Ledipasvir / sofosbuvir (Harvoni) for the treatment of chronic hepatitis C in children

Introduction

The fixed combination ledipasvir / sofosbuvir (trade name: Harvoni) has been approved in Germany since July 2020 for the treatment of hepatitis C in children between the ages of 3 and 12 years. The aim of this treatment is to keep the from multiplying and prevent complications of hepatitis C.

Hepatitis C viruses (HCV) attack the liver, where they can lead to an acute inflammation. There are seven known types of hepatitis C , referred to as genotypes 1 to 7. In up to 80 out of 100 people with hepatitis C, the immune system is unable to successfully fight the on its own. These people develop a permanent (chronic) of the liver which can cause cirrhosis. The liver then starts working less effectively.

As long as the effect on liver performance isn't noticeable, it is known as “compensated cirrhosis." Signs of advanced ("decompensated") cirrhosis include fluid retention in the abdominal cavity, blood clotting problems, and neurological and psychiatric problems, sometimes even as severe as a coma. The risk of liver cancer increases too. It is currently believed that a treatment can reduce the risk of such complications if patients consistently have no detectable virus in their blood following that treatment.

The fixed combination ledipasvir / sofosbuvir has only been approved for the treatment of chronic hepatitis C genotypes 1, 3, 4, 5 and 6.

Application

Ledipasvir / sofosbuvir is available in the form of tablets, in two doses: 45 mg of ledipasvir with 200 mg of sofosbuvir, and 90 mg of ledipasvir with 400 mg of sofosbuvir. The combination of drugs is taken once a day independently of meals. The dose depends on the child’s body weight.

Children who have difficulty swallowing tablets or who weigh less than 17 kilograms can take this drug combination in the form of granules. These are available in two doses: 33.75 mg of ledipasvir with 150 mg of sofosbuvir, and 45 mg ledipasvir with 200 mg sofosbuvir.

The treatment lasts between 8 and 24 weeks. This depends on the severity of the illness, among other things. Depending on the type of and course of the disease, the drug may be combined with ribavirin.

Other treatments

Watchful waiting is the standard treatment option for children who have chronic hepatitis C.

Assessment

In 2020, the Institute for Quality and Efficiency in Health Care (IQWiG, Germany) wanted to find out whether the combination of ledipasvir and sofosbuvir has any advantages or disadvantages compared with watchful waiting in children who have chronic hepatitis C.

For this purpose, IQWiG analyzed three studies that provided at least preliminary answers to some questions. The studies looked into treatment with ledipasvir / sofosbuvir, but didn't compare it directly with another treatment. The results of these kinds of studies are generally less conclusive.

Children with chronic hepatitis C can be divided into different groups based on the genotype of their , the stage of the disease, and their previous treatments. The studies provided data about the following group:

  • Children who are receiving first-line treatment or a subsequent treatment for chronic hepatitis C genotypes 1 or 4 and do not have liver cirrhosis.

The researchers at IQWiG analyzed the data of 157 children in total: All of the children took ledipasvir / sofosbuvir once a day for 12 or 24 weeks. This is what was found:

Secondary diseases: The studies did not last long enough to examine the influence of ledipasvir / sofosbuvir on secondary diseases (diseases caused by the hepatitis C ). But early estimates suggest that the combination of ledipasvir / sofosbuvir offers an advantage here:

  • At least 95 out of 100 children had no detectable viruses in their blood 12 weeks after the treatment ended.
  • One of the studies also provided data that was taken over a period of 24 weeks: After 24 weeks, about 98 out of 100 children had no detectable viruses in their blood.

The watchful waiting approach is not known to, or expected to, have this kind of benefit. But it's not possible to tell exactly how big this advantage of ledipasvir / sofosbuvir actually is.

Life expectancy: None of the participants passed away during the studies.

Severe side effects and stopping treatment due to side effects: Less than 1 out of 100 children had a severe side effect. Less than 1 out of 100 children stopped the treatment due to side effects.

More information

This information summarizes the main results of a review produced by the Institute for Quality and Efficiency in Health Care (IQWiG, Germany). The review was commissioned by the German Federal Joint Committee (G-BA) as part of the “early benefit assessment of medications.” On the basis of this review and the hearings received, G-BA passed a resolution on the added benefit of ledipasvir / sofosbuvir (Harvoni) in children.

Institute for Quality and Efficiency in Health Care (IQWiG, Germany). Ledipasvir/sofosbuvir (chronic hepatitis C in children) - Benefit assessment according to §35a Social Code Book V. Dossier assessment; Commission A20-63. October 29, 2020. (IQWiG reports; Volume 987).

IQWiG health information is written with the aim of helping people understand the advantages and disadvantages of the main treatment options and health care services.

Because IQWiG is a German institute, some of the information provided here is specific to the German health care system. The suitability of any of the described options in an individual case can be determined by talking to a doctor. informedhealth.org can provide support for talks with doctors and other medical professionals, but cannot replace them. We do not offer individual consultations.

Our information is based on the results of good-quality studies. It is written by a team of health care professionals, scientists and editors, and reviewed by external experts. You can find a detailed description of how our health information is produced and updated in our methods.

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Created on January 27, 2021
Next planned update: 2024

Authors/Publishers:

Institute for Quality and Efficiency in Health Care (IQWiG, Germany)

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