POLPHITT / POLSTS
A non-commercial clinical trial for children and adolescents with primary malignant liver tumours (HB/HCC) – POLPHITT, together with the otoprotective substudy POLSTS.
About the study
POLPHITT (Paediatric Hepatic International Tumour Trial) is a non-commercial clinical trial involving children and adolescents with primary malignant liver tumours – hepatoblastoma (HB) and hepatocellular carcinoma (HCC) – together with the POLSTS substudy.
The study is conducted through collaboration between specialised paediatric oncology centres across Poland, coordinated by the Medical University of Gdańsk and involving centres in Kraków, Warsaw, Wrocław, Szczecin, Zabrze, Katowice, Białystok and Poznań.
POLPHITT
The primary objective of POLPHITT is to establish the most appropriate treatment strategy for specific groups of patients with hepatoblastoma or hepatocellular carcinoma. The study aims to reduce treatment intensity in patients with favourable prognosis in order to minimise short- and long-term side effects, while intensifying surgical approaches and introducing new treatment pathways for children with high-risk disease.
POLSTS
POLSTS is an otoprotective substudy for paediatric patients with liver cancer treated with cisplatin. It evaluates the use of sodium thiosulfate (STS), a thiol-containing compound with reducing properties, to help prevent cisplatin-induced hearing loss.
Why is this important?
Cisplatin is one of the key medicines used in the treatment of hepatoblastoma, but its use may be associated with kidney toxicity and hearing loss. Hearing impairment can affect a child’s daily functioning, including learning ability, social and emotional development, and fatigue during school activities, even when only high-frequency hearing is affected.
How does STS work? (POLSTS)
The exact mechanism through which STS reduces cisplatin-related nephrotoxicity and hearing loss is not fully understood. Proposed explanations include its ability to act as a free-radical scavenger and to neutralise platinum compounds. Some studies have also suggested direct protective effects on cochlear hair cells, potentially reducing the toxic effects of platinum-based chemotherapy on hearing.
Potential clinical benefits and project highlights
- identification of somatic alterations and potentially harmful germline variants associated with liver cancer; transcriptome sequencing will provide gene expression profiles useful for molecular tumour classification and may help identify novel gene fusions and alternative splicing events;
- identification of prognostic and predictive epigenetic biomarkers.
Study population (POLPHITT / POLSTS)
Patients aged ≤18 years with newly diagnosed primary malignant liver tumours: hepatoblastoma (HB) or hepatocellular carcinoma (HCC). The study plans to enrol 30 participants.
Inclusion criteria
- clinical diagnosis of HB and histologically confirmed HB or HCC;
-
histological confirmation of HB is required unless the patient meets all other criteria but:
- is too ill to safely undergo a biopsy;
- has a tumour causing critical organ dysfunction (e.g. respiratory failure, abdominal compartment syndrome or urinary tract obstruction);
- has uncorrectable coagulopathy.
- age ≤18 years;
- written informed consent.
Exclusion criteria
- any previous chemotherapy or current anticancer treatment;
- relapsed disease;
- previous solid organ transplantation (other than orthotopic liver transplantation – OLT);
- uncontrolled infection;
- inability to comply with the study protocol for any reason;
- another malignant disease;
- pregnancy or breastfeeding.
Eligibility is assessed individually on the basis of medical records and protocol-defined criteria.
Participating centres
POLPHITT/POLSTS is conducted in specialised paediatric oncology centres across Poland.
Gdańsk
University Clinical Centre in Gdańsk
Department of Paediatrics, Haematology and Oncology
Joanna Stefanowicz, MD, PhD
Warsaw
The Children’s Memorial Health Institute
Department of Oncology
Ewa Święszkowska, MD, PhD
Szczecin
University Clinical Hospital No. 1, Pomeranian Medical University
Department of Paediatrics, Oncology and Paediatric Immunology
Paweł Wawrykow, MD, PhD
Wrocław
University Clinical Hospital in Wrocław
Department of Bone Marrow Transplantation, Oncology and Paediatric Haematology
Katarzyna Wilczyńska, MD
Kraków
University Children’s Hospital in Kraków
Department of Paediatric Oncology and Haematology
Katarzyna Pawińska-Wąsikowska, MD, PhD
Poznań
Karol Jonscher Clinical Hospital, Poznań University of Medical Sciences
Department of Paediatric Oncology, Haematology and Transplantology
Patrycja Marciniak-Stępak, MD, PhD
Study recruitment
If you would like to learn more about study eligibility or participation, please contact the study team directly.
study coordinator Hanna Garnier, MD, PhD
coordinator assistant Ewelina Wojciechowska, MD
tel. 58 584 49 60
centre Department of Paediatric Surgery and Urology, Medical University of Gdańsk
Organisational information
The project is funded by the Polish state budget through the Medical Research Agency (MRA).
- project number: 2021/ABM/01/00009;
- funding call: ABM/2021/1;
- agreement number: 2021/ABM/01/00009-00.
Frequently asked questions
No. Participation is entirely voluntary. Families are encouraged to ask any questions before making a decision, and choosing not to participate does not affect the child’s access to treatment or medical care.
Yes. Participants may withdraw from the study at any time without providing a reason. This does not affect access to treatment or future medical care.
Participation includes examinations and visits related to diagnosis, treatment and follow-up. Depending on the clinical situation, these may include blood tests, imaging procedures, hearing assessments, cardiac examinations and follow-up visits.
Follow-up after treatment continues for at least two years. During this period, the study team monitors the child’s health, potential treatment-related side effects and the risk of disease recurrence.
Many procedures are part of standard clinical care. Some participants may require additional visits, blood sampling or monitoring. For patients participating in POLSTS, hearing assessments are an important part of the study.
No direct benefit can be guaranteed for every participant. However, the study aims to improve treatment outcomes and reduce treatment-related side effects in children and adolescents with liver cancer.
Participants remain under continuous medical supervision. Any significant side effects or important new information related to treatment will be discussed with patients and their families, and further management will be decided individually.
You may contact the study team at any time. Contact details are provided in the study contact section.
Study team
principal investigator
Piotr Czauderna, MD, PhD, Professor
study coordinator
Hanna Garnier, MD, PhD
coordinator assistant
Ewelina Wojciechowska, MD
Key information
project number
2021/ABM/01/00009
funding call
ABM/2021/1
agreement number
2021/ABM/01/00009-00
study population
children and adolescents ≤18 years (HB/HCC)
planned enrolment
30 participants
status
recruiting
Contact
Department of Paediatric Surgery and Urology
Medical University of Gdańsk
ul. Smoluchowskiego 17
80-214 Gdańsk, Poland
tel. 58 584 49 60
e-mail:
phitt.poland@gmail.com
website:
polphitt-polsts.gumed.edu.pl