Journal of Pediatric Hematology/Oncology最新文献

{"title":"Second Malignant Neoplasms in Long-term Retinoblastoma Survivors: Retrospective Cohort Study of 491 Patients in Turkey.","authors":"Eren Müngen, İrem Koç, Hayyam Kiratli, Ali Varan","doi":"10.1097/MPH.0000000000003039","DOIUrl":"10.1097/MPH.0000000000003039","url":null,"abstract":"<p><strong>Background: </strong>It has been reported that the risk of second malignant neoplasms (SMNs) in long-term follow-up patients with heritable retinoblastoma (Rb) is significantly increased compared with patients with non-heritable Rb and the general population. In this study, we investigated the types, frequencies, clinical and pathologic features, potential risk factors, and outcomes of SMNs occurring in a large group of retinoblastoma patients that were diagnosed, treated, and followed up for a long time in our Pediatric Oncology unit.</p><p><strong>Methods: </strong>Our study comprehensively analyzed records of Rb patients followed up at Hacettepe University Pediatric Oncology Department over a 51-year period from January 1972 to January 2023. We determined the number, rate, and time of diagnosis of various SMN types and investigated potential risk factors that could lead to the development of SMNs.</p><p><strong>Results: </strong>A total of 491 patients were included in this study. Median age at the time of retinoblastoma diagnosis was 1.25 (range, 0.02 to 12.08) years. Of these cases, 313 (63.7%) were unilateral, 174 (35.4%) were bilateral, and 4 (0.9%) were trilateral Rb. Enucleation was performed in 348 (70.9%) cases. A total of 334 cases received systemic chemotherapy with different protocols. Intra-arterial chemotherapy (IAC) was administered in 101 (20.6%) patients. Radiotherapy was administered in 76 (15.5%) patients. After enucleation, 56 (11.4%) patients were followed up without further treatment. SMNs occurred in 13 (2.6%) of 491 patients. Among these, 9 (69.2%) patients were considered as having heritable Rb. Enucleation was performed in 10 cases. Only 1 patient received radiotherapy and 12 patients received systemic chemotherapy. Most common subtype of SMNs was osteosarcoma (n=7; 53.8%), followed by acute myeloid leukemia (AML) (n=3; 23.1%), acute lymphoblastic leukemia (ALL) (n=1; 7.7%), Wilms tumor (n=1; 7.7%), and colon adenocarcinoma (n=1; 7.7%). The median time from the diagnosis of Rb to the onset of SMN was 136 (range, 24 to 250) months. Among the patients that underwent IAC, no patient developed SMN. Of the 491 patients, 41 (8.3%) died and 450 are still alive. Of the 13 patients with SMN, 8 (61.5%) died and 5 are still alive. Five-year OS of our study group was 91% and it was significantly lower in patients with SMNs compared with those without SMNs ( P =0.001).</p><p><strong>Conclusion: </strong>In this study, overall survival (OS) was lower in patients with bilateral retinoblastoma compared with unilateral cases, and similarly reduced in patients who received radiotherapy compared with those who did not. In addition, the development of second malignant neoplasms (SMNs) was significantly higher in hereditary retinoblastoma patients than in non-hereditary cases. These findings highlight the importance of careful long-term monitoring and tailored follow-up strategies in patients at increased risk.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e161-e167"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144004834","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":"Agrobacterium radiobacter -induced Port Catheter Bacteremia in a Pediatric Acute Lymphoblastic Leukemia Patient: A Rare Clinical Case.","authors":"Fatma Burçin Kurtipek, Dilek Kaçar, Bedia Dinç, Aslinur Özkaya Parlakay, Neşe Yarali","doi":"10.1097/MPH.0000000000003045","DOIUrl":"10.1097/MPH.0000000000003045","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"260-261"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144031467","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: A Right Orbital Tumor.","authors":"Bun Sereyleak, Has Sothearak, Sam Lyvannak, Thy Bunpaov, Khauv Phara, Yin Sopheakbot, Ven Ratanak, Jason Jarzembowski, Frank Goulding Keller, Bruce Camitta","doi":"10.1097/MPH.0000000000003060","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003060","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2025-06-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144475746","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":"Adolescent and Young Adult Oncology in the United States in 2025: Finding Its Place in the Oncology World Part 2 of 2.","authors":"Peter H Shaw, Sharon M Castellino, Ryan Guerrettaz, Nicholas Yeager, Bradley Zebrack, Rachel Brandon, Archie Bleyer","doi":"10.1097/MPH.0000000000003066","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003066","url":null,"abstract":"<p><p>In 2015, this core of authors wrote a \"state of the union\" overview of AYA oncology care at the time titled \"Adolescent and Young Adult (AYA) Oncology in the United States: A Specialty in Its Late Adolescence.