Table 1.
Demographic and Clinical Characteristics of the Patients at Baseline.*
| Characteristic | Patisiran (N = 181) | Placebo (N = 178) |
|---|---|---|
| Median age at screening (range) — yr | 76 (47–85) | 76 (41–85) |
| Male sex — no. (%) | 161 (89) | 160 (90) |
| Race — no. (%)† | ||
| White | 138/180 (77) | 140/174 (80) |
| Asian | 23/180 (13) | 15/174 (9) |
| Black | 16/180 (9) | 15/174 (9) |
| Other | 3/180 (2) | 4/174 (2) |
| Wild-type ATTR amyloidosis — no. (%) | 144 (80) | 144 (81) |
| Median time since diagnosis of ATTR amyloidosis (range) — yr | 0.8 (0.0–6.0) | 0.4 (0.0–10.0) |
| Treatment with tafamidis — no. (%) | ||
| At baseline | 46 (25) | 45 (25) |
| Started during 12-month double-blind period | 5 (3) | 3 (2) |
| ATTR amyloidosis stage — no. (%)‡ | ||
| Stage 1 | 124 (69) | 120 (67) |
| Stage 2 | 46 (25) | 45 (25) |
| Stage 3 | 11 (6) | 13 (7) |
| Polyneuropathy disability score — no. (%) | ||
| 0: no impairment | 96 (53) | 109 (61) |
| I: preserved walking, sensory disturbances | 63 (35) | 55 (31) |
| II: impaired walking without need for a stick or crutches | 22 (12) | 14 (8) |
| NYHA class — no. (%) | ||
| Class I | 10 (6) | 15 (8) |
| Class II | 156 (86) | 150 (84) |
| Class III | 15 (8) | 13 (7) |
| Median 6-minute walk distance (IQR) — m | 358.0 (295.0–420.0) | 367.7 (300.0–444.3) |
| Score on the KCCQ-OS§ | 69.8+21.2 | 70.3+20.7 |
| Laboratory values | ||
| Median NT-proBNP level (IQR) — pg/ml | 2008.0 (1135.0–2921.0) | 1813.0 (952.0–3079.0) |
| Median high-sensitivity troponin I level (IQR) — pg/ml¶ | 64.0 (38.6–92.0) | 60.2 (38.2–103.1) |
| Median mBMI (IQR)‖ | 1147.0 (988.4–1273.8) | 1134.0 (1018.7–1259.1) |
| Median eGFR (IQR) — ml/min/1.73 m2 | 71 (58–83) | 67 (51–84) |
| Median creatinine (IQR) — mg/dl | 1.0 (0.9–1.2) | 1.0 (0.8–1.4) |
| Coexisting conditions — no. (%) | ||
| Diabetes mellitus | 30 (17) | 25 (14) |
| Hypertension | 84 (46) | 101 (57) |
| Concomitant medication — no. (%) | ||
| Diuretic | 168 (93) | 164 (92) |
| Mineralocorticoid receptor antagonist | 92 (51) | 74 (42) |
| Beta-blocker | 73 (40) | 77 (43) |
| ACEI, ARB, or ARNI | 82 (45) | 71 (40) |
| SGLT2 inhibitor | 8 (4) | 7 (4) |
Plus-minus values are means ±SD. Percentages may not total 100 because of rounding. ACEI denotes angiotensinconverting-enzyme inhibitor, ARB angiotensin-receptor blocker, ARNI angiotensin receptor-neprilysin inhibitor, ATTR transthyretin-mediated, eGFR estimated glomerular filtration rate, IQR interquartile range, NT-proBNP N-terminal pro- B-type natriuretic peptide, NYHA New York Heart Association, and SGLT2 sodium-glucose cotransporter-2.
Race was reported by the patients.
Risk stratification was based on the levels of the serum biomarkers NT-proBNP and an eGFR. Stage 1 (lower risk) was defined by an NT-proBNP level of 3000 pg per milliliter or less and eGFR of at least 45 ml per minute per 1.73 m2. Stage 2 (intermediate risk) included all patients who did not meet the criteria for stages 1 or 3. Stage 3 (higher risk) was defined by an NT-proBNP greater than 3000 pg per milliliter and an eGFR of less than 45 ml per minute per 1.73 m2.
Kansas City Cardiomyopathy Questionnaire-Overall Summary (KCCQ-OS) scores range from 0 to 100, with a score of 0 to 24 indicating very poor to poor quality of life, 25 to 49 poor to fair, 50 to 74 fair to good, and 75 to 100 good to excellent.
Troponin I levels were assessed at baseline in a total of 174 patients in the patisiran group and in 172 patients in the placebo group.
The modified body-mass index (mBMI) was calculated as the conventional BMI (weight in kilograms divided by the square of the height in meters) multiplied by the serum albumin level in grams per liter.