A Promising Treatment for Polymyalgia Rheumatica

Brief Report: A Prospective Open-Label Phase IIa Trial of Tocilizumab in the Treatment of Polymyalgia Rheumatica

Lally L, Forbess L, Hatzis C, Spiera R
Arthritis Rheumatol. 2016;68:2550-2554

Study Summary

In this article, Lally and colleagues present the results of an open-label trial of the anti-interleukin (IL)-6 receptor agent tocilizumab in 10 patients with isolated polymyalgia rheumatica (PMR). All patients were diagnosed at inclusion with PMR based on the Healy criteria and were also retrospectively determined to meet classification by the American College of Rheumatology/European League Against Rheumatism criteria. The patients had been on steroids for less than 1 month, and those who had doses higher than 30 mg/day were excluded. Patients were given tocilizumab 8 mg/kg monthly for 1 year, and steroids were rapidly tapered according to a set protocol. The primary endpoint was the proportion of patients in whom PMR was in remission without steroid treatment. Safety and tolerability were also assessed. The authors also retrospectively created a comparator group of 10 patients with PMR who were treated according to usual care (ie, steroids but no tocilizumab).

One patient withdrew after an infusion reaction (second dose), leaving nine patients in the analyses. All nine met the primary endpoint and were off steroids and had relapse-free remission at 6 months. In addition, disease remained in remission in these patients who completed the full 15-month trial, including at the last study visit performed 3 months after the final tocilizumab dose.

From a safety standpoint, one patient discontinued therapy due to an infusion reaction labeled as "mild." Another patient had episodic neutropenia that led to holding infusions at weeks 40 and 48, although no infections were noted. Otherwise, the most frequent adverse event was upper respiratory tract infections, which occurred in five patients and were considered mild by the investigators.

None of the 10 "comparator" subjects who were similar at baseline in terms of age, sex, and mean initial steroid dose were in remission at 6 months in absence of steroids. Adverse events were not evaluated in this group.