Glucocerebrosidase (Taliglucerase alfa Injection) is a plant-derived recombinant enzyme replacement therapy that has been approved in children and adults with Type-1 Gaucher disease.

Glucocerebrosidase (Taliglucerase alfa Injection) Uses:

• Gaucher disease:

  • It is used in the management of adult and pediatric patients with a confirmed diagnosis of type 1 Gaucher disease.

Glucocerebrosidase (Taliglucerase alfa Injection) Dose in Adults

It is important to give pretreatment with antihistamines and corticosteroids for the prevention of subsequent infusion reactions in patients with an infusion reaction requiring symptomatic treatment. However, during clinical studies, patients were not routinely pre-medicated prior to infusion.

Glucocerebrosidase (Taliglucerase alfa Injection) Dose in the treatment of Gaucher disease (type 1):

• IV: it is given as 60 units/kg every 14 days. The dosing is individualized based on disease intensity.

• Conversion from imiglucerase:

  • Start taliglucerase alfa using the patient’s same prior imiglucerase dose and administer after every 14 days.

It is noteworthy that conversion to taliglucerase alfa is based on a single study of patients stabilized on a biweekly imiglucerase dose for more than 6 months.

Glucocerebrosidase (Taliglucerase alfa Injection) Dose in  Childrens

Pretreatment should be done with antihistamines, antipyretics or corticosteroids can be considered for the prevention of subsequent infusion reactions in patients with an infusion reaction requiring symptomatic treatment. However, during clinical studies, patients were not routinely pre-medicated before infusion.

Glucocerebrosidase (Taliglucerase alfa Injection) Dose in the treatment of Gaucher disease (type 1):

• Children ≥4 years and Adolescents:

  • IV: 60 units/kg/dose every 2 weeks

  • Conversion from imiglucerase:

    • Start taliglucerase alfa using the patient’s same previous imiglucerase dose and administer every 14 days.
    • The dose can be adjusted if needed to achieve and maintain therapeutic goals. The conversion to taliglucerase alfa is based on a single study of patients stabilized on a biweekly imiglucerase dose for more than 6 months.

Taliglucerase alfa (Elelyso) Pregnancy Category: B

• Studies on animals for reproduction have not revealed any adverse effects.
• Pregnancy may exacerbate type I Gaucher Disease or cause new symptoms.
• If the disease is not controlled, women with type I Gaucher disease are at greater risk of miscarriage or spontaneous abortion.
• Negative pregnancy outcomes such as hepatosplenomegaly or low platelet count may occur.
• This could lead to increased bleeding and postpartum hemorhage that requires transfusion.

Use of Taliglucerase alfa during lactation:

• However, it has not been confirmed in breast milk.
• Nursing infants would likely have their digestion systems degraded by any enzymes they ingest.
• Consider the benefits of breastfeeding to your infant versus the possibility of bone density loss in your mother.
• According to literature, breast-feeding should be considered during therapy. It must also take into consideration the risks to the infant as well as the benefits to the mother.

Dose in Kidney Disease:

There are no dose adjustment requirement ins renal diseases.

Dose in Liver disease:

There are no specific dose adjustment required in hepatic impairment.

Common Side Effects of Glucocerebrosidase (Taliglucerase alfa Injection):

• Central Nervous System:

  • Headache

• Gastrointestinal:

  • Vomiting

• Hypersensitivity:

  • Hypersensitivity Reaction

• Immunologic:

  • Antibody Formation

• Neuromuscular & Skeletal:

  • Arthralgia
  • Limb Pain

• Respiratory:

  • Pharyngitis

Less Common Side Effects of Glucocerebrosidase (Taliglucerase alfa Injection):

• Cardiovascular:

  • Flushing

• Central Nervous System:

  • Dizziness
  • Fatigue

• Dermatologic:

  • Pruritus
  • Urticaria
  • Skin Rash

• Gastrointestinal:

  • Nausea
  • Abdominal Pain

• Hypersensitivity:

  • Fixed Drug Eruption
  • Anaphylaxis

• Neuromuscular & Skeletal:

  • Back Pain

Contraindication to Glucocerebrosidase (Taliglucerase alfa Injection):

• There are no contraindications in US labeling. Canadian labeling does not contain any contraindications.
• However, severe hypersensitivity to taliglucerasealfa or any component should be avoided.

Warnings and precautions

• Formation of antibodies:

  • It has been observed that IgG anti-drug antibody (ADA) was developed. It is unknown if this has any clinical significance.
  • Antibody development should be closely monitored in patients who have developed immune reactions or infusion reactions to taliglucerasealfa, or who have had an immunological response after receiving other enzyme replacement therapies. It is unknown if antibodies are associated with a greater risk of infusion reactions.

• CNS effects

  • The therapy has been associated with dizziness and fatigue. 
  • Patients should exercise caution when operating machinery or driving.

• Hypersensitivity/anaphylactoid reactions

  • Anaphylaxis and severe hypersensitivity reactions have been reported. 
  • These reactions can occur up to three hours after infusion.
  • It is important to have the right medical assistance available.
  • The severity and type of reaction should be taken into consideration. 
  • For mild reactions, it may include temporarily stopping or slowing down the infusion or pre-medicating for antihistamines or steroids.
  • Pretreatment can prevent future reactions.
  • Observe your patient before and after the infusion.
  • If you experience severe reactions, stop the infusion immediately and seek appropriate treatment. Restart the infusion under strict supervision.

Monitoring parameters:

• Hemoglobin and platelet count.
• Angiotensin-converting enzyme levels.
• Tartrate-resistant acid phosphatase.
• Chitotriosidase and IgG anti-drug antibody formation (in patients who experience, or previously experienced, immune or infusion reactions to enzyme replacement therapy).
• Liver and spleen volume.
• Bone density.
• ECG, echocardiogram, and chest x-ray.

How to administer Glucocerebrosidase (Taliglucerase alfa Injection)?

IV:

• Administer IV over a minimum infusion time of 60 minutes (usual infusion time: 60 to 120 minutes).
• Administer using a low protein-binding infusion set with a 0.2-micron in-line filter.
• Initiate infusion at a rate of 1.2 mL/minute; the rate may be increased, but not to exceed 2.2 mL/minute depending upon the patient tolerance.

Mechanism of action of Glucocerebrosidase (Taliglucerase alfa Injection):

• Taliglucerase Alfa is an analog to glucocerebrosidase.
• It is made using recombinant DNA technology and plant (carrot cell) culture.
• Gaucher's disease is known to be deficient in glucocerebrosidase. 
• It is necessary to catalyze the hydrolysis glucocerebroside into glucose and ceramide. This reduces liver size and improves anemia.

Half-life elimination:

• Pediatric patients: 33 to 37 minutes.
• Adults: 19 to 29 minutes.
• Its half life is dose dependent and increases with increasing the dose.

International Brand Names of Taliglucerase alfa:

• Eleliso
• Elelyso
• Eleyso
• Uplyso

Taliglucerase alfa Brand Names in Pakistan:

No Brands Available in Pakistan.