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Rare Tumors in Children and Adolescents - the STEP Working Group's Evolution to a Prospective Registry
Felicitas Hippert1, Lena Desing2, Sonja Diez3
1Clinic of Pediatrics, Municipal Hospital Dortmund, Dortmund, Germany.
Abstract:
Background Very rare tumors (VRT) in children and adolescents have such a low incidence that until recently, they have not been integrated into the clinical and scientific network of pediatric oncology. Data is very limited and consistent treatment strategies are missing. Thus, VRTs are classic orphan diseases. To counteract this problem, the Arbeitsgemeinschaft für Seltene Tumorerkrankungen in der Pädiatrie (STEP) was founded. Here we report on patient recruitment during the first 10 years. Patients Patients aged up to 18 years and not included in any other clinical trial or GPOH registry were included in this analysis. Methods Data was collected from 2008 to 2018 by means of a standardized form. The recorded diagnoses were descriptively analyzed focusing on histology, localization, and year of report. Results A total of 623 patients with VRTs were registered. During 2008-2014, the annual number of registrations was around 40 and is around 90 since 2015. Most frequent diagnoses included tumors of the skin (n=150), tumors of the gastrointestinal tract (n=102), tumors of the gonads (n=77), the ENT region (n=68), and miscellaneous tumors (n=107). Discussion With the establishment of central structures for clinical consultation and documentation of VRTs, the number of registrations increased. Comprehensively, VRTs are as common as other classic pediatric oncology tumors, but extremely heterogeneous in terms of localization, histology, and prognosis. By a centralized and complete registration and analysis of VRTs, also in collaboration with international partners, it is possible to develop treatment strategies and thus greatly increase treatment quality.
Insights
The Arbeitsgemeinschaft für Seltene Tumorerkrankungen in der Pädiatrie (STEP) network has increased rare tumor registrations in children and adolescents. Centralized data collection is vital for developing treatment strategies for these pediatric orphan diseases.
Area of Science:
- Pediatric Oncology
- Rare Diseases
- Clinical Network Development
Background:
- Very rare tumors (VRTs) in children and adolescents lack integrated clinical networks and treatment strategies due to low incidence.
- VRTs are considered classic orphan diseases, necessitating specialized approaches.
- The Arbeitsgemeinschaft für Seltene Tumorerkrankungen in der Pädiatrie (STEP) was established to address these challenges.
Purpose of the Study:
- To report on patient recruitment within the STEP network over its first 10 years of operation.
- To analyze the characteristics of registered very rare tumors in pediatric patients.
- To evaluate the impact of a centralized network on rare tumor data collection.
Main Methods:
- Descriptive analysis of patient data collected from 2008 to 2018 using standardized forms.
- Inclusion criteria: patients up to 18 years old, not in other trials or registries.
- Focus on histology, localization, and year of report for recorded diagnoses.
Main Results:
- A total of 623 pediatric patients with VRTs were registered.
- Annual registrations increased from approximately 40 (2008-2014) to 90 (since 2015).
- Most frequent VRTs included skin (n=150), gastrointestinal (n=102), gonadal (n=77), and ENT (n=68) tumors.
Conclusions:
- Establishment of central structures for VRT consultation and documentation significantly increased patient registrations.
- VRTs, though heterogeneous, are as common as other pediatric oncology tumors.
- Centralized registration and international collaboration are crucial for developing effective treatment strategies and improving care quality for pediatric rare tumors.
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