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View article: Real-world data for tisagenlecleucel in patients with R/R B-ALL: subgroup analyses from the CIBMTR registry
Real-world data for tisagenlecleucel in patients with R/R B-ALL: subgroup analyses from the CIBMTR registry Open
Since the first approval of tisagenlecleucel in 2017, pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL) may receive this CD19-directed chimeric antigen receptor T-cell therapy. We repor…
View article: Proteomic Characterization of Pediatric Acute Lymphoblastic Leukemias Sheds Light on Subtype-Specific Molecular Mechanisms
Proteomic Characterization of Pediatric Acute Lymphoblastic Leukemias Sheds Light on Subtype-Specific Molecular Mechanisms Open
Acute lymphoblastic leukemia (ALL) accounts for approximately one third of cancers diagnosed in children every year. Molecular subtyping has significantly improved patient stratification and treatment selection for pediatric leukemia patie…
View article: Successful classification of clinical pediatric leukemia genetic subtypes via structural variant detection using HiFi long-read sequencing
Successful classification of clinical pediatric leukemia genetic subtypes via structural variant detection using HiFi long-read sequencing Open
Gene fusions are common primary drivers of pediatric leukemias and are the result of underlying structural variant (SVs). Current clinical workflows to detect such alterations rely on a multimodal approach, which often increases analysis t…
View article: GWAS Identifies Pharmacogenetic Markers Associated with Premedication-Related Pegaspargase Hypersensitivity and Toxicities in Pediatric and Adolescent Young Adults (AYAs) with Acute Lymphoblastic Leukemia or Lymphoma
GWAS Identifies Pharmacogenetic Markers Associated with Premedication-Related Pegaspargase Hypersensitivity and Toxicities in Pediatric and Adolescent Young Adults (AYAs) with Acute Lymphoblastic Leukemia or Lymphoma Open
Background: Pediatric acute lymphoblastic leukemia (ALL) patients have significantly improved outcomes over the last few decades with overall survival (OS) rates exceeding 90%. Several factors, including incorporation of asparaginase thera…
View article: A Genomics Driven, Inducible Pluripotent Stem Cell Model of Infant Lymphoblastic Leukemia
A Genomics Driven, Inducible Pluripotent Stem Cell Model of Infant Lymphoblastic Leukemia Open
Acute lymphoblastic leukemia in infants (iALL) is a high-risk subtype of childhood leukemia with poor survival outcomes despite intensive therapies. Rearrangement of KMT2A (KMT2A-r) occurs in 70% of cases and is associated with chemotherap…
View article: A Retrospective Study of Inotuzumab Ozogamicin in Individuals with Down Syndrome and B-Cell Acute Lymphoblastic Leukemia
A Retrospective Study of Inotuzumab Ozogamicin in Individuals with Down Syndrome and B-Cell Acute Lymphoblastic Leukemia Open
Background: Individuals with Down syndrome (DS) have a 20-fold increased risk of B-cell acute lymphoblastic leukemia (B-ALL), and experience more frequent relapse and toxicities during ALL therapy, including infection-related mortality. In…
View article: Evaluation of Hi-C Sequencing for Detection of Gene Fusions in Hematologic and Solid Tumor Pediatric Cancer Samples
Evaluation of Hi-C Sequencing for Detection of Gene Fusions in Hematologic and Solid Tumor Pediatric Cancer Samples Open
Hi-C sequencing is a DNA-based next-generation sequencing method that preserves the 3D genome conformation and has shown promise in detecting genomic rearrangements in translational research studies. To evaluate Hi-C as a potential clinica…
View article: Menin inhibitors in pediatric acute leukemia: a comprehensive review and recommendations to accelerate progress in collaboration with adult leukemia and the international community
Menin inhibitors in pediatric acute leukemia: a comprehensive review and recommendations to accelerate progress in collaboration with adult leukemia and the international community Open
Aberrant expression of HOX and MEIS1 family genes, as seen in KMT2A-rearranged, NUP98-rearranged, or NPM1-mutated leukemias leads to arrested differentiation and leukemia development. HOX family genes are essential gatekeepers of physiolog…
View article: Azacitidine as epigenetic priming for chemotherapy is safe and well-tolerated in infants with newly diagnosed KMT2A-rearranged acute lymphoblastic leukemia: Children’s Oncology Group trial AALL15P1
Azacitidine as epigenetic priming for chemotherapy is safe and well-tolerated in infants with newly diagnosed KMT2A-rearranged acute lymphoblastic leukemia: Children’s Oncology Group trial AALL15P1 Open
