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Journal Articles

Hematopoietic stem cell transplantation in 746 children with osteopetrosis: A study of the inborn errors working party of the EBMT

Robert Chiesa, Michael H. Albert, Patrick Gilbert, Jeroen Knippenberg, Irina Zaidman, Hawazen Alsaedi, Fulvio Porta, R.F. Wynn, Rakefet Sidlik Muskatel, Serap Aksoylar, Barış Kuşkonmaz, Zohreh Nademi, Sarah Lawson, Akif Yesilipek, Kerstin Felgentreff, Marta Gonzalez Vicent, Juliana Silva, Tatyana Bykova, O. Alphan Kupesiz, Marco Zecca, Maura Faraci, Amir Hamidieh, Mayada Abu Shanap, Etai Adam, Tariq Ghafoor, Musa Karakükcü, Natalia Maximova, Sophie van Lancker, Catherine Paillard, Marleen Renard, Franco Locatelli, Martin Castelle, Persis Amrolia, Srividhya Senthil, Cécile Renard, Mohammed Essa, Benedicte Bruno, Cristina Díaz-de-Heredia, Ingrid Furlan, Renata Formankova, Matthias Felber, Nuno Miranda, Kanchan Rao, Baris Malbora, Jacek Winiarski, Robbert Bredius, Beki James, Jean-Sebastien Diana, Melek Erdem, Amal Alseraihy, Melissa Gabriel, Bernd Gruhn, Tania Masmas, Alessandra Biffi, Katarzyna Drabko, Krzysztof Kalwak, David Bueno, Manfred Hoenig, Carlos Vaz, Katharina Wustrau, Benedicte Brichard, Samppa Ryhänen, Anna Villa, Benedicte Neven, Mehtap Sirin, Despina Moshous, Ansgar Schulz

Blood (2025) 146 (Supplement 1): 165.

https://doi.org/10.1182/blood-2025-165

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

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

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

Central Nervous System Disorders (CNSD) Following Hematopoietic Stem Cell Transplantation (HSCT): A Prospective Study from the Infectious Diseases Working Party (IDWP) and the Transplant Complications Working Party (TCWP), European Society of Blood and Marrow Transplantation (EBMT)

Blood (2024) 144 (Supplement 1): 3497.

https://doi.org/10.1182/blood-2024-194695

Published: 2024

Journal Articles

Analysis of Outcomes after Second Allogeneic Stem Cell Transplant in Relapsed Acute Myeloid Leukaemia in Children: A Study from the EBMT Pediatric Diseases Working Party

Blood (2024) 144 (Supplement 1): 2182.

https://doi.org/10.1182/blood-2024-207029

Published: 2024

Journal Articles

Outcomes of HLA-mismatched HSCT with TCRαβ/CD19 depletion or post-HSCT cyclophosphamide for inborn errors of immunity

Blood (2024) 144 (5): 565–580.

https://doi.org/10.1182/blood.2024024038

Published: 2024

Includes: Supplemental data

Journal Articles

CD371-positive pediatric B-cell acute lymphoblastic leukemia: propensity to lineage switch and slow early response to treatment

Blood (2024) 143 (17): 1738–1751.

https://doi.org/10.1182/blood.2023021952

Published: 2024

Includes: Supplemental data

Journal Articles

The Transcription Factors NFATc1 and NFATc2 Control Glucocorticoid Resistance in Pediatric T-Cell Acute Lymphoblastic Leukemia

Giulia Veltri, Alberto Peloso, Alice Cani, Elena Mariotto, Giulia Borile, Loris Russo, Chiara Frasson, Matilde Cescon, Egidio Iorio, Sonia Minuzzo, Panagiotis Ntziachristos, Alessandra Biffi, Barbara Buldini, Martina Pigazzi, Stefano Indraccolo, Silvia Bresolin, Valentina Serafin

Blood (2023) 142 (Supplement 1): 420.

