Longer disease progression milestone-free time in people with amyotrophic lateral sclerosis treated versus not treated with intravenous edaravone: results from an administrative claims analysis
Aim: To estimate time-to-progression milestones in people with amyotrophic lateral sclerosis (PALS) treated versus not treated with intravenous (IV) edaravone (Radicava R IV, Mitsubishi Tanabe Pharma America [MTPA], hereafter ”IV edaravone”) in a real-world setting. Background: IV edaravone is US...
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| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Becaris Publishing Limited
2025-01-01
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| Series: | Journal of Comparative Effectiveness Research |
| Subjects: | |
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| Summary: | Aim: To estimate time-to-progression milestones in people with amyotrophic lateral sclerosis (PALS)
treated versus not treated with intravenous (IV) edaravone (Radicava R IV, Mitsubishi Tanabe Pharma
America [MTPA], hereafter ”IV edaravone”) in a real-world setting. Background: IV edaravone is US FDA
approved for the treatment of ALS and was shown in clinical trials to slow the rate of physical functional
decline. Patients & methods: This retrospective observational analysis included PALS continuously enrolled
in Optum’s Clinformatics Data Mart between 8 August 2017 and 31 December 2021. Cases treated
with IV edaravone and controls not treated with IV edaravone were propensity score matched for: age,
sex, race, US region of residence, pre-index disease duration, insurance, riluzole prescription; and preindex
claims for cardiovascular disease, artificial nutrition/gastrostomy tube, noninvasive ventilation and
all-cause hospitalization. The index date was the first IV edaravone claim for cases; for controls, the
index date was randomly assigned after IV edaravone market availability. Restricted mean time lost
was calculated for the following disease progression milestones: new use of canes/walkers/wheelchairs,
artificial nutrition, noninvasive ventilation, invasive ventilation, speech-generating devices and hospice.
Results: Cases (n = 395) were matched to controls (n = 395). Cases had less restricted mean time lost,
indicating longer disease progression milestone-free time, for all disease progression milestones. From 0
to 24 months post index, more cases (n = 129) than controls (n = 103) reported no milestones and more
controls (n = 232) than cases (n = 131) reported deaths. Conclusion: In a US-based real-world setting, IV
edaravone-treated PALS had a longer time to disease progression milestone events and fewer deaths in 2
years compared with PALS not treated with IV edaravone. |
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| ISSN: | 2042-6313 |