Revolutionary hepatitis C therapy now available for children in Poland

What is hepatitis C? 

Hepatitis C is a liver disease caused by the hepatitis C virus (HCV). If left untreated, it naturally progresses to cirrhosis. In cases of significant fibrosis, cancerous changes may also occur. Hepatitis C can lead to a variety of extrahepatic manifestations, including reduced quality of life. 

How do children become infected?

Most children acquire the virus from their infected mothers. We know that every mother wants to give birth to a healthy child. When a baby is born infected and the mother knows she was the source of the infection, it can be emotionally devastating. We see that such mothers often live with guilt and elevated stress levels. 

What is the likelihood of transmission from mother to child? 

Women typically learn they are infected during routine prenatal testing. They immediately go on to ask, “Will my child be healthy?” Fortunately, the risk of mother-to-child transmission is relatively low at about 6%. However, for families who fall into that 6%, the disease becomes a 100% reality. 

Does infection always lead to illness in children?

In general, 60–70% of those infected develop chronic infection, which leads to chronic hepatitis. However, 30–40% – primarily young children – can spontaneously clear the virus, typically by the age of 4 to 7. 

Is that why hepatitis C in children was previously considered not to require treatment? 

Yes, that was the prevailing view for many years. Until recently, children were not included in therapeutic programs. It was assumed that the disease progressed slowly and mildly in pediatric patients. But many children born with HCV carry the virus for life. Over time, liver damage inevitably occurs. 

Does that mean hepatitis C in children can be dangerous?

Absolutely. Large-scale studies from the UK show that if a child becomes infected, there is a 32% chance they will develop cirrhosis by the age of 32. That amounts to one in three adults in their early 30s living with the long-term consequences of pediatric HCV infection, which is not a low number. This is especially true because these are young people who should be healthy and professionally active.

How have children with hepatitis C been treated so far? 

For many years, interferon-based therapies were used for both adults and children. Interferon is a substance that stimulates the immune system to fight the virus, but its effectiveness was only around 50–60%. It had to be administered via injections over long periods – up to a year – and caused many side effects. In children, it could impair growth and development, and sometimes these effects persisted even after treatment ended. Around a decade ago, direct-acting antiviral (DAA) therapies – interferon-free treatments that directly target the virus – revolutionized adult care. These therapies are nearly 100% effective, have minimal side effects, and only require 2–3 months of treatment. Unfortunately, until recently, these advanced treatments were not available for the youngest patients. This is because pediatric drug research and approvals are often delayed by several years compared to those for adults.  

Why is that? Are clinical trials involving children more difficult?  

Children are considered a particularly vulnerable population. A child is not simply a small adult but a developing organism with critical developmental milestones. Treatment must not interfere with this development. Therefore, we typically wait for evidence from adult trials to confirm a drug’s safety and efficacy before initiating pediatric studies.

You led the PANDAA-PED study, which evaluated interferon-free therapy in children. What was your approach?

Our entire team was excited about this project from the beginning. We were testing a drug already known to be highly effective in adults. Parents of affected children had been waiting for this opportunity, and it felt like salvation for many of them. We approached the study with great hope, assuming the treatment would be equally effective in children. However, assessing safety was our top priority.

Which children were eligible for the study, and how large was the group?

Globally, an estimated 56 million people are infected with HCV, but only about 3 million are pediatric patients, scattered around the world. Moreover, most people with hepatitis C are unaware they are infected. That makes it challenging to gather a large pediatric study cohort. Our study included 50 children aged 6 to 18 from across Poland—past, current, and waiting patients. 

Did the children in the study show any symptoms of illness?

Stereotypically, one might expect a teenager with lifelong hepatitis C to look jaundiced, stunted, developmentally delayed, cachectic, with ascites even. In reality, most of our patients were thriving and appeared healthy, and many played sports like soccer. Of course, some reported symptoms such as gastrointestinal discomfort, fatigue, or excessive sleepiness. But in general, hepatitis C remains asymptomatic for a long time. 

How did the study proceed? Did the children stay in the hospital? How was the medication administered?

Children visited our clinic, sometimes from distant parts of Poland. The first visit was the most comprehensive, involving full diagnostic testing to assess the disease stage and a thorough explanation of study protocols. We created a space where parents could ask all their questions. I often say it is the physician’s job to worry about treatment, not the patients’ or their families’. Once enrolled, patients received the medication (sofosbuvir/velpatasvir) in tablet form—a monthly supply to take home. They returned each month for follow-up and the next dose. At each visit, we examined the children, checked for the presence of the virus, and asked about any side effects. The full course of treatment lasted three months. The most critical visit occurred three months after completing therapy. That’s the standard benchmark for treatment success: if the virus remains undetectable three months post-treatment, the patient is considered cured. The assumption is that if the virus were still present in the body, it would multiply enough within three months to become detectable. 

What were the final results?

The treatment with sofosbuvir/velpatasvir was 100% effective, that is every child was cured. Hepatitis C is the only chronic disease we can cure completely with just three months of oral medication. That’s extraordinary. Working on this project brought immense satisfaction to our entire team. And the gratitude of the parents is added value. Nothing compares to that. 

You mentioned testing for the virus. Did you also assess the children’s mental well-being? 

Our primary goal was clinical: to offer access to modern treatment and prove its efficacy and safety. However, we also wanted to explore additional benefits like improved quality of life for the children and their families. Before and after treatment, parents and children completed quality-of-life questionnaires. Analysis showed significant improvement in overall health and activity scores after therapy.

Will the children who participated require further follow-up? 

The final study visit occurred one year after treatment, confirming long-term efficacy and no adverse effects on development. However, follow-up is still necessary for children who showed signs of liver damage before treatment. We know that fibrosis, once present, can carry a continued risk of carcinogenesis, even after viral elimination. For these patients, semiannual monitoring is essential. For those whose livers were healthy before and remained healthy post-treatment, further follow-up is not strictly necessary. Still, we offer annual check-ups – and many families choose to take advantage of them. 

Are there any special recommendations for children after treatment, such as dietary restrictions?  

No special precautions are required following successful HCV treatment. As with any healthy child, we recommend a balanced lifestyle that includes physical activity. 

Is this modern interferon-free therapy now available for children in Poland?

Yes – that was our ultimate goal. Our study has resulted in five published papers showing 100% efficacy, an excellent safety profile, and no negative impact on child development during or after treatment. In some cases, liver changes even began to reverse during treatment. These findings strongly support including pediatric patients in Poland’s National Health Fund (NFZ) therapeutic program. Based on our results, the Medical Research Agency submitted a formal application to the Ministry of Health. A positive recommendation was issued by the President of the Agency for Health Technology Assessment and Tariff System (AOTMiT). As a result, beginning April 1, 2025, the hepatitis C interferon-free therapeutic program in Poland has been expanded to include pediatric patients as young as three years old. 

Interview by: Iwona Kołakowska
Photo: Michał Teperek
Communication and Promotion Office