Granulocyte colony-stimulating factor therapy as a bridging treatment for pediatric decompensated liver cirrhosis prior to liver transplantation: an open-label randomized clinical trial

Background: Decompensated cirrhosis (DC) in children is the main indication of liver transplantation (LT). The lack of access to liver transplantation and impending clinical complications while awaiting transplantation affect the morbidity and mortality in pretransplant patients. Granulocyte colony-stimulating factor (G-CSF) therapy has shown promising results in adult decompensated cirrhosis as a potential bridging treatment. Our study aimed to identify the effect of G-CSF on pediatric end-stage liver disease (PELD) score, liver function, CD34+ cell mobilization, nutritional status, survival, and short-term side effects in children awaiting LT. Methods: This study was an open-label, randomized controlled trial that included patients with decompensated liver cirrhosis between 3 months and 12 years of age. The intervention group received 12 courses of G-CSF subcutaneous injection (5 μg/kg/day) plus standard medical treatment (SMT) for liver cirrhosis. Results were obtained regarding PELD scores, liver function, CD34+ cell mobilization, changes in leukocyte and neutrophil counts, nutritional status, survival, and side effects within three months. Results: Thirty-five pediatric patients were randomized into the intervention (17 patients) and control (18 patients) groups. During the trial, 14 (82%) in the intervention group completed the treatment. The median ages of the patients in the intervention and control groups were 18 and 14.5 months, respectively. The study’s primary outcome identified no statistically significant difference in PELD scores between the intervention and control groups after G-CSF treatment. Liver function tests that showed significant changes in the intervention group compared to the control group were from improvements in alanine aminotransferase (ALT) levels. Other liver function tests, nutritional status, and survival did not. CD34+ cell mobilization was increased in the intervention group compared with the control group, but there was no significant difference. Minor side effects of G-CSF were observed in the intervention group. Conclusion: Multiple doses of G-CSF did not improve the PELD score, nutritional status, and survival after three months but significantly showed temporary improvement in ALT level.