Intravenous immunoglobulin vs observation in childhood immune thrombocytopenia: a randomized controlled trial

Heitink‐Pollé, Katja M. J.; Uiterwaal, Cuno S.P.M.; Porcelijn, Leendert; Tamminga, R. Y. J.; Smiers, Frans J.; Woerden, Nicole L. van; Wesseling, J; Vidarsson, Gestur; Laarhoven, Annemieke G.; Haas, Masja de; Bruin, Marrie C.A.

doi:10.1182/blood-2018-02-830844

Cited by 75 publications

(101 citation statements)

References 33 publications

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“…Immune thrombocytopenia (ITP) is an acquired immune‐mediated disorder, characterised by an isolated thrombocytopenia which results in an increased risk of bleeding. In children, bleeding symptoms are usually mild (Neunert et al , ) and the spontaneous remission rate is high, with 75–80% of children recovering within 12 months of presentation (Imbach et al , ; Heitink‐Pollé et al , , ). In light of this, most children in the UK with ITP do not receive therapy directed at raising the platelet count, but are managed on an expectant ‘watch and wait’ policy (Grainger et al , ).…”

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confidence: 99%

Fatigue, executive function and psychological effects in children with immune thrombocytopenia: a cross‐sectional study

et al. 2020

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Childhood ITP is often considered to be a relatively mild haematological disorder, with only a minority of patients requiring treatment for troublesome bleeding. Over recent years, wider effects of the condition have been identified in some adults, particularly relating to fatigue and cognitive impairment. In this study, we sought to investigate such effects in a group of children with ITP and further our understanding of their psychological profile. Children attending routine haematology outpatient clinics and their parents were asked to complete standardised questionnaires designed to assess a range of psychological and cognitive factors. Although the majority of children had some scores within the normal range, a significantly high proportion had difficulties with fatigue (70Á6%), emotional and behavioural symptoms (25Á7%) or executive functioning (19Á4%). Quality of life and subjective evaluation of the illness (appraisal) correlated significantly with each of these domains, but bleeding severity and platelet count did not. Our findings provide valuable insight into the broader impact of childhood ITP, which could aid in providing holistic care, potentially contribute to decisions regarding medical treatment, and guide future research.

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confidence: 99%

Fatigue, executive function and psychological effects in children with immune thrombocytopenia: a cross‐sectional study

et al. 2020

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“…The preliminary results, presented in abstract form at the 2016 ASH annual meeting, indicated no difference in the rates of persistent disease at 6 months between the two groups (10.2% in the IVIg group and 10.4% in the observation group). 42 The study did show a lower incidence of grade 4 or 5 bleeding in IVIg group compared with placebo (8% vs 1%); however, given the low rates of bleeding, the number needed to treat does not justify exposure of all children to IVIg. There have been no additional randomized trials in pediatric ITP with a primary aim of preventing chronic disease.…”

Section: Pediatric Considerationsmentioning

confidence: 86%

Management of newly diagnosed immune thrombocytopenia: can we change outcomes?

Neunert

2017

Blood Advances

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Immune thrombocytopenia resulting from antibody-mediated platelet destruction combined with impaired platelet production is a common cause of thrombocytopenia. The decision to treat newly diagnosed patients is based on several factors including ceasing hemorrhagic manifestations, increasing the platelet count, prevention of bleeding, and inducing remission.Current standard first-line therapy is a course of corticosteroids. Although this treatment paradigm increases the platelet count in the majority of patients, a high percentage relapse after discontinuation of corticosteroid therapy. For this reason, intensification of first-line therapy that results in superior long-term remission rates would be desirable. This manuscript focuses primarily on adults with idiopathic thrombocytopenic purpura (ITP), highlighting pediatric data and practice when applicable. The primary aim is to outline upfront strategies for treatment-naive patients with ITP to enhance remission rates, taking into account assessment of the risks and benefits of these approaches.

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“…In this issue of Blood, Hu et al elegantly demonstrate that steroid receptor coactivator-3 (SRC-3) is essential for maintenance of hematopoietic stem cell (HSC) homeostasis via repression of mitochondrial biogenesis by acetyltransferase GCN5-mediated posttranslational modification of peroxisome proliferator-activated receptor g coactivator 1-a (PGC-1a). 1 The metabolic phenotype of a cell is emerging as a critical determinant of the fate of stem cells. This is also true for HSCs with a number of players in the metabolic rewiring of HSCs already described.…”

Section: Mitochondria Confirmed As Drivers Of Hsc Fatementioning

confidence: 99%

“…Finally, the health-related quality of life (HRQoL) analysis of 107 of the 200 TIKI trial patients (published separately) 8 showed a significant improvement in HRQoL in patients achieving a complete platelet response as was stated in the complete report. 1 Importantly, these were all prospective observations from a trial with an apparent very high rate of compliance: all patients were included in the analysis. One important caveat was that no premedication was given with the IVIg infusions.…”

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confidence: 99%

IVIg and FcγRIIb in kids with ITP: individualizing therapy

Despotovic

Bussel

2018

Blood

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In this issue of Blood, Heitink-Pollé et al present the long-awaited results of a very important recent study, the TIKI (treatment with or without IV immunoglobulin [IVIg] for kids with immune thrombocytopenia [ITP]) trial: a randomized, controlled trial comparing IVIg to observation in children with ITP at diagnosis, with the primary end point of evaluating the rate of chronic ITP in treated and untreated children. 1 10. Shrestha S, Wiener HW, Olson AK, et al. Functional FCGR2B gene variants influence intravenous immunoglobulin response in patients with Kawasaki disease.

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Intravenous immunoglobulin vs observation in childhood immune thrombocytopenia: a randomized controlled trial

Abstract: Key Points In children with newly diagnosed ITP, IVIg treatment at diagnosis does not result in a lower rate of chronic ITP. Upfront treatment with IVIg led to faster recovery and less severe bleeding events.

Cited by 75 publications

References 33 publications

Fatigue, executive function and psychological effects in children with immune thrombocytopenia: a cross‐sectional study

Fatigue, executive function and psychological effects in children with immune thrombocytopenia: a cross‐sectional study

Management of newly diagnosed immune thrombocytopenia: can we change outcomes?

IVIg and FcγRIIb in kids with ITP: individualizing therapy

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