PDF(Pubmed)
Abstract：
Youths with sickle cell anemia (SCA) are at risk of pain crises, stroke, and early death. Complications can be reduced by the oral disease-modifying medication hydroxyurea, and in 2014, the National Heart, Lung, and Blood Institute published revised guidelines that hydroxyurea should be offered to youths aged 9 months and older with SCA regardless of disease severity.
To describe changes in hydroxyurea use among youths with SCA before and after release of the National Heart, Lung, and Blood Institute guidelines.
This cross-sectional study was conducted using administrative data from 2010 to 2018 from Michigan and New York State (NYS) Medicaid programs. The study population included youths aged 1 to 17 years with SCA enrolled in the Michigan or NYS Medicaid programs for at least 1 year (Michigan: 2010-2018; NYS: 2012-2018). Youths with SCA were identified using validated claims-based definitions. Data were analyzed from June to October 2020.
The main outcome was hydroxyurea use characterized as mean annual counts of days\' supply of filled hydroxyurea prescriptions. Rates of hydroxyurea use over time were assessed using regression models (Michigan: zero-inflated negative binomial; NYS: negative binomial). Models included indicators for periods before and after guideline release.
A total of 4302 youths with SCA (2236 males [52.0%]; 2676 born 2005-2017 [62.2%]; 150 Hispanic [3.5%], 2929 non-Hispanic Black [68.0%], and 389 non-Hispanic White [9.0%]) contributed 12 565 person-years. The mean (SD) annual days\' supply of hydroxyurea was 47.2 (93.6) days per youth in Michigan and 97.4 (137.0) days per youth in NYS. In Michigan, there was an increase in the odds of having nonzero days\' supply after the guidelines were released (odds ratio, 1.52; 95% CI, 1.07-2.14). In NYS, no change was seen in the mean days\' supply of filled hydroxyurea.
These findings suggest that hydroxyurea was substantially underused among youths with SCA, despite establishment as the primary disease-modifying therapy for SCA, and that there was incomplete clinician or patient uptake of newly released guidelines. Results suggest that expanding use of hydroxyurea may require a multifaceted approach that includes addressing multiple system- and patient-level barriers.
To describe changes in hydroxyurea use among youths with SCA before and after release of the National Heart, Lung, and Blood Institute guidelines.
This cross-sectional study was conducted using administrative data from 2010 to 2018 from Michigan and New York State (NYS) Medicaid programs. The study population included youths aged 1 to 17 years with SCA enrolled in the Michigan or NYS Medicaid programs for at least 1 year (Michigan: 2010-2018; NYS: 2012-2018). Youths with SCA were identified using validated claims-based definitions. Data were analyzed from June to October 2020.
The main outcome was hydroxyurea use characterized as mean annual counts of days\' supply of filled hydroxyurea prescriptions. Rates of hydroxyurea use over time were assessed using regression models (Michigan: zero-inflated negative binomial; NYS: negative binomial). Models included indicators for periods before and after guideline release.
A total of 4302 youths with SCA (2236 males [52.0%]; 2676 born 2005-2017 [62.2%]; 150 Hispanic [3.5%], 2929 non-Hispanic Black [68.0%], and 389 non-Hispanic White [9.0%]) contributed 12 565 person-years. The mean (SD) annual days\' supply of hydroxyurea was 47.2 (93.6) days per youth in Michigan and 97.4 (137.0) days per youth in NYS. In Michigan, there was an increase in the odds of having nonzero days\' supply after the guidelines were released (odds ratio, 1.52; 95% CI, 1.07-2.14). In NYS, no change was seen in the mean days\' supply of filled hydroxyurea.
These findings suggest that hydroxyurea was substantially underused among youths with SCA, despite establishment as the primary disease-modifying therapy for SCA, and that there was incomplete clinician or patient uptake of newly released guidelines. Results suggest that expanding use of hydroxyurea may require a multifaceted approach that includes addressing multiple system- and patient-level barriers.
摘要：
患有镰状细胞性贫血（SCA）的年轻人有疼痛危机的风险，中风,和早逝。改善口腔疾病的药物羟基脲可以减少并发症，2014年，国家心脏，肺，血液研究所发布了修订后的指南，即无论疾病严重程度如何，均应向9个月及以上的SCA青少年提供羟基脲。
■为了描述在国家心脏发布之前和之后，患有SCA的年轻人使用羟基脲的变化，肺，和血液研究所指南。
■这项横断面研究是使用2010年至2018年密歇根州和纽约州（NYS）医疗补助计划的行政数据进行的。研究人群包括1至17岁的青少年，SCA在密歇根州或NYS医疗补助计划中注册至少1年（密歇根州：2010-2018;NYS：2012-2018）。使用经过验证的基于声明的定义来识别患有SCA的年轻人。数据从2020年6月至10月进行了分析。
■主要结果是羟基脲的使用，其特征为填充羟基脲处方的平均年供应天数。使用回归模型（密歇根州：零膨胀负二项；NYS：负二项）评估了羟基脲随时间的使用率。模型包括指南发布前后的指标。
■共有4302名患有SCA的年轻人（2236名男性[52.0％]；2676名2005-2017年出生[62.2％]；150名西班牙裔[3.5％]，2929非西班牙裔黑人[68.0%]，389名非西班牙裔白人[9.0%]）贡献了12565人年。在密歇根州，羟基脲的平均（SD）年供应天数为每位青年47.2（93.6）天，在NYS为每位青年97.4（137.0）天。在密歇根,指南发布后，非零日供应的几率增加了(赔率比，1.52；95%CI，1.07-2.14）。在纽约，填充羟基脲的平均日供应量没有变化。
■这些发现表明，在患有SCA的年轻人中，羟基脲的使用严重不足，尽管已确立为SCA的主要疾病修饰疗法，并且临床医生或患者对新发布的指南的吸收不完全。结果表明，扩大羟基脲的使用可能需要多方面的方法，包括解决多个系统和患者水平的障碍。
■为了描述在国家心脏发布之前和之后，患有SCA的年轻人使用羟基脲的变化，肺，和血液研究所指南。
■这项横断面研究是使用2010年至2018年密歇根州和纽约州（NYS）医疗补助计划的行政数据进行的。研究人群包括1至17岁的青少年，SCA在密歇根州或NYS医疗补助计划中注册至少1年（密歇根州：2010-2018;NYS：2012-2018）。使用经过验证的基于声明的定义来识别患有SCA的年轻人。数据从2020年6月至10月进行了分析。
■主要结果是羟基脲的使用，其特征为填充羟基脲处方的平均年供应天数。使用回归模型（密歇根州：零膨胀负二项；NYS：负二项）评估了羟基脲随时间的使用率。模型包括指南发布前后的指标。
■共有4302名患有SCA的年轻人（2236名男性[52.0％]；2676名2005-2017年出生[62.2％]；150名西班牙裔[3.5％]，2929非西班牙裔黑人[68.0%]，389名非西班牙裔白人[9.0%]）贡献了12565人年。在密歇根州，羟基脲的平均（SD）年供应天数为每位青年47.2（93.6）天，在NYS为每位青年97.4（137.0）天。在密歇根,指南发布后，非零日供应的几率增加了(赔率比，1.52；95%CI，1.07-2.14）。在纽约，填充羟基脲的平均日供应量没有变化。
■这些发现表明，在患有SCA的年轻人中，羟基脲的使用严重不足，尽管已确立为SCA的主要疾病修饰疗法，并且临床医生或患者对新发布的指南的吸收不完全。结果表明，扩大羟基脲的使用可能需要多方面的方法，包括解决多个系统和患者水平的障碍。
