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Zolgensma contains the main active ingredient, Onasemnogene abeparvovec, which is indicated for the treatment of spinal muscular atrophy in patients under 2 years of age. However, this therapy is very expensive and not yet popular in Vietnam.
1. Uses of Zolgensma
Zolgensma is a related viral vector-based gene therapy indicated for the treatment of pediatric patients under 2 years of age with spinal muscular atrophy. Spinal muscular atrophy is a rare disease that causes motor impairment and premature death from respiratory muscle paralysis, especially in young children. To treat this terrible disease, doctors around the world will use gene replacement therapy - Zolgensma. However, this promising therapy is still a dream for most Vietnamese patients because of the high cost.
2. Dosage of the drug Zolgensma
The dose of Zolgensma is determined according to the patient's body weight. The recommended dose is 1.1 × 1014 vg/kg. Patients with renal impairment : No dose adjustment of Zolgensma in patients with renal impairment Patients with hepatic impairment : The manufacturer does not instruct the dose adjustment of Zolgensma in patients with hepatic impairment . However, patients with pre-existing liver failure may be at increased risk of hepatotoxicity. Therefore, special caution should be exercised when using this drug in this population.
3. How to take Zolgensma
3.1. Pre-infusion precautions Due to the increased risk of serious immune reactions, Zolgensma should be withheld in patients with concomitant infections until the infection has resolved. Assess liver function, creatinine, blood count Perform baseline testing for the presence of anti-AAV9 antibodies One day prior to Zolgensma infusion, initiate systemic corticosteroids equivalent to oral prednisolone 1 mg/kg daily days for a total of 30 days. Thaw Zolgensma before use. Zolgensma will thaw in about 12 hours if refrigerated or in about 4 hours at room temperature. After thawing, Zolgensma is a clear to slightly opalescent, colorless to translucent white, grainless liquid. Visually inspect the vial prior to infusion. Do not use the vial if there are particles or discoloration. Do not shake the vial. Withdraw the appropriate amount of drug from the vial into a syringe, remove air from the syringe, cap the syringe and store at room temperature until infusion to the patient. Use Zolgensma within 8 hours of drawing the medicine into the syringe. Do not refreeze the drug. 3.2. How to infusion Use Zolgensma as a single intravenous infusion. Place a catheter into a vein (usually a peripheral vein in an arm or leg). IV infusion for 60 minutes Wash the line with saline after the infusion is complete.
4. Zolgensma side effects
Patients using Zolgensma may experience side effects including:
Frequency > 10%: Increase in serum alanine aminotransferase, increase in serum aspartate aminotransferase, appearance of antibodies Frequency 1% to 10%: Vomiting , thrombocytopenia, thrombotic microangiopathy, acute liver failure, acute hepatotoxicity, fever, elevated troponin in the blood
5. What are the precautions when using Zolgensma?
At the end of the 30-day period of systemic corticosteroid therapy, liver function tests include assessment of ALT, AST, total bilirubin, and prothrombin time. For patients with normal clinical findings, total bilirubin, prothrombin time, and ALT and AST levels less than 2 times the upper limit of normal, the corticosteroid dose should be tapered gradually over the next 28 days. Do not abruptly discontinue systemic corticosteroids. If liver function abnormalities persist, continue systemic corticosteroids (equivalent to oral prednisolone at 1 mg/kg/day) until AST and ALT values are both below 2 times the upper limit of normal. , then gradually reduce the dose of corticosteroid over 28 days or longer as needed. If abnormal liver function persists, consult a gastroenterologist or hepatologist. Use of Zolgensma in premature infants who have not reached gestational age is not recommended because concomitant treatment with corticosteroids may adversely affect neurodevelopment in the infant. Zolgensma should be withheld until the child reaches gestational age. Thrombocytopenia: Monitor platelet counts prior to Zolgensma infusion and weekly for the first month, then weekly for the second and third months until the platelet count returns to baseline. Thrombotic microangiopathy (TMA): If patient has clinical and/or laboratory abnormalities, consult a pediatric hematologist or pediatrician immediately for appropriate management. clinical. Increased troponin-I: Monitor troponin-I prior to infusion of Zolgensma and weekly for the first month, then monthly for the second and third months until troponin-I levels return to normal. Certain vaccines, such as measles, mumps and rubella (MMR) and varicella are contraindicated in patients receiving steroids at immunosuppressive doses (20 mg/day or 2 mg/kg/day). prednisolone for at least 2 weeks). Therefore, it is necessary to adjust the vaccination schedule to match the use of corticosteroids before and after Zolgensma infusion. Zolgensma therapy should be carefully considered in patients with hepatic impairment. Cases of severe liver injury and acute liver failure have been reported in patients receiving Zolgensma and with pre-existing liver disease. Contact a physician immediately if the patient's skin and/or whites turn yellow. Above is an overview of the drug Zolgensma. If you have questions or need more information about your medication, you should contact your doctor.
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