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Article
Rituximab administration in pediatric patients with newly diagnosed acute lymphoblastic leukemia
Polyethylene glycol (PEG)-asparaginase (pegaspargase) is a key agent in chemotherapy for acute lymphoblastic leukemia (ALL), but recipients frequently experience allergic reactions. We hypothesized that by dec...
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Article
Decitabine combined with minimally myelosuppressive therapy for induction of remission in pediatric high-risk acute myeloid leukemia with chromosome 5q deletion: a report of three cases
Cases of pediatric acute myeloid leukemia (AML) with complex karyotypes including chromosome 5 abnormalities are rare and have a very poor prognosis. Management of AML with monosomy 5/del(5q) has been inconsis...
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Article
Open AccessNTAL is associated with treatment outcome, cell proliferation and differentiation in acute promyelocytic leukemia
Non-T cell activation linker (NTAL) is a lipid raft-membrane protein expressed by normal and leukemic cells and involved in cell signaling. In acute promyelocytic leukemia (APL), NTAL depletion from lipid raft...
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Article
Survival and risk factors for mortality in pediatric patients with acute myeloid leukemia in a single reference center in low–middle-income country
Despite advances in therapy and care for children with acute myeloid leukemia (AML), survival rates for children in low- and middle-income countries (LMICs) remain poor. We studied risk factors for mortality a...
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Article
Open AccessA high-throughput screen indicates gemcitabine and JAK inhibitors may be useful for treating pediatric AML
Improvement in survival has been achieved for children and adolescents with AML but is largely attributed to enhanced supportive care as opposed to the development of better treatment regimens. High risk subty...
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Article
Open AccessA phase II clinical trial of adoptive transfer of haploidentical natural killer cells for consolidation therapy of pediatric acute myeloid leukemia
Consolidation therapies for children with intermediate- or high-risk acute myeloid leukemia (AML) are urgently needed to achieve higher cure rates while limiting therapy-related toxicities. We determined if ad...
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Article
Open AccessThe genomic landscape of pediatric myelodysplastic syndromes
Myelodysplastic syndromes (MDS) are uncommon in children and have a poor prognosis. In contrast to adult MDS, little is known about the genomic landscape of pediatric MDS. Here, we describe the somatic and ger...
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Article
Predictors of thrombohemorrhagic early death in children and adolescents with t(15;17)-positive acute promyelocytic leukemia treated with ATRA and chemotherapy
Clinical trials on childhood acute promyelocytic leukemia (APL) report early death (ED) rates of 3–8%, but predictors of thrombohemorrhagic (TH)-ED are not well understood. In a retrospective study, we aimed t...
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Reference Work Entry In depth
Childhood Adrenocortical Carcinoma
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Article
Open AccessMolecular cytogenetic studies characterizing a novel complex karyotype with an uncommon 5q22 deletion in childhood acute myeloid leukemia
Deletions in the long arm of chromosome 5 or loss of the whole chromosome are rare in childhood Acute Myeloid Leukemia (AML) patients. It is also unknown if the wide variety of breakpoints have diverging impli...
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Article
All-trans retinoic acid with daunorubicin or idarubicin for risk-adapted treatment of acute promyelocytic leukaemia: a matched-pair analysis of the PETHEMA LPA-2005 and IC-APL studies
Front-line treatment of acute promyelocytic leukaemia (APL) consists of all-trans retinoic acid (ATRA) and anthracycline-based chemotherapy. In this setting, a comparison of idarubicin and daunorubicin has never ...
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Article
Genomic landscape of paediatric adrenocortical tumours
Paediatric adrenocortical carcinoma is a rare malignancy with poor prognosis. Here we analyse 37 adrenocortical tumours (ACTs) by whole-genome, whole-exome and/or transcriptome sequencing. Most cases (91%) sho...
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Article
Internal tandem duplication of the FLT3 gene confers poor overall survival in patients with acute promyelocytic leukemia treated with all-trans retinoic acid and anthracycline-based chemotherapy: an International Consortium on Acute Promyelocytic Leukemia study
Activating internal tandem duplication (ITD) mutations in the fms-like tyrosine kinase 3 (FLT3) gene (FLT3-ITD) are associated with poor outcome in acute myeloid leukemia, but their prognostic impact in acute pro...
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Living Reference Work Entry In depth
Childhood Adrenocortical Carcinoma
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Article
The genomic landscape of hypodiploid acute lymphoblastic leukemia
Linda Holmfeldt, Lei Wei et al. report whole-genome and exome sequencing of 40 childhood hypodiploid acute lymphoblastic leukemias.
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Article
Spinal epidural lipomatosis in children with hematologic malignancies
Abnormal fat deposition in the epidural space or spinal epidural lipomatosis (SEL) due to corticosteroid treatment or obesity may cause obstruction to cerebrospinal fluid flow. Little is known about SEL in pat...
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Article
Open AccessErratum to: Association of the germline TP53 R337H mutation with breast cancer in southern Brazil
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Article
Open AccessInherited germline TP53 mutation encodes a protein with an aberrant C-terminal motif in a case of pediatric adrenocortical tumor
Childhood adrenocortical tumor (ACT), a very rare malignancy, has an annual worldwide incidence of about 0.3 per million children younger than 15 years. The association between inherited germline mutations of ...
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Chapter
TP53 Molecular Genetics
The TP53 tumor suppressor normally functions as a transcription factor within a stress response signaling pathway to activate genes that block cell proliferation, repair DNA, inhibit angiogenesis, and induce a...
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Chapter
Adrenocortical Cancer in Children
Adrenocortical cancer (ACC) is a rare but aggressive childhood endocrine neoplasm. Its incidence in children is extremely low (only 0.2% of pediatric cancers) [1], and most pediatric oncologists see few cases ...