{"title":"Pyrites: Adnexal Mass and Hypercalcemia in a Teenage Girl.","authors":"Pritam Singha Roy, Jaydip Bhaumik, Anand Bardia, Partha S Bhattacharyya, Anisha Gehani, Arpita Bhattacharyya","doi":"10.1097/MPH.0000000000003223","DOIUrl":"10.1097/MPH.0000000000003223","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":"48 5","pages":"256-258"},"PeriodicalIF":0.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148308643","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}
Linah Omer, Nicholas J Gloude, Catherine McGuinn, Taizo A Nakano, Kathleen Overholt, Elizabeth Ogando-Rivas, Kasiani C Myers, Larisa A Broglie, Kirsty Hillier, Edo Schaefer, Jennifer A Rothman, Sasidhar Goteti, Yigal Dror, María I Cancio, Gloria P Contreras Yametti, Michaela Cada, Kristin A Shimano, Steven W Allen, Jeffrey M Lipton, Peter Kurre, Paul Castillo, Jill L O de Jong
{"title":"A Survey of Provider Practices for Diagnosis and Treatment of Pediatric Moderate Aplastic Anemia From the North American Pediatric Aplastic Anemia Consortium.","authors":"Linah Omer, Nicholas J Gloude, Catherine McGuinn, Taizo A Nakano, Kathleen Overholt, Elizabeth Ogando-Rivas, Kasiani C Myers, Larisa A Broglie, Kirsty Hillier, Edo Schaefer, Jennifer A Rothman, Sasidhar Goteti, Yigal Dror, María I Cancio, Gloria P Contreras Yametti, Michaela Cada, Kristin A Shimano, Steven W Allen, Jeffrey M Lipton, Peter Kurre, Paul Castillo, Jill L O de Jong","doi":"10.1097/MPH.0000000000003209","DOIUrl":"10.1097/MPH.0000000000003209","url":null,"abstract":"<p><p>Pediatric moderate aplastic anemia (MAA) lacks defined diagnostic criteria and a clear standard of care due to limited understanding of its pathophysiology and natural history. To understand current diagnostic and management practices for pediatric patients with MAA, a survey of the North American Pediatric Aplastic Anemia Consortium (NAPAAC) was conducted among 104 providers across 57 institutions. The survey results show that the approach to MAA remains inconsistent. The survey demonstrates broad variability regarding the working definition, diagnostic work-up, and therapeutic management of children with MAA. The diagnostic work-up and treatment options for children with MAA are largely driven by management guidelines for pediatric severe aplastic anemia (SAA). Treatment triggers and preferred therapy types varied widely among respondents. Curated next-generation sequencing panels and whole exome/whole genome sequencing were included by only 55% and 9% of respondents, respectively, suggesting the need to more broadly consider inherited bone marrow failure syndromes in the differential diagnosis of these patients. Based on the most commonly reported practices across NAPAAC institutions, we have included a proposed diagnostic and management algorithm in this manuscript. Effective, risk-adapted treatment for children with MAA requires a better understanding of the biology, natural history, and treatment outcomes in this heterogeneous population.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"230-236"},"PeriodicalIF":0.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147973222","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}
Courtney M Vaughn, Catherine Evers Smith, Laura G Schuettpelz
{"title":"Successful Treatment of Relapsed Pediatric Central Nervous System ALK+ Anaplastic Large Cell Lymphoma With Lorlatinib Monotherapy.","authors":"Courtney M Vaughn, Catherine Evers Smith, Laura G Schuettpelz","doi":"10.1097/MPH.0000000000003224","DOIUrl":"10.1097/MPH.0000000000003224","url":null,"abstract":"<p><strong>Background: </strong>Pediatric Anaplastic Lymphoma Kinase-driven (ALK+) Anaplastic Large Cell Lymphoma (ALCL) offers an exciting opportunity for targeted therapy. Standard frontline chemotherapy for ALK+ ALCL is associated with severe toxicities; ALK inhibition may provide an opportunity to decrease this toxicity and improve relapse outcomes.</p><p><strong>Observation: </strong>We present a patient with both a systemic and CNS relapse of her ALK+ ALCL who achieved complete remission with ALK inhibitor monotherapy.</p><p><strong>Conclusion: </strong>This case supports studying the inclusion of newer ALK inhibitors in upfront therapy for pediatric ALK+ ALCL and supports the use of Lorlatinib to treat CNS relapse of ALK+ ALCL.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e209-e211"},"PeriodicalIF":0.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148218276","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}
