Granulocyte colony stimulating factor(Lipoxen Technologies Ltd.)

Management of pediatric acute megakaryoblastic leukemia (AMKL) without Down syndrome (non‐DS‐AMKL) remains challenging due to suboptimal induction responses and poor survival. This study assessed the efficacy of fludarabine, cytarabine, granulocyte colony‐stimulating factor, and idarubicin (FLAG‐IDA) in non‐DS‐AMKL and evaluated the role of risk‐adapted consolidation strategies, including hematopoietic stem cell transplantation (HSCT) during first complete remission (CR1), guided by genetic profiles and measurable residual disease (MRD) dynamics. In this multicenter retrospective study from China, we analyzed 58 non‐DS‐AMKL patients treated with FLAG‐IDA ( n  = 50) or daunorubicin, cytarabine, etoposide (DAE) ( n  = 8) induction, followed by risk‐adapted consolidation. FLAG‐IDA demonstrated CR rates comparable to those with DAE (first‐course: 70.0% vs. 87.5%, p  = 0.423; cumulative: 82.0% vs. 87.5%, p  = 1.000). Compared with other AML subtypes, non‐DS‐AMKL showed inferior 5‐year overall survival (OS) (61.7% vs. 78.2%, p  = 0.001) and event‐free survival (EFS) (58.5% vs. 68.8%, p  = 0.045), attributable to a higher relapse rate (34.8% vs. 19.5%, p  = 0.002). High‐risk genetics predicted worse outcomes. HSCT in CR1 significantly improved survival for high‐risk or poor responders. CR with MRD negativity after second induction predicted superior OS and EFS ( p  < 0.001). Risk‐adapted HSCT and MRD‐guided stratification are critical for non‐DS‐AMKL management.

Thrombocytopenia and leukopenia are among the most common hematological abnormalities in patients with chronic liver disease (CLD), particularly among those with cirrhosis. Thrombocytopenia and leukopenia are associated with increased risks of bleeding, infection, treatment delay, and poor clinical outcomes. Their pathogenesis is multifactorial, comprising hypersplenism‐related sequestration, decreased synthesis of hematopoietic growth factors, such as thrombopoietin, bone marrow suppression, immune‐mediated destruction, and systemic inflammation. This review summarizes the current understanding of the mechanisms underlying cytopenias in CLD and discusses available therapeutic strategies, including platelet transfusion, spleen‐targeted interventions, thrombopoietin‐based treatments, thrombopoietin receptor agonists, and granulocyte colony‐stimulating factor. Particular attention is given to the efficacy and safety profiles of these treatment modalities and their potential risks, such as thromboembolic events due to thrombopoietin receptor agonists. Importantly, effective management of cytopenias in CLD should be tailored based on disease severity, etiology of cirrhosis, dominant pathophysiological mechanisms, thrombotic risk, and the urgency of invasive procedures. Integrating these factors into clinical decision‐making may improve the outcomes of patients and optimize treatment choices. Future studies should focus on risk‐stratified treatment algorithms, long‐term safety of emerging agents, and combination therapeutic strategies.