About this Research Topic
Hemophagocytic lymphohistiocytosis (HLH) in the pediatric population represents a severe, life-threatening immune dysregulation syndrome, marked by extreme inflammation and cytokine elevation. This hyperinflammation is triggered by T-lymphocyte overactivation and defective natural killer cell function, contributing to both primary and secondary disease forms. Primary HLH, typically seen in young children, requires aggressive treatment like hematopoietic stem cell transplantation (HSCT) following chemotherapy-induced remission. Secondary HLH, however, often relies on a regimen of dexamethasone, cyclosporine, and etoposide. Despite these interventions, treatment-resistant and relapsed cases of HLH pose critical challenges, highlighting an urgent need for novel therapeutic approaches.
This Research Topic aims to explore state-of-the-art targeted therapies for severe and refractory HLH. Focusing on innovative treatment modalities, it seeks to uncover how monotherapies or their combination with standard treatments like chemotherapy or HSCT can improve patient outcomes. Emphasis on a robust understanding of genetic underpinnings and disease mechanisms that lead to treatment resistance will guide the discovery of more effective interventions.
To gather further insights into these complex treatment strategies, we welcome articles addressing, but not limited to, the following themes:
- Exploring genetic defects and mechanisms contributing to HLH refractoriness.
- Evaluating the efficacy of novel targeted therapies, whether as standalone treatments or in combination with existing protocols.
- Assessing the integration of stem cell transplantation with new pharmacologic therapies to enhance survival rates.
Please note: Manuscripts consisting solely of bioinformatics, computational analysis, or predictions of public databases which are not accompanied by validation (independent clinical or patient cohort, or biological validation in vitro or in vivo, which are not based on public databases) are not suitable for publication in this journal.
This Research Topic aims to explore state-of-the-art targeted therapies for severe and refractory HLH. Focusing on innovative treatment modalities, it seeks to uncover how monotherapies or their combination with standard treatments like chemotherapy or HSCT can improve patient outcomes. Emphasis on a robust understanding of genetic underpinnings and disease mechanisms that lead to treatment resistance will guide the discovery of more effective interventions.
To gather further insights into these complex treatment strategies, we welcome articles addressing, but not limited to, the following themes:
- Exploring genetic defects and mechanisms contributing to HLH refractoriness.
- Evaluating the efficacy of novel targeted therapies, whether as standalone treatments or in combination with existing protocols.
- Assessing the integration of stem cell transplantation with new pharmacologic therapies to enhance survival rates.
Please note: Manuscripts consisting solely of bioinformatics, computational analysis, or predictions of public databases which are not accompanied by validation (independent clinical or patient cohort, or biological validation in vitro or in vivo, which are not based on public databases) are not suitable for publication in this journal.
Keywords: hemophagocytic lymphohistiocytosis, targeted therapy, refractory disease, relapse, pediatric population, pediatric cancer
Important Note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.
