Zusammenfassung
Innerhalb des klinischen Phänotyps Typ-1-Diabetes besteht eine beträchtliche Heterogenität: Die genetische Anfälligkeit ist komplex, die Progressionsraten unterscheiden sich deutlich, ebenso wie die Wirksamkeit therapeutischer Maßnahmen. Neue Präventionsstrategien sollen mithilfe von Biomarkern zu personalisierten Kombinationstherapien in Analogie zum Vorgehen in der Kinderonkologie führen. Bekanntlich beginnt der Typ-1-Diabetes mit der Autoimmunität, dem Nachweis von 2 oder mehr persistierenden diabetesassoziierten Antikörpern (Stadium 1). Eine zusätzliche subklinische Dysglykämie wird als Stadium 2 bezeichnet, der manifeste Typ-1-Diabetes als Stadium 3. Sowohl genetische wie immunologische Parameter ermöglichen eine Früherkennung von Kindern und Jugendlichen. Da 9 von 10 betroffenen Kindern aus Familien ohne andere Familienmitglieder mit Typ-1-Diabetes stammen, sind bevölkerungsbezogene Screeningstrategien zur Prävention des Typ-1-Diabetes erforderlich. Untersuchungen zur psychologischen Belastung durch Screeningprozeduren zeigten, dass die große Mehrheit der Familien mit dem Wissen um das erhöhte Diabetesrisiko ihres Kindes nach kurzer Zeit relativ unbelastet leben konnte. Im Forschungsnetzwerk Globale Plattform zur Prävention des Autoimmunen Diabetes (GPPAD) werden Primärprävention mit oralem Insulin (POINT-Studie) oder Probiotika (S1NTIA-Studie) untersucht. Das europäische Forschungsnetzwerk „An innovative approach towards understanding and arresting Type 1 diabetes“ (INNODIA) entwickelt ein einheitliches Studienprotokoll („Masterprotokoll“), um ab dem Patientenalter von 5 Jahren die Untersuchung neuer Einzelsubstanzen und Kombinationstherapien zu beschleunigen. Sekundäre Präventionsansätze zeigen in ersten Pilotstudien vielversprechende Ergebnisse zu Verlängerung der Remissionsphase oder Verzögerung des Krankheitsbeginns bei Risikopopulationen.
Abstract
There is considerable heterogeneity within the clinical phenotype of type 1 diabetes: the genetic susceptibility is complex and progression rates markedly differ, as does the efficacy of therapeutic measures. New prevention strategies should use biomarkers to lead to personalized combination treatment analogous to the approach used in pediatric oncology. It is well known that type 1 diabetes starts with autoimmunity and the detection of two or more persistent diabetes-associated antibodies (stage 1). An additional subclinical dysglycemia is termed stage 2 and manifest type 1 diabetes is termed stage 3. Both genetic and immunological parameters enable early detection in children and adolescents. Because 9 out of 10 affected children come from families without other family members having type 1 diabetes, population-based screening strategies are needed for prevention of type 1 diabetes. Studies on the psychological burden of screening procedures showed that the vast majority of families were able to live relatively unburdened by the knowledge of their child’s increased diabetes risk after a short period of time. The Global Platform for Prevention of Autoimmune Diabetes (GPPAD) research network is investigating primary prevention with oral insulin (POInT study) or probiotics (S1NTIA study). The European research network “an innovative approach towards understanding and arresting type 1 diabetes” (INNODIA) is developing a standardized study protocol (master protocol) to accelerate the investigation of new single agents and combination therapies in patients over 5 years of age. Secondary prevention approaches are showing promising results in initial pilot studies to prolong the remission phase or delay disease onset in populations at risk.
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O. Kordonouri, T. Danne und K. Lange geben an, dass kein Interessenkonflikt besteht.
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Kordonouri, O., Danne, T. & Lange, K. Neue Wege zur Prävention des Typ-1-Diabetes bei Kindern. Monatsschr Kinderheilkd 169, 930–940 (2021). https://doi.org/10.1007/s00112-021-01274-x
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DOI: https://doi.org/10.1007/s00112-021-01274-x