Holly J Edington, Nicholas P DeGroote, Jason Stevenson, Shanmuganathan Chandrakasan, Tamara P Miller, Sharon M Castellino
{"title":"Immunoglobulin Supplementation Practices and Effects During Pediatric B-Cell Acute Lymphoblastic Leukemia Therapy.","authors":"Holly J Edington, Nicholas P DeGroote, Jason Stevenson, Shanmuganathan Chandrakasan, Tamara P Miller, Sharon M Castellino","doi":"10.1097/MPH.0000000000003203","DOIUrl":"10.1097/MPH.0000000000003203","url":null,"abstract":"<p><p>Although some children with B-cell acute lymphoblastic leukemia (B-ALL) receive intravenous immunoglobulin (IVIG) during conventional therapy, supplementation practices vary, and data describing the indications and their impact are lacking. In this single-institution cohort of children with B-ALL receiving conventional chemotherapy, we evaluate associations of patient and disease characteristics with IVIG supplementation, describe infection-related outcomes by IVIG receipt, and describe outcomes after IVIG supplementation. Multivariable logistic regression models identified factors associated with IVIG receipt. Of 373 eligible patients with B-ALL who did not receive immune therapy, 251 (67.3%) had an IgG level checked, and 114 (30.6%) received IVIG. The median IgG nadir was lower in IVIG recipients versus nonrecipients (404 vs. 675 mg/dL, P<0.01). IVIG recipients were younger at diagnosis (4 vs. 6 y, P<0.01) and had a larger number of severe infections per 1000 leukemia treatment days (4.2 vs. 2.5, P<0.01). In adjusted models, the odds of IVIG administration were lower for non-white patients (OR=0.43, 95% CI=0.22-0.83), higher for patients with >2 severe infections during treatment (OR=2.57, 95% CI=1.28-5.18), and higher for National Cancer Institute standard risk patients with IgG nadir <500 mg/dL (OR=7.45, 95% CI=3.54-15.70). For IVIG recipients, the rate of emergency department visits, severe infections, and febrile neutropenia episodes was 5.4, 1.9, and 1.1 per 1000 treatment days, respectively, during IVIG supplementation. Among children with B-ALL receiving conventional chemotherapy, clinical characteristics differ between IVIG recipients and nonrecipients. Uniform IgG measurements during therapy may guide supplementation decisions, especially in the era of frontline immunotherapy.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":"48 5","pages":"e175-e182"},"PeriodicalIF":0.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148308572","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}
Haley Hultquist, Alyssa Rodriguez, My H Vu, Paul S Gaynon, Alexandra E Kovach
{"title":"Initial Cerebrospinal Fluid Blast Clearance Rate in Pediatric B-Lymphoblastic Leukemia is Associated With Overall Survival.","authors":"Haley Hultquist, Alyssa Rodriguez, My H Vu, Paul S Gaynon, Alexandra E Kovach","doi":"10.1097/MPH.0000000000003207","DOIUrl":"10.1097/MPH.0000000000003207","url":null,"abstract":"<p><p>Children with central nervous system (CNS) involvement by B-lymphoblastic leukemia (B-ALL) at diagnosis have higher risk features and greater rates of relapse than those without CNS involvement. This study sought to elucidate the relationship between cerebrospinal fluid (CSF) blast clearance rate with overall survival (OS) and relapse in pediatric B-ALL. Patients with CNS involvement of newly diagnosed B-ALL were stratified into 3 CSF clearance groups by days after initial lumbar puncture: fast (1 to 7 d), intermediate (8 to 15 d), and slow (15+ d) clearers. Among 246 new patients, 28% (69) were CSF+ at diagnosis (87% [60] CNS2, 13% [9] CNS3); 55% (38) fast, 35% (24) intermediate, and 10% (7) slow clearers. The hazard ratio for OS was 5.8 (95% CI: 1.2- 27.9, P =0.029) times higher in slow clearers compared with fast clearers, and 17.4 times higher compared with intermediate clearers (95% CI: 1.7-178, P =0.016). There were no significant differences in risk group ( P =0.3) or relapse rates ( P =0.5). These data suggest that among pediatric patients with CNS+ B-ALL disease at diagnosis, those who cleared CSF faster had better OS compared with patients who were slower at clearing the CSF of blasts. A study of the functional dynamics of underlying differences in CSF clearance rates may inform therapeutic modification.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"237-241"},"PeriodicalIF":0.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147774649","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}
