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Chapters

Individualized Prognosis in Childhood Immune Thrombocytopenia

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i Introduction


Chapter 1

Childhood Immune Thrombocytopenia 

1.1 Morbidity and mortality
1.2 Disease mechanisms
1.3 Diagnosis
1.4 Prognosis
1.5 Clinical management
1.6 Treatment options
1.7 Modification of ITP disease courses
1.8 Towards individualized prognosis and treatment
1.8.1 Proposed prediction markers of ITP disease courses
1.9 Statistical learning
1.10 Research questions and scope of the thesis

ii Genetic and immune parameters for prognosis

Chapter 2

Age at diagnosis shapes the prognosis of childhood ITP medRxiv

Chapter 3

Transient and chronic childhood immune thrombocytopenia are distinctly affected by Fc-g receptor polymorphisms abstract

Chapter 4

Platelet autoantibody immunoassays in childhood ITP: a systematic review abstract

Chapter 5

Antigen-specific autoantibodies indicate prognosis and IVIg treatment response in childhood immune thrombocytopenia abstract

Chapter 6

ITGB1-expressing CD4+ T effector cell response associates with chronic childhood immune thrombocytopenia

iii Pathophysiology and mechanisms of ITP

Chapter 7

IgG-Fc glycosylation before and after rituximab treatment in immune thrombocytopenia abstract

Chapter 8

Anti-glycoprotein Iba autoantibodies do not impair circulating thrombopoietin levels in immune thrombocytopenia patients abstract

iv Models for prognostication

Chapter 9

A clinical prediction score for transient versus persistent childhood immune thrombocytopenia

Chapter 10

Biological stratification of clinical disease courses in childhood immune thrombocytopenia

Chapter 11

Intravenous Immunoglobulins (IVIg) in childhood immune thrombocytopenia: towards personalized medicine abstract

v Discussion

Chapter 12

General discussion

12.1 Prediction models for ITP prognosis
12.1.1 Individualized prediction of ITP spontaneous recovery and IVIg treatment responses
12.2 Insights into ITP pathophysiology
12.2.1 Genetic heterogeneity
12.2.2 Platelet autoantibodies
12.2.3 Cellular immune response
12.2.4 Age heterogeneity
12.2.5 Further thoughts on mechanisms
12.3 Clinical implications
12.3.1 Diagnosis of ITP
12.3.2 Prognosis versus diagnosis
12.3.3 Additional diagnostic tests
12.3.4 Treatment
12.3.5 Questions of generalizability
12.3.6 Follow-up studies
12.4 A roadmap to advance ITP research & care
12.4.1 Dogmalysis
12.4.2 Better clinical study design
12.4.3 Focus on patient-centered outcomes
12.4.4 Towards a reporting standard

Chapter 13

Conclusions