Journal of Pediatric Hematology/Oncology最新文献

{"title":"Clinicohematological and Genetic Profile of Hereditary Spherocytosis in Children: An Experience From a North Indian Center.","authors":"Ankita Chakraborty, Manas Kalra, Anupam Sachdeva, Jyoti Kotwal, Sabina Langer, Amrita Saraf, Pallavi Rana, Surbhi Dahiya, Vandana Arya, Satish Aggarwal, Alpana Prasad","doi":"10.1097/MPH.0000000000003205","DOIUrl":"10.1097/MPH.0000000000003205","url":null,"abstract":"<p><strong>Background: </strong>Hereditary spherocytosis (HS) is a genetically and clinically diverse red cell membrane disorder, with limited clinical and molecular data on pediatric patients from India.</p><p><strong>Methods: </strong>This ambispective study, conducted at a North Indian tertiary center over a period of 6 years, evaluated HS patients for clinical, laboratory, genetic, and treatment outcomes.</p><p><strong>Results: </strong>Forty-seven patients (33 males, 14 females; mean age 12.1±8.7 yrs) were analyzed. Family history was contributory in 40.4%. Around 72.3% patients required at least one PRBC transfusion by the time of analysis, while 9 were transfusion dependent. Hemolytic facies and growth retardation were seen in 25.6% and 30.7%, while gallstones were found in 42.5%. MCHC was 32.2±1.92% and was not found to be a useful screening test. Incubated osmotic fragility testing (iOFT) was positive in 36 cases (sensitivity 76.6%), while eosin-5-maleimide (EMA) binding by flow cytometry was positive in 41 cases (sensitivity 87.2%), detecting all cases missed or equivocal on iOFT and demonstrating superior diagnostic yield. The remaining 6 cases were confirmed by genetic testing, which served as the definitive diagnostic modality. Among 34 patients tested genetically, mutations were found in ANK1 (n=14), SPTB (n=12), SLC4A1 (n=3), EPB42 (n=4), and SPTA1 (n=1). Beyond classic HS mutations, additional findings included PKLR mutations (n=2), hereditary elliptocytosis (n=2), dehydrated stomatocytosis (n=2), and Gilbert syndrome (n=4) with cholelithiasis. Genetic testing refined diagnoses, reclassifying one Congenital Dyserythropoietic Anemia (CDA) case as HS and 4 HS cases as alternative etiologies.</p><p><strong>Conclusion: </strong>This study underscores the clinical and genetic heterogeneity of Indian HS, demonstrating the value of combining the EMA dye test with genetic sequencing. The disease impacted facial features and growth, while splenectomy showed good outcomes in severe cases.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"217-223"},"PeriodicalIF":0.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147774573","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":"COVID-19 and Influenza Vaccine Acceptance and Hesitancy Among Caregivers of Children With Cancer.","authors":"Alastair Murray, Palcah Shibale, Jennifer Wilkes, Janet A Englund, Michael A Bender, Jeffrey R Harris, Annika M Hofstetter","doi":"10.1097/MPH.0000000000003199","DOIUrl":"10.1097/MPH.0000000000003199","url":null,"abstract":"<p><strong>Introduction: </strong>Children with cancer are immunocompromised and at increased risk for severe outcomes from infections such as COVID-19 and influenza. Vaccination is recommended, but caregiver perceptions in this high-risk group are not well characterized.</p><p><strong>Methods: </strong>We conducted a cross-sectional survey of English- and Spanish-speaking caregivers of pediatric oncology patients at a large urban pediatric hospital between March and June 2022, during evolving COVID-19 vaccine authorization and recommendations for children. The survey assessed vaccine-related knowledge, attitudes, and hesitancy regarding COVID-19 and influenza. Responses were linked to the child's electronic health record. COVID-19 vaccine acceptance, defined as documented or caregiver-reported receipt, or intention to vaccinate if age-ineligible, was the primary outcome. Influenza vaccine acceptance was a secondary outcome. Associations were analyzed using multivariable logistic regression.</p><p><strong>Results: </strong>Of 442 eligible caregivers, 100 (23%) participated. COVID-19 vaccine acceptance was 71%, influenza acceptance was 82%, and 57% of caregivers were vaccine-hesitant. Vaccine hesitancy was negatively associated with COVID-19 vaccine acceptance. In contrast, perceived vaccine safety, efficacy, prior influenza vaccination, and concern about cancer during the pandemic were positively associated.</p><p><strong>Conclusion: </strong>Caregiver attitudes significantly influenced vaccine acceptance. Interventions that strengthen provider communication, address safety and efficacy concerns, and acknowledge caregiver perceptions may help reduce hesitancy in this vulnerable population.