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Background:Paroxysmal Nocturnal Hemoglobinuria (PNH) is an ultra-rare (0.5-1.5 cases per million), acquired, life-threatening disease characterized by complement-mediated hemolysis and thrombosis (Parker et. al. NORD 2023). PNH-related symptoms are multifactorial and may negatively impact cognitive function, including a difficulty in thinking clearly or “brain fog” (Daly et. al. JPRO 2021). Pegcetacoplan (PEG), a C3 inhibitor therapy, has been assessed in its efficacy for treating PNH and improving hemoglobin (Hb) levels in clinical trials; however, there is limited evidence on the effect of PEG treatment on cognitive function in individuals living with PNH in a real-world setting. Notably, the Patient-Reported Outcomes Measurement System (PROMIS) is a validated system of questionnaires used to reliably assess patient reported outcomes, including cognitive function. The minimal important change (MIC), the smallest change in score that patients perceive as important, is between a 2-6 improvement in T-score for PROMIS measures (Terwee et. al. Qual. Life Res. 2021).

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Gastrointestinal (GI) adverse events (AEs) associated with DMF can result in premature treatment discontinuation.

IntroductionIn real world comparative effectiveness studies of relapsing-remitting multiple sclerosis (RRMS) patients, treatment with delayed-release dimethyl fumarate (DMF) compared with fingolimod (FTY) for ≤2 years was associated with no statistically significant differences in relapse outcomes. We assessed the real-world effectiveness of DMF compared with FTY in RRMS patients at 12 months.MethodsEFFECT (NCT02776072) was an observational, international, retrospective, single time point, medical record review study undertaken to evaluate the effectiveness of DMF and other disease-modifying therapies (DMTs). Patient eligibility criteria included age ≥18 years, diagnosis of RRMS, treatment naive or 1 prior DMT (interferon or glatiramer acetate), DMT treatment initiation after ,December 2010and≥12 months of medical record data following DMT initiation. Endpoints included Kaplan-Meier estimated proportion of patients relapsed at 12 months and annualised relapse rate (ARR). Substantive baseline covariates were used in estimating propensity score. The data were divided into 4 strata using propensity score. After assessing for balance in baseline covariates between treatment groups, Kaplan-Meier estimates and estimate of treatment effects were pooled across strata of propensity score.ResultsOf the 826 DMF and 785 FTY patients enrolled at sites in 17 countries, 816 and 781 patients respectively, were included in the full analysis set. Treatment groups were balanced after propensity score stratification. At 12 months, 86% of DMF-treated patients and 94% of FTY-treated patients remained on therapy. In the trimmed full analysis set, the estimated proportion of DMF patients that relapsed at 12 months was 12% compared with 13% for FTY patients; hazard ratio (95% CI) 1.07 (0.78, 1.46; p=0.6926). At 12 months after treatment initiation, the adjusted ARR ratio (95% CI) was 1.09 (0.80, 1.49; p=0.5754) for patients treated with DMF compared with FTY.ConclusionOver 12 months, treatment with DMF versus FTY was associated with no statistically significant difference on relapse outcomes.