Granulocyte-Colony Stimulating Factor (G-CSF) as Optimizing Therapy for Pediatric Liver Transplantation

University of Indonesia (UI)

Status and phase: Completed; Phase 3

Conditions: Severe Malnutrition; Liver Cirrhosis; Undernutrition; PELD

Treatments: Drug: G-CSF (Filgrastim)

Study type: Interventional

Funder types: Other

Identifiers

NCT04113317

FakultasKUI

Details and patient eligibility

About

This study compares the effect of human recombinant Granulocyte-Colony Stimulating Factor (G-CSF) in pediatric patients with liver cirrhosis with a control group. The study aims to observe improvement of (Pediatric End-stage Liver Disease) PELD score and nutritional status prior to liver transplantation procedure. In addition to the intervention, standard treatments for liver cirrhosis are also given for both groups. G-CSF is administered for 12 times.

Condition of disease:

Pediatric patient aged 3 months to 12 years old Liver cirrhosis Undernourished / Severe malnutrition PELD score 10-25

Intervention:

Drug: Recombinant Human G-CSF

Phase:

Phase 3

Full description

This open-label randomized control trial conducted in Cipto Mangunkusumo Hospital, Jakarta, Indonesia, aimed to determine the effect of G-CSF to improve PELD score and nutritional status of pediatric patients with liver cirrhosis prior to liver transplantation. A total of 52 pediatric patients with liver cirrhosis, malnutrition and PELD score between 10-25 will be collected and divided into intervention group and control group. The intervention group will be given a subcutaneous injection of G-CSF 5μg/kg/day for 5 days consecutively, followed by a single dose every 3 days up to 12 times throughout the trial. Additionally, both the intervention and control group receive standard liver cirrhosis treatment. Randomization is done using block randomization.

The patients will be clinically monitored and evaluated for anthropometric changes (body weight, mid-arm circumference, mid-arm muscle circumference, and triceps skinfold thickness), complete blood count, absolute neutrophil count, liver function tests, cytokines (IL-10, TNF-α), Procalcitonin, hepatocyte growth factor (HGF), CD34+ cell count and PELD score (albumin, bilirubin, prothrombin and international normalized ratio values).

Data analysis will be performed using IBM SPSS Statistics version 20.0.0.

Enrollment: 52 patients

Sex: All

Ages: 3 months to 12 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Age between 3 months to 12 years old
• Conscious state
• Without the presence of fever (temperature: <37,5°C)
• Decompensated liver cirrhosis
• Liver cirrhosis due to various etiology
• Undernourished or severe malnutrition
• PELD score between 10-25

Exclusion criteria

• Malignancy (liver origin or other types)
• History of undergoing any organ transplantation procedure
• Acute liver failure
• Failure of any organ other than the liver
• Encephalopathy
• Severe infection such as bacterial peritonitis and pneumonia

Trial design

Primary purpose: Supportive Care

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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52 participants in 2 patient groups

Treatment Group

Experimental group

Description:

Subcutaneous injection of G-CSF with dose of 5μg/kg/day for 5 days consecutively, in addition to a single dose every 3 days up to 12 times, as well as liver cirrhosis standard regimen

Treatment:

Drug: G-CSF (Filgrastim)

Control Group

No Intervention group

Description:

Liver cirrhosis standard treatment only