Mycophenolate mofetil versus cyclophosphamide for remission induction in childhood polyarteritis nodosa: An open label, randomised, Bayesian, non-inferiority trial.



Brogan, Paul A, Arch, Barbara ORCID: 0000-0001-6060-8091, Hickey, Helen ORCID: 0000-0003-0467-0362, Anton, Jordi, Iglesias, Este, Baildam, Eileen, Mahmood, Kamran, Cleary, Gavin, Moraitis, Elena, Papadopoulou, Charalampia
et al (show 13 more authors) (2021) Mycophenolate mofetil versus cyclophosphamide for remission induction in childhood polyarteritis nodosa: An open label, randomised, Bayesian, non-inferiority trial. Arthritis & rheumatology (Hoboken, N.J.).

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Abstract

<h4>Objective</h4>Cyclophosphamide (CYC) is used in clinical practice off-label for induction of remission of childhood polyarteritis nodosa (cPAN). Mycophenolate mofetil (MMF) might offer a less toxic alternative: we explored their relative effectiveness in a randomised controlled trial (RCT).<h4>Methods</h4>International, open-label, Bayesian, RCT investigating the relative effectiveness of MMF and CYC for remission induction in cPAN. Eleven newly-diagnosed patients were randomised (1:1) to MMF or intravenous-CYC; all received the same glucocorticoid regimen. The primary endpoint was remission within 6-months whilst compliant with glucocorticoid taper. Bayesian distributions for remission rates were established a priori for MMF and CYC by experienced clinicians, and updated to posterior distributions on trial completion.<h4>Results</h4>Baseline disease activity/features were similar between groups. The primary remission endpoint occurred in 4/6 patients (67%) in the MMF group and 4/5 patients (80%) in the CYC group. Time to remission was shorter in the MMF group (median 7.4 weeks versus 17.5 weeks for CYC). No relapses occurred in either group within 18-months. Two serious infections were probably related to MMF. Physical and psychosocial quality-of-life scores were superior in the MMF group compared to CYC at 6-and 18-months. Combining the prior expert opinion with results from MYPAN provided posterior estimates of remission of 71% (90% CrI 51-83%) for MMF; and 75% (90% CrI 57-86%) for CYC.<h4>Conclusion</h4>Taking the prior opinion and the study results together, rates of remission induction in cPAN on MMF and CYC are similar, and MMF might be associated with better health-related quality of life than CYC.

Item Type: Article
Divisions: Faculty of Health and Life Sciences
Faculty of Health and Life Sciences > Institute of Population Health
Depositing User: Symplectic Admin
Date Deposited: 17 May 2021 09:27
Last Modified: 06 Sep 2022 07:30
DOI: 10.1002/art.41730
URI: https://livrepository.liverpool.ac.uk/id/eprint/3123023