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Journal Articles

Outcome of children with B-cell acute lymphoblastic leukemia with hypodiploidy or BCR::ABL1 fusion undergoing allogeneic HSCT

Blood blood.2025030951.

https://doi.org/10.1182/blood.2025030951

Published: 2025

Includes: Supplemental data

Journal Articles

Allogeneic hematopoietic cell transplantation in dyskeratosis congenita: Short and long- term outcome of 183 consecutive patients transplanted at EBMT centers

Blood (2025) 146 (Supplement 1): 2490.

https://doi.org/10.1182/blood-2025-2490

Published: 2025

Journal Articles

Chemotherapy-free treatment for children and adolescents with newly diagnosed acute promyelocytic leukemia (APL): Results of the prospective ICC-APL-02 study.

Blood (2025) 146 (Supplement 1): 880.

https://doi.org/10.1182/blood-2025-880

Published: 2025

Journal Articles

Comparison of outcomes in pediatric patients with acute leukemia undergoing allogeneic HLA-haploidentical hematopoietic stem cell transplantation (HSCT) after in vitro a/b T- and CD19-cell depletion or In Vivo administration of post-transplant cyclophosphamide: An EBMT pediatric working party study

Blood (2025) 146 (Supplement 1): 515.

https://doi.org/10.1182/blood-2025-515

Published: 2025

Journal Articles

Reduced post-transplant alpha-diversity and domination by enterococcus faecium are associated with increased mortality in pediatric recipients of allogeneic hematopoietic cell transplantation.

Blood (2025) 146 (Supplement 1): 513.

https://doi.org/10.1182/blood-2025-513

Published: 2025

Journal Articles

A novel B7-H3-specific binder enables effective CAR T-cell therapy in an Acute Myeloid Leukemia experimental model

Pamela De Falco, Alessio Ottaviani, Michele Veneruso, Marilena Margiotta, Simona Manni, Marika Guercio, Francesca Del Bufalo, Roberta Castriconi, Cristina Bottino, Danielle Kirkey, Soheil Meshinchi, Pietro Merli, Marco Becilli, Biagio De Angelis, Concetta Quintarelli, Franco Locatelli

Blood (2025) 146 (Supplement 1): 6095.

https://doi.org/10.1182/blood-2025-6095

Published: 2025

Journal Articles

First results of exagamglogene autotemcel in pediatric patients aged 5-11 years with transfusion-dependent β-thalassemia or sickle cell disease with recurrent severe vaso-occlusive crises

Blood (2025) 146 (Supplement 1): 379.

https://doi.org/10.1182/blood-2025-379

Published: 2025

Journal Articles

Unfavorable outcomes in pediatric PAX5r B-cell precursor acute lymphoblastic leukemia: FLT3 targeting as a potential novel treatment strategy

Blood (2025) 146 (Supplement 1): 760.

https://doi.org/10.1182/blood-2025-760

Published: 2025

Journal Articles

Emapalumab induces rapid, durable responses and reliable bridging to curative HSCT in patients with primary HLH: Pooled analysis of prospective trials NI-0501-04, NI-0501-05 and NI-0501-09

Michael Jordan, Franco Locatelli, Didier Halimi, Elena Burillo, Brian Jamieson, Andrew Danquah, Emmanuel Monnet

Blood (2025) 146 (Supplement 1): 4758.

https://doi.org/10.1182/blood-2025-4758

Published: 2025

Journal Articles

Safety and efficacy of surovatamig (AZD0486) in adolescent and adult patients with relapsed or refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL): Updated Results from the Phase 1/2 SYRUS study

Blood (2025) 146 (Supplement 1): 3345.

https://doi.org/10.1182/blood-2025-3345

Published: 2025

Journal Articles

CD19 antigen: From tumor target to safety switch able to improve the safety profile of CAR T cell therapy

Simona Manni, Laura Iaffaldano, Maria Cecilia Quadraccia, Mariasole Aurigemma, Marika Guercio, Simona Caruso, Maria Antonietta De Ioris, Giada Del Baldo, Danielle Kirkey, Soheil Meshinchi, Pietro Merli, Marco Becilli, Francesca Del Bufalo, Biagio De Angelis, Franco Locatelli, Concetta Quintarelli

