Managed Care Hemo

National Hemophilia Foundation

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Hemophilia Education for Managed Care and Payer Professionals

Providers

Advisor Recommended Article:

The viability and necessity of APRN‐led care models in the clinical management of haemophilia and other inherited bleeding and clotting disorders

Federizo A, Shullick M, Witkop M. The viability and necessity of APRN‐led care models in the clinical management of haemophilia and other inherited bleeding and clotting disorders. Haemophilia. 2018;24:563–569.
A. Federizo M. Shullick M. Witkop
Available via: https://onlinelibrary.wiley.com/doi/pdf/10.1111/hae.13536

Abstract
Inhibitor formation is among the most severe complications of hemophilia treatment. With a cumulative incidence of ∼30% in those with severe hemophilia A and ∼3% in those with severe hemophilia B, inhibitors are caused by a T-cell response directed against infused coagulation factor; these inhibitors neutralize factor VIII or IX activity and disrupt normal hemostasis. Inhibitor patients become unresponsive to standard factor treatment and, as an alternative, use bypass treatment (eg, recombinant factor VIIa or factor VIII inhibitor bypass activity). However, response to bypass agents is poorer and the burden of disease is higher, with greater morbidity, hospitalization, cost, and mortality, than in noninhibitor patients. Furthermore, inhibitor formation interferes with prophylaxis to prevent bleeding episodes and is a contraindication to gene therapy. Thus, more effective therapies for inhibitor patients are greatly needed. In the last several years, there has been an explosion of novel alternative hemostatic agents for hemophilia patients with and without inhibitors. These agents take advantage of technologic manipulation of coagulation factors and natural anticoagulants to promote hemostasis. The approaches include the following: (1) mutants or mimics of coagulation factors, rendering them resistant to natural anticoagulants; or (2) knock-down or disruption of natural anticoagulants, preventing degradation of coagulation factors. The purpose of this article was to review these novel alternative hemostatic agents and their mechanisms of action, as well as the preliminary pharmacokinetic, safety, and efficacy data available from early-phase clinical trials.

 


How to Thrive in the Current Health Care Ecosystem: Recommendations for Providers to Address Payer Challenges

Free On-demand CE Activity for HTC Physicians, Nurses, and Social Workers

For complete CE information and to begin this activity, please go to:

ManagedCareHemo.com/ThriveCE/

Jointly provided by the National Hemophilia Foundation, Impact Education, LLC, and Postgraduate Institute for Medicine.
This activity is supported by independent educational grants from Genentech, CSL Behring, and Shire.


Advisor Recommended Article:

Novel approaches to hemophilia therapy: successes and challenges

Hematology 2017 2017:605-609.
Arruda VR, Doshi BS, Samelson-Jones BJ.
Available via: https://www.ncbi.nlm.nih.gov/pubmed/29018078

Abstract
New therapies for hemophilia A and hemophilia B will likely continue to change clinical practice. Ranging from extended half-life to nonfactor products and gene therapy, these innovative approaches have the potential to enhance the standard of care by decreasing infusion frequency to increase compliance, promoting prophylaxis, offering alternatives to inhibitor patients, and easing route of administration. Each category has intrinsic challenges that may limit the broader application of these promising therapies. To date, none specifically address the challenge of dispersing treatment to the developing world.


Advisor Recommended Article:

Novel alternate hemostatic agents for patients with inhibitors: beyond bypass therapy

Hematology 2017 2017:605-609.
Ragni MV.
Available via: https://www.ncbi.nlm.nih.gov/pubmed/29222310