\" Since then, the landscape of cancer care in this unique population has changed, with encouraging improvement in some areas and persistent challenges in others. Ten years later, we have decided to update our review to demonstrate how far we have come in caring for 15 to 39-year-olds with cancer in the United States and how much further we need to go to truly improve both their short and long-term outcomes. Back in 2015, we described the field as in its late adolescence, still trying to define itself. With this 2-part review, we hope to demonstrate that as a subspecialty, it has grown up but is still trying to firmly establish its place in the larger world of oncology, much like a young adult that has moved away from home and is establishing its own identity in a changing world. In the first part of our update on the state of adolescent and young adult (AYA) oncology in the United States in 2025, we reviewed the epidemiology of AYA cancers (those in 15 to 39-year-olds) as well as the advancements in the management of acute lymphoblastic leukemia, Hodgkin lymphoma, non-Hodgkin lymphoma, and sarcomas in this population. In this second part, we review the topics of clinical trial enrollment, models of care, the psychosocial impact of cancer in this population and survivorship.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2025-06-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144266447","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":"Sustained Clinical Remission of BRAF V600E-Mutated Langerhans Cell Histiocytosis With Multiorgan Involvement in an Infant Treated With Dabrafenib.","authors":"Franziska Cuntz, Jean Donadieu, Susanne Holzhauer","doi":"10.1097/MPH.0000000000003014","DOIUrl":"10.1097/MPH.0000000000003014","url":null,"abstract":"<p><strong>Background: </strong>Above 50% of LCH cases show BRAF-mutations, which can be targeted by dabrafenib in refractory disease.</p><p><strong>Observations: </strong>Here, we report on a patient with neonatal multisystem, BRAF-mutated LCH refractory to conventional treatment with vinblastine and prednisolone. Duodenal involvement rendered oral nutrition impossible, and the patient was severely ill with pancytopenia, hepatic dysfunction, cholestasis, and septic episodes. After initiation of targeted therapy with dabrafenib, the patient achieved sustained clinical remission.</p><p><strong>Conclusions: </strong>Multisystem LCH is a rare and potentially life-threatening disease that can mimic various neonatal conditions. A high index of suspicion is necessary for diagnosis. Timely initiation of targeted therapy may prevent irreversible organ damage.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"210-213"},"PeriodicalIF":0.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143557221","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":"Patterns and Predictors of Hydroxyurea Use Among Californians Living With Sickle Cell Disease.","authors":"Melinda Rushing, Sophia Horiuchi, Mariam Kayle, Sarah L Reeves, Alexander K Glaros, Susan Paulukonis","doi":"10.1097/MPH.0000000000003027","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003027","url":null,"abstract":"<p><p>Hydroxyurea is the primary disease-modifying therapy for sickle cell disease (SCD), yet adherence is low. Our objective was to identify patterns and predictors of hydroxyurea adherence among Medicaid enrollees with SCD. Children and adults with SCD who received Medicaid benefits between 2009 and 2018 in California were included. Monthly hydroxyurea possession ratios were calculated using filled hydroxyurea prescriptions. Group-based trajectory modeling was applied to identify hydroxyurea possession trajectories and multinomial logistic regression modeling to evaluate predictors of hydroxyurea possession group membership: prior acute care visits due to VOCs, prescriber specialty, and participant sex and age. 713 participants (48% in the 0 to 17 age group, 50% male) had 3 distinct hydroxyurea possession groups: persistently high (n=263, 37%), moderate to low (n=253, 35%), and low to no possession (n=197, 28%). The 18 to 24 and 25+ age groups had greater odds of being in the moderate to low (OR: 2.62, 1.70) and low to no (OR: 3.60, 2.45) than the persistently high possession group compared with the 0 to 17 age group when adjusted for prior VOCs. Children had greater odds of being in the persistently high hydroxyurea possession group compared with young adults and adults, suggesting there are protective factors at this age that promotes better hydroxyurea adherence.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":"47 4","pages":"169-176"},"PeriodicalIF":0.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144026452","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":"Alternaria Invasive Infection in Children With Hemato-Oncological Disease: A National Multicenter Report.","authors":"Aviv Sever, Chen Rosenberg Danziger, Nimrod Sachs, Salvador Fisher, Efraim Bilavsky, Gilad Sherman, Yael Shachor-Meyouhas, Galia Grisaru, Haim Ben Zvi","doi":"10.1097/MPH.0000000000003016","DOIUrl":"10.1097/MPH.0000000000003016","url":null,"abstract":"<p><p>Invasive fungal diseases significantly impact hemato-oncology pediatric patients, with Aspergillus and Candida being the primary culprits. However, pediatric Alternaria infections remain understudied. This study aims to characterize Alternaria infections in pediatric hemato-oncology cases nationwide. This retrospective multicenter observational study reviewed medical records from Israel's 5 largest tertiary pediatric centers between 2011 and 2023. We identified 22 patients aged 4 to 18 years with invasive Alternaria infection. Predominant diagnoses were acute lymphoid leukemia (55%) and acute myeloid leukemia (23%), with 86% presenting neutropenic fever. Alternaria infections manifested as invasive rhinosinusitis (77%), skin lesions resembling ecthyma (14%), and pulmonary infection (9%). Notably, 76% of sinusitis cases exhibited suggestive symptoms. Voriconazole treatment led to a 90% recovery rate, irrespective of surgery. Two fatalities were unrelated to the infections. This study, the largest on Alternaria infections in children, emphasizes their occurrence in leukemia patients with neutropenic fever, showcasing common clinical presentations and a favorable prognosis despite underlying diseases.