Villegas Ana, Núñez Ramiro, Gaya Anna, Cuevas-Ruiz María Victoria, Bosch José Miguel, Carral Anna, Arrizabalaga Beatriz, Gómez-Roncero María Isabel, Mora Asunción, Bravo Pilar, Lavilla Esperanza, Monteserín Carmen, Hernández Belén, Martínez-Barranco Pilar, Jarque Isidro, Urquía María Anunciación, García-Donas Gloria, Brunet Salut, González Fernando Ataulfo, Urbano Álvaro
Hematology Service, Hospital Clínico San Carlos, Plaza de Cristo Rey s/n, 28040, Madrid, Spain.
Hematology Service, Universidad Complutense Madrid, Madrid, Spain.
Ann Hematol. 2017 Oct;96(10):1727-1733. doi: 10.1007/s00277-017-3059-x. Epub 2017 Jul 26.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening blood disease. With the advent of eculizumab treatment, renal function has substantially improved, although no data from real-world clinical practice are available. An observational, retrospective, multicenter study was conducted in Spain on clinical data obtained from outpatient visits of patients with PNH (Spanish PNH Registry) who had experienced acute (ARF) or chronic (CRF) renal failure. Of the 128 patients registered (April 2014), 60 were diagnosed with classic PNH. Twenty-seven (45.0%) patients with a mean age of 48.5 (±16.2) years had renal failure, ARF or CRF, and were included in this study. Near half of the patients (n = 13; 48.1%) presented with ARF alone, 33.3% (n = 9) had CRF with episodes of ARF, while 18.5% (n = 5) were diagnosed with CRF alone. For patients with diagnosis of PNH and renal failure (n = 27), the median time to the first ARF episode was 6.5 (CI 95%; 2.2, 14.9) years, whereas the median to the diagnosis of CRF was 14.5 (CI 95%; 3.8, 19.2) years after the diagnosis of PNH. Patients with ARF (n = 22) were treated with eculizumab and did not experience new episodes of ARF, except for one patient with sepsis. Of the patients with CRF, two received treatment without experiencing further episodes of ARF. Sixteen patients who completed treatment (11 with ARF and 5 with ARF + CRF) recovered from the episode of ARF or from CRF. Of the remaining patients treated with eculizumab, one patient improved from stages III to II, three patients stabilized without showing disease progression, and one patient progressed from stages III to IV. Treatment with eculizumab in PNH patients has beneficial effects on renal function, preventing ARF and progression to CRF.
阵发性睡眠性血红蛋白尿（PNH）是一种罕见的、危及生命的血液疾病。随着依库珠单抗治疗的出现，肾功能有了显著改善，尽管尚无来自真实世界临床实践的数据。在西班牙针对从阵发性睡眠性血红蛋白尿患者（西班牙PNH登记处）门诊就诊获取的临床数据开展了一项观察性、回顾性、多中心研究，这些患者曾经历急性肾衰竭（ARF）或慢性肾衰竭（CRF）。在登记的128例患者（2014年4月）中，60例被诊断为典型PNH。27例（45.0%）平均年龄为48.5（±16.2）岁的患者患有肾衰竭，包括急性肾衰竭或慢性肾衰竭，并被纳入本研究。近半数患者（n = 13；48.1%）仅出现急性肾衰竭，33.3%（n = 9）患有慢性肾衰竭且伴有急性肾衰竭发作，而18.5%（n = 5）仅被诊断为慢性肾衰竭。对于诊断为PNH且患有肾衰竭的患者（n = 27），首次急性肾衰竭发作的中位时间为6.5（95%置信区间；2.2，14.9）年，而慢性肾衰竭诊断的中位时间为阵发性睡眠性血红蛋白尿诊断后14.5（95%置信区间；3.8，19.2）年。急性肾衰竭患者（n = 22）接受了依库珠单抗治疗，除1例败血症患者外，未出现新的急性肾衰竭发作。在慢性肾衰竭患者中，2例接受治疗后未出现急性肾衰竭的进一步发作。16例完成治疗的患者（11例急性肾衰竭患者和5例急性肾衰竭合并慢性肾衰竭患者）从急性肾衰竭发作或慢性肾衰竭中康复。在其余接受依库珠单抗治疗的患者中，1例患者从III期改善至II期，3例患者病情稳定未出现疾病进展，1例患者从III期进展至IV期。阵发性睡眠性血红蛋白尿患者使用依库珠单抗治疗对肾功能有有益影响，可预防急性肾衰竭及进展为慢性肾衰竭。
