10 Rheumatology Advances From 2016

Kevin D. Deane, MD, PhD


December 19, 2016

Odds and Ends

New Guidelines Regarding Antimalarials and Eye Toxicity

In 2016, the American Academy of Ophthalmology published revised guidelines for the use and follow-up of antimalarials related to prevention of ocular disease.[28] The new recommendations suggested that the prevalence of some form of retinal toxicity was 1% at 5 years and < 2% at 10 years, but almost 20% after 20 years of use. These estimates were based on a large retrospective study that utilized clinical data and retinal images.[29]

According to these new guidelines, risk for antimalarial ocular toxicity was minimized if hydroxychloroquine dosages < 5 mg/kg/day were used and were based on actual body weight rather than ideal weight; this differs from prior recommendations that recommended a dosage < 6.5 mg/kg/day based on ideal body weight. It will be of interest to see how these guidelines are followed in real-world care by rheumatologists, especially in light of emerging data that underdosing of hydroxychloroquine may result in increased risk for disease flares.[30]

Growing Use of 'Practical Trials'

Practical (or pragmatic) clinical trials examine clinically relevant alternatives, typically in real-world clinical settings and in the absence of blinding.[31] They are typically cheaper and easier to implement than randomized, controlled, blinded trials. Although they result in less robust data than controlled trials, they can still provide very valuable information about the effects of therapies in the real world.

A growing number of these types of trials are being performed in rheumatology. As an example, the CareRA study in Europe demonstrated the effectiveness of a combination of methotrexate and tapering steroids in early RA.[32] Given the budget constraints on medical research worldwide, these types of trials may be more commonly seen in rheumatology, and therefore the rheumatology community should be familiar with them.

Immune Checkpoint Inhibitors Creating New Autoimmunity

Immune-checkpoint inhibitors, such as anti-programmed death 1 agents (eg, nivolumab and pembrolizumab) and antibodies that block the CTLA4 receptor (eg, ipilimumab), are being increasingly used to improve treatment of a variety of cancers, including melanoma and lung cancer.[33] However, several immune-mediated manifestations are associated with the use of these agents; these include rashes, arthritis, hepatitis, nephritis, colitis, and pneumonitis, as well as vitiligo, thyroid disease, and diabetes,[34] with vitiligo, thyroid disease, and diabetes more commonly seen with anti-CTLA4 therapy. Typically, patients improve with suspension of immune-checkpoint inhibitor therapy and short-term use of corticosteroids; studies are under way to identify the best treatment regimens and the potential long-term effects of suspension of therapy and use of steroids on the anticancer effectiveness of these agents.[33,34]

For the most part, cancer specialists are managing these treatment-related manifestations of immune-checkpoint inhibitors. However, rheumatologists should be aware of these manifestations, because they may be asked to evaluate patients to ensure that no primary rheumatic disease is present or to help with management.


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