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Factor IX


General


Pronunciation

(FAK ter nyne)


Brand Names: U.S.

  • AlphaNine ‚ ® SD
  • BeneFix ‚ ®
  • Mononine ‚ ®

Indications


Use: Labeled Indications

Prevention and control of bleeding in patients with factor IX deficiency (hemophilia B or Christmas disease)

NOTE: Contains either nondetectable levels of factors II, VII, and X (AlphaNine ‚ ®, Mononine ‚ ®) or only factor IX (BeneFIX ‚ ®). Therefore, NOT INDICATED for replacement therapy of any other clotting factor besides factor IX or for reversal of anticoagulation due to either vitamin K antagonists or other anticoagulants (eg, dabigatran), hemophilia A patients with factor VIII inhibitors, or patients in a hemorrhagic state caused by reduced production of liver-dependent coagulation factors (eg, hepatitis, cirrhosis).


Contraindications


Hypersensitivity to mouse protein (Mononine ‚ ®) or hamster protein (BeneFix ‚ ®)


Dosing and Administration


Dosing: Adult

NOTE: Contains either nondetectable levels of factors II, VII, and X (AlphaNine ‚ ®, Mononine ‚ ®) or only factor IX (BeneFIX ‚ ®). Therefore, NOT INDICATED for replacement therapy of any other clotting factor besides factor IX or for reversal of anticoagulation due to either vitamin K antagonists or other anticoagulants (eg, dabigatran), hemophilia A patients with factor VIII inhibitors, or patients in a hemorrhagic state caused by reduced production of liver-dependent coagulation factors (eg, hepatitis, cirrhosis).

Control bleeding in patients with factor IX deficiency (hemophilia B or Christmas disease): Dosage is expressed in units of factor IX activity; dosing must be individualized based on severity of factor IX deficiency, extent and location of bleeding, and clinical status of patient:

Formula for units required to raise blood level %:

AlphaNine ‚ ® SD, Mononine ‚ ®: IV:

Number of factor IX units required = body weight (in kg) x desired factor IX level increase (as %) x 1 unit/kg

For example, to attain a 100% level in a 70 kg patient who has a baseline level of 20%: Number of factor IX units needed = 70 kg x 80% x 1 unit/kg = 5600 units

BeneFix ‚ ®: IV: Number of factor IX units required = body weight (in kg) x desired factor IX level increase (as %) x 1.3 units/kg

Guidelines: As a general rule, the level of factor IX required for different conditions is as follows; Note: The following recommendations may vary from those found within prescribing information or practitioner preference.

Primary prophylaxis: 25-40 units/kg twice weekly (World Federation of Hemophilia, 2005) or 40-100 units/kg 2 or 3 times weekly (National Hemophilia Foundation, MASAC recommendation, 2007); however, the optimum regimen has yet to be defined.

Minor hemorrhage (eg, bruising, cuts/scrapes, uncomplicated joint hemorrhage):

Desired levels of factor IX for hemostasis: 15% to 30%

Frequency of dosing: Every 12-24 hours if necessary

Duration of treatment: 1-2 days

Moderate hemorrhage (eg, epistaxis, oropharyngeal bleeds, dental extractions, hematuria):

Desired levels of factor IX for hemostasis: 25% to 50%

Frequency of dosing: Every 12-24 hours

Duration of treatment: 2-7 days

Major hemorrhage (eg, joint and muscle [especially large muscles] hemorrhage, intracranial or intraperitoneal hemorrhage), major trauma, or surgical prophylaxis:

Desired levels of factor IX for hemostasis: 50% to 100% (depending on the clinical situation, desired factor IX level may be reduced following active treatment period for hemorrhage or >48 hours postop)

Frequency of dosing: Every 12-24 hours or every 18-30 hours, depending on half-life and measured factor IX levels (after 3-5 days, maintain at least 20% activity)

Duration of treatment: 7-10 days, depending upon nature of insult


Dosing: Geriatric

Refer to adult dosing.


Dosing: Pediatric

NOTE: Contains either nondetectable levels of factors II, VII, and X (AlphaNine ‚ ®, Mononine ‚ ®) or only factor IX (BeneFIX ‚ ®). Therefore, NOT INDICATED for replacement therapy of any other clotting factor besides factor IX or for reversal of anticoagulation due to either vitamin K antagonists or other anticoagulants (eg, dabigatran), hemophilia A patients with factor VIII inhibitors, or patients in a hemorrhagic state caused by reduced production of liver-dependent coagulation factors (eg, hepatitis, cirrhosis).

