Non-Cyclic Rozanolixizumab Administration in Complex Generalized Myasthenia Gravis

Rozanolixizumab, an inhibitor of the neonatal Fc receptor (FcRn), is used to treat patients with generalized myasthenia gravis (gMG) refractory to standard therapies. However, the approved six-week cyclic regimen may be associated with a "wearing-off" effect and disease fluctuations. We evaluated the non-cyclic administration of rozanolixizumab in patients with complex gMG.

We retrospectively studied eight patients with anti-acetylcholine receptor antibody-positive complex gMG treated at Bordeaux University Hospital. "Complex" was defined by refractoriness, contraindications, intolerance to immunosuppressants or highly active MG. Patients transitioned to weekly or fortnightly rozanolixizumab after relapse during intermittent therapy. The primary outcome was ≥ 2-point improvement in Myasthenia Gravis Activities of Daily Living (MG-ADL) score at 3 months. Clinical data and serum immunoglobulin G (IgG) levels were collected at baseline, 3, 6, and 12 months.

The cohort (mean age 70.5 years; 75% female) had a mean baseline MG-ADL score of 10.8, which improved rapidly to 4.1 at 3 months. Hospitalization days dropped from 15.8/month pre-treatment to zero by 6-12 months. Corticosteroids were tapered from 30.3 to 13.3 mg/day at 1 year. Mean serum IgG levels declined by almost 50% and stabilized. Only one discontinuation occurred due to gastrointestinal symptoms; no serious infections or severe adverse events were observed.

Fortnightly rozanolixizumab yielded sustained clinical benefits, reduced hospitalizations, and enabled steroid tapering in complex gMG patients. This approach may prevent cyclical relapses seen with intermittent dosing. Larger studies are needed to confirm efficacy and long-term safety.