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Phase III Results: Telaprevir, Boceprevir Improve HCV Cure Rates

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Potential for Misuse Is ‘Huge’

Before next year’s American Association for the Study of Liver Diseases meeting,

Dr. Paul J. Pockros

"we are going to see the approval of two direct-acting antiviral drugs – telaprevir and boceprevir. We anticipate the widespread use of these drugs in the United States and the European Union, as they’ve been shown to improve sustained virologic response rates by approximately 75% in treatment-naive patients," Dr. Paul Pockros said during the meeting. Unfortunately, there is also a "huge potential" for misuse of these drugs, owing to prescribing physicians’ poor understanding of the therapeutic populations, inadequate viral-assay testing, poor side-effect management, and lack of monitoring for antiviral resistance, he said.

The designs of the phase III trials on which the Food and Drug Administration approval will be based, as well as the resulting treatment regimens, are fairly complex, said Dr. Pockros, "so there will be lots of opportunities to screw things up." For example, he hypothesized, "I am sure that some patients are going to be put on telaprevir for 44 weeks with a 4-week pegylated interferon/ribavirin lead-in [even though the lead-in strategy was evaluated for boceprevir, not telaprevir], and other patients might be put on boceprevir for 12 weeks with no lead-in."

For optimal safety and efficacy, Dr. Pockros stressed, physicians must have a good understanding of the treatment regimens, particularly in special populations, and they must actively and frequently monitor for antiviral resistance. Additionally, physicians should anticipate problems with adherence, which is already an issue for some patients on standard therapy, he said, noting that – despite the shorter treatment duration – adding a third drug with its own set of side effects to the mix may not be realistic for patients with a history of poor compliance.

The objective for physicians should be "to keep our eyes on the ball," said Dr. Pockros. "Our primary goal moving forward with all of the new drugs is going to be eradicating the virus."

Paul J. Pockros, M.D., is head of the division of gastroenterology/hepatology and director of the Liver Research Consortium at the Scripps Clinic in La Jolla, Calif. He disclosed relationships with Vertex and Tibotec, which are involved in the development of telaprevir, and Merck, which manufactures boceprevir.


 

All the patients underwent a 4-week lead-in phase of standard therapy followed by a random assignment to continue standard therapy alone or in conjunction with 800 mg of boceprevir taken three times daily. The treatment duration for patients in the boceprevir arm with undetectable HCV RNA at study weeks 8 and 12 was 36 weeks, whereas those patients in whom HCV RNA was detectable at study week 8, but undetectable at study week 12, stopped boceprevir at week 36 but continued standard therapy for an additional 12 weeks, for a total treatment duration of 48 weeks, Dr. Bacon said. Patients in the control group were treated for 48 weeks.

The SVR rates at 24 weeks after treatment conclusion were significantly higher in the boceprevir groups, compared with the control group. In the response-guided and fixed-duration boceprevir groups, the SVR rates were 59% and 66%, respectively, compared with 21% in the control patients, he said.

In all study arms, "previous relapsers and previous null responders fared better than prior nonresponders," Dr. Bacon said, noting that the respective SVR rates for previous relapsers, null responders, and nonresponders were 29%, 7%, and 0% in the control group; 69%, 40%, and 33% in the response-guided therapy group; and 75%, 52%, and 34% in the fixed-duration group.

Although the rates of anemia were significantly higher in the boceprevir arms, "the rate of treatment discontinuation related to side effects was similar across all three arms," Dr. Bacon reported, possibly because the use of erythropoietin was allowed to treat anemia, he said.

The findings of this study answer an important question about response-guided therapy "by confirming that many patients can be treated successfully with a treatment duration that is reduced by 3 months relative to the current standard of care treatment," Dr. Bacon said.

Boceprevir with a standard therapy lead-in strategy was also evaluated in the SPRINT-2 study involving HCV genotype 1 treatment-naive patients, according to Dr. Fred Poordad of Cedars-Sinai Medical Center in Los Angeles. The trial included 1,097 patients who underwent a similar 4-week standard therapy lead-in strategy as defined above, followed by the addition of placebo for 44 more weeks or by the addition of boceprevir, either for 24 more weeks for patients with undetectable HCV RNA at week 8 or for 24 more weeks plus 20 additional weeks of standard therapy for patients with detectable HCV RNA at week 8, but not at week 24, Dr. Poordad explained. Patients with detectable HCV RNA at week 24 were discontinued for futility, he said.

"In both the response-guided and fixed-treatment arms, boceprevir increased viral cure rates significantly, by approximately 70%," Dr. Poordad stated. Specifically, the SVR rate was 63% in the 28-week response-guided group, 66% in the 48-week fixed-duration group, and 38% in the 48-week control group, he said.

In a cohort analysis of treatment response for the study’s 159 black patients, the relative improvement in SVR rates remained significantly improved in the boceprevir arms, although the differences were not as robust, Dr. Poordad said. In this subgroup, the respective SVR rates in the response-guided therapy, fixed-duration therapy, and control groups were 42%, 53%, and 23%, respectively.

The rationale for using a lead-in strategy "is to help physicians identify patient responsiveness to interferon before adding boceprevir," Dr. Poordad explained. This can provide an early indication of the likelihood of treatment success. The advantage of subsequent response-guided therapy, he noted, is that it enables physicians to be flexible in managing their patients’ therapy "by adapting treatment duration to individual patient response."

All the sources disclosed relationships with numerous pharmaceutical companies. Among them, Dr. Jacobson and Dr. Sherman disclosed relationships with Vertex Pharmaceuticals, which manufactures telaprevir. Dr. Jacobson also has a relationship with Tibotec, which also is involved with the development of telaprevir. Dr. Poordad and Dr. Bacon also disclosed relationships with Merck, which manufactures boceprevir. Dr. Pockros disclosed relationships with Vertex and Tibotec, which are involved in the development of telaprevir, and Merck, which manufactures boceprevir.

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