Clinical Study

ZonMW - Improve diagnosis and treatment of psychotic disorder of auto-immune origin (patient study)

Psychotic syndromes such as schizophrenia are serious disorders with far-reaching effects on health, which have consequences for daily life of patients and their families. More than 1% of the population has a serious psychotic disorder, with often a first onset between 15 and 30 years of age. Treatment of psychotic disorders is still mainly symptomatic since the pathogenesis of these diseases is heterogeneous; genetic and environmental factors play a role that is still poorly understood. In a small subgroup of patients, there are strong indications that autoantibodies are responsible for causing a psychotic episode suggesting an autoimmune disease as the underlying cause. The aim of this study is to establish the prevalence of autoantibodies against neuronal surface proteins in an early onset psychotic patient’s cohort. This will improve the diagnosis of this specific patient group and would be an important achievement because, fortunately, a standard immunosuppressive treatment is available, which has been successfully applied in other autoimmune diseases. In this way, patients could benefit by standard immunosuppressive treatment in the clinic. Our study has been approved in 2016 by the METC of the MUMC+.

 

Clinical trial for MG 

Although myasthenia gravis (MG) is one of the best-characterized autoimmune diseases, standard therapies are ineffective in 10-15% of generalized MG cases, necessitating advanced treatment options. The role of the thymus is well-documented, particularly in acetylcholine receptor-positive (AChR) MG patients with thymic follicular hyperplasia (TFH). The thymus is considered a key source of anti-AChR-producing plasma cells. However, recent research suggests that the bone marrow (BM) may serve as a long-term survival niche, sustaining disease activity and contributing to refractory disease. This trial aims to characterize immune cell populations in the thymus, BM, and circulation to develop an in vitro tool for predicting treatment response in AChR-MG. Additionally, we seek to evaluate 3T MRI as a non-invasive method for identifying TFH, potentially improving patient selection for thymectomy. This is a non-randomized controlled trial including 150 participants who are willing to undergo thymectomy, donate blood samples and undergo clinical assessments for disease progression at multiple time points. The cohort consists of four patient groups: 1) AChR-MG+ 2) AChR-MG+ with thymoma 3) AChR-MG- with thymoma and 4) AChR-MG- without thymoma. Of the total participants, 75 are expected to consent to BM donation and 20 AChR-MG non thymomatous patients will be selected for 3T-MRI imaging of the thymus. From the collected specimens, the compositions of immune cell populations and (auto)antibody production will be analyzed in the presence and absence of immunomodulatory drugs. In vitro findings will be correlated with the clinical status of participants at enrollment and follow-up assessments. Additionally, 3T-MRI will be used to assess the presence and severity of TFH, with outcomes correlated to clinical status at enrollment and follow up assessments.

You can find more information in OMON: In vitro tests met bloedcellen om te voorspellen welk medicijn geschikt zal zijn in individuele patiënten met MG | Onderzoek met mensen