Infants less than 1 year old diagnosed with KMT2A-rearranged (KMT2A-r) acute lymphoblastic leukemia (ALL) are at high risk of remission failure, relapse, and death due to leukemia, despite intensive therapies. Infant KMT2A-r ALL blasts are…
View article: Evaluation of Hi-C sequencing for the detection of gene fusions in hematologic and solid pediatric cancer samples
Evaluation of Hi-C sequencing for the detection of gene fusions in hematologic and solid pediatric cancer samples Open
HiC sequencing is a DNA-based next-generation sequencing method that preserves the 3D conformation of the genome and has shown promise in detecting genomic rearrangements in translational research studies. To evaluate HiC as a potential cl…
View article: Optimized cytogenetic risk-group stratification of <i>KMT2A</i>-rearranged pediatric acute myeloid leukemia
Optimized cytogenetic risk-group stratification of <i>KMT2A</i>-rearranged pediatric acute myeloid leukemia Open
A comprehensive international consensus on the cytogenetic risk-group stratification of KMT2A-rearranged (KMT2A-r) pediatric acute myeloid leukemia (AML) is lacking. This retrospective (2005-2016) International Berlin-Frankfurt-Münster Stu…
View article: Severus: accurate detection and characterization of somatic structural variation in tumor genomes using long reads
Severus: accurate detection and characterization of somatic structural variation in tumor genomes using long reads Open
Most current studies rely on short-read sequencing to detect somatic structural variation (SV) in cancer genomes. Long-read sequencing offers the advantage of better mappability and long-range phasing, which results in substantial improvem…
View article: Asparaginase-Associated Toxicities and Hypersensitivity Reactions in Pediatric and Adolescent Down Syndrome Patients with Acute Lymphoblastic Leukemia or Lymphoma
Asparaginase-Associated Toxicities and Hypersensitivity Reactions in Pediatric and Adolescent Down Syndrome Patients with Acute Lymphoblastic Leukemia or Lymphoma Open
Children with Down syndrome acute lymphoblastic leukemia (DS-ALL) have worse prognosis compared to those with non-DS-ALL, largely due to higher treatment related mortality. Pegaspargase (PEG) is integral in protocols for the treatment of p…
View article: Single-Cell RNA Sequencing Facilitates Study of Cancer Mechanisms across Pediatric Leukemias
Single-Cell RNA Sequencing Facilitates Study of Cancer Mechanisms across Pediatric Leukemias Open
Leukemia is the most common pediatric cancer, diagnosed in over 3,000 children annually in the US. While the prognosis for many pediatric leukemias has significantly improved with the addition of DNA sequencing to the diagnosis and treatme…
View article: Minimal residual disease predicts outcomes in <i>KMT2A</i>‐rearranged but not <i>KMT2A</i>‐germline infant acute lymphoblastic leukemia: Report from Children's Oncology Group study AALL0631
Minimal residual disease predicts outcomes in <i>KMT2A</i>‐rearranged but not <i>KMT2A</i>‐germline infant acute lymphoblastic leukemia: Report from Children's Oncology Group study AALL0631 Open
We measured minimal residual disease (MRD) by multiparameter flow cytometry at three time points (TP) in 117 infants with KMT2A (lysine [K]‐specific methyltransferase 2A)‐rearranged and 58 with KMT2A ‐germline acute lymphoblastic leukemia …
View article: Measurable Residual Disease and Fusion Partner Independently Predict Survival and Relapse Risk in Childhood <i>KMT2A</i>-Rearranged Acute Myeloid Leukemia: A Study by the International Berlin-Frankfurt-Münster Study Group
Measurable Residual Disease and Fusion Partner Independently Predict Survival and Relapse Risk in Childhood <i>KMT2A</i>-Rearranged Acute Myeloid Leukemia: A Study by the International Berlin-Frankfurt-Münster Study Group Open
PURPOSE A previous study by the International Berlin-Frankfurt-Münster Study Group (I-BFM-SG) on childhood KMT2A-rearranged ( KMT2A-r) AML demonstrated the prognostic value of the fusion partner. This I-BFM-SG study investigated the value …
View article: Real-world use of tisagenlecleucel in infant acute lymphoblastic leukemia
Real-world use of tisagenlecleucel in infant acute lymphoblastic leukemia Open
Infants with B-cell acute lymphoblastic leukemia (B-ALL) have poor outcomes because of chemotherapy resistance leading to high relapse rates. Tisagenlecleucel, a CD19-directed chimeric antigen receptor T-cell (CART) therapy, is US Food and…
View article: Outstanding outcomes in infants with <i>KMT2A</i>-germline acute lymphoblastic leukemia treated with chemotherapy alone: results of the Children’s Oncology Group AALL0631 trial
Outstanding outcomes in infants with <i>KMT2A</i>-germline acute lymphoblastic leukemia treated with chemotherapy alone: results of the Children’s Oncology Group AALL0631 trial Open
Not available.
