Paternal Somatic Mosaicism of a Novel Frameshift Mutation in ELANE Causing Severe Congenital Neutropenia.

abstract：BACKGROUND:Soft tissue sarcomas (STS) are a heterogeneous group of mesenchymal malignancies that occur throughout the lifespan. The impact of age on disease features and outcome is unclear. METHODS:We analyzed the clinical features and outcome of all STS cases registered between 1973 and 2006 in the SEER database. RE...

journal_title：Pediatric blood & cancer

authors： Ferrari A,Sultan I,Huang TT,Rodriguez-Galindo C,Shehadeh A,Meazza C,Ness KK,Casanova M,Spunt SL

更新日期：2011-12-01 00:00:00

abstract：:Most publications report the adverse (negative) health issues in childhood cancer survivors. Presenting information to the newly diagnosed patient in a positive manner is advocated, while noting that recurrence is the most likely adverse event. Re-analysis of population-based studies on life-threatening toxicities fro...

journal_title：Pediatric blood & cancer

authors： Fryer C

更新日期：2011-12-15 00:00:00

abstract：:Germline or acquired mutations involving the GATA-binding protein gene (GATA2) have been linked to a variety of clinical conditions. In addition, patients harboring GATA2 mutations have a striking predisposition to develop myeloid malignancies, such as myelodysplastic syndrome or acute myeloid leukemia, but not acute ...

journal_title：Pediatric blood & cancer

authors： Esparza O,Xavier AC,Atkinson TP,Hill BC,Whelan K

更新日期：2019-06-01 00:00:00

abstract：:We evaluated the in vitro glucocorticoid (GC) responsiveness of 117 pediatric acute myeloid leukemia cells by considering GC resistance, GC-induced proliferation, and GC-induced differentiation. None of the samples was highly GC sensitive, and only 15% were intermediately sensitive. GC-induced differentiation was not ...

journal_title：Pediatric blood & cancer

authors： Klein K,Haarman EG,de Haas V,Zwaan ChM,Creutzig U,Kaspers GL

更新日期：2016-08-01 00:00:00

abstract：PURPOSE:To assess health and musculoskeletal function in survivors of pediatric sarcomas. PATIENTS AND METHODS:Thirty-two individuals treated for Ewing sarcoma family of tumors (ESFT), rhabdomyosarcoma (RMS), or non-rhabdomyosarcoma soft tissue sarcomas (NR-STS) with multi-modality therapy were enrolled on this cross-...

journal_title：Pediatric blood & cancer

authors： Mansky P,Arai A,Stratton P,Bernstein D,Long L,Reynolds J,Chen D,Steinberg SM,Lavende N,Hoffman K,Nathan PC,Parks R,Augustine E,Chaudhry U,Derdak J,Wiener L,Gerber L,Mackall C

更新日期：2007-02-01 00:00:00

abstract：BACKGROUND:Abandonment of treatment is one of the toughest challenges to deal with in pediatric oncology. It leads to unnecessary mortality and morbidity in patients from low- and middle-income countries. PROCEDURE:The objective of our retrospective study was to determine the prevalence and predictors for abandonment ...

journal_title：Pediatric blood & cancer

authors： Siddiqui DE,Ashraf MS,Iftikhar S,Belgaumi AF

更新日期：2018-02-01 00:00:00

abstract：BACKGROUND:Right hemicolectomy (RHC) in patients with neuroendocrine tumors (NET) of the appendix has been advocated for tumors more than 2 cm, or with mesoappendix/vessel invasion, or with raised mitotic activity. No study demonstrated prolonged survival with RHC over appendectomy alone. The aim of this study was to c...

journal_title：Pediatric blood & cancer

authors： de Lambert G,Lardy H,Martelli H,Orbach D,Gauthier F,Guérin F

更新日期：2016-04-01 00:00:00

abstract：BACKGROUND:Low expression of HLA class II antigens has been associated with more aggressive disease in several human malignancies including adult adrenocortical tumors (ACT), but their clinical relevance in pediatric ACT needs to be investigated. PROCEDURE:This study analyzed the expression profile of three class II h...

