Pub Date : 2026-01-23DOI: 10.1016/j.phoj.2025.100801
Carlos Tourinho Lapa Filho , Mônica Pinheiro de Almeida Verissimo , Thais Novaes Ferreira
Hemoglobin SC Disease is among the main hemoglobinopathies related to sickle cell anemia (only surpassed in prevalence by Hemoglobin SS Disease). Although considered a pathology of lesser severity and complications, information on its clinical manifestations seem to lack support in consistent studies. In the pediatric population, data is even more scarce. The present study carried out an integrative review of the clinical manifestations reported in pediatric patients with hemoglobin SC disease, with their age of occurrence, the most prevalent manifestations, evolution of pathology and associated morbidity. The articles analyzed were able to provide guidance on the issues examined, but the data lacked consistency. New and greater number of prospective studies are necessary to increase the level of evidence for the clinical findings presented in this entity.
{"title":"Hemoglobin SC disease and its clinical manifestations in pediatric patients: where is the data?","authors":"Carlos Tourinho Lapa Filho , Mônica Pinheiro de Almeida Verissimo , Thais Novaes Ferreira","doi":"10.1016/j.phoj.2025.100801","DOIUrl":"10.1016/j.phoj.2025.100801","url":null,"abstract":"<div><div>Hemoglobin SC Disease is among the main hemoglobinopathies related to sickle cell anemia (only surpassed in prevalence by Hemoglobin SS Disease). Although considered a pathology of lesser severity and complications, information on its clinical manifestations seem to lack support in consistent studies. In the pediatric population, data is even more scarce. The present study carried out an integrative review of the clinical manifestations reported in pediatric patients with hemoglobin SC disease, with their age of occurrence, the most prevalent manifestations, evolution of pathology and associated morbidity. The articles analyzed were able to provide guidance on the issues examined, but the data lacked consistency. New and greater number of prospective studies are necessary to increase the level of evidence for the clinical findings presented in this entity.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"11 1","pages":"Article 100801"},"PeriodicalIF":0.0,"publicationDate":"2026-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146079063","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Somatic malignant transformation (SMT) is defined as the development of non germ cell malignant neoplasm within a teratoma. Malignant change can occur in any of the various components of the three germ cell layers. In adults, more than 80 % of all malignant transformations are squamous cell carcinoma, the rest being adenocarcinoma and carcinoid tumor. In children, SMT are very uncommon and reports are limited.
We report a case series of three children who presented with SMT of a teratoma. We also undertook a literature search of published reports on SMT in children.
{"title":"Somatic malignant transformation of teratoma in children and adolescents - Case series with literature review","authors":"Yamini Krishnan , Smitha Bhaskaran , Venma Jojo , Pavitra Subramanian , Gazel S. , Krishnan V.P.","doi":"10.1016/j.phoj.2025.100800","DOIUrl":"10.1016/j.phoj.2025.100800","url":null,"abstract":"<div><div>Somatic malignant transformation (SMT) is defined as the development of non germ cell malignant neoplasm within a teratoma. Malignant change can occur in any of the various components of the three germ cell layers. In adults, more than 80 % of all malignant transformations are squamous cell carcinoma, the rest being adenocarcinoma and carcinoid tumor. In children, SMT are very uncommon and reports are limited.</div><div>We report a case series of three children who presented with SMT of a teratoma. We also undertook a literature search of published reports on SMT in children.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"11 1","pages":"Article 100800"},"PeriodicalIF":0.0,"publicationDate":"2026-01-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145980995","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2026-01-09DOI: 10.1016/j.phoj.2026.100803
S. Suganya , M. Mohammed Shakeel , Jaswanthini , Suganth , Karthik Kumar , S. Kalpana , Hemachitra
Background
Acute lymphoblastic leukemia (ALL) is the most common childhood cancer. With improved risk stratification and MRD-guided therapy, the overall prognosis of ALL is more than 90 %. Nevertheless, Induction mortality remains a significant clinical concern in low- and middle-income countries (LMIC), with invasive fungal infection (IFI) of increasing concern.
Case report
We present a case of a 9-year-old boy with B-cell acute lymphoblastic leukemia (B-ALL) who developed persistent fever due to IFI during induction chemotherapy. Despite hematologic remission, he developed IFI of the kidneys with subacute thyroiditis suspicious for disseminated infection, necessitating modification of chemotherapy and prolonged antifungal therapy.
