Study of Cytokines in Children With Opsoclonus-Myoclonus Syndrome
Märksõnad
Abstraktne
Kirjeldus
In this translational research, immunological mechanisms that underlie the assault of the immune system on the brain in paraneoplastic opsoclonus-myoclonus syndrome (OMS) are under evaluation. To test our principal hypothesis that there is an imbalance of pro-inflammatory (Th1) and anti-inflammatory (Th2) cytokines in OMS, a comprehensive cytokine panel will be measured by enzyme-linked immunosorbent assay (ELISA) and multiplexed fluorescent bead-based immunoassay detection (LUMINEX 100 Lab MAP system)in blood and cerebrospinal fluid (CSF) of 400 children. To test the second hypothesis that cytokines could serve as biomarkers of disease activity in OMS, cytokine concentrations will be correlated with clinical variables, such as disease severity, OMS duration, prior relapses, and remissions, as well as immunological variables, such as lymphocyte subset analysis. The cytokine 'biomarker profile' could aid decision making for early intervention by identifying children at high risk for relapse and poor outcome and allow targeting of the most implicated inflammatory cytokines by cytokine therapies. To test our third hypothesis that lack of response to immunotherapy is due in part to failure to increase the expression of anti-inflammatory Th2 cytokines, we will make paired pre/post comparisons of the impact of immunotherapies given in the course of clinical care [steroids, adrenocorticotropin (ACTH), intravenous immunoglobulins (IVIg), rituximab, chemotherapy, other drugs, combinations] on the cytokine and clinical profile. This research could lead to the application of commercially-available cytokines and cytokine blockers or to the development of new ones for OMS.
Kuupäevad
| Viimati kinnitatud: | 04/30/2017 |
| Esmalt esitatud: | 12/07/2008 |
| Hinnanguline registreerumine on esitatud: | 12/08/2008 |
| Esmalt postitatud: | 12/09/2008 |
| Viimane värskendus on esitatud: | 05/18/2017 |
| Viimati värskendus postitatud: | 05/22/2017 |
| Õppe tegelik alguskuupäev: | 12/31/2007 |
| Eeldatav esmane lõpetamise kuupäev: | 11/30/2016 |
| Eeldatav uuringu lõpetamise kuupäev: | 11/30/2016 |
Seisund või haigus
Faas
Käerühmad
| Arm | Sekkumine / ravi |
|---|---|
| Pediatric case-controls These are children who underwent lumbar puncture and blood drawing for diagnostic testing for non-inflammatory neurological or non-neurological disorders, and whose samples were retrieved from the clinical lab under a linked Institutional Review Board (IRB) protocol. | |
| Pediatric OMS These are patients treated by the P.I. based on clinical decision making, not a clinical trial (this is an observational study). The types of treatments are varied, and, on the initial evaluation, the patients may be untreated or already tried on various immunotherapies. They range from monotherapy with steroids, ACTH, or IVIg, to disease modifying agents, such as rituximab, cyclophosphamide, and other chemotherapy, typically adjunctively or as combination therapy. |
Abikõlblikkuse kriteeriumid
| Õppimiseks sobivad vanused | 1 Year To 1 Year |
| Uuringuks kõlblikud sood | All |
| Proovivõtumeetod | Probability Sample |
| Võtab vastu tervislikke vabatahtlikke | Jah |
| Kriteeriumid | Inclusion Criteria: - Clinical diagnosis of OMS Exclusion Criteria: - Equivocal diagnosis - Contraindications to lumbar puncture - Treatment with agents outside the scope of the study |
Tulemus
Esmased tulemusnäitajad
1. Reduction in inflammatory cytokines [6 and 12 months]
Sekundaarsed tulemusmõõdud
1. Correlation of cytokine concentration and clinical severity score. [6 and 12 months]
