Mycophenolate Mofetil Versus Cyclophosphamide for Remission Induction in Childhood Polyarteritis Nodosa: An Open‐Label, Randomized, Bayesian Noninferiority Trial

Objective Cyclophosphamide (CYC) is used in clinical practice off-label for the induction of remission childhood polyarteritis nodosa (PAN). Mycophenolate mofetil (MMF) might offer a less toxic alternative. This study was undertaken to explore relative effectiveness CYC and MMF treatment randomized controlled trial (RCT). Methods an international, open-label, Bayesian RCT investigate PAN. Eleven patients with newly diagnosed PAN were (1:1) receive or intravenous CYC; all received same glucocorticoid regimen. The primary end point within 6 months while compliant taper. distributions rates established priori by experienced clinicians updated posterior on completion. Results Baseline disease activity features similar between 2 groups. met 4 (67%) group 5 (80%) group. Time shorter compared (median 7.1 weeks versus 17.6 weeks). No relapses occurred either 18 months. Two serious infections found be likely linked treatment. Physical psychosocial quality-of-life scores superior at Combining prior expert opinion results from present provided estimates 71% (90% credibility interval [90% CrI] 51, 83) 75% CrI 57, 86). Conclusion results, taken together opinion, indicate that are treatment, associated better health-related quality life than