Aviv Sever, Tomer Portnoy, Michael Gurevich, Yuri Paschovich, Shira Amar, Helen Toledano
{"title":"Excellent Outcome of Residual Pulmonary Metastases in Initially High Burden Metastatic Hepatoblastoma: A Case Series.","authors":"Aviv Sever, Tomer Portnoy, Michael Gurevich, Yuri Paschovich, Shira Amar, Helen Toledano","doi":"10.1097/MPH.0000000000003197","DOIUrl":"10.1097/MPH.0000000000003197","url":null,"abstract":"<p><strong>Background: </strong>SIOPEL-4 protocol has improved survival for metastatic hepatoblastoma. Intensified adjuvant chemotherapy is given to those with persistent pulmonary nodules post primary tumour resection. Multiple (≥10), bilateral pulmonary metastases at diagnosis, and cumulative nodule size have been linked to poorer prognosis. Reports suggest intensifying upfront treatment for these patients compared with those with lower initial metastatic burden.</p><p><strong>Observations: </strong>We report 3 cases with ≥20 bilateral, large, pulmonary metastases at diagnosis and persistent lesions postadjuvant chemotherapy. Despite high-risk features, all achieved long-term remission.</p><p><strong>Conclusions: </strong>Risk prediction may be better assessed by markers of treatment response than by initial metastatic load.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e200-e204"},"PeriodicalIF":0.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147623058","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":"Beacon Bootcamp: A Near-Peer, Experiential Approach to Chemotherapy Prescribing Education in Pediatric Hematology/Oncology Fellowship.","authors":"Paula Sulikowski, Jennifer C Yu","doi":"10.1097/MPH.0000000000003211","DOIUrl":"10.1097/MPH.0000000000003211","url":null,"abstract":"<p><p>Pediatric hematology/oncology fellows at our institution lacked formal training in prescribing chemotherapy and supportive care. This pilot study aimed to assess the feasibility of implementing a structured curriculum in a fellowship program and to gain preliminary insights into its impact on fellows' confidence and competence in placing chemotherapy and supportive care orders in the Epic Beacon system. We developed Beacon Bootcamp using Kern 6-Step Approach to Curriculum Development. General and targeted needs assessments informed the learning objectives. We held individual practice sessions, grounded in Kolb experiential learning theory, as one of our educational strategies and piloted a near-peer teaching model. The curriculum was implemented during the 2024 to 2025 academic year, and outcomes were compared with a baseline cohort from the prior academic year. Fellows who participated in Beacon Bootcamp reported higher levels of comfort with prescribing chemotherapy and supportive care compared with the previous cohort, which did not receive formal training. We observed a lower rate of order corrections required following the intervention (21% vs. 38%); however, the small sample size limited statistical analysis. Our findings suggest that Beacon Bootcamp improves fellows' confidence and may reduce prescribing errors. Its impact could be greater if implemented during the first quarter of the fellowship. Further evaluation in a larger cohort is needed to more definitively assess its effectiveness.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e150-e154"},"PeriodicalIF":0.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147973206","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}
Divyaswathi Citla-Sridhar, Jianzhong Hu, Robert F Sidonio, Sanjay Ahuja
{"title":"Bleeding Phenotypes in Inherited Platelet Function Disorders: Insights From the ATHNdataset.","authors":"Divyaswathi Citla-Sridhar, Jianzhong Hu, Robert F Sidonio, Sanjay Ahuja","doi":"10.1097/MPH.0000000000003213","DOIUrl":"10.1097/MPH.0000000000003213","url":null,"abstract":"<p><strong>Background: </strong>Inherited platelet function disorders (IPFDs) are rare, heterogeneous conditions that pose diagnostic and management challenges. While bleeding in Glanzmann thrombasthenia (GT) and Bernard-Soulier syndrome (BSS) is well characterized, milder or unclassified IPFDs remain poorly defined.</p><p><strong>Objective: </strong>To characterize the bleeding phenotype, treatment patterns, and diagnostic distribution of IPFDs across the United States using the national ATHNdataset.