journal_title：Pediatric blood & cancer

authors： Leite FA,Lira RC,Fedatto PF,Antonini SR,Martinelli CE Jr,de Castro M,Neder L,Ramalho LN,Tucci S Jr,Mastelaro MJ,Seidinger AL,Cardinalli IA,Yunes JA,Brandalise SR,Tone LG,Scrideli CA

更新日期：2014-11-01 00:00:00

abstract：BACKGROUND:Pegasparaginase (PEG) is important for treatment of Acute Lymphoblastic Leukemia (ALL). Despite conjugation to polyethylene glycol to reduce immunogenicity, allergic reactions still occur and may be severe. Traditionally, PEG is given via intramuscular (IM) injection but recent protocols have shown it can be...

journal_title：Pediatric blood & cancer

authors： Pidaparti M,Bostrom B

更新日期：2012-09-01 00:00:00

abstract：:Unstable hemoglobin variants represent a rare etiology of congenital hemolytic anemia. Without a high index of suspicion, plus proper laboratory testing and interpretation, the correct diagnosis can be elusive. We report on five children who were initially thought to have other congenital disorders such as hereditary ...

journal_title：Pediatric blood & cancer

authors： Yates AM,Mortier NA,Hyde KS,Hankins JS,Ware RE

更新日期：2010-12-15 00:00:00

abstract：:Undifferentiated embryonal sarcoma of the liver (UESL) is a rare aggressive mesenchymal pediatric tumor. Previously, reported outcomes have been very poor. Here, we report a single-center experience of five patients with UESL treated with upfront gross total resection and adjuvant chemotherapy. We have a median follow...

journal_title：Pediatric blood & cancer

authors： Mathias MD,Ambati SR,Chou AJ,Slotkin EK,Wexler LH,Meyers PA,Magnan H

更新日期：2016-12-01 00:00:00

abstract：BACKGROUND:Testicular germ cell tumors (T-GCTs) occur from infancy to adulthood, and are the most common solid tumor in adolescent and young adult males. Traditionally, pediatric T-GCTs were perceived as more indolent than adult T-GCTs. However, there are few studies comparing these groups and none that specifically ev...

journal_title：Pediatric blood & cancer

authors： Cost NG,Lubahn JD,Adibi M,Romman A,Wickiser JE,Raj GV,Sagalowsky AI,Margulis V

更新日期：2014-03-01 00:00:00

abstract：BACKGROUND:Juvenile xanthogranuloma (JXG) is the most common non-Langerhans cell histiocytosis in children. The mortality and morbidity of JXG with extracutaneous lesions remain unclear. METHODS:Data of patients aged < 18 years who were diagnosed with JXG between 2001 and 2010 were retrospectively collected through a ...

journal_title：Pediatric blood & cancer

authors： Maeda M,Morimoto A,Shioda Y,Asano T,Koga Y,Nakazawa Y,Kanegane H,Kudo K,Ohga S,Ishii E,Histiocytosis Study Group of the Japanese Society of Pediatric Hematology\/Oncology.

更新日期：2020-07-01 00:00:00

abstract：BACKGROUND:Studies on mitochondrial DNA copy number reveal an increase or decrease in copy number that appears to be cancer specific, but data on acute lymphoblastic leukemia have been inconsistent regarding the significance of changes in mitochondrial DNA copies. The purpose of this pilot study was to analyze mitochon...

journal_title：Pediatric blood & cancer

authors： Jain A,Bakhshi S,Thakkar H,Gerards M,Singh A

更新日期：2018-03-01 00:00:00

abstract：BACKGROUND:Kaposi sarcoma (KS) is the most common HIV-associated malignancy in sub-Saharan Africa. The presentation and outcomes of pediatric KS are not well understood. PROCEDURE:We performed a retrospective cohort analysis of 81 HIV-infected children with KS at the Baylor Children's Clinical Centres of Excellence in...