Control bleeding in patients with factor IX deficiency (hemophilia B or Christmas disease): Dosage is expressed in units of factor IX activity; dosing must be individualized based on severity of factor IX deficiency, extent and location of bleeding, and clinical status of patient:

AlphaNine ‚ ® SD, Mononine ‚ ®: IV: Refer to adult dosing.

BeneFix ‚ ®: IV:

Children <15 years:

Formula for units required to raise blood level %:

Number of factor IX units required = body weight (in kg) x desired factor IX level increase (as %) x 1.4 units/kg

Children ≥15 years: Refer to adult dosing.

Note: See Guidelines" section in adult dosing.


Dosing: Renal Impairment

No dosage adjustment provided in manufacturer 's labeling.


Dosing: Hepatic Impairment

No dosage adjustment provided in manufacturer 's labeling; use with caution.


Reconstitution

Refer to instructions for individual products. Diluent and factor IX should come to room temperature before combining.


Administration

Solution should be infused at room temperature

IV administration only: Should be infused slowly: The rate of administration should be determined by the response and comfort of the patient.

AlphaNine ‚ ® SD: Administer IV at a rate not exceeding 10 mL/minute

BeneFix ‚ ®: Administer IV over several minutes

Mononine ‚ ®: Administer IV at a rate of ~2 mL/minute. Administration rates of up to 225 units/minute have been regularly tolerated without incident (when reconstituted as directed to ~100 units/mL).


Storage

When stored at refrigerator temperature, 2 ‚ °C to 8 ‚ °C (36 ‚ °F to 46 ‚ °F), factor IX is stable for the period indicated by the expiration date on its label. Avoid freezing which may damage container for the diluent.

AlphaNine ‚ ® SD: May also be stored at room temperature not to exceed 30 ‚ °C (86 ‚ °F) for up to 1 month. Reconstituted solution should be used within 3 hours of preparation.

BeneFix ‚ ®: May also be stored at room temperature not to exceed 25 ‚ °C (77 ‚ °F) for up to 6 months. Reconstituted solution should be at room temperature and used within 3 hours of preparation.

Mononine ‚ ®: May also be stored at room temperature not to exceed 25 ‚ °C (77 ‚ °F) for up to 1 month. Reconstituted solution should be at room temperature and used within 3 hours of preparation.


Dosage Forms/Strengths


Dosage Forms

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Injection, powder for reconstitution [recombinant]:

BeneFix ‚ ®: 250 units, 500 units, 1000 units, 2000 units [contains polysorbate 80 and sucrose 0.8%; derived from or manufactured using Chinese hamster ovary cells]

Injection, powder for reconstitution [human derived]:

AlphaNine ‚ ® SD: 500 units, 1000 units, 1500 units [contains polysorbate 80 and trace amounts of factors II, VII, and X]

Mononine ‚ ®: 500 units, 1000 units [contains polysorbate 80, mouse protein, and trace amounts of factors II, VII, and X]


Drug Interactions

Aminocaproic Acid: May enhance the adverse/toxic effect of Factor IX. Specifically, use of this combination may increase the risk of thrombosis. Avoid combination


Monitoring Parameters

Factor IX levels, aPTT; BP, HR, signs of hypersensitivity reactions; screen for factor IX inhibitors when patient is to undergo surgery or if suboptimal response to treatment occurs


Adverse Reactions


Frequency not defined.

Cardiovascular: Cyanosis, flushing, hypotension, chest tightness, thrombosis

Central nervous system: Chills, dizziness, drowsiness, fever (including transient fever following rapid administration), headache, lethargy, lightheadedness, somnolence

Dermatologic: Angioedema, photosensitivity reaction, rash, urticaria

Gastrointestinal: Abnormal taste, diarrhea, nausea, vomiting

Hematologic: Disseminated intravascular coagulation (DIC)

Hepatic: Alkaline phosphatase increased, ALT increased, AST increased

Local: Injection site reactions: Cellulitis, discomfort, pain, phlebitis, stinging

