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HLA-haploidentical stem cell transplantation in children with inherited bone marrow failure syndromes: A retrospective analysis on behalf of EBMT severe aplastic Anemia and pediatric diseases working parties Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-18 Stefano Giardino, Dirk-Jan Eikema, Brian Piepenbroek, Mattia Algeri, Mouhab Ayas, Maura Faraci, Abdelghani Tbakhi, Marco Zecca, Mohammed Essa, Bénédicte Neven, Yves Bertrand, Gaurav Kharya, Tatiana Bykova, Sarah Lawson, Mario Petrini, Alexander Mohseny, Fanny Rialland, Beki James, Anca Colita, Mony Fahd, Simone Cesaro, Ansgar Schulz, Katharina Kleinschmidt, Krzysztof Kałwak, Selim Corbacioglu, Carlo
Haploidentical stem cell transplantation (haplo-SCT) represents the main alternative for children with inherited bone marrow failure syndrome (I-BMF) lacking a matched donor. This retrospective study, conducted on behalf