Journal of Pediatric Hematology/Oncology最新文献

{"title":"Importance of Ambulatory Blood Pressure Monitoring in Childhood Cancer Survivors.","authors":"Ali A Ozguven, Fatos Alkan, Aysen T Yildirim, Yesim Yigit, Huseyin Gulen, Senol Coskun","doi":"10.1097/MPH.0000000000003127","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003127","url":null,"abstract":"<p><p>Hypertension is a significant morbidity among childhood cancer survivors (CCS). Ambulatory blood pressure monitoring (ABPM) allows for continuous 24-hour blood pressure assessment and provides valuable insights into abnormal nocturnal dipping (aND) and ambulatory arterial stiffness index (AASI). This study aimed to evaluate the prevalence of hypertension and prehypertension, as well as, the rates of aND, and AASI in CCS. Ambulatory blood pressure monitoring was performed in 49 patients who had completed cancer treatment at least 1 year prior. Among all patients, the prevalence of hypertension and prehypertension was 10% and 16%, respectively. The solid tumor group demonstrated a significantly higher prevalence of prehypertension, nephrotoxic drug exposure, and cumulative doses of endothelium-toxic agents compared with the hematologic malignancy group. However, no significant difference in hypertension prevalence was observed between the 2 groups. Notably, a diagnosis of Wilms tumor was significantly associated with prehypertension. According to ABPM findings, aND was observed in 61% of patients. The higher prevalence of prehypertension and aND in CCS compared with healthy children, along with the observed association between hypertension and AASI, highlights the importance of ABPM in the long-term cardiovascular monitoring of this population. The increased prevalence of prehypertension in patients with solid tumors may be attributed to the cumulative dosage of drugs used during treatment.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145238819","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":"Unclassified Versus Well-Defined Platelet Function Disorders: A Multicenter Comparison of Bleeding Patterns and Treatment.","authors":"Divyaswathi Citla-Sridhar, Beverly Spray, Robert Sidonio, Michael Silvey, Sanjay Ahuja","doi":"10.1097/MPH.0000000000003134","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003134","url":null,"abstract":"<p><strong>Introduction: </strong>Platelet function disorders (PFDs) are caused by abnormalities in platelet receptors, granules, and signaling pathways. While severe conditions like Glanzmann Thrombasthenia (GT) and Bernard-Soulier Syndrome (BSS) have well-characterized bleeding phenotypes, other PFDs remain less defined. This study aimed to describe the bleeding phenotype in different PFDs at the time of diagnosis and during longitudinal follow-up and compare bleeding symptoms and health care utilization for bleed management.</p><p><strong>Methods: </strong>This retrospective multicenter study analyzed data from 129 patients diagnosed with PFDs at 3 Hemophilia Treatment Centers in the United States from 2015 to 2020. Data included demographics, bleeding symptoms, and treatment utilization. Statistical comparisons of bleeding symptoms, frequency, and treatment across PFDs were performed using the χ2 or the Fisher exact tests.</p><p><strong>Results: </strong>Among 129 patients, 8 had GT, 2 had BSS, 40 had platelet storage pool disorder, 7 had platelet secretion defect, and 72 had PFD not otherwise specified (NOS). Epistaxis was the most common symptom at diagnosis, except in platelet secretion defects, where soft tissue bleeding predominated. Heavy menstrual bleeding affected 31.7% of females. Over a 5-year period, epistaxis remained frequent in GT and PFD NOS. GT had the highest treatment burden, with 86.2% of bleeds requiring treatment. Hospitalizations were significantly greater in GT and platelet secretion defects.</p><p><strong>Conclusion: </strong>Individuals with PFD NOS and platelet secretion defects can experience serious bleeding. Life-threatening bleeds occur in PFDs beyond GT and BSS, necessitating thorough evaluation, close follow-up, and careful perioperative planning. Unclassified platelet disorders require further evaluation along with genetic testing to prevent excessive blood loss.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145238794","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":"Teratoid Wilms Tumor: Report of a Rare Histologic Variant With Review of Literature.","authors":"Anju Verma, Ankur Mandelia, Lily Pal, Tarun Kumar, Nishant Agarwal","doi":"10.1097/MPH.0000000000003132","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003132","url":null,"abstract":"<p><p>Wilms tumor (WT) is the most common pediatric renal neoplasm, and Teratoid Wilms' tumor (TWT) is a rare histologic variant of WT, which consists predominantly of well-differentiated heterologous mesenchymal and/or epithelial elements. We report a case of TWT in a toddler who presented with an incidentally detected abdominal lump. Trucut biopsy was suggestive of WT with rhabdomyomatous differentiation. Six weeks of neoadjuvant chemotherapy was given with minimal response. Radical nephroureterectomy with lymph node sampling was performed and histopathology was suggestive of TWT. The child is asymptomatic and disease-free at follow-up.