Therapy Trials and Registries in Paediatric Oncology
Since approximately 1970, cooperative, multicentric clinical trials have been conducted for children and adolescents with cancer. Due to the rarity of such diseases, such trials are necessary to obtain valid data and thus improve treatment results.
Author: Prof. Dr. med. U. Creutzig, Editor: Julia Dobke, English Translation: Dr. med. Gesche Riabowol (nee Tallen), Last modification: 2024/10/25 https://kinderkrebsinfo.de/doi/e1758
Given the rarity of malignant diseases in childhood and adolescence, paediatric oncologists have already been collaborating in the 1970s to share their experiences in treating paediatric malignancies. Six years after Donald Pinkel (Memphis/USA) had presented his treatment results for children with ALL in Munich in 1968, nearly identical results were achieved in the first cooperative trial for treating ALL in Germany. The BFM (Berlin, Frankfurt, Münster) study group founded by H. Riehm in 1976 gained international recognition due to its treatment concept and high cure rate in ALL.
Advances in cancer treatment in children and adolescents are largely due to multicentre cooperative therapy trials. Hence, the Society for Paediatric Oncology and Hematology (GPOH) considers the promotion of therapy studies as one of its main tasks. For the standardisation of first-line treatment, the term "therapy optimising trial" has been established for many diseases. Treatment according to therapy optimising trials is considered the standard of patient care in paediatric oncology and haematology both nationally and internationally.
As of today, paediatric oncologists in more than 70 clinics throughout Germany, Austria, and Switzerland participate in this network. During the last few years, more and more cooperative, multicentric trials have been conducted within a European or even worldwide network. This has led to impressively improved and internationally recognised treatment results.
The majority of these so-called GPOH trials or studies (a "GPOH trial" is assigned to a study group conducted by the GPOH and is therefore subject to GPOH study rules) are phase III or Phase IV trials. There are also individual GPOH studies that are early clinical trials, but which, unlike the majority of studies, are led by an academic sponsor, i.e. whose study management/sponsorship is not the responsibility of a pharmaceutical company. These studies are also known as IITs (investigator initiated trials). All other early clinical trials can be found in the Phase I/II trials section.
The number of GPOH registries is increasing as well. These are also supervised by a study chair/principal investigator and serve the prospective, national or international, structured data acquisition such as patient information, and consent, based on the registry‘s protocol. Registries are used, for example, to provide access to reference diagnostics, to document and optimise patient care, to participate in accompanying research projects and to provide data and biomaterial for scientific evaluations.