Journal of Pediatric Hematology/Oncology最新文献

Acute Myeloblastic Leukemia in a Child With Duchenne Muscular Dystrophy: A Novel Case and Brief Review of the Literature.

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-03-17 DOI: 10.1097/MPH.0000000000003019

Daniel S Cirotski, Lindsey Sawyer, Wilson File, Linda Pegram

引用次数: 0

Dupilumab for Atopic Dermatitis-like Chronic Graft-versus-Host Disease in Three Pediatric Hematopoietic Cell Transplant Recipients.

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-03-11 DOI: 10.1097/MPH.0000000000003018

Savannah L Ishee, Meredith M Jenkins, Margaret M Jones, James A Connelly

{"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":"https://doi.org/10.1097/MPH.0000000000003018","url":null,"abstract":"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.","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2025-03-11","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}

引用次数: 0

Ex Vivo Drug Screening: An Emerging Paradigm in the Treatment of Childhood Cancer.

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-03-10 DOI: 10.1097/MPH.0000000000003017

Anees Ahmed, Ellen Cox, Louis Lane, Ola Rominiyi, Sarah Danson, Helen E Bryant, Greg Wells, David King

{"title":"Ex Vivo Drug Screening: An Emerging Paradigm in the Treatment of Childhood Cancer.","authors":"Anees Ahmed, Ellen Cox, Louis Lane, Ola Rominiyi, Sarah Danson, Helen E Bryant, Greg Wells, David King","doi":"10.1097/MPH.0000000000003017","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003017","url":null,"abstract":"Developing and providing the right therapy for the right patient (or personalized targeted treatments) is key to reducing side-effects and improving survival in childhood cancers. Most efforts aiming to personalize childhood cancer treatment use genomic analysis of malignancies to identify potentially targetable genetic events. But it is becoming clear that not all patients will have an actionable change, and in those that do there is no additional way to determine if treatments will be effective. Ex vivo drug screening is a laboratory technique used to test the effects of various drugs or compounds, on biological tissues or cells that have been removed from an organism. This information is then used to predict which cancer treatments will be most effective based on the therapeutic response in the tissue or cells removed from that individual. Its utility in personalizing treatments in childhood cancer is increasingly recognized. In this review we describe the different methods for ex vivo drug screening and the advantages and disadvantages of each technique. We also present recent evidence that ex vivo screening may have utility in a variety of childhood malignancies including an overview of current clinical trials appraising its use. Finally, we discuss the research questions and hurdles that must be overcome before ex vivo screening can be widely used in pediatric oncology.","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2025-03-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143630576","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}

引用次数: 0

Sustained Clinical Remission of BRAF V600E-Mutated Langerhans Cell Histiocytosis With Multiorgan Involvement in an Infant Treated With Dabrafenib.

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-03-06 DOI: 10.1097/MPH.0000000000003014

Franziska Cuntz, Jean Donadieu, Susanne Holzhauer

引用次数: 0

Alternaria Invasive Infection in Children With Hemato-Oncological Disease: A National Multicenter Report.

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-03-06 DOI: 10.1097/MPH.0000000000003016

Aviv Sever, Chen Rosenberg Danziger, Nimrod Sachs, Salvador Fisher, Efraim Bilavsky, Gilad Sherman, Yael Shachor-Meyouhas, Galia Grisaru, Haim Ben Zvi

{"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":"https://doi.org/10.1097/MPH.0000000000003016","url":null,"abstract":"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.","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2025-03-06","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}

引用次数: 0

Full House Agglutination - An Interesting Finding in Mycoplasma Pneumoniae Infection. 满屋凝集--肺炎支原体感染中的有趣发现

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-03-05 DOI: 10.1097/MPH.0000000000003015

Jayashree D Kulkarni, Arjun Kashyap

引用次数: 0

Novel SMARCA4 Variant in an Infant With Atypical Teratoid Rhabdoid Tumor.

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-03-03 DOI: 10.1097/MPH.0000000000003004

Shria K Haldipurkar, Sudarshawn N Damodharan, Pamela Rathbun, Kai Lee Yap, Nitin Wadhwani, Angela J Waanders

引用次数: 0

Hereditary Pyropoikilocytosis as a Modifier of Sickle Cell Disease Severity.