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145238751","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":"A Rare Case of Plasmacytoma-like Post-transplant Lymphoproliferative Disorder in a Pediatric Hematopoietic Stem Cell Transplant Recipient Treated With Daratumumab.","authors":"Anne-Charlotte Teyssier, Alexandre Quoc Dinh Nguyen, Dorothée Dal Soglio, Sophie Turpin, Sonia Cellot, Michel Duval, Pierre Teira, Henrique Bittencourt","doi":"10.1097/MPH.0000000000003120","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003120","url":null,"abstract":"<p><p>Post-transplant lymphoproliferative disorder (PTLD) is a complication of immunosuppressive therapy following solid organ transplantation (SOT) and hematopoietic stem cell transplantation (HSCT). Although PLTD usually presents as B-cell proliferation, plasmacytoma-like PTLD, a rare subtype of monomorphic PTLD, has been described, mostly in SOT recipients. Only 2 cases of this disease entity have been previously reported in patients after HSCT. While the treatment of choice for PTLD is the reduction of immunosuppression combined with rituximab (anti-CD20 monoclonal antibody), the optimal treatment for PTLD with plasmacellular differentiation, which is often CD20-negative, is unknown. We present a case of monomorphic plasmacytoma-like PTLD in a child who received an allogeneic HSCT for relapsed acute lymphoblastic leukemia. He was successfully treated with a myeloma-based approach using an anti-CD38 monoclonal antibody, daratumumab.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145238579","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":"Congenital Undifferentiated Retroperitoneal Sarcoma With YWHAE::NUTM2B Fusion From the Abdomen to the Brain.","authors":"Marleni R Torres Nunez, David Livingston, Julieann C Lee, Zach Abramson, Soniya Pinto, Faizan Malik, Selene C Koo, Jessica Gartrell","doi":"10.1097/MPH.0000000000003129","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003129","url":null,"abstract":"<p><strong>Background: </strong>YWHAE::NUTM2B fusion-positive undifferentiated sarcomas in infants have been recently described as molecular characterization increases to classify challenging solid tumor cases. The impact of this molecular distinction on clinical behavior remains unclear, posing significant challenges for treatment decision-making.</p><p><strong>Methods: </strong>Data were obtained from the patient's medical record, including the diagnostic workup and management. A review of previously published cases was performed..</p><p><strong>Results: </strong>We present a case of congenital undifferentiated retroperitoneal sarcoma harboring a YWHAE::NUTM2B fusion, highlighting the potential significance of brain imaging surveillance in disease management.</p><p><strong>Conclusion: </strong>This case, combined with others, reveals a potential pattern of central nervous system (CNS) metastasis within this molecular subgroup, thus highlighting the importance of brain surveillance imaging and the relevance of CNS-penetrant chemotherapy-based regimens.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145238758","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":"Off-label Use of Dinutuximab Beta in Combination With Chemotherapy in Patients With Ewing Sarcoma: A Retrospective Single-center Case Series.","authors":"Neofit J Spasov, Frank Dombrowski, Holger Lode","doi":"10.1097/MPH.0000000000003126","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003126","url":null,"abstract":"<p><p>Surface expression of the disialoganglioside subtype GD2 has been observed on Ewing sarcoma (ES) cells, making it a suitable target for immunotherapy with the anti-GD2 antibody dinutuximab beta (DB). Here we report our experience of using DB in a cohort of 13 patients with GD2-positive, metastatic ES, in both the frontline (n=9) and relapsed/refractory (n=4) settings, when added to standard chemotherapeutic regimens. Outcomes were compared with 24 patients, primarily with localized ES, who were also treated at our center with standard therapy alone (without DB). Patients treated with DB had a median overall survival (OS) of 1877 days in the frontline setting and 810 days in the relapsed/refractory setting. Median time to progression was 1811 days and 782 days, respectively. In contrast, those treated with standard therapy alone in our center demonstrated a median OS of 1547 days and 210 days in the frontline and relapsed/refractory setting, respectively, with median times of progression of 1261 days and 113 days. DB treatment was well tolerated, with no new or unexpected adverse events reported. Anti-GD2 immunotherapy with DB represents a promising therapeutic option to improve outcomes in patients with metastatic ES, in both the frontline and relapsed/refractory settings.