Is seripalase (BRINEURA) necessary for long-term use? Analysis of the pros and cons of long-term medication

Seripase (BRINEURA) is an enzyme replacement therapy drug used to treat neuronal zero-aminase deficiency (CLN2 disease) in infants and young children. This disease is an inherited lysosomal storage disease in which patients lack functional enzymes, leading to gradual degenerative damage to the nervous system. Therefore, the therapeutic goal of seripalase is to continuously supplement the missing enzyme to delay disease progression and improve quality of life. This means that seripase generally needs to be used regularly for a long time, usually by intracerebroventricular injection every two weeks to maintain its efficacy.

An obvious advantage of long-term medication is that it can continuously replenish enzymes that are lacking in the body and delay the neurodegenerative process of the disease. Research and clinical practice have shown that patients who continue to receive seripalase treatment have a significantly slower rate of decline in motor ability, language ability and cognitive function, and their quality of life is significantly improved compared with patients who are not treated or who receive short-term treatment. In addition, long-term treatment can also reduce the frequency and severity of epileptic seizures, prolong the ability to live independently, and have a certain effect on reducing family and nursing burdens.

However, there are certain risks and inconveniences associated with long-term use of seripase. Because the drug requires intracerebroventricular injection, long-term treatment increases the frequency of surgical procedures and the risk of infection, bleeding, or local nerve damage. In addition, some patients may experience allergic reactions or immunogenic reactions, leading to the production of antibodies and affecting the efficacy of the drug. Economic cost is also an important consideration for long-term treatment. Seripase is expensive, and each treatment costs a lot, which puts a heavy financial burden on the family.

In clinical application, the pros and cons of long-term medication need to be weighed. The doctor will develop an individualized plan based on the patient's condition, treatment response and tolerance to minimize the occurrence of side effects while ensuring the therapeutic effect. For families, they should cooperate with the medical team in follow-up and care, and regularly assess the patient's neurological function and overall health.

Reference materials:https://www.drugs.com/