UNASSIGNED: Agalsidase beta-treated adult patients (aged ≥16 years) from the Fabry Registry and adult untreated patients from a natural history cohort were matched 1:1 and X:X (with one occurrence and multiple occurrences of each untreated patient, respectively) by sex, phenotype, age and (for eGFR slope analysis) baseline eGFR. Outcomes included eGFR slope over 5 years and composite clinical event risk (cardiovascular, cerebrovascular or renal event, or death) over 10+ years. As a surrogate indicator of therapeutic response in paediatric patients, the percentage experiencing normalization in plasma globotriaosylceramide (GL-3) from treatment initiation was assessed in patients aged 2 to <16 years.
UNASSIGNED: Overall, eGFR slopes for 1:1-matched untreated and treated adult patients [122 pairs (72.1% male)] were -3.19 and -1.47 mL/min/1.73 m2/year, respectively (reduction in rate of decline = 53.9%, P = .007), and for X:X-matched [122 untreated/950 treated (59.4% male)] were -3.29 and -1.56 mL/min/1.73 m2/year, respectively (reduction in rate of decline = 52.6%, P < .001). Agalsidase beta treatment was associated with lower risk of clinical events, with hazard ratios of 0.41 (P = .003) and 0.67 (P = .008) for 1:1-matched and X:X-matched analyses, respectively. Plasma GL-3 declined markedly in paediatric patients and normalized in most within 6 months of treatment initiation.
UNASSIGNED: Agalsidase beta treatment preserves kidney function and delays progression to severe clinical events among adult patients with FD. Plasma GL-3 levels analysed in paediatric patients showed normalization of elevated pre-treatment levels in most patients.
■法布里登记处的阿胶糖苷酶β治疗的成年患者(年龄≥16岁)和自然史队列中未经治疗的成年患者进行1:1和X:X匹配(每位未经治疗的患者发生一次和多次发生,分别)按性别,表型,年龄和(用于eGFR斜率分析)基线eGFR。结果包括5年eGFR斜率和复合临床事件风险(心血管,脑血管或肾脏事件,或死亡)超过10年。作为儿科患者治疗反应的替代指标,在2岁至<16岁的患者中,评估了从治疗开始开始时血浆中的globotriaosceramide(GL-3)经历正常化的百分比.
■总的来说,1:1匹配的未经治疗和治疗的成年患者的eGFR斜率[122对(72.1%男性)]为-3.19和-1.47mL/min/1.73m2/年,分别(下降率=53.9%,P=.007),对于X:X匹配[122未处理/950处理(59.4%男性)]为-3.29和-1.56mL/min/1.73m2/年,(降幅分别为52.6%,P<.001)。半乳糖苷酶β治疗与较低的临床事件风险相关,1:1匹配和X:X匹配分析的风险比为0.41(P=0.003)和0.67(P=0.008),分别。儿科患者的血浆GL-3显着下降,大多数在治疗开始后6个月内恢复正常。
■阿胶糖苷酶β治疗可保护成年FD患者的肾功能并延迟严重临床事件的进展。在儿科患者中分析的血浆GL-3水平显示大多数患者中升高的治疗前水平正常化。