Homozygous c.130-131 ins A (pW44X) mutation in the HAX1 gene as the most common cause of congenital neutropenia in Turkey: Report from the Turkish Severe Congenital Neutropenia Registry.

abstract：:The identification and referral of candidate patients for phase I trials relies heavily on pediatric oncologists who must balance their own perceptions of phase I trials with the desires of the patient and his/her family. A survey was sent to 419 physicians practicing pediatric oncology at 30 different institutions. R...

journal_title：Pediatric blood & cancer

authors： Gilliam MB,Madan-Swain A,Adams JM,Pressey JG

更新日期：2013-08-01 00:00:00

abstract：BACKGROUND:Sickle cell disease (SCD) is an inherited blood disorder characterized by a chronic hemolytic anemia that can contribute to fatigue and global cognitive impairment in patients. The study objective was to report on the feasibility, reliability, and validity of the PedsQL™ Multidimensional Fatigue Scale in SCD...

journal_title：Pediatric blood & cancer

authors： Panepinto JA,Torres S,Bendo CB,McCavit TL,Dinu B,Sherman-Bien S,Bemrich-Stolz C,Varni JW

更新日期：2014-01-01 00:00:00

abstract：:Pediatric renal cell carcinoma (RCC) is a rare cancer that can be associated with inherited diseases including tuberous sclerosis complex (TSC) caused by germline mutations in TSC1 or TSC2. Somatic mutations in TSC1 and TSC2 have also been reported in adult RCC, which predict response to mTOR inhibitors. Here, we pres...

journal_title：Pediatric blood & cancer

authors： Potter SL,Venkatramani R,Wenderfer S,Graham BH,Vasudevan SA,Sher A,Wu H,Wheeler DA,Yang Y,Eng CM,Gibbs RA,Roy A,Plon SE,Parsons DW

更新日期：2017-05-01 00:00:00

abstract：:The majority of children with cancer live in low- and middle-income countries (LMICs) with little or no access to cancer treatment. The purpose of the paper is to describe the current status of childhood cancer treatment in Africa, as documented in publications, dedicated websites and information collected through sur...

journal_title：Pediatric blood & cancer

authors： Kruger M,Hendricks M,Davidson A,Stefan CD,van Eyssen AL,Uys R,van Zyl A,Hesseling P

更新日期：2014-04-01 00:00:00

abstract：:Pediatric cancer survivors have increased risk of obesity, hypertension, dyslipidemia, and type 2 diabetes, leading to premature cardiovascular disease (CVD). Multiple tissues that are involved in glucose homeostasis and lipid metabolism are adversely affected by chemotherapy. This review highlights the relevant tissu...

journal_title：Pediatric blood & cancer

authors： Rosen GP,Nguyen HT,Shaibi GQ

更新日期：2013-12-01 00:00:00

abstract：:Children with cancer and their families experience shifts in spiritual wellness from diagnosis through treatment and survivorship or bereavement. An interdisciplinary team conducted a systematic review of quantitative and qualitative research on spiritual assessments, interventions, and outcomes in childhood cancer fo...

journal_title：Pediatric blood & cancer

authors： Robert R,Stavinoha P,Jones BL,Robinson J,Larson K,Hicklen R,Smith B,Perko K,Koch K,Findley S,Weaver MS

更新日期：2019-09-01 00:00:00

abstract：:Congenital hypotransferrinemia (OMIM 209300) is an extremely rare disorder of inherited iron metabolism. Since its description in 1961, only 16 cases have been reported. The defective gene and molecular defect causing this disorder and clinicolaboratory findings seen in the homozygous and heterozygous states have been...

journal_title：Pediatric blood & cancer

authors： Aslan D

更新日期：2018-02-01 00:00:00

abstract：BACKGROUND:The introduction of the United Kingdom Medical Research Council's 10th AML trial (MRC AML 10) protocol incorporating high-dose anthracycline therapy has improved outcome of children with acute myeloid leukemia (AML). In this study, we review the results of childhood AML therapy in a Singapore university hosp...

