Phase 1 study of pomalidomide in children with recurrent, refractory, and progressive central nervous system tumors: A Pediatric Brain Tumor Consortium trial.

abstract：BACKGROUND:Alveolar soft part sarcomas (ASPS) are generally chemo- and radio-resistant mesenchymal tumours, with no standardized treatment guidelines. We describe the clinical behaviour of paediatric ASPS and compare these features to previously reported adult series. PATIENTS AND METHODS:The clinical data of 51 child...

journal_title：Pediatric blood & cancer

authors： Orbach D,Brennan B,Casanova M,Bergeron C,Mosseri V,Francotte N,Van Noesel M,Rey A,Bisogno G,Pierron G,Ferrari A

更新日期：2013-11-01 00:00:00

abstract：:Male breast cancer (MBC) is unusual, especially in young adults. Most cases of MBC as a secondary malignancy relate to the previous treatment with ionizing radiation. MBC can be associated with mutations in hereditary cancer predisposition syndrome genes (i.e., BRCA2); however, no such association has been reported in...

journal_title：Pediatric blood & cancer

authors： Hagelstrom RT,Ford J,Reiser GM,Nelson M,Pickering DL,Althof PA,Sanger WG,Coccia PF

更新日期：2016-03-01 00:00:00

abstract：:A 6-year-old male with chronic granulomatous disease, who was transplanted with bone marrow and exhibited increasing mixed chimerism, subsequently received two donor lymphocyte infusions (DLI). Two weeks after the second DLI, the patient developed acute graft-versus-host disease (GVHD) and progressive pancytopenia tha...

journal_title：Pediatric blood & cancer

authors： Kato I,Umeda K,Awaya T,Yui Y,Niwa A,Fujino H,Matsubara H,Watanabe K,Heike T,Adachi N,Endo F,Mizukami T,Nunoi H,Nakahata T,Adachi S

更新日期：2010-02-01 00:00:00

abstract：:Neuroblastoma is the most common extracranial solid tumor of childhood, and the outcomes for children with high-risk and relapsed disease remain poor. However, new international strategies for risk stratification and for treatment based on novel tumor targets and including immunotherapy are being employed in attempts ...

journal_title：Pediatric blood & cancer

authors： Zage PE,Louis CU,Cohn SL

更新日期：2012-07-01 00:00:00

abstract：BACKGROUND:Griscelli syndrome type 2 (GS2), the X-linked lymphoproliferative (XLP) and the Chédiak-Higashi (CHS) syndromes are diseases that all may develop hemophagocytic syndromes. We wanted to investigate whether the treatment protocols for hemophagocytic lymphohistiocytosis (HLH) can also be used for these syndrome...

journal_title：Pediatric blood & cancer

authors： Trottestam H,Beutel K,Meeths M,Carlsen N,Heilmann C,Pasić S,Webb D,Hasle H,Henter JI

更新日期：2009-02-01 00:00:00

abstract：:We report two cases of primary CNS lymphoma (PCNSL) treated with high-dose methotrexate. Though standard adult treatment of PCNSL incorporates whole-brain radiotherapy, the literature suggests it may be possible to delay or avoid radiotherapy and the associated increased risk of neurologic sequelae in pediatric patien...

journal_title：Pediatric blood & cancer

authors： Shah AC,Kelly DR,Nabors LB,Oakes WJ,Hilliard LM,Reddy AT

更新日期：2010-12-01 00:00:00

abstract：BACKGROUND:Asparaginase (Asp) and corticosteroid (CS) treatment in patients with acute lymphoblastic leukaemia (ALL) is associated with an increased risk of thrombotic events. OBJECTIVE:Characterization of global haemostatic phenotypes of patients with ALL during Asp therapy. PROCEDURE:Thrombin generation (TG) was mo...

journal_title：Pediatric blood & cancer

authors： Rozen L,Noubouossie D,Dedeken L,Huybrechts S,Lê PQ,Ferster A,Demulder A

更新日期：2017-02-01 00:00:00

abstract：:Immune-mediated hemolytic anemia is a well-recognized complication of hematopoietic stem cell transplantation. We report on a 6-year-old boy with X-linked adrenoleukodystrophy who developed severe delayed alloimmune hemolytic anemia associated with immune-mediated neutropenia and thrombocytopenia following major ABO i...

journal_title：Pediatric blood & cancer

authors： Chao MM,Levine JE,Ferrara JL,Cooling L,Cooke KR,Hutchinson RJ,Yanik GA

更新日期：2008-04-01 00:00:00

abstract：:We describe here the outcomes of reduced-toxicity alternate-donor stem cell transplant (SCT) with posttransplant cyclophosphamide (PTCy) for primary immunodeficiency disorders (PIDs) in eight children (haploidentical-seven and matched unrelated donor-one). The conditioning was with serotherapy (alemtuzumab-3/rabbit-an...

