[Pubmed] Phase II Trial in AChR Ab+ Myasthenia Gravis of Transition from Intravenous to Subcutaneous Immunoglobulin: The
Posté : 13 févr. 2023 12:00
Eur J Neurol. 2023 Feb 13. doi: 10.1111/ene.15745. Online ahead of print.
ABSTRACT
BACKGROUND/OBJECTIVES: Data on maintenance therapy with SCIg in myasthenia gravis (MG) is limited. We report on transitioning AChR Ab+ MG patients on stable IVIg regimens as part of routine clinical care to SCIg 1:1.2.
METHODS: This multi-center North American open-label prospective investigator-initiated study had two components: IVIg Screening Period enrolling patients already on IVIg as part of routine clinical care (ISP; Weeks -10 to -1), followed by transition of stable MG subjects to SCIg of Experimental Treatment Period (ETP; Weeks 0 to 12). We hypothesized that more than 65% of patients entering the ETP would have a stable Quantitative Myasthenia Gravis (QMG) score from Week 0 to Week 12. Secondary outcome measures included other efficacy measures, safety, tolerability, IgG levels and treatment satisfaction.
RESULTS: We recruited 23 patients in the ISP and 22 entered the ETP. Twelve subjects (54.5%) were female and eighteen (81.8%) were white, with mean age 51.4±17 years. We obtained Week 12 ETP QMG data on 19 of 22; one subject withdrew from ETP owing to clinical deterioration and two subjects withdrew due to dislike of needles. On primary analysis, 19 of 22 participants (86.4%; 95% CI:0.72-1.00) were treatment successes using last observation carried forward (p=0.018). Secondary efficacy measures supported MG stability. SCIg was safe and well tolerated, and IgG levels were stable. Treatment satisfaction was comparable between ISP and ETP.
DISCUSSION: MG patients on IVIg as part of their routine clinical care remained stable on monthly IVIg dosage, and most maintained similar disease stability on SCIg.
PMID:36779862 | DOI:10.1111/ene.15745
Source: https://pubmed.ncbi.nlm.nih.gov/3677986 ... t6+86293ac