SSM Health Cardinal Glennon Children's Hospital, St. Louis, Missouri.
Children's Hospital and Clinics of Minnesota, Minneapolis, Minnesota.
Pediatr Blood Cancer. 2019 Jul;66(7):e27650. doi: 10.1002/pbc.27650. Epub 2019 Feb 7.
Hydroxyurea (HU) reduces complications and improves quality and duration of life in sickle cell disease. Evidence supports the use of HU starting after nine months of age.
We performed a retrospective study of patients starting HU at less than five years of age between January 1, 2008, and December 31, 2016. We evaluated clinical events, laboratory data, and toxicity between three different age groups: cohort 1 (0-1 year), cohort 2 (1-2 years), and cohort 3 (2-5 years).
Sixty-five patients were included in the analysis. The mean age was 7.2 months (n = 35), 19.5 months (n = 13), and 35.5 months (n = 17) for cohorts 1, 2, and 3, respectively. Cohort 1 had higher hemoglobin (P = 0.0003) and MCV (P = 0.0199) and lower absolute reticulocyte count (P = 0.0304) at 24 months of age compared with cohort 3. The absolute neutrophil count (ANC) was lower compared with both older cohorts (P = 0.0364, 0.0025). The mean baseline hemoglobin F in cohort 1 was 31.5% compared with 19.7% and 16.5% in cohorts 2 and 3, respectively (P = 0.002, P < 0.0001). The mean duration of therapy was 31.3 months, 57.6 months (P = 0.018), and 29.1 months (P = 0.401), respectively. Mean Hb F levels remained higher in cohort 1 (29.9%) compared with cohorts 2 and 3 (20.4%, P = 0.007; 20.6%, P = 0.003). Cohort 1 experienced fewer hospitalizations (P = 0.0025), pain crises (P = 0.0618), and transfusions (P = 0.0426). There was no difference in toxicity between groups.
HU is safe and effective in patients 5 to 12 months of age and generated a more robust response compared with initiation in older patients.
羟基脲 (HU) 可降低镰状细胞病的并发症发生率，改善生活质量和延长寿命。有证据支持在九个月大后开始使用 HU。
我们对 2008 年 1 月 1 日至 2016 年 12 月 31 日期间，在五岁以下开始使用 HU 的患者进行了回顾性研究。我们评估了三个不同年龄组（队列 1：0-1 岁；队列 2：1-2 岁；队列 3：2-5 岁）之间的临床事件、实验室数据和毒性。
共纳入 65 例患者进行分析。队列 1、2 和 3 的平均年龄分别为 7.2 个月（n=35）、19.5 个月（n=13）和 35.5 个月（n=17）。与队列 3 相比，队列 1 在 24 个月时的血红蛋白（P=0.0003）和 MCV（P=0.0199）更高，绝对网织红细胞计数（P=0.0304）更低。与两个年龄较大的队列相比，绝对中性粒细胞计数（ANC）更低（P=0.0364，0.0025）。队列 1 的基线血红蛋白 F 平均值为 31.5%，而队列 2 和 3 分别为 19.7%和 16.5%（P=0.002，P<0.0001）。治疗的平均持续时间分别为 31.3 个月、57.6 个月（P=0.018）和 29.1 个月（P=0.401）。与队列 2 和 3 相比，队列 1 的平均 Hb F 水平仍较高（29.9%，P=0.007；20.6%，P=0.003）。队列 1 住院治疗（P=0.0025）、疼痛危象（P=0.0618）和输血（P=0.0426）次数更少。各组之间的毒性无差异。
HU 在 5 至 12 个月大的患者中是安全且有效的，与开始治疗时年龄较大的患者相比，其产生的反应更强烈。
