Implementing data on targeted therapy from the INFORM registry platform for children with relapsed cancer in Sweden
(2024) In Frontiers in Oncology 14.- Abstract
Background: Advances in treatment of childhood malignancies have improved overall cure rates to 80%. Nevertheless, cancer is still the most common cause of childhood mortality in Sweden. The prognosis is particularly poor for relapse of high-risk malignancies. In the international INFORM registry, tumor tissue from patients with relapsed, refractory, or progressive pediatric cancer as well as from very-high risk primary tumors is biologically characterized using next-generation sequencing to identify possible therapeutic targets. We analyzed data from Swedish children included in the INFORM registry concerning patient characteristics, survival, sequencing results and whether targeted treatment was administered to the children based on... (More)
Background: Advances in treatment of childhood malignancies have improved overall cure rates to 80%. Nevertheless, cancer is still the most common cause of childhood mortality in Sweden. The prognosis is particularly poor for relapse of high-risk malignancies. In the international INFORM registry, tumor tissue from patients with relapsed, refractory, or progressive pediatric cancer as well as from very-high risk primary tumors is biologically characterized using next-generation sequencing to identify possible therapeutic targets. We analyzed data from Swedish children included in the INFORM registry concerning patient characteristics, survival, sequencing results and whether targeted treatment was administered to the children based on the molecular findings. Methods: A registry-based descriptive analysis of 184 patients included in the INFORM registry in Sweden during 2016–2021. Results: The most common diagnoses were soft tissue and bone sarcomas followed by high grade gliomas [including diffuse intrinsic pontine glioma (DIPG)]. Complete molecular analysis was successful for 203/212 samples originating from 184 patients. In 88% of the samples, at least one actionable target was identified. Highly prioritized targets, according to a preset scale, were identified in 48 (24%) samples from 40 patients and 24 of these patients received matched targeted treatment but only six children within a clinical trial. No statistically significant benefit in terms of overall survival or progression free survival was observed between children treated with matched targeted treatment compared to all others. Conclusion: This international collaborative study demonstrate feasibility regarding sequencing of pediatric high-risk tumors providing molecular data regarding potential actionable targets to clinicians. For a few individuals the INFORM analysis was of utmost importance and should be regarded as a new standard of care with the potential to guide targeted therapy.
(Less)
- author
- Wallin, Sofia ; Øra, Ingrid LU ; Prochazka, Gabriela ; Sandgren, Johanna ; Björklund, Caroline ; Ljungman, Gustaf ; Vogt, Hartmut ; Ek, Torben ; van Tilburg, Cornelis M. and Nilsson, Anna
- organization
- publishing date
- 2024
- type
- Contribution to journal
- publication status
- published
- subject
- keywords
- molecular diagnostic techniques, molecular targeted therapy, pediatric cancer, pediatric oncology, precision medicine
- in
- Frontiers in Oncology
- volume
- 14
- article number
- 1340099
- publisher
- Frontiers Media S. A.
- external identifiers
-
- pmid:38357207
- scopus:85184879877
- DOI
- 10.3389/fonc.2024.1340099
- language
- English
- LU publication?
