Regarding medication for TRAPS:
ACR: Enbrel (Etanercept) in
TNF-Receptor Associated Periodic Fever Syndrome
Doctor's Guide
November 14, 2001
By Bruce Sylvester
SAN FRANCISCO, CA. -- November 14, 2001 -- Enbrel (etanercept) therapy
appears to safely decrease the severity, duration, and frequency of inflammatory
symptoms in patients with tumour necrosis factor receptor associated fever
syndrome (TRAPS).
Researchers reported the findings yesterday (Nov. 13) at the American College
of Rheumatology (ACR) annual meeting in San Francisco, California.
"The [tumour necrosis factor] TNF receptor is one of the major mediators
of inflammation in the human body. So we are not only learning more about
treatment for the small number of TRAPS patients, but also more about the
crucial TNF receptor pathway," said Keith Hull, MD, lead researcher and
clinical fellow at the National Institutes of Arthritis Musculoskeletal
and Skin Diseases in Bethesda, Maryland.
"TRAPS is rare but it is one of the few diseases in which there is a mutation
in the TNF receptor pathway. So our research will become a model for studying
the TNF receptor pathway. Hopefully it will lead to a better understanding
of inflammatory pathways in the body, so we cam aim research at a broader
group of inflammatory diseases, like rheumatoid arthritis," Dr. Hull said.
TRAPS is an autoinflammatory disorder resulting from mutations in TNFRSF1A,
the gene that encodes the 55 kDa receptor for TNF. Clinically, patients
present with recurring inflammatory attacks consisting of fever, rash, conjunctivitis,
abdominal pain, and myalgia that are often severely disabling.
Nonsteroidal anti-inflammatory drug (NSAID) therapy is largely ineffective
in TRAPS patients, while glucocorticoids are somewhat more effective but
have significant long-term toxicities.
National Institutes of Health researchers undertook a six-month, open-labeled
pilot study to determine the safety and efficacy of standard doses of Enbrel
to alleviate symptoms of TRAPS.
Fifteen symptomatic patients with known mutations in TNF RSF1A were enrolled
into an Institutional Review Board-approved protocol consisting of four
three-month phases: baseline observation; Enbrel, 25 mg for adults or 0.4
mg/kg for children, twice weekly; Enbrel, same dosing, three times per week;
and finally withdrawal of Enbrel.
Patients recorded symptoms and use of medications using a standardised
diary throughout each phase of the study. Additionally, researchers took
blood draws during each phase to assess C-reactive protein (CRP), erythrocyte
sedimentation rate (ESR), and serum amyloid-A (SAA) levels from patients
at baseline, between attacks and during attack episodes.
Results from five patients who have completed the study reveal the ability
of twice-weekly Enbrel to significantly decrease symptoms (derived using
an attack score) by 74 percent from pretreatment levels, and by 87 percent
when administered three times weekly. Patients returned to pretreatment symptom
levels following the withdrawal of Enbrel.
Enbrel dose-dependently decreased the baseline CRP (49 percent and 76 percent,
respectively), ESR (23 percent and 47 percent, respectively), the amount
of NSAIDs (61 percent and 87 percent, respectively) and glucocorticoids (80
percent and 85 percent, respectively) taken to relieve symptoms. The only
reported adverse effect was that of minor local injection site irritation.
"We were encouraged by its ability to decrease reported symptoms in 66
percent of patients, the first group of subjects to complete the protocol,"
Hull said.
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