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Efficacy of etanercept in the tumor necrosis factor receptor-associated periodic syndrome: a prospective, open-label, dose-escalation study.

ABSTRACT: OBJECTIVE:To investigate the efficacy of etanercept in improving the symptoms and underlying inflammation in patients with tumor necrosis factor receptor-associated periodic syndrome (TRAPS). METHODS:Fifteen patients with TRAPS were enrolled in a prospective, open-label, dose-escalation study. Patients recorded attacks, symptom severity, and use of ancillary medications in a daily diary. Blood samples were collected during each period and measured for levels of acute-phase reactants. Between 7 years and 9 years after the conclusion of the initial study, patients completed a followup survey and were evaluated to determine the long-term outcome of etanercept treatment. RESULTS:Etanercept treatment significantly attenuated the total symptom score and reduced the frequency of symptoms. Etanercept also reduced levels of acute-phase reactants, particularly during asymptomatic periods. During a 10-year followup period, patients continued to receive etanercept for a median of 3.3 years, with a number of patients switching to anti-interleukin-1? receptor therapy or not receiving biologic agents, most frequently citing injection site reactions and lack of efficacy as reasons for discontinuation. However, patients continuing to receive etanercept had reduced symptoms at followup. CONCLUSION:Etanercept reduces symptoms and serum levels of inflammatory markers of TRAPS in a dose-dependent manner, but does not completely normalize symptoms or acute-phase reactant levels. Although long-term adherence to etanercept is poor, continuing to receive etanercept may provide continued symptomatic benefit.

SUBMITTER: Bulua AC

PROVIDER: S-EPMC3882089 | biostudies-literature | 2012 Mar

REPOSITORIES: biostudies-literature

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Efficacy of etanercept in the tumor necrosis factor receptor-associated periodic syndrome: a prospective, open-label, dose-escalation study.

Bulua Ariel C AC Mogul Douglas B DB Aksentijevich Ivona I Singh Harjot H He David Y DY Muenz Larry R LR Ward Michael M MM Yarboro Cheryl H CH Kastner Daniel L DL Siegel Richard M RM Hull Keith M KM

Arthritis and rheumatism 20120301 3

<h4>Objective</h4>To investigate the efficacy of etanercept in improving the symptoms and underlying inflammation in patients with tumor necrosis factor receptor-associated periodic syndrome (TRAPS).<h4>Methods</h4>Fifteen patients with TRAPS were enrolled in a prospective, open-label, dose-escalation study. Patients recorded attacks, symptom severity, and use of ancillary medications in a daily diary. Blood samples were collected during each period and measured for levels of acute-phase reactan ...[more]

PMID: 22006113

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Project description:Background: Systemic sclerosis (SSc) is a chronic autoimmune disease. We tested the safety of a single intravenous injection of intrafamilial allogeneic bone marrow-derived mesenchymal stromal cells (BM-MSC) to treat severe SSc. Methods: We conducted the first prospective, monocentric, dose-escalation, phase I/II clinical trial investigating the feasibility and safety of a single allogeneic BM-MSC infusion in 20 severe SSc patients who were refractory or had a contraindication to conventional immunosuppressive therapy or autologous hematopoietic stem cell transplant. Patients were assigned to an infusion of either 1x10^6 BM-MSC/kg or 3x10^6 BM-MSC/kg obtained from 20 independent intrafamilial donors. Primary endpoint was the rate of grade ≥ 3 Severe Adverse Events (SAE; NCI Common Terminology Criteria for Adverse Events (v5.0)) in the first 10 days post-BM-MSC infusion. Secondary endpoints included adequacy of BM-MSC production, medium-term SAE, as well as clinical and immunological response. Findings: No Grade 3 ≥ SAE occurred in the first 10 days following MSC infusion in the 20 SSc patients. A significant improvement in modified Rodnan Skin Score (p<0.0001) was observed after BM-MSC infusion, which peaked at 3 months and was sustained for 1 year. No changes in health-related quality of life or pulmonary function were observed. CD8pos T-Cell and NK cell counts slightly increased after infusion (p<0.05) and two patients developed de novo donor-specific anti-HLA. Circulating TGF-β level at baseline was significantly higher in non-responder compared to responder patients (p<0.05). A pattern of low IDO activity, CCL2 production, and HLA-DR expression in BM-MSC batches under in vitro stimulation by IFN-beta was associated with a lack of clinical response in recipient SSc patients. Interpretation: A single infusion of allogeneic BM-MSC transplant was safe in severe SSc patients and triggered short-term disease modifying effects. Clinical response was associated with both the recipients biological features and the functional properties of infused BM-MSC.

Dataset Information

Efficacy of etanercept in the tumor necrosis factor receptor-associated periodic syndrome: a prospective, open-label, dose-escalation study.

Publications

Efficacy of etanercept in the tumor necrosis factor receptor-associated periodic syndrome: a prospective, open-label, dose-escalation study.

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