Department of Medicine

University of Pittsburgh

Margaret V. Ragni, MD, MPH


Professor of Medicine, Division Hematology/Oncology

Director, Hemophilia Center of Western PA

Office: Hemophilia Center WPA,3636 Blvd of the Allies
Pittsburgh, PA 15213
Phone: 412-209-7288
Fax: 412-209-7281

BS, Chatham College, Pittsburgh, PA, 1971
MD, University Pittsburgh School of Medicine, 1975
MPH, Graduate School of Public Health, 1993

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

Area of Specialization/Research Interest  
Epidemiology of HIV/HCV infection in hemophilia and liver disease progression, liver transplantation
Immunogenetics of hemophilia inhibitors and prevention of inhibitors
Dendritic cell tolerance in hemophilia mouse and inhibitor formation
Genetics and clinical phenotype of VWD
New treatments for Von Willebrand Disease
Thrombophilia and duration/management anticoagulation
F.VIII, VWF, F.IX and cardiovascular risk in aging hemophiliacs
Bleeding disorders in women
VWD in children

Dr. Ragni is Professor of Medicine and Clinical Translational Science, Division Hematology/Oncology and Director, Hemophilia Center of Western PA. She is PI on a T35 student research grant and collaborates on an NHLBI genotype-phenotypes von Willebrand study; an NIAID-funded organ transplantation in HIV, an NHLBI-funded hemophilia gene transfer, and an NHLBI-funded Transfusion Medicine/Hemostasis clinical trials network. She is a member of the Medical and Scientific Advisory Board, National Hemophilia Foundation and of the NHLBI Hemostasis & Thrombosis study section. She chaired the Hemophilia/VWD Subcommittee of the NHLBI State of the Science Symposium 2009, developing four clinical trial concepts, three submitted. She is a member of the ASH Development Committee, Contributing Editor to The Hemologist, and on the Editorial Board of Haemophilia. She was nominated to FDA Blood Products Advisory Committee and serves as Co-chair, Hemostasis Thrombosis Research Society Research Committee.

Recent Grants
Title: R01 Comparative Effectiveness in Diagnosis of VWD
Role: Co-Investigator
Amount: $80,000 direct
Funding Agency:
Start Year: 2013
End Year: 2018
Title: AAV8-hFIX-101 Gene Transfer in Patients with Hemophilia B
Role: Co-Investigator, Local PI
Amount: $200,928
Funding Agency: Center for Molecular & Cellular Biology, Penn
Start Year: 2012
End Year: 2014
Title: U01 Cross-Sectional Analysis of Cardiovascular Disease in the Hemophilia Population
Role: Co-Investigator
Amount: $ 37,000 direct
Funding Agency: Centers for Disease Control (CDC)
Start Year: 2012
End Year: 2014
Title: U34 Feasibility of Hemophilia INHIBIT Trial
Role: PI
Amount: $459,000
Funding Agency: NHLBI
Start Year: 2012
End Year: 2014
Title: R34 Hemophilia Adult Prophylaxis Study
Role: PI
Amount: $450,000 direct
Funding Agency: NHLBI
Start Year: 2011
End Year: 2013
Title: R01 Phase I/II AAV - 2/8 Gene Transfer IX in Hemophilia B
Role: Local PI
Amount: $19,906 direct
Funding Agency: NHLBI, St. Jude
Start Year: 2010
End Year: 2013
Title: 2-P01 Molecular and Clinical Understanding of Von Willebrand Disease
Role: Co-Investigator, local PI on Multicenter Grant
Amount: $165,745 direct
Funding Agency: NHLBI - Program Project Grant Specialized Center of Clinically Oriented Research
Start Year: 2005
End Year: 2017
Title: 2-T35 Training Students in Biomedical Research in Hematology
Role: PI
Amount: $198,100 direct
Funding Agency: NHLBI
Start Year: 2005
End Year: 2016

Recent Peer-Reviewed Publications
Title: Phase II clinical trial of recombinant interleukin-11 (rhIL-11, Neumega) in DDAVP-unresponsive von Willebrand disease and mild hemophilia A.
Author(s): Ragni MV, Novelli E, Murshed A, Merricks EP, Kloos M, NIchols TM
Year: 2013
Journal: Thrombosis Haemostasis
Volume: 109
Page(s): 248-254
Title: Phase 3 study of long-acting recombinant factor IX Fc
Author(s): Powell J, Pasi J, Ragni M, Ozelo M, Valentino L, Mahlangu J, Manco-Johnson M, Josephson N, Perry D
Year: 2013
Journal: N Engl J Med, provisional acceptance pending response to comments
Title: Pharmacokinetics and safety of a nevel recombinant human von Willebrand factor manufactured with a plasma-free method: a multicenter prospective clinical trial.
Author(s): Mannucci PM, Kempton C, Miller C, Romond E, Shapiro A, Birschmann I, Ragni MV, Gill JC, Yee TT
Year: 2013
Journal: Blood
Title: Liver transplant outcomes in HIV-infected hemophilic men.
Author(s): Ragni MV, DeVera M, Roland ME, Wong MT, Stosor V, Sherman KE, Hardy D, Blumberg E, Fung F
Year: 2013
Journal: Haemophilia
Volume: 19
Page(s): 134-140
Title: Anticoagulation in ambulatory cancer patients: A cost-effectiveness analysis.
Author(s): Pishko A, Smith KJ, Ragni MV
Year: 2012
Journal: Thrombosis Haemostasis
Volume: 108
Page(s): 303-310
Title: Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients
Author(s): Powell JS, Josephson NJ, Quon D, Ragni MV, Cheng G, McKinney B, Jiang H, Li L, Dumont JA
Year: 2012
Journal: Blood
Volume: 119
Page(s): 3031-3037
Title: Recombinant factor IX Fc fusion (rFIXFc) demonstrates safety and prolonged activity in hemophilia B patients
Author(s): Shapiro AD, Ragni MV, Valentino L, Key NS, Josephson NC, Powell JS, Cheng G, Thrompson A, Goyal J
Year: 2012
Journal: Blood
Volume: 119
Page(s): 666-672
Title: Pulmonary embolism in sickle cell disease: A case-control study.
Author(s): Novelli EM, Huynh C, Gladwin MT, Moore CG, Ragni MV
Year: 2012
Journal: Journal of Thrombosis Haemostasis
Volume: 10
Page(s): 760-766
Title: Plasma thrombospondin-1 is increased in sickle cell disease and associated with hydroxyurea use, lower rates of hemolysis, and increased vaso-oclusive episodes
Author(s): Novelli EM, Kato GJ, Ragni MV, Zhang Y, Hildesheim ME, Nouraie M, Barge S, Meyer MP, Cortese-Hassett A
Year: 2012
Journal: American Journal of Hematology
Volume: 87
Page(s): 326-330
Title: Outcomes of liver transplantation in HCV-HIV co-infnected recipients.
Author(s): Terrault nA, Roland ME, Schiano T, Dove L, Wong MT, Poordad F, Ragni MV, Burin B, Simon D
Year: 2012
Journal: Liver Transplant
Volume: 18
Page(s): 716-726
For Pub Med search results, click here.