Based on the genotype characteristics and genotype-phenotype-treatment prognosis data of Chinese WD patients, this study intends to further optimize the treatment regimen of Chinese WD patients and formulate individualized treatment regimens for each genotype, so as to further improve the prognosis of patients.
The purpose of the study is to evaluate treatment regimen efficacy based on different mutations, and Zinc treatment in presymptomatic WD patients.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
400
Dosage Form: DMPS: 500-1000mg per day,DMPS Frequency:BID,DMPS Duration: 6 days;
Dosage Form: Penicillamine: 250-1500mg per day, Frequency:TID,Duration: 5 years;
Dosage Form: DMSA: 750-1000mg per day,DMSA Frequency:BID,DMSA Duration: 5 years; Dosage Form: DMSA: 35mg/kg per day,DMSA Frequency:BID,DMSA Duration: 5 years;
econd Affiliated Hospital,Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
RECRUITINGSerum ceruloplasmin
Serum ceruloplasmin concentration will be analysed using a validated assay
Time frame: Five years
24-hour urine copper
24-hour urinary copper excretion is to be measured
Time frame: Five years
serum copper
serum copper will be analysed using a validated assay
Time frame: Five years
White blood cell
The white blood cell will be evaluated using a validated assay
Time frame: Five years
Platelet count
The platelet count will be evaluated using a validated assay
Time frame: Five years
urine protein level
The urine protein level will be collected using a validated assay
Time frame: Five years
Alanine transaminase
The concentration of alanine transaminase will be collected using a validated assay
Time frame: Five years
blood creatinine
The concentration of blood creatinine will be tested using a validated assay
Time frame: Five years
international normalized ratio
The international normalized ratio will be analyzed
Time frame: Five years
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Dosage Form: Zinc gluconate: 140mg per time,Zinc Frequency:BID,Zinc Duration: 5 years; Dosage Form: Zinc gluconate: 140mg per time,Zinc Frequency:TID,Zinc Duration: 5 years; Dosage Form: Zinc gluconate: 210mg per time,Zinc Frequency:TID,Zinc Duration: 5 years;
hepatic fibrosis markers test
The concentrations of procollagen III, collage IV and hyaluronidase will be evaluated using a validated assay
Time frame: Five years
bone mineral density test
The bone mineral density will be tested using the dual energy X ray absorptiometry
Time frame: Five years
Abdominal ultrasound
The abdominal ultrasound will be collected
Time frame: Five years
Urinary ultrasound
The urinary ultrasound will be analyzed in patients
Time frame: Five years
Cranial MRI scan
The cranial MRI scan will be analyzed in patients
Time frame: Five years
Unified Wilson's disease rating scale
The Unified Wilson's disease rating scale consist of three subscales, including neurological part (0\~112), liver functional part (0\~36) and mental state part (0\~76). Three subscale scores are summed to compute a total score. The higher values represent a worse outcome
Time frame: Five years