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Journal Pediatr Blood Cancer

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ONCOPEDS: A mobile application to improve early diagnosis and timely referral in childhood cancer in a low- and middle-income country-A pilot study.

Liliana Vásquez, Jacqueline Montoya, Cecilia Ugaz, Ligia Ríos, Esmeralda León, Iván Maza, Essy Maradiegue, Sharon Chávez, Fanny Tarrillo, Rosdali Diaz, Claudia Pascual, Ninoska Rojas, Mariela Tello, Carla Moore, Darshi Shah, Bernardette Cotrina, Juan Bartolo, Jorge Perez, Víctor Palacios,

<h4>Background</h4>Diagnosis delay in children and adolescents with cancer is a public health problem in Peru that leads to high rates of advanced disease and mortality. We aimed to assess the implementation feasibility and potential utility of ONCOpeds®, a mobile application that provides consultations with pediatric oncologists, in reducing the latency ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28908]

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Genetic factors involved in delayed methotrexate elimination in children with acute lymphoblastic leukemia.

Yu Cheng, Mao-Hua Chen, Qian Zhuang, Bi-Juan Lin, Ying-Ying Chen, Ling Yang, Mao-Bai Liu, Wan-Cai Que, Hong-Qiang Qiu,

<h4>Background</h4>Delayed excretion of methotrexate can lead to life-threatening toxicity that may result in treatment cessation, irreversible organ damage, and death. Various factors have been demonstrated to influence the pharmacokinetic process of methotrexate, including genetic and nongenetic factors.<h4>Methods</h4>We investigated the genetic factors primarily related to the metabolic pathway of methotrexate in ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28858]

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Suboptimal outcome for patients with biliary rhabdomyosarcoma treated on low-risk clinical trials: A report from the Children's Oncology Group.

Jamie M Aye, Wei Xue, Joshua D Palmer, David O Walterhouse, Michael A Arnold, Todd E Heaton, Rajkumar Venkatramani,

<h4>Background</h4>Biliary rhabdomyosarcoma (RMS) is the most common biliary tumor in children. The biliary tract is classified as a favorable primary site. Therefore, patients with localized biliary RMS were included in two consecutive low-risk studies, D9602 and ARST0331, by the Children's Oncology Group (COG). The outcome for these patients treated with ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28914]

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Comparison of chemotherapy with or without asparaginase for extranodal nasal NK/T-cell lymphoma in children and adolescents.

Zijun Zhen, Huiqiang Huang, Tongyu Lin, Zhiming Li, Xi Zhen, Zhongjun Xia, Jia Zhu, Suying Lu, Feifei Sun, Juan Wang, Junting Huang, Xiaofei Sun,

<h4>Purpose</h4>As extranodal natural killer/T-cell lymphoma (ENKTL) occurs rarely in children and adolescents, standardized therapy is yet to be determined. This study aimed to describe the clinical features and determine the optimal chemotherapy regimen for childhood ENKTL.<h4>Methods</h4>The treatment outcomes of radiotherapy combined with asparaginase-based (P-GEMOX or P-GMED) or asparaginase-absent chemotherapy regimens ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28901]

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Active learning for fellows: The hematopathology "unknown case".

Rahela Aziz-Bose, Gerald Wertheim, Leslie Kersun,

The pediatric hematology/oncology fellowship program at the Children's Hospital of Philadelphia set out to create a case-based learning curriculum for common hematologic malignancies that would apply principles of adult learning theory and improve fellows' retention of information in a supportive, goal-oriented learning environment. A framework we employed in developing this ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28895]

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General cancer screening practices among adult survivors of retinoblastoma: Results from the Retinoblastoma Survivor Study.

Joanne F Chou, Jennifer S Ford, Ruth A Kleinerman, David H Abramson, Jasmine H Francis, Charles A Sklar, Kevin C Oeffinger, Leslie L Robison, Ira J Dunkel, Danielle Novetsky Friedman,

We assessed breast, cervical, and colorectal cancer screening practices in adult retinoblastoma (Rb) survivors and non-Rb controls. We found that most Rb survivors adhered to general population cancer screening recommendations. Rates did not differ among Rb survivors and non-Rb controls, or among survivors by laterality, even though bilateral survivors reported ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28873]

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Parental decision making regarding consent to randomization on Children's Oncology Group AALL0932.

