Guercio, Marika Orlando, Domenico Di Cecca, Stefano Sinibaldi, Matilde Boffa, Iolanda Caruso, Simona Abbaszadeh, Zeinab Camera, Antonio Cembrola, Biancamaria Bovetti, Katia
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Published in
Haematologica
The prognosis of many patients with chemotherapy-refractory or multiply relapsed CD30+ non-Hodgkin Lymphoma (NHL) or Hodgkin lymphoma (HL) still remains poor, and novel therapeutic approaches are warranted to address this unmet clinical need. In light of this consideration, we designed and pre-clinically validated a Chimeric Antigen Receptor (CAR) ...
D'Altri, Teresa Wilhelmson, Anna S Schuster, Mikkel B Wenzel, Anne Kalvisa, Adrija Pundhir, Sachin Meldgaard Hansen, Anne Porse, Bo T
Published in
Haematologica
ASXL1 is one of the most commonly mutated genes in myeloid malignancies, including Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML). In order to further our understanding of the role of ASXL1 lesions in malignant hematopoiesis, we generated a novel knock-in mouse model carrying the most frequent ASXL1 mutation identified in MDS patie...
Loftus, Joseph P Yahiaoui, Anella Brown, Patrick A Niswander, Lisa M Bagashev, Asen Wang, Min Schauf, Allyson Tannheimer, Stacey Tasian, Sarah K
Published in
Haematologica
Survival of infants with KMT2A-rearranged (R) acute lymphoblastic leukemia (ALL) remains dismal despite intensive chemotherapy. We observed constitutive phosphorylation of spleen tyrosine kinase (SYK) and associated signaling proteins in infant ALL patient-derived xenograft (PDX) model specimens and hypothesized that the SYK inhibitor entospletinib...
Karampini, Ellie Bürgisser, Petra E Olins, Jenny Mulder, Aat A Jost, Carolina R Geerts, Dirk Voorberg, Jan Bierings, Ruben
Published in
Haematologica
Von Willebrand factor (VWF) is a multimeric hemostatic protein that is synthesized in endothelial cells, where it is stored for secretion in elongated secretory organelles, so-called Weibel-Palade bodies (WPBs). Hemostatic activity of VWF is strongly tied to WPB length, but how endothelial cells control the dimensions of their WPBs is unclear. In t...
Mina, Roberto Bonello, Francesca Petrucci, Maria Teresa Liberati, Anna Marina Conticello, Concetta Ballanti, Stelvio Musto, Pellegrino Olivieri, Attilio Benevolo, Giulia Capra, Andrea
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Published in
Haematologica
Despite remarkable advances in the treatment of multiple myeloma in the last decades, the prognosis of patients harboring high-risk cytogenetic abnormalities remains dismal as compared to that of standard-risk patients. Proteasome inhibitors demonstrated to partially ameliorate the prognosis of high-risk patients. We pooled together data from two p...
Hanna, Bola S Yazdanparast, Haniyeh Demerdash, Yasmin Roessner, Philipp M Schulz, Ralph Lichter, Peter Stilgenbauer, Stephan Seiffert, Martina
Published in
Haematologica
Ibrutinib is a bruton's tyrosine kinase (BTK) inhibitor approved for the treatment of multiple B-cell malignancies, including chronic lymphocytic leukemia (CLL). In addition to blocking B-cell receptor signaling and chemokine receptor-mediated pathways in CLL cells, that are known drivers of disease, ibrutinib also affects the microenvironment in C...
Shi, Yuzhe Beckett, Melanie C Blair, Helen J Tirtakusuma, Ricky Nakjang, Sirintra Enshaei, Amir Halsey, Christina Vormoor, Josef Heidenreich, Olaf Krippner-Heidenreich, Anja
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Published in
Haematologica
T-cell Acute Lymphoblastic Leukemia (T-ALL) is frequently characterized by glucocorticoid (GC) resistance, which is associated with inferior outcomes, thus highlighting the need for novel therapeutic approaches for GC resistant T-ALL. The pTCR/TCR signaling pathways play a critical role in cell fate decisions during physiological thymocyte developm...
Lewis, Thomas Corcoran, David B Thurston, David E Giles, Peter J Ashelford, Kevin Walsby, Elisabeth J Fegan, Christopher D Pepper, Andrea G S Miraz Rahman, Khondaker Pepper, Chris
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Published in
Haematologica
Chronic lymphocytic leukemia (CLL) and multiple myeloma (MM) are incurable hematological malignancies that are pathologically linked with aberrant NF-κB activation. In this study, we identified a group of novel C8-linked benzofused Pyrrolo[2,1-c][1,4]benzodiazepines (PBD) monomeric hybrids capable of sequence-selective inhibition of NF-κB with low ...
Logue, Jennifer M Zucchetti, Elisa Bachmeier, Christina A Krivenko, Gabriel S Larson, Victoria Ninh, Daniel Grillo, Giovanni Cao, Biwei Kim, Jongphil Chavez, Julio C
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Published in
Haematologica
CD19 CAR T-cell therapy with axicabtagene ciloleucel (axi-cel) for relapsed or refractory (R/R) large B cell lymphoma (LBCL) may lead to durable remissions, however, prolonged cytopenias and infections may occur. In this single center retrospective study of 85 patients, we characterized immune reconstitution and infections for patients remaining in...
Wang, Wei Khoury, Joseph D Miranda, Roberto N Jorgensen, Jeffrey L Xu, Jie Loghavi, Sanam Li, Shaoying Pemmaraju, Naveen Nguyen, Than Medeiros, L Jeffrey
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Published in
Haematologica
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematopoietic neoplasm whose immunophenotype remains incompletely characterized, particularly in terms of distinction from reactive plasmacytoid dendritic cells (PDCs). This limitation complicates detection of low-level involvement by BPDCN as well as minimal residual disease (MRD) asse...
