Steroid-sparing effect of corticorelin acetate in peritumoral cerebral edema is associated with improvement in steroid-induced myopathy

Abstract

Purpose: To compare the safety and efficacy of corticorelin acetate (CrA) and placebo in patients with malignant brain tumors requiring chronic administration of dexamethasone (DEX) to control the signs and symptoms of peritumoral brain edema (PBE).

Patients and methods: Prospective, randomized, double-blind study of 200 patients with PBE on a stable dose of DEX. Initially, DEX dose was decreased by 50% over a 2-week period and then held at this level for 3 weeks. The primary end point was the proportion of patients who responded to treatment-patients who achieved a ≥ 50% DEX reduction from baseline and achieved stable or improved neurologic examination score and Karnofsky performance score at week 2, and then continued to respond at week 5.

Results: One hundred patients received subcutaneous injections of 1 mg twice per day of CrA and 100 patients received placebo for the duration of the study period. Although results did not attain statistical significance (at the P < .05 level), a clinically important difference in the proportion of responders between the CrA group (57.0%) and the placebo group (46.0%; P = .12) was observed. In addition, the maximum percent reduction in DEX dose achieved during the double-blind 12-week study was significantly greater in the CrA group (62.7%) than in placebo group (51.4%; P < .001). Patients receiving CrA demonstrated an improvement in myopathy and were less likely to develop signs of Cushing syndrome.

Conclusion: CrA enables a reduction in steroid requirement for patients with PBE and is associated with a reduction in the incidence and severity of common steroid adverse effects, including myopathy.

Trial registration: ClinicalTrials.gov NCT00088166.