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The Who? What? Guide

Special note

As a member of the bleeding disorders community you should be aware that pharmaceutical corporations and home care providers are profit-based organizations. They will compete for your business. Always consult with your hemophilia treatment center (HTC) or hematologist regarding pharmaceutical product selection. There are no “generic brands” of factor replacement products. Likewise consult with your HTC or hematologist when selecting a home care provider, especially if you need in-home nursing or infusion support.

The Bleeding Disorders Foundation of North Carolina (BDFNC) does not endorse any treatment products, manufacturers, home care services or individual medical providers. The companies and products included in this document are listed as a public service only. Listing is based on generally available information at the time of publication. Any inclusion or omission should not be interpreted as an endorsement or absence thereof.

BDFNC does not engage in the practice of medicine and under no circumstances recommends particular treatment for specific individuals. Dose schedules and other treatment regimens are continually revised and new side-effects recognized. Information listed here is provided for general reference only, and does not replace the advice of a medical advisor and/or product insert information. Any treatment must be designed according to the needs of the individual and the resources available.

Non-profit organizations serving the bleeding disorders community

BDFNC: Bleeding Disorders Foundation of NC (BDFNC) is a non-profit organization dedicated to improving the quality of life of persons affected by bleeding disorders through advocacy, education, promotion of research, and supportive programs and services. BDFNC is a chapter of the National Hemophilia Foundation, and a member organization of the Hemophilia Federation of America. In addition to broad community services, BDFNC offers special programs for the Latino community, women and girls, and families newly diagnosed with a bleeding disorder.

130 Iowa Lane
Suite 203
Cary, NC 27511
www.bleedingdisordersnc.org (this site)
info@bleedingdisordersnc.org
919-319-0014

NBDF (formerly NHF): The National Bleeding Disorders Foundation (NBDF), formerly National Hemophilia Foundation (NHF), is dedicated to finding better treatments and cures for bleeding and clotting disorders and to preventing the complications of these disorders through education, advocacy, and research.

HFA: The Hemophilia Federation of America is the national community-based advocacy organization serving all people living with bleeding disorders and their families across the United States.

ATHN: American Thrombosis & Hemostasis Network, a non-profit organization committed to advancing and improving care for individuals affected by bleeding and thrombotic disorders.

Accessia Health (formerly Patient Services, Inc. – PSI): Accessia Health helps chronically ill patients with unaffordable medical expenses.

Patient Advocate Foundation: The Patient Advocate Foundation offers financial assistance and advocacy to help patients afford essential medications, treatments, and healthcare.

HTCs: Hemophilia Treatment Centers in North Carolina

Hemophilia Treatment Centers (HTCs) are usually affiliated with major medical centers and provide specialized, family-centered comprehensive care for those affected by bleeding and clotting disorders. Services include diagnosis, medical evaluation and management, 24-hour phone accessibility, and educational programs for medical professionals, patients and schools. HTCs are part of a federally-funded national HTC network, and participate in clinical trials and other leading edge research initiatives.

Please refer to the treatment centers section of this web site for an up to date list of medical facilities in the North Carolina area.

Common bleeding disorders terms

  • Coagulation – the process by which blood forms clots through the action of different clotting factors. Factors are often referred to with Roman numerals: VII (7), VIII (8), IX (9), X (10), etc.
  • Hemophilia A (classical hemophilia) – a deficiency in coagulation factor VIII (8). May be categorized as mild, moderate, or severe.
  • Hemophilia B (Christmas disease) – a deficiency in coagulation factor IX (9). May be categorized as mild, moderate, or severe.
  • Von Willebrand disease (vWD) – a bleeding disorder in which von Willebrand factor (VWF), a blood protein, is either deficient or defective.
  • Inhibitor – an antibody to infused clotting factor concentrates, making standard treatments ineffective.
  • Plasma – the liquid portion of the blood that contains the clotting factors (total of 13 including VIII and IX).
  • Platelets – cells that circulate within our blood and bind together when they recognize damaged blood vessels.
  • PTT, PT – Partial Thromboplastin Time, Prothrombin Time – tests used to investigate unexplained bleeding or clotting.
  • TT – Thrombin time (clotting time) test.
  • CBC – Complete blood count – a test to evaluate the cellular portions of the blood.

Pharmaceutical corporations producing medications used in the treatment of bleeding disorders

These companies produce treatment products but do not sell directly to the consumer. Products listed are approved for use in the US.
  • Bayer – Jivi® | Kovaltry®
  • BioMarin – Roctavian®
  • CSL Behring – Afstyla® | Corifact® | Hengenix® | Humate-P® | Idelvion® | RiaSTAP®
  • Genentech – Hemlibra®
  • Grifols – Alphanate® | AlphaNine®SD | Profilnine®
  • HEMA Biologics – Sevenfact®
  • Kedrion Biopharma – Coagadex® | Koate® | Ryplazim®
  • Medexus Pharma – Ixinity®
  • Novo Nordisk – Alhemo® | Esperoct® | Novoeight® | NovoSeven® RT | Rebinyn® | Tretten®
  • Octapharma Plasma – Fibryga® | Nuwiq® | Wilate®
  • Pfizer – BeneFIX® | Beqvez® | Hympavzi® | Xyntha® | Xyntha Solofuse®
  • Sanofi – Alprolix® | Altuviiio® | Eloctate®
  • STAQ Pharma – DDAVP® Nasal Spray (Compounded)
  • Takeda – Advate® | Adynovate® | FEIBA® | Hemofil M® | Obizur® | Recombinate® | Rixubis® | Vonvendi®)
  • Various manufacturers – Amicar® (aminocaproic acid) | Cyklokapron® | Lysteda® (tranexamic acid) | DDAVP (desmopressin) injection or nasal spray

All pharmaceutical trademarks are the property of their respective manufacturers, who are not affiliated with BDFNC.