journal_title：Pediatric blood & cancer

authors： Cox CM,El-Mallawany NK,Kabue M,Kovarik C,Schutze GE,Kazembe PN,Mehta PS

更新日期：2013-08-01 00:00:00

abstract：BACKGROUND:Primary hemophagocytic lymphohistiocytosis (HLH) represents a group of inherited hyperinflammatory immunodeficiencies, including familial HLH (FHL), Griscelli syndrome type 2 (GS2), and X-linked lymphoproliferative syndrome (XLP). We previously reported an annual incidence of suspected primary HLH in Sweden ...

journal_title：Pediatric blood & cancer

authors： Meeths M,Horne A,Sabel M,Bryceson YT,Henter JI

更新日期：2015-02-01 00:00:00

abstract：BACKGROUND:Drug shortages require clinical teams to decide how to allocate drugs in limited supply among their patients. Ethical frameworks are invaluable for promoting rational approaches to drug distribution, but gaps remain between ethical theory and clinical application. The goal of this work was to explore how dec...

journal_title：Pediatric blood & cancer

authors： Russell HV,Bernhardt MB,Berg S

更新日期：2017-07-01 00:00:00

abstract：:Opsoclonus, myoclonus, ataxia syndrome (OMA) is a severe neurologic disorder often associated with neuroblastoma. It is challenging to treat and can have long-term neurologic sequelae. Current recommended therapies include intravenous immunoglobulin, corticosteroids, rituximab, and chemotherapy (cyclophosphamide). We ...

journal_title：Pediatric blood & cancer

authors： Johnston DL,Murray S,Irwin MS,Doyle J,Schechter T

更新日期：2018-08-01 00:00:00

abstract：BACKGROUND:Various projects dedicated specifically to adolescents and young adults (AYA) with cancer have been developed in recent years. A critical aspect of such programs is the ability to demonstrate its value, and therefore how to measure desired outcomes. METHODS:A list of metrics to consider for demonstrating th...

journal_title：Pediatric blood & cancer

authors： Ferrari A,Silva M,Veneroni L,Magni C,Clerici CA,Meazza C,Terenziani M,Spreafico F,Chiaravalli S,Casanova M,Luksch R,Catania S,Schiavello E,Biassoni V,Podda M,Bergamaschi L,Puma N,Indini A,Proserpio T,Massimino M

更新日期：2016-12-01 00:00:00

abstract：BACKGROUND:Children with cancer may be at an increased risk of infection with hepatitis B virus (HBV) when levels of hepatitis B antibodies are reduced owing to chemotherapy-induced immunosuppression. This study evaluated the changes in HBV antibody status and HBV infections after chemotherapy in children with acute ly...

journal_title：Pediatric blood & cancer

authors： Choi YB,Lee NH,Yi ES,Kim YJ,Koo HH

更新日期：2019-12-01 00:00:00

abstract：BACKGROUND:Over the past decade, PET-CT has been used to assess rhabdomyosarcoma (RMS) in children. However, the role of PET-CT in staging RMS is unknown. PROCEDURE:Thirty subjects with RMS, median age 7.3 years, underwent PET-CT before therapy. PET-CTs and conventional imaging (CI) were independently reviewed by two ...

journal_title：Pediatric blood & cancer

authors： Federico SM,Spunt SL,Krasin MJ,Billup CA,Wu J,Shulkin B,Mandell G,McCarville MB

更新日期：2013-07-01 00:00:00

abstract：BACKGROUND:M6620 is a novel inhibitor of the DNA damage repair enzyme ATR, and has potentiated the activity of cisplatin and irinotecan in non-small cell lung cancer and colon cancer xenografts, respectively. PROCEDURES:M6620 was tested in vitro at concentrations ranging from 1.0 nM to 10.0 μM and at 75 nM in combinat...

journal_title：Pediatric blood & cancer

authors： Kurmasheva RT,Kurmashev D,Reynolds CP,Kang M,Wu J,Houghton PJ,Smith MA

更新日期：2018-02-01 00:00:00

abstract：BACKGROUND:Total body irradiation (TBI) is an important component of hematopoietic stem cell transplant (SCT) for pediatric malignancies. With increasing survival rates, late effects of SCT become more important. Younger children may be at particular risk of late effects of radiation and SCT. METHODS:We retrospectivel...