journal_title：Pediatric blood & cancer

authors： Rastogi N,Katewa S,Thakkar D,Kohli S,Nivargi S,Yadav SP

更新日期：2018-01-01 00:00:00

abstract：:2-Chlorodeoxyadenosine (2-CdA) has been successfully used in children to treat refractory Langerhans cell histiocytosis and juvenile xanthogranuloma (JXG) as salvage therapy. Although 2-CdA is generally well-tolerated, with temporary myelosuppression as the primary dose-limiting toxicity, prolonged myelosuppressive, a...

journal_title：Pediatric blood & cancer

authors： Yamada K,Yasui M,Sawada A,Inoue M,Nakayama M,Kawa K

更新日期：2012-02-01 00:00:00

abstract：:Leydig cell tumors account for 3% of testicular tumors and have never been reported after treatment for Ewing's sarcoma. We report the unusual occurrence of a patient who developed a Leydig cell tumor of the testis 18 years after successful treatment for Ewing's sarcoma. Additional monitoring for second malignancies m...

journal_title：Pediatric blood & cancer

authors： Butros LJ,Phillip S,Chou A,Meyers PA,Huvos AG,Healey JH,Russo P,Gorlick RG

更新日期：2007-12-01 00:00:00

abstract：BACKGROUND:Children with acute lymphoblastic leukemia (ALL) who fail to achieve complete remission (CR) after induction therapy (induction failure: IF) have a poor prognosis; however, there have been few prospective studies in patients with IF. PATIENTS AND METHODS:Between April 1997 and March 2005, 27 of 1,237 leukem...

journal_title：Pediatric blood & cancer

authors： Suzuki N,Yumura-Yagi K,Yoshida M,Hara J,Nishimura S,Kudoh T,Tawa A,Usami I,Tanizawa A,Hori H,Ito Y,Miyaji R,Oda M,Kato K,Hamamoto K,Osugi Y,Hashii Y,Nakahata T,Horibe K,Japan Association of Childhood Leukemia Study

更新日期：2010-01-01 00:00:00

abstract：BACKGROUND:The views and clinical practice of children's cancer units were surveyed regarding management of central venous catheter (CVC) occlusion (CVC-occlusion), CVC-related thrombosis (CVC-thrombosis) and thromboembolism (CVC-thromboembolism). PROCEDURE:A questionnaire was sent to all 22 United Kingdom Children's ...

journal_title：Pediatric blood & cancer

authors： Skinner R,Koller K,McIntosh N,McCarthy A,Pizer B,United Kingdom Children's Cancer Study Group (UKCCSG).,Paediatric Oncology Nursing Forum (PONF) Supportive Care Group.

更新日期：2008-04-01 00:00:00

abstract：:Optic pathway gliomas represent approximately 5% of all pediatric intracranial tumors. While these tumors are most frequently low grade astrocytomas, they follow a highly variable clinical course, and accordingly, there is much debate regarding their optimal management. Their propensity to occur in very young children...

journal_title：Pediatric blood & cancer

authors： Jahraus CD,Tarbell NJ

更新日期：2006-05-01 00:00:00

abstract：:A contemporaneous presentation of a second breast cancer in a mother and an extremity rhabdomyosarcoma (RMS) in her daughter led to the diagnosis of the Li Fraumeni syndrome (LFS). Although the association between LFS and RMS in young patients is well recognised 1 there are no guidelines as to how this knowledge shoul...

journal_title：Pediatric blood & cancer

authors： Trahair T,Andrews L,Cohn RJ

更新日期：2007-03-01 00:00:00

abstract：BACKGROUND:A feature often seen in adolescent patients with cancer is a lengthy symptom interval, especially in comparison with children. It has been suggested that inadequate awareness of cancer risk among adolescents may play an important role in this study. METHODS:The Società Italiana Adolescenti con Malattie Onco...

journal_title：Pediatric blood & cancer

authors： Magni C,Segrè C,Finzi C,Veneroni L,Clerici CA,Massimino M,Casanova M,Martinella V,Chiaravalli S,Ricci A,Biondi A,Ferrari A

更新日期：2016-08-01 00:00:00

abstract：:We evaluated the effects of body mass index (BMI) on the risk of thromboembolism (TE) in children (<18 years) with hematological malignancies during the period 1990-2009 (n = 359). Obesity was prevalent in 12% of patients: 6% versus 17% prior to and after the year 2000 (P = 0.02). Sixty-one (17%) patients developed TE...

journal_title：Pediatric blood & cancer

authors： Tuckuviene R,Christensen AL,Chan AK,Athale U

更新日期：2012-08-01 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:Positron emission tomography (PET) and gallium scans facilitate diagnosis and staging, evaluation of response to therapy, and monitoring for relapse in Hodgkin lymphoma (HL), but have not been compared in pediatric HL. PROCEDURE:We performed concurrent PET and gallium scans on 44 pediatric HL patients at di...

journal_title：Pediatric blood & cancer

authors： Hines-Thomas M,Kaste SC,Hudson MM,Howard SC,Liu WA,Wu J,Kun LE,Shulkin BL,Krasin MJ,Metzger ML

