journal
https://read.qxmd.com/read/38644612/extramedullary-infiltration-in-pediatric-acute-myeloid-leukemia-results-from-the-therapeutically-applicable-research-to-generate-effective-treatments-target-initiative
#1
JOURNAL ARTICLE
Weiya Li, Mingyue Shi, Pan Zhou, Ying Liu, Xiaobo Liu, Xingjun Xiao, Suqiong Zuo, Yanliang Bai, Kai Sun
BACKGROUND: The outcome of extramedullary infiltration (EMI) in pediatric acute myeloid leukemia (AML) is controversial, and little is known about the implications of stem cell transplantation (SCT) and gemtuzumab ozogamicin (GO) treatment on patients with EMI. METHODS: We retrieved the clinical data of 713 pediatric patients with AML from the Therapeutically Applicable Research to Generate Effective Treatments (TARGET) dataset, and analyzed the clinical and prognostic characteristics of patients with EMI at diagnosis and relapse...
April 21, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38644611/early-diagnosis-of-sickle-cell-retinopathy-by-using-ocular-coherence-tomography-in-pediatric-population-7-18-years-in-central-india
#2
JOURNAL ARTICLE
Pooja Soni, Bhavna Dhingra, Samendra Karkhur, Narendra K Chaudhary, Abhijit P Pakhare
BACKGROUND: Sickle cell disease (SCD) is the commonest inherited blood disorder leading to complications occurring due to vaso-occlusion including sight-threatening retinopathy. Retinopathy can be managed if diagnosed early and vision loss can be prevented. Since, very less data are available from India, hence, this study was conducted in children (7-18 years) with SCD to diagnose retinopathy by using ocular coherence tomography (OCT) in subclinical stages. METHODS: This cross sectional single-center study was performed in 7-18 years age group children with SCD without any visual symptoms...
April 21, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38644609/two-siblings-with-fanconi-anemia-fancq-ercc4-xpf-presenting-with-tumor-mimicking-lesions-in-the-brain-and-acute-neurological-deterioration
#3
JOURNAL ARTICLE
Zeynep Canan Özdemir, Coşkun Yarar, Çiğdem Öztunalı, Ersin Tötret, Kürşat Bora Çarman, Özcan Bör
The complementation Q group (FANCQ) subtype of Fanconi anemia (FA) caused by the ERCC4/XPF mutation is very rare. Two siblings, aged 13 and 10 with Fanconi phenotypic features, presented with right hemiparesis and focal-onset seizures. In both cases, cranial magnetic resonance imaging (MRI) showed mass-like lesions accompanied by peripheral edema and calcification. In one case, oral steroid treatment and surgical excision were performed, while in the other case, the cranial lesion regressed just with steroid treatment and without surgery...
April 21, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38644606/late-onset-lymphopenia-during-radiation-is-associated-with-an-increased-risk-of-tumor-recurrence-in-newly-diagnosed-pediatric-medulloblastoma
#4
JOURNAL ARTICLE
Holly B Lindsay, Michael E Scheurer, Anthony K Allam, Bryony J Lucas, Susan L McGovern, Murali Chintagumpala, Arnold C Paulino
BACKGROUND: Recent data found a correlation between lymphopenia occurring early during craniospinal radiation therapy (RT) and risk of disease recurrence in newly diagnosed childhood medulloblastoma. However, the population included patients who received chemotherapy prior to or during RT. Here, we investigate the effect of lymphopenia during RT in patients with newly diagnosed pediatric medulloblastoma who were chemotherapy-naïve. PROCEDURE: We analyzed 79 patients with newly diagnosed medulloblastoma (ages 2-21 years) treated between 1997 and 2013 with craniospinal RT...
April 21, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38644601/quality-of-life-in-children-and-adolescents-after-treatment-for-acute-lymphoblastic-leukemia-according-to-the-nopho-all2008-protocol
#5
JOURNAL ARTICLE
Nina Mogensen, Ulrika Kreicbergs, Birgitte Klug Albertsen, Päivi M Lähteenmäki, Mats Heyman, Arja Harila
BACKGROUND: The improved outcome of childhood acute lymphoblastic leukemia (ALL) over the last decades has increased the importance of assessing late effects and health-related quality of life (HRQoL), particularly when evaluating and comparing outcomes in clinical trials. This study aimed to assess HRQoL in children treated for ALL according to the NOPHO ALL2008 protocol. PROCEDURE: Children, aged 1 to less than 18 years at diagnosis, alive in first remission, and their parents, were asked to complete PedsQL 4...
