# NACFC2021 – Orkambi and Symdeko May Improve Childrens Liver Health


Treatment with Orkambi (lumacaftor / ivacaftor) and / or Symdeko (tezacaftor / ivacaftor) may reduce liver damage in children and adolescents with cystic fibrosis (CF) who have liver damage, new data show.

“We detected improvements in validated non-invasive biomarkers of hepatic fibrosis [scarring] and cirrhosis in children and adolescents treated with “these therapies,” the researchers wrote in a poster presented at the 2021 North American Cystic Fibrosis Conference (NACFC), to be held virtually Nov. 2-5.

The results of the research were discussed in a presentation titled “Impact of lumacaftor / ivacaftor and tezacaftor / ivacaftor on children’s liver health. “

Orkambi and Symdeko are both combination therapies containing two CFTR modulators – drugs designed to increase the functionality of the CFTR protein, the defect of which causes cystic fibrosis, in patients with specific mutations causing the disease.

Cystic fibrosis is generally considered to be primarily a lung disorder, given the build-up of abnormally sticky and thick mucus it creates. But liver disease can be a serious problem in cystic fibrosis. This is because cirrhosis, a form of advanced liver fibrosis (scarring), is the third most common cause of death in people with the genetic disease.

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Although Orkambi and Symdeko have been shown to be effective in improving lung function in eligible patients, their effect on liver health is not well understood.

Now, a team of scientists from Stanford University in California has conducted an analysis of data from 50 children and adolescents with cystic fibrosis who were treated with Orkambi and / or Symdeko at their center. All patients were treated for at least six months and their liver health was assessed before and after treatment. These patients had a mean age of 11.2 years at the start of CFTR modulator therapy.

Of the 50 children, 12 had liver damage, defined as evidence of liver damage based on imaging and / or abnormal levels of certain markers of liver health.

Collectively analyzing data from all patients, the researchers noted no substantial difference in the occurrence of abnormal liver health tests before and after starting CFTR modulator therapy. More patients were treated with Orkambi only than with Symdeko only, but a total of 18 participants received both therapies.

“Significant changes” were noted in children with liver damage, the researchers said. Specifically, the data indicated that before starting CFTR modulator therapy, the platelet levels in these children decreased over time. Platelets are cell fragments involved in clotting and are also important indicators of liver and kidney health.

Notably, after patients started treatment, their platelet levels generally stabilized, according to Zachary Sellers, MD, PhD, a professor at Stanford University who presented the results to NACFC.

The data also suggests that levels of ALT and GGT, two markers of liver damage, decreased after treatment with CFTR modulators in children with liver damage, Sellers said. Such changes have not been seen in children without liver damage. Levels of GPR, or GGT-to-Platelet Ratio, a marker of liver scarring, were also reduced with treatment.

“We have found that treatment with Orkambi and / or Symdeko improves specific biomarkers… of liver damage,” Sellers said.

A separate to study, published in August, also found that Orkambi may decrease markers of liver damage in patients with liver damage, Sellers noted.

Collectively, these data suggest that “CFTR modulators may benefit cystic fibrosis patients with hepatic impairment, even regardless of whether or not the patients have existing lung disease,” he concluded.

Editor’s Note: The Cystic Fibrosis News Today team will be covering the 2021 Cystic Fibrosis Virtual Conference (NACFC) from November 2-5. Go here to see the latest stories from the conference.

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