Heart failure & cardiomyopathies
Effect of Patisiran on Stroke Volume in Hereditary Transthyretin-Mediated Amyloidosis: Insights from Pressure–Volume Analysis of the APOLLO Study
Authors' conclusion
Patisiran may delay progression of LV chamber dysfunction starting at 9 months of therapy.
Study context
- Population
- Hereditary ATTR amyloidosis with polyneuropathy, pre-specified cardiac subpopulation of APOLLO
- Comparison
- Patisiran vs placebo
- Primary endpoint
- Change in LV stroke volume by transthoracic echocardiography
- Follow-up
- 9 and 18 months
Methodological detailspre-specified cardiac subpopulation, phase 3
- Pre-specified cardiac subpopulation
- Phase 3
Primary result
As reported in the abstract
LS mean change in LV SV · 9 months
Patisiran
−0.3
least-squares mean change · SE 1.2 · ml
Placebo
−5.4
least-squares mean change · SE 1.9 · ml
P = 0.021
Further findings
| Outcome | Patisiran | Placebo | P value |
|---|---|---|---|
| LS mean change in LV SV · 18 months | least-squares mean change −1.7 (SE 1.3) ml | least-squares mean change - 8.1 (SE 2.3) ml | P = 0.016 |
Mechanism (non-invasive pressure-volume analysis)
- Decline in left ventricular (LV) stroke volume (SV) driven by diminished LV capacitance in placebo relative to patisiran
Original publication
Effect of Patisiran on Stroke Volume in Hereditary Transthyretin-Mediated Amyloidosis: Insights from Pressure–Volume Analysis of the APOLLO Study. Eur J Heart Fail. Published 6 February 2023. doi:10. 1002/ ejhf. 2783
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