A patient who has undergone Enzyme replacement therapy for chronic liver disease will likely have a limited quality of life. There is a very low risk of severe liver injury with protine replacement medication, although some agents can cause minor elevations in serum enzyme levels. None of the agents have been associated with acute liver failure or chronic hepatitis. However, some patients may develop serious adverse reactions, including rash or anaphylaxis.
Enzyme replacement medication involves replacing the missing or deficient enzyme in the body with an infusion of an protine preparation. This replacement enzyme can be purified from human or animal tissues or developed by novel recombinant techniques. Patients receive this replacement enzyme intravenously or parenterally. Typically, the patient will need to stop taking other medications during this therapy. Some people may experience allergic reactions, rash, difficulty breathing, or anaphylaxis.
Enzyme replacement medication has been shown to improve the symptoms of patients with lysosomal storage disease. It has been used to treat a variety of other disorders, including Fabry disease and Pompe disease. It has also been used to treat mucopolysaccharidoses. It may even reverse the progression of some diseases. Despite the risks, protine replacement therapy can greatly improve the quality of life for patients suffering from lysosomal storage disease.
The effectiveness of protine replacement medication depends on the enzyme that is replaced. For MPS, recombinant protine are administered via intravenous infusions every week. Unlike oral enzymes, ERT is lifelong. Nevertheless, there are some cases of severe infusion reactions. Most patients receive ERT infusions in the hospital setting. However, home treatment is now available for some MPS patients. The effectiveness of enzyme replacement medication depends on its efficacy.
While Enzyme replacement medication is generally effective for non-neuronopathic forms of the disease, it isn't as effective for the neuronopathic forms. Because protine cannot cross the blood-brain barrier, they are only effective for treating patients with Gaucher disease, such as type 2 or type 3. However, the benefits of ERT are significant and deserve a try. There are many people who have successfully benefited from this treatment.
A patient who initially improves during ERT may regress. The development of inhibitory antibodies is also possible in patients with Gaucher disease. These antibodies bind to the Enzyme that is being infused. However, this is rare and less than one percent of patients with Gaucher disease develop inhibitory antibodies. Hence, nurses must be vigilant and take necessary precautions. When an inhibitory antibody develops, the patient may experience adverse reactions to ERT.
Some studies have shown that ERT reduces urinary GAG concentration. However, this effect is short-lived. The treatment can also decrease the size of the liver and spleen. This decrease in volume is expected from the beginning. ERT also has the potential to improve respiratory function and facilitate diaphragm excursions. It is therefore advisable to discuss ERT with doctor before starting it. Thus, growing prevalence of liver disease to foster the ERT market growth in the near future.