Dataset Information

FcRn inhibitors for myasthenia gravis.

ABSTRACT: This is a protocol for a Cochrane Review (intervention). The objectives are as follows: To evaluate the benefits and harms of FcRn inhibitors for the maintenance treatment of myasthenia gravis in adult participants compared with control treatment (placebo, standard-of-care therapy, an alternative FcRn inhibitor, or an alternative immunomodulatory therapy). We will evaluate the efficacy of the treatment by the effect on disease severity and functional impairment, as assessed using a measurement tool validated for use in myasthenia gravis. Where possible, we will assess whether the effects of FcRn inhibitors differ according to different participant subgroups or different treatment regimens, or both. These data will be used to inform policymakers on the participant subgroups most likely to benefit from treatment and the most efficacious treatment regimen.

SUBMITTER: White LM

PROVIDER: S-EPMC12371811 | biostudies-literature | 2025 Aug

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

FcRn inhibitors for myasthenia gravis.

White Laura M LM Clay Fiona J FJ Forbes Anne-Marie AM Keh Ryan Yann Shern RYS Lilleker James B JB Spillane Jennifer J Storms Karen K Dodd Katherine C KC Sussman Jon J

The Cochrane database of systematic reviews 20250822

<h4>Objectives</h4>This is a protocol for a Cochrane Review (intervention). The objectives are as follows: To evaluate the benefits and harms of FcRn inhibitors for the maintenance treatment of myasthenia gravis in adult participants compared with control treatment (placebo, standard-of-care therapy, an alternative FcRn inhibitor, or an alternative immunomodulatory therapy). We will evaluate the efficacy of the treatment by the effect on disease severity and functional impairment, as assessed us ...[more]

PMID: 40843812

Similar Datasets

Project description:Myasthenia gravis (MG) is an autoimmune disease affecting the neuromuscular junction, whose clinical hallmark is muscle weakness and early fatigability. Azathioprine (AZA) is commonly used in Myasthenia Gravis therapy. AZA is a purine antagonist, which inhibits the cell cycle in the resting and DNA synthesis phases. It is usually used as an immunosuppressant to block T- and B-cell proliferation. AZA, bioconverted to 6-mercaptopurine by glutathione S-transferase (GST), can be metabolized either through the hypoxanthine phosphoribosyl transferase pathway to active 6-thioguanine nucleotides (6-TGN) or through the thiopurine S-methyltransferase (TPMT) pathway to inactive methyl-thiopurine metabolites. The incorporation of active 6-TGNs into DNA, causing breaks in DNA strands resulting in interference with RNA production and thereby protein synthesis, is responsible for the drug effect. The response to AZA is determined by which metabolic pathway is being favored. While balanced use of both HPRT and TPMT pathways results in responsiveness to AZA, hyperactivity of TPMT skews the balance towards the TPMT pathway and results in unresponsiveness to AZA with no pharmacological effect. In contrast, low TPMT activity skews the balance towards the HPRT pathway, resulting in increasing side-effects due to the accumulation of 6-TGNs. Current treatments for MG therapy are often inadequate because less than 40% of patients achieve complete remission with available drugs. This reflects the lack of drugs acting on target sites for which there is strong evidence of pathogenicity and the inability to identify responder patients. No criteria for responsiveness are available, exposing patients to unpredictable failures and unpredictable side effects. These individuals are at particular risk for adverse drug reaction (ADRs) or therapeutic failure. Genetic profiling with the Affymetrix drug metabolizing enzymes and transports genotyping array offers the ability to determine 1,931 variants and 225 genes involved in drug metabolism and disposition. Accordingly, the study of well-known drug-metabolizing genes can be involved in the specific metabolic pathway of a drug, which is more likely to define genotype-phenotype association and thereby genotype profiles relevant to drug response that can be applied as predictive biomarkers for pharmacological treatment.

Dataset Information

FcRn inhibitors for myasthenia gravis.

Publications

FcRn inhibitors for myasthenia gravis.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets