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View article: Isatuximab in combination with chemotherapy for pediatric patients with relapsed/refractory acute lymphoblastic leukemia or acute myeloid leukemia: The ISAKIDS study
Isatuximab in combination with chemotherapy for pediatric patients with relapsed/refractory acute lymphoblastic leukemia or acute myeloid leukemia: The ISAKIDS study Open
Children with relapsed acute leukemia have a poor prognosis; current relapse treatments are toxic, and novel treatments are needed. The anti‐CD38 antibody isatuximab is approved for relapsed‐refractory multiple myeloma in adults. We presen…
View article: Opportunities and Challenges With CAR T-cell Treatment of Children and Young Adults With B-Cell Acute Lymphoblastic Leukemia: Review and Recommendations From the Westhafen Intercontinental Group
Opportunities and Challenges With CAR T-cell Treatment of Children and Young Adults With B-Cell Acute Lymphoblastic Leukemia: Review and Recommendations From the Westhafen Intercontinental Group Open
Chimeric antigen-receptor T-cells (CAR T-cells) targeted at pediatric B-cell precursor acute lymphoblastic leukemia (B-ALL) have changed the paradigm for treatment of relapsed and refractory B-ALL. We present a comprehensive review and rec…
View article: Harmonized immune recovery monitoring after HCT: evidence and practical guidance from the Westhafen Intercontinental Group
Harmonized immune recovery monitoring after HCT: evidence and practical guidance from the Westhafen Intercontinental Group Open
Allogeneic hematopoietic cell transplantation (allo-HCT) is a curative option for patients with high-risk malignancies and nonmalignant disorders. Long-term survival depends on robust immune reconstitution (IR), which governs overall immun…
View article: Impact of minimal residual disease on the outcome of hematopoietic stem cell transplantation for childhood acute lymphoblastic leukemia within the FORUM Trial
Impact of minimal residual disease on the outcome of hematopoietic stem cell transplantation for childhood acute lymphoblastic leukemia within the FORUM Trial Open
In the randomized cohort of the international phase-III FORUM trial, which showed the superiority of total-body irradiation (TBI) over chemotherapy-based conditioning prior to hematopoietic stem cell transplantation (HSCT) for pediatric ac…
View article: Vitamin D status in children undergoing allogeneic hematopoietic stem cell transplantation: A prospective exploratory study
Vitamin D status in children undergoing allogeneic hematopoietic stem cell transplantation: A prospective exploratory study Open
Objectives Allogeneic hematopoietic stem cell transplantation (HSCT) is associated with nutritional risks. This study describes vitamin D status and intake in children undergoing HSCT and compares results to healthy children. Methods In a …
View article: Update on long-term outcomes of a cohort of patients with TCF3::HLF positive acute lymphoblastic leukemia treated with blinatumomab and stem cell transplantation
Update on long-term outcomes of a cohort of patients with TCF3::HLF positive acute lymphoblastic leukemia treated with blinatumomab and stem cell transplantation Open
Not available.
