Table 1.
Observational Studies Reporting Outcomes of Different Interventions for Patients with Non-Complicated ATTR CA
| Reference | Intervention (Therapy) | Design | Size (N) | Population | Diagnosis | Outcomes | Adverse Events |
|---|---|---|---|---|---|---|---|
| Aus dem Siepen et al, 201526 | Four capsules of 1200 mg green tea extract | Retrospective | 25 | ATTRwt-CA | Biopsy | No significant improvements | Death, no compliance, transplantation (nine patients) |
| Benson et al, 201727 | ASO; IONIS-TTR℞ | Open-label | 22 | ATTRv and ATTRwt CA + chronic heart failure + Left ventricular (LV) wall thickness ≥ 1.3 cm | Biopsy | Disease stabilization (Significant reduction in LV mass, BNP, and 6MWT and improved NYHA class) | No significant changes in creatinine and platelets |
| Castano et al, 201228 | Diflunisal 250 mg twice daily for one year (with proton pump inhibitor for 1 year or a histamine receptor antagonist) | Open-label | 13 | ATTR CA | Biopsy | Disease stabilization | No change in ejection fraction, LV mass, creatinine levels, BNP, and troponin |
| Dasgupta et al, 202029 | Inotersen | Open-label | 33 | ATTRv and ATTRwt CA + heart failure + LV wall thickness ≥ 1.3 cm + NYHA class I–III | NR | Disease stabilization (Significant reduction in LV mass and thickness, BNP, and 6MWT) | NR |
| Ikram et al, 201830 | Diflunisal | Retrospective | 23 | ATTR CA | Cardiovascular imaging technique, endomyocardial biopsy with tissue typing, pyrophosphate nuclear scintigraphy with confirmatory genetic testing | Safety | No bleeding and no increase in creatinine, GIT side effects in 13% (discontinued treatment) |
| Kristen et al, 201231 | 1.5–2 L Green tea per day for one year | Retrospective | 19 | ATTR CA | Biopsy | Reduced progression of amyloidosis (LV mass reduced in 12.5%) | Death, no compliance, transplantation (five patients) |
| Minamisawa et al, 201932 | Patisiran | Post-hoc analysis of APOLLO | 126 | ATTRv-related polyneuropathy + LV wall thickness ≥ 13 cm | NR | Disease stabilization (Improved global longitudinal strain at 18 months) | NR |
| Obici et al, 201233 | Doxycycline 100 twice daily + TUDCA 250 thrice daily for one year | Open-label Phase II | 20 | ATTRv and ATTRwt CA | NR | Safety | No remarkable adverse outcomes were noted |
| Solomon et al, 201934 | Patisiran | Post-hoc analysis of APOLLO | 126 | ATTRv-related polyneuropathy + LV wall thickness ≥ 13 cm | NR | Reduced all-cause mortality, hospitalizations, NT-proBNP, and LV wall thickness | NR |
| Wixner et al, 201735 | Doxycycline 200 once daily + UDCA 750 once daily for one year | Prospective | 28 | ATTRv and ATTRwt CA | NR | Disease stabilization with no significant improvements | 30% of patients discontinued treatment |