The following article is part of conference coverage from the 34th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) in Berlin, Germany. Neurology Advisor’s staff will be reporting breaking news associated with research conducted by leading experts in neurology. Check back for the latest news from ECTRIMS 2018.

Treatment of real-world patients with multiple sclerosis showed that early initiation of dimethyl fumarate significantly reduced annualized relapse rates compared with no treatment or prior treatment with other disease-modifying therapies. This research was presented at the 34th Congress of the European Committee for Treatment and Research in Multiple Sclerosis 2018, held October 10-12, 2018, in Berlin, Germany.


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The authors of this post-hoc interim analysis of the ongoing ESTEEM study sought to evaluate the real-world efficacy of delayed-release dimethyl fumarate in patients with multiple sclerosis who had been treated within 3 years of diagnosis, treated with only 1 prior interferon-beta/glatiramer acetate, or who were treatment-naive.


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The ESTEEM study included 3075 patients who were newly prescribed dimethyl fumarate to manage relapsing-remitting multiple sclerosis. Efficacy outcomes were measured using annualized relapse rates, which were obtained using a repeated-measure negative binomial model. Relapse rates were assessed for the overall population, newly diagnosed patients with no prior treatment and who initiated dimethyl fumarate within 1 year of diagnosis (n=770), early multiple sclerosis patients who initiated dimethyl fumarate within 3 years of diagnosis (n=1291), and patients who switched treatment from interferon-beta/glatiramer acetate to dimethyl fumarate (n=1915).

The results showed that 73% of patients enrolled in ESTEEM were receiving treatment at the time of the interim analysis. The investigators followed these patients for a median 13 months. For each subgroup, unadjusted annualized relapse rates for the year before starting dimethyl fumarate therapy were compared with those for the 24 months post dimethyl fumarate initiation: overall population, 0.8 (95% CI, 0.78-0.83) vs 0.15 (95% CI, 0.14-0.16); newly diagnosed patients, 1.12 (95% CI, 1.07-1.17) vs 0.17 (95% CI, 0.14-0.21); early multiple sclerosis patients, 1.03 (95% CI, 0.99-1.08) vs 0.17 (95% CI, 0.14-0.19); and patients who switched treatments, 0.69 (95% CI, 0.65-0.73) vs 0.16 (95% CI, 0.14-0.17). This represents an 81% lower relapse rate for the overall population (85% for newly diagnosed patients, 84% for early patients, and 77% for patients who switched therapies).

For patients with multiple sclerosis, annualized relapse rates were significantly reduced after treatment with dimethyl fumarate compared with the period before treatment initiation. The researchers concluded that these findings confirm the real-world effectiveness of dimethyl fumarate for treatment of multiple sclerosis early in the disease course.

Disclosures: Multiple authors declare associations with the pharmaceutical industry. Please see original reference for a full list of authors’ disclosures.

For more coverage of ECTRIMS 2018, click here.

Reference

Macdonell R, Giles K, Balashov K, et al. Real-world efficacy of delayed-release dimethyl fumarate in early multiple sclerosis: interim results from ESTEEM. Presented at: 34th Congress of the European Committee for Treatment and Research in Multiple Sclerosis. October 10-12, 2018; Berlin, Germany. Poster P595.

Teprotumumab, an insulin-like growth factor-I receptor (IGF-IR) inhibitor, has shown comparable efficacy in proptosis reduction in patients with thyroid eye disease (TED) with low inflammation as previously found in patients with high inflammatory activity, according to study results presented at the American Academy of Ophthalmology 2021 Annual Meeting, held November 12-15, 2021 in New Orleans.

Teprotumumab, a human monoclonal antibody, was recently approved by the US Food and Drug Administration (FDA) for the treatment of TED, also known as Graves’ disease. TED is a rare and debilitating autoimmune disorder that can lead to proptosis, or bulging of the eyes, and cause one or both eyes to move out of the sockets. Proptosis can become chronic and be resistant to therapy, but there’s strong evidence that suggests IGF-IR can play a role in the pathogenesis of TED via the blocking of pathologic immune responses in active TED.

The objective of the current study was to determine the efficacy of teprotumumab proptosis reduction in patients with TED with low inflammation compared with patients with high inflammation activity who previously did not respond to treatment or had a disease flare in two pivotal randomized trials.

The open-label OPTIC-X trial included a total of 15 patients (47.2±1 6.0 years, 10 women) with a TED duration of 11.7±2.2 months with a baseline proptosis of 22.3 ± 2.9 mm. Patients with TED who were previously randomized to placebo in the OPTIC RCT and continued to have proptosis at the end of the study were also included. Those with a baseline clinical activity score (CAS) <4 were part of the analysis.

In the trial, patients received 8 teprotumumab infusions over a 24-week period. The primary endpoint was a teprotumumab response of ≥2-mm proptosis reduction.

At week 24, the researchers noted 13 patients (86.7%) were responders with a mean proptosis reduction of 2.9±1.7 mm. Among patients with CAS <3, 10 of 12 (83.3%) were responders with a mean proptosis reduction of 2.6 ± 1.4 mm.

“The results suggest comparable teprotumumab proptosis efficacy in TED patients with low-level inflammation as measured by CAS as previously demonstrated in those with high inflammatory activity,” the researchers concluded.

Patients and their families are advocating for their right to access the latest medical research papers to help find potential treatments and understand health conditions. Yet, many research articles cost an average of $35 each or require a membership in order to access the database to read them.

This is particularly burdensome for families with loved ones who have rare diseases and are already paying substantial medical fees.

Although there are ways around the system like contacting the lead study investigator to obtain a copy of the article or heading over to a local library that has a subscription to the research database, these options can be time-consuming for families who feel like they are already fighting against the clock.

“It is patients who make new research findings possible through their participation in studies,” said Jean Slutsky, chief engagement and dissemination officer at the Patient-Centered Outcomes Research Institute (PCORI) in Washington, DC. “Patients and their caregivers are the true end-users of research results. They need research findings to help make informed decisions about their care and to share fully in making these decisions with their healthcare providers.”


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Scientific publishing companies and the research community have been at odds over this issue for years. The company Elsevier, for example, took 40% of its income as profit last year. Academics have found this particularly unsettling because much of its research is dependent on taxpayer dollars.

With calls for open access, the patient-centered research trend is growing. JAMA dedicates a portion of its site to clinical articles with less jargon and more accessibility to the public. PubMed Central also offers millions of pieces free for readers.

PCORI, a group dedicated to funding research to help inform patients of everyday healthcare decisions, has been working to help alleviate some of these obstacles. They provide funding for open access, peer-reviewed articles so anyone can access the information. They are also helping patients and their families synthesize research studies into digestible abstracts.

“Journal articles and research reports are usually written by and for clinical and scientific audiences and contain scientific language and graphs that can be difficult for laypeople to understand fully,” said Ms Slutsky. “PCORI is therefore developing two easier-to-use abstracts of about 500 words each on results from all the studies we fund. One of these abstracts is written for laypeople and the other is for clinical professionals.”

Reference

Mak A. Who gets to read the research we pay for? Slate. August 21, 2018. https://slate.com/technology/2018/08/who-gets-to-read-the-research-taxpayers-fund.html. Accessed September 19, 2018.

Reference

Douglas RS, Francis-Sedlak M. Teprotumumab efficacy in TED patients with low inflammation as measured by clinical activity score. Paper presented at: American Academy of Ophthalmology 2021 Annual Meeting; November 12-15, 2021; New Orleans. Abstract PA059.

This article originally appeared on Ophthalmology Advisor