Table 1.

Endpoints of autonomic significance in tafamidis studies and related cohorts

Study	Autonomic endpoints	Outcome
FX-005 clinical trial	Norfolk QOL-DN score	NS in the ITT population
	mBMI	Positive effect for EE population
	Small-fiber function	Significantly deteriorated in placebo group Significantly preserved in tafamidis group
FX-006 clinical trial	Norfol QOL, mBMI, small-fiber function	Positive rate of change
FX1A-201 clinical trial	Norfolk QOL-DN	Negligible changes
	Nutritional status	Maintained over 12 months
FX1A-303 clinical trial	Norfolk-QOL-DN	The change in TQOL compared to no treatment at all was much lower in both groups of patients
	mBMI	Positive effect for both groups (tafamidis–tafamidis and placebo–tafamidis)
Post-hoc analysis [8]	Combination with NIS-LL with small-fiber unction summated 3	Significantly preserved
Post-hoc analysis [15]	mBMI	Sustained delay in deterioration
Real-world experience [10]	CADT	Deteriorated in 21%
	Development of OH	2 (%) patients
Real-world experience [5]	mBMI	Did not change at 36 months
	Kumamoto Scale	Stable in 33% and improved in 4%
Real-world experience [13]	Neurophysiological score of small-fiber (SFNS)	Deteriorated significantly at 36 months
	BMI	Significant increase in BMI at 30–36 months