Nishtha Chandra
Corticosteroid Dose Tapering in Patients with Generalised Myasthenia Gravis on Zilucoplan: An Interim Analysis of RAISE-XT
Autori
- SOFIA MARINI (NEUROLOGIA, FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI, ROME, ITALY – NEUROLOGY)
- MIRIAM FREIMER (DEPARTMENT OF NEUROLOGY, THE OHIO STATE UNIVERSITY WEXNER MEDICAL CENTER, COLUMBUS, OH, USA – NEUROLOGY)
- CHANNA HEWAMADDUMA (ACADEMIC NEUROSCIENCE UNIT, SHEFFIELD TEACHING HOSPITALS FOUNDATION TRUST, SHEFFIELD, UK; SHEFFIELD INSTITUTE FOR TRANSLATIONAL NEUROSCIENCES (SITRAN), UNIVERSITY OF SHEFFIELD, SHEFFIELD, UK – NEUROLOGY)
- MARIA ISABEL DA SILVA LEITE (NUFFIELD DEPARTMENT OF CLINICAL NEUROSCIENCES, UNIVERSITY OF OXFORD, OXFORD, UK – NEUROLOGY)
- RAPHAËLLE BEAU LEJDSTROM (UCB PHARMA, BULLE, SWITZERLAND – NEUROLOGY)
- BABAK BOROOJERDI (UCB PHARMA, MONHEIM, GERMANY – NEUROLOGY)
- FIONA GRIMSON (UCB PHARMA, SLOUGH, UK – NEUROLOGY)
- NATASA SAVIC (UCB PHARMA, BULLE, SWITZERLAND – NEUROLOGY)
- JAMES FRANCIS HOWARD JR (DEPARTMENT OF NEUROLOGY, THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL, CHAPEL HILL, NC, USA – NEUROLOGY)
Presentatore
SOFIA MARINI (NEUROLOGIA, FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI, ROME, ITALY)
Modalità
Poster Session
Abstract
“Background
In the Phase 3 RAISE study (NCT04115293), zilucoplan significantly improved myasthenia gravis (MG)-specific outcomes in patients with acetylcholine receptor autoantibody-positive generalised MG. After the first 12 weeks of the ongoing, open-label extension study, RAISE-XT (NCT04225871), corticosteroid dose could be changed per the investigator’s discretion. Here, we evaluate changes in corticosteroid dose during zilucoplan treatment in RAISE-XT.
Methods
In RAISE-XT, adults who completed a qualifying double-blind study (NCT03315130/NCT04115293) self-administered daily subcutaneous zilucoplan 0.3mg/kg, either continuing previous zilucoplan treatment or switching from placebo. Primary outcome was incidence of treatment-emergent adverse events (TEAEs). This post hoc analysis assessed proportions of patients who discontinued/reduced or increased their corticosteroid dose relative to double-blind baseline and change from baseline (CFB) in Myasthenia Gravis Activities of Daily Living (MG-ADL) score at Week 60.
Results
200 patients enrolled. At Week 60, 30% (n=18/60) and 22% (n=12/54) of patients receiving corticosteroids in the zilucoplan and placebo-switch groups, respectively, had reduced or discontinued corticosteroids. Among these patients, mean dose reductions were 14mg and 16mg; mean (standard deviation [SD]) CFB in MG-ADL score was −5.00 (3.96) and −5.67 (6.89), respectively. At Week 60, 12% (n=7/60) and 7% (n=4/54) of patients in the zilucoplan and placebo-switch groups, respectively, had increased corticosteroid dose (~12mg mean dose increase in both groups) with mean (SD) CFB in MG-ADL score of −4.86 (2.55) and −9.75 (4.57). TEAEs occurred in 188 (94.0%) patients (data cut-off: 08 September 2022).
Conclusions
While receiving zilucoplan, discontinuation or reduction of concomitant corticosteroids was possible with maintained efficacy. Funding: UCB Pharma.”