View article: Comparable outcomes after busulfan- or treosulfan-based conditioning for allo-HSCT in children with ALL: results of FORUM
Comparable outcomes after busulfan- or treosulfan-based conditioning for allo-HSCT in children with ALL: results of FORUM Open
The superiority of total body irradiation (TBI)–based vs chemotherapy conditioning for allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children with acute lymphoblastic leukemia (ALL) has been established in the internati…
View article: Comparison of outcomes of immunosuppressive therapy with rabbit versus horse antithymocyte globulin and cyclosporine a in children with acquired severe aplastic anemia
Comparison of outcomes of immunosuppressive therapy with rabbit versus horse antithymocyte globulin and cyclosporine a in children with acquired severe aplastic anemia Open
sponsorship: The authors acknowledge all the patients, their families, treating physicians from participating centers and reference pathologists and cytogeneticists of the EWOG-SAA. The authors thank Ms. Alexandra Fisher and Ms. Annemarie …
View article: Pharmacokinetics, pharmacodynamics, efficacy, and safety of ravulizumab in pediatric paroxysmal nocturnal hemoglobinuria
Pharmacokinetics, pharmacodynamics, efficacy, and safety of ravulizumab in pediatric paroxysmal nocturnal hemoglobinuria Open
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disease of uncontrolled terminal complement activation leading to intravascular hemolysis, thrombotic events and increased morbidity and mortality. This phase 3, open-label, s…
View article: Epigenetic aging of human blood cells is influenced by the age of the host body
Epigenetic aging of human blood cells is influenced by the age of the host body Open
Allogenic hematopoietic stem cell transplantation is a therapeutic procedure performed over a wide range of donor and recipient age combinations, representing natural experiments of how the age of the recipient affects aging in transplante…
View article: GENOTYPE/PHENOTYPE ASSOCIATIONS IN 174 INDIVIDUALS WITH GERMLINE GATA2 MUTATIONS
GENOTYPE/PHENOTYPE ASSOCIATIONS IN 174 INDIVIDUALS WITH GERMLINE GATA2 MUTATIONS Open
Background and aims: Germline GATA2 variants are associated with a pleiotropic Mendelian disorder predisposing to myelodysplastic syndrome (MDS) with variable penetrance and atypical presentations. Uncommon phenotypes continue to emerge, r…
View article: Biallelic inactivation of the <scp><i>NF1</i></scp> tumour suppressor gene in juvenile myelomonocytic leukaemia: Genetic evidence of driver function and implications for diagnostic workup
Biallelic inactivation of the <span><i>NF1</i></span> tumour suppressor gene in juvenile myelomonocytic leukaemia: Genetic evidence of driver function and implications for diagnostic workup Open
Summary Juvenile myelomonocytic leukaemia (JMML) is characterized by gene variants that deregulate the RAS signalling pathway. Children with neurofibromatosis type 1 (NF‐1) carry a defective NF1 allele in the germline and are predisposed t…
View article: Comparable Outcome after Busulfan- or Treosulfan-Based Conditioning Regimen in Children Above 4 Years of Age with ALL Undergoing Allogeneic HSCT. Results from the Prospective International Forum-Trial
Comparable Outcome after Busulfan- or Treosulfan-Based Conditioning Regimen in Children Above 4 Years of Age with ALL Undergoing Allogeneic HSCT. Results from the Prospective International Forum-Trial Open
Purpose: Total body irradiation (TBI) has proved to be the “gold standard” as part of the conditioning regimen before allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with acute lymphoblastic leukemia (ALL). …
View article: Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study
Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study Open
Allogeneic hematopoietic stem cell transplantation (HSCT) is highly effective for treating pediatric high-risk or relapsed acute lymphoblastic leukemia (ALL). For young children, total body irradiation (TBI) is associated with severe late …
View article: P501: ISATUXIMAB PLUS CHEMOTHERAPY FOR PEDIATRIC RELAPSED/REFRACTORY ACUTE LYMPHOBLASTIC LEUKEMIA OR ACUTE MYELOID LEUKEMIA (ISAKIDS): INTERIM EFFICACY ANALYSIS
P501: ISATUXIMAB PLUS CHEMOTHERAPY FOR PEDIATRIC RELAPSED/REFRACTORY ACUTE LYMPHOBLASTIC LEUKEMIA OR ACUTE MYELOID LEUKEMIA (ISAKIDS): INTERIM EFFICACY ANALYSIS Open
