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Open AccessEconomic evaluation of anti-CD19 CAR T-cell pathway for large B-cell lymphomas in the real-life setting: the experience of an Italian hub center in the first three years of activity
Poor literature report actual and detailed costs of chimeric antigen receptor (CAR) T-cell pathway in a real-life setting. We retrospectively collect data for all patients with relapsed/refractory aggressive l...
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Article
Open AccessRuxolitinib for the treatment of acute and chronic graft-versus-host disease in children: a systematic review and individual patient data meta-analysis
Steroid-refractory graft-versus-host disease (SR-GvHD) represents a major complication of pediatric allogenic hematopoietic stem cell transplantation. Ruxolitinib, a selective JAK 1–2 inhibitor, showed promisi...
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Article
Letermovir for Cytomegalovirus infection in pediatric patients undergoing allogenic hematopoietic stem cell transplantation: a real-life study by the Infectious Diseases Working Group of Italian Association of Pediatric Hematology-Oncology (AIEOP)
Letermovir prophylaxis revolutionized the approach to Cytomegalovirus infection in adult hematopoietic stem cell transplant (HCT), while data in pediatric setting are still lacking. We retrospectively analyzed...
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Response to: meta-analysis on allogeneic transplant for treating pediatric patients with acute myeloid leukemia in first remission: reanalysis of primary data
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Open AccessSecond allogeneic stem cell transplantation can rescue a significant proportion of patients with JMML relapsing after first allograft
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Article
Open AccessERCC6L2-related disease: a novel entity of bone marrow failure disorder with high risk of clonal evolution
ERCC excision repair 6 like 2 (ERCC6L2) gene encodes for different helicase-like protein members of the Snf2 family involved in transcription-coupled nucleotide excision repair and in cell proliferation. Germline...
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Antibiotic prophylaxis and management of infections in pediatric hematopoietic stem cell transplantation: a survey from the Stem Cell Transplant and the Infectious Disease Working Groups of the AIEOP network
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Open AccessAllogeneic hematopoietic stem cell transplantation for pediatric acute myeloid leukemia in first complete remission: a meta-analysis
Identification of pediatric patients with acute myeloid leukemia (AML) candidates to receive allogeneic hematopoietic stem cell transplantation (allo-HSCT) in first complete remission (CR1) is still a matter o...
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Open AccessHematopoietic stem cell transplantation in children and adolescents with GATA2-related myelodysplastic syndrome
GATA2 deficiency is a heterogeneous multi-system disorder characterized by a high risk of develo** myelodysplastic syndrome (MDS) and myeloid leukemia. We analyzed the outcome of 65 patients reported to the ...
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Open AccessThe altered transcriptome of pediatric myelodysplastic syndrome revealed by RNA sequencing
Pediatric myelodysplastic syndrome (PMDS) is a very rare and still poorly characterized disorder. In this work, we identified novel potential targets of PMDS by determining genes with aberrant expression, whic...
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Open AccessSynonymous GATA2 mutations result in selective loss of mutated RNA and are common in patients with GATA2 deficiency
Deficiency of the transcription factor GATA2 is a highly penetrant genetic disorder predisposing to myelodysplastic syndromes (MDS) and immunodeficiency. It has been recognized as the most common cause underly...
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Open AccessHh/Gli antagonist in acute myeloid leukemia with CBFA2T3-GLIS2 fusion gene
CBFA2T3-GLIS2 is a fusion gene found in 17% of non-Down syndrome acute megakaryoblastic leukemia (non-DS AMKL, FAB M7) and in 8% of pediatric cytogenetically normal acute myeloid leuke...
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Open AccessIdentification of the NUP98-PHF23 fusion gene in pediatric cytogenetically normal acute myeloid leukemia by whole-transcriptome sequencing
The genomic landscape of children with acute myeloid leukemia (AML) who do not carry any cytogenetic abnormality (CN-AML) is particularly heterogeneous and challenging, being characterized by different clinica...