- yes
- additional info
- Funding Information: The author(s) declare financial support was received for the research, authorship, and/or publication of this article. We are grateful for the financial support from the Swedish Childhood Cancer Fund (BCF), Entrepreneurs for good and ARMEC Lindeberg Foundation directed to inclusion and follow up of children in Sweden included in the Inform registry. The INFORM program is financially supported by the German Cancer Research Center (DKFZ), the German Cancer Consortium (DKTK), the German Federal Ministry of Education and Research (BMBF), the German Federal Ministry of Health (BMG), the Ministry of Science, Research and the Arts of the State of Baden-Württemberg (MWK BW); the German Cancer Aid (DKH), the German Childhood Cancer Foundation (DKS), RTL television, the aid organization BILD hilft e.V. (Ein Herz für Kinder) and the generous private donation of the Scheu family. Acknowledgments Funding Information: The authors would like to thank the local study nurses in the Swedish university centers who organized sampling of tumor material. The Swedish Childhood Tumor Biobank (BTB) has been instrumental in handling samples from the different centra, extracting DNA/RNA and organizing the shipment to Heidelberg, Germany. We are grateful for the financial support from the Swedish Childhood Cancer Fund (BCF), Entrepreneurs for good and ARMEC Lindeberg Foundation. The INFORM program is financially supported by the German Cancer Research Center (DKFZ), the German Cancer Consortium (DKTK), the German Federal Ministry of Education and Research (BMBF), the German Federal Ministry of Health (BMG), the Ministry of Science, Research and the Arts of the State of Baden-Württemberg (MWK BW); the German Cancer Aid (DKH), the German Childhood Cancer Foundation (DKS), RTL television, the aid organization BILD hilft e.V. (Ein Herz für Kinder) and the generous private donation of the Scheu family. Publisher Copyright: Copyright © 2024 Wallin, Øra, Prochazka, Sandgren, Björklund, Ljungman, Vogt, Ek, van Tilburg and Nilsson.
- id
- 518fc0b1-b8ea-4037-b64f-1b22504ad786
- date added to LUP
- 2024-02-29 00:17:50
- date last changed
- 2024-04-23 16:18:07
@article{518fc0b1-b8ea-4037-b64f-1b22504ad786,
abstract = {{<p>Background: Advances in treatment of childhood malignancies have improved overall cure rates to 80%. Nevertheless, cancer is still the most common cause of childhood mortality in Sweden. The prognosis is particularly poor for relapse of high-risk malignancies. In the international INFORM registry, tumor tissue from patients with relapsed, refractory, or progressive pediatric cancer as well as from very-high risk primary tumors is biologically characterized using next-generation sequencing to identify possible therapeutic targets. We analyzed data from Swedish children included in the INFORM registry concerning patient characteristics, survival, sequencing results and whether targeted treatment was administered to the children based on the molecular findings. Methods: A registry-based descriptive analysis of 184 patients included in the INFORM registry in Sweden during 2016–2021. Results: The most common diagnoses were soft tissue and bone sarcomas followed by high grade gliomas [including diffuse intrinsic pontine glioma (DIPG)]. Complete molecular analysis was successful for 203/212 samples originating from 184 patients. In 88% of the samples, at least one actionable target was identified. Highly prioritized targets, according to a preset scale, were identified in 48 (24%) samples from 40 patients and 24 of these patients received matched targeted treatment but only six children within a clinical trial. No statistically significant benefit in terms of overall survival or progression free survival was observed between children treated with matched targeted treatment compared to all others. Conclusion: This international collaborative study demonstrate feasibility regarding sequencing of pediatric high-risk tumors providing molecular data regarding potential actionable targets to clinicians. For a few individuals the INFORM analysis was of utmost importance and should be regarded as a new standard of care with the potential to guide targeted therapy.</p>}},
author = {{Wallin, Sofia and Øra, Ingrid and Prochazka, Gabriela and Sandgren, Johanna and Björklund, Caroline and Ljungman, Gustaf and Vogt, Hartmut and Ek, Torben and van Tilburg, Cornelis M. and Nilsson, Anna}},
keywords = {{molecular diagnostic techniques; molecular targeted therapy; pediatric cancer; pediatric oncology; precision medicine}},
language = {{eng}},
publisher = {{Frontiers Media S. A.}},
series = {{Frontiers in Oncology}},
title = {{Implementing data on targeted therapy from the INFORM registry platform for children with relapsed cancer in Sweden}},
url = {{http://dx.doi.org/10.3389/fonc.2024.1340099}},
doi = {{10.3389/fonc.2024.1340099}},
volume = {{14}},
year = {{2024}},
}