English: acute immune thrombocytopenic purpura, ITP. Nederlands: acute idiopathische thrombocytopenische purpura, ITP.
Conditions
Interventions
The intervention consists of one dose of 0.8 g/kg bodyweight Nanogam ®, a liquid intravenous immunoglobulin, manufactured by Sanquin.
Besides the primary intervention, quality of life questionnaires
Sponsors
University Medical Center Utrecht (UMCU), Wilhelmina Children's Hospital (WKZ)
Eligibility
Inclusion criteria
Inclusion criteria: General inclusion criteria: 1. Children aged 3 months -16 years, presenting to a pediatrician with newly diagnosed acute ITP 2. Platelet count < 20 x 10 9 /L 3. Bleeding tendency < grade 4 (Buchanan) 4. No prior immunomodulating treatment within 4 weeks before diagnosis 5. Sufficient comprehension of the Dutch language 6. Signed informed consent by parents and/ or patients
Exclusion criteria
Exclusion criteria: General exclusion criteria: 1. 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 2. Immunomodulating treatment (IVIG, corticosteroids) within 4 weeks before diagnosis 3. History of allergic reactions against human plasma, plasma products or intravenous immunoglobulin 4. Severe or life threatening bleeding at presentation: grade 4 or 5 (Buchanan) 5. A patient known with IgA deficiency with IgA antibodies 6. A patient known with renal insufficiency 7. Insufficient comprehension of the Dutch language 8. No informed consent
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| - Main study parameter is the percentage of patients developing chronic ITP, defined by a platelet count of < 150 x 10^9/l six months after diagnosis. | — |
Secondary
| Measure | Time frame |
|---|---|
| - Clinical parameters during the course of the disease, eg: bleeding score graded according to the revised grading system developed by Buchanan, and time between onset of symptoms and recovery of platelet numbers. - 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. - 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. | — |
Contacts
Public ContactK.M.J. Heitink-Pollé
University Medical Center Utrecht (UMCU), Wilhelmina Children's Hospital (WKZ) Department of Paediatric Heamatology and Oncology Huispost KC 03.063.0 Post Box 85090
Outcome results
None listed