the Allies
Pittsburgh, PA 15213
 
Phone: 412-209-7288
Fax: 412-209-7281
E-mail: ragni@dom.pitt.edu
Education and Training
Education
BS, Chatham College, Pittsburgh, PA, 1971
MD, University Pittsburgh School of Medicine, 1975
MPH, Graduate School of Public Health, 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. My 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). I served as Co-Chair State of the NHLBI Science SOS Hemophilia & VWD Subcommittee to design future trials, with three U34 trials funded NHLBI, and three R01 clinical trials in preparation, including one to prevent inhibitors in hemophilia A, one to reduce VWD menorrhagia, and one to prevent VWD postpartum bleeding. I have collaborated on multi-center organ transplant HIV trials (NIAID), hemophilia gene therapy trials (NHLBI); VWD genotype-phenotype studies (NHLBI); novel therapeutics (siRNA-AT3, emcizumab (ACE910) and extended half-life protein trials (VIIa, VIII, IX) for hemophilia, and rhIL-11 and recombinant VWF for VWD.
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 of 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 initiates to improve and optimize care and push science forward. We also provide national and international consultation to many patients and caregivers, and we participate in a World Federation of Hemophilia-supported Twinning Program with a hemophilia center in Tanzania, sharing knowledge, resources, and hands on care. HCWP has been supported by long-standing federal and state grants from the CDC/ATHN Hemophilia Treatment Center Region III and the Pennsylvania Health Department.
Educational Interest
As co-Chair of the American Society of Hematology (ASH) Annual Meeting Education Program in 2014, I had
the opportunity to create a program which was also adapted to 3-day programs in South America and Asia. I serve as speaker for ASH & FDA-new drug symposia, and for symposia focusing on novel therapeutics in hemophilia and von Willebrand disease. I have served as principal investigator of an NIH T35 training grant to train medical students in clinical research (pending renewal). I am a long-term member of the National Hemophilia Foundation Medical Advisory Committee (MASAC) which discusses and writes medical advisories for physicians and patient community in the area of rare bleeding disorders. I also serve on the Advisory Board of the Foundation for Women and Girls with Blood Disorders, which plans, executes, and distributes talks, education, seminars, and webinars for women and their providers. I mentor students, residents, and fellows in these clinical research projects. I served as past Chair, Curriculum Committee, School of Medicine, directed the Medicine, Ethics, Society course in the Medical School, and teach basic coagulation and hemostasis to medical students, and am a current member of the Curriculum Committee for the Division Hematology/Oncology Fellowship Program.
Publications
For my complete bibliography, Click Here.
Selected Publications:
Powell J, Pasi J, Ragni M, Ozelo M, Valentino L, Mahlangu J, Manco-Johnson M, Josephson N, Perry D. Phase 3 study of long-acting recombinant factor IX Fc. N Engl J Med, provisional acceptance pending response to comments. 2013.
Mannucci PM, Kempton C, Miller C, Romond E, Shapiro A, Birschmann I, Ragni MV, Gill JC, Yee TT. Pharmacokinetics and safety of a nevel recombinant human von Willebrand factor manufactured with a plasma-free method: a multicenter prospective clinical trial. Blood. 2013.
Ragni MV, Novelli E, Murshed A, Merricks EP, Kloos M, NIchols TM. Phase II clinical trial of recombinant interleukin-11 (rhIL-11, Neumega) in DDAVP-unresponsive von Willebrand disease and mild hemophilia A. Thrombosis Haemostasis. 2013; 109: 248-254.
Ragni MV, DeVera M, Roland ME, Wong MT, Stosor V, Sherman KE, Hardy D, Blumberg E, Fung F. Liver transplant outcomes in HIV-infected hemophilic men. Haemophilia. 2013; 19: 134-140.
Pishko A, Smith KJ, Ragni MV. Anticoagulation in ambulatory cancer patients: A cost-effectiveness analysis. Thrombosis Haemostasis. 2012; 108: 303-310.
Novelli EM, Huynh C, Gladwin MT, Moore CG, Ragni MV. Pulmonary embolism in sickle cell disease: A case-control study. Journal of Thrombosis Haemostasis. 2012; 10: 760-766.
