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Journal of Pediatric Hematology/oncology

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https://www.readbyqxmd.com/read/29901560/health-status-and-health-related-quality-of-life-measurement-in-pediatric-cancer-clinical-trials-an-examination-of-the-dfci-00-01-acute-lymphoblastic-leukemia-protocol
#1
Charlene Rae, William Furlong, David Feeny, Rana Couchman, Lewis Silverman, Stephen Sallan, Caroline Laverdiere, Luis Clavell, Bruno Michon, Kara Kelly, Eric Larsen, Eleanor Pullenayegum, Uma Athale, Ronald Barr
Health-related quality of life (HRQL) improved progressively during therapy and beyond in children treated for acute lymphoblastic leukemia on the Dana-Farber Cancer Institute (DFCI) 95-01 protocol. This study aimed to validate that trajectory in a successor study (DFCI 00-01) and to compare the HRQL of patients in the 2 studies. Children aged above 5 years were assessed during each phase of treatment (N=4) and 2 years after completion of therapy. Health status and HRQL were measured using Health Utilities Index (HUI) instruments, HUI2 and HUI3...
June 12, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29901559/aneursymal-bone-cyst-an-uncommon-paraspinal-tumor-in-children
#2
Nilay Shah, Thomas Scharschmidt, Bonita Fung, Miriam Conces, Bhuvana A Setty
Paraspinal tumors with benign histology in the absence of trauma rarely arise in children. Treatment of such benign tumors, in contrast to malignancies, generally consists of surgical resection of the lesion with confirmation of histology via pathologic evaluation. We present a pediatric case of an atraumatic paraspinal mass with a histologic diagnosis of aneurysmal bone cyst, and USP6 gene rearrangement supporting the histologic diagnosis. The patient underwent gross total resection of the paraspinal lesion with no additional intervention...
June 12, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29889805/early-axial-growth-outcomes-of-pediatric-patients-receiving-proton-craniospinal-irradiation
#3
Brian De, Oren Cahlon, Kevin Sine, Dennis Mah, Eugen B Hug, Suzanne L Wolden
Guidelines on proton craniospinal irradiation (p-CSI) target volume selection in children are lacking. We examined the impact of target volume selection on growth of children receiving p-CSI at a institution. Records of 58 patients who received p-CSI were reviewed. Median age at treatment initiation was 8 years (range, 2 to 18 y). Spinal target volumes included whole vertebral body (WVB) in 67% and partial vertebral body (PVB) in 33%. Height z-scores before and after p-CSI were assessed using Centers for Disease Control and Prevention stature-for-age charts...
June 8, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29889804/immune-thrombocytopenia-in-a-child-with-refractory-langerhans-cell-histiocytosis-following-cladribine-containing-therapy
#4
Chow Yee Lai, Tristan Pettit
In this report, we present a young infant with multisystem Langerhans cell histiocytosis, who after cladribine and cytarabine salvage treatment developed immune thrombocytopenia (IT). On review of the literature, there were no previous reports of Langerhans cell histiocytosis-associated IT. Treatment of the IT with intravenous immunoglobulin and oral corticosteroids was unsuccessful. Eltrombopag, in combination with a 4-day course of dexamethasone was commenced as second-line therapy. Platelet recovery occurred 10 days after initiation of eltrombopag...
June 8, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29889803/successful-treatment-of-pediatric-epstein-barr-virus-positive-aggressive-natural-killer-cell-leukemia
#5
Bo Kyung Kim, Kyung Taek Hong, Hyoung Jin Kang, Hong Yul An, Jung Yoon Choi, Che Ry Hong, Kyung Duk Park, Dong Soon Lee, Hee Young Shin
Epstein-Barr virus (EBV)-positive aggressive natural killer-cell leukemia (ANKL) is a rare malignancy of mature natural killer cells, with a very poor survival rate. Patients have a rapidly declining clinical course and a poor prognosis, with a median survival of only a few months. Herein, we describe a 16-year-old boy who was diagnosed with EBV-positive ANKL and successfully treated using combination chemotherapy and a subsequent allogeneic hematopoietic stem cell transplantation (alloHSCT). The patient is disease free 4 years and 9 months after alloHSCT...
