About this Research Topic
Hemophagocytic lymphohistiocytosis (HLH) is a potentially life-threatening disease, that occurs in children, characterized by hyperinflammation consecutive to immune dysregulation. Patients with HLH have elevations in numerous cytokines including IL-2, IL-6, IL-10, and IL-18, and IFN-gamma due to the hyperactivation of T-lymphocytes and defective natural killer cell function. HLH is classified into primary and secondary forms, but mechanisms are intricated, and sometimes secondary forms can be associated with genetic defects. Primary HLH is described in young children, as of poor outcome and hematopoietic stem cell transplantation (HSCT) is the best chance for cure, usually performed after inducing a remission of the disease with chemotherapy and immunosuppression. Secondary HLH in the pediatric population is primarily treated with protocols based on dexamethasone, cyclosporine, and etoposide. In some situations, even secondary HLH cases might need consolidation with HSCT. Refractory and relapsed HLH is always challenging to the pediatric oncologist, as there are no specific second-line therapies and the approach is individualized. Some patients might not be suitable for HSCT, or might not be good candidates for chemotherapy.
Several options are available and targeted therapies are new approaches that are currently highly explored. More and more research are made regarding isolated cases or series of cases that have had a good response after targeted therapies as monotherapies or associated with the chemotherapeutic protocols in both forms, primary or secondary. Therapies target key cytokines, such as IFN gamma, IL-1, IL-6, or the inhibition of JAK1/2-STAT1 pathways. A better understanding of the underlying genetic defects or mechanisms that lead to severe and refractory disease and the implementation of more potent targeted therapies accordingly could improve the survival rate of these patients.
We welcome Original Research, Review, Mini Review and Perspective articles on themes including, but not limited to:
• describing mechanisms or genetic defects that might lead to the refractoriness of the disease
• prove that novel targeted therapies administered as monotherapies or associated with chemotherapy-based protocols or even stem cell transplantation lead to a better outcome in patients who have either severe or refractory disease.
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.
Several options are available and targeted therapies are new approaches that are currently highly explored. More and more research are made regarding isolated cases or series of cases that have had a good response after targeted therapies as monotherapies or associated with the chemotherapeutic protocols in both forms, primary or secondary. Therapies target key cytokines, such as IFN gamma, IL-1, IL-6, or the inhibition of JAK1/2-STAT1 pathways. A better understanding of the underlying genetic defects or mechanisms that lead to severe and refractory disease and the implementation of more potent targeted therapies accordingly could improve the survival rate of these patients.
We welcome Original Research, Review, Mini Review and Perspective articles on themes including, but not limited to:
• describing mechanisms or genetic defects that might lead to the refractoriness of the disease
• prove that novel targeted therapies administered as monotherapies or associated with chemotherapy-based protocols or even stem cell transplantation lead to a better outcome in patients who have either severe or refractory disease.
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
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