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Hemophilia Education for Managed Care and Payer Professionals

Introductory Video

Latest Expert Interview

Emerging Therapies for the Management of Inhibitors

Inhibitor development in persons with hemophilia represent one of the costliest complications among all chronic diseases. Patients who develop an inhibitor are vulnerable to potentially severe bleeding episodes despite ongoing factor infusions and are twice as likely to be hospitalized for a bleeding complication as those without an inhibitor. In addition to the clinical and economic burden of the inhibitor itself, traditional management options, such as immune tolerance induction (ITI) or the on-demand or prophylactic use of bypassing agents, may likewise carry high costs. The recent approval of the humanized bispecific antibody emicizumab-kxwh signaled the advent of a new era of non-factor options for the management of inhibitors. In addition to offering decreased bleeding episodes, these novel therapies are given subcutaneously rather than intravenously, representing simplified home therapy.

  • Inhibitor to FVIII only
    • The approval of emicizumab-kxwh was based on data from two clinical trials: an adult and adolescent trial (HAVEN 1) and a pediatric trial (HAVEN 2)
    • For patients receiving emicizumab-kxwh prophylaxis in HAVEN 1, the annualized bleeding rate (ABR) was 2.9 (95% CI; 1.7, 5.0) compared with 23.3 (95% CI: 12.3, 43.9) for patients not receiving prophylaxis corresponding to an 87% ABR reduction (95% CI: 72.3%, 94.3%), p<0.0001
    • In HAVEN 2, the interim analysis ABR for emicizumab-kxwh-treated bleeds was 0.2 (95% CI: 0.1, 0.6) versus an ABR of 2.9 for all bleeds (95% CI: 1.8, 4.9)
    • The recommended dose of emicizumab-kxwh is 3 mg/kg by subcutaneous injection once weekly for the first 4 weeks, followed by 1.5 mg/kg once weekly
  • Inhibitor to FVIII or FIX
    • Concizumab, a humanized monoclonal antibody against tissue factor pathway inhibitor (TFPI), is currently in phase 3 clinical trials for the management of patients with inhibitors
    • In spiked samples from hemophilia patients, peak thrombin and endogenous thrombin potential (ETP) increased concentration dependently, reaching near-normal levels at concizumab concentrations >10 nm
    • Repeated SC doses of concizumab in healthy subjects increased both peak thrombin and ETP; these effects were sustained throughout the dosing interval


Oldenburg J, Mahlangu JN, Kim B, et al. Emicizumab Prophylaxis in Hemophilia A with Inhibitors. N Engl J Med. 2017;377:809-818.

Young G, Sidonio RF, Liesner R, et al. HAVEN 2 Updated Analysis: Multicenter, Open-Label, Phase 3 Study to Evaluate Efficacy, Safety and Pharmacokinetics of Subcutaneous Administration of Emicizumab Prophylaxis in Pediatric Patients with Hemophilia A with Inhibitors. Blood. 2017;130(Suppl 1):85.

Waters EK, Sigh J, Friedrich U, Hilden I, Sørensen BB. Concizumab, an anti-tissue factor pathway inhibitor antibody, induces increased thrombin generation in plasma from haemophilia patients and healthy subjects measured by the thrombin generation assay. Haemophilia. 2017;23:769–776.

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Q1: What best describes your primary role?

Q2: Approximately how many patients with hemophilia are being managed in your plan or practice setting?

Q3: How do you perceive the value of available and emerging non-factor agents for the management of inhibitors in light of disease burden and cost of therapies?

Q4: Which of the below topics do you most need further information on?

Introductory Video

Advisory Board

Jennifer Maahs, RN, PNP, MSN

Nurse Practitioner
Indiana Hemophilia and Thrombosis Center

Vanita K. Pindolia, PharmD, BCPS, MBA

Vice President, Ambulatory Clinical Pharmacy Programs_PCM
Henry Ford Health System/Health Alliance Plan of Michigan

Steven W. Pipe, MD

Director, Division of Pediatric Hematology and Oncology
Pediatric Medical Director, Hemophilia and Coagulation Disorders Program
University of Michigan

Marion Koerper, MD

Professor Emerita on Recall
Department of Pediatric Hematology
School of Medicine University of California, San Francisco
(UCSF) School of Medicine
Medical Advisor
National Hemophilia Foundation

Diane J. Nugent, MD Chair, Hematology

Medical Director, Hematology and Blood and Donor Services
Division Chief, Hematology
Children’s Hospital of Orange County

Celynda G. Tadlock, PharmD, MBA

Vice President Pharmacy Business Development, Aetna
President, Coventry Prescription Management Serivces, Inc.