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首页> 外文期刊>Orphanet journal of rare diseases >Biological parameters predictive of percent dense red blood cell decrease under hydroxyurea
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Biological parameters predictive of percent dense red blood cell decrease under hydroxyurea

机译：预测羟基脲下密集红细胞减少百分比的生物学参数

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Background Dense red blood cells (DRBCs) are associated with chronic clinical manifestations of sickle-cell–disease (SCD). Hydroxyurea (HU) decreases the percent (%) DRBCs, thereby improving its therapeutic benefits, especially the prevention of SCD clinical complications, but parameters influencing %DRBCs remain unknown. The purpose of this study was to determine predictive biological parameters of %DRBC decline under HU. Methods Factors affecting the %DRBC decrease in SCD patients HU-treated for ≥6 months were analyzed. Biological parameters and the %DRBCs were determined before starting HU and after ≥6 months of HU intake. Bivariate analyses evaluated the impact of each biological parameter variation on %DRBC changes under treatment. Multivariate analyses assessed the correlations between the decreased %DRBCs and biological parameters. Results The %DRBCs declined by 40.95% after ≥6 months on HU. That decrease was associated with less hemolysis, however in several analyses on this group of patients we did not find a statistically significant correlation between decrease in %DRBCs and increase in HbF. Initial %DRBC values were the most relevant parameter to predict %DRBC decline. Conclusion Our results strengthen the known HU efficacy in SCD management statistically independently of the classical HbF biological response. Decreasing %DRBCs is essential to limiting chronic SCD symptoms related to DRBCs and predictive factors might help prevent those manifestations. The results of this study provide new perspectives on indication for HU use, i.e., to prevent SCD-induced organ damage.
机译：背景密集的红细胞（DRBC）与镰状细胞疾病（SCD）的慢性临床表现有关。羟基尿素（HU）降低了DRBC的百分比（％），从而提高了其治疗效果，尤其是预防SCD临床并发症，但是影响％DRBC的参数仍然未知。这项研究的目的是确定HU下％DRBC下降的预测生物学参数。方法分析经HU治疗≥6个月的SCD患者％DRBC降低的影响因素。在开始HU之前和HU摄入≥6个月后，确定生物学参数和％DRBC。双变量分析评估了每种生物学参数变化对治疗中％DRBC变化的影响。多变量分析评估了降低的％DRBC与生物学参数之间的相关性。结果HU≥6个月后，％DRBC下降了40.95％。减少与溶血减少有关，但是在这组患者的数项分析中，我们没有发现％DRBC减少与HbF增加之间存在统计学上的显着相关性。初始％DRBC值是预测％DRBC下降的最相关参数。结论我们的结果在统计学上独立于经典的HbF生物反应，在统计学上增强了HU在SCD管理中的功效。减少％DRBC对限制与DRBC相关的慢性SCD症状至关重要，预测因素可能有助于预防这些症状。这项研究的结果为使用HU的适应症即预防SCD引起的器官损害提供了新的见解。

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