Laronidase (Intravenous)

Laronidase is used in alpha-L-iduronidase deficient patients as a replacement enzyme treatment to treat mucopolysaccharide disorders involving storage of this enzyme.

Overview

Laronidase is the drug name of the enzyme iduronidase, which is produced by the ovaries of Chinese hamsters for sale as Aldurazyme. This medication is given as an injection into the veins to treat patients who lack the enzyme alpha-L-iduronidase. The lack of this enzyme creates a condition known as Hurlers syndrome or Hurler-Schele syndrome in patients.

Hurler or Hurler-Schele syndrome, also called mucopolysaccharidosis type I or MPS I, is a build-up of glycosaminoglycans in the body due to the low amounts of alpha-L-iduronidase. Hurler's disease or syndrome is considered the most severe type of mucopolysaccharidosis, which is a genetic disease. Patients suffer from a progressive physical deterioration, mental retardation and often die before they are ten years old due to respiratory or cardiac complications of the disease.

Laronidase, as a replacement for the enzyme lacking in these children, has been proven to reduce the pain and other symptoms that often accompany this condition. Patients also often undergo bone marrow transplants or umbilical cord blood transfusions to treat their conditions as well. There is no known cure for this disease, but a combination of these treatments can help patients live a more normal life.

Conditions Treated

  • Type 1 Mucopolysaccharidosis

Type Of Medicine

  • Hydrolytic Lysosomal Glycosaminoglycan-specific enzyme

Side Effects

Patients may experience unwanted health effects upon the introduction of this enzyme therapy to their body. Report the following symptoms to your doctor, should they occur after treatment:

  • Pain in stomach or abdomen
  • Pus accumulation
  • Pain in back
  • Stools with blood or tar-like appearance
  • Gums bleeding
  • Urine has signs of blood
  • Vision is blurry
  • Pain or tightness in chest
  • Chills
  • Stools are clay-colored
  • Confused demeanor
  • Urine is dark in color
  • Dizzy, drowsy or sleepy
  • Swelling of the face
  • Rapid, irregular heart rhythm
  • Elevated body temperature
  • Red, flushed face
  • Headache
  • Injection area infection, tenderness, redness, itching, lumps, hives, soreness, swelling, warmth or tingling
  • Itchy skin
  • Fainting or becoming lightheaded when rising from seated or lying position
  • Little to no appetite
  • Vomiting
  • Nausea
  • Skin paleness
  • Tiny red dots on skin
  • Gasping for breath
  • Rash on skin
  • Sweating
  • Tender, swollen or red areas of infection
  • Difficulty breathing
  • Halitosis
  • Bruising or bleeding that is unusual
  • Fatigue
  • Weak muscles
  • Vomiting with signs of blood
  • Yellow tinge to skin or eyes
  • Coughing
  • Hives or other swollen patches on sex organs, face, lips, eyes, tongue, hands, throat, feet or legs
  • Loud breathing, wheezing
  • Skin is flushed and red
  • Chest feels tight

Other health symptoms related to a dose of Laronidase, while not dangerous to overall health, can be problematic. Consult your physician on how to alleviate these symptoms if they do not disappear over time:

  • Discolored skin with blistering, coldness, burning or bleeding
  • Loss of sight
  • Aches or pains in the body
  • Vision changes
  • Pains or aches in body
  • Needles and pins, tingling, numb sensations
  • Diarrhea
  • Congested ears
  • Difficulty moving
  • Sensation of pressure
  • Vocal changes or loss
  • Pain or stiffness in muscles
  • Congested nose, sneezing, nose running
  • Joint pain or stiffness
  • Hyperactive muscle reflexes
  • Throat soreness
  • Swollen feet or legs
  • Puffy, swollen face
  • Spider or varicose veins

These are the generally reported effects that some patients experience when given a dose of Laronidase; your experience may vary. Report any changes to your demeanor or health to your physician right away for your own safety.

Dosage

The recommended dosage of Laronidase consists of a treatment regimen that will be carried out in a professional medical setting. You will be administered this medication through intravenous infusion methods by a doctor or nurse or other trained professional.

