Journal of Pediatric Hematology/Oncology最新文献

{"title":"Rare Pancreatitis-Related Complications of L-Asparaginase in Pediatric Acute Lymphoblastic Leukemia: A Case With Pseudocyst and Panniculitis and Literature Review.","authors":"Fatma B Kurtipek, Çağri Damar, Elif Gökçek, Belgin Gülhan, Müjdem N Azili, Neşe Yarali","doi":"10.1097/MPH.0000000000003110","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003110","url":null,"abstract":"<p><strong>Background: </strong>L-asparaginase is an essential chemotherapeutic agent for treating pediatric acute lymphoblastic leukemia (ALL), but its use is commonly linked to severe complications, including pancreatitis. Pancreatic pseudocysts and panniculitis are a rare complication of L-asparaginase-induced pancreatitis.</p><p><strong>Case presentation: </strong>We report the case of a 20-month-old girl diagnosed with pre-B ALL who developed pancreatitis, complicated by pseudocyst formation and panniculitis, following L-asparaginase therapy. The patient had clinical deterioration and the development of a mechanical ventilation requirement; septic shock was initially suspected. However, due to the appearance of cutaneous findings a few days later, radiologic investigations were performed, revealing the formation of intra-abdominal pseudocysts and subcutaneous fat necrosis. Consequently, a delayed diagnosis of pancreatitis was made, which was managed conservatively with a multidisciplinary approach. Eventually, the pseudocyst resolved with close clinical and radiologic monitoring.</p><p><strong>Discussion: </strong>Pancreatic pseudocyst formation in pediatric oncology patients is uncommon but necessitates prompt recognition and an individualized treatment strategy. Panniculitis secondary to pancreatitis is even rarer and may serve as an early cutaneous clue to underlying pancreatic pathology. Conservative management of pseudocysts, supported by interventional radiology and close follow-up, can be effective in select cases, reducing the risk associated with surgical intervention.</p><p><strong>Conclusion: </strong>This case underscores the importance of heightened clinical suspicion for pancreatic complications in pediatric patients receiving L-asparaginase. A multidisciplinary team approach is essential for effective diagnosis and treatment. Additional research is warranted to refine treatment guidelines for L-asparaginase-induced pancreatitis and its complications.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144958115","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":"Comparing Tolerability and Toxicity of Calaspargase-Pegol and Pegaspargase in Pediatric Leukemia Patients.","authors":"Jinjoo Kang, Srivarshini Kanukollu, Angelica Bevinetto, Rosamarie Corless, Arlene Redner","doi":"10.1097/MPH.0000000000003098","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003098","url":null,"abstract":"<p><p>As of December 2022, calaspargase-pegol is the standard long-acting asparaginase formulation for patients younger than 22 years of age with acute leukemia. This 3-year retrospective evaluation compared real-world safety and efficacy of calaspargase-pegol versus pegaspargase in pediatric patients. Incidence and severity of hypersensitivity reactions, silent inactivation, and common asparaginase-associated adverse effects were collected. A total of 103 patients were included, of whom 50 received calaspargase-pegol and 58 received pegaspargase. Similar rates of symptomatic hypersensitivity reactions (20% vs. 10.3%; P=0.16) and silent inactivation (16% vs. 5.2%; P=0.18) were seen with calaspargase-pegol versus pegaspargase, respectively. However, 13 patients in the calaspargase-pegol group required conversion to short-acting asparaginase compared with 6 patients in the pegaspargase group (26% vs. 10.3%; P=0.03). Most adverse effects were comparable between the 2 groups, except for a lower rate of hyperbilirubinemia with calaspargase-pegol compared with pegaspargase (34% vs. 58.6%; P=0.01). Overall, no significant differences were found between the formulations in terms of rate of hypersensitivity reactions and silent inactivation. However, calaspargase-pegol did require more conversions to short-acting asparaginase therapy. Additional monitoring and preventative measures should be utilized to prevent calaspargase-pegol-associated adverse effects.