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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Becaris Publishing Limited
2025-01-01
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| Series: | Journal of Comparative Effectiveness Research |
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| author | James D Berry Melissa Hagan Jeffrey Zhang Ying Liu Malgorzata Ciepielewska |
| author_facet | James D Berry Melissa Hagan Jeffrey Zhang Ying Liu Malgorzata Ciepielewska |
| author_sort | James D Berry |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-d110065490e24c248576eb43aa6dbdf9 |
| institution | Kabale University |
| issn | 2042-6313 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | Becaris Publishing Limited |
| record_format | Article |
| series | Journal of Comparative Effectiveness Research |
| spelling | doaj-art-d110065490e24c248576eb43aa6dbdf92025-01-27T15:17:38ZengBecaris Publishing LimitedJournal of Comparative Effectiveness Research2042-63132025-01-0114210.57264/cer-2024-0007Longer disease progression milestone-free time in people with amyotrophic lateral sclerosis treated versus not treated with intravenous edaravone: results from an administrative claims analysisJames D Berry0https://orcid.org/0000-0003-0898-6081Melissa Hagan1https://orcid.org/0009-0004-6677-0662Jeffrey Zhang2https://orcid.org/0000-0003-0890-0444Ying Liu3Malgorzata Ciepielewska4Healey Center for ALS, Massachusetts General Hospital, Neurology, Boston, MA, 02114 USAMitsubishi Tanabe Pharma America, Inc., Health Economics and Outcomes Research (HEOR), Medical Affairs, Jersey City, NJ, 07310 USAPrinceton Pharmatech, LLC, Biostatistics, Princeton, NJ, 08550 USAPrinceton Pharmatech, LLC, Biostatistics, Princeton, NJ, 08550 USAMitsubishi Tanabe Pharma America, Inc., Health Economics and Outcomes Research (HEOR), Medical Affairs, Jersey City, NJ, 07310 USAAim: 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.administrative claims databaseamyotrophic lateral sclerosisdisease progressionedaravonemilestonespropensity score matchingreal-world datarestricted mean time lostsurvival |
| spellingShingle | James D Berry Melissa Hagan Jeffrey Zhang Ying Liu Malgorzata Ciepielewska 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 Journal of Comparative Effectiveness Research administrative claims database amyotrophic lateral sclerosis disease progression edaravone milestones propensity score matching real-world data restricted mean time lost survival |
| title | 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 |
| title_full | 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 |
| title_fullStr | 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 |
| title_full_unstemmed | 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 |
| title_short | 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 |
| title_sort | 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 |
| topic | administrative claims database amyotrophic lateral sclerosis disease progression edaravone milestones propensity score matching real-world data restricted mean time lost survival |
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