Objective:

2

To evaluate the clinical efficacy and safety of Congrong Shujing Granules (苁蓉舒痉颗粒

CSGs) in treating patients with Parkinson's disease (PD) and Chinese medicine (CM) syndrome of Shen (Kidney) essence deficiency

and to investigate the potential mechanism involving efficacy through a transcriptome sequencing approach.

Methods:

2

Eligible PD patients with syndrome of Shen essence deficiency were randomly assigned to a treatment group or a control group by a random number table

and were treated with CSGs combined with Western medicine (WM)

or placebo combined with WM

respectively. Both courses of treatment lasted for 12 weeks. The Unified Parkinson's Disease Rating Scale (UPDRS) score

the PD Question-39 (PDQ-39) score

CM Syndrome Scale score

and drug usage of all patients were evaluated before and after treatment. Safety was evaluated by clinical laboratory tests and electrocardiographs. Blood samples from 6 patients in each group were collected before and after the trial and used for transcriptomic analysis by gene ontology analysis and Kyoto Encyclopedia of Genes and Genomes pathway analysis. Differentially expressed genes were validated using reverse transcription-polymerase chain reaction.

Results:

2

A total of 86 PD patients were selected from the Third Affiliated People's Hospital of Fujian University of Traditional Chinese Medicine between January 2017 and December 2017. Finally

72 patients completed the trial

including 35 in the treatment group and 37 in the control group. When compared with the control group after treatment

patients in the treatment group showed significant decreases in UPDRS sub-Ⅱ score

PDQ-39 score

CM syndrome score

and Levodopa equivalent dose (

P

<

0.05). During the treatment course

no significant changes were observed in safety indicators between the two groups (

P

>

0.05). A possible mechanism of clinical efficacy was proposed that involved regulating cell metabolism-related processes and ribosome-related pathways. Treatment with CSGs had shown to affect relevant gene loci for PD

including AIDA

ANKRD36BP2

BCL2A1

BCL2L11

FTH1P2

GCH1

HPRT1

NFE2L2

RMRP

RPS7

TGFBR1

WIPF2

and COX7B.

Conclusions:

2

CSGs combined with WM can be used to treat PD patients with CM syndrome of Shen essence deficiency with a good safety. The possible mechanism of action and relevant gene loci were proposed. (Registration No. ChiCTR-IOR-16008394)