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-08-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144958165","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":"Comparing Iron Prophylaxis Strategies in Infants: Is Sucrosomial Iron a Better Alternative?","authors":"Sultan Okur Acar, Neryal Tahta","doi":"10.1097/MPH.0000000000003111","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003111","url":null,"abstract":"<p><p>Iron deficiency and iron deficiency anemia (IDA) are prevalent during infancy. Timely iron prophylaxis can prevent associated irreversible complications. As part of the \"Iron Like Turkey\" program initiated in April 2004, iron prophylaxis is administered from the fourth month of life for at least 5 months. This study aims to evaluate the implementation of iron prophylaxis in our country and compare the effectiveness of different iron preparations used in prophylaxis. Between January 2021 and January 2024, the iron prophylaxis status of 248 healthy, full-term, breastfed infants 9 to 15 months old who were referred to the Pediatric Hematology outpatient clinic of Manisa City Hospital for various reasons was assessed. Iron prophylaxis was administered to 204 (82.3%) infants, of whom 92 (44.1%) had irregular usage. Among those receiving iron prophylaxis, 110 (53.9%) used Fe+3, while 94 (46.1%) used Sucrosomial Iron (SI). No infants received Fe+2 for prophylaxis. Iron deficiency anemia (IDA) was present in 142 (57.3%) cases. No significant difference was found between Fe+3and Sucrosomial Iron in terms of adherence and duration of prophylaxis (P>0.05). However, hemoglobin (Hb), mean corpuscular volume (MCV), ferritin, and transferrin saturation levels were significantly lower in the Sucrosomial Iron group compared with the Fe+3 group (P<0.05). Iron prophylaxis is an easy and effective method to prevent IDA. Both mothers during pregnancy and infants should receive prophylaxis on time and for an adequate duration. Further research is needed to evaluate the efficacy of different iron formulations used in prophylaxis.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144958105","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":"Iron and Vitamin B12 Levels in School-Age Children.","authors":"Isıl Culha Hosceylan, Tunç Kallemoglu, Mine Basibuyuk, Nalan Karabayir","doi":"10.1097/MPH.0000000000003100","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003100","url":null,"abstract":"<p><strong>Objective: </strong>Iron and vitamin B12 deficiencies remain widespread among school-age children, contributing to growth and developmental issues. This study aimed to investigate iron and vitamin B12 levels in school-age children.</p><p><strong>Materials and methods: </strong>A cross-sectional study was conducted at a Family Medicine Unit in Istanbul. Hemogram, serum iron, total iron-binding capacity, ferritin, and vitamin B12 levels of 6 to 18-year-old children under regular follow-up were retrospectively evaluated.</p><p><strong>Results: </strong>The study included 234 children (115 boys, 119 girls; mean age: 9.9±3.2 y). Anemia prevalence was 9.4%, significantly higher in girls (13.4%) than boys (5.2%). Iron deficiency anemia was detected in 3.4% without significant gender or age variation. Ferritin levels were below 12 ng/mL in 56.3% of cases, with a significant difference between genders. Mean vitamin B12 level was 261.5±162.9 pg/mL (boys: 217.3±173.9; girls: 275.2±151.1). Vitamin B12 deficiency affected 40.6% of children, particularly boys and those age 12 to 18 years of age (P<0.05). No correlation was found between vitamin B12 levels and other parameters except hemoglobin.</p><p><strong>Conclusion: </strong>While anemia is more common in school-age girls, vitamin B12 deficiency has been found to be more common in adolescent boys. Regular monitoring of anemia, iron and vitamin B12 levels in school-age children is critical for a healthy future.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144958093","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":"Palliative Chemotherapy is Well Tolerated in a Patient With Metastatic Colorectal Cancer Due to Bloom Syndrome With a Novel BLM Mutation.","authors":"Jacqueline Baca, Katherine Anderson, Thomas Lahiri, Amelia Sybenga, Timothy Higgins, Jessica L Heath","doi":"10.1097/MPH.0000000000003108","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003108","url":null,"abstract":"<p><strong>Background: </strong>Bloom syndrome is an autosomal recessive condition of genomic instability caused by increased sister-chromatid exchange, which results in a predisposition to a variety of cancers at a young age. The molecular alterations in Bloom Syndrome predisposing to chromosomal instability alter the expected response to and toxicities of chemotherapy in patients with this condition.