Conclusion
This case highlights the diagnostic challenges in IFI management in pediatric leukemia in LMIC.
{"title":"Cryptococcosis in B-cell acute lymphoblastic leukemia with suspected dissemination: A case report","authors":"S. Suganya , M. Mohammed Shakeel , Jaswanthini , Suganth , Karthik Kumar , S. Kalpana , Hemachitra","doi":"10.1016/j.phoj.2026.100803","DOIUrl":"10.1016/j.phoj.2026.100803","url":null,"abstract":"<div><h3>Background</h3><div>Acute lymphoblastic leukemia (ALL) is the most common childhood cancer. With improved risk stratification and MRD-guided therapy, the overall prognosis of ALL is more than 90 %. Nevertheless, Induction mortality remains a significant clinical concern in low- and middle-income countries (LMIC), with invasive fungal infection (IFI) of increasing concern.</div></div><div><h3>Case report</h3><div>We present a case of a 9-year-old boy with B-cell acute lymphoblastic leukemia (B-ALL) who developed persistent fever due to IFI during induction chemotherapy. Despite hematologic remission, he developed IFI of the kidneys with subacute thyroiditis suspicious for disseminated infection, necessitating modification of chemotherapy and prolonged antifungal therapy.</div></div><div><h3>Conclusion</h3><div>This case highlights the diagnostic challenges in IFI management in pediatric leukemia in LMIC.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"11 1","pages":"Article 100803"},"PeriodicalIF":0.0,"publicationDate":"2026-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145928648","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2026-01-08DOI: 10.1016/j.phoj.2025.100802
Elpis Mantadakis, Sonia Alexiadou
Primary hemophagocytic lymphohistiocytosis (pHLH) is a fatal, autosomal recessive hyperinflammatory syndrome. Infections, neoplasia, and underlying autoimmune and autoinflammatory conditions trigger secondary HLH. In pHLH, immunosuppression with dexamethasone and etoposide has been used as the standard of care before hematopoietic transplantation, the only curative option. However, approximately one-third of the patients do not achieve remission and die before transplantation. As a result, the focus has shifted to novel biologic agents and we discuss these here. Emapalumab (Gamifant®, a monoclonal antibody that neutralizes free and receptor-bound interferon-γ), has been approved in the US for HLH since 2020 based on its efficacy in 34 patients. The European Medicines Agency has refused marketing authorization, expressing concerns regarding its efficacy, since most responding patients required additional therapy. As the JAK/STAT pathway is critical for cytokine signaling and has a significant role in HLH, its suppression by ruxolitinib (Jakafi®, a potent anti-inflammatory inhibitor) has been successful in animal models of HLH. Over the last 6 years, several clinical studies, mainly from China, have studied ruxolitinib's use in HLH. All have shown ruxolitinib to be safe and effective, minimizing or avoiding exposure to chemotherapy. Beyond emapalumab and ruxolitinib, other agents have shown promise in managing aspects of HLH, including interleukin-1 receptor antagonists, such as anakinra, tocilizumab (Actemra®, a recombinant humanized monoclonal antibody against the interleukin-6 receptor), alemtuzumab (Campath® and Lemtrada®, a monoclonal antibody against CD52), and finally, tadekinig alpha, a recombinant interleukin-18 binding protein with a high affinity for IL-18, a major inflammatory cytokine. In summary, a growing number of biological agents are expected to play a key role in the treatment of HLH in the immediate future.