Kellee Parker, Erika Cottrell, Linda Stork, Susan Lindemulder,

<h4>Background</h4>Within pediatric oncology, parental decision making regarding participation in clinical trials that aim to reduce therapy to mitigate side effects is not well studied. The recently completed Children's Oncology Group trial for standard-risk acute lymphoblastic leukemia (AALL0932) included a reduction in maintenance therapy, and required consent for randomization immediately prior ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28907]

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Acquired factor XIII deficiency in a child with pure erythroid leukemia.

Guillaume Feugray, Nimrod Buchbinder, Gérard Buchonnet, Odile Fenneteau, Paul Billoir, Véronique Le Cam Duchez,

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28890]

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Donor characteristics and intraoperative total nucleated cell count influence hematopoietic progenitor cell yield of healthy donor bone marrow grafts.

Jacob Kalin, Anh Thy H Nguyen, Benjamin Oshrine,

<h4>Background</h4>Bone marrow graft cell content impacts engraftment potential after allogeneic hematopoietic cell transplantation (alloHCT). Surrogates, such as intraoperative total nucleated cell count (ioTNC), are of unclear utility in predicting final graft characteristics. In addition, demographic and clinical factors may influence graft cellular profile and recipient engraftment.<h4>Procedure</h4>We retrospectively reviewed marrow harvests ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28902]

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Age at diagnosis as a prognostic factor in South African children with neuroblastoma.

Jaques van Heerden, Tonya M Esterhuizen, Marc Hendricks, Janet Poole, Ané Büchner, Gita Naidu, Jan du Plessis, Barry van Emmenes, Ronelle Uys, G P Hadley, Mariana Kruger,

<h4>Purpose</h4>Low- and middle-income countries (LMICs) reported a higher median age at diagnosis of neuroblastoma (NB) compared to high-income countries. The aim was to determine if the optimal age at diagnosis, which maximizes the difference in overall survival between younger versus older patients in the South African population was similar to ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28878]

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Acute hepatic encephalopathy and multiorgan failure in sickle cell disease and COVID-19.

Giulia M Martone, Priyanka M Nanjireddy, Robin A Craig, Andrew J Prout, Meghan A Higman, Kara M Kelly, Steven J Ambrusko,

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28874]

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Disease-specific hospitalizations among 5-year survivors of Wilms tumor: A Nordic population-based cohort study.

Stine Høgsholt, Peter Haubjerg Asdahl, Trine Gade Bonnesen, Anna Sällfors Holmqvist, Laura Madanat-Harjuoja, Laufey Tryggvadottir, Andrea Bautz, Vanna Albieri, Daniel Green, Jeanette Falck Winther, Henrik Hasle,

<h4>Background</h4>With modern therapy, over 90% of Wilms tumor patients can expect to become long-term survivors, and focus on morbidity and late effects become increasingly important. We provide a novel evaluation and insight to subsequent hospitalizations in 5-year survivors of Wilms tumor.<h4>Methods</h4>As part of the Adult Life after Childhood Cancer in ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28905]

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Cancer incidence and survival trends among infants in the United States from 1975 to 2014.

Haijun Wang, Maria Carmenza Mejia, Sandra J Gonzalez, Roger J Zoorob, Weiwen Chai, Xianglin L Du,

<h4>Background</h4>Cancer among infants (<1 year old) has unique epidemiologic, clinical, and genetic characteristics compared with cancer in older children. Nonetheless, data on secular trends in infant cancer incidence and survival in the United States is sparse.<h4>Methods</h4>Population-based data from nine areas of the Surveillance Epidemiology and End Results (SEER) were used to ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28917]

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Bone marrow trephine biopsies from posterior superior iliac crest in neonates.

