Ryan Wang, Shervin Pejhan, Qi Zhang, Robert Siddaway, Cynthia Hawkins, S Danielle MacNeil, Glenn Bauman, Sandrine de Ribaupierre, Chantel Cacciotti
{"title":"Hybrid Treatment Approach for a Rare Middle Cranial Fossa Intracranial Tumor in a Pediatric Patient: A Case Report.","authors":"Ryan Wang, Shervin Pejhan, Qi Zhang, Robert Siddaway, Cynthia Hawkins, S Danielle MacNeil, Glenn Bauman, Sandrine de Ribaupierre, Chantel Cacciotti","doi":"10.1097/MPH.0000000000003049","DOIUrl":"10.1097/MPH.0000000000003049","url":null,"abstract":"<p><p>Pediatric central nervous system (CNS) tumors are often classified by distinct histologic and molecular features; however, some tumors remain unclassified, resulting in diagnostic and therapeutic challenges. We report a case of a previously healthy 3-year-old female who presented with right eyelid ptosis and headache. Imaging revealed a right middle cranial fossa mass. Following surgery and histopathologic and molecular analyses, the diagnosis was a malignant neoplasm with mixed neural and myoblastic differentiation, not elsewhere classified based on the current World Health Organization (WHO) classification. We describe a unique hybrid treatment approach for this rare tumor consisting of rhabdomyosarcoma and embryonal treatment regimens.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e203-e208"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144015835","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Optimizing Outcomes in Childhood Mature B-cell Non-Hodgkin Lymphoma: Insights Into Staging, Risk Stratification, and Response Evaluation.","authors":"Ali H Algiraigri","doi":"10.1097/MPH.0000000000003046","DOIUrl":"10.1097/MPH.0000000000003046","url":null,"abstract":"<p><p>Staging and risk stratification are critical components in optimizing treatment for pediatric mature B-cell non-Hodgkin lymphoma (B-NHL), an aggressive malignancy with high cure rates when appropriately managed. This review examines contemporary approaches to staging and risk classification through case-based scenarios, emphasizing the practical and patient-centered applications of recent advancements in diagnostic imaging, risk assessment, and biochemical profiling. Through detailed clinical cases, we examine the role of PET-CT and other imaging modalities in accurate staging, outline key criteria for risk categorization, and highlight factors such as tumor burden, central nervous system (CNS) involvement, and early treatment response that influence therapeutic intensity. We also discuss the integration of early response assessments, including PET scans, within current response-adapted treatment frameworks. By illustrating the nuances of individualized staging and risk stratification across diverse patient presentations, this review offers clinicians practical, evidence-based guidance to enhance treatment efficacy while minimizing long-term toxicity in pediatric B-NHL.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e138-e143"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144014278","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Gashaw Arega, Syed Ibrahim Bukhari, Sadaf Altaf, Asim Fakhruddin Belagaumi
{"title":"Pediatric Cancer Survivorship Program in Low-income and Middle-income Countries; A Dire Need of Comprehensive Approach to a Healthy Life After Cancer: Perspective and Literature Review.","authors":"Gashaw Arega, Syed Ibrahim Bukhari, Sadaf Altaf, Asim Fakhruddin Belagaumi","doi":"10.1097/MPH.0000000000003044","DOIUrl":"10.1097/MPH.0000000000003044","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e210-e214"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144078679","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Refika S Dokuzboy, Dilek Çetin, Ali U Tuğcu, Şerife S Oğuz
{"title":"Discovering a Rare Connection: Hereditary Thrombotic Thrombocytopenic Purpura and Large Placental Lakes.","authors":"Refika S Dokuzboy, Dilek Çetin, Ali U Tuğcu, Şerife S Oğuz","doi":"10.1097/MPH.0000000000003053","DOIUrl":"10.1097/MPH.0000000000003053","url":null,"abstract":"<p><p>Hereditary thrombotic thrombocytopenic purpura (hTTP) is a rare genetic disorder caused by mutations in the ADAMTS13 (a disintegrin-like and metalloprotease with thrombospondin type 1 motif, member 13) gene, leading to deficient or absent ADAMTS13 activity. Without ADAMTS13, ultralarge von Willebrand factor (ULVWF) molecules are not properly cleaved, resulting in the formation of platelet-rich thrombi, platelet consumption, organ ischemia, and microangiopathic hemolytic anemia. We report a female newborn who presented with respiratory distress, jaundice, anemia, and thrombocytopenia. Prenatal ultrasonography revealed a large placental lake. She was diagnosed with hTTP and successfully treated with fresh frozen plasma (FFP) transfusion. Genetic analysis revealed a pathogenic homozygous mutation in the ADAMTS13 gene. To our knowledge, this report is the first to document large placental lakes in a newborn with hTTP, suggesting a potential link between fetal ADAMTS13 deficiency and abnormal placentation.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"246-249"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144159732","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Esther E Knapp, Jennifer Hall, Alyssa A Middleton, Carla A Rich, Kyle B Brothers
