Key result
Tafamidis and technetium-scintigraphy approval linked to a ~5-fold increase in ATTR-positive cardiac biopsies.
Why the study?
The impact of the approval of tafamidis and technetium-scintigraphy on the diagnosis of ATTR cardiomyopathy in Japan was not well characterized.
Did the approval of tafamidis and technetium-scintigraphy increase the diagnosis of ATTR cardiomyopathy in Japan?
Observational (n=5,400)
Yes
Did the approval of tafamidis and technetium-scintigraphy increase the diagnosis of ATTR cardiomyopathy in Japan?
Effect estimate: 4.9 times increase
The approval of targeted therapies and specific imaging modalities for ATTR cardiomyopathy significantly increased disease awareness and diagnostic biopsy rates in Japan.
Supports heightened ATTR cardiomyopathy vigilance post-approval; leaves open whether biopsy trends reflect incidence or awareness.
BACKGROUND: In 2019, 2020 and 2022, the Japanese Government approved the use of tafamidis and two technetium-scintigraphies for transthyretin amyloid (ATTR) cardiomyopathy, and announced the patient criteria for tafamidis therapy. In 2018, we had started a nation-wide pathology consultation of amyloidosis. OBJECTIVE: To reveal the impact of approval of tafamidis and technetium-scintigraphy on the diagnosis of ATTR cardiomyopathy. METHODS: antibodies. Proteomic analysis was performed when the typing diagnosis by immunohistochemistry was unavailable. RESULTS: Out of 5400 consultation cases received from April 2018 to July 2022, the type of amyloidosis by immunohistochemistry was determined in 4119 of the 4420 Congo-red positive cases. The incidences of AA, ALκ, ALλ, ATTR, Aβ2M and others were 3.2, 11.3, 28.3, 54.9, 0.6 and 1.8%, respectively. Out of 2208 cardiac biopsy cases received, 1503 cases were ATTR positive. There were 4.0 and 4.9 times more total cases and ATTR-positive cases, respectively, in the last 12 months as compared to the first 12 months. CONCLUSIONS: The approval of tafamidis and technetium-scintigraphy raised the awareness of ATTR cardiomyopathy, leading to an upsurge in ATTR-positive cardiac biopsy cases.
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Naiki et al. (2023) conducted an observational in Amyloidosis (n=5,400). Approval of tafamidis and technetium-scintigraphy vs. Pre-approval period (first 12 months) was evaluated on Number of ATTR-positive cardiac biopsy cases (4.9 times increase). The approval of tafamidis and technetium-scintigraphy was associated with a 4.9-fold increase in ATTR-positive cardiac biopsy cases in the last 12 months compared to the first 12 months.
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