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Influence of a clinical trial in the decision-making processes of patients with amyotrophic lateral sclerosis

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Abstract

Background

Amyotrophic lateral sclerosis (ALS) is an incurable neurological disease, and patients diagnosed with ALS have a survival time of 2–5 years without life-sustaining therapy. Decision-making processes for the acceptance or decline of percutaneous endoscopic gastrostomy (PEG) and tracheostomy with invasive ventilation (TIV) therapy are complex and multifaceted. In this study, we examined whether participation or no participation in clinical trials of ALS had an influence on the decision-making processes of ALS patients.

Methods

Fifty-seven consecutive ALS participants were recruited. Two participants did not wish to participate in any clinical trials, and Twenty-two participants were enrolled in clinical trials. Twenty-three participants wished to participate but could not be enrolled in any of the clinical trials because they exceeded the number of participants in these trials or they met the exclusion criteria.

Result

At baseline, there was no significant difference in the preference rates for PEG and TIV between the participant and non-participant groups, but after the double-blind period/6 months, both preference rates were significantly higher in the non-participant group than in the participant group. Notably, the rate of preferred TIV in the participant group drastically decreased after the double-blind period. A single regression analysis revealed that participation in clinical trials had a strong influence on the change of TIV preference for 6 months.

Conclusion

Participation in a clinical trial decreases the willingness to prolong life after the clinical trial. The present results are meaningful when designing clinical trials and discussing life-sustaining treatments with ALS patients.

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Data are available for collaborative studies with qualified investigators after inquiry.

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Acknowledgements

The authors would like to thank the staff of Keio University Hospital.

Funding

This research was supported by the Ministry of Education, Culture, Sports, Science and Technology of Japan (No. 21H02812) (to DI), Foundation of Japan Amyotrophic Lateral Sclerosis Association, Ice Bucket Challenge Grant (Japan Amyotrophic Lateral Sclerosis Association) (to DI and HO), Serika Fund (to DI and HO) and a grant support from Japan Agency for Medical Research and Development [Research on Practical Application of Innovative Pharmaceutical and Medical Devices for Rare and Intractable Diseases (Grant No. JP 18ek0109395, JP 19ek0109395, JP 20ek0109395, JP 18ek0109329, JP 19ek0109329, JP 20ek0109329) to HO].

Author information

Authors and Affiliations

  1. Department of Neurology, Keio University School of Medicine, 35 Shinanomachi, Shinjuku-ku, Tokyo, 160-8582, Japan

    Chris Kato, Satoru Morimoto, Shinichi Takahashi, Yugaku Daté, Kensuke Okada, Jin Nakahara & Daisuke Ito

  2. Department of Physiology, Keio University School of Medicine, Tokyo, 160-8582, Japan

    Satoru Morimoto, Shinichi Takahashi & Hideyuki Okano

  3. Department of Neurology and Stroke, Saitama Medical University International Medical Center, Saitama, Japan

    Shinichi Takahashi

Authors
  1. Chris Kato
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  2. Satoru Morimoto
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  3. Shinichi Takahashi
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  4. Yugaku Daté
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  5. Kensuke Okada
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  6. Hideyuki Okano
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  7. Jin Nakahara
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  8. Daisuke Ito
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Contributions

Study conception and design were conducted by DI. Data processing and statistical analyses were conducted by CK, SM, ST, YD, KO, HO, and JN. The first draft of the manuscript was written by CK, and all authors reviewed, critiqued, and edited subsequent versions of the manuscript. All authors approved the final manuscript.

Corresponding author

Correspondence to Daisuke Ito.

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Conflicts of interest

On behalf of all authors, the corresponding author states that there is no conflict of interest, except that HO is a founder scientist and a Scientific Advisory Board of SanBio Co. Ltd. and K Pharma Inc and DI is the principal investigator of the VALOR study [ClinicalTrials.gov Identifier: NCT02623699].

Ethical approval

The study design and protocol were approved by the Ethics Committee for Human Research of the Keio University School of Medicine (#20200287).

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Kato, C., Morimoto, S., Takahashi, S. et al. Influence of a clinical trial in the decision-making processes of patients with amyotrophic lateral sclerosis. J Neurol 269, 2634–2640 (2022). https://doi.org/10.1007/s00415-021-10862-6

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  • DOI: https://doi.org/10.1007/s00415-021-10862-6

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