April 21, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38644596/impact-of-sociodemographic-factors-stress-and-communication-on-health-related-quality-of-life-in-survivors-of-pediatric-cancer
#6
JOURNAL ARTICLE
Valdeoso Patterson, Anna Olsavsky, Dana Garcia, Malcolm Sutherland-Foggio, Kathryn Vannatta, Kemar V Prussien, Heather Bemis, Bruce E Compas, Cynthia A Gerhardt
BACKGROUND: While most research has largely focused on medical risks associated with reduced health-related quality of life (HRQOL) in survivors, sociodemographic and family factors may also play a role. Thus, we longitudinally examined sociodemographic factors and family factors associated with survivor HRQOL, including adolescent's cancer-specific stress, mother's general stress, and mother-adolescent communication. METHODS: Mothers (N = 80) and survivors (ages 10-23, N = 50) were assessed 5 years following initial diagnosis...
April 21, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38644595/association-of-elevated-tricuspid-regurgitation-velocity-with-cerebrovascular-and-kidney-disease-in-children-with-sickle-cell-disease
#7
JOURNAL ARTICLE
Chibuzo Ilonze, Parul Rai, Najibah Galadanci, Rima Zahr, Victoria I Okhomina, Guolian Kang, Dakshin Padmanabhan, Jeffrey Lebensburger, Ammar Saadoon Alishlash
BACKGROUND: Tricuspid regurgitation velocity (TRV), measured by echocardiography, is a surrogate marker for pulmonary hypertension. Limited pediatric studies have considered the association between TRV and surrogate markers of end-organ disease. METHODS: We conducted a cross-sectional study that evaluated the prevalence of elevated TRV ≥2.5 m/s and its associations with renal and cerebrovascular outcomes in children with sickle cell disease (SCD) 1-21 years of age in two large sickle cell cohorts, the University of Alabama at Birmingham (UAB) sickle cell cohort, and the Sickle Cell Clinical Research and Intervention Program (SCCRIP) cohort at St...
April 21, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38637906/two-cases-of-hepatoblastoma-in-bohring-opitz-syndrome-an-emerging-association
#8
LETTER
Kritika Patel, Shelly McQuaid, Tyler Ketterl, Daniel J Benedetti, Elizabeth Sokol
No abstract text is available yet for this article.
April 18, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38637875/pleuroparenchymal-fibroelastosis-as-a-late-complication-of-childhood-cancer-therapy-a-case-series
#9
JOURNAL ARTICLE
Priya H Marathe, Valeria Santibanez, Paul A Meyers, Maria L Padilla, Danielle N Friedman
Pleuroparenchymal fibroelastosis (PPFE) is a rare interstitial pneumonia with distinct clinicopathologic features. It has been associated with exposure to hematopoietic stem cell transplantation (HSCT) and classical alkylating agents. Here, we highlight PPFE as a late complication of childhood cancer therapy by describing the cases of four survivors of childhood cancer with a diagnosis of treatment-related PPFE. All patients received high-dose alkylating agents. PPFE should be considered in the differential diagnosis of restrictive lung disease in patients with history of exposure to alkylating agents or HSCT...
April 18, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38637871/bilateral-wilms-tumor-with-anaplasia-a-report-from-the-children-s-oncology-group-study-aren0534
#10
JOURNAL ARTICLE
Rodrigo L P Romao, Jennifer H Aldrink, Lindsay A Renfro, Elizabeth A Mullen, Andrew J Murphy, Jack Brzezinski, Marcus M Malek, Daniel J Benedetti, Nicholas G Cost, Ethan Smith, Jeffrey S Dome, Andrew M Davidoff, Amy Treece, Lauren N Parsons, Conrad V Fernandez, Brett Tornwall, Robert C Shamberger, Arnold Paulino, John A Kalapurakal, James I Geller, Peter F Ehrlich
INTRODUCTION: The purpose of this study is to examine the outcomes in children with anaplastic bilateral Wilms tumor (BWT) from study AREN0534 in order to define potential prognostic factors and areas to target in future clinical trials. METHODS: Demographic and clinical data from AREN0534 study patients with anaplasia (focal anaplasia [FA], or diffuse anaplasia [DA]) were compared. Event-free survival (EFS) and overall survival (OS) were reported using Kaplan-Meier estimation with 95% confidence bands, and differences in outcomes between FA and DA compared using log-rank tests...
