Granulocyte Colony-stimulating Factor as Bridging Therapy for Pediatric Decompensated Liver Cirrhosis Prior to Liver Transplantation: an Open-label Randomised Controlled Trial

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Abstract

Decompensated cirrhosis in children is a leading indication of liver transplantation (LT). Granulocyte-colony stimulating factor (G-CSF) therapy has shown promising results in adult decompensated cirrhosis. Our study aimed to investigate the effect of G-CSF on liver function, Pediatric End-stage liver disease (PELD) score, CD34+ cells mobilization, nutritional status, short-term side effects, and survival in children indicated for liver transplantation (LT).

We performed an open-label, randomized controlled trial with decompensated liver cirrhosis between 3 months to 12 years old. The intervention group received a subcutaneous injection of G-CSF (5 μg/kg/day) for twelve courses in addition to standard medical treatment (SMT) for liver cirrhosis. We measured liver function, PELD scores, CD34+ cell mobilization, the change of leucocyte and neutrophil count, nutritional status, side effects, and survival within three months.

Thirty-five pediatric patients were randomized into 17 interventional groups and 18 control groups. During the trial, 14 (82%) of the interventional group completed the intervention course. The median age was 18 months in the interventional group and 14.5 months in the control group. The alanine aminotransferase (ALT) level showed significant improvement in the intervention group, while other liver parameters, PELD score, nutritional status, and survival, did not. CD34+ cells mobilization rose in the interventional group but was statistically insignificant. Minor side effects of G-CSF were found in the intervention group.

Multiple doses of G-CSF significantly improve ALT but did not improve PELD score, nutritional status, and survival in three months.
Original languageEnglish
Pages (from-to)1-16
JournalResearch Square
DOIs
Publication statusPublished - 7 Sept 2021

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