{"title":"An update in the diagnosis and therapy of hemophagocytic lymphohistiocytosis","authors":"Elpis Mantadakis, Sonia Alexiadou","doi":"10.1016/j.phoj.2025.100802","DOIUrl":"10.1016/j.phoj.2025.100802","url":null,"abstract":"<div><div>Primary hemophagocytic lymphohistiocytosis (pHLH) is a fatal, autosomal recessive hyperinflammatory syndrome. Infections, neoplasia, and underlying autoimmune and autoinflammatory conditions trigger secondary HLH. In pHLH, immunosuppression with dexamethasone and etoposide has been used as the standard of care before hematopoietic transplantation, the only curative option. However, approximately one-third of the patients do not achieve remission and die before transplantation. As a result, the focus has shifted to novel biologic agents and we discuss these here. Emapalumab (Gamifant®, a monoclonal antibody that neutralizes free and receptor-bound interferon-γ), has been approved in the US for HLH since 2020 based on its efficacy in 34 patients. The European Medicines Agency has refused marketing authorization, expressing concerns regarding its efficacy, since most responding patients required additional therapy. As the JAK/STAT pathway is critical for cytokine signaling and has a significant role in HLH, its suppression by ruxolitinib (Jakafi®, a potent anti-inflammatory inhibitor) has been successful in animal models of HLH. Over the last 6 years, several clinical studies, mainly from China, have studied ruxolitinib's use in HLH. All have shown ruxolitinib to be safe and effective, minimizing or avoiding exposure to chemotherapy. Beyond emapalumab and ruxolitinib, other agents have shown promise in managing aspects of HLH, including interleukin-1 receptor antagonists, such as anakinra, tocilizumab (Actemra®, a recombinant humanized monoclonal antibody against the interleukin-6 receptor), alemtuzumab (Campath® and Lemtrada®, a monoclonal antibody against CD52), and finally, tadekinig alpha, a recombinant interleukin-18 binding protein with a high affinity for IL-18, a major inflammatory cytokine. In summary, a growing number of biological agents are expected to play a key role in the treatment of HLH in the immediate future.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"11 1","pages":"Article 100802"},"PeriodicalIF":0.0,"publicationDate":"2026-01-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145908861","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
The aim of the study was to analyze the profile and outcomes of children with acute myeloid leukemia (AML) who underwent hematopoietic stem cell transplantation (HSCT) in the last 20 years at our center.
Patients and methods
A retrospective cross-sectional analysis was performed on children between 0 and 17.99 years of age with AML who underwent HSCT from January 2002 and December 2021 at Apollo Hospital in Chennai, South India, with a minimum follow up of 3 years.
Results
Fifty-five children underwent HSCT with a diagnosis of AML and high risk cytogenetics, or for those with favorable cytogenetics: MRD positivity post induction in 29/55 (52.7 %), relapsed AML in 15/55 (27.3 %) and refractory AML in 11/55 (20 %). Twenty-one of the 55 children underwent haploidentical HSCT (38 %), 16/55 (29 %) matched sibling donor (MSD) HSCT and 18/55 (32 %) matched unrelated donor (MUD) HSCT, with engraftment in 51/55 (92.7 %) children. Overall survival was 37/55 (67.1 %) at the end of 3 years, 15/21 (71 %) patients with haploidentical HSCT, 13/18 (72 %) with MUD HSCT, and 9/16 (56 %) with MSD HSCT (p value = 0.002). Relapse was documented in 4/21 (19 %) patients with haplo-HSCT, 3/18 (16 %) with MUD HSCT and 3/16 (18 %) with MSD HSCT, with an overall relapse rate of 10/55 (18 %). Relapse rates were 2/23 (8 %) among those with chronic GVHD versus 8/32 (25 %) among those without (p value = 0.01).
Conclusion
The outcomes in our cohort were promising in high risk, relapsed and refractory AML post-HSCT with an overall survival of 67.1 %. Alternate donor transplants are equally efficacious and have reliable outcomes in terms of survival. Chronic GVHD did offer protection against relapse. Potential limitations of the study were the relatively short follow up, and selection bias in patients selected for HSCT.
