Feriozzi Sandro, Schwarting Andreas, Sunder-Plassmann Gere, West Michael, Cybulla Markus
Nephrology and Dialysis, Belcolle Hospital, Strada Sammartinese snc, Viterbo, Italy.
Am J Nephrol. 2009;29(5):353-61. doi: 10.1159/000168482. Epub 2008 Oct 31.
The aim of this study was to determine the effects of enzyme replacement therapy with agalsidase alpha on renal function in patients with Fabry nephropathy. Serum creatinine data were collected from 165 adult patients during 3 years of treatment. Serum creatinine increased in all men whereas it was stable in women, except in stage II renal disease (Kidney Disease Outcomes Quality Initiative). The estimated glomerular filtration rate (eGFR) declined in males with stage I and II (from 115.0 +/- 22.2 to 98.3 +/- 27.3 and from 76.5 +/- 8.1 to 66.3 +/-21.6 ml/min/1.73 m(2), respectively; both p < 0.01), whereas eGFR was stable in stage III. In females, eGFR was stable in stages I and III, and decreased in stage II (from 72.5 +/- 8.3 to 67.3 +/- 13.6 ml/min/1.73 m(2); p = 0.01). The 24-hour proteinuria was <1 g in all patients, and most patients (96%) were treated with angiotensin receptor blockers (ARBs) or angiotensin-converting enzyme (ACE) inhibitors. Agalsidase alpha in combination with ACE inhibitors/ARB may be effective in slowing the deterioration in renal function in Fabry nephropathy.
本研究的目的是确定α - 半乳糖苷酶替代疗法对法布里肾病患者肾功能的影响。在3年的治疗期间，收集了165例成年患者的血清肌酐数据。所有男性患者的血清肌酐均升高，而女性患者血清肌酐稳定，但处于II期肾病的女性患者除外（肾脏病预后质量倡议组织标准）。I期和II期男性患者的估算肾小球滤过率（eGFR）下降（分别从115.0±22.2降至98.3±27.3以及从76.5±8.1降至66.3±21.6 ml/min/1.73 m²；p均<0.01），而III期男性患者的eGFR稳定。在女性患者中，I期和III期的eGFR稳定，II期下降（从72.5±8.3降至67.3±13.6 ml/min/1.73 m²；p = 0.01）。所有患者的24小时蛋白尿均<1 g，大多数患者（96%）接受了血管紧张素受体阻滞剂（ARB）或血管紧张素转换酶（ACE）抑制剂治疗。α - 半乳糖苷酶与ACE抑制剂/ARB联合使用可能有效减缓法布里肾病患者肾功能的恶化。
