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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

Correction of ineffective erythropoiesis and durable clinical benefit with exagamglogene autotemcel for transfusion-dependent β-thalassemia

Blood (2025) 146 (Supplement 1): 6089.

https://doi.org/10.1182/blood-2025-6089

Published: 2025

Journal Articles

Outcomes in pediatric patients with HSCT-TMA treated with ravulizumab

Michelle Schoettler, David Bueno, Sonali Chaudhury, Jude Fry, Eric Bachman, Min Qin, Elsa Konig, Aviva Krauss, Sarah Lawson, Yoshiyuki Takahashi, Christopher Dvorak, Franco Locatelli

Blood (2025) 146 (Supplement 1): 1052.

https://doi.org/10.1182/blood-2025-1052

Published: 2025

Journal Articles

Use of emapalumab is associated with rapid and sustained benefits in primary HLH subgroups, including CNS involvement and previously untreated patients: Pooled analysis of prospective trials NI-0501-04, NI-0501-05 and NI-0501-09

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

Blood (2025) 146 (Supplement 1): 2979.

https://doi.org/10.1182/blood-2025-2979

Published: 2025

Journal Articles

HEM-ismart: An international proof-of-concept therapeutic stratification trial of molecular anomalies in relapsed or refractory hematological malignancies in children

Blood (2025) 146 (Supplement 1): 1582.

https://doi.org/10.1182/blood-2025-1582

Published: 2025

Journal Articles

The need for a subsequent HCT is a late & continuous risk and with significant impact in overall survival in haemoaglobinopathies: Real-world data from 2000–2022. an analysis of the european society for blood and marrow transplantation (EBMT) hemoglobinopathy working party.

Josu de la Fuente, Donatella Baronciani, Mathilde Fekom, Arnaud Dalissier, Marco Zecca, Franco Locatelli, Giorgio La Nasa, Raffaella Cerretti, Tariq Ghafoor, Giuseppe Visani, Hakan Ozdogu, Fabio Ciceri, Ashrafsadat Mousavi, Sandrine Bremathas, Selim Corbacioglu, Emanuele Angelucci

Blood (2025) 146 (Supplement 1): 178.

https://doi.org/10.1182/blood-2025-178

Published: 2025

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

Outcomes of 544 patients with t(6;9) Acute Myeloid Leukemia undergoing allogeneic stem cell transplantation: An EBMT Study on behalf of the acute leukemia working party (ALWP) and the pediatric diseases working party (PDWP)

Blood (2025) 146 (Supplement 1): 6066.

https://doi.org/10.1182/blood-2025-6066

Published: 2025

Journal Articles

Prevalence, clinical implications and impact of stem cell transplantation in UBTF-TD positive AML

Valeria Ceolin, Rhonda Ries, Jack H. Peplinski, Danielle Kirkey, Manuela Spadea, Vincenzo Apolito, Katherine Tarlock, Grace Hawkins, Melia Blankenfeld, Isabel Lira, Nika Panahi, Bruce Liu, Christian Thiede, Franco Locatelli, Francesco Saglio, Franca Fagioli, Soheil Meshinchi

Blood (2025) 146 (Supplement 1): 221.

https://doi.org/10.1182/blood-2025-221

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

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

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

Correction of ineffective erythropoiesis and durable clinical benefit with exagamglogene autotemcel for transfusion-dependent β-thalassemia

Outcomes in pediatric patients with HSCT-TMA treated with ravulizumab

Use of emapalumab is associated with rapid and sustained benefits in primary HLH subgroups, including CNS involvement and previously untreated patients: Pooled analysis of prospective trials NI-0501-04, NI-0501-05 and NI-0501-09

HEM-ismart: An international proof-of-concept therapeutic stratification trial of molecular anomalies in relapsed or refractory hematological malignancies in children

The need for a subsequent HCT is a late & continuous risk and with significant impact in overall survival in haemoaglobinopathies: Real-world data from 2000–2022. an analysis of the european society for blood and marrow transplantation (EBMT) hemoglobinopathy working party.

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

Outcomes of 544 patients with t(6;9) Acute Myeloid Leukemia undergoing allogeneic stem cell transplantation: An EBMT Study on behalf of the acute leukemia working party (ALWP) and the pediatric diseases working party (PDWP)

Prevalence, clinical implications and impact of stem cell transplantation in UBTF-TD positive AML

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

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

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