Neuromuscular & skeletal: Neck tightness, paresthesia, rigors

Ocular: Visual disturbance

Respiratory: Allergic rhinitis, asthma, cough, dyspnea, hypoxia, laryngeal edema, lung disorder

Miscellaneous: Allergic reaction, anaphylaxis, burning sensation in jaw/skull, factor IX inhibitor development, hypersensitivity reaction

Postmarketing and/or case reports: HAV seroconversion, inadequate response/recovery, nephrotic syndrome (associated with immune tolerance induction), parvovirus B19 seroconversion, renal infarction, superior vena cava syndrome (neonates)


Warnings/Precautions


Concerns related to adverse effects:

- Antibody formation: The development of factor IX antibodies (or inhibitors) has been reported with factor IX therapy (usually occurs within the first 10-20 exposure days); the risk of severe hypersensitivity reactions occurring may be greater in these patients. When clinical response is suboptimal or patient is to undergo surgical procedure, screen for inhibitors. Patients with severe gene defects (eg, gene deletion or inversion) are more likely to develop inhibitors (WFH, 2005).

- Hypersensitivity reactions: Hypersensitivity and anaphylactic reactions have been reported with use. Delayed reactions (up to 20 days after infusion) in previously untreated patients may also occur. Due to potential for allergic reactions, the initial ~10-20 administrations should be performed under appropriate medical supervision. Hypersensitivity reactions may be associated with factor IX inhibitor development; patients experiencing allergic reactions should be evaluated for factor IX inhibitors.

- Thrombotic events: Observe closely for signs or symptoms of intravascular coagulation or thrombosis; risk is generally associated with the use of factor IX complex concentrates (containing therapeutic amounts of additional factors); however, potential risk exists with use of factor IX products (containing only factor IX). Use with caution when administering to patients with liver disease, postoperatively, neonates, or patients at risk of thromboembolic phenomena, disseminated intravascular coagulation or patients with signs of fibrinolysis due to the potential risk of thromboembolic complications.

Disease-related concerns:

- Hepatic impairment: Use with extreme caution in patients with hepatic impairment due to the risk of thromboembolic complications.

Dosage form specific issues:

- Human plasma (AlphaNine ‚ ® SD, Mononine ‚ ®): Product of human plasma; may potentially contain infectious agents which could transmit disease. Screening of donors, as well as testing and/or inactivation or removal of certain viruses, reduces the risk. Infections thought to be transmitted by this product should be reported to the manufacturer.

Other warnings/precautions:

- Appropriate use: Contains either nondetectable levels of factors II, VII, and X (AlphaNine ‚ ®, Mononine ‚ ®) or only factor IX (BeneFIX ‚ ®). Therefore, factor IX products are NOT INDICATED for replacement therapy of any other clotting factor besides factor IX. In addition, factor IX concentrate products are NOT INDICATED for reversal of anticoagulation due to either vitamin K antagonists or other anticoagulants (eg, dabigatran), hemophilia A patients with factor VIII inhibitors, or patients in a hemorrhagic state caused by reduced production of liver-dependent coagulation factors (eg, hepatitis, cirrhosis).

- Immune tolerance induction: Safety and efficacy have not been established in immune tolerance induction with factor IX products. Nephrotic syndrome has occurred following immune tolerance induction in patients with factor IX inhibitors and a history of allergic reactions to therapy.


Pregnancy Risk Factor

C


Pregnancy Considerations

Animal reproduction studies have not been conducted. Safety and efficacy in pregnant women have not been established. Use during pregnancy only if clearly needed. Parvovirus B19 or hepatitis A, which may be present in plasma-derived products, may affect a pregnant woman more seriously than a nonpregnant woman.


Actions


Pharmacology

Replaces deficient clotting factor IX. Hemophilia B, or Christmas disease, is an X-linked inherited disorder of blood coagulation characterized by insufficient or abnormal synthesis of the clotting protein factor IX. Factor IX is a vitamin K-dependent coagulation factor which is synthesized in the liver. Factor IX is activated by factor XIa in the intrinsic coagulation pathway. Activated factor IX (IXa), in combination with factor VII:C activates factor X to Xa, resulting ultimately in the conversion of prothrombin to thrombin and the formation of a fibrin clot. The infusion of exogenous factor IX to replace the deficiency present in hemophilia B temporarily restores hemostasis.


Half-Life Elimination

IX component: Adults: 21-31 hours; children: 14-28 hours

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