{"title":"Genomic Sequencing in Pediatric Oncology: Perspectives of Parents and Adolescents on Ethical Issues.","authors":"Esther E Knapp, Jennifer Hall, Alyssa A Middleton, Carla A Rich, Kyle B Brothers","doi":"10.1097/MPH.0000000000003048","DOIUrl":"10.1097/MPH.0000000000003048","url":null,"abstract":"<p><p>This study aimed to elicit the perspectives of adolescents with cancer, and parents of children with cancer regarding tumor and germline genomic sequencing in pediatric oncology. Semistructured interviews were conducted with adolescents and parents/guardians of children with cancer. The interview guide included potential benefits of genomic sequencing, heritable cancer predisposition conditions, impacts on family relationships, and secondary findings. Participants reported several ways they believed sequencing results could be valuable, including more targeted therapy and knowledge regarding heritable variants. However, opinions varied on what information would be useful, with many participants reporting an interest only in those results that would directly impact therapy. Several parents were inclined to feel guilty about their children having cancer, whether genetically linked or not. Adolescent patients tended to be protective towards their parents and generally felt they would not blame their parents, even for an inherited condition. Many participants felt they lacked the knowledge to make a completely informed decision on results derived from complex scientific technology. These findings will help inform the process of obtaining informed consent for genomic sequencing in children with cancer.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"224-229"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144102267","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Farrah Gaston, Blase Polite, Steven Ullenius, Sean Fitzgerald, Madhuri Bajaj
{"title":"Sporadic Mucinous Adenocarcinoma of the Colon in an Adolescent Male: A Case Report and Review of the Literature.","authors":"Farrah Gaston, Blase Polite, Steven Ullenius, Sean Fitzgerald, Madhuri Bajaj","doi":"10.1097/MPH.0000000000003031","DOIUrl":"10.1097/MPH.0000000000003031","url":null,"abstract":"<p><p>Colorectal cancer (CRC) is commonly seen in adult patients but makes up <1% of cancers in pediatric patients, with the largest known pediatric study having a sample size of 81 patients taken over a span of roughly 40 years. Patients typically present with nonspecific symptoms such as abdominal pain, nausea, and vomiting, which can result in the diagnosis being missed. Histopathology is typically unfavorable with signet ring colon cancer and mucinous adenocarcinoma with signet ring cell features being common. Treatment guidelines are based largely on adult protocols and include surgical resection followed by chemotherapy, with the addition of bevacizumab or cetuximab in higher-risk populations. In our case, we present a 17-year-old male with abdominal pain and weight loss who was found to have metastatic sporadic mucinous adenocarcinoma with signet ring cell features. Oncotype next-generation sequencing was found to be microsatellite stable, human epithelial growth factor receptor 2 negative, Kristen rat sarcoma viral oncogene homolog/v-raf murine sarcoma viral oncogene homolog B1/neuroblastoma reticular activating system wild type, and low tumor mutation burden with 3.7 mutations per megabase. He was treated with FOLFIRINOX at 70% dosing without bevacizumab due to recurrent admissions for small bowel obstructions and surgeries. Unfortunately, his cancer did not respond, so he proceeded with palliative chemotherapy. He expired 12 months later. This case raises the need for research on the biology of CRC in pediatric patients, as it is different from adults. It also underlines the necessity of the development of guidelines on CRC treatment in pediatric patients by initiating pediatric clinical trials or lowering the age of adult trials to under 18 years of age.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"219-223"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144015842","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Mahmoud M Elzembely, Yousra Sayed, Heba Abdel-Razik Sayed