Sandip Bartakke, Umesh Lukade, Shrividya Sethuratnam,

Cytopenias are common among neonates in neonatal intensive care units (NICU). Although, bone marrow aspirations (BMAs) are often performed as part of diagnostic workup, but trephine bone marrow biopsies (BMBs) have not been reported from living neonates. BMB is indispensable to accurately assess the cellularity and architecture. There is paucity ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28893]

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The role of interferon-gamma and its signaling pathway in pediatric hematological disorders.

Pietro Merli, Concetta Quintarelli, Luisa Strocchio, Franco Locatelli,

Interferon-gamma (IFN-γ) plays a key role in the pathophysiology of hemophagocytic lymphohistiocytosis (HLH), and available evidence also points to a role in other conditions, including aplastic anemia (AA) and graft failure following allogeneic hematopoietic stem cell transplantation. Recently, the therapeutic potential of IFN-γ inhibition has been documented; emapalumab, an anti-IFN-γ ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28900]

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Pretransplant myeloid and immune suppression, reduced toxicity conditioning with posttransplant cyclophosphamide: Initial outcomes of novel approach for matched unrelated donor hematopoietic stem cell transplant for hemoglobinopathies.

Gaurav Kharya, Atish N Bakane, Archana M Rauthan,

Hematopoietic stem cell transplant (HSCT) is currently the only curative option for thalassemia major (TM) and sickle cell disease (SCD). We report our experience of using pretransplant immune suppression (PTIS), augmented Johns Hopkins conditioning, and posttransplant cyclophosphamide (PTCy) as graft-versus-host disease (GvHD) prophylaxis for matched unrelated donor (MUD) transplant in ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28909]

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Female reproductive function after treatment of childhood acute lymphoblastic leukemia.

Roxanne Roshandel, Marloes van Dijk, Annelies Overbeek, Gertjan Kaspers, Cornelis Lambalk, Catharina Beerendonk, Dorine Bresters, Margriet van der Heiden-van der Loo, Marry van den Heuvel-Eibrink, Leontien Kremer, Jacqueline Loonen, Helena van der Pal, Cecile Ronckers, Wim Tissing, Birgitta Versluys, Flora van Leeuwen, Marleen van den Berg, Eline van Dulmen-den Broeder, ,

<h4>Background</h4>The aim was to evaluate self-reported reproductive characteristics and markers of ovarian function in a nationwide cohort of female survivors of childhood acute lymphoblastic leukemia (ALL), because prior investigations have produced conflicting data.<h4>Procedure</h4>Self-reported reproductive characteristics were assessed by questionnaire among 357 adult 5-year survivors, treated between 1964 and 2002, and ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28894]

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Metastatic progression in infants diagnosed with stage 4S neuroblastoma. A study of the Italian Neuroblastoma Registry.

Stefano Parodi, Stefania Sorrentino, Andrea Di Cataldo, Annalisa Tondo, Anna Maria Fagnani, Patrizia Perri, Anna Rita Gigliotti, Giovanni Erminio, Maria Valeria Corrias, Bruno De Bernardi,

<h4>Purpose</h4>Stage 4S neuroblastoma, a tumor affecting infants, is characterized by the capacity to regress spontaneously and high cure rate. About a third of these infants undergo tumor progression requiring antitumor treatment and 10-15% eventually die. In case of metastatic progression, it may occur either at 4S sites (mainly liver) or ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28904]

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Domino donor lymphocyte infusion for secondary poor graft function after HLA-mismatched allogeneic stem cell transplantation between HLA-identical sibling pairs with congenital immunodeficiency.

Satoru Hamada, Taichi Uehara, Jiro Miyamoto, Shinobu Kiyuna, Tokiko Oshiro, Takeshi Yagi, Shingo Kurokawa, Nobuyuki Hyakuna, Koichi Nakanishi,

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28851]

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Medulloblastoma and familial adenomatous polyposis: Good prognosis and good quality of life in the long-term?