Topic: 3. Acute myeloid leukemia - Biology & Translational Research Background: Elevated CD38 expression is present in acute pediatric leukemias, making CD38 a potential target for treating children with acute lymphoblastic leukemia (ALL) …
View article: PB2501: INSIGHT-1: A PHASE I FIRST-IN-HUMAN TCR TRIAL OF GENETICALLY MODIFIED T-CELLS TARGETING TDT IN CHILDREN AND ADULTS WITH T- AND B-CELL ACUTE LYMPHOBLASTIC LEUKEMIA/LYMPHOMA (TRIAL-IN-PROGRESS)
PB2501: INSIGHT-1: A PHASE I FIRST-IN-HUMAN TCR TRIAL OF GENETICALLY MODIFIED T-CELLS TARGETING TDT IN CHILDREN AND ADULTS WITH T- AND B-CELL ACUTE LYMPHOBLASTIC LEUKEMIA/LYMPHOMA (TRIAL-IN-PROGRESS) Open
Topic: 25. Gene therapy, cellular immunotherapy and vaccination - Clinical Background: Immunotherapy with chimeric antigen receptor (CAR) T-cells targeting CD19 (CD19 CAR-T) has revolutionized the curative treatment options for chemorefrac…
View article: Supplementary Data from Next-Generation Sequencing of Minimal Residual Disease for Predicting Relapse after Tisagenlecleucel in Children and Young Adults with Acute Lymphoblastic Leukemia
Supplementary Data from Next-Generation Sequencing of Minimal Residual Disease for Predicting Relapse after Tisagenlecleucel in Children and Young Adults with Acute Lymphoblastic Leukemia Open
Supplementary Data from Next-Generation Sequencing of Minimal Residual Disease for Predicting Relapse after Tisagenlecleucel in Children and Young Adults with Acute Lymphoblastic Leukemia
View article: Data from Next-Generation Sequencing of Minimal Residual Disease for Predicting Relapse after Tisagenlecleucel in Children and Young Adults with Acute Lymphoblastic Leukemia
Data from Next-Generation Sequencing of Minimal Residual Disease for Predicting Relapse after Tisagenlecleucel in Children and Young Adults with Acute Lymphoblastic Leukemia Open
We assessed minimal residual disease (MRD) detection and B-cell aplasia after tisagenlecleucel therapy for acute lymphoblastic leukemia (ALL) to define biomarkers predictive of relapse (N = 143). Next-generation sequencing (NGS) MRD detect…
View article: Data from Next-Generation Sequencing of Minimal Residual Disease for Predicting Relapse after Tisagenlecleucel in Children and Young Adults with Acute Lymphoblastic Leukemia
Data from Next-Generation Sequencing of Minimal Residual Disease for Predicting Relapse after Tisagenlecleucel in Children and Young Adults with Acute Lymphoblastic Leukemia Open
We assessed minimal residual disease (MRD) detection and B-cell aplasia after tisagenlecleucel therapy for acute lymphoblastic leukemia (ALL) to define biomarkers predictive of relapse (N = 143). Next-generation sequencing (NGS) MRD detect…
View article: Supplementary Data from Next-Generation Sequencing of Minimal Residual Disease for Predicting Relapse after Tisagenlecleucel in Children and Young Adults with Acute Lymphoblastic Leukemia
Supplementary Data from Next-Generation Sequencing of Minimal Residual Disease for Predicting Relapse after Tisagenlecleucel in Children and Young Adults with Acute Lymphoblastic Leukemia Open
Supplementary Data from Next-Generation Sequencing of Minimal Residual Disease for Predicting Relapse after Tisagenlecleucel in Children and Young Adults with Acute Lymphoblastic Leukemia
View article: Data from N-myc and Noncoding RNAs in Neuroblastoma
Data from N-myc and Noncoding RNAs in Neuroblastoma Open
Neuroblastoma is a pediatric tumor of the sympathetic nervous system. Amplification and overexpression of the MYCN proto-oncogene occurs in approximately 20% of neuroblastomas and is associated with advanced stage disease, rapid tumor prog…
View article: Data from N-myc and Noncoding RNAs in Neuroblastoma
Data from N-myc and Noncoding RNAs in Neuroblastoma Open
Neuroblastoma is a pediatric tumor of the sympathetic nervous system. Amplification and overexpression of the MYCN proto-oncogene occurs in approximately 20% of neuroblastomas and is associated with advanced stage disease, rapid tumor prog…
View article: Supplementary Table 2 from N-myc and Noncoding RNAs in Neuroblastoma
Supplementary Table 2 from N-myc and Noncoding RNAs in Neuroblastoma Open
PDF file - 86K, Correlation between miRNA and N�myc expression in NB cell lines
View article: Supplementary Table 1 from N-myc and Noncoding RNAs in Neuroblastoma
Supplementary Table 1 from N-myc and Noncoding RNAs in Neuroblastoma Open
PDF file - 62K, Differentially expressed miRNAs between MNA and non�MNA primary tumors