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Effect of leuprorelin in bulbar function of spinal and bulbar muscular atrophy patients: observational study for 1 year

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Abstract

Background

This study aimed to investigate the effect of androgen suppression therapy using leuprorelin focused on the bulbar function of patients with spinal and bulbar muscular atrophy (SBMA).

Methods

Genetically confirmed SBMA patients who consented to participate in this observational study were enrolled. Leuprorelin was subcutaneously injected every 12 weeks. Videofluoroscopic swallowing study was performed at baseline and after androgen suppression therapy for 1 year. The primary outcome measures were the changes in the vallecular residue and pyriform sinus residue. The videofluoroscopic swallowing study data were analyzed and interpreted by two experienced physiatrists.

Results

A total of 40 patients with SBMA were analyzed in this study. The inter-rater reliability testing showed good agreement for the pharyngeal residue (ICC = 0.84) and videofluoroscopic dysphagia scale (ICC = 0.75). The vallecular residue and pyriform sinus residue after swallowing 9 mL yogurt were significantly reduced (26.8 ± 22.6 to 14.6 ± 14.5, p < 0.001, 14.9 ± 16.9 to 7.6 ± 9.9, p < 0.001, respectively). The swallowing subscore of amyotrophic lateral sclerosis functional rating scale-revised improved after androgen suppression therapy (3.3 ± 0.5 to 3.5 ± 0.6, p = 0.041).

Conclusions

Leuprorelin significantly reduced the pharyngeal residue in patients with SBMA after 1 year of treatment without any serious adverse events and longitudinal studies are needed to confirm these results.

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Acknowledgements

This work was supported by the National Research Foundation of Korea (NRF) grant funded by the Korea government (Ministry of Science and ICT) (No. 2017R1C1B5076264).

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Authors and Affiliations

  1. Department of Physical Medicine and Rehabilitation, Dong-A University College of Medicine, Busan, Republic of Korea

    Min-Gu Kang

  2. Department of Rehabilitation Medicine, School of Medicine, Kyungpook National University, Kyungpook National University Chilgok Hospital, Daegu, Republic of Korea

    Dae-Won Gwak & Yu-Sun Min

  3. Department of Neurology, School of Medicine, Kyungpook National University, Kyungpook National University Chilgok Hospital, Daegu, Republic of Korea

    Hee-Jin Cho & Jin-Sung Park

Authors
  1. Min-Gu Kang
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  2. Dae-Won Gwak
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  3. Hee-Jin Cho
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  4. Yu-Sun Min
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  5. Jin-Sung Park
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Contributions

Study concept and design: JSP and YSM; collection and analysis of data: MGK, DWG and HJC; interpretation of study data: MGK, DWG, HJC, YSM, and JSP; drafting the manuscript: MGK and JSP; editing the manuscript: MGK and JSP.

Corresponding author

Correspondence to Jin-Sung Park.

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Conflicts of interest

None.

Ethics approval

This study was approved by the Institutional Review Board of the Kyungpook National University Chilgok Hospital approved this study (KNUCH 2018-01-009).

Informed consent

All participants provided written informed consent.

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Kang, MG., Gwak, DW., Cho, HJ. et al. Effect of leuprorelin in bulbar function of spinal and bulbar muscular atrophy patients: observational study for 1 year. J Neurol 268, 3344–3351 (2021). https://doi.org/10.1007/s00415-021-10503-y

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  • DOI: https://doi.org/10.1007/s00415-021-10503-y

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