April 18, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38637852/ikzf1-plus-alterations-contribute-to-outcome-disparities-in-hispanic-latino-children-with-b-lymphoblastic-leukemia
#11
JOURNAL ARTICLE
Alexandra E Kovach, Maximilian Wengyn, My H Vu, Andrew Doan, Gordana Raca, Deepa Bhojwani
BACKGROUND: Compared to other ethnicities, Hispanics/Latinos (H/L) have a high incidence of acute lymphoblastic leukemia (ALL), enrichment of unfavorable ALL genetic subtypes, and worse outcomes, even after correcting for socioeconomic factors. We previously demonstrated increased incidence of the high-risk genetic drivers IKZF1 deletion and IGH::CRLF2 rearrangement in H/L compared to non-H/L children with B-ALL. Here in an expanded pediatric cohort, we sought to identify novel genetic drivers and secondary genetic alterations in B-ALL associated with H/L ethnicity...
April 18, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38627891/discovering-needs-for-palliative-care-in-children-with-cancer-in-indonesia
#12
JOURNAL ARTICLE
Alexandra Widita Swipratami Pangarso, Sri Mulatsih, Mei Neni Sitaresmi, Susanne Verhulst, Gertjan Kaspers, Saskia Mostert
BACKGROUND: Although most children with cancer die in low- and middle-income countries, palliative care receives limited attention in these settings. This study explores parents' perspectives on experiences and needs of children dying from cancer. METHODS: Home visits were conducted to interview parents of children, who were treated for cancer at an Indonesian academic hospital and died between 2019 and 2020, using semi-structured questionnaires. RESULTS: Parents of 49 children (response rate 74%) were interviewed...
April 16, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38627882/event-free-survival-in-relapsed-and-refractory-rhabdomyosarcoma-treated-on-cooperative-group-phase-ii-trials-a-report-from-the-children-s-oncology-group
#13
JOURNAL ARTICLE
Jonathan Metts, Wei Xue, Zhengya Gao, Sapna Oberoi, Aaron R Weiss, Rajkumar Venkatramani, Douglas J Harrison
BACKGROUND: Novel therapies are needed for relapsed and refractory rhabdomyosarcoma (RRMS). Phase II clinical trials in RRMS have typically utilized radiologic response as the primary activity endpoint, an approach that poses several limitations in RRMS. In this analysis, we aimed to estimate an event-free survival (EFS) endpoint for RRMS that could be used as a benchmark for future studies. PROCEDURE: We performed a retrospective study of patients with RRMS enrolling on 13 single-agent phase II Children's Oncology Group and legacy group trials from 1997 to 2016...
April 16, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38622789/disease-modifying-therapies-for-sickle-cell-disease-decisional-needs-and-supports-among-adolescents-and-young-adults
#14
JOURNAL ARTICLE
Ke Ding, Benjamin Bear, Erica Sood, Melissa Alderfer, Lori Crosby, Aimee K Hildenbrand
BACKGROUND: Shared decision-making is one promising solution to addressing barriers in use of disease-modifying therapies for adolescents and young adults (AYAs) with sickle cell disease (SCD). A thorough understanding of decisional needs can guide the development of decisional supports and promote shared decision-making. PROCEDURE: Informed by the Ottawa Decision Support Framework (ODSF), we conducted a qualitative analysis to assess decisional needs and supports reported by AYAs with SCD, their caregivers, and healthcare providers...
April 15, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38616403/boron-neutron-capture-therapy-prolongs-survival-in-a-patient-with-a-recurrent-malignant-peripheral-nerve-sheath-tumor-a-case-report
#15
LETTER
Yuko Watanabe, Yi-Wei Chen, Hiroshi Igaki, Ayumu Arakawa, Kayoko Tao, Masanaka Sugiyama, Miho Nakajima, Nami Shirakawa, Shunsuke Yanagisawa, Yasuji Miyakita, Akihiko Yoshida, Kayako Isohashi, Koji Ono, Yoshitaka Narita, Chitose Ogawa
No abstract text is available yet for this article.