{"title":"Twenty-years of hematopoietic stem cell transplantation in childhood acute myeloid leukemia: a single-center study from South India","authors":"Anuraag Reddy Nalla, Aarathi Viswanathan, Kavitha Ganesan, Anupama Nair, Vijayshree Muthukumar, Minakshi Balwani, Nithya Seshadri, Ramya Uppuluri, Revathi Raj","doi":"10.1016/j.phoj.2025.100478","DOIUrl":"10.1016/j.phoj.2025.100478","url":null,"abstract":"<div><h3>Background</h3><div>The aim of the study was to analyze the profile and outcomes of children with acute myeloid leukemia (AML) who underwent hematopoietic stem cell transplantation (HSCT) in the last 20 years at our center.</div></div><div><h3>Patients and methods</h3><div>A retrospective cross-sectional analysis was performed on children between 0 and 17.99 years of age with AML who underwent HSCT from January 2002 and December 2021 at Apollo Hospital in Chennai, South India, with a minimum follow up of 3 years.</div></div><div><h3>Results</h3><div>Fifty-five children underwent HSCT with a diagnosis of AML and high risk cytogenetics, or for those with favorable cytogenetics: MRD positivity post induction in 29/55 (52.7 %), relapsed AML in 15/55 (27.3 %) and refractory AML in 11/55 (20 %). Twenty-one of the 55 children underwent haploidentical HSCT (38 %), 16/55 (29 %) matched sibling donor (MSD) HSCT and 18/55 (32 %) matched unrelated donor (MUD) HSCT, with engraftment in 51/55 (92.7 %) children. Overall survival was 37/55 (67.1 %) at the end of 3 years, 15/21 (71 %) patients with haploidentical HSCT, 13/18 (72 %) with MUD HSCT, and 9/16 (56 %) with MSD HSCT (p value = 0.002). Relapse was documented in 4/21 (19 %) patients with haplo-HSCT, 3/18 (16 %) with MUD HSCT and 3/16 (18 %) with MSD HSCT, with an overall relapse rate of 10/55 (18 %). Relapse rates were 2/23 (8 %) among those with chronic GVHD versus 8/32 (25 %) among those without (p value = 0.01).</div></div><div><h3>Conclusion</h3><div>The outcomes in our cohort were promising in high risk, relapsed and refractory AML post-HSCT with an overall survival of 67.1 %. Alternate donor transplants are equally efficacious and have reliable outcomes in terms of survival. Chronic GVHD did offer protection against relapse. Potential limitations of the study were the relatively short follow up, and selection bias in patients selected for HSCT.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 4","pages":"Article 100478"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145839265","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-12-01DOI: 10.1016/j.phoj.2025.100790
{"title":"Erratum regarding incorrect article type for previously published articles","authors":"","doi":"10.1016/j.phoj.2025.100790","DOIUrl":"10.1016/j.phoj.2025.100790","url":null,"abstract":"","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 4","pages":"Article 100790"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145839267","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-12-01DOI: 10.1016/j.phoj.2025.100788
Martin Sterba , Petra Pokorna , Michal Kyr , Tomas Merta , Katerina Jendrisakova , Sarka Kozakova , Ondrej Slaby , Peter Mudry , Jaroslav Sterba
Background
We present a case of a 12-month-old female patient with metastatic neuroblastoma who was transferred to our institution after disease progression following two cycles of standard treatment in Ukraine. Upon admission, the child was critically ill, presenting with a large abdominal mass, severe cachexia, deep vein thrombosis with limb oedema, and oxygen dependency due to limited chest excursions.
Case report
After initial stabilization, comprehensive genomic profiling revealed an SRGAP3:NTRK2 gene fusion in the tumor sample. An individualized combination of standard chemotherapy, targeted treatment with larotrectinib, and metronomic chemotherapy was administered. This regimen resulted in a radiologically confirmed partial response and a notable clinical improvement.
Conclusion
This case documents a rare instance of NTRK2 fusion-driven neuroblastoma successfully treated with a regimen containing a pan-TRK inhibitor that proved to be well-tolerated and effective.
{"title":"NTRK2 fusion-driven neuroblastoma treated with targeted therapy: A case report","authors":"Martin Sterba , Petra Pokorna , Michal Kyr , Tomas Merta , Katerina Jendrisakova , Sarka Kozakova , Ondrej Slaby , Peter Mudry , Jaroslav Sterba","doi":"10.1016/j.phoj.2025.100788","DOIUrl":"10.1016/j.phoj.2025.100788","url":null,"abstract":"<div><h3>Background</h3><div>We present a case of a 12-month-old female patient with metastatic neuroblastoma who was transferred to our institution after disease progression following two cycles of standard treatment in Ukraine. Upon admission, the child was critically ill, presenting with a large abdominal mass, severe cachexia, deep vein thrombosis with limb oedema, and oxygen dependency due to limited chest excursions.</div></div><div><h3>Case report</h3><div>After initial stabilization, comprehensive genomic profiling revealed an <em>SRGAP3</em>:<em>NTRK2</em> gene fusion in the tumor sample. An individualized combination of standard chemotherapy, targeted treatment with larotrectinib, and metronomic chemotherapy was administered. This regimen resulted in a radiologically confirmed partial response and a notable clinical improvement.</div></div><div><h3>Conclusion</h3><div>This case documents a rare instance of <em>NTRK2</em> fusion-driven neuroblastoma successfully treated with a regimen containing a pan-TRK inhibitor that proved to be well-tolerated and effective.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 4","pages":"Article 100788"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145693238","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-11-15DOI: 10.1016/j.phoj.2025.100779
Dana Ashkenazi Lustig , Shifra Ash , Myriam Ben-Arush
Background/objectives
Langerhans cell histiocytosis (LCH) is a rare pediatric neoplasm frequently involving bone. While various therapeutic strategies exist, including surgery and systemic therapy, some lesions—particularly unifocal bone lesions—have demonstrated a capacity for spontaneous healing. This review explores the clinical characteristics, proposed mechanisms, and therapeutic implications of self-healing in pediatric LCH bone lesions.