The manufacturer recommends 0.58 milligrams per kilogram of patient's body weight be administered weekly through an IV. The infusion time is three to four hours. Patients who weigh less than 20 kilograms will receive a total of 100 millilitres of the solution with the medication incorporated. Those who weight more than 20 kilograms will get 250 millilitres of the solution with the medication incorporated.

Interactions

Patients who have had past sensitive reactions to other medications or to Laronidase itself should make this known to their healthcare professional. Additionally, those patients who exhibit sensitive reactions to any animals, foods, perfumes, preservatives or dyes should also let their doctor know of these sensitivities. Review the patient informational brochure that your doctor will provide to you before your first dose of this treatment.

Children under the age of five have not been studied to provide data on how effective Laronidase is on their symptoms. Use in young children has not been established to be safe or effective and is up to the discretion of the attending physician.

The geriatric population has not been studied for use of this drug to treat any conditions. Enzyme therapy with Laronidase may not be appropriate in this age group.

With regard to the use of Laronidase during pregnancy, no safety data has been provided with regard to the development of the fetus during treatment. Use of this drug on women who are pregnant should only be done if the patient clearly needs the treatment and no alternatives are available.

Laronidase may or may not be transported from a treated mother's breast milk to her infant, it is not known at this time. Caution should be taken if considering Laronidase for any form of treatment on a nursing mother.

Laronidase may interact poorly with other medications, including those found over-the-counter, some herbal and vitamin therapies and holistic remedies. For this reason, it is advised that patients disclose any substances they take prior to being treated with Laronidase.

Discuss any foods, tobacco products or alcoholic beverages and their use with Laronidase treatment with your medical team before you partake of any of these substances. Their use may have an effect on how Laronidase reacts with your system.

Patients with health conditions that involve the lungs or breathing capability may not be candidates for treatment with Laronidase or at least should be given this medication with caution. Inform your physician of your full medical history including lung diseases prior to your treatment with Laronidase, as your dose may need to be adjusted.

Warnings

Regular visits to your physician will be required during your enzyme replacement therapy with Laronidase. You will be tested to determine whether this therapy is working to combat the symptoms of your condition without causing any unwanted health issues.

Children under five years old, geriatric patients, women who are pregnant or those that are breastfeeding have not been determined to be candidates for therapy with this medication. The safety of the treatment has not been established in these groups.

A life-threatening reaction known as anaphylaxis is a risk when being treated with Laronidase. Report symptoms such as swelling of the facial features, throat or tongue, itching, skin rash, being lightheaded, difficulty breathing or tightness in your chest and irregular heart rhythms to your physician immediately, should they occur.

Storage

This medication will be stored in a professional medical setting, following all recommendations by the manufacturer for the safe treatment of the medication. The manufacturer recommends that doses of Laronidase be refrigerated but not allowed to freeze. Do not shake the medication and do not use it if it is expired. After diluting the medication with the solution for intravenous administration, it should be used immediately or discarded after 36 hours of refrigerated storage. Observe all expiry dates indicated on the packaging, as there are no preservatives of any kind added to Laronidase doses. Local requirements for safe disposal of expired medication should be followed.

Summary

Hurler's syndrome, a classification of MPS I disease, is a metabolic disease in which the patient is deficient in lysosomal enzymes. These enzymes are required for the normal bodily function of breaking down glycosaminoglycan molecules, which are carbohydrate strains found in the joints, bones, cartilage, corneas, skin and other cells of the body. Typically this disease is first spotted in children, who exhibit signs of slower growth rates and diseases involving their organs. Patients who are not treated typically survive only to age 10 before succumbing to lung or cardiac conditions associated with the disease.

Laronidase has been used as an enzyme replacement therapy in these patients along with bone marrow or umbilical cord blood transplants. With this therapy, patients can experience a more normal life with less pain and symptoms of their disease. This medication is given as an intravenous infusion, in an amount calculated based on the patient's weight and age. The infusion time for the medication is typically over a three to four hour period, taking place in a medical setting such as a clinic or hospital.

Patients with respiratory diseases or those who are very young are not candidates for enzyme replacement therapy with Laronidase, nor are pregnant and breastfeeding women or geriatric patients. Risks associated with this treatment include respiratory problems and sensitivity to the medication known as anaphylaxis.