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Results The study included 27 patients, 18 males and 9 females, 29-77 years of age. Baseline data are presented for 14 patients with a Polyneuropathy Disability (PND) Score I and 13 patients with a PND Score II or greater. Characterized by FAP Stage, 24 patients are FAP Stage 1 and 3 patients are FAP Stage 2. Two patients (PND Score II or greater) reported a total of six hospitalizations due to FAP in the past 12 months, each for 3 or more nights in duration. Mean EQ-5D scores were 0.82 (PND Score I) and 0.74 (PND Score II or greater). Patients reported their perceived health status on the EQ-VAS with mean scores of 75 (PND Score I) and 60 (PND Score II or greater). Ten patients (8/10 PND Score II or greater) reported they cannot work because of FAP (mean 61 years of age). Patients also reported inability to perform various ADLs. Most commonly, 77% of patients with PND Score II or greater cannot stand for hours (14% in PND Score I) and 69% cannot run (21% in PND Score I).

Patients with hereditary ATTR amyloidosis experience an increasing burden of illness as the disease progresses