Sarthak Chakrabarti, Sourish K Roy, Sanjiban Patra, Surabhi Jajodia, Rajat Khandelwal, Indrajeet K Tiwary, Mahesh K Goenka
{"title":"Significant Gastrointestinal Bleeding Due to Jejunal Metastasis as the Harbinger of Choriocarcinoma Syndrome in an Adolescent Boy.","authors":"Sarthak Chakrabarti, Sourish K Roy, Sanjiban Patra, Surabhi Jajodia, Rajat Khandelwal, Indrajeet K Tiwary, Mahesh K Goenka","doi":"10.1097/MPH.0000000000003275","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003275","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148977127","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":"Comments on \"Initial Cerebrospinal Fluid Blast Clearance Rate in Pediatric B-Lymphoblastic Leukemia Is Associated With Overall Survival\".","authors":"Munazza Khan, Wamiq Saeed","doi":"10.1097/MPH.0000000000003269","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003269","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148930118","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}
Mehmet Fatih Orhan, Öznur Tiryaki, Aslı Akdeniz Kudubeş, Hamide Zengin, Köksal Delibaş
{"title":"Audiological Outcomes Following Platinum-Based Chemotherapy in Pediatric Oncology Patients.","authors":"Mehmet Fatih Orhan, Öznur Tiryaki, Aslı Akdeniz Kudubeş, Hamide Zengin, Köksal Delibaş","doi":"10.1097/MPH.0000000000003270","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003270","url":null,"abstract":"<p><strong>Purpose: </strong>This study aimed to retrospectively evaluate the audiological monitoring of ototoxicity developing in pediatric oncology patients receiving platinum-based chemotherapy.</p><p><strong>Methods: </strong>This retrospective study included 14 patients aged 1 month to 18 years who received platinum-based chemotherapy at the pediatric hematology/oncology clinic between 2015 and 2023 and had pre- and post-treatment audiometric evaluations. Audiometric evaluations were performed in the 250 to 6000 Hz frequency range, and hearing loss was classified according to American Speech-Language-Hearing Association criteria.</p><p><strong>Results: </strong>A general increase in hearing thresholds was observed post-treatment, with the magnitude varying by frequency. While significant changes were detected in low and midfrequencies, the most pronounced increase was observed in high frequencies (4000 to 6000 Hz), and the effect size was large at these frequencies. In the assessment of high-frequency hearing loss, 85.7% of patients had hearing loss. When comparing low and high frequencies, the increase in hearing thresholds was significantly greater at high frequencies. In comparisons between the right and left ears, a tendency toward greater hearing loss was observed in the left ear; however, this difference was not statistically significant.</p><p><strong>Conclusion: </strong>Platinum-based chemotherapy may be associated with clinically relevant hearing threshold shifts in pediatric oncology patients, particularly at high frequencies. Regular audiological monitoring is important for the early identification and management of hearing loss.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148900841","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}
Adnan Deniz, Sevgi Tale, Gökçe Cırdı, Ayfer Sakarya Güneş, Leman Tekin Orgun, Emine Zengin, Bülent Kara
{"title":"Central Nervous System Immune Reconstitution Inflammatory Syndrome During Neutrophil Recovery in Pediatric Acute Myeloid Leukemia: A Clinical and Laboratory Observation.","authors":"Adnan Deniz, Sevgi Tale, Gökçe Cırdı, Ayfer Sakarya Güneş, Leman Tekin Orgun, Emine Zengin, Bülent Kara","doi":"10.1097/MPH.0000000000003260","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003260","url":null,"abstract":"<p><strong>Background: </strong>Central nervous system (CNS) immune reconstitution inflammatory syndrome (IRIS) is well characterized in HIV but exceedingly rare in pediatric hematologic malignancies.</p><p><strong>Observation: </strong>An 11.5-year-old girl with acute myeloid leukemia (AML) and probable invasive pulmonary mold infection developed an expansive right parieto-occipital lesion with a 14 mm midline shift during neutrophil recovery. Evaluation excluded leukemic infiltration and active infection. Probable CNS IRIS was diagnosed; dexamethasone with continued antifungal therapy produced rapid clinical and radiologic recovery, with sustained AML remission at 29 months.</p><p><strong>Conclusions: </strong>CNS IRIS should be considered in pediatric AML with unexplained neurological deterioration during neutrophil recovery; timely corticosteroid therapy can be life-saving.