Terrault nA, Roland ME, Schiano T, Dove L, Wong MT, Poordad F, Ragni MV, Burin B, Simon D. Outcomes of liver transplantation in HCV-HIV co-infnected recipients. Liver Transplant. 2012; 18: 716-726.
Novelli EM, Kato GJ, Ragni MV, Zhang Y, Hildesheim ME, Nouraie M, Barge S, Meyer MP, Cortese-Hassett A. Plasma thrombospondin-1 is increased in sickle cell disease and associated with hydroxyurea use, lower rates of hemolysis, and increased vaso-oclusive episodes. American Journal of Hematology. 2012; 87: 326-330.
Powell JS, Josephson NJ, Quon D, Ragni MV, Cheng G, McKinney B, Jiang H, Li L, Dumont JA. Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients. Blood. 2012; 119: 3031-3037.
Shapiro AD, Ragni MV, Valentino L, Key NS, Josephson NC, Powell JS, Cheng G, Thrompson A, Goyal J. Recombinant factor IX Fc fusion (rFIXFc) demonstrates safety and prolonged activity in hemophilia B patients. Blood. 2012; 119: 666-672.
Sponsored Research/Activities
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: Gene-Therapy, Open-Label, Dose-Escalation Study of SPK-9001
Role: Principal Investigator
Funding Agency: Spark Therapeutics
Start Year: 2015
End Year: 2017
Title: ALN-AT3SC-001 A Phase 1 Single-ascending and Multiple-ascending Dose, Safety, Tolerability and Pharmacokinetics Study of Subcutaneously Administered ALN-AT3SC in Healthy Adult Volunteers and Hemophilia A or B Patients
Role: Principal Investigator
Funding Agency: Anylan Pharmaceuticals
Grant Number: DRUG
Start Year: 2015
End Year: 2017
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: Feasibility of the Von Willebrand Disease Minimize Trial
Role: Principal Investigator
Funding Agency: National Heart, Lung, & Blood Institute
Grant Number: U34 HL119582
Start Year: 2013
End Year: 2016
Title: Zimmerman Program for the Molecular and Clinical Biology of VWD
Role: Principal Investigator
Funding Agency: Blood Center of Wisconsin/National Heart, Lung, & Blood Institute
Grant Number: P01 HL081588
Start Year: 2012
End Year: 2017
Title: A Phase I Safety Study in Subjects with Severe Hemophilia B (Factor IX Deficiency) Using a Single-Stranded, Adeno-Associated Pseudotype 8 Viral Vector to Deliver the Gene for Human Factor IX (AAV8-hFIX19-101)
Role: Principal Investigator
Funding Agency: Spark Therapeutics
Grant Number: DRUG
Start Year: 2012
End Year: 2016
Title: Feasibility of the Hemophilia INHIBIT Trial
Role: Principal Investigator
Funding Agency: National Heart, Lung, & Blood Institute
Grant Number: U34 HL114674
Start Year: 2012
End Year: 2015
Title: Pathophysiology of Anti-Factor VIII Inhibitor Development in Patients with Hemophilia A Undergoing Surgery
Role: Principal Investigator
Funding Agency: Emory University/National Heart, Lung, & Blood Institute
Grant Number: K23 HL105785
Start Year: 2011
End Year: 2016
Title: Hemophilia Adult Prophylaxis Study
Role: Principal Investigator
Funding Agency: National Heart, Lung, & Blood Institute
Grant Number: R34 HL105870
Start Year: 2011
End Year: 2015
Title: A Phase 1 Safety Study in Subjects with Severe Hemophilia B (Factor IX Deficiency) Using Adeno-Associated Viral Vector ("AAV Vector") to Deliver the Gene for Human Factor IX into the Liver Coupled with Transient Immunomodulation
Role: Principal Investigator
Funding Agency: Spark Therapeutics
Grant Number: DRUG
Start Year: 2009
End Year: 2016
Title: Extended Safety Evaluation in Subjects Who Have Participated in Chiron Technologies Center for Gene Therapy Retrovirus Protocols
Role: Principal Investigator
Funding Agency: Novartis Pharmaceutical
Grant Number: DRUG
Start Year: 2002
End Year: 2015
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 of Hematology, 2016
Medical Student Research Mentoring Merit Award, University of Pittsburgh, 2016
Editoiral Board, Associate Editor, Blood Advances, 2016