Table 1. Comparison of the design of 2 randomised controlled trials for vasculitis: MYPAN versus MYCYC.
Trial name and study population | MYPAN a : PAN b in children (≥4 and ≤ 18 years) | MYCYC c : ANCA d associated vasculitis in adults & children (8 children, 132 adults) |
---|---|---|
Hypothesis | MMF e is not inferior (<10% absolute difference) to intravenous CYC f for induction of remission | MMF is not inferior (<12% absolute difference) to intravenous CYC for induction of remission |
Entry criteria | i) Must fulfil classification criteria for PAN; ii) 1or more major PVAS g items and/or 3 or more minor PVAS items; iii) Must be newly diagnosed patients | i) Chronic inflammatory disease lasting at least 4 weeks; characteristic histology on biopsy and/or a positive ANCA; ii) 1 or more major BVAS h items and/or 3 or more minor PVAS items; iii) Must be newly diagnosed patients |
Primary endpoint | Remission within 6 months defined as PVAS 0/63 on 2 consecutive readings at least one month apart on protocol steroid taper | Remission within 6 months defined as BVAS 0/63 on 2 consecutive readings at least one month apart on protocol steroid taper |
aMYPAN: Mycophenolate mofetil for childhood polyarteritis nodosa;
bPAN: polyarteritis nodosa;
cMYCYC: Mycophenolate mofetil versus cyclophosphamide for ANCA associated vasculitis;
dANCA: anti neutrophil cytoplasmic antibodies;
eMMF: Mycophenolate mofetil;
fCYC: cyclophosphamide;
gPVAS: Paediatric Vasculitis Activity Score;
hBVAS: Birmingham Vasculitis Activity Score.