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-08-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148673895","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":"MTHFD1 Deficiency in Two Unrelated Children: Highlights on Phenotypic Spectrum and Response to Folic Acid Therapy.","authors":"Fajer Altammar, Haidy Elsayed, Mohamed Elshinawy, Waleed Al-Herz","doi":"10.1097/MPH.0000000000003238","DOIUrl":"10.1097/MPH.0000000000003238","url":null,"abstract":"<p><strong>Background and aims: </strong>Methylene-tetrahydrofolate dehydrogenase 1 (MTHFD1) deficiency is a rare inborn error of immunity (IEI) involving defects in folate metabolism. It can present with combined immunodeficiency and variable phenotypic features, including recurrent bacterial infections, megaloblastic anemia, and failure to thrive.</p><p><strong>Methods: </strong>We describe 2 unrelated Kuwaiti children with MTHFD1 deficiency caused by a homozygous pathogenic variant [c.517C>T (Arg173Cys)].</p><p><strong>Results: </strong>Both cases presented with unexplained megaloblastic anemia, recurrent sinopulmonary infections, failure to thrive, and developmental delay. The first patient, a 10-year-old girl, was diagnosed with combined immunodeficiency and autoimmune thyroiditis. Treatment with folic acid resulted in significant clinical improvement. The second patient, a 3-year-old girl, presented with sepsis secondary to progressive lobar pneumonia and bone marrow failure. Despite intensive treatment, including broad-spectrum antibiotics, antifungals, IVIG, and dexamethasone for suspected hemophagocytic lymphohistiocytosis, she developed severe complications and passed away on day 18 of PICU admission.</p><p><strong>Conclusion: </strong>As there is no specific clinical or laboratory phenotype for most IEIs, we emphasize the importance of molecular diagnosis through urgent genetic testing in patients with suspected MTHFD1 deficiency. Precision therapy with prompt folate or folinic acid supplementation can significantly improve outcomes, as evidenced by the survival of one patient with minimal intervention.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e241-e244"},"PeriodicalIF":0.9,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148258563","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":"Frequent Use of Hematologic Testing in Children Admitted for Nonaccidental Trauma.","authors":"Meghan McCormick, Ramasubramanian Kalpatthi","doi":"10.1097/MPH.0000000000003236","DOIUrl":"10.1097/MPH.0000000000003236","url":null,"abstract":"<p><p>Nonaccidental trauma (NAT) can present with bleeding symptoms. Judicious use of hematologic testing is recommended to evaluate for medical causes of bleeding, while acknowledging inherited bleeding disorders and NAT may be present concurrently. A retrospective chart review using the Pediatric Health Information System database identified pediatric patients <18 years of age with an ICD diagnostic code for NAT associated with admission. Using laboratory charge data, we identified the hematologic testing sent and the number of encounters with expanded hematologic testing (EHT), defined as testing beyond CBC, PT, PTT, Factor IX, and von Willebrand testing (and fibrinogen/d-dimer in cases of intracranial hemorrhage). In 9561 admissions meeting inclusion criteria, laboratory testing was sent in 91.9% of encounters and EHT in 35.9% of encounters. The most common EHT test being fibrinogen. EHT was associated with a significant increase in laboratory-associated charges. Only 69 children (0.7%) were later identified to have an underlying bleeding disorder, most commonly von Willebrand Disease. Factors associated with EHT included hematology consultation, young age, higher income, and private health insurance. Despite few patients diagnosed with underlying bleeding disorders, EHT was frequently obtained. Sociodemographic features may influence testing decisions. Adherence to guidelines for evaluation may help reduce disparities.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e250-e254"},"PeriodicalIF":0.9,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148352198","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":"Clinical Presentation and Treatment Outcomes of Pediatric Acute Promyelocytic Leukemia: A Study From a Developing Country.","authors":"Naila Rafiq, Ghulam Q Pathan, Sadia Muhammad, Neelum Mansoor","doi":"10.1097/MPH.0000000000003231","DOIUrl":"10.1097/MPH.0000000000003231","url":null,"abstract":"<p><p>Acute promyelocytic leukemia (APL) is a highly curable form of acute myeloid leukemia (AML) when treated early with all-trans retinoic acid (ATRA) and arsenic trioxide (ATO). Resource constraints in low- and middle-income countries (LMICs) may affect results. This study examined the clinical characteristics and outcomes of pediatric patients with APL receiving an ATRA-ATO-based regimen. The retrospective review included 1- to 16-year-olds diagnosed between January 2019 and December 2022. Morphology and PML-RARA detection by fluorescence in situ hybridization (FISH) confirmed the diagnosis. Patients were stratified by initial white cell count (WCC) (low-risk <10×10 9 /L; high-risk ≥10×10 9 /L). Data were retrieved from electronic medical records. Event-free survival (EFS) rates were obtained using the Kaplan-Meier analysis. Fifty patients (mean age: 10.7 y; 56% male) were evaluated. Low-risk was 32%, and high-risk was 68%. Thrombocytopenia and hypofibrinogenemia were common. All patients had PML-RARA FISH-positives, and 100% of evaluated patients attained morphologic and molecular remissions. Eleven (22%) patients died, mostly from hemorrhage. Abandonment and relapses were observed in 10% and 8% of patients, respectively. Two-year EFS was 60% (low-risk 81%; high-risk 50%). The ATRA-ATO regimen showed substantial remission rates, but early deaths remain a concern. Increased survival rates in resource-constrained environments require improved early detection, referral, and supportive care.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"277-282"},"PeriodicalIF":0.9,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148252036","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}
Elizabeth L McKone, Kristofer W Roberts, William S Harmsen, William G Breen, Anita Mahajan, Wendy A Allen-Rhoades, Peter S Rose, Nadia N Laack, Safia K Ahmed
{"title":"Local Control and Survival Outcomes in Pediatric Nonrhabdomyosarcoma Soft Tissue Sarcoma: The Mayo Clinic Experience.","authors":"Elizabeth L McKone, Kristofer W Roberts, William S Harmsen, William G Breen, Anita Mahajan, Wendy A Allen-Rhoades, Peter S Rose, Nadia N Laack, Safia K Ahmed","doi":"10.1097/MPH.0000000000003202","DOIUrl":"10.1097/MPH.0000000000003202","url":null,"abstract":"<p><strong>Objective: </strong>This single institutional analysis explored outcomes and factors associated with local control and survival in pediatric patients with nonrhabdomyosarcoma soft tissue sarcoma (NRSTS).</p><p><strong>Methods: </strong>Patients 18 years of age or younger diagnosed between October 1990 and November 2021 who received external beam radiotherapy (EBRT) were retrospectively identified. Overall survival (OS) and disease-free survival (DFS) were analyzed using Kaplan-Meier methods and univariate Cox models.</p><p><strong>Results: </strong>Among 51 included patients, there were 33 extremity tumors (65%), with 80% T1/T2 and 90% M0. Surgery in 49/51 (96%) resulted in 32 (65%) R0 resections. All received EBRT, with brachytherapy or intraoperative radiotherapy boosts in 21/51 (41%). Thirty-four (66.7%) received chemotherapy. With 5.7 years median follow-up, 5-year OS and DFS were 70.7% and 67.9%, respectively. Positive margins were significantly associated with worse OS. Two-year local failure incidence was 18.1% for EBRT versus 5.3% for EBRT+boost (HR 3.67, P =0.24). Among 6 local failures, 5 received EBRT alone, including 3 with positive margins. Two treated with EBRT+boost had positive margins, neither with local recurrence.</p><p><strong>Conclusion: </strong>Multimodality treatment of pediatric NRSTS results in 5-year OS and DFS around 70%. Although complete resection remains essential, and the modern incidence of R1 resection has declined, IORT/brachytherapy boost may continue to have a role in selected patients with a high local recurrence risk.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"261-269"},"PeriodicalIF":0.9,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148330540","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}
Gokalp Rustem Aksoy, Hatice Zeynep Terzi, Yunus Murat Akcabelen, Aytul Temuroglu, Arzu Oto
{"title":"An Unexpected Malignancy Behind Pediatric Microangiopathic Hemolytic Anemia: Gastric Signet Ring Cell Carcinoma.","authors":"Gokalp Rustem Aksoy, Hatice Zeynep Terzi, Yunus Murat Akcabelen, Aytul Temuroglu, Arzu Oto","doi":"10.1097/MPH.0000000000003240","DOIUrl":"10.1097/MPH.0000000000003240","url":null,"abstract":"<p><strong>Background: </strong>In the pediatric population, gastrointestinal (GIS) cancers are rare, and paraneoplastic syndromes associated with these malignancies are even more uncommon.</p><p><strong>Observations: </strong>A 15-year-old patient with a clinical presentation of microangiopathic hemolytic anemia (MAHA) resistant to plasma exchange was found to have metastatic adenocarcinoma in a biopsy of a supraclavicular lymphadenopathy. Subsequent endoscopy revealed gastric signet ring cell carcinoma.</p><p><strong>Conclusions: </strong>This case highlights that malignancy should be suspected in pediatric patients with MAHA who are unresponsive to plasma exchange and have normal ADAMTS-13 activity.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e259-e263"},"PeriodicalIF":0.9,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148302156","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}