Zusammenfassung
Die Behandlung kleinwüchsiger Mädchen mit Ullrich-Turner-Syndrom (UTS) mit Wachstumshormon (WH) zur Verbesserung des Längenwachstums ist in Deutschland seit 1991 zugelassen. Die bisher empfohlene Dosis liegt bei etwa 50 µg/kgKG/Tag (tägliche subkutane Injektionen). Durch den Vergleich unterschiedlicher WH-Dosen konnte in den letzten Jahren nachgewiesen werden, dass diejenigen UTS-Patientinnen eine bessere Größenzunahme aufwiesen, die mit einer höheren als der bisher üblichen GH-Dosis behandelt worden waren. Daher hat die Europäische Zulassungsbehörde EMEA vor kurzem auf Antrag der Firma Novo Nordisk für die Behandlung der Betroffenen eine WH-Dosis von 46–67 µg/kgKG/Tag zugelassen. Der vorliegende Bericht fasst die Ergebnisse eines Workshops von Pädiatrischen Endokrinologen zusammen, welcher zum aktuellen Stand der WH-Therapie bei UTS unter Berücksichtigung dieser neuen Option abgehalten wurde. Im Rahmen des Workshops wurden verschiedene Aspekte zur WH-Therapie diskutiert wie aktueller Stand der Therapie, Hintergrund für die Dosiserhöhung, Erhöhung der Dosis generell oder nur in besonderen Situationen, mögliche Risiken, Dosisanpassung, Vorgehen in der Pubertät, Beendigung der Therapie und Transition in die Erwachsenenmedizin.
Abstract
Therapy with recombinant human growth hormone (GH) of short-statured girls with Ullrich Turner syndrome (UTS) has been authorized in Germany since 1991. To date, the daily recommended GH dose has been 50 µg/kg (s.c. injection). By comparing different GH doses, it could be shown in recent years that height gain was much better in those girls who were treated with a higher GH dose than girls treated with the recommended dose. Therefore, upon request of the company Novo Nordisk, the European Medicines Agency (EMEA) recently authorized a GH dose of 46–67 µg/kg/day for the treatment of this patient population. The current report summarizes the results of an expert workshop of pediatric endocrinologists, which was held to review the present position of GH therapy in girls with Turner syndrome in light of the higher GH dose. The workshop addressed issues such as the current status of therapy, the background to the dose increase, whether the dose should be increased in general or only in certain cases, possible risks, dosage adjustments, approaches in puberty, as well as ending therapy and transition to adult medicine.
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Danksagung
Wir danken der Firma Novo Nordisk GmbH, insbesondere Frau Dr. Pfeifer, Herrn Dr. Schmidt und Herrn Prof. Dr. Knittel, für die Organisation des Workshops.
Interessenkonflikt
Der korrespondierende Autor weist auf folgende Beziehung/en hin: Die Autoren und Teilnehmer des Workshops wurden von der Firma Novo Nordisk Pharma GmbH eingeladen, die den Workshop am 25.02.2009 in Frankfurt am Main organisierte. Für Präsentationen einzelner Teilnehmer wurde ein Honorar gezahlt, außerdem wurden die Reisekosten für die Teilnehmer/-innen übernommen.
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Dörr, HG., Ranke, M. Therapie mit Wachstumshormon bei Ullrich-Turner-Syndrom. Monatsschr Kinderheilkd 158, 63–70 (2010). https://doi.org/10.1007/s00112-009-2146-z
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DOI: https://doi.org/10.1007/s00112-009-2146-z