Journal of Pediatric Hematology/Oncology最新文献

{"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}

{"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}

{"title":"Adjuvant Etoposide for Very High-Risk PFA Ependymoma: A Case Report.","authors":"Jacob Silverman, Melissa Mégalli, Emilie Giguère, Sarah Elbaz, Melanie Letourneau, Catherine Achim, Cynthia Hawkins, William Legallou, Panagiota Giannakouros, Sébastien Perreault, Geneviève Legault, Eric Bouffet, Nada Jabado, Hallie Coltin, Louis Crevier, Samuele Renzi","doi":"10.1097/MPH.0000000000003080","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003080","url":null,"abstract":"<p><strong>Background: </strong>Ependymomas of the posterior fossa type A (PF-A) with a combined chromosome 1q gain and 6q loss are associated with an extremely high risk of recurrence and a very poor outcome.</p><p><strong>Observations: </strong>We report the case of a 4-year-old girl who received adjuvant oral etoposide for 1 year after conventional treatment (surgery and focal radiation). The patient remains in clinical and radiologic remission 2.5 years post-diagnosis.</p><p><strong>Conclusions: </strong>This approach of using oral etoposide could be considered in ultra-high-risk 6q loss PF-A ependymoma to try and decrease the risk of relapse, awaiting further evaluation in a clinical trial.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144540609","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":"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}

{"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}

{"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}

{"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}

{"title":"Second Malignant Neoplasms in Long-term Retinoblastoma Survivors: Retrospective Cohort Study of 491 Patients in Turkey.","authors":"Eren Müngen, İrem Koç, Hayyam Kiratli, Ali Varan","doi":"10.1097/MPH.0000000000003039","DOIUrl":"10.1097/MPH.0000000000003039","url":null,"abstract":"<p><strong>Background: </strong>It has been reported that the risk of second malignant neoplasms (SMNs) in long-term follow-up patients with heritable retinoblastoma (Rb) is significantly increased compared with patients with non-heritable Rb and the general population. In this study, we investigated the types, frequencies, clinical and pathologic features, potential risk factors, and outcomes of SMNs occurring in a large group of retinoblastoma patients that were diagnosed, treated, and followed up for a long time in our Pediatric Oncology unit.</p><p><strong>Methods: </strong>Our study comprehensively analyzed records of Rb patients followed up at Hacettepe University Pediatric Oncology Department over a 51-year period from January 1972 to January 2023. We determined the number, rate, and time of diagnosis of various SMN types and investigated potential risk factors that could lead to the development of SMNs.</p><p><strong>Results: </strong>A total of 491 patients were included in this study. Median age at the time of retinoblastoma diagnosis was 1.25 (range, 0.02 to 12.08) years. Of these cases, 313 (63.7%) were unilateral, 174 (35.4%) were bilateral, and 4 (0.9%) were trilateral Rb. Enucleation was performed in 348 (70.9%) cases. A total of 334 cases received systemic chemotherapy with different protocols. Intra-arterial chemotherapy (IAC) was administered in 101 (20.6%) patients. Radiotherapy was administered in 76 (15.5%) patients. After enucleation, 56 (11.4%) patients were followed up without further treatment. SMNs occurred in 13 (2.6%) of 491 patients. Among these, 9 (69.2%) patients were considered as having heritable Rb. Enucleation was performed in 10 cases. Only 1 patient received radiotherapy and 12 patients received systemic chemotherapy. Most common subtype of SMNs was osteosarcoma (n=7; 53.8%), followed by acute myeloid leukemia (AML) (n=3; 23.1%), acute lymphoblastic leukemia (ALL) (n=1; 7.7%), Wilms tumor (n=1; 7.7%), and colon adenocarcinoma (n=1; 7.7%). The median time from the diagnosis of Rb to the onset of SMN was 136 (range, 24 to 250) months. Among the patients that underwent IAC, no patient developed SMN. Of the 491 patients, 41 (8.3%) died and 450 are still alive. Of the 13 patients with SMN, 8 (61.5%) died and 5 are still alive. Five-year OS of our study group was 91% and it was significantly lower in patients with SMNs compared with those without SMNs ( P =0.001).</p><p><strong>Conclusion: </strong>In this study, overall survival (OS) was lower in patients with bilateral retinoblastoma compared with unilateral cases, and similarly reduced in patients who received radiotherapy compared with those who did not. In addition, the development of second malignant neoplasms (SMNs) was significantly higher in hereditary retinoblastoma patients than in non-hereditary cases. These findings highlight the importance of careful long-term monitoring and tailored follow-up strategies in patients at increased risk.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e161-e167"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144004834","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":"Agrobacterium radiobacter -induced Port Catheter Bacteremia in a Pediatric Acute Lymphoblastic Leukemia Patient: A Rare Clinical Case.","authors":"Fatma Burçin Kurtipek, Dilek Kaçar, Bedia Dinç, Aslinur Özkaya Parlakay, Neşe Yarali","doi":"10.1097/MPH.0000000000003045","DOIUrl":"10.1097/MPH.0000000000003045","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"260-261"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144031467","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":"Severe Hypertriglyceridemia Secondary to Long-Acting Asparaginase in Pediatric Patients With Acute Lymphoblastic Leukemia.","authors":"Amr Elgehiny, Alexander Hsu, David McCall, Amber Gibson, Branko Cuglievan, Cesar Nunez, Miriam B Garcia, Luz Castellanos, Alexander Funck, Shehla Razvi","doi":"10.1097/MPH.0000000000003077","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003077","url":null,"abstract":"<p><p>Pegylated asparaginase is now standard in US treatment protocols for acute lymphoblastic leukemia (ALL). However, they are associated with significant side effects, including severe hypertriglyceridemia. In this case series, we report 8 patients with severe (triglyceride >1000 mg/dL) hypertriglyceridemia after receiving long-acting asparaginase for ALL and describe their clinical course. The 8 patients included 3 females and 5 males (aged 2 to 14 y; median=12 y); 7 were Hispanic and 1 was Middle Eastern. The median time from dose to peak hypertriglyceridemia was 17 days and to resolution was 25 days. Presentations included isolated hypertriglyceridemia, pseudohyponatremia, hypoglycemia, and lipemia interfering with complete blood count results. Median length of hospitalization was 3.5 days. Management included hydration, a low-fat diet, omega-3 supplements, fenofibrates, statins, and levocarnitine. An insulin drip was used in 2 patients in the intensive care unit. Asparaginase treatment continued per protocol after triglyceride levels were <1000 mg/dL. In conclusion, severe hypertriglyceridemia can occur after long-acting asparaginase and is typically asymptomatic and transient, not requiring a pause or modification in treatment. We recommend monitoring for hypertriglyceridemia closely in patients with risk factors who are resuming long-acting asparaginase therapy after triglyceride levels fall <1000 mg/dL.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2025-06-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144497382","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}