</p><p><strong>Observations: </strong>We report a 16-year-old patient with previously undiagnosed Bloom syndrome who presented with metastatic mucinous adenocarcinoma and tolerated palliative chemotherapy, initially with modified FOLFOX and subsequently with FOLFIRI, both at 50% dose reduction, without significant toxicity.</p><p><strong>Conclusions: </strong>Dose modified multiagent chemotherapy was well tolerated in an adolescent patient with Bloom Syndrome and metastatic colorectal cancer.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144958137","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":"The Top 50 Most-Cited Studies of Extended Half-Life Factors in Hemophilia.","authors":"Ayşe Şimşek","doi":"10.1097/MPH.0000000000003101","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003101","url":null,"abstract":"<p><strong>Objective: </strong>In recent years, one of the most significant advancements in hemophilia treatment has been the introduction of extended half-life (EHL) factors. This study aims to identify the 50 most influential articles on EHL factor use in hemophilia treatment and analyze their key characteristics.</p><p><strong>Methods: </strong>A comprehensive literature search was conducted without restrictions on publication date, country, or language using the Boolean operator \"OR.\" A total of 665 documents published between 2005 and 2024 were identified from the Web of Science (WOS) Core Collection as of December 3, 2024. Articles were ranked in descending order of citation count.</p><p><strong>Results: </strong>The 50 most-cited articles were published between 2008 and 2020, with the most productive years being 2016 and 2019, contributing 25 articles. These studies accumulated 4130 citations, averaging 82.6 citations per article. The most-cited paper, published in 2014, received 388 citations, and had the highest citation density (35.27 citations per year). The 50 articles encompassed 6 study types: reviews, phase II to III clinical trials, experimental/ex vivo studies, prospective and retrospective observational studies, and questionnaire-based studies. Review articles were the most prevalent (22 articles), while the most-cited study was a phase II to III clinical trial. No systematic reviews, meta-analyses, or randomized controlled trials were among the top-cited papers.</p><p><strong>Conclusion: </strong>The literature on EHL factors in hemophilia has expanded over the past 2 decades, with notable growth between 2016 and 2019. Haemophilia was the most prominent journal in this field.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144958096","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":"Hepatitis-Associated Aplastic Anemia in Children: Unraveling Clinical Mysteries in a Single-Center Case Series-More Questions Than Answers!","authors":"Muayad Alali, Kiet Tat, Kathleen Overholt","doi":"10.1097/MPH.0000000000003105","DOIUrl":"10.1097/MPH.0000000000003105","url":null,"abstract":"<p><p>Hepatitis-associated aplastic anemia (HAAA) is a rare but potentially life-threatening form of acquired aplastic anemia. Since 2022, our center has observed an increase in HAAA cases. This study examines pediatric HAAA cases to enhance understanding of its presentation, diagnosis, and treatment outcomes, aiming to guide future research and care protocols. A retrospective review was conducted on 5 pediatric HAAA patients treated between 2022 and 2023 at a tertiary children's hospital in the Midwestern United States. Data included clinical presentation, diagnostics, bone marrow and liver pathology, treatments, and clinical course. Immunohistochemical analysis was performed on liver biopsies. As a result, none of the 5 patients developed liver failure. One patient had a genetic mutation associated with an immune-mediated disease; other genetic tests were negative. Histopathology revealed consistent CD8 T-cell infiltration in the liver and bone marrow, with a median CD4/CD8 ratio of 0.5. The median interval from hepatitis onset to pancytopenia was 7 to 9 weeks, with a median follow-up of 2.5 years. Four patients developed severe aplastic anemia (sAA), and 1 had nonsevere aplastic anemia (NSAA). Steroid therapy was insufficient in 4 cases, necessitating antithymocyte globulin (ATG) and cyclosporine. Due to nonresponse, 4 patients required stem cell transplantation (SCT). HAAA can rapidly progress to sAA, highlighting the importance of early, aggressive intervention. Equine ATG and cyclosporine should be initiated promptly, but refractory cases often require SCT. Further research is essential to refine therapeutic strategies and improve outcomes.