TNF receptor-associated periodic syndrome: Etanercept shows only limited benefits
A study has shown that Etanercept ( Embrel ) reduces the frequency and severity of symptoms of TNF receptor-associated periodic syndrome ( TRAPS ), a rare inherited condition characterized by recurrent fevers, abdominal pain and skin rashes.
The study, published in Arthritis & Rheumatism, also points out the need for the development of additional therapies to more thoroughly ease symptoms and prevent long-term complications of the disease.
The study was released by researchers at the National Institute of Arthritis and Musculoskeletal and Skin Diseases ( NIAMS ), a part of the National Institutes of Health.
TRAPS is associated with mutations in the gene coding for tumor necrosis factor receptor 1 ( TNFR1 ), a critical molecule in receiving inflammatory signals in the body's immune system.
Etanercept is one of a class of drugs that block tumor necrosis factor, a protein implicated in the harmful inflammation in TRAPS, as well as a number of common rheumatic diseases, including rheumatoid arthritis.
While the drug has been used in the treatment of TRAPS for about 10 years, this is the first formal study to look at its effectiveness long-term.
The study was conceived in 2001 by Keith Hull, then a rheumatology fellow in the NIAMS under the supervision of Daniel Kastner, one of the discoverers of the TNF receptor mutations in TRAPS, and now the scientific director of the NIH's National Human Genome Research Institute.
The initial study enrolled 15 patients with TRAPS. Each patient kept a daily diary of attacks, symptom severity, and use of additional medicines, and had periodic blood tests to measure acute phase reactants, proteins that are produced by the liver to fight infection and serve as markers of inflammation in the blood.
While on treatment with Etanercept, patients reported lower symptom scores and a greater number of symptom-free days each week.
Etanercept has also reduced levels of acute phase reactants, particularly during asymptomatic periods.
To find out whether Etanercept was effective as a long-term treatment, researchers contacted all 15 patients treated with Etanercept seven to nine years after the conclusion of the initial study period.
Despite the overall beneficial effects of Etanercept, most patients discontinued the drug during the follow-up period due to a perceived lack of efficacy or painful injection site reactions, which could be related to the skin manifestations of the disease.
The three patients who remained on the drug, however, continued to report benefits, suggesting that for some, the drug can be an effective long-term treatment option.
However, the study did not show whether Etanercept could prevent the long-term consequences of TRAPS, chiefly a condition called amyloidosis, in which inflammatory proteins build up in the body, damaging the kidneys, heart and other organs.
Researchers are concerned that Etanercept may not prevent amyloidosis because inflammatory markers were not completely suppress.
Patients are still having some residual symptoms, attacks of fevers and rashes.
Researchers are still looking for other pathways to target in this disease. ( Xagena )
Source: NIH / National Institute of Arthritis and Musculoskeletal and Skin Diseases, 2011
The FDA ( U.S. Food and Drug Administration ) has approved Darzalex Faspro ( Daratumumab and Hyaluronidase-fihj ), a...
hATTR amyloidosis with polyneuropathy: Vutrisiran has improved progression of neuropathy, quality of life, and gait speed
HELIOS-A phase 3 study of Vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated ( ATTR )...
HELIOS-A study: Vutrisiran has reversed polyneuropathy manifestations with improvements in neuropathy, quality of life, and gait speed
Full positive results from the HELIOS-A phase 3 study of Vutrisiran, an investigational RNAi therapeutic in development for the treatment...
Light chain amyloidosis: depth of response prior to autologous stem cell transplantation to predict survival
The role of induction therapy prior to autologous stem cell transplant ( ASCT ) in immunoglobulin light chain ( AL...
Tafamidis in transthyretin amyloid cardiomyopathy: effects on transthyretin stabilization and clinical outcomes
Transthyretin ( TTR ) amyloidosis is a progressive systemic disorder caused by misfolded TTR monomers that cumulatively deposit in the...
Cerebral beta-amyloidosis and neurodegeneration among people with normal cognitive function aged 50–89 years
As preclinical Alzheimer's disease becomes a target for therapeutic intervention, the overlap between imaging abnormalities associated with typical ageing and...
Transthyretin amyloidosis is caused by the deposition of hepatocyte-derived transthyretin amyloid in peripheral nerves and the heart. A therapeutic approach...
Diflunisal, an anti-inflammatory drug, has shown success in treatment of patients with familial transthyretin amyloidosis
A study led by researchers from the Amyloidosis Center at Boston University School of Medicine ( BUSM ) and Boston...
The FDA ( Food and Drug Administration ) has approved a drug to help ease the suffering faced by those...