Pilot study of the effect of romiplostim on child health‐related quality of life (HRQoL) and parental burden in immune thrombocytopenia (ITP)

Klaassen, Robert J.; Mathias, Susan D.; Buchanan, George R.; Bussel, James B.; Deuson, Robert; Young, Nancy L.; Collier, Anderson B.; Bomgaars, Lisa; Blanchette, Victor S.

doi:10.1002/pbc.23312

Cited by 42 publications

(43 citation statements)

References 18 publications

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“…Klaassen et al studied HRQoL using the KIT in 22 children with chronic ITP participating in a pilot study of romiplostin for children with chronic ITP. 7 Parents of children receiving romiplostin (n=17) showed significant improvement of KIT scores, whereas parents of children receiving placebo did not (n=5; P=0.008). Zilber et al performed a retrospective cross-sectional study in 17 Israeli children with newly diagnosed and persistent ITP and 34 parents of children with ITP.…”

Section: Discussionmentioning

confidence: 96%

“…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][7][8][9][10][11] but large prospective studies with longitudinal generic as well as disease-specific HRQoL measurements are lacking.…”

Section: Introductionmentioning

confidence: 99%

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Health-related quality of life in children with newly diagnosed immune thrombocytopenia

et al. 2014

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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...

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Section: Discussionmentioning

confidence: 96%

Section: Introductionmentioning

confidence: 99%

Health-related quality of life in children with newly diagnosed immune thrombocytopenia

et al. 2014

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“…The effect of romiplostim was found to be dose dependent, showing the effect by 5th day and diminished after the 28th day in animal studies. Significant improvement in platelet count was found both in children and adults, suffering from ITP when administrated with romiplostim subcutaneously (Kuter et al, , 2012Klaassen et al, 2012). However, in younger children, the response was less robust.…”

Section: Thrombopoetin Mimeticsmentioning

confidence: 81%

Erythropoietin and thrombopoietin mimetics: Natural alternatives to erythrocyte and platelet disorders

Gutti

Pasupuleti

Sahu

et al. 2016

Critical Reviews in Oncology/Hematology

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“…Quality -of-life investigations showed a significant decrease of parental burden in the romiplostim group. 29 The child self-reported quality of life was not statistically different from placebo. A further study evaluating the safety and efficacy of long-term dosing of romiplostim in pediatric subjects with ITP is still recruiting.…”

Section: New Strategiesmentioning

confidence: 98%

Chronic Immune Thrombocytopenia in Children: Who Needs Splenectomy?

Schifferli

Kühne

2013

Seminars in Hematology

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Pilot study of the effect of romiplostim on child health‐related quality of life (HRQoL) and parental burden in immune thrombocytopenia (ITP)

Cited by 42 publications

References 18 publications

Health-related quality of life in children with newly diagnosed immune thrombocytopenia

Health-related quality of life in children with newly diagnosed immune thrombocytopenia

Erythropoietin and thrombopoietin mimetics: Natural alternatives to erythrocyte and platelet disorders

Chronic Immune Thrombocytopenia in Children: Who Needs Splenectomy?

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