journal_title：Pediatric blood & cancer

authors： Tan RM,Quah TC,Aung L,Liang S,Kirk RC,Yeoh AE

更新日期：2007-03-01 00:00:00

abstract：:We report a 10-year-old male with Hb E/Beta thalassemia disease who developed chronic graft-versus-host disease (cGVHD) of antiphospholipid antibody syndrome after successful allogeneic stem cell transplantation (SCT). He exhibited a recurrent ischemic stroke on day 368 post-SCT while on cyclosporine A, azathioprine, ...

journal_title：Pediatric blood & cancer

authors： Sirachainan N,Pakakasama S,Hongeng S,Chuansumrit A,Tuntiyatorn L,Vilaiyuk S

更新日期：2011-07-15 00:00:00

abstract：:Hepatoblastoma is a malignant pediatric liver tumor. The currently used diagnostic serum marker for hepatoblastoma, α-fetoprotein (AFP), is not always reliable in infants with hepatoblastoma, due to the physiologically elevated levels of AFP in this age group. In this report, we show that Delta-like 1 homolog (DLK1), ...

journal_title：Pediatric blood & cancer

authors： Falix FA,Aronson DC,Lamers WH,Hiralall JK,Seppen J

更新日期：2012-10-01 00:00:00

abstract：BACKGROUND:A desperate need for novel therapies in pediatric ependymoma (EPN) exists, as chemotherapy remains ineffective and radiotherapy often fails. EPN have significant infiltration of immune cells, which correlates with outcome. Immune checkpoint inhibitors provide an avenue for new treatments. This study characte...

journal_title：Pediatric blood & cancer

authors： Witt DA,Donson AM,Amani V,Moreira DC,Sanford B,Hoffman LM,Handler MH,Levy JMM,Jones KL,Nellan A,Foreman NK,Griesinger AM

更新日期：2018-05-01 00:00:00

abstract：BACKGROUND:In malaria-endemic countries in West Africa, sickle cell disease (SCD) contributes to childhood mortality. Historically, Liberia had regions wherein hemoglobin S and beta-thalassemia trait were mutually exclusive. Data on hemoglobinopathies in the Monrovia, the capital, are outdated and do not reflect urban ...

journal_title：Pediatric blood & cancer

authors： Tubman VN,Marshall R,Jallah W,Guo D,Ma C,Ohene-Frempong K,London WB,Heeney MM

更新日期：2016-04-01 00:00:00

abstract：:Immunotherapy with the anti-GD2 monoclonal antibody ch14.18, or dinutuximab, represents an important therapeutic advance in the treatment of pediatric high-risk neuroblastoma and is now considered part of standard of care in this patient population. To date, transverse myelitis as a result of dinutuximab therapy has n...

journal_title：Pediatric blood & cancer

authors： Ding YY,Panzer J,Maris JM,Castañeda A,Gomez-Chiari M,Mora J

更新日期：2018-01-01 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：:VPS45-associated severe congenital neutropenia (SCN) is a rare disorder characterized by life-threating infections, neutropenia, neutrophil and platelet dysfunction, poor response to filgrastim, and myelofibrosis with extramedullary hematopoiesis. We present a patient with SCN due to a homozygous c.1403C>T (p.P468L) m...

journal_title：Pediatric blood & cancer

authors： Shah RK,Munson M,Wierenga KJ,Pokala HR,Newburger PE,Crawford D

更新日期：2017-09-01 00:00:00

abstract：BACKGROUND:We assessed the safety and efficacy of ifosfamide and vinorelbine (IV) as a less toxic and effective reinduction regimen for pediatric patients with relapsed or refractory Hodgkin Lymphoma. PROCEDURE:This multi-center Children's Oncology Group phase II pilot study enrolled patients <30 years of age with bio...