View article: MicroRNA Content of Ewing Sarcoma Derived Extracellular Vesicles Leads to Biomarker Potential and Identification of a Previously Undocumented EWS-FLI1 Translocation
MicroRNA Content of Ewing Sarcoma Derived Extracellular Vesicles Leads to Biomarker Potential and Identification of a Previously Undocumented EWS-FLI1 Translocation Open
Objective: Ewing Sarcoma Family of Tumors (ESFT) are a highly aggressive pediatric bone and soft tissue malignancy with poor outcomes in the refractory and recurrent setting. Over 90% of Ewing Sarcoma (ES) tumors are driven by the pathogno…
View article: Single-cell multiomics reveals increased plasticity, resistant populations, and stem-cell–like blasts in <i>KMT2A</i>-rearranged leukemia
Single-cell multiomics reveals increased plasticity, resistant populations, and stem-cell–like blasts in <i>KMT2A</i>-rearranged leukemia Open
KMT2A-rearranged (KMT2A-r) infant acute lymphoblastic leukemia (ALL) is a devastating malignancy with a dismal outcome, and younger age at diagnosis is associated with increased risk of relapse. To discover age-specific differences and cri…
View article: Gemtuzumab Ozogamicin Improves Event-Free Survival and Reduces Relapse in Pediatric <i>KMT2A</i> -Rearranged AML: Results From the Phase III Children's Oncology Group Trial AAML0531
Gemtuzumab Ozogamicin Improves Event-Free Survival and Reduces Relapse in Pediatric <i>KMT2A</i> -Rearranged AML: Results From the Phase III Children's Oncology Group Trial AAML0531 Open
PURPOSE We investigated the impact of the CD33-targeted agent gemtuzumab ozogamicin (GO) on survival in pediatric patients with KMT2A-rearranged ( KMT2A-r) acute myeloid leukemia (AML) enrolled in the Children's Oncology Group trial AAML05…
View article: Single-cell multi-omics reveals elevated plasticity and stem-cell-like blasts relevant to the poor prognosis of<i>KMT2A</i>-rearranged leukemia
Single-cell multi-omics reveals elevated plasticity and stem-cell-like blasts relevant to the poor prognosis of<i>KMT2A</i>-rearranged leukemia Open
Summary Infant ALL is a devastating malignancy caused by rearrangements of the KMT2A gene ( KMT2A-r ) in approximately 70% of patients. The outcome is dismal and younger age at diagnosis is associated with increased risk of relapse. To dis…
View article: Decitabine and Vorinostat with Chemotherapy in Relapsed Pediatric Acute Lymphoblastic Leukemia: A TACL Pilot Study
Decitabine and Vorinostat with Chemotherapy in Relapsed Pediatric Acute Lymphoblastic Leukemia: A TACL Pilot Study Open
Purpose: Treatment failure from drug resistance is the primary reason for relapse in acute lymphoblastic leukemia (ALL). Improving outcomes by targeting mechanisms of drug resistance is a potential solution. Patients and Methods: We report…
View article: Abstract 3649: Allelic imbalance in<i>KMT2A</i>-rearranged infant acute lymphoblastic leukemia
Abstract 3649: Allelic imbalance in<i>KMT2A</i>-rearranged infant acute lymphoblastic leukemia Open
BackgroundInfant acute lymphoblastic leukemia (ALL) is a malignant disorder with poor clinical outcome. It is well-known that infant ALL cases with rearrangement of the KMT2A gene (KMT2A-r) have an even poorer prognosis than non-KMT2A-r ca…