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"185-189"},"PeriodicalIF":0.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143567473","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":"Catheter-related Bloodstream Infection in Pediatric Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation.","authors":"Seval Özen, Volkan Köse, Yunus M Akçabelen, Fatih Üçkardeş, Saliha Kanik Yüksek, Özlem A Bilir, Şerife M Kanbur, Belgin Gülhan, Gülsüm I Bayhan, Ikbal O Bozkaya, Asli N Ö Parlakay, Namik Y Özbek","doi":"10.1097/MPH.0000000000003025","DOIUrl":"10.1097/MPH.0000000000003025","url":null,"abstract":"<p><p>The aim of this study was to identify catheter-related bloodstream infection (CRBSI) episodes, to determine the causative agents and antibiotic susceptibility profiles, demographic characteristics, and clinical outcomes of patients treated in the pediatric bone marrow transplant (BMT) unit between November 2019 and July 2022. Forty patients were included in the study. The median patient age was 7.5 years (range: 1.5 to 19.9 y) and the most common underlying disease was ALL (77.5%). CRBSI was found to be significantly higher in haploidentic donors ( P <0.001). When CRBSI was confirmed, 65% of the patients were neutropenic with a median duration of 17.5 days (range: 3 to 150). It was found that the mean time to CVC infection was 22 days (range: 5 to 118). As a result of multivariate logistic analysis (OR: 1.038 [95% CI: 1.007-1.070], P =0.018) of the time of infection of the catheter and mortality, it was determined that the mortality rate increased as the duration of the catheter remained. CRBSI was detected in 41.2% of transplanted patients and the overall mortality rate attributed to this complication was 10%. Among the patients, 22 (55%) were colonized before hematopoietic stem cell transplantation (HSCT), and Gram-negative agents (n=15, 68%) mostly accounted for colonization. Gram-negative pathogens (60%) were found to be statistically significantly more common in CRBSI ( P <0.01). The most common causative agent was K. pneumoniae (n=13, 32.5%). Of the Gram-negative isolates (n=24), 17 (70.8%) were multidrug-resistant organisms (MDRO). A fluoroquinolone (80%) was used for antibiotic prophylaxis. Among patients with CRBSI, 65% had a fluoroquinolone-resistant isolate. We found a high rate of quinolone resistance among CRBSI isolates after the use of fluoroquinolone prophylaxis at our unit.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"155-160"},"PeriodicalIF":0.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143763227","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":"Dupilumab for Atopic Dermatitis-like Chronic Graft-versus-Host Disease in Three Pediatric Hematopoietic Cell Transplant Recipients.","authors":"Savannah L Ishee, Meredith M Jenkins, Margaret M Jones, James A Connelly","doi":"10.1097/MPH.0000000000003018","DOIUrl":"10.1097/MPH.0000000000003018","url":null,"abstract":"<p><p>Atopic dermatitis (AD)-like graft-versus-host disease (GVHD) is a chronic form of skin GVHD with features that include erythema, xerosis, scaling, and pruritus. Patients often require treatment with systemic immunosuppression and aggressive topical therapies for relief. Long-term effects of chronic immunosuppression are undesirable and alternative therapies are needed. A retrospective review of 3 patients receiving dupilumab (DUP) for AD-like cGVHD was conducted. Data collected included demographics, transplant history, and GVHD management and outcomes. Information relating to DUP included dose, route, frequency, and safety based on dermatologic reactions, ocular toxicities, and infections. Patients had differing underlying conditions, transplant types, cell sources, and GVHD prophylactic therapies. Three patients received tacrolimus and topical corticosteroids for GVHD treatment, and 1 also received sirolimus and ruxolitinib. After the initial DUP dose, all patients experienced improvement in their GVHD. To date, all patients have complete remission of their skin cGVHD and have weaned off other therapies. No patients experienced dermatologic or ocular toxicities, and no infections were reported. DUP was efficacious and safe for treating AD-like cGVHD in our 3 pediatric patients. Further investigations are warranted to determine the appropriate placement in therapy.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e128-e130"},"PeriodicalIF":0.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143630575","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":"Full House Agglutination - An Interesting Finding in Mycoplasma pneumoniae Infection.","authors":"Jayashree D Kulkarni, Arjun Kashyap","doi":"10.1097/MPH.0000000000003015","DOIUrl":"10.1097/MPH.0000000000003015","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"208-209"},"PeriodicalIF":0.9,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143557204","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}