journal_title：Pediatric blood & cancer

authors： Trippett TM,Schwartz CL,Guillerman RP,Gamis AS,Gardner S,Hogan S,London WB,Chen L,de Alarcon P

更新日期：2015-01-01 00:00:00

abstract：BACKGROUND:Studies have shown that children with cancer globally lack access to palliative care. Little is known regarding physicians' perceptions of palliative care, treatment access, and self-reported competence in providing palliative care. PROCEDURE:Members of the Global Neuroblastoma Network (online tumor board) ...

journal_title：Pediatric blood & cancer

authors： Balkin EM,Thompson D,Colson KE,Lam CG,Matthay KK

更新日期：2016-05-01 00:00:00

abstract：BACKGROUND:The efficacy of combination chemotherapy with methotrexate (MTX) and asparaginase is not well known in relapsed and refractory acute leukemia after contemporary therapy. PROCEDURE:A retrospective study of pediatric patients with relapsed or refractory acute myeloid leukemia (AML) who received MTX and aspara...

journal_title：Pediatric blood & cancer

authors： Buaboonnam J,Cao X,Pauley JL,Pui CH,Ribeiro RC,Rubnitz JE,Inaba H

更新日期：2013-07-01 00:00:00

abstract：BACKGROUND:Hemophagocytic Lymphohistiocytosis (HLH) is characterized by uncontrolled inflammation that is generally fatal without immune modulating chemotherapy. At Texas Children's Hospital, we have observed significant central nervous system (CNS) toxicity in several patients treated for HLH according to the Histiocy...

journal_title：Pediatric blood & cancer

authors： Thompson PA,Allen CE,Horton T,Jones JY,Vinks AA,McClain KL

更新日期：2009-05-01 00:00:00

abstract：BACKGROUND:Ultrasound-guided (USG) cannulation of the brachiocephalic vein (BCV) is gaining worldwide consensus for central venous access in children. This study reports a 20-month experience with this approach in children. METHODS:All patients who underwent percutaneous USG central venous catheter (CVC) positioning i...

journal_title：Pediatric blood & cancer

authors： Avanzini S,Mameli L,Disma N,Zanaboni C,Dato A,Montobbio G,Montagnini L,Bevilacqua M,Pierri F,Conte M,Amoroso L,Pala G,Pestarino S,Castagnola E,Molinari AC,Micalizzi C,Morreale G,Mattioli G,Pini Prato A

更新日期：2017-02-01 00:00:00

abstract：PURPOSE:To report the final analysis of survival outcomes for children with newly diagnosed high-grade glioma (HGG) treated on the "Head Start" (HS) II and III protocols with chemotherapy and intent to avoid irradiation in children <6 years old. PATIENTS AND METHODS:Between 1997 and 2009, 32 eligible children were enr...

journal_title：Pediatric blood & cancer

authors： Espinoza JC,Haley K,Patel N,Dhall G,Gardner S,Allen J,Torkildson J,Cornelius A,Rassekh R,Bedros A,Etzl M,Garvin J,Pradhan K,Corbett R,Sullivan M,McGowage G,Stein D,Jasty R,Sands SA,Ji L,Sposto R,Finlay JL

更新日期：2016-10-01 00:00:00

abstract：BACKGROUND:Although chemotherapy has improved outcome of osteosarcoma, 30-40% of patients succumb to this disease. Survivors experience substantial morbidity and mortality from anthracycline-induced cardiotoxicity. We hypothesized that the cardioprotectant dexrazoxane would (i) support escalation of the cumulative doxo...

journal_title：Pediatric blood & cancer

authors： Schwartz CL,Wexler LH,Krailo MD,Teot LA,Devidas M,Steinherz LJ,Goorin AM,Gebhardt MC,Healey JH,Sato JK,Meyers PA,Grier HE,Bernstein ML,Lipshultz SE

