ARTICLES
1525
Qualità of Life
IntroductionImmune thrombocytopenia (ITP) is an autoimmune disorder characterized by isolated thrombocytopenia (peripheral blood platelet count <100x10 9 /L) in the absence of other disorders associated with thrombocytopenia.1 Only 20-25% of children with ITP will develop chronic disease, currently defined as thrombocytopenia <100x10 9 /L lasting for more than 12 months. 1 In the case of no or mild bleeding the management consists of careful observation and restriction of activities that carry a risk of severe bleeding, regardless of platelet count.
2Severe bleeding, occurring in only 3-6% of children, 3 requires treatment with corticosteroids, intravenous immunoglobulin or anti-D immunoglobulin, either alone or in combination.Despite the transient and often benign course of the disease, many clinicians observe that ITP has a significant impact on health-related quality of life (HRQoL). 4 Recently published management guidelines state that HRQoL issues should be taken into account while making decisions on management in childhood ITP.2,5 However, these statements are based on clinical experience rather than results of research since HRQoL studies in childhood ITP are scarce. Recently, several clinical studies addressing HRQoL in children with ITP have been performed, 6-11 but large prospective studies with longitudinal generic as well as disease-specific HRQoL measurements are lacking.For this reason we decided to study HRQoL as part of a prospective study in children with newly diagnosed ITP: the TIKI study (Therapy with or without Intravenous Immunoglobulin for Kids with acute ITP), a multicenter randomized clinical trial to determine whether early administration of intravenous immunoglobulin can prevent a chronic course of the disease. In this study, children receive either a single dose of intravenous immunoglobulin or careful observation and treatment only in the case of severe bleeding. The final results of the primary outcome of this study are awaited and have not been published yet. The aim of the current HRQoL study was to relate generic as well as disease-specific HRQoL scores of children with newly diagnosed ITP to type of treatment, bleeding severity and clinical course of the disease, to analyze changes in HRQoL scores over time and to compare generic HRQoL scores of children with newly diagnosed ITP with already published data from a reference group of Dutch school children.
12
Methods
PatientsChildren aged 3 months to 16 years with newly diagnosed ITP, a platelet count below 20x10 9 /L and with mild to moderate bleeding were eligible for inclusion in the TIKI study. Despite its generally transient and benign course, childhood immune thrombocytopenia has a large impact on health-related quality of life. Recently published guidelines state that quality of life should be taken into account while making decisions on management in childhood immune thrombocytopenia. We, therefore, assessed healthrelated quality of life in children with newly diagnosed immune thrombocytopeni...