Patients with transthyretin amyloidosis enrolled in THAOS between 2018 and 2021 continue to experience substantial diagnostic delay

Figures & data

Table 1. Demographics of symptomatic patients in THAOS, by enrolment time period and genotype.

	ATTRv amyloidosis				ATTRwt amyloidosis
	2007–2010	2011–2014	2015–2017	2018–2021	2007–2010	2011–2014	2015–2017	2018–2021
	n = 478	n = 1043	n = 646	n = 728	n = 70	n = 230	n = 278	n = 732
Male, n (%)	275 (57.5)	601 (57.6)	414 (64.1)	462 (63.5)	69 (98.6)	215 (93.5)	264 (95.0)	684 (93.4)
Age at symptom onset, n	476	1040	646	728	70	229	278	732
Median (10th, 90th percentile)	39.5 (26.5, 66.5)	44.0 (27.0, 67.6)	53.2 (29.0, 72.0)	57.0 (33.5, 73.5)	74.0 (59.7, 82.2)	71.8 (60.5, 82.5)	72.0 (60.1, 82.5)	74.5 (59.5, 84.4)
Age at enrolment	478	1043	646	728	70	230	278	732
Median (10th, 90th percentile)	47.7 (30.8, 72.7)	51.1 (31.8, 72.9)	59.3 (37.6, 75.8)	64.1 (40.1, 77.8)	76.9 (67.5, 84.5)	76.1 (67.2, 84.3)	76.8 (67.0, 85.4)	79.5 (69.7, 87.5)
Symptom duration at enrolment	476	1040	646	728	70	229	278	732
Median (10th, 90th percentile)	4.3 (0.9, 12.7)	4.0 (0.8, 14.4)	3.9 (0.7, 16.5)	4.1 (0.8, 14.9)	3.1 (0.5, 12.1)	2.1 (0.3, 10.8)	2.5 (0.4, 14.8)	3.4 (0.4, 14.8)
Time from symptom onset to diagnosis	342	978	608	687	35	215	278	717
Median (10th, 90th percentile)	1.6 (0.1, 7.0)	1.8 (0.0, 9.2)	2.2 (0.0, 10.4)	2.4 (0.0, 11.7)	1.2 (0.2, 9.7)	1.2 (0.0, 10.3)	1.5 (0.0, 13.7)	2.3 (0.0, 13.7)

One patient enrolled in the time period 2018–2021 had missing variant data and was not categorised by genotype.

ATTRv amyloidosis: hereditary transthyretin amyloidosis; ATTRwt amyloidosis: wild-type transthyretin amyloidosis; THAOS: Transthyretin Amyloidosis Outcomes Survey.

Data availability statement

Upon request, and subject to review, Pfizer will provide the data that support the findings of this study. Subject to certain criteria, conditions and exceptions, Pfizer may also provide access to the related individual de-identified participant data. See https://www.pfizer.com/science/clinical-trials/trial-data-and-results for more information.