Blood (2025) 146 (Supplement 1): 2541.

https://doi.org/10.1182/blood-2025-2541

Published: 2025

Journal Articles

Long-term follow-up demonstrates durable clinical benefits of exagamglogene autotemcel for sickle cell disease with recurrent vaso-occlusive crises: Final results of climb SCD-121

Blood (2025) 146 (Supplement 1): 2537.

https://doi.org/10.1182/blood-2025-2537

Published: 2025

Journal Articles

Soluble factors in the AML secretome drive macrophage polarization toward an M2-like state promoting an immunosuppressive microenvironment

Giorgia Longo, Claudia Tregnago, Sara Perpinello, Ambra Da Ros, Maddalena Benetton, Giulia Borella, Elisabetta Campodoni, Monica Sandri, Riccardo Masetti, Alessandra Biffi, Franco Locatelli, Martina Pigazzi

Blood (2025) 146 (Supplement 1): 1407.

https://doi.org/10.1182/blood-2025-1407

Published: 2025

Journal Articles

Next generation sequencing profiling of pediatric Acute Myeloid Leukemia reveals distinct genetic landscapes across cytogenetic subgroups

Maddalena Benetton, Claudia Tregnago, Katia Polato, Diego Calvetti, Veronica Zampieri, Manuela Tumino, Barbara Buldini, Alessandra Biffi, Valeria Paganelli, Pietro Merli, Giuseppe Menna, Franca Fagioli, Carmelo Rizzari, Franco Locatelli, Martina Pigazzi

Blood (2025) 146 (Supplement 1): 5268.

https://doi.org/10.1182/blood-2025-5268

Published: 2025

Journal Articles

KMT2A::AF6 fusion protein localizes to PML nuclear bodies and undergoes ATO-induced degradation: A potential novel therapeutic approach for KMT2A::AF6-rearranged Acute Myeloid Leukemia

Claudia Tregnago, Sara Perpinello, Ambra Da Ros, Maddalena Benetton, Giorgia Longo, Helmut Hanenberg, Jan-Henning Klusmann, Dirk Reinhardt, Franco Locatelli, Martina Pigazzi

Blood (2025) 146 (Supplement 1): 753.

https://doi.org/10.1182/blood-2025-753

Published: 2025

Journal Articles

Venetoclax and azacytidine in childhood primary advanced myelodysplastic syndromes, refractory/relapsed Acute Myeloid Leukemia and therapy-related myeloid diseases

Francesco Baccelli, Pietro Merli, Gennaro Pagano, Marco Becilli, Katia Girardi, Valeria Paganelli, Davide Leardini, Edoardo Muratore, Francesca Vendemini, Giuliana Beneduce, Manuela Tumino, Francesco Saglio, Elena Sieni, Elena Mastrodicasa, Francesco Paolo Tambaro, Riccardo Masetti, Franco Locatelli

Blood (2025) 146 (Supplement 1): 289.

https://doi.org/10.1182/blood-2025-289

Published: 2025

Journal Articles

NUP98::NSD1 fusion is associated with poor outcomes in Acute Myeloid Leukemia regardless of co-occurring mutations and treatment regimen

Katherine Tarlock, Danielle Kirkey, Rhonda Ries, Jack H. Peplinski, Todd Alonzo, Robert Gerbing, Jim Wang, Todd Cooper, Alan Gamis, Richard Aplenc, Martina Pigazzi, Franco Locatelli, Jessica Pollard, Soheil Meshinchi

Blood (2025) 146 (Supplement 1): 1702.

https://doi.org/10.1182/blood-2025-1702

Published: 2025

Journal Articles

ITCC-101/APAL2020D: A randomized Phase 3 trial of fludarabine/cytarabine/gemtuzumab ozogamycin with or without venetoclax in children with relapsed Acute Myeloid Leukemia

Blood (2025) 146 (Supplement 1): 1656.

https://doi.org/10.1182/blood-2025-1656

Published: 2025

Journal Articles

A single cell multiomic approach to dissect immunophenotypic plasticity in B-cell precursor acute lymphoblastic leukemia (BCP-ALL) pediatric patients