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144883066","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":"Standardized Neurologic Assessment Documentation for Daily Nelarabine Administration: A Bedside Questionnaire.","authors":"Andrew Wahba, Anil George, Eric S Schafer, Alexandra Stevens, Mary C Shapiro","doi":"10.1097/MPH.0000000000003109","DOIUrl":"10.1097/MPH.0000000000003109","url":null,"abstract":"<p><p>Nelarabine is an essential component in the treatment of pediatric T-cell lymphoblastic malignancies. Rare high-grade neurologic toxicities have been reported, but nelarabine discontinuation when early symptoms arise mitigates poor outcomes. Currently, no standard assessment of neurotoxicity exists. Our patient safety initiative implemented a novel 7-question neurologic assessment tool performed daily during nelarabine courses, which prompted early identification of neurotoxicity and allowed for appropriate interventions. Investigating 31 patients over 18 months demonstrated improved documentation from 65.9% to 97.2% (Δ+31.3%). Nine doses were omitted due to discovered neurotoxicity. Tool implementation advanced our ability to optimize patient care during nelarabine administration.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144883068","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":"Naxitamab-combination Therapy for the Treatment of Patients With Refractory and/or Relapsed High-risk Neuroblastoma.","authors":"Erin Murphy Trovillion, Meghan Michael, Lauren Brown, Katlin Phillips, Javier Oesterheld, Giselle Saulnier-Sholler","doi":"10.1097/MPH.0000000000003096","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003096","url":null,"abstract":"<p><p>Approved treatments for patients with refractory and/or relapsed (R/R) high-risk (HR) neuroblastoma are limited, and there is a need for new treatment combinations. In this case series, 4 patients were treated with the anti-GD2 monoclonal antibody naxitamab and granulocyte-macrophage colony-stimulating factor (GM-CSF) in combination with cyclophosphamide and topotecan between August 2021 and December 2022. This combined chemoimmunotherapy regimen was well tolerated in these heavily pretreated patients with R/R HR neuroblastoma who had few treatment options and overall poor prognoses.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-08-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144821673","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":"Moving the Needle Forward for Low-Risk and Intermediate-Risk Pediatric Rhabdomyosarcoma in LMIC: Outcomes From a Cancer Center in Pakistan.","authors":"Alina Sadaf, Sadia Anjum, Sana B Rizvi, Rabia M Wali","doi":"10.1097/MPH.0000000000003089","DOIUrl":"10.1097/MPH.0000000000003089","url":null,"abstract":"<p><p>Pediatric rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in children. In low- and middle-income countries (LMIC) such as Pakistan, several challenges in the diagnosis and treatment of RMS may lead to poor outcomes. A retrospective chart review was conducted from January 1, 2017 to January 1, 2022, to identify patients with low-risk and intermediate-risk RMS at a tertiary cancer center in Pakistan. Curative treatment was given per the standard arms of the Children's Oncology Group (COG) studies ARST0331 and ARST1431 without maintenance chemotherapy. Forty-eight patients were eligible for survival analysis. The most common tumor region was parameningeal (31.3%). The 3-year overall survival (OS) was 92.9% for the 14 low-risk patients and 70.6% for the 34 intermediate-risk patients. Eleven patients died (~23%; 3 due to sepsis, 8 due to disease progression/relapse), 11 (23%) abandoned treatment, 16 (33%) had disease progression, and 3 had relapse. There was a preponderance of intermediate-risk RMS in this cohort, with 3-year OS approaching that of high-income countries but with a lower PFS and EFS. Sepsis-related deaths, treatment abandonment, and malnutrition remain significant challenges.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-08-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144883067","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}