Hydroxyurea at escalated dose versus fixed low-dose hydroxyurea in adults with sickle cell disease.

Hydroxyurea at escalated dose versus fixed low-dose hydroxyurea in adults with sickle cell disease.
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在患有镰状细胞病的成人中,增加剂量的羟基脲与固定低剂量的羟基脲。

DOI:
10.1111/ejh.14138
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发表时间:
2024
影响因子:
3.1
通讯作者:
Ataga,KennethI
Ataga,KennethI
中科院分区:
医学3区
文献类型:
--
作者:
Ogu,UgochiO;Mukhopadhyay,Ayesha;Patel,Kruti;Nelson,MarquitaN;Strahan,KayLeeS;Wu,Lin;Smeltzer,MatthewP;Ataga,KennethI

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羟基脲可降低镰状细胞病(SCD)血管闭塞并发症的发生率,增加血红蛋白,并降低死亡率。尽管目前的指南建议升级到最大耐受剂量(MTD),但固定低剂量羟基尿素的使用在低资源国家很常见。我们进行了一项系统的综述和荟萃分析,以评估递增剂量羟基脲与固定小剂量羟基脲在成人SCD中的疗效。9项研究包括定量合成,4项评估固定低剂量,5项评估递增剂量的羟基尿素。固定低剂量和递增剂量研究中羟基尿素的平均日剂量分别为~10和22 mg/kg。在升级和固定的低剂量研究中,血管闭塞危象发生率的估计没有差异(p= .73)。在固定低剂量研究中,从基线到随访的平均血红蛋白差异大于递增剂量研究(1.07 g/dL vs.0.54 g/dL,p= .01)。胎儿血红蛋白的平均估计值没有差异。尽管有限的合格研究和几个结果的研究之间的效果有很大的异质性,但对于成人SCD患者最合适的羟基尿素剂量方案,临床上似乎是平衡的。成人SCD患者MTD和固定小剂量羟基脲的对照研究是必需的。
Hydroxyurea reduces the frequency of vaso‐occlusive complications, increases hemoglobin, and decreases mortality in sickle cell disease (SCD). Although current guidelines recommend escalation to maximum tolerated dose (MTD), the use of fixed low‐dose hydroxyurea is common in low‐resource countries. We conducted a systematic review and meta‐analysis to evaluate the efficacy of escalated doses versus fixed low‐dose of hydroxyurea in adults with SCD. Nine studies were included in the quantitative synthesis, four evaluating fixed low‐dose and five evaluating escalated doses of hydroxyurea. Average daily doses of hydroxyurea in the fixed low‐dose and escalated dose studies were ~10 and 22 mg/kg, respectively. There was no difference in the estimate of vaso‐occlusive crisis rate between escalated and fixed low‐dose studies (p= .73). The mean difference in hemoglobin from baseline to follow‐up was greater for fixed low‐dose than escalated dose studies (1.07 g/dL vs. 0.54 g/dL,p= .01). No difference was seen in the mean estimate of fetal hemoglobin. Despite limited eligible studies and substantial heterogeneity of effect between the studies for several outcomes, there appears to be clinical equipoise regarding the most appropriate hydroxyurea dosing regimen in adults with SCD. Controlled studies of hydroxyurea at MTD versus fixed low‐dose in adults with SCD are required.
新生儿镰状细胞病筛查:对死亡率的影响。
DOI: --
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