As part of the primary endpoint, the response rate was determined according to the total improvement score using the 2016 ACR/European League Against Rheumatism (EULAR) myositis response criteria. Fifty percent of patients had minimal improvement and 50% of patients had moderate improvement in disease activity as measured by these criteria. The medication was well tolerated, with no significant adverse effects.
Paik et al. noted this study demonstrated clinical effcacy of a pan-jakinib using validated myositis response criteria and that randomized controlled trials are indicated to further explore this treatment.5
Researchers have demonstrated the clinical efficacy of a pan-jakinib using validated myositis response criteria. More randomized controlled trials are necessary to further explore this treatment option.
Reexamining Treatments
Dr. Chenoy pointed out that what’s old is new again. Although tacrolimus was already being studied in patients with refractory polymyositis and interstitial lung disease (ILD) in 1999, calcineurin inhibitors are once more a subject of interest, as demonstrated by a study comparing prednisolone and tacrolimus with prednisolone and cyclosporin A in the treatment of ILD in patients with dermatomyositis and polymyositis.6
This prospective, multicenter, open-label, randomized phase 2 trial had a primary endpoint of progression-free survival in the intention-to-treat population after 52 weeks of follow-up. Although the difference was not statistically significant, patients in the group treated with prednisolone and tacrolimus had a higher rate of progression-free survival than the group treated with prednisolone and cyclosporin A (87% vs. 71%, P=0.16). In addition, the patients in the group treated with prednisolone and tacrolimus had a higher rate of overall survival compared with the group treated with prednisolone and cyclosporin A (97% vs. 93%, P=0.50). The forced vital capacity (FVC) did significantly increase in both groups.7
In 1992, plasma exchange and leukopheresis were studied in the treatment of patients with polymyositis and dermatomyositis.8 These treatments are again relevant as part of a combined treatment regimen for patients with anti-melanoma differentiation-associated gene 5 (anti-MDA-5) antibody-positive dermatomyositis, which is frequently associated with a rapidly progressive form of ILD. Patients with new-onset disease were treated with high-dose glucocorticoids (i.e., 1 mg/kg/day of prednisolone), tacrolimus and intravenous cyclophosphamide. Plasmapheresis was used if a patient’s condition worsened after the initial treatment regimen. This group was compared with a historical control group that had received high-dose glucocorticoids and the step-up addition of immunosuppressants, if indicated.
The most significant finding: The combined immunosuppressive group showed significantly higher six-month survival when compared with the historical control group (89% vs. 33%), indicating that up-front combined therapy may benefit patients with anti-MDA-5-antibody-positive disease with aggressive features.9
