AI Article Synopsis

  • AJHP is publishing peer-reviewed manuscripts promptly online before final formatting, indicating that these versions are not the final published articles.
  • The study investigates the diagnosis and treatment pathway of transthyretin cardiac amyloidosis (ATTR-CA), highlighting a significant gap between estimated prevalence and actual treatment rates among at-risk patients.
  • Results showed that 58.8% of diagnosed patients were prescribed tafamidis, with 80.7% of those starting treatment, while factors like younger age, gender, and race influenced prescription and treatment initiation rates.

Article Abstract

Purpose: 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.

Conclusion: 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.

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Source
http://dx.doi.org/10.1093/ajhp/zxae183DOI Listing

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Article Synopsis
  • AJHP is publishing peer-reviewed manuscripts promptly online before final formatting, indicating that these versions are not the final published articles.
  • The study investigates the diagnosis and treatment pathway of transthyretin cardiac amyloidosis (ATTR-CA), highlighting a significant gap between estimated prevalence and actual treatment rates among at-risk patients.
  • Results showed that 58.8% of diagnosed patients were prescribed tafamidis, with 80.7% of those starting treatment, while factors like younger age, gender, and race influenced prescription and treatment initiation rates.
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  • Tafamidis was approved for treating hereditary and wild-type transthyretin amyloid cardiomyopathy (ATTRwt-CM) in May 2019, based on the ATTR-ACT clinical trial findings.
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Advancement in the diagnosis and treatment of transthyretin amyloid cardiomyopathy has made great strides in recent years. Novel therapeutics for transthyretin amyloidosis such as tafamidis, patisiran, and inotersen have shown significant benefits in a not-so-rare disease but come with high listing price tags ranging from a quarter to more than a half million dollars per year. These costs create significant financial barriers for the majority of patients, especially those with existing Medicare insurance plans.

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