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e160-e166"},"PeriodicalIF":0.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147941817","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":"Internalizing and Externalizing Problems and Psychiatric Comorbidities in Children With Hereditary Spherocytosis.","authors":"Seda Erbaş, Mustafa Özay, Hilal Yazici Kopuz, Berhan Akdağ","doi":"10.1097/MPH.0000000000003225","DOIUrl":"10.1097/MPH.0000000000003225","url":null,"abstract":"<p><p>This study evaluated internalizing and externalizing problems, as well as psychiatric comorbidities, in children with hereditary spherocytosis (HS). The study cohort comprised children followed at the Department of Pediatric Hematology at Gaziantep City Hospital (n=27). Psychiatric diagnoses were assessed using a semi-structured interview, and parents completed the Strengths and Difficulties Questionnaire (SDQ) to evaluate internalizing and externalizing symptoms in their children. Based on the semi-structured interview, 29.6% of patients (n=8) met diagnostic criteria for attention-deficit/hyperactivity disorder (ADHD), followed by enuresis (22.2%, n=6). Compared with the control group, children with HS demonstrated higher parent-reported SDQ scores for both internalizing ( P =0.042) and externalizing problems ( P =0.017). Among the SDQ subdomains, children with HS had significantly higher hyperactivity/inattention scores than controls ( P =0.004). No significant between-group differences were identified in other domains, including emotional symptoms, conduct problems, and peer relationship problems. These findings suggest that children with HS may experience a greater burden of internalizing and externalizing difficulties than their healthy peers and may also be at increased risk of psychiatric comorbidities, particularly ADHD. Larger multicenter studies are warranted to confirm and extend these findings.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e134-e137"},"PeriodicalIF":0.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148218227","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":"Pediatric High-Risk Neuroblastoma Treated With Autologous Stem Cell Transplant-Experience From a Tertiary Cancer Center in India.","authors":"Helie Raval, Dale Montes, Maharshi Trivedi, Chinmay Doctor, Harsha Panchal","doi":"10.1097/MPH.0000000000003215","DOIUrl":"10.1097/MPH.0000000000003215","url":null,"abstract":"<p><p>High-risk neuroblastoma (HR-NB) remains a therapeutic challenge in low- and middle-income countries. We retrospectively analyzed the clinical characteristics, treatment response, transplant outcomes, event-free survival, and overall survival of pediatric patients with HR-NB treated with intensive multimodality therapy, including autologous stem cell transplant (ASCT) at a resource-limited tertiary cancer center between January 2013 and January 2024. Children aged 18 years or younger received rapid-COJEC induction chemotherapy, followed by surgical resection when feasible, ASCT with busulfan-melphalan conditioning, radiotherapy, and differentiation therapy with 13-cis-retinoic acid. Eighteen patients were included, with a male-to-female ratio of 2:1 and a median age of 4 years. The adrenal gland was the most common primary site in 55.55% of patients. Bone marrow and bone metastases were present in 72.2% and 44.4% of patients, respectively. Complete response after induction was achieved in 44.4% of patients. Median neutrophil engraftment occurred at 13 days. At a median follow-up of 17 months, 55.5% of patients were alive in remission. Relapse occurred in 50%, with most succumbing to the disease. There was no treatment-related mortality. Two-year event-free and overall survival were 55.3% and 65.6%, respectively. Outcomes were comparable to other resource-limited settings, and the lack of complete response after induction did not adversely affect event-free survival.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e196-e199"},"PeriodicalIF":0.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148218142","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":"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}

{"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}

{"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}

{"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}

{"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}

{"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}