{"title":"Compliance of Pediatric Cancer Survivors to Attend Long-term Follow-up Clinic in Tertiary Cancer Center in Egypt: A Retrospective Study.","authors":"Mahmoud M Elzembely, Yousra Sayed, Heba Abdel-Razik Sayed","doi":"10.1097/MPH.0000000000003047","DOIUrl":"10.1097/MPH.0000000000003047","url":null,"abstract":"<p><strong>Background: </strong>Current treatment protocols have significantly improved the 5-year survival rate for childhood cancers. Our study aims to describe the adherence of survivors in Egypt to transition from oncology care to survivorship care and nonadherence risk factors.</p><p><strong>Methods: </strong>Medical charts of patients in the Pediatric Long-Term Follow-Up (LTFU) Clinic at South Egypt Cancer Institute, Assiut University, were reviewed from 2015 to 2022 and compared with patients who finished treatment between 2014 and 2021 and did not attend the LTFU clinic. Records were reviewed for the number of LTFU clinic attendants, demographic data, logistic data, and medical factors.</p><p><strong>Results: </strong>We found that LTFU clinic attendants during the study period were 405 (38.6%) of 1050 finished treatment from 2014 to 2021. Median age of survivors was 8 years (8 mo to 22 y). LTFU attendants included 285 (70.4%) with hematologic malignancies. Educated parents were 283 (70%), with 44 (15.7%) having university degrees. Multivariate logistic regression showed that older age, unemployed parents, and living >100 km from the hospital were significantly related to nonattendance at the LTFU clinic.</p><p><strong>Conclusion: </strong>We concluded that programs need to be established in developing countries to improve survivors' adherence to transitioning from oncology care to survivorship care.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"230-234"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144248388","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Ursula Tanriver, Angelina Hutter, Malte Kohns, Nicolas von der Weid, Alexandra Schifferli
{"title":"Invasive Fungal Infections in Pediatric Hematology-Oncology: A 12-Year Single-center Observational Study and the Need for a Clinical Classification System.","authors":"Ursula Tanriver, Angelina Hutter, Malte Kohns, Nicolas von der Weid, Alexandra Schifferli","doi":"10.1097/MPH.0000000000003035","DOIUrl":"10.1097/MPH.0000000000003035","url":null,"abstract":"<p><p>Pediatric patients undergoing chemotherapy or allogeneic hematopoietic stem cell transplantation are at high risk for invasive fungal infections (IFIs). Diagnosing IFIs is challenging due to nonspecific clinical presentations, invasiveness of diagnostic procedures, and the low sensitivity of microbiological assays. This retrospective 12-year observational study examines incidence, diagnostic challenges, fungal pathogens, and course of IFI cases in pediatric patients with hematologic malignancies or receiving allogeneic hematopoietic stem cell transplantation at the University Children's Hospital of Basel. Of the 163 patients reviewed, 39 (23.9%) received antifungal therapy based on clinical suspicion of IFI. Applying the EORTC/MSGERC diagnostic classification system, only 19 cases (11.6% cumulative incidence) were confirmed as proven or probable IFIs. Within this subgroup, a mortality rate of 21% underscores the severe consequences associated with advanced disease and profound immunosuppression. In addition, we introduce a novel clinical classification system to complement the EORTC/MSGERC criteria, with the aim of enhancing early diagnosis and risk stratification in this high-risk population. Our findings highlight the diagnostic challenges in pediatric patients, including the low specificity of radiologic findings and the limited utility of microbiological tests in guiding treatment decisions. Addressing these gaps is crucial for improving outcomes and advancing care for children with IFIs.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e192-e198"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143988098","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Khadija Belcadi, Zineb Isfaoun, Naoual El Ansari, Meryem Lakhrissi, Naoufal Fejjal, Maria El Kababri, Mohamed El Khorassani, Laila Hessissen, Amina Kili
{"title":"Hyperbaric Oxygen Therapy in Doxorubicin Extravasation: A Promising Future Treatment?: A Case Report.","authors":"Khadija Belcadi, Zineb Isfaoun, Naoual El Ansari, Meryem Lakhrissi, Naoufal Fejjal, Maria El Kababri, Mohamed El Khorassani, Laila Hessissen, Amina Kili","doi":"10.1097/MPH.0000000000003038","DOIUrl":"10.1097/MPH.0000000000003038","url":null,"abstract":"<p><p>Doxorubicin extravasation is a serious complication that can lead to severe local necrosis, aesthetic damage, and injury to underlying nerves, tendons, and muscles. Its management has been the subject of numerous studies, but the optimal treatment remains controversial. The limited access to dexrazoxane makes the management even more challenging. We report the case of a 17-month-old infant treated for an alveolar rhabdomyosarcoma of the soft tissues in the gluteal region, who experienced doxorubicin extravasation in the forearm. This incident was successfully managed with initial fasciotomy followed by hyperbaric oxygen therapy. We highlight the beneficial effects of hyperbaric oxygen therapy as a complementary treatment in cases of doxorubicin extravasation, supported by current data from the literature.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e177-e180"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144015327","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}