Subcutaneous Efgartigimod PH20 Treatment in Participants With Generalized Myasthenia Gravis in ADAPT-SC+: Interim Analyses on Quality of Life, Efficacy, Tolerability, and Long-Term Safety

Samantha Griffiths

Subcutaneous Efgartigimod PH20 Treatment in Participants With Generalized Myasthenia Gravis in ADAPT-SC+: Interim Analyses on Quality of Life, Efficacy, Tolerability, and Long-Term Safety

Autori

TUAN VU (DEPARTMENT OF NEUROLOGY, UNIVERSITY OF SOUTH FLORIDA, MORSANI COLLEGE OF MEDICINE, TAMPA, FLORIDA, USA – )
LAURA FIONDA (NEUROMUSCULAR AND RARE DISEASE CENTRE, DEPARTMENT OF NEUROSCIENCE, MENTAL HEALTH AND SENSORY ORGANS (NESMOS), SAPIENZA UNIVERSITY OF ROME, SANT’ANDREA HOSPITAL, ROME, ITALY – NEUROLOGY)
JAMES F. HOWARD (DEPARTMENT OF NEUROLOGY, THE UNIVERSITY OF NORTH CAROLINA, CHAPEL HILL, NORTH CAROLINA, USA – )
DENIS KOROBKO (STATE BUDGETARY HEALTHCARE INSTITUTION OF NOVOSIBIRSK REGION “”STATE NOVOSIBIRSK REGIONAL CLINICAL HOSPITAL,”” NOVOSIBIRSK, RUSSIA – )
MAREK SMILOWSKI (DEPARTMENT OF HEMATOLOGY AND BONE MARROW TRANSPLANTATION, MEDICAL UNIVERSITY OF SILESIA, KATOWICE, POLAND – )
FIEN GISTELINCK (ARGENX, GHENT, BELGIUM – )
SOPHIE STEELAND (ARGENX, GHENT, BELGIUM – )
JAN NOUKENS (CURARE CONSULTING BV, THE NETHERLANDS – )
JANA PODHORNA (ARGENX, GHENT, BELGIUM – )
YUEBING LI (CLEVELAND CLINIC, CLEVELAND, OHIO, USA – )
KIMIAKI UTSUGISAWA (DEPARTMENT OF NEUROLOGY, HANAMAKI GENERAL HOSPITAL, HANAMAKI, JAPAN – )
FRANCESCO SACCÀ (NRSO DEPARTMENT, GENESIS CENTER, FEDERICO II UNIVERSITY OF NAPLES, NAPLES, ITALY – )
HEINZ WIENDL (DEPARTMENT OF NEUROLOGY, UNIVERSITY OF MÜNSTER, MÜNSTER, GERMANY – )
JAN L. DE BLEECKER (GHENT UNIVERSITY HOSPITAL, GHENT, BELGIUM – )
RENATO MANTEGAZZA (DEPARTMENT OF NEUROIMMUNOLOGY AND NEUROMUSCULAR DISEASES, FONDAZIONE ISTITUTO NEUROLOGICO CARLO BESTA, MILAN, ITALY – )

Presentatore

LAURA FIONDA (NEUROMUSCULAR AND RARE DISEASE CENTRE, DEPARTMENT OF NEUROSCIENCE, MENTAL HEALTH AND SENSORY ORGANS (NESMOS), SAPIENZA UNIVERSITY OF ROME, SANT’ANDREA HOSPITAL, ROME, ITALY)

Modalità

Oral Communication

Abstract

“Introduction
In ADAPT-SC, subcutaneous (SC) efgartigimod PH20 (coformulated with recombinant human hyaluronidase PH20) demonstrated noninferior total IgG reduction to efgartigimod intravenous, resulting in similar clinical improvement in participants with generalized myasthenia gravis (gMG). Participants completing ADAPT-SC, or enrolled in ADAPT+, could roll over into the ongoing, open-label extension, ADAPT-SC+.

Objective
Evaluate quality of life (QoL), efficacy, tolerability, and long-term safety of efgartigimod PH20 SC in participants with gMG in ADAPT-SC+.

Methods
Efgartigimod PH20 SC 1000 mg was administered in cycles of 4 once-weekly injections. Subsequent cycles were initiated ≥4 weeks from the last dose based on clinical evaluation.

Results
As of March 2022, 164 participants received ≥1 dose of efgartigimod PH20 SC. Most participants completed ≈3 cycles. Mean(SD) study duration was 170(59) days (72 patient-years). Mean(SE) MG-QoL15r scores (range 0–30, higher=worse QoL) improved from 13.7(0.52) at baseline to 8.8(0.50) at week 4, cycle 1. Similarly, EQ-5D-5L VAS scores (range 0–100, higher=better QoL) improved from 59.3(1.46) to 73.0(1.35). Improvements in QoL and total MG-ADL scores were consistent across multiple subsequent cycles. Speed of onset, magnitude, and repeatability of MG-ADL improvements were similar to efgartigimod IV in ADAPT/ADAPT+. The most frequent adverse events were injection site erythema (25.6%), headache (15.2%), and COVID-19 (11.6%). All injection site reactions (ISRs) were mild/moderate, most resolved spontaneously, and incidence decreased with subsequent cycles.

Conclusions
Treatment with efgartigimod PH20 SC improves QoL and function in participants with gMG. Safety and tolerability of efgartigimod PH20 SC was similar to efgartigimod IV, except mild/moderate ISRs, none of which led to treatment discontinuation.”