Patients on edaravone had significantly higher rates of all-cause hospitalization, and about 60% discontinued the drug, Dr. Francesca Cunningham of the Department of Veterans Affairs in Hines, Illinois, and colleagues found.
Those who were on edaravone for six months or longer were more than twice as likely to be hospitalized for ALS-related causes, the researchers report in JAMA Network Open.
"This surveillance evaluation identified that patients using edaravone (mostly with riluzole) had unexpected outcomes, which raises questions about its benefit in Veterans Health Administration patients and underscores the importance of further studies," Dr. Cunningham and her team conclude.
The U.S. Food and Drug Administration approved the free-radical scavenger edaravone, an intravenous medication, for treating ALS in 2017. Marketed as Radicava by Mitsubishi Tanabe Pharma America, the drug costs about $150,000 for a year of treatment.
Dr. Cunningham and her team looked at 369 VHA patients with ALS who received at least one prescription for edaravone between 2017 and 2019. Most (71.3%) were also on riluzole. They also matched 223 patients on edaravone to 669 on riluzole alone by propensity score.
Acute all-cause hospitalization events occurred in 35.4% of the edaravone group and 22% of the riluzone-only patients (P<0.001). The mean time to hospitalization was 44.5 days for edaravone vs. 68.2 days for riluzone only, while the mean time to discontinuation was 93.8 days with edaravone and 161.8 with riluzole alone.
Amyotrophic Lateral Sclerosis Functional Rating Scale (ALS-FRS-R) scores declined by a median of 8 points from baseline for the patients on riluzole alone, versus a 2-point drop for patients on edaravone.
Among chronic users, the hazard ratios for ALS hospitalization and percutaneous endoscopic gastrostomy (PEG) tube placement were 2.51 and 3.04, respectively, for edaravone versus riluzole - both significant risk increases. Mortality rates were similar for the two groups.
"It is important to use real-world data and pharmacovigilance efforts to assess medications in your healthcare system to better understand utilization, safety and effectiveness," Dr. Cunningham told Reuters Health by email. "These data assist doctors and patients in shared decision-making to provide realistic expectations of how a drug performs in real-world settings rather than in clinical trial settings."
The findings should not raise "major concerns" about prescribing edaravone, "but should encourage more detailed studies to be conducted to determine the effectiveness of edaravone in ALS patients," she added.
"To confirm these findings, robust research designs with in-depth medical record review are needed to better characterize drug exposure, reasons for discontinuation, outcomes, and covariates. The VA will continue to track and monitor the safe and appropriate use of edaravone and provide timely information to optimize veteran ALS care," she and her colleagues conclude.
Mitsubishi Tanabe Pharma America did not respond to a request for comment by press time.
By Anne Harding
SOURCE: https://bit.ly/3nvFmI6 JAMA Network Open, online October 5, 2020.
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