journal_title：Pediatric blood & cancer

authors： Mulcahy Levy JM,Tello T,Giller R,Wilkening G,Quinones R,Keating AK,Liu AK

更新日期：2013-04-01 00:00:00

abstract：:Imatinib can be safely discontinued in adults with chronic myeloid leukemia (CML) where there is a prolonged complete molecular response (CMR). No data are available in the pediatric population. Six children with CML discontinued imatinib by themselves. Only three of them were in CMR but for <2 years. A significant in...

journal_title：Pediatric blood & cancer

authors： Millot F,Claviez A,Leverger G,Corbaciglu S,Groll AH,Suttorp M

更新日期：2014-02-01 00:00:00

abstract：BACKGROUND:MYCN amplification marks poor prognosis in neuroblastoma (NB) tumors. In evaluating the mechanisms by which retinoic acid (RA) or nerve growth factor (NGF) decrease cell number in MYCN amplified NB cells, we have identified a number of proteins whose expression either decreases (E2F, CDC2, CDK6, cyclin depen...

journal_title：Pediatric blood & cancer

authors： Woo CW,Tan F,Cassano H,Lee J,Lee KC,Thiele CJ

更新日期：2008-02-01 00:00:00

abstract：:The incompatibility causing fetal and neonatal alloimmune thrombocytopenia (FNAIT) results from a fetus inheriting a paternal human platelet antigen (HPA), which is different from the maternal HPA. We present a unique case of FNAIT in a pregnancy involving an oocyte recipient mother with Turner syndrome. This is the f...

journal_title：Pediatric blood & cancer

authors： Barg AA,Ifrah AD,Strauss T,Simchen MJ,Orvieto R,Rosenberg N,Kenet G

更新日期：2017-08-01 00:00:00

abstract：:Clinically apparent jaundice is unusual in patients with beta-thalassemia major. Co-inheritance of Gilbert syndrome has been reported to cause hyperbilirubinemia in these subjects. Crigler-Najjar syndrome is another rare disorder of bilirubin metabolism caused by mutation in the gene coding the enzyme UGT1A1. We repor...

journal_title：Pediatric blood & cancer

authors： Aggarwal V,Seth A,Sharma S,Aneja S,Sammarco P,Fabiano C

更新日期：2010-04-01 00:00:00

abstract：:We report the case of a drug interaction between methotrexate (MTX) and chloral hydrate (CH) observed in a child treated for acute leukemia. Significantly slower MTX clearance and increased MTX exposure occurred on the first three courses of a high-dose chemotherapy when co-administered with CH despite normal renal fu...

journal_title：Pediatric blood & cancer

authors： Dao K,Ivanyuk A,Buclin T,Beck-Popovic M,Diezi M

更新日期：2013-03-01 00:00:00

abstract：BACKGROUND:Primary epithelial lung malignancies are rare in childhood and adolescence. We reviewed the Memorial Sloan-Kettering Cancer Center experience with these tumors to better understand their histology, time to diagnosis, treatment, and outcome. PROCEDURE:A retrospective review was performed on all patients 21 y...

journal_title：Pediatric blood & cancer

authors： Lal DR,Clark I,Shalkow J,Downey RJ,Shorter NA,Klimstra DS,La Quaglia MP

更新日期：2005-10-15 00:00:00

abstract：:Here, we present the case of a pediatric patient with newly diagnosed hepatocellular carcinoma causing central biliary obstruction and persistently elevated bilirubin of 3.0-4.3 mg/dl despite placement of bilateral internal-external biliary drains. The tumor was not resectable, and the patient was not a candidate for ...

journal_title：Pediatric blood & cancer

authors： Hiniker SM,Rangaswami A,Lungren MP,Thakor AS,Concepcion W,Balazy KE,Kovalchuk N,Donaldson SS

更新日期：2017-06-01 00:00:00