OBJECTIVE: Compared to estimated population prevalence rates, relatively few patients at risk are diagnosed with and treated for transthyretin cardiac amyloidosis (ATTR-CA). Where along the clinical pathway patient drop-off occurs, as well as the association of drop-off with patient sociodemographic characteristics, remains unknown.
METHODS: Using data from a healthcare system-wide cardiovascular imaging repository and specialty pharmacy, we characterized the clinical pathway from diagnosis with pyrophosphate scintigraphy (PYP) to tafamidis prescription, initiation, and adherence. Standardized differences (d values of ≥0.20, indicating at least a small effect size) were used to compare sociodemographics (age, sex, race, Area Deprivation Index) among patients with PYP-identified ATTR-CA by tafamidis prescription status and among patients prescribed tafamidis by initiation status. Tafamidis adherence was measured with the proportion of days covered (PDC).
RESULTS: Of 97 patients with ATTR-CA, 58.8% were prescribed tafamidis, with 80.7% of those initiating therapy. Patients with ATTR-CA prescribed tafamidis were younger than those not prescribed tafamidis (d = -0.30). Utilization of a specialty pharmacy resulted in enrichment of treatment in subgroups traditionally undertreated in cardiovascular medicine, with higher rates of tafamidis initiation among women (100% initiation), patients of Black/African American race (d = 0.40), and those living in more economically disadvantaged areas (d ≥ 0.30). Adherence was high (PDC of >80%) in 88.4% of those initiating tafamidis.
CONCLUSIONS: These findings highlight the tremendous opportunity for more robust ATTR-CA clinical programs, identifying potential patient subgroups that should be targeted to reduce disparities. For patients diagnosed with ATTR-CA, utilization of a specialty pharmacy process appears to ensure equitable provision of tafamidis therapy.
目标:与估计的人口患病率相比,相对较少的有风险的患者被诊断为甲状腺素运载蛋白心脏淀粉样变性(ATTR-CA)并接受治疗.在沿着临床路径发生患者下车的地方,以及下车与患者社会人口统计学特征的关联,仍然未知。
方法:使用来自医疗保健系统范围的心血管成像库和专业药房的数据,我们表征了从焦磷酸盐闪烁显像(PYP)诊断到塔法米米处方的临床路径,initiation,和坚持。标准化差异(d值≥0.20,表明至少有一个小的影响大小)用于比较社会人口统计学(年龄,性别,种族,区域剥夺指数)通过tafamidis处方状态具有PYP识别的ATTR-CA的患者和通过起始状态规定的tafamidis的患者。用覆盖天数的比例(PDC)测量Tafamidis的粘附性。
结果:在97例ATTR-CA患者中,58.8%的人是处方tafamidis,80.7%的人开始治疗。使用ATTR-CA处方tafamidis的患者比未使用tafamidis的患者年轻(d=-0.30)。使用专业药物导致传统上在心血管医学中治疗不足的亚组的治疗丰富,妇女中Tafamidis的启动率较高(100%启动),黑人/非裔美国人种族患者(d=0.40),以及生活在经济困难地区的人(d≥0.30)。88.4%的起始tafamidis的粘附性高(PDC>80%)。
结论:这些发现突出了更强大的ATTR-CA临床计划的巨大机会,确定应针对的潜在患者亚组,以减少差异。对于被诊断为ATTR-CA的患者,使用专业药学流程似乎可以确保公平提供tafamidis治疗。