Protein C Concentrate (Human) for Protein C Deficiency - A Complete Guide

Overview:

On March 30, 2007, the United States Food and Drug Administration (FDA) authorized Protein C to treat severe congenital protein C deficiency. Protein C is a main coagulation system inhibitor that reduces thrombin production. A hypercoagulability condition caused by a severe congenital protein C deficiency typically manifests at birth as purpura fulminans (acute purpuric rash characterized by microvasculature coagulation, resulting in skin discoloration and purpuric lesions) and severe venous and arterial thrombosis (blood clot blocking arteries). It is typical to observe repeated thrombotic episodes. Numerous concentrations of human protein C inactivated by viruses have been created during the 1990s. These concentrates are the recommended intervention for treating and preventing severe congenital protein C deficient clinical symptoms. Blood naturally contains protein C, an anticoagulant essential for controlling blood clotting.

Drug Group:

Protein C concentrate (human) is a medication in the blood thinner or anticoagulant drug group.

Available Doses and Dosage Forms:

Usually, the drug is supplied in ampules or vials for intravenous (IV) delivery. Doses are often recommended based on the severity of the patient's protein C insufficiency, weight, and response to treatment.

Protein C concentrate is available in single-dose vials reconstituted with five milliliters or ten milliliters of sterile water for injection, respectively, to provide a single dose of human Protein C at a concentration of 100 IU/mL (international units per milliliter). The vials contain 500 (blue color bar) or 1000 (green color bar) nominal international units (IU) of human Protein C.

• Dosage at First: 100 to 120 IU/kg (international units per kilogram). The next three doses are 60 to 80 IU/kg every six hours.

• Dosage for Maintenance: 45 to 60 IU/kg every six or 12 hours.

• Extended Prophylaxis: Every 12 hours, 45 to 60 IU/kg.

For Patients:

What Is Protein C Deficiency?

An uncommon hereditary condition known as protein C deficiency occurs when the body cannot create enough functional protein C, the blood's natural anticoagulant. A lack of protein C can raise the chance of an aberrant blood clot forming since it is essential for controlling blood clotting.

The body's capacity to properly regulate blood coagulation is compromised in those with protein C deficiency due to low amounts of functional protein C. A higher risk of aberrant blood clot formation as a result of this imbalance might result in thromboembolic events and disorders such as deep vein thrombosis (DVT) (blood clots in deep veins) and pulmonary embolism (blood clots in the arteries of the lung). Blood clots that develop during thromboembolic episodes can clog blood arteries as they pass through the circulation and pose a major health risk.

How Does Protein C Concentrate (Human) Work?

• Protein C concentrate (human) is made from human plasma and includes protein C in its active form.

• Giving to those lacking in protein C helps restore the lost protein.

• As an anticoagulant, the drug helps control blood clotting and avoid producing too many clots.

What Is the Dosage of Protein C Concentrate (Human)?

The degree of protein C deficiency, the patient's weight, and each patient's reaction to treatment can all affect the protein C concentration (human) dose.

• The first dose for acute episodes or short-term prophylaxis may be 100 to 120 IU/kg.

• Thereafter, 60 to 80 IU/kg doses may be given every six hours.

• The 45 to 60 IU/kg maintenance dose may be given every six or 12 hours.

• One possible maintenance dosage for long-term prevention might be 45 to 60 IU/kg every 12 hours.

How Effective Is Protein C Concentrate (Human)?

• For people with a protein C deficit, protein C concentrate (human) is typically helpful in controlling and avoiding thromboembolic episodes.

• Individual differences in effectiveness may exist; thus, careful monitoring and dose modifications are necessary to guarantee the best possible outcomes.

• Frequent assessment of protein C levels and contact with medical specialists aid in determining the efficacy of the treatment and facilitating any required modifications.

What Are the Things to Inform the Doctor Before Taking the Drug?

• Medical History: Give a thorough account of the past and present thromboembolic episodes, liver or renal illnesses, and any other pertinent problems.

• Medications: Tell the doctor about all the drugs used, including over-the-counter and prescription ones, vitamins, and herbal therapies.

