羟基脲对死亡率和发病率的影响在成人镰状细胞性贫血:风险和收益9年的治疗。

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斯坦伯格MH,巴顿F,卡斯特罗O, Pegelow CH,半刚石SK, Kutlar, Orringer E,贝尔维尤R, Olivieri N, Eckman J, Varma M,拉米雷斯G,阿德勒B,史密斯W,卡洛斯·T Ataga K, DeCastro L, C毕格罗,Saunthararajah Y,电动高架索道M, Vichinsky E,碎屑,Shurin年代,桥梁K, Waclawiw M, D,债券Terrin M

羟基脲对死亡率和发病率的影响在成人镰状细胞性贫血:风险和收益9年的治疗。

《美国医学协会杂志》上。2003年4月2;289 (13):1645 - 51。doi: 10.1001 / jama.289.13.1645。

PubMed ID
12672732 (在PubMed
]
文摘

背景:羟基脲增加胎儿血红蛋白(住宅)和水平减少发病率从疾患患者并发症镰状细胞性贫血(SCA)。高住宅水平降低发病率和死亡率。目的:确定羟基脲减弱患者死亡率SCA。设计:长期观察随访研究SCA患者死亡率的最初参与随机、双盲、安慰剂对照的多中心研究羟基脲在镰状细胞性贫血要看更多有关憩苑(),在1992 - 1995年进行的,以确定羟基脲降低血管阻塞性事件。要看更多有关憩苑的病人的随访中,1996 - 2001年进行的,病人可以继续,停止或启动羟基脲。数据收集的试验和后续期间。设置:住院病人和门诊病人在21镰状细胞转诊中心在美国和加拿大。病人:二百九十九只成年患者频繁参加后续的痛苦的事件。后续数据通过2001年5月完成了233名病人。干预:要看更多有关憩苑的,患者被随机分配接受羟基脲(n = 152)或安慰剂(n = 147)。 MAIN OUTCOME MEASURE: Mortality, HbF levels, painful episodes, acute chest syndrome, and blood cell counts. The randomized trial was not designed to detect specified differences in mortality. RESULTS: Seventy-five of the original 299 patients died, 28% from pulmonary disease. Patients with reticulocyte counts less than 250 000/mm3 and hemoglobin levels lower than 9 g/dL had increased mortality (P =.002). Cumulative mortality at 9 years was 28% when HbF levels were lower than 0.5 g/dL after the trial was completed compared with 15% when HbF levels were 0.5 g/dL or higher (P =.03 ). Individuals who had acute chest syndrome during the trial had 32% mortality compared with 18% of individuals without acute chest syndrome (P =.02). Patients with 3 or more painful episodes per year during the trial had 27% mortality compared with 17% of patients with less frequent episodes (P =.06). Taking hydroxyurea was associated with a 40% reduction in mortality (P =.04) in this observational follow-up with self-selected treatment. There were 3 cases of cancer, 1 fatal. CONCLUSIONS: Adult patients taking hydroxyurea for frequent painful sickle cell episodes appear to have reduced mortality after 9 of years follow-up. Survival was related to HbF levels and frequency of vaso-occlusive events. Whether indications for hydroxyurea treatment should be expanded is unknown.

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