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-09-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145069878","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":"Nutrition Management of Children With Central Nervous System Tumors: A Survey of Pediatric Oncology Dietitians in the United States.","authors":"Rachel Hill, Karen Ringwald-Smith, Caroline Hambuchen","doi":"10.1097/MPH.0000000000003125","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003125","url":null,"abstract":"<p><strong>Purpose: </strong>Central nervous system (CNS) tumors include benign and malignant tumors of the brain and spinal cord. Nutrition risk for children with CNS tumors varies widely based on the diagnosis and treatment plan. Research supports proactive interventions for high-risk patients. However, assessment and intervention strategies vary widely between institutions, and the absence of nutrition management guidelines is a barrier to providing optimal care to these patients. Our group endeavored to survey experienced pediatric oncology dietitians throughout the United States regarding nutrition management practices and then measure agreement with practice recommendations for nutrition care.</p><p><strong>Methods: </strong>Thirteen dietitians were recruited to participate in a virtual conference concerning nutrition practices in pediatric CNS patients. Two lead dietitians performed thematic analysis of the discussions and developed 16 practice recommendations from the panel. Participants were then invited to provide feedback on the practice recommendations before rating their level of agreement on a final version of the practice recommendations.</p><p><strong>Results: </strong>Strong agreement (greater than 80% voting agree or strongly agree) was reached on 16/16 of the practice recommendations.</p><p><strong>Conclusion: </strong>Appropriate nutrition care is gaining attention as an essential element of pediatric cancer therapy. These survey results can guide nutrition care for children with CNS tumors.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145069817","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":"Mixed Chimerism and Clinical Outcome of Hematopoietic Stem Cell Transplantation in Glanzmann Thrombasthenia: Experience on 2 Siblings and Literature Review.","authors":"Eman T Al-Antary, Meera Chitlur, Manisha Gadgeel, Süreyya Savaşan","doi":"10.1097/MPH.0000000000003121","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003121","url":null,"abstract":"<p><p>Glanzmann thrombasthenia (GT) is a rare autosomal recessive platelet disorder characterized by abnormalities in platelet aggregation, resulting from quantitative or qualitative defects in integrins αIIb and β3. Currently, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only potentially curative therapeutic approach for severe GT. In this report, we present 2 children with GT that underwent successful allo-HSCT, along with 2008 to 2022 data from the Center for International Blood and Marrow Transplant Research and a summary of the existing literature providing further evidence that allo-HSCT can be a curative approach that prevents severe and life-threatening bleeding in GT. Our cases empathize the importance of monitoring flow cytometric platelet integrin αIIb (GPIIb) and β3 (GPIIIa) detection in identifying potential late graft rejection, which is a more direct assessment of platelet populations, particularly in the case of pretransplant presence of platelet antiglycoprotein GPIIb/IIIa complex antibodies.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145030052","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":"Asparaginase Premedication With Hydrocortisone Decreases Hypersensitivity Reactions.","authors":"Ryan Guerrettaz, Megan Wegter, Melodee Liegl, Amy Y Pan, Kalen McCabe, Rebecca Volmy, Michael Burke","doi":"10.1097/MPH.0000000000003128","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003128","url":null,"abstract":"<p><strong>Background: </strong>While pegylated Escherichia coli asparaginase (PEG) is an integral component of leukemia and lymphoma treatment, hypersensitivity reactions (HSR) remain a common adverse event, often resulting in adjustments to the treatment regimen, increasing the burden on patients and families. HSR to asparaginase often indicates a transition to Erwinia asparaginase (ERW), which requires patients to return to the hospital 6 times for subcutaneous injections to replace one dose of IV PEG. Previous trials have demonstrated rates of HSR to pegylated E. coli asparaginase (PEG) anywhere from 0.5% to 25%. At our institution, despite premedication with antihistamine medications such as diphenhydramine and famotidine, we have observed a similar range of patients develop treatment-limiting HSR to PEG. In addition, nonallergic infusion reactions (IR) often overlap symptomatically with HSR, making it difficult to identify true HSR, thus leading to the conservative clinical decision to remove asparaginase from the treatment plan. There are reports of adding hydrocortisone to the premedication regimen for patients receiving PEG, but this has been reported mainly in the rechallenge setting.