更新日期：2008-08-01 00:00:00

abstract：BACKGROUND:ABT-751, an orally bioavailable sulfonamide, binds beta-tubulin to inhibit microtubule polymerization. We described response and event-free survival (EFS) in children with neuroblastoma and other solid tumors receiving ABT-751, assessed in vitro cytotoxicity of ABT-751 and evaluated the effect of ABT-751 on ...

journal_title：Pediatric blood & cancer

authors： Meany HJ,Sackett DL,Maris JM,Ward Y,Krivoshik A,Cohn SL,Steinberg SM,Balis FM,Fox E

更新日期：2010-01-01 00:00:00

abstract：:Hemophilia is an excellent example in medicine where clinical translation of basic science discoveries has transformed the gloomy outlook of the disease. This review provides an overview of clinical advances in hemophilia management with a specific focus on the molecular heterogeneity of the disease and progress in ma...

journal_title：Pediatric blood & cancer

authors： Sharathkumar AA,Carcao M

更新日期：2011-12-01 00:00:00

abstract：:The BRAF V600E missense mutation is known to be present in a subset of central nervous system tumors. We report a patient with a BRAF V600E mutated pilomyxoid astrocytoma who failed multiple conventional chemotherapy regimens. Treatment with vemurafenib, a molecularly targeted therapy against the mutant BRAF V600E kin...

journal_title：Pediatric blood & cancer

authors： Skrypek M,Foreman N,Guillaume D,Moertel C

更新日期：2014-11-01 00:00:00

abstract：:Severe congenital neutropenia (SCN) is a bone marrow failure disease with an autosomal dominant inheritance from mutations in ELANE. Here, we report a 7-week-old Korean male with SCN. His elder sister died from pneumonia at 2 years. Direct sequencing of ELANE in the proband identified a heterozygous novel frameshift m...

journal_title：Pediatric blood & cancer

authors： Kim HJ,Song MJ,Lee KO,Kim SH,Kim HJ

更新日期：2015-12-01 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:Thiotepa and carboplatin are known to be active in central nervous system tumors. Topotecan potentiates the anti-cancer effects of alkylators and crosses the blood-brain barrier. We present ten patients with recurrent or progressive central nervous system malignancies treated on a myeloablative regimen using...

journal_title：Pediatric blood & cancer

authors： Gilheeney SW,Khakoo Y,Souweidane M,Wolden S,Boulad F,Dunkel IJ

更新日期：2010-04-01 00:00:00

abstract：BACKGROUND:Aberrant gangliosides are produced and shed by some tumors into the extracellular milieu. Their concentration is related to disease progression in children with neuroblastoma and in experimental models. The mechanism for this tumor promoting effect is not known. PURPOSE:Here, we investigated the effect of g...

journal_title：Pediatric blood & cancer

authors： Jabbar AA,Kazarian T,Hakobyan N,Valentino LA

更新日期：2006-03-01 00:00:00

abstract：BACKGROUND:Transcranial Doppler ultrasound (TCD) has been demonstrated to be a powerful predictor of stroke risk due to sickle cell disease (SCD) in pediatric populations. Little is known about how this healthcare innovation has disseminated into preventive care for SCD. The objective of this study was to determine TCD...

journal_title：Pediatric blood & cancer

authors： Raphael JL,Shetty PB,Liu H,Mahoney DH,Mueller BU

更新日期：2008-11-01 00:00:00

abstract：BACKGROUND:Children with cancer have high utilization of the emergency department (ED), but little is known about which outcomes are most important to them and their caregivers when they seek care in the ED. PROCEDURE:A qualitative evaluation of ED experience for children with cancer and their caregivers was performed...

journal_title：Pediatric blood & cancer

authors： Mueller EL,Cochrane AR,Lynch DO,Cockrum BP,Wiehe SE

更新日期：2019-10-01 00:00:00

abstract：BACKGROUND:Since there is no validated assay to monitor disease in children with neuroblastoma (NB), we tested whether NB specific cell-free RNA could be detected in their plasma samples. Moreover, with the aim of reducing patients' discomfort, we compared this assay to a recently standardized procedure that uses a lar...

journal_title：Pediatric blood & cancer

authors： Corrias MV,Pistorio A,Cangemi G,Tripodi G,Carlini B,Scaruffi P,Fardin P,Garaventa A,Pistoia V,Haupt R

更新日期：2010-07-01 00:00:00

abstract：BACKGROUND:The treatment of central nervous system (CNS) germ cell tumors (GCT) remains controversial. The purpose of this study was to demonstrate efficacy of a chemotherapy only strategy, with less morbidity, when compared to regimens with irradiation. METHODS:Between January 2001 and December 2004 newly diagnosed p...

journal_title：Pediatric blood & cancer

authors： da Silva NS,Cappellano AM,Diez B,Cavalheiro S,Gardner S,Wisoff J,Kellie S,Parker R,Garvin J,Finlay J

更新日期：2010-03-01 00:00:00