更新日期：2016-01-01 00:00:00

abstract：:Phytosterolemia is a rare autosomal recessive sterol storage disease caused by mutations in ABCG5 and ABCG8 genes. A 9-year-old Turkish boy who was presented with exclusively hematologic abnormalities had elevated plant sterol levels. Sequencing of ABCG5 and ABCG8 genes revealed a novel homozygous IVS10-1 G>T mutation...

journal_title：Pediatric blood & cancer

authors： Kaya Z,Niu DM,Yorulmaz A,Tekin A,Gürsel T

更新日期：2014-08-01 00:00:00

abstract：:As increasing numbers of childhood cancer patients are surviving, the long-term complications of the disease and its treatment have become ever more increasingly important. Reduced bone mineral density and increased fracture risk have been reported during and after treatment of children with cancer. The causes of oste...

journal_title：Pediatric blood & cancer

authors： van der Sluis IM,van den Heuvel-Eibrink MM

更新日期：2008-02-01 00:00:00

abstract：BACKGROUND:The identification of tissue-specific differentially methylated regions (tDMRs) is key to our understanding of mammalian development. Research has indicated that tDMRs are aberrantly methylated in cancer and may affect the oncogenic process. PROCEDURE:We used the MassARRAY EpiTYPER system to determine the q...

journal_title：Pediatric blood & cancer

authors： Sugito K,Kawashima H,Uekusa S,Yoshizawa S,Hoshi R,Furuya T,Kaneda H,Hosoda T,Masuko T,Ohashi K,Ikeda T,Koshinaga T,Fujiwara K,Igarashi J,Ghosh S,Held WA,Nagase H

更新日期：2013-03-01 00:00:00

abstract：BACKGROUND:Stroke is a severe complication of sickle cell anemia (SCA). The role of glucose-6-phosphate dehydrogenase (G6PD) deficiency in the development of stroke in children with SCA is controversial. PROCEDURE:The aim of this study was to investigate the association of clinical ischemic stroke, high-risk transcran...

journal_title：Pediatric blood & cancer

authors： Belisário AR,Rodrigues Sales R,Evelin Toledo N,Velloso-Rodrigues C,Maria Silva C,Borato Viana M

更新日期：2016-06-01 00:00:00

abstract：:The youngest siblings may be both emotionally vulnerable and often neglected members of the family of a childhood cancer patient. The prompt identification of signs of distress in these subjects allows trained caregivers to intervene with personalized, age-appropriate, attention, and care. A narrative approach, based ...

journal_title：Pediatric blood & cancer

authors： Massimo LM,Wiley TJ

更新日期：2008-03-01 00:00:00

abstract：BACKGROUND:Incidence of venous thromboembolism (VTE) in children is reported to be increasing. We examined thrombophilia testing results in children with VTE that presented in inpatient and outpatient settings to explore patterns of thrombophilia testing. PROCEDURE: PATIENTS/METHODS:Children, ages 0-20 years with VTE...

journal_title：Pediatric blood & cancer

authors： Mahajerin A,Obasaju P,Eckert G,Vik TA,Mehta R,Heiny M

更新日期：2014-03-01 00:00:00

abstract：BACKGROUND:Acinic cell carcinoma (AciCC) is rare in children; therefore, reaching a consensus on its management is challenging and radiotherapy is limited by concerns about long-term toxicity. The purpose of this study is to analyze the effectiveness and safety of surgery plus postoperative 125 I interstitial brachythe...

journal_title：Pediatric blood & cancer

authors： Li J,Zhang J,Lyu XM,Huang MW,Zheng L,Zhang JG

更新日期：2020-07-01 00:00:00

abstract：BACKGROUND:The presence of central nervous system (CNS) disease in pediatric acute myeloid leukemia (AML) is often thought to confer a worse prognosis. This study examined the outcome of children with AML who had CNS disease at diagnosis. METHODS:Patients enrolled on Children's Cancer Group protocols 2861, 2891, 2941,...

journal_title：Pediatric blood & cancer

authors： Johnston DL,Alonzo TA,Gerbing RB,Lange BJ,Woods WG

更新日期：2010-09-01 00:00:00