• Allergies: Since Protein C concentrate is made from human plasma, please report any known allergies, especially to human plasma.

How Is Protein C Concentrate (Human) Administered?

• Administration Route: Intravenous (IV) injection of Protein C concentrate usually results in direct bloodstream administration. The injection site must be cleansed using an antiseptic solution to avoid infection.

• Healthcare Setting: Trained medical personnel handle the administration, often at a healthcare center.

• Dose Instructions: Adhere to the healthcare provider's recommended dose and administration regimen.

What Are the Side Effects of Protein C Concentrate (Human)?

• Allergic Reactions: Breathing difficulties, swelling, rash, itching, and hives.

• Fever and Lightheadedness: One of the adverse effects might be a fever.

• Reactions at the Injection Site: Swelling, discomfort, or redness.

• Additional Unfavorable Effects: Report any unexpected or severe symptoms, such as changes in blood pressure, nausea, vomiting, or dizziness.

Dietary Considerations:

Protein C concentrate (human) is generally unrelated to dietary limitations. Maintaining a good, balanced diet is necessary to promote general well-being.

As some dietary components may affect blood clotting, talk to a healthcare professional about any dietary concerns or changes.

Missed Dose:

In the event of a missed dose of protein C concentrate (human), contacting healthcare providers immediately for guidance is recommended. Doubling the dose to compensate for a missed one should only be done if directed by healthcare professionals.

Overdose:

If an overdose is suspected or symptoms of excessive bleeding occur, seeking emergency medical attention is advised. Overdose may elevate the risk of bleeding complications, necessitating prompt intervention.

Storage:

• Protein C concentrate (human) should not be frozen; instead, it should be kept refrigerated.

• If the drug has been exposed to temperatures outside of the approved range or frozen, do not use it.

• Make sure kids cannot get their hands on the medicine.

• Protein C concentrate (human) should not be used after the expiration date printed on the container.

For Doctors:

Indication:

Protein C concentrate (human) is an anticoagulant prescribed for preventing and treating venous thrombosis and purpura fulminans in newborns, children, and adult patients with severe congenital protein C deficiency.

Dose:

The degree of protein C shortage, body weight, and the particular clinical circumstance are some examples of the unique aspects that may affect the dose of protein C concentrate in humans.

1. Initial Dosage: 100 to 120 international units per kilogram. The following three dosages are 60 to 80 IU/kg every six hours.

2. Maintenance Dosage: 45 to 60 IU/kg every six to 12 hours.

Dosing Considerations:

• Considering the severity of the deficit and the intended therapy (acute episode, short-term prophylaxis, or long-term prophylaxis), healthcare experts will establish the right dosage for each patient.

• Monitoring protein C levels to modify the dosage as needed could be required.

• Initial and subsequent dosages depend on the patient's weight and are given at predetermined intervals for acute episodes or short-term prevention.

• A maintenance dosage administered at various intervals is part of long-term prophylaxis.

• Dosing recommendations should be based on each patient's reaction and the clinical situation.

• It is important to adhere to the recommended dose and administration guidelines given by medical authorities.

What Are the Pharmacological Aspects of Protein C Concentrate (Human)?

1. Pharmacodynamics:

• Anticoagulant Activity: By blocking particular clotting factors, especially factors Va and VIIIa, Protein C concentrate (human) demonstrates anticoagulant qualities.

• Control of Coagulation: Protein C helps balance procoagulant and anticoagulant activity in the circulation by controlling the coagulation cascade.

2. Mechanism:

• Source: Protein C concentrate, derived from human plasma, has protein C in its active state.

• Administration: Protein C concentrate restores the missing protein C's regulatory role in coagulation by supplementing it intravenously.

• Anticoagulant Action: Activated Protein C has an anticoagulant action by inhibiting clotting factors, which restricts blood clot formation and averts thromboembolic episodes.

3. Pharmacokinetics:

• Distribution: Following ingestion, the bloodstream contains protein C concentrate.

• Metabolism: The body's physiological functions in protein C are the main focus of protein C concentrate metabolism.

• Excretion: Since the drug is used to replenish a natural protein that is lacking rather than being metabolized in the conventional sense, excretion information is usually not relevant.