</p><p><strong>Methods: </strong>We conducted a retrospective cohort study using the ATHNdataset, a deidentified registry maintained by the American Thrombosis and Hemostasis Network. Clinical outcomes, including bleeding events, laboratory data, treatments, and procedures, were analyzed for participants with IPFDs between 2013 and 2022 and with active follow-up from January 2021 to December 2022.</p><p><strong>Results: </strong>Among 2302 individuals with IPFDs, 8.1% as GT, 2.4% as BSS, 7.0% as granule defects, and 81.6% as IPFD-other. Among 985 participants with active follow-up, 236 bleeding events were reported across 251 patients. The most common events were epistaxis (42.8%), followed by soft tissue (14.4%) and oral bleeding (13.9%). Notably, intracranial hemorrhage occurred in 8 patients and joint bleeds in 82, reflecting significant morbidity across all subtypes. Median ISTH-BAT scores were elevated-13 in GT, 16 in dense-granule defects, and 7 in unclassified IPFDs-exceeding reported population norms. Regarding hemostatic management, antifibrinolytics were used in over half of all patients, with desmopressin and platelet transfusions being used less frequently.</p><p><strong>Conclusion: </strong>By defining the clinical spectrum, treatment utilization, and residual diagnostic uncertainty across IPFDs, this study underscores the importance of improving diagnostic precision and care for patients with platelet function disorders in the modern era.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"207-216"},"PeriodicalIF":0.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147973204","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":"High-Dose Intravitreal Topotecan for Retinoblastoma With Subretinal Seeds: A Well-Tolerated and Efficacious Approach.","authors":"Ranveer Palia, Waylon Alvarado, Kerri Becktell, Aparna Ramasubramanian, Nicole Liberio","doi":"10.1097/MPH.0000000000003221","DOIUrl":"10.1097/MPH.0000000000003221","url":null,"abstract":"<p><strong>Background: </strong>High-dose intravitreal topotecan (90 μg) has emerged as a promising treatment option for refractory or recurrent retinoblastoma with active tumor and subretinal seeds, offering localized disease control while minimizing systemic toxicity.</p><p><strong>Observations: </strong>In this 5-patient case series, tumor regression was achieved in 4 cases without significant adverse effects. One patient required enucleation due to disease progression. Timely administration and consistent follow-up were identified as key factors influencing treatment outcomes.</p><p><strong>Conclusions: </strong>High-dose intravitreal topotecan demonstrates meaningful therapeutic potential in achieving localized tumor control with an acceptable safety profile, supporting further investigation into its role in managing refractory retinoblastoma.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"242-245"},"PeriodicalIF":0.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148211600","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":"Checkpoint Inhibitor Pneumonitis With Pneumomediastinum Following Nivolumab-AVD in Classic Hodgkin Lymphoma: A Case Report.","authors":"Siddarth R Ganesh, Aaron D Hodes, Burton E Appel","doi":"10.1097/MPH.0000000000003220","DOIUrl":"10.1097/MPH.0000000000003220","url":null,"abstract":"<p><strong>Background: </strong>Immune checkpoint inhibitors have transformed cancer treatment but may cause pulmonary immune-related toxicities even after therapy completion.</p><p><strong>Observations: </strong>A young patient with classic Hodgkin lymphoma developed checkpoint inhibitor pneumonitis with pneumomediastinum following nivolumab-AVD. She presented with progressive dyspnea, and imaging revealed right lower lobe consolidation with mediastinal air extending into the neck. Symptoms improved rapidly with intravenous corticosteroids, followed by a taper.</p><p><strong>Conclusion: </strong>This case describes a rare presentation of pulmonary toxicity in a younger patient and reflects the need for vigilance as immunotherapy use expands across age groups and malignancies.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e186-e188"},"PeriodicalIF":0.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148218172","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}