ashington [US],: To assess the effectiveness of novel medicines and gauge the severity of the deadly disease cystic fibrosis , researchers have created a test that’s urine that measure it.
The test can also reveal the extent to which the patient’s medical treatment is of benefit. This has been shown by a new study from Aarhus University and Aarhus University Hospital that has just been published in the scientific journal Annals of Internal Medicine.
Now, by focusing on the kidneys and the urine, the researchers have developed a simple test, which likely shows how effective the treatment is for the individual patient.
Better and easier than testing sweat
Until now, measurement of the chloride concentration in sweat has been the most widely used method of evaluating a patient’s CFTR function. However, this method is time-consuming, requires experienced staff, has great intra-individual variation, and does not sufficiently reflect the severity of the disease. It is thus an important discovery that the excretion of bicarbonate in the urine can be used to characterise CFTR function and thus disease severity.
There are high expectations that the new medication will significantly improve the health of patients. In this study, the researchers examined 50 adult patients with the disease both before and after treatment with Kaftrio.
Another important finding is a clear correlation between the results of the urine test and disease severity, such as the degree of reduced lung function.
The new treatment is expected to significantly improve the life expectancy and quality of life of patients with cystic fibrosis. The study by Aarhus University and Aarhus University Hospital finds that after six months of treatment with Kaftrio, patients on average achieve approximately 70 percent of the base excretion rate seen in healthy control subjects.
“We know that the medicine is absorbed, metabolised and excreted differently from person to person. If, with a simple test, we can monitor the effects of the medicine for the individual patient, then we expect that patients will be able to achieve a better treatment result,” said Jens Leipziger.