Pharmaceutical products commonly prescribed in the US for treatment of bleeding disorders

This list is provided here only as a general reference, not as a list of approved and/or potentially appropriate products. Except as noted, treatment is administered by injection into a vein. Some are plasma-derived (made from human blood); others are made using a recombinant DNA process. Always consult your physician about the most appropriate treatment product for your condition.

Please note: In addition to the disorders listed below, there are other more rare conditions that cause abnormal bleeding. These disorders – including acquired factor deficiencies and platelet disorders – and their pharmaceutical treatments are beyond the scope of this document. Please refer to the MASAC recommendations below for additional information.

Important References:

► for hemophilia A:

plasma-derived: Alphanate® | Koate® | Hemofil® M | Humate-P®

recombinant: Advate® | Adynovate® | Afstyla® | Altuviiio® | Eloctate® | Esperoct® | Jivi® | Kovaltry® | Novoeight® | Nuwiq® | Recombinate® | Xyntha® | Xyntha Solofuse®

therapeutic antibody for prevention of bleeding: Hemlibra® (administered subcutaneously)

gene therapy: Roctavian®

► for hemophilia A with inhibitor:

plasma-derived: Feiba®

recombinant: NovoSeven® RT | Sevenfact®

therapeutic antibody: Hemlibra® (administered subcutaneously)

► for hemophilia B:

plasma-derived: Alphanine® SD | Profilnine®

recombinant: Alprolix® | BeneFIX® | Idelvion® | Ixinity® | Rebinyn® | Rixubis®

gene therapy: Beqvez® | Hemgenix®

► for hemophilia B with inhibitor:

plasma-derived: Feiba®

recombinant: NovoSeven®RT | Sevenfact®

► for vWD:

plasma-derived: Alphanate® | Humate-P® | Wilate®

recombinant: Vonvendi®

► for mild vWD or mild hemophilia A:

other: Aminocaproic acid | Desmopressin (DDAVP) – a synthetic version of a naturally occurring hormone that releases clotting factors from blood vessel linings; administered nasally or by injection or intravenous | tranexamic acid

► for factor I deficiency:

plasma-derived: Riastap®, Fibryga®

► for factor II deficiency:

plasma-derived: Profilnine®

► for factor V deficiency:

plasma-derived: plasma transfusion |

other: aminocaproic acid | tranexamic acid

► for factor VII deficiency:

recombinant: Sevenfact® | NovoSeven®

► for factor X deficiency:

plasma-derived: Coagadex®, Profilnine®

► for factor XI deficiency:

plasma-derived: plasma

recombinant: NovoSeven®

other: aminocaproic acid | tranexamic acid

► for factor XIII deficiency:

plasma-derived: Corifact®

recombinant: Tretten® (A-subunit)

► for Glanzmann’s thrombasthenia:

plasma-derived: platelets

recombinant: NovoSeven®

other: aminocaproic acid | tranexamic acid

► for qualitative platelet disorders:

plasma-derived: platelets

other: DDAVP® | tranexamic acid

► for storage pool disorders:

plasma-derived: platelets

other: aminocaproic acid | DDAVP® | tranexamic acid

► for hereditary antithrombin deficiency:

plasma-derived: Thrombate III®

All pharmaceutical trademarks are the property of their respective manufacturers, who are not affiliated with BDFNC.

Home care companies providing services to people with bleeding disorders

These are for-profit “specialty pharmacy” businesses that sell medications and supplies to patients with bleeding disorders. Many of these companies provide additional services, including home delivery, pharmacist consultations, financial consultation and assistance, and in-home nursing and infusion services. Home care providers do not prescribe medications or give medical recommendations, nor do they make endorsements or referrals to a specific HTC, hematologist, or medical provider.

There are numerous home care companies operating in North Carolina. Your HTC will have the most current information about the providers that best fit your needs.

340B Program

The 340B Drug Pricing Program is a federal program that allows Hemophilia Treatment Centers (HTCs) to purchase clotting factor at a discount for their patients. Participating HTCs rely on this program to fund ongoing support for the specialized comprehensive services to people with bleeding disorders.

The Charlotte Festival & Walk is on June 6, register and donate today!

The Charlotte Festival & Walk has passed, but there is still time to donate!

The BDFNC office will be closed from December 22 to January 4 for the holiday season. Please note: All assistance programs will be paused (you can still apply) and applications will be reviewed once the office re-opens.