</p><p><strong>Methods: </strong>We conducted a retrospective analysis evaluating the benefits of adding hydrocortisone to a universal 2-drug premedication regimen of famotidine and diphenhydramine. Data were gathered via chart audit comparing a group of patients with a 2-drug regimen (diphenhydramine and famotidine) to a group of patients with a 3-drug regimen of diphenhydramine, famotidine, and hydrocortisone. Analysis included patients with any lymphoid malignancy treated with a frontline PEG-containing treatment regimen. A 1-year time interval was used to collect data for the preintervention and postintervention groups. All patients who received IV PEG were reviewed. HSRs or infusion reactions were reviewed independently to confirm and grade using Common Terminology Criteria for Adverse Events (CTCAE), v5 of allergic reactions.</p><p><strong>Results: </strong>In 85 patients analyzed over a 2-year period, 50 were in group 1 and given a 2-drug regimen of diphenhydramine and famotidine before PEG infusion. The rate of HSR in group 1 was 38% with 12% experiencing IRs. Group 2 contained 35 patients who received a 3-drug regimen with diphenhydramine, famotidine, and hydrocortisone. The rate of HSR in group 2 was 11% with another 11% experiencing IRs. Based on this data, rates of IR remained unchanged, while rates of HSR decreased significantly by more than 25% (P=0.007) with the addition of hydrocortisone to the premedication regimen.</p><p><strong>Conclusions: </strong>Our retrospective cohort study provided preliminary evidence that the addition of hydrocortisone to a 2-drug premedication regimen of diphenhydramine and famotidine can decrease the rates of hypersensitivity reactions to IV PEG.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145030047","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":"Rhabdomyosarcoma in Pediatric Patients Under 2 Years: Clinical Features and Outcome.","authors":"Mohammad Alzoubi, Adam Diab, Nada Odeh, Noor Al-Assaf, Shrouq Amer, Raghad Darweesh, Ahmad Kh Ibrahimi, Mohammad Boheisi, Iyad Sultan, Hadeel Halalsheh","doi":"10.1097/MPH.0000000000003124","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003124","url":null,"abstract":"<p><strong>Background: </strong>Rhabdomyosarcoma (RMS) typically responds well to a combination of treatments with favorable prognosis in children 1 to 9 years old. However, infants may fare worse due to receiving less aggressive local therapy for concerns about long-term effects of surgery/radiation. This study investigates the clinical characteristics, treatment approach, and survival outcomes of RMS in children under 2.</p><p><strong>Methods: </strong>We reviewed retrospectively children younger than 2 years with newly diagnosed RMS treated from January 2002 until December 2022 at King Hussein Cancer Center. Demographics, clinical characteristics, and outcomes were analyzed. Statistical analysis included descriptive statistics and survival analysis using Kaplan-Meier methods. All cases were reviewed in a multidisciplinary clinic comprising experienced radiotherapists and surgeons.</p><p><strong>Results: </strong>We identified 34 cases of RMS in patients younger than 2 years at diagnosis. The median age was 13 months, with 70.6% males. The most common tumor site was bladder/prostate (N=13, 38%), followed by orbit (N=5, 14.7%), the predominant subtype was embryonal (N=30). Risk-stratification categorized 17.6% as low-risk and 79.4% as intermediate-risk. Twenty-five patients had tumors >5 cm, with metastasis in 6 (17.6%). All patients received neo-adjuvant chemotherapy, local control was by radiotherapy only (n=12, 35.3%), combined surgery and radiotherapy (n=11, 32.4%), or surgery alone (n=3, 8.8%). The 5-year event-free and overall survival rates were 55.1% and 57.5%, respectively. Fourteen patients experienced relapse/progression, with local relapse the most common pattern. TNM stage, clinical group, metastasis at diagnosis, and radiotherapy use significantly impacted survival.</p><p><strong>Conclusions: </strong>Children under 2 years of age with RMS face significant challenges, with high local recurrence rates and suboptimal survival outcomes compared with older pediatric patients. Our findings highlight the need for tailored treatment approaches that balance effective local control with minimizing long-term toxicity.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145030025","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}