Giulia Gomiero, Alberto Peloso, Elena Varotto, Pamela Scarparo, Chiara Frasson, Alice Cani, Grazia Fazio, Giovanni Cazzaniga, Franco Locatelli, Carmelo Rizzari, Alessandra Biffi, Michael Dworzak, Silvia Bresolin, Barbara Buldini

Blood (2025) 146 (Supplement 1): 36.

https://doi.org/10.1182/blood-2025-36

Published: 2025

Journal Articles

DNA methylation episignature as a novel diagnostic tool for diamond-blackfan anemia

Paola Quarello, Karim Karimi, Slavica Trajkova, Emanuela Garelli, Tommaso Pippucci, Serena Rizzo, Marco Zecca, Francesca Fioredda, Angelica Barone, Simone Cesaro, Maria Gabelli, Francesca Torchio, Giulia Zucchetti, Maria Elena Cantarini, Paola Corti, Franco Locatelli, Franca Fagioli, Bekim Sadikovic, Alfredo Brusco

Blood (2025) 146 (Supplement 1): 4673.

https://doi.org/10.1182/blood-2025-4673

Published: 2025

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Outcome of children with B-cell acute lymphoblastic leukemia with hypodiploidy or BCR::ABL1 fusion undergoing allogeneic HSCT

Allogeneic hematopoietic cell transplantation in dyskeratosis congenita: Short and long- term outcome of 183 consecutive patients transplanted at EBMT centers

Chemotherapy-free treatment for children and adolescents with newly diagnosed acute promyelocytic leukemia (APL): Results of the prospective ICC-APL-02 study.

Comparison of outcomes in pediatric patients with acute leukemia undergoing allogeneic HLA-haploidentical hematopoietic stem cell transplantation (HSCT) after in vitro a/b T- and CD19-cell depletion or In Vivo administration of post-transplant cyclophosphamide: An EBMT pediatric working party study

Reduced post-transplant alpha-diversity and domination by enterococcus faecium are associated with increased mortality in pediatric recipients of allogeneic hematopoietic cell transplantation.

A novel B7-H3-specific binder enables effective CAR T-cell therapy in an Acute Myeloid Leukemia experimental model

First results of exagamglogene autotemcel in pediatric patients aged 5-11 years with transfusion-dependent β-thalassemia or sickle cell disease with recurrent severe vaso-occlusive crises

Unfavorable outcomes in pediatric PAX5r B-cell precursor acute lymphoblastic leukemia: FLT3 targeting as a potential novel treatment strategy

Emapalumab induces rapid, durable responses and reliable bridging to curative HSCT in patients with primary HLH: Pooled analysis of prospective trials NI-0501-04, NI-0501-05 and NI-0501-09

Safety and efficacy of surovatamig (AZD0486) in adolescent and adult patients with relapsed or refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL): Updated Results from the Phase 1/2 SYRUS study

CD19 antigen: From tumor target to safety switch able to improve the safety profile of CAR T cell therapy

Long-term follow-up demonstrates durable clinical benefits of exagamglogene autotemcel for sickle cell disease with recurrent vaso-occlusive crises: Final results of climb SCD-121

Soluble factors in the AML secretome drive macrophage polarization toward an M2-like state promoting an immunosuppressive microenvironment

Next generation sequencing profiling of pediatric Acute Myeloid Leukemia reveals distinct genetic landscapes across cytogenetic subgroups

KMT2A::AF6 fusion protein localizes to PML nuclear bodies and undergoes ATO-induced degradation: A potential novel therapeutic approach for KMT2A::AF6-rearranged Acute Myeloid Leukemia

Venetoclax and azacytidine in childhood primary advanced myelodysplastic syndromes, refractory/relapsed Acute Myeloid Leukemia and therapy-related myeloid diseases

NUP98::NSD1 fusion is associated with poor outcomes in Acute Myeloid Leukemia regardless of co-occurring mutations and treatment regimen

ITCC-101/APAL2020D: A randomized Phase 3 trial of fludarabine/cytarabine/gemtuzumab ozogamycin with or without venetoclax in children with relapsed Acute Myeloid Leukemia

A single cell multiomic approach to dissect immunophenotypic plasticity in B-cell precursor acute lymphoblastic leukemia (BCP-ALL) pediatric patients

DNA methylation episignature as a novel diagnostic tool for diamond-blackfan anemia

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