Maura Massimino, Stefano Signoroni, Luna Boschetti, Veronica Biassoni, Elisabetta Schiavello, Andrea Ferrari, Filippo Spreafico, Monica Terenziani, Stefano Chiaravalli, Nadia Puma, Luca Bergamaschi, Maria Teresa Ricci, Laura Cattaneo, Giovanna Gattuso, Francesca Romana Buttarelli, Francesca Gianno, Evelina Miele, Geraldina Poggi, Marco Vitellaro,

<h4>Introduction</h4>Mutations of the APC (adenomatous polyposis coli) gene correlate mainly with familial adenomatous polyposis (FAP), but can occasionally be pathogenic for medulloblastoma (MBL) wingless-related integration site (WNT) subtype, the course of which has only recently been described.<h4>Methods</h4>We retrieved all patients with documented germline APC mutations and a diagnosis of MBL ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28912]

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Effect of COVID-19 on anakinra-induced remission in homozygous STX11 hemophagocytosis lymphohistiocytosis.

Anshul Vagrecha, Hiren B Patel, Tania Mamdouhi, Suchitra Acharya, Abena Appiah-Kubi, Banu Aygun, Adrianna Vlachos, Lawrence C Wolfe, Jeffrey M Lipton, Randy Q Cron, Carolyn Fein Levy,

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28897]

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Discrepancies between F-18-FDG PET/CT findings and conventional imaging in Langerhans cell histiocytosis.

Justin Ferrell, Susan Sharp, Ashish Kumar, Michael Jordan, Jennifer Picarsic, Adam Nelson,

<h4>Background</h4>Accurate risk stratification of Langerhans cell histiocytosis (LCH) is essential as management can range from conservative in single system, low risk for central nervous system (CNS) involvement lesions to intensive chemotherapy for multisystem or high-risk disease. Additionally, being able to differentiate metabolically active from inactive lesions is essential for both ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28891]

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Sarcopenia is a prognostic outcome marker in children with high-risk hepatoblastoma.

Annika Ritz, Julian Kolorz, Jochen Hubertus, Julia Ley-Zaporozhan, Dietrich von Schweinitz, Sibylle Koletzko, Beate Häberle, Irene Schmid, Roland Kappler, Michael Berger, Eberhard Lurz,

<h4>Background</h4>Children with hepatoblastoma (HB) are at risk of sarcopenia due to immobility, chemotherapy, and malnutrition. We hypothesized that children with HB have a low preoperative total psoas muscle area (tPMA), reflecting sarcopenia, which negatively impacts outcome.<h4>Procedure</h4>Retrospective study of children (1-10 years) with hepatoblastoma treated at a large university children's hospital from ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28862]

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Clear cell sarcoma of the kidney in Austrian children: Long-term survival after relapse.

Waltraud Friesenbichler, Roswitha Lüftinger, Gabriele Kropshofer, Martin Henkel, Gabriele Amann, Rhoikos Furtwängler, Norbert Graf, Leo Kager,

<h4>Introduction</h4>Clear cell sarcoma of the kidney (CCSK) is a rare malignant childhood renal tumour. Recently, the central nervous system (CNS) was found to be the most frequent site of relapse associated with a poor outcome. Optimal treatment strategies are scarce.<h4>Patients and methods</h4>Retrospective data analysis of all Austrian children with CCSK. ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28860]

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A phase 1 study of entinostat in children and adolescents with recurrent or refractory solid tumors, including CNS tumors: Trial ADVL1513, Pediatric Early Phase-Clinical Trial Network (PEP-CTN).

Andrew Bukowinski, Bill Chang, Joel M Reid, Xiaowei Liu, Charles G Minard, Jane B Trepel, Min-Jung Lee, Elizabeth Fox, Brenda J Weigel,

<h4>Background</h4>Entinostat is an oral small molecule inhibitor of class I histone deacetylases (HDAC), which has not previously been evaluated in pediatrics. We conducted a phase I trial to determine the maximum tolerated dose/recommended phase 2 dose (MTD/RP2D), toxicity profile, pharmacokinetics (PK), and pharmacodynamics (PD) of entinostat in children with relapsed ... Read more >>

Pediatr Blood Cancer (Pediatric blood & cancer)
[2021, :e28892]

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