Toxicity:

There is little information available on the toxicity of human protein C concentrate. People should seek medical treatment if they develop any unexpected symptoms or indicators of an adverse response, such as lightheadedness, itching, or rash. Throughout therapy, it is critical to keep an eye out for any indications of allergic reactions, severe bleeding, or other side effects.

Clinical Studies:

Research on protein C concentrate (human) in clinical settings may shed light on the product's tolerance, safety, and effectiveness.

In these trials, the drug's effectiveness in different patient populations is frequently evaluated, dose regimens are assessed, and side events are tracked. A protein C concentrate safety profile was developed based on 121 individuals from clinical trials and compassionate usage in cases of severe congenital protein C deficiency.

The exposure period varied from one day to eight years. The investigator concluded that one patient's lightheadedness and hypersensitivity/allergic responses (rash and itching) were attributable to protein C concentrate.

In clinical investigations, no inhibitory antibodies against protein C concentrate have been found. Nevertheless, the possibility of generating antibodies cannot be completely ruled out.

What Are the Contraindications of Protein C Concentrate (Human)?

• Known Allergies: Do not take this medication with a history of allergies to human plasma or any ingredients in Protein C concentrate.

• Hypersensitivity: Protein C concentrate (human) should not be used by patients who have previously experienced hypersensitivity responses.

• Thrombosis Risk: There may be instances where a medication is contraindicated due to a thrombosis risk. Before starting therapy, patients who have a higher risk of thromboembolic events may need to be carefully evaluated.

Warnings and Precautions:

1. Hypersensitivity: Protein C concentrate could include tiny amounts of heparin or mouse protein during manufacture. It is impossible to eliminate allergic responses to heparin and/or mouse protein. If indications of hypersensitivity appear, stop the injection or infusion. Treatment for anaphylactic shock should follow current medical guidelines.

2. Transmissible Contagious Agents: Since Protein C concentrate is made from human plasma, it could spread infectious organisms. Viruses like the variant of Creutzfeldt-Jakob disease (vCJD) agent are among the risks.

3. Bleeding Incidents: Studies in the clinical setting have shown bleeding events that may be related to taking anticoagulants simultaneously. It is impossible to exclude Protein C concentrate's involvement in bleeding incidents. The risk of bleeding from tissue plasminogen activator (tPA) may rise when protein C concentrate and tPA are used concurrently.

4. Thrombocytopenia Induced by Heparin (HIT): Protein C concentrate may cause thrombocytopenia due to its tiny levels of heparin. Rapid thrombocyte count reduction is linked to HIT. It might be challenging to diagnose HIT, particularly in acute-phase individuals who have a severe congenital protein C deficit. A platelet count should be taken immediately, and stopping protein C concentrate should be considered.

5. Reduced Sodium Intake and Renal Damage: The maximum daily dose of protein C concentrate contains more than 200 milligrams (mg) of salt, which should be disclosed to patients following a low-sodium diet. Those with renal impairment need to be closely watched for salt excess.

What Are the Drug Interactions of Protein C Concentrate (Human)?

• Anticoagulants: Using anticoagulants concurrently with other anticoagulant drugs may make bleeding more likely. Adjustments to the dose and close observation may be required.

• Platelet Aggregation Inhibitors: Careful evaluation and alterations to treatment regimens may be necessary for a pharmaceutical interaction that impacts platelet function.

Specific Considerations:

• Pregnancy and Lactation: It is unknown if protein C concentrate can damage a fetus or impair a pregnant woman's ability to reproduce. Not researched for use in childbirth or labor. There is no evidence on protein C concentrate in human milk, how it affects breastfed infants, or how it affects milk production. Consider the advantages of breastfeeding for health and development in addition to the mother's clinical requirement for protein C concentrate. Analyze the possible negative effects of protein C concentrate or the underlying maternal disease on the breastfed child.

• Pediatrics: There may be differences in the safety and effectiveness of protein C concentrate (human) between pediatric and adult populations. It could be necessary to modify the dose for pediatric patients.

• Liver Impairment: Patients using protein C concentrate may need to have their doses altered or their liver or kidneys closely monitored.