| 제목 |
Effect of Edaravone in Patients with advanced Amyotrophic Lateral Sclerosis |
| 소속 |
Gangnam Severance Hospital, Yonsei University College of Medicine, Seoul, Department of Rehabilitation Medicine and Rehabilitation Institute of Neuromuscular Disease1, Gangnam Severance Hospital, Yonsei University College of Medicine, Seoul, Pulmonary Rehabilitation Center2, CHA Bundang Medical Center, CHA University, Department of Rehabilitation Medicine3, Graduate School of Yonsei University, Department of Physical Therapy4, Severance Hospital, Yonsei University College of Medicine, Seoul, Department of Rehabilitation Medicine5 |
| 저자 |
Eunyoung Kim1,2*, Mi Ri Suh3, Bitnarae Kim2,4, Won Ah Choi1,2, Sanghoon Shin5, Seong-Woong Kang1,2† |
Introduction
In 2017, the FDA approved Edaravone as a neuroprotective agent to slow the progression of Amyotrophic Lateral Sclerosis (ALS). The precise mechanism of Edaravone in the treatment of ALS is not known, but its therapeutic effect is considered to be done by its antioxidant properties. The former clinical trials have been performed on ALS patients in less advanced status only (Japan ALS severity classification grade 1-3; forced vital capacity (FVC) in ratio to normal predicted value (FVCPred%) of ≥60%). In the previous clinical trials published in 2014, the average change in ALSFRS score was -5.7 ± 0.85 decline, and the next study presented only at the 2016 conference showed a decrease in average ALSFRS score -6.52 ± 1.78. Therefore we included more advanced ALS patients with a FVCPred% of less than 60% to study the efficacy of Edaravone in these patients.
Case Report
Since January of 2017 to June of 2018, 19 patients were enrolled in the Edaravone treatment. 60 mg of Edaravone is administered daily intravenously for the first 14 days which is followed by 14 days of rest. In the second to sixth cycles, the same dose of Edaravone is given for 10 days in every cycle, with a 14-day rest period between each cycles. Eleven patients finished the treatment of 6 cycles, 2 patients were dropped out and 3 patients are still continuing the treatment. Among the 11 patients who finished the total 6 cycles, 7 patients (M:F=5:2, mean age = 58.6±8.8 years) had FVCPred% of less than 60% at initial. These seven patients started the Edaravone infusion approximately 2.17±1.58 years after symptom onset and 0.99±1.02 years after diagnosis. For all subjects, pulmonary function(FVC and peak cough flow), ALS functional rating scale (ALSFRS) and manual muscle test were checked at the beginning and end of the Edaravone infusion for every cycle. We also performed the regular laboratory tests to screen drug-related adverse outcomes.
The mean ALSFRS score was 30.1 ± 8.1 at baseline and 24.7 ± 5.4 after 6 cycles of Edaravone administration. The mean change in ALSFRS score from baseline was -7.2 ± 6.6. The mean baseline FVC Pred% in the sitting position was 36.1 ± 12.4 and it became 25.7 ± 12.7 after therapy. The mean baseline FVC Pred% and after 6 months of treatment in the supine position was 28.3 ± 5.5 and 22.5 ± 8.9 respectively. Not any major drug-related side effects were found.
Conclusion
The mean change in ALSFRS scores of patients with more advanced ALS was significantly reduced compared to the values reported in previous clinical trials. We can conclude that Edaravone can be successfully treated in ALS patients with a FVCPred% of less than 60%. Nowadays, we are enrolling more patients, following up serial ALSFRS scores and pulmonary function parameters, aiming to compare the efficacy of Edaravone depending on the types of onset and on the methods of therapies(combined with exercise therapy Vs. Edaravone alone). |
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Figure 1. Changes of ALSFRS score at each cycle of Edaravone therapy
Table 1. FVC and ALSFRS score in each patient under Edaravone therapy
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