June 8, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29889802/microrna-17-microrna-19b-microrna-146a-microrna-302d-expressions-in-hepatoblastoma-and-clinical-importance
#6
Çiğdem Ö Ecevit, Safiye Aktaş, Hülya Tosun Yildirim, Bengü Demirağ, Ayşe Erbay, İrfan Karaca, Ahmet Çelik, Ayşe Banu Demir, Ayşe Pinar Erçetin, Nur Olgun
Hepatoblastoma (HB) is the most common liver malignancy in children. The prognosis changes according to the histologic subtypes of HB. In the present study, we aimed to characterize the expression level of selected microRNAs (miRNAs) in HB as well as in histologic subtypes, and to consider the association with the prognosis. A total of 22 HB tumor samples, subtyped as fetal (n=16) and embryonal (n=6), and 10 nontumorous surrounding liver samples were evaluated in this study. Expressions of miR-17, miR-146a, miR-302d, and miR-19b were analyzed in 22 HB tumor samples and 10 nontumorous surrounding liver samples by quantitative real-time polymerase chain reaction...
June 8, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29889801/the-efficiency-and-toxicity-of-mifamurtide-in-childhood-osteosarcoma
#7
Nurdan Tacyildiz, Sonay Incesoy Ozdemir, Emel Unal, Melda Berber, Handan Dincaslan, Gulsan Yavuz
The aim of the present study was to evaluate the efficiency and side effects of mifamurtide in childhood osteosarcoma (OS). In total, 477 doses of 2 mg/m intravenous (IV) mifamurtide, along with paracetamol as a premedication, were given to 15 patients with primary nonmetastatic OS after complete surgical resection and to 3 patients with progressive OS. The most common side effects encountered in the patients were chills and fever (17/18). These reactions were observed in 4 patients during the administration of each dose, in a single patient during the last administration, and in the remaining 12 patients during the first or initial 2 administrations...
June 8, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29877907/heterozygosity-for-cmt-type-4-predicts-a-severe-vincristine-induced-polyneuropathy-phenotype-a-case-report-and-review-of-literature
#8
Andrew Sy, Jerry Cheng, Robert Cooper, Lisa Mueller
Vincristine (VCR) is a common chemotherapeutic agent used in the treatment of multiple types of pediatric tumors. VCR's adverse effects are well documented and commonly involve peripheral neuropathy via axonal degeneration. Neuropathic severity is dose-dependent, with sensory deficits occurring with as little as 4 mg cumulative dose. Severe peripheral neuropathy is generally rare, but its effects become additive when given to patients with undiagnosed hereditary peripheral neuropathy such as Charcot-Marie-Tooth...
June 5, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29864110/hematologic-malignancies-associated-with-mediastinal-germ-cell-tumors-10-years-experience-at-thailand-s-national-pediatric-tertiary-referral-center
#9
Panjarat Sowithayasakul, Phakatip Sinlapamongkolkul, Jitsupa Treetipsatit, Nassawee Vathana, Nattee Narkbunnam, Kleebsabai Sanpakit, Jassada Buaboonnam
Mediastinal germ cell tumor (MGCT), which accounts for 1% to 3% of extragonadal germ cell tumors, has unique manifestations; it is associated with several types of hematologic malignancy, particularly myeloid neoplasm. The aim of this study was to report the 10-year incidence, clinical characteristics, and outcomes of MGCT at Thailand's national pediatric tertiary referral center. This retrospective study included patients diagnosed with MGCT at the Department of Pediatrics, Siriraj Hospital during 2005 to 2014...
June 1, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29863582/novel-therapy-for-pediatric-angiosarcoma-with-cytoreductive-surgery-and-hyperthermic-intraperitoneal-chemotherapy
#10
Lindsey Winer, Francisco I Macedo, Abdullah Alfawaz, Antonio Sommariva, Giovanni Cecchetto, Holly L Neville, Mecker G Möller
The management of pediatric abdominopelvic angiosarcoma remains unclear due to limited clinical experience. Herein, we presented the first 2 pediatric patients with abdominal angiosarcoma who were treated with cytoreductive surgery (CRS) and hyperthermic intraperitoneal chemotherapy (HIPEC) after neoadjuvant therapy. The first patient is alive with recurrent disease at 1-year follow-up and the second patient remains disease free after 1 year. CRS and HIPEC should be considered as a therapeutic option in the management of pediatric abdominal angiosarcomas...