Abstract
Inhibitor formation is among the most severe complications of hemophilia treatment. With a cumulative incidence of ∼30% in those with severe hemophilia A and ∼3% in those with severe hemophilia B, inhibitors are caused by a T-cell response directed against infused coagulation factor; these inhibitors neutralize factor VIII or IX activity and disrupt normal hemostasis. Inhibitor patients become unresponsive to standard factor treatment and, as an alternative, use bypass treatment (eg, recombinant factor VIIa or factor VIII inhibitor bypass activity). However, response to bypass agents is poorer and the burden of disease is higher, with greater morbidity, hospitalization, cost, and mortality, than in noninhibitor patients. Furthermore, inhibitor formation interferes with prophylaxis to prevent bleeding episodes and is a contraindication to gene therapy. Thus, more effective therapies for inhibitor patients are greatly needed. In the last several years, there has been an explosion of novel alternative hemostatic agents for hemophilia patients with and without inhibitors. These agents take advantage of technologic manipulation of coagulation factors and natural anticoagulants to promote hemostasis. The approaches include the following: (1) mutants or mimics of coagulation factors, rendering them resistant to natural anticoagulants; or (2) knock-down or disruption of natural anticoagulants, preventing degradation of coagulation factors. The purpose of this article was to review these novel alternative hemostatic agents and their mechanisms of action, as well as the preliminary pharmacokinetic, safety, and efficacy data available from early-phase clinical trials.


Advisor Recommended Article:

Alternative therapies for the management of inhibitors

Haemophilia (2016), 22 (Suppl. 5), 36–41
Shima M, Lillicrap D, Kruse-Jarres R.
Available via: https://www.ncbi.nlm.nih.gov/pubmed/27405674

Abstract
The development of inhibitors to factor VIII (FVIII) or factor IX (FIX) remains a major treatment complication encountered in the treatment of haemophilia. Not all patients with even the same severity and genotype develop inhibitors suggesting an underlying mechanism of tolerance against FVIII- or FIX-related immunity. One mechanism may be central tolerance observed in patients in whom the FVIII mutation enables some production of the protein. The other is a peripheral tolerance mechanism which may be evident in patients with null mutation. Recently, recombinant porcine FVIII (rpFVIII, Obixur, OBI-1, BAX801) has been developed for the haemostatic treatment of both congenital haemophilia with inhibitor (CHAWI) and acquired haemophilia A (AHA). In 28 subjects with AHA with life-/limb-threatening bleeding, rpFVIII reduced or stopped bleeding in all patients within 24 h. The cross-reactivity of anti-human FVIII antibodies to rpFVIII remains around 30-50%. Recently, new therapeutics based on the quite novel concepts have been developed and clinical studies are ongoing. These are humanized asymmetric antibody mimicking FVIIIa function by maintaining a suitable interaction between FIXa and FX (Emicizumab, ACE910), and small interfering RNAs (siRNA, ALN-AT3) suppress liver production of AT through post-transcriptional gene silencing and a humanized anti-TFPI monoclonal antibody (Concizumab). Their main advantages are longer half-life, subcutaneous applicability and efficacy irrespective of the presence of inhibitors which will make it easier to initiate more effective treatment especially early childhood.


Comprehensive Care Sustainability Collaborative Provided By NHF

Established in 2014, the Comprehensive Care Sustainability Collaborative (CCSC) was formed with the goal to overcome communication gaps between payers and providers. Growing threats to the future sustainability of the HTC integrated care model served as the impetus for NHF to launch the CCSC initiative. CCSC has acquired valuable insights that can be accessed on the website www.CCSChemo.com. The site contains educational materials, tools and other resources that can be leveraged by HTCs to improve the recognition of HTC-derived value.


 

Free On-demand CE Activity for Physicians, Pharmacists, and Nurses

This activity features two tracks with up to 1.5 credit hours each

Don’t Miss This Activity’s Associated Expert Interview With:

Michael Zeglinski, RPh
Senior Vice President, Specialty Pharmacy
OptumRx®/BriovaRx®

For complete CE information and to begin this activity, please go to:
www.managedcarehemo.com/optimization/

Jointly provided by the National Hemophilia Foundation, Impact Education, LLC, and Medical Education Resources, Inc.
This activity is supported by independent educational grants from Novo Nordisk, Inc., Grifols, and Shire.