Methods
A systematic literature search was conducted in March 2025 using PubMed, Google Scholar, and SciSpace databases. Studies were selected following PRISMA guidelines, with inclusion criteria focusing on pediatric patients with LCH presenting bone involvement, particularly those demonstrating spontaneous regression post-biopsy, curettage, or conservative management. Data on clinical presentation, treatment approaches, and proposed pathophysiological mechanisms were extracted and analysed.
Results
Spontaneous resolution of bone lesions was most commonly observed in unifocal eosinophilic granuloma. Approximately 5–10 % of pediatric LCH cases demonstrated lesion regression post-biopsy or after minimal intervention. Proposed mechanisms include biopsy-induced inflammation, immune system modulation, apoptosis via the Fas/Fas-L pathway, and disruption of the MAPK signalling cascade—especially in the presence of BRAF V600E mutations. Nanoparticle-based delivery of targeted agents, such as anti-BCL2 or cytokine inhibitors, represents an emerging therapeutic strategy with potential to replicate this natural healing process.
Conclusions
Spontaneous healing is a clinically significant but underreported aspect of pediatric LCH, with implications for reducing invasive treatment. Understanding the molecular and immunological drivers of lesion regression may guide future personalized therapies. Further research into targeted drug delivery and apoptosis modulation could shift the therapeutic paradigm, offering fewer toxic alternatives to systemic chemotherapy in select patients.
{"title":"Pediatric Langerhans Cell Histiocytosis of Bone: A systematic review of the unknown mechanism of self-healing","authors":"Dana Ashkenazi Lustig , Shifra Ash , Myriam Ben-Arush","doi":"10.1016/j.phoj.2025.100779","DOIUrl":"10.1016/j.phoj.2025.100779","url":null,"abstract":"<div><h3>Background/objectives</h3><div>Langerhans cell histiocytosis (LCH) is a rare pediatric neoplasm frequently involving bone. While various therapeutic strategies exist, including surgery and systemic therapy, some lesions—particularly unifocal bone lesions—have demonstrated a capacity for spontaneous healing. This review explores the clinical characteristics, proposed mechanisms, and therapeutic implications of self-healing in pediatric LCH bone lesions.</div></div><div><h3>Methods</h3><div>A systematic literature search was conducted in March 2025 using PubMed, Google Scholar, and SciSpace databases. Studies were selected following PRISMA guidelines, with inclusion criteria focusing on pediatric patients with LCH presenting bone involvement, particularly those demonstrating spontaneous regression post-biopsy, curettage, or conservative management. Data on clinical presentation, treatment approaches, and proposed pathophysiological mechanisms were extracted and analysed.</div></div><div><h3>Results</h3><div>Spontaneous resolution of bone lesions was most commonly observed in unifocal eosinophilic granuloma. Approximately 5–10 % of pediatric LCH cases demonstrated lesion regression post-biopsy or after minimal intervention. Proposed mechanisms include biopsy-induced inflammation, immune system modulation, apoptosis via the Fas/Fas-L pathway, and disruption of the MAPK signalling cascade—especially in the presence of BRAF V600E mutations. Nanoparticle-based delivery of targeted agents, such as anti-BCL2 or cytokine inhibitors, represents an emerging therapeutic strategy with potential to replicate this natural healing process.</div></div><div><h3>Conclusions</h3><div>Spontaneous healing is a clinically significant but underreported aspect of pediatric LCH, with implications for reducing invasive treatment. Understanding the molecular and immunological drivers of lesion regression may guide future personalized therapies. Further research into targeted drug delivery and apoptosis modulation could shift the therapeutic paradigm, offering fewer toxic alternatives to systemic chemotherapy in select patients.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 4","pages":"Article 100779"},"PeriodicalIF":0.0,"publicationDate":"2025-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145528765","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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