May 31, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29863581/central-venous-catheter-associated-venous-thromboembolism-in-children-with-hematologic-malignancy
#11
Sara-Jane N Onyeama, Sheila J Hanson, Mahua Dasgupta, Kathryn Baker, Pippa M Simpson, Rowena C Punzalan
In pediatric oncology, the diagnosis of a hematologic malignancy and presence of a central venous catheter (CVC) have been identified as significant risk factors for the development of a venous thromboembolism (VTE). There remain little data regarding CVC factors associated with CVC-related VTE. Using the VTE and oncology database in a quaternary care center, a retrospective cohort study was conducted in children below 18 years old with hematologic cancer from November 5, 2012 to April 4, 2016. Patient, CVC factors, and VTE occurrence were analyzed to identify significant patient and CVC factors associated with the development of clinically identified CVC-related VTE...
May 31, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29846281/identification-of-a-novel-mutation-in-the-sec23b-gene-associated-with-congenital-dyserythropoietic-anemia-type-ii-through-the-use-of-next-generation-sequencing-panel-in-an-undiagnosed-case-of-nonimmune-hereditary-hemolytic-anemia
#12
Sultan Aydin Koker, Tuba H Karapinar, Yeşim Oymak, Paola Bianchi, Elisa Fermo, Salih Gozmen, Canan Vergin
Congenital dyserythropoietic anemias (CDAs) are rare hereditary blood disorders characterized by ineffective erythropoiesis, hemolysis, and erythroblast morphologic abnormalities in the bone marrow. The 3 main types of CDA, I to III, and variant types of CDA, IV-VIII, have been described. The causative genes have been identified as CDAN1, C15ORF41, SEC23B, KIF23, KLF1, and GATA1. CDA type II is the most frequent form. Typical symptoms are jaundice, hepatosplenomegaly, mild-to-severe normocytic anemia, and inadequate reticulocyte response...
May 25, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29846280/brincidofovir-cmx001-toxicity-associated-with-epithelial-apoptosis-and-crypt-drop-out-in-a-hematopoietic-cell-transplant-patient-challenges-in-distinguishing-drug-toxicity-from-gvhd
#13
Claire J Detweiler, Sarah B Mueller, Anthony D Sung, Jennifer L Saullo, Vinod K Prasad, Diana M Cardona
Brincidofovir (CMX001) is an oral agent with activity against double-strand DNA viruses undergoing clinical trials in immunocompromised patients. We report a patient clinically diagnosed with brincidofovir-related gastrointestinal (GI) toxicity and his histologic findings. A 2-year-old boy with medulloblastoma undergoing autologous hematopoietic cell transplantation developed adenovirus viremia 9 days posttransplant. After initial treatment with intravenous cidofovir he was started on oral brincidofovir as part of a clinical trial...
May 25, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29794646/plasma-hepcidin-levels-in-healthy-children-review-of-current-literature-highlights-limited-studies
#14
Surjeet Kumar, Prateek Bhatia, Richa Jain, Bhavneet Bharti
BACKGROUND: There are few studies that highlight pediatric hepcidin reference ranges especially from Asian subcontinent. In current study, plasma from 131 children (72 boys and 59 girls; 1 to 12 y) was analyzed for hepcidin-25 by enzyme-linked immunosorbent assay. OBSERVATIONS: The median (interquartile range) plasma hepcidin in boys was 21.89 ng/mL (16.50 to 51.70 ng/mL) and girls was 21.95 ng/mL (19.20 to 47.70 ng/mL). No statistically significant difference (P=0...
May 22, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29794645/evaluation-of-the-use-of-fosaprepitant-for-the-prevention-of-chemotherapy-induced-nausea-and-vomiting-in-pediatric-patients
#15
Sarah Timaeus, Joshua Elder, Kyle Franco
A neurokinin-1 (NK1) receptor antagonist is recommended with a 5-HT3 receptor antagonist and dexamethasone for prevention of chemotherapy-induced nausea and vomiting (CINV) in adult patients receiving highly emetogenic chemotherapy. Data for fosaprepitant use in pediatric patients is lacking. A retrospective chart review was conducted using an electronic medical record to characterize the use of fosaprepitant in patients aged 10 months to 18 years at a single institution from August 2015 to January 2017. Thirty-nine patients received fosaprepitant 4 mg/kg (maximum, 150 mg) for prevention of CINV, and 35 were included in the analysis...