April 14, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38616383/epstein-barr-virus-associated-smooth-muscle-tumor-in-a-female-with-ataxia-telangiectasia-a-case-report
#16
LETTER
Beatrice Rivalta, Paola Zangari, Lucia Pacillo, Emma Concetta Manno, Veronica Santilli, Gioacchino Andrea Rotulo, Nicola Cotugno, Chiara Rossetti, Silvia Vallese, Maria Giovanna Paglietti, Paolo Tomà, Valerio Pardi, Alessandro Inserra, Paola Francalanci, Giuseppe Maria Milano, Rita Alaggio, Caterina Cancrini, Andrea Finocchi, Paolo Palma, Donato Amodio
No abstract text is available yet for this article.
April 14, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38616378/concurrent-primary-mediastinal-germ-cell-tumor-and-acute-myeloid-leukemia-case-report-of-sustained-remission-and-review-of-the-literature
#17
LETTER
Kristina Zalewski, Evan Cantor, Roland Chu, Rajen Mody, Greg Yanik, Laura Sedig
No abstract text is available yet for this article.
April 14, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38616361/characteristics-and-treatment-of-acute-myeloid-neoplasms-with-cutaneous-involvement-in-infants-up-to-6%C3%A2-months-of-age-a-retrospective-study
#18
JOURNAL ARTICLE
Juliette Renaud, Bianca F Goemans, Franco Locatelli, Martina Pigazzi, Shelagh Redmond, Claudia E Kuehni, Alice Destaillats, Todd A Alonzo, Robert B Gerbing, Alan Gamis, Richard Aplenc, Raffaele Renella, Todd Cooper, Francesco Ceppi
BACKGROUND: Myeloid neoplasms account for 50% of cases of pediatric leukemias in infants. Approximately 25%-50% of patients with newborn leukemia have cutaneous extramedullary disease (EMD). In less than 10% of patients, aleukemic leukemia cutis or isolated extramedullary disease with cutaneous involvement (cEMD) occurs when skin lesions appear prior to bone marrow involvement and systemic symptoms. Interestingly, in acute myeloid leukemia with cutaneous EMD (AML-cEMD) and cEMD, spontaneous remissions have been reported...
April 14, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38616355/iron-overload-disorders-growth-and-gonadal-dysfunction-in-childhood-and-adolescence
#19
REVIEW
Marta Tenuta, Biagio Cangiano, Giulia Rastrelli, Francesco Carlomagno, Francesca Sciarra, Andrea Sansone, Andrea M Isidori, Daniele Gianfrilli, Csilla Krausz
Hemochromatosis (HC) is characterized by the progressive accumulation of iron in the body, resulting in organ damage. Endocrine complications are particularly common, especially when the condition manifests in childhood or adolescence, when HC can adversely affect linear growth or pubertal development, with significant repercussions on quality of life even into adulthood. Therefore, a timely and accurate diagnosis of these disorders is mandatory, but sometimes complex for hematologists without endocrinological support...
April 14, 2024: Pediatric Blood & Cancer
https://read.qxmd.com/read/38613166/good-engraftment-after-reduced-intensity-targeted-busulfan-based-conditioning-and-matched-related-donor-hematopoietic-cell-transplantation-in-hemoglobinopathies
#20
JOURNAL ARTICLE
Maximilian Klink, Matthias Felber, Ulrike Zeilhofer, Mathias Hauri-Hohl, Markus Schmugge, Daniel Drozdov, Tayfun Güngör
BACKGROUND: Hematopoietic cell transplantation (HCT) is an established curative therapy for transfusion-dependent thalassemia (TDT) and sickle cell disease (SCD). The latest American Society of Hematology guidelines recommend myeloablative preparative regimen in patients under 18 years of age. PROCEDURE: The objective was to demonstrate safety and efficacy of a reduced intensity conditioning (RIC) regimen including high-dose fludarabine, anti-thymocyte globulin, and targeted busulfan as a single alkylator to sub-myeloablative exposures...
April 12, 2024: Pediatric Blood & Cancer
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