Teprotumumab is shown to be effective for proptosis in thyroid eye diseases

In-office treatment of patients with multiple sclerosis showed that early treatment with dimethyl fumarate significantly reduced annual relapse rates compared to 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 from October 10-12, 2018 in Berlin.

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The authors of this interim post hoc analysis of the ongoing ESTEEM trial attempted to assess the true efficacy of sustained-release dimethyl fumarate in patients with multiple sclerosis treated within 3 years of diagnosis and previously treated with only 1 interferon . Beta / glatiramer acetate, or that were treatment naive.

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The ESTEEM study enrolled 3,075 patients newly prescribed dimethyl fumarate for the treatment of relapsing-remitting multiple sclerosis. Efficacy outcomes were measured using annualized relapse rates obtained using a negative repeated measurement binomial model. Recurrence rates were determined for the total population, newly diagnosed patients without prior treatment who started dimethyl fumarate within 1 year of diagnosis (n = 770), patients with early multiple sclerosis who started dimethyl fumarate within 3 years of diagnosis (n = 1291), rated. , and patients who switched treatment from interferon-beta / glatiramer acetate to dimethyl fumarate (n = 1915).

The results showed that 73% of the patients enrolled in ESTEEM were receiving treatment at the time of the interim analysis. The investigators followed these patients for a median of 13 months. For each subgroup, the unadjusted annualized recurrence rates for the year before the start of dimethyl fumarate therapy were compared with those for the 24 months after the start of dimethyl fumarate therapy: total 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); Patients with early multiple sclerosis, 1.03 (95% CI, 0.99-1.08) vs. 0.17 (95% CI, 0.14-0.19); and patients who switched treatment, 0.69 (95% CI, 0.65-0.73) vs. 0.16 (95% CI, 0.14-0.17). This corresponds to an 81% lower relapse rate for the general population (85% in newly diagnosed patients, 84% in early patients, and 77% in patients who have switched therapy).

In patients with multiple sclerosis, the annualized recurrence rates after treatment with dimethyl fumarate were significantly reduced compared to the pre-treatment period. The researchers concluded that these results confirm the true effectiveness of dimethyl fumarate in treating multiple sclerosis in the early course of the disease.

Disclosure: Several authors explain links to the pharmaceutical industry. For a full list of author disclosures, see the original reference.

Click here to learn more about ECTRIMS 2018.


R. Macdonell, K. Giles, K. Balashov, et al. Effective efficacy of sustained-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. 10-12 October 2018; Berlin, Germany. Poster P595.

Teprotumumab, an insulin-like growth factor I receptor (IGF-IR) inhibitor, has shown comparable efficacy in reducing proptosis in patients with thyroid eye disease (TED) with low inflammation that the previous study found in patients with high inflammatory activity Results presented at the American Academy of Ophthalmology’s 2021 Annual Meeting, held in New Orleans from November 12-15, 2021.

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 disease that can cause proptosis or bulging of the eyes and cause one or both eyes to move out of their sockets. Proptosis can become chronic and resistant to therapy, but there is strong evidence that IGF-IR may play a role in the pathogenesis of TED by blocking pathological immune responses in active TED.

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

The open-label OPTIC-X study included a total of 15 patients (47.2 ± 1 6.0 years, 10 women) with a TED duration of 11.7 ± 2.2 months and a baseline proptosis of 22.3 ± 2 , 9 mm. Patients with TED who were previously randomized to placebo in the OPTIC RCT and who continued to have proptosis at the end of the study were also included. Those with a baseline clinical activity score (CAS) <4 were included in the analysis.

In the study, patients received 8 infusions of teprotumumab over a period of 24 weeks. The primary endpoint was response to teprotumumab with a propotosis reduction of ≥ 2 mm.

At week 24, the researchers found that 13 patients (86.7%) were responders with a mean proptosis reduction of 2.9 ± 1.7 mm. Of the 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 a comparable effectiveness of teprotumumab proptosis in TED patients with low inflammation, measured by CAS, as previously shown in patients with high inflammatory activity,” the researchers concluded.

Patients and their families advocate their right to access the latest medical research papers to find potential treatments and understand health conditions. However, many research articles cost an average of $ 35 each or require membership to access and read the database.

This is particularly stressful for families with relatives who suffer from rare diseases and who are already paying high medical costs.

Although there are ways to bypass the system, such as: For example, contacting the lead study director to get a copy of the article or going 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 battling against the clock .

“It is patients who enable new research by participating 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 real end users of the research results. They need research results in order to make informed decisions about their care and to fully share these decisions with their healthcare providers. “

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Scientific publishers and academia have been arguing about this question for years. The Elsevier company, for example, took 40% of its income as profit last year. Scientists find this particularly worrying, as much of the research relies on taxpayers ’money.

With calls for open access, the trend of patient-centered research is growing. JAMA dedicates part of its website to clinical articles with less technical jargon and more accessible to the public. PubMed Central also offers millions of articles for free to readers.

PCORI, a group dedicated to funding research to educate patients about everyday health choices, has worked to overcome some of these barriers. They provide funding for peer-reviewed open access articles so that everyone can access the information. They also help patients and their families compose research studies into digestible summaries.

“Journal articles and research reports are typically written by and for clinical and scientific audiences, and contain scientific language and graphics that can be difficult for laypeople to understand,” said Ms. Slutsky. “PCORI is therefore developing two more user-friendly abstracts of around 500 words each on the results of all of the studies we sponsor. One of these abstracts is for laypeople and the other for clinical professionals. “


Mak A. Who can read the research we pay for? Slate. August 21, 2018. Accessed September 19, 2018.


Douglas RS, Francis-Sedlak M. Efficacy of Teprotumumab in TED Patients with Low Inflammation as Measured by Clinical Activity Score. Paper presented at the 2021 annual meeting of the American Academy of Ophthalmology; 12-15 November 2021; New Orleans. Summary PA059.

This article originally appeared on Ophthalmology Advisor

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