May 22, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29794644/increased-survival-for-children-with-acute-myeloid-leukemia-results-from-improved-postrelapse-treatment
#16
Aditi Vedi, Richard Mitchell, Sunita Shanmuganathan, Cecilia Oswald, Glenn M Marshall, Toby Trahair, Siva Sivarajasingam, David S Ziegler
BACKGROUND: The treatment for pediatric acute myeloid leukemia (AML) has not changed significantly over the past 3 decades, yet outcomes have improved with cure rates increasing from 30% to over 60% of all newly diagnosed children over this period. This improvement in survival has been attributed to both treatment intensification and improved supportive care over the decades, although the precise impact of each remains unknown. PATIENTS AND METHODS: We retrospectively analyzed a unique cohort of 276 patients with de novo AML diagnosed in childhood, all treated with the same chemotherapy protocol over a 25-year period from 1986 to 2012...
May 22, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29794643/pediatric-gastrointestinal-posttransplant-lymphoproliferative-disorder-incidence-clinical-characteristics-and-impact-of-major-surgical-interventions-upon-overall-survival
#17
Claudia Dziegielewski, Rodrigo Contreras, Sheila Weitzman, Justin T Gerstle
Posttransplant lymphoproliferative disorder (PTLD) is a severe complication of solid organ transplantation. A common site for PTLD development is the gastrointestinal (GI) tract. The purpose of this study was to evaluate the incidence, clinical features, and overall survival of pediatric patients with GI-PTLD, and to assess whether major surgical interventions increased mortality. Records of pediatric transplant patients who developed GI-PTLD between January 2000 and June 2015 were retrospectively reviewed at our institution...
May 22, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29771864/cold-pressor-pain-response-in-children-with-cancer
#18
Brooke N Jenkins, Ryan J Roemer, Ariana Martinez, Tara K Torres, Michelle A Fortier
OBJECTIVE: The goal of this study was to examine pain responses in pediatric patients with cancer. METHOD: Children (ages 6 to 18) undergoing treatment for cancer (N=68) completed the cold pressor task. RESULTS: Average pain tolerance was 118.22 seconds (SD=101.18) and 40% of the children kept their hand in the water the entire 4-minute ceiling. On a 0 to 10 numeric rating scale, children reported a pain severity of 5.07 (SD=3.47) at their first report of pain, a pain severity of 5...
May 16, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29771863/upshaw-schulman-syndrome-with-c-2728c-t-mutation-in-adamts13-gene
#19
Shahzadi Resham, Zehra Fadoo, Bushra Moiz
Congenital thrombotic thrombocytopenic purpura is a rare autosomal recessive disorder presenting with hemolytic anemia, thrombocytopenia, micro vascular thrombosis, and end organ damage. Here, we present a case of a 7-year-old girl having recurrent neonatal hemolysis, developmental delay, frequent seizures, and thrombocytopenia. Characteristic clinical picture and gene sequencing of a disintegrin and metalloproteinase with thrombospondin motifs 13 confirmed the diagnosis of Upshaw-Schulman syndrome. She was treated successfully with plasma infusion...
May 16, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29771862/efficacy-and-safety-of-direct-acting-antiviral-therapy-for-chronic-hepatitis-c-in-thalassemic-children
#20
Shivadatta Padhi, Sudhir Maharshi, Gaurav K Gupta, Kapil Garg, Sandeep Nijhawan
OBJECTIVES: There is limited data on the efficacy and safety of directly acting antiviral therapy (DAA) for chronic hepatitis C in pediatric population. The aim was to assess the efficacy and safety of DAA in chronic hepatitis C β-thalassemic major pediatric patients. METHODS: Prospective study was conducted from September 2015 to January 2017. All β-thalassemic major chronic hepatitis C pediatric patients with age between 5 and 14 years were included in this study...
May 16, 2018: Journal of Pediatric Hematology/oncology
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