Study of Tacrolimus Used for Pediatric Patients With Nephrotic Syndrome Based on Pharmacogenomics and Metabonomics

Guangzhou Women and Children's Medical Center

Status: Unknown

Conditions: Nephrotic Syndrome

Treatments: Other: therapeutic drug monitoring

Study type: Observational

Funder types: Other

Identifiers

NCT02602873

683292136

Details and patient eligibility

About

Tacrolimus is recommended to be the first line therapeutic medication within the several immunosuppressive agents when treating refractory pediatric nephrotic syndrome, because of its definite efficacy and low toxicity. But there are still some key problems which hinder the using of tacrolimus in clinic, such as its narrow therapeutic widow, great individual difference of pharmacokinetics. Routine therapeutic drug monitoring(TDM) is needed in practice. But the disadvantage of TDM is hysteresis, which could lead to treatment failure or toxicity. To find out the reasons of great pharmacokinetic difference between patients and find out the individual proper dosage before administration are important for the clinical using of tacrolimus.

It is hot in research of tacrolimus in organ transplant field, such as the association between gene polymorphisms of cytochrome P-450 3A4, 3A5 and multiple drug resistant gene(MDR1) and concentration of tacrolimus. However, there is few study about pharmacogenomics and metabonomics of tacrolimus in patients of nephrotic syndrome.

The aim is to study the relationships between pharmacogenomics, metabonomics of tacrolimus and its efficacy, toxicity and blood concentration in patients of nephrotic syndrome, to find out the exact dosage before administration, to provide reference to individual drug administration.

Full description

Investigators will collect data about efficacy and adverse drug reaction(ADR) such as time of efficacy when evaluated, how to evaluate ADR, data of demography, etc.

Enrollment: 150 estimated patients

Sex: All

Ages: 1 month to 14 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. patients with refractory nephrotic syndrome;
2. patients age ≤14y.

Exclusion criteria

1. patients are sensitive to steroid;
2. combined therapy with other immunosuppressive agent;
3. combined using drugs which maybe interact the concentration of tacrolimus;
4. with other malignant disease, such as tumor.

Trial design

150 participants in 2 patient groups

good efficacy

Description:

using therapeutic drug monitoring to adjust the dose of tacrolimus. patients can reach effective outcome.

Treatment:

Other: therapeutic drug monitoring

poor efficacy

Description:

using therapeutic drug monitoring to adjust the dose of tacrolimus. patients can not reach effective outcome.

Treatment:

Other: therapeutic drug monitoring