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Klin Padiatr 2022; 234(03): 146-153
DOI: 10.1055/a-1675-3145
Original Article

Rare Tumors in Children and Adolescents – the STEP Working Groupʼs Evolution to a Prospective Registry

Seltene Tumorerkrankungen bei Kindern und Jugendlichen – der Weg der STEP-Arbeitsgruppe zu einem prospektiven Register
Felicitas Hippert
1   Clinic of Pediatrics, Municipal Hospital Dortmund, Dortmund, Germany
,
Lena Desing
2   Clinic of Pediatrics, Hospital Weiden, Weiden, Germany
,
Sonja Diez 
3   Pediatric Surgery, Erlangen University Hospital, Erlangen, Germany
,
Andrea Witowski
1   Clinic of Pediatrics, Municipal Hospital Dortmund, Dortmund, Germany
,
Benedikt Bernbeck
1   Clinic of Pediatrics, Municipal Hospital Dortmund, Dortmund, Germany
,
Michael Abele
4   Pediatric Hematology/Oncology, University Hospital Tubingen Department of Pediatrics, Tubingen, Germany
,
Christian Seitz
4   Pediatric Hematology/Oncology, University Hospital Tubingen Department of Pediatrics, Tubingen, Germany
,
Friederike Erdmann
5   Division of Childhood Cancer Epidemiology, Institute of Medical Biostatistics, Epidemiology and Informatics (IMBEI), University Medical Centre of the Johannes Gutenberg University Mainz, Mainz, Germany
,
Ines Brecht
4   Pediatric Hematology/Oncology, University Hospital Tubingen Department of Pediatrics, Tubingen, Germany
,
Dominik T. Schneider 
1   Clinic of Pediatrics, Municipal Hospital Dortmund, Dortmund, Germany
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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.

Zusammenfassung

Hintergrund Seltene Tumoren (VRT) im Kindes- und Jugendalter haben eine so niedrige Inzidenz, dass sie bisher nicht in das klinische und wissenschaftliche Netzwerk der Kinderonko-logie integriert waren. Die Datenlage ist sehr begrenzt und einheitliche Behandlungsstrategien fehlen. Somit sind VRT klassische Orphan Diseases. Um dieser Problematik entgegen zu wirken, wurde die Arbeitsgemeinschaft für Seltene Tumorerkrankungen in der Pädiatrie (STEP) gegründet. Hier berichten wir über die Patientenrekrutierung während der ersten 10 Jahre.Patienten Einschlusskriterien waren ein Patientenalter bis zum 18. Lebensjahr und eine Nichterfassung in anderen Therapieoptimierungsstudien oder Registern der GPOH. Methoden Die Erfassung erfolgte von 2008 bis 2018 anhand eines standardisierten Dokumentationsbogens. Die Meldungen wurden deskriptiv bezüglich Histologie, Lokalisation und Meldejahr ausgewertet. Ergebnisse Es wurden insgesamt 623 Patienten mit VRT registriert. Zwischen 2008-2014 schwankte die jährliche Meldezahl um 40, seit 2015 liegt sie bei ca. 90. Am häufigsten wurden Tumoren der Haut (n=150), Tumoren des Gastrointestinaltraktes (n=102), der Gonaden (n=77), des HNO-Bereichs (n=68) sowie verschiedene Tumoren (n=107) gemeldet. Diskussion Durch zentrale Strukturen für die klini-sche Beratung und Dokumentation von VRT steigt die Zahl erfasster seltener Tumoren. Insgesamt sind VRT vergleichbar häufig wie andere klassische kinderonkologische Tumoren, hinsichtlich Lokalisation, Histologie und Prognose aber extrem heterogen. Durch die zentrale und vollständige Erfassung und Analyse von VRT, gemeinsam mit internationalen Partnern, kann es gelingen, Behandlungsstrategien zu entwickeln und damit die Behandlungsqualität erheblich zu verbessern.

Key words

childhood cancer - rare tumors - epidemiology - melanoma - colorectal carcinoma - registries

Schlüsselwörter

Krebs bei Kindern - seltene Tumoren - Epidemiologie - Melanome - kolorektales Karzinom - Registerstudien


Publication History

Received: 10 August 2021
Received: 14 October 2021

Accepted: 18 October 2021

Article published online:
19 November 2021

© 2021. Thieme. All rights reserved.

Georg Thieme Verlag
Rüdigerstraße 14, 70469 Stuttgart, Germany

 

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