Experiences with treating PBC with obeticholic acid in Brno's CKTCH
MUDr. Libuše Husová, Ph.D., shared the current insights from the therapy of patients with primary biliary cholangitis (PBC) using obeticholic acid (OCA) gained at the Center for Cardiovascular and Transplantation Surgery in Brno (CKTCH) during the XLVIII May Hepatology Days.
Monitored Cohort and Treatment Progress
CKTCH began using OCA for treating patients with PBC in August 2019, shortly after reimbursement was established. So far, they have administered it to 15 female patients, with an average age of 59 years (40–77). The initial liver condition varied among these patients, ranging from mild fibrosis to cirrhosis (2 patients). The initial daily dose of OCA was 5 mg.
After 3 months, therapy was discontinued for 5 patients because they did not meet reimbursement criteria (a 10% reduction in ALP from the original value). This was not due to adverse effects (AEs) or intolerance to the treatment. Two patients were placed on the liver transplant waiting list 3/4 year after stopping obeticholic acid.
The remaining 10 patients continued the therapy, and after another 3 months, i.e., 6 months of treatment, the daily dose was increased to 10 mg for 2 patients due to insufficient response.
To date, 7 patients have undergone a one-year treatment follow-up. Four of them continue the treatment, and three had to stop, again solely due to not meeting reimbursement criteria (no AEs were observed, and there was no progression of PBC or decline in liver function). Three other women have been undergoing treatment for a shorter period.
Main Findings After One Year of OCA Treatment
- Some women experienced a reduction in bilirubin levels, with no patient exceeding the upper limit of normal.
- ALP values decreased markedly in the first months, sometimes by more than 50% of the value after the first 3 months. The decline between the 6th and 12th months was much slower.
- For two patients with initial liver stiffness F3, liver function normalized after one year.
Effective and Well-Tolerated Therapy
Dr. Husová concluded by stating that treatment with obeticholic acid is undoubtedly a good choice for these indicated patients. It is very well tolerated, has minimal adverse effects, and none were observed in the monitored patients. She noted that all patients reported a reduction in fatigue and reminded that therapy discontinuation occurred only due to not meeting reimbursement criteria, not due to intolerance or AEs. However, treatment is restricted to specific centers. It can be indicated in patients who have documented intolerance to ursodeoxycholic acid (UDCA) or have been taking UDCA for at least 1 year and meet one of the following conditions after 12 months of treatment: serum ALP activity > 1.67 times the upper limit of normal, or elevated total serum bilirubin concentration to < 2 times the upper limit of normal.
Eva Srbová
editor proLékaře.cz
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