Ethnic Minorities More Likely to Be Ineligible for Kaftrio, UK Study Finds

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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People from ethnic minority backgrounds were significantly less likely to be eligible for the cystic fibrosis therapy Kaftrio (ivacaftor/tezacaftor/elexacaftor) than white patients, according to a recent U.K. study.

Given that the therapy is the most effective CF therapy on the market, “the CF community should urgently address the unmet need for effective targeted therapies” for ineligible patients, the researchers added.

The study, “Who are the 10%? — Non eligibility of cystic fibrosis (CF) patients for highly effective modulator therapies,” was published in Respiratory Medicine. 

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CF is caused by mutations in the CFTR gene, leading to reduced or impaired function of the CFTR protein. CFTR modulators are a class of treatments that work in various ways to increase CFTR’s function for patients with certain types of mutations.

Vertex Pharmaceuticals‘ Kaftrio (marketed as Trikafta in the U.S.) is a highly effective CFTR modulator that’s approved in the U.K for patients ages 6 and up with at least one F508del mutation, the most common CF-causing mutation.

With Kaftrio’s emergence, an estimated 90% of CF patients will have access to a highly effective therapy.

However, about 10% of patients are still ineligible for Kaftrio due to their genetic profile, age, or lung transplant status, highlighting a need to better understand the clinical characteristics of this population to develop more effective treatments. In particular, the question remains as to whether people from ethnic minority backgrounds are more likely to be ineligible for the treatment.

Researchers in the U.K. investigated this question by evaluating CF patient data from the UK Cystic Fibrosis Registry in October 2020. 

Overall, 9,887 patients were included in the analysis. Of them, 852 people, or 8.6%, did not have a copy of the F508del mutation and were ineligible for Kaftrio treatment.

Results showed no overall differences between treatment-eligible or -ineligible patients in terms of sex, socioeconomic status, nutritional status, or country in which patients received treatment.

The Kaftrio-ineligible patients were significantly older, with a mean age of 25.2 years compared with a mean age of 22.2 years in the eligible group.

In general, clinical data indicated that those in the treatment-ineligible group may be less severely affected than those with an F508del mutation. Specifically, more of these patients had sufficient levels of pancreatic enzymes, lower prevalence of chronic Pseudomonas aeruginosa infections, and a lower prevalence of CF-related diabetes. Their best lung performance measurement was also significantly higher than those seen in the treatment-eligible group.

Significant differences were observed in overall ethnicity between treatment-eligible vs. -ineligible patients. Specifically, 95.6% of treatment-eligible patients were white compared with 70% of the treatment-ineligible patients. More patients without an F508del mutation were of Asian background (19.2%).

The percentages of other ethnic minority groups, including Black, mixed, or other, were also higher in the treatment-ineligible group.

“This disparity in access to the most effective treatments for ethnic minority patients will negatively impact a group who already face disparate health outcomes,” the researchers wrote.

“Understanding the scale of this difference will help to focus information shared with patients and families, ensuring that optimal standard CF care is given to these higher risk groups, and deciding research priorities in investigating new treatment and organisation of clinical services,” they added.

More ethnic minorities are needed in clinical trials to support more equal access to new and effective therapies, the researchers noted.