Hamostaseologie 2009; 29(02): 171-176
DOI: 10.1055/s-0037-1617015
Rewiew
Schattauer GmbH

Reassessment of treatment modalities for paediatric patients with chronic immune thrombocytopenia

Bewertung von Therapieoptionen bei Kindern und Jugendlichen mit chronischer Immun - thrombozytopenie
H. J. Laws
1   Department of Paediatric Oncology, Haematology and Clinical Immunology, University Hospital Duesseldorf
,
G. Janssen
1   Department of Paediatric Oncology, Haematology and Clinical Immunology, University Hospital Duesseldorf
,
A. Borkhardt
1   Department of Paediatric Oncology, Haematology and Clinical Immunology, University Hospital Duesseldorf
› Author Affiliations
Further Information

Publication History

Publication Date:
29 December 2017 (online)

Summary

Approximately 70% of children have the acute form of immune thrombocytopenia (ITP), which is defined by recovery within six months of presentation with or without treatment. Chronic ITP is to be reserved for patients with platelets < 100 000/μl for more than twelve months and exclusion of other diagnosis like systemic lupus erythematosus or bone marrow failures. In children, the chance of spontaneous recovery is 52% after diagnosis of chronic ITP. The Intercontinental Childhood ITP Study group recommends that children without bleeding may not require therapy regardless of their platelet count. Whereas in patients with bleeding symptoms first line therapy is defined and includes steroids or immunoglobuline, second line therapy in refractory patients with significant hemorrhagic problems is unclear. Guidelines recommend splenectomy, but for more than 50 years patients and physicians look for pharmacological alternatives. It may be that rituximab is a promising option which has been proven to be effective with few adverse effects. Till now the treatment has focused on immunomodulation. Research has now focused on stimulating platelet production. In this review we discuss old and new therapy modalities for children with cITP.

Zusammenfassung

Unabhängig von der Therapie haben 70% der Kinder innerhalb von sechs Monaten nach der Diagnosestellung einer akuten Immunthrombozytopenie (ITP) eine normale Thrombozytenzahl. Der Begriff chronische ITP (cITP) ist nur zu verwenden, wenn länger als 12 Monate die Thrombozytenzahl unter 100 000/μl liegt und andere Erkrankungen (z. B. systemischer Lupus erythematodes, beginnendes Knochenmark versagen, von-Wille-brand-Syndrom 2b) ausgeschlossen wurden. 52% der Kinder mit cITP erreichen wieder eine normale Thrombozytenzahl. Die Intercontinal Childhood ITP Study Group empfiehlt, dass nur Kinder mit relevanter Blutungsneigung behandelt werden – unabhängig von der Thrombozytenzahl. Während für Patienten mit Blutungssymptomen als First-line-Therapie Cortison und Immunglobuline gut definiert und etabliert sind, gelten die Empfehlungen für die Folgetherapie bei anhaltenden Blutungen als weniger akzeptiert. So empfehlen die amerikanischen und britischen Therapierichtlinien zwar die Splenektomie, doch seit mehr als 50 Jahren wird nach Alternativen gesucht. Rituximab ist möglicherweise eine viel versprechende Therapieoption, die bereits erfolg-reich eingesetzt wurde und wenig unerwünschte Arzneimittelwirkungen zeigte. Bisher lag der Fokus der Therapie in einer Immunmodulation. Mit Romiplostin und Elthrombopag stehen nun zwei Medikamente zur Verfügung, die die Thrombozytenbildung stimulieren. In dieser Übersicht diskutieren wir etablierte und neue Therapiemodalitäten für Kinder mit cITP.

 
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