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-03-03 DOI: 10.1097/MPH.0000000000003012

Megan Lantz, Lily Dolatshahi

引用次数: 0

Rare Inherited Coagulation Deficiencies: A Single-center Study. 罕见的遗传性凝血功能缺陷：一项单中心研究。

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-03-01 Epub Date: 2024-12-16 DOI: 10.1097/MPH.0000000000002985

Özlem Terzi, Sadik Sami Hatipoğlu

{"title":"Rare Inherited Coagulation Deficiencies: A Single-center Study.","authors":"Özlem Terzi, Sadik Sami Hatipoğlu","doi":"10.1097/MPH.0000000000002985","DOIUrl":"10.1097/MPH.0000000000002985","url":null,"abstract":"Background: Rare factor deficiency (RFD) is characterized by a deficiency of factor (F)I, FII, FV, FVII, FX, FXI, FXII, FXIII, or a combined deficiency of FV+FVIII or vitamin K-dependent factors. The prevalence of RFD ranges from 1/1,000,000 to 3,000,000. Combined deficiencies of vitamin K-related factors have been described in 30 families worldwide, and these patients can present with a wide range of clinical symptoms, from mucocutaneous bleeding to life-threatening symptoms such as central nervous system and gastrointestinal bleeding.Objective: This study aimed to contribute to the literature on RFD.Material and methods: This retrospective study analyzed data from 43 children with RFD.Results: The most common factor deficiencies were FVII (n=13); whereas the other deficiencies were FI (n=1), FV (n=2), FV+FVIII (n=2), FX (n=6), FXI (n=5), FXII (n=9), FXIII (n=3), and vitamin K-dependent combined factor deficiency (n=2). Acute and severe bleeding was controlled by treatment in 6 patients, and 12 patients with recurrent bleeding symptoms received prophylaxis. RFDs were more common in regions with high rates of consanguineous marriage, and in our study, 16 (16/43) of the cases were found to have consanguineous marriages between parents.Conclusions: It is important to improve genetic counseling and access to testing for family members with RFD due to autosomal recessive inheritance. Delays in diagnosis and treatment and lack of adequate prevention are important risk factors for life-threatening bleeding.","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e90-e95"},"PeriodicalIF":0.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142882411","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}

引用次数: 0

An Atypical Case of Neonatal Alloimmune Thrombocytopenia. 新生儿同种免疫性血小板减少症1例。

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-03-01 Epub Date: 2025-01-10 DOI: 10.1097/MPH.0000000000002988

Gülsen Mutluoglu, Barbara De Muynck, Marie-Paule Emonds, Tom Van Maerken

{"title":"An Atypical Case of Neonatal Alloimmune Thrombocytopenia.","authors":"Gülsen Mutluoglu, Barbara De Muynck, Marie-Paule Emonds, Tom Van Maerken","doi":"10.1097/MPH.0000000000002988","DOIUrl":"10.1097/MPH.0000000000002988","url":null,"abstract":"Fetal and neonatal alloimmune thrombocytopenia (FNAIT) results from maternal antibodies targeting fetal platelets during pregnancy, often causing hemorrhagic manifestations detectable antenatally or shortly after birth. We report an atypical form of FNAIT with delayed onset in a healthy, breastfed male infant who developed diffuse petechiae 2 weeks after birth due to severe thrombocytopenia. The mother was shown to be negative for the human platelet antigen-1a (HPA-1a) allele but had anti-HPA-1a IgG antibodies, while the father and newborn were HPA-1a positive, confirming the diagnosis. Despite intravenous immunoglobulins and platelet transfusions, the recovery was slow. Analysis of breast milk demonstrated the presence of anti-HPA-1a IgG antibodies. The unusual clinical presentation 2 weeks after birth and the slow platelet recovery under appropriate treatment suggest postnatal transfer of maternal anti-HPA-1a antibodies or B lymphocytes producing these antibodies to the newborn, which may possibly have occurred through breastfeeding. Further research is needed to validate these findings and understand the role of breast milk in provoking the disease. Early detection and management remain essential to prevent serious complications associated with FNAIT.","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e125-e127"},"PeriodicalIF":0.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142962294","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}

引用次数: 0