Zusammenfassung
Hämophilie, eine angeborene geschlechtsgebundene Blutgerinnungsstörung, ist eine seltene Erkrankung mit weltweit vergleichbarer Prävalenz, die unbehandelt zu früher Behinderung führt und deren Behandlung per Substitution des fehlenden Gerinnungsfaktors kostenintensiv ist. Ziel jüngerer Forschung ist es, die biopsychosozialen Konsequenzen von Krankheit und Behandlung aus Sicht der Betroffenen zu beschreiben, neue Therapieoptionen auszuloten, den Nutzen der Behandlung zu quantifizieren und die Versorgung der Patienten über verschiedene Länder zu vergleichen. Hierzu wurden krankheitsübergreifende und krankheitsspezifische Instrumente zur Beurteilung der Lebensqualität von Kindern mit Hämophilie entwickelt und in Studien eingesetzt. Im vorliegenden Beitrag werden nach einer Darstellung der klinischen Aspekte der Hämophilie und der Erfassung der Lebensqualität 2 multinationale Studien zur Lebensqualität von Kindern und Jugendlichen mit Hämophilie im europäischen Vergleich vorgestellt. In der HAEMO-QoL-Studie wurden 339 Kinder im Alter ab 4 Jahren mit Hämophilie aus 6 europäischen Ländern zu ihrer gesundheitsbezogenen Lebensqualität befragt. In der ESCHQoL-Studie wurden 1424 Personen mit Hämophilie aus 21 Europäischen Ländern untersucht, davon 444 Kinder ab 4 Jahren. In beiden Studien wurden zudem klinische Daten, Angaben zur Versorgung und psychosoziale Determinanten wie Krankheitsbewältigung, Lebenszufriedenheit und soziale Unterstützung erhoben. Zwischen den einzelnen Ländern ergaben sich signifikante Unterschiede bzgl. der Lebensqualität der Kinder. Zudem beeinflussen psychosoziale Faktoren die Lebensqualität. Von besonderer Bedeutung für die Lebensqualität der Kinder scheinen nach bisherigen Analysen die ausreichende Versorgung mit Faktorpräparaten auf der klinischen Seite und die soziale Unterstützung auf der psychosozialen Seite zu sein. Die bestehenden Länderunterschiede in der Lebensqualitätsbewertung stellen eine gesundheitspolitische Herausforderung dar.
Abstract
Haemophilia, an inherited x-linked bleeding disorder, is a rare disease with comparable prevalence worldwide, which leads to early disability if untreated, and which, if treated via substitution of blood clotting factor, is associated with high treatment costs. Recent research has focused on bio-psychosocial consequences of treatment from the patient’s perspective, in order to identify new treatment approaches, to quantify the patient benefit, and to compare haemophilia care across countries. For this, generic and disease-specific instruments have been developed and are currently being used in studies. The present paper describes clinical aspects of haemophilia as well as the assessment of quality of life and proceeds to introduce two recent studies on quality of life of children and adolescents with haemophilia in Europe. The HAEMO-QoL study investigated the quality of life of 339 children with haemophilia age 4 and above from six European countries. A total of 1,424 patients from 21 European countries participated in the ESCHQoL study, among these were 444 children aged 4 and above years. In both studies clinical data, information about health care and psychosocial determinants such as coping, life satisfaction and social support were assessed. Significant differences in quality of life between countries were found. Psychosocial determinants were associated with quality of life. Current analyses suggest that important determinants for quality of life are availability of factor concentrate from the clinical side and social support from the psychosocial side. The difference in quality of life across countries is a future major health-political challenge.
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Bullinger, M., Gringeri, A. & von Mackensen, S. Lebensqualität von jungen Patienten mit Hämophilie in Europa. Bundesgesundheitsbl. 51, 637–645 (2008). https://doi.org/10.1007/s00103-008-0539-0
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DOI: https://doi.org/10.1007/s00103-008-0539-0