Acute Idiopathic Thrombocytopenic Purpura (ITP) in children MedDRA version: 14.1 Level: LLT Classification code 10023095 Term: ITP System Organ Class: 10005329 - Blood and lymphatic system disorders
Conditions
Interventions
Trade Name: Nanogam
Pharmaceutical Form: Solution for infusion
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentra
Sponsors
University Medical Center Utrecht
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: General inclusion criteria - Children aged 3 months -16 years, presenting to a pediatrician with newly diagnosed acute ITP and - Platelet count =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: A patient presenting with any of the following criteria will not be included in the study: General exclusion criteria - clinical features that are not compatible with the diagnosis of acute ITP, for example: presence of other auto-immune phenomena, organomegaly, other cytopenias besides thrombocytopenia or features susceptible for infectious disease like hepatitis, Epstein-Barr virus or HIV - immunomodulating treatment (IVIG, corticosteroids) within 4 weeks before diagnosis - history of allergic reactions against human plasma, plasma products or intravenous immunoglobulin - Severe or life threatening bleeding at presentation: grade 4 or 5 (Buchanan) - No informed consent
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to investigate the hypothesis that early IVIG treatment in children with newly diagnosed acute ITP reduces the risk of development of chronic disease. ;Secondary Objective: Secondary objectives are: 1. To evaluate the clinical parameters during the course of the disease, eg: bleeding score and time between onset of symptoms and recovery of platelet numbers. 2. Comparing the HRQoL in parents and patients with acute ITP who did and did not have IVIG and in those that do and do not develop chronic ITP. 3. Estimation of variability of biological parameters of the immune system of the patient that are supposed to be involved in the differences in outcome between acute vs. chronic disease as well as between response on IVIG treatment vs. non response. These include: A) the genetic polymorphisms of the activating and inhibiting IgG-Fc receptor and other inhibiting immune receptors. B) Immunoglobulin glycosylation variability within the platelet auto antibodies and its changes during time, as well as the changes due to IVIG treatment. C) Quantity and function of regulatory T cells. ;Primary end point(s): Primary study endpoint is development of chronic ITP, defined by a platelet count of < 150 x 10^9/l six months after diagnosis. | — |
Countries
Netherlands
Outcome results
None listed