https://doi.org/10.1182/blood-2023-181548

Published: 2023

Journal Articles

Characterization of the Molecular Landscape of Pediatric Acute Myeloid Leukemia: A Retrospective AIEOP AML2013/01 Study

Maddalena Benetton, Pietro Merli, Nicola Santoro, Luca Lo Nigro, Claudia Tregnago, Ambra Da Ros, Katia Polato, Veronica Zampieri, Barbara Michielotto, Samuela Francescato, Anna Leszl, Riccardo Masetti, Barbara Buldini, Alessandra Biffi, Franca Fagioli, Carmelo Rizzari, Franco Locatelli, Martina Pigazzi

Blood (2023) 142 (Supplement 1): 1580.

https://doi.org/10.1182/blood-2023-186458

Published: 2023

Journal Articles

Pre-Transplant Levels of Multiparametric Flow-Cytometry Measurable Residual Disease (MFC-MRD) Predict Outcomes in Children with Acute Myeloid Leukemia Given Allogeneic Hematopoietic Stem Cell Transplantation

Barbara Buldini, Pietro Merli, Elena Varotto, Riccardo Masetti, Maria Gabelli, Marco Zecca, Franca Fagioli, Pamela Scarparo, Mattia Algeri, Alessandra Biffi, Martina Pigazzi, Daria Pagliara, Franco Locatelli

Blood (2023) 142 (Supplement 1): 1590.

https://doi.org/10.1182/blood-2023-189080

Published: 2023

Journal Articles

Plasma S-Evs Mirnas in Pediatric B Lineage Leukemia: Transforming Factors of Bone Marrow Niche

Alice Cani, Carlotta Caterina Damanti, Federica Lovisa, Enrico Gaffo, Giulia Borile, Elisa Carraro, Pina Fusco, Elena Varotto, Stefania Bortoluzzi, Alessandra Biffi, Barbara Buldini, Carmelo Rizzari, Marta Pillon, Lara Mussolin, Silvia Bresolin

Blood (2023) 142 (Supplement 1): 4081.

https://doi.org/10.1182/blood-2023-188680

Published: 2023

Journal Articles

Specific Cytogenetic Abnormalities at Diagnosis Predict Survival after Hematopoietic Cell Transplant in Poor-Risk Pediatric Acute Myeloid Leukemia: A PDWP/EBMT Study

Blood (2022) 140 (Supplement 1): 1882–1884.

https://doi.org/10.1182/blood-2022-162596

Published: 2022

Journal Articles

Preclinical Development of a CAR-T Cell Approach Targeting the CD84 Antigen Associated to Pediatric Acute Myeloid Leukemia

Martina Pigazzi, Olivia Marini, Elena Porcù, Giovanni Faggin, Elena Varotto, Paolo Rizzardi, Barbara Buldini, Franco Locatelli, Alessandra Biffi

Blood (2022) 140 (Supplement 1): 10247–10248.

https://doi.org/10.1182/blood-2022-166790

Published: 2022

Journal Articles

CD72 Is a Pan-Tumor Antigen Associated with Childhood Acute Leukemia

Barbara Buldini, Giovanni Faggin, Elena Porcù, Pamela Scarparo, Katia Polato, Claudia Tregnago, Elena Varotto, Paolo Rizzardi, Carmelo Rizzari, Franco Locatelli, Alessandra Biffi, Martina Pigazzi

Blood (2022) 140 (Supplement 1): 4974–4975.

https://doi.org/10.1182/blood-2022-169040

Published: 2022

Journal Articles

The Impact of Pre-Transplant Extramedullary Disease on the Outcome of Allogeneic Hematopoietic Cell Transplantation for Acute Myeloid Leukemia in Children- on Behalf of PDWP/EBMT

Blood (2022) 140 (Supplement 1): 12938–12940.

https://doi.org/10.1182/blood-2022-162484

Published: 2022

Journal Articles

The Role of Hemoglobin and Hemolysis on Transcranial Doppler Velocities in Children with Sickle Cell Disease: Data from a Natural History Cohort

Giulia Reggiani, Beatrice Coppadoro, Vania Munaretto, Renzo Manara, Claudio Baracchini, Alessio Pieroni, Federica Viaro, Irene Agodoa, Anne Beaubrun, Alessandra Biffi, Laura Sainati, Raffaella Colombatti

Blood (2021) 138 (Supplement 1): 3092.

https://doi.org/10.1182/blood-2021-148043

Published: 2021

Journal Articles

Quality of Life in Children, Adolescents and Young Adults with Sickle Cell Disease and Their Caregivers during Standard of Care and after Bone Marrow Transplantation: A Single Center Report

Vania Munaretto, Giulia Reggiani, Chiara Munerol, Elizabeth Maran, Marina Perdibon, Anna Chiara Frigo, Alessandra Biffi, Laura Sainati, Raffaella Colombatti

Blood (2021) 138 (Supplement 1): 3032.

https://doi.org/10.1182/blood-2021-151297

Published: 2021

Journal Articles

Outcome of Allogeneic HSCT after Chemo-Based Conditioning in Infants with Acute Myeloid Leukemia in First Complete Remission: A Multicenter EBMT-PDWP Study

Blood (2021) 138 (Supplement 1): 2862.

https://doi.org/10.1182/blood-2021-145858

Published: 2021

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Hematopoietic stem cell transplantation in 746 children with osteopetrosis: A study of the inborn errors working party of the EBMT

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

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

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

Central Nervous System Disorders (CNSD) Following Hematopoietic Stem Cell Transplantation (HSCT): A Prospective Study from the Infectious Diseases Working Party (IDWP) and the Transplant Complications Working Party (TCWP), European Society of Blood and Marrow Transplantation (EBMT)

Analysis of Outcomes after Second Allogeneic Stem Cell Transplant in Relapsed Acute Myeloid Leukaemia in Children: A Study from the EBMT Pediatric Diseases Working Party

Outcomes of HLA-mismatched HSCT with TCRαβ/CD19 depletion or post-HSCT cyclophosphamide for inborn errors of immunity

CD371-positive pediatric B-cell acute lymphoblastic leukemia: propensity to lineage switch and slow early response to treatment

The Transcription Factors NFATc1 and NFATc2 Control Glucocorticoid Resistance in Pediatric T-Cell Acute Lymphoblastic Leukemia

Characterization of the Molecular Landscape of Pediatric Acute Myeloid Leukemia: A Retrospective AIEOP AML2013/01 Study

Pre-Transplant Levels of Multiparametric Flow-Cytometry Measurable Residual Disease (MFC-MRD) Predict Outcomes in Children with Acute Myeloid Leukemia Given Allogeneic Hematopoietic Stem Cell Transplantation

Plasma S-Evs Mirnas in Pediatric B Lineage Leukemia: Transforming Factors of Bone Marrow Niche

Specific Cytogenetic Abnormalities at Diagnosis Predict Survival after Hematopoietic Cell Transplant in Poor-Risk Pediatric Acute Myeloid Leukemia: A PDWP/EBMT Study

Preclinical Development of a CAR-T Cell Approach Targeting the CD84 Antigen Associated to Pediatric Acute Myeloid Leukemia

CD72 Is a Pan-Tumor Antigen Associated with Childhood Acute Leukemia

The Impact of Pre-Transplant Extramedullary Disease on the Outcome of Allogeneic Hematopoietic Cell Transplantation for Acute Myeloid Leukemia in Children- on Behalf of PDWP/EBMT

The Role of Hemoglobin and Hemolysis on Transcranial Doppler Velocities in Children with Sickle Cell Disease: Data from a Natural History Cohort

Quality of Life in Children, Adolescents and Young Adults with Sickle Cell Disease and Their Caregivers during Standard of Care and after Bone Marrow Transplantation: A Single Center Report

Outcome of Allogeneic HSCT after Chemo-Based Conditioning in Infants with Acute Myeloid Leukemia in First Complete Remission: A Multicenter EBMT-PDWP Study

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