Erhebungseinheit für seltene pädiatrische Erkrankungen in Deutschland (ESPED) – 25 Jahre pädiatrische Epidemiologie: Eine Bestandsaufnahme

Zusammenfassung

Hintergrund Die Erhebungseinheit für seltene pädiatrische Erkrankungen in Deutschland (ESPED) wurde 1992 mit dem Ziel gegründet, Daten zur Inzidenzbestimmung und zum Krankheitsverlauf seltener pädiatrischer Erkrankungen zu erheben.

Methoden und Fragestellung Retrospektive Analyse der von 1992–2017 durch ESPED erhobenen epidemiologischen Daten bezüglich untersuchter Krankheitsbilder und -gruppen sowie systematische Analyse sämtlicher nationaler und internationaler ESPED-Publikationen. Zudem wurden die Kriterien zur Evaluation von Surveillance-Systemen des Center of Disease Control and Prevention (CDC) auf ESPED bzgl. Einfachheit, Flexibilität, Rechtzeitigkeit, Qualität der Daten, Repräsentanz und Vollständigkeit, Akzeptanz und Stabilität angewandt.

Ergebnisse Im Zeitraum 1992–2017 wurden prospektiv 96 seltene Krankheitsentitäten untersucht. Die 3 größten Krankheitsgruppen waren: Infektiologie (n=30), Neuropädiatrie (n=14) und Hämatologie/Hämostaseologie (n=10). Es wurden 337 Publikationen in Kooperation mit ESPED in nationalen und internationalen Fachzeitschriften veröffentlicht. Bei den 192 Publikationen mit Impact-Faktor (IF) betrug der mediane IF 2,587 (Range 0,032–28,409). Die höchsten IF wurden in den Bereichen Endokrinologie/Stoffwechsel (n=130; medianer IF=3,534), Infektiologie (n=83; medianer IF=3,131) und Hämatologie/Hämostaseologie (n=37; medianer IF=2,497) erzielt. Unsere Untersuchung zeigte, dass ESPED die definierten CDC-Qualitätskriterien erfüllt.

Schlussfolgerung ESPED ist ein sinnvolles, qualitativ hochwertiges Instrument zur Erfassung der Inzidenz sowie zur Beschreibung klinischer Erscheinungsformen von seltenen pädiatrischen Erkrankungen. Dies spiegelt sich in Publikationen in hochkarätigen nationalen und internationalen Fachzeitschriften wider.

Abstract

Background The German Paediatric Surveillance Unit (ESPED) was founded in 1992 to generate incidence data and detailed clinical descriptions of rare, childhood-onset diseases.

Methods Retrospective analysis of the ESPED epidemiological data collection from 1992–2017, and analysis of all published national and international publications originating from ESPED surveys. Center of Disease Control and Prevention (CDC) criteria for evaluating surveillance systems (simplicity, flexibility, timeliness, usefulness, data quality, representativeness, stability and acceptability) were adopted and applied to available ESPED data.

Results Between 1992 and 2017 ESPED completed 96 prospective studies on rare diseases in children. The 3 most frequent clinical entities were: Infectious/communicable disease (n=30), neurological diseases (n = 14) and hematologic diseases (n=10). Studies resulted in 337 publications in national and international journals. The median impact factor of the 192 journal publications with (impact factor) was 2,587 (range 0,032–28,409). The highest impact factors were seen in the fields of endocrinology/metabolism (n=130; median IF=3,534), infectious diseases (n=83; median IF=3,131) and hematology (n=37; median IF=2,497). Our analysis indicates that ESPED surveys meet CDC quality standards.

Conclusion ESPED surveys are an important contributor in the field of clinical epidemiology in children with rare diseases. The high quality of ESPED surveys is reflected by high-impact publications in both national and international journals.

Schlüsselwörter

Inzidenz - Surveillance Systeme - seltene Erkrankungen - Capture-Recapture Methode - Center for Disease Control and Prevention (CDC) - International Network of Paediatric Surveillance Units (INoPSU)

Key words

incidence - surveillance systems - rare diseases - capture-recapture method - Center for Disease Control and Prevention (CDC) - International Network of Paediatric Surveillance Units (INoPSU)

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