Efficacy of faecal microbiota transplantation in patients with progressive supranuclear palsy-Richardson's syndrome: a phase 2, single centre, randomised clinical trialResearch in context

Haiyan Tian; Jiuqi Wang; Renyi Feng; Rui Zhang; Han Liu; Chi Qin; Lin Meng; Yongkang Chen; Yu Fu; Dongxiao Liang; Xin Yuan; Yanping Zhai; Qingyong Zhu; Lingjing Jin; Junfang Teng; Xuebing Ding; Xuejing Wang

EClinicalMedicine (Apr 2023)

Efficacy of faecal microbiota transplantation in patients with progressive supranuclear palsy-Richardson's syndrome: a phase 2, single centre, randomised clinical trialResearch in context

Haiyan Tian,
Jiuqi Wang,
Renyi Feng,
Rui Zhang,
Han Liu,
Chi Qin,
Lin Meng,
Yongkang Chen,
Yu Fu,
Dongxiao Liang,
Xin Yuan,
Yanping Zhai,
Qingyong Zhu,
Lingjing Jin,
Junfang Teng,
Xuebing Ding,
Xuejing Wang

Affiliations

Haiyan Tian: Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, 450052, China; Institute of Parkinson and Movement Disorder, Zhengzhou University, Zhengzhou, Henan, 450052, China
Jiuqi Wang: Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, 450052, China; Institute of Parkinson and Movement Disorder, Zhengzhou University, Zhengzhou, Henan, 450052, China
Renyi Feng: Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, 450052, China; Institute of Parkinson and Movement Disorder, Zhengzhou University, Zhengzhou, Henan, 450052, China
Rui Zhang: Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, 450052, China; Institute of Parkinson and Movement Disorder, Zhengzhou University, Zhengzhou, Henan, 450052, China
Han Liu: Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, 450052, China; Institute of Parkinson and Movement Disorder, Zhengzhou University, Zhengzhou, Henan, 450052, China
Chi Qin: Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, 450052, China; Institute of Parkinson and Movement Disorder, Zhengzhou University, Zhengzhou, Henan, 450052, China
Lin Meng: Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, 450052, China; Institute of Parkinson and Movement Disorder, Zhengzhou University, Zhengzhou, Henan, 450052, China
Yongkang Chen: Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, 450052, China; Institute of Parkinson and Movement Disorder, Zhengzhou University, Zhengzhou, Henan, 450052, China
Yu Fu: Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, 450052, China; Institute of Parkinson and Movement Disorder, Zhengzhou University, Zhengzhou, Henan, 450052, China
Dongxiao Liang: Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, 450052, China; Institute of Parkinson and Movement Disorder, Zhengzhou University, Zhengzhou, Henan, 450052, China
Xin Yuan: Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, 450052, China; Institute of Parkinson and Movement Disorder, Zhengzhou University, Zhengzhou, Henan, 450052, China
Yanping Zhai: Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, 450052, China; Institute of Parkinson and Movement Disorder, Zhengzhou University, Zhengzhou, Henan, 450052, China
Qingyong Zhu: Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, 450052, China; Institute of Parkinson and Movement Disorder, Zhengzhou University, Zhengzhou, Henan, 450052, China
Lingjing Jin: Department of Neurology and Neurological Rehabilitation, Shanghai Yangzhi Rehabilitation Hospital, School of Medicine, Tongji University, Shanghai, 200092, China
Junfang Teng: Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, 450052, China; Institute of Parkinson and Movement Disorder, Zhengzhou University, Zhengzhou, Henan, 450052, China; Corresponding author. Department of Neurology, The First Affiliated Hospital of Zhengzhou University, No.1 Jianshe Dong Road, ErQi District, Zhengzhou, Henan, 450052, China.
Xuebing Ding: Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, 450052, China; Institute of Parkinson and Movement Disorder, Zhengzhou University, Zhengzhou, Henan, 450052, China; Corresponding author. Department of Neurology, The First Affiliated Hospital of Zhengzhou University, No.1 Jianshe Dong Road, ErQi District, Zhengzhou, Henan, 450052, China.
Xuejing Wang: Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, 450052, China; Institute of Parkinson and Movement Disorder, Zhengzhou University, Zhengzhou, Henan, 450052, China; Corresponding author. Department of Neurology, The First Affiliated Hospital of Zhengzhou University, No.1 Jianshe Dong Road, ErQi District, Zhengzhou, Henan, 450052, China.

Journal volume & issue: Vol. 58
p. 101888

Abstract

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Summary: Background: Faecal microbiota transplantation (FMT) has demonstrated efficacy in treating gastrointestinal (GI) diseases, such as Clostridium difficile infection (CDI) and inflammatory bowel disease (IBD). GI dysfunction is a frequent and occasionally dominating symptom of progressive supranuclear palsy-Richardson's syndrome (PSP-RS). However, it is not known whether FMT has clinical efficacy for PSP-RS. Methods: This 36-week, randomised, placebo-controlled, parallel-group, phase 2 clinical trial was performed at a university tertiary referral hospital in China. From August 15 2021 to December 31 2021, a total of 68 newly diagnosed patients with PSP-RS (male 40 [59%], female 28 [41%]) who had never received any antiparkinsonian medications were enrolled and randomly assigned to receive either healthy donor FMT (n = 34, FMT group) or a mixture of 0.9% saline and food colouring (E150c) as sham transplantation (n = 34, placebo group) through transendoscopic enteral tubing (TET). Two days after oral antibiotics, participants received 1 week of transplantation. After an interval of 4 weeks, retransplantation was performed. Then, the last transplantation was given after another interval of 4 weeks, and the participants were followed up for 24 weeks (week 36). Clinicaltrials.gov identifier: ChiCTR-2100045397. Findings: Among 68 patients who were randomised (mean age, 67.2 (SD 5.1); 40 [59%] were male, 28 [41%] were female), 63 participants completed the trial. Efficacy analyses were performed on the intention-to-treat (ITT) analysis set. At week 16, the mean PSP Rating Scale (PSPRS) scores (the primary outcome) improved from 40.1 (SD 7.6) to 36.9 (SD 5.9) in the FMT group, whereas the scores changed from 40.1 (SD 6.9) to 41.7 (SD 6.2) in the placebo group, for a treatment benefit of 4.3 (95% CI, 3.2–5.4) (P < 0.0001). After 3-cycle intervention, symptoms of constipation, depression, and anxiety (the secondary outcome) improved significantly at week 16 in the FMT group compared with the placebo group, the majority of which were maintained at the 24-week follow-up (week 36). Interpretation: Our findings suggest that, compared with placebo, FMT treatment significantly improved motor and nonmotor symptoms in patients with PSP-RS, as well as reduced intestinal inflammation and enhanced the intestinal barrier by regulating the intestinal microbiota composition. Funding: The National Natural Science Foundation of China (No. 82122022, 82171248, 81873791, and 82230084), Natural Science Foundation of Henan Province for Excellent Young Scholars (no. 202300410357), and Henan Province Young and Middle-Aged Health Science and Technology Innovation Talent Project (YXKC2020033).

Published in EClinicalMedicine

ISSN: 2589-5370 (Online)
Publisher: Elsevier
Country of publisher: United Kingdom
LCC subjects: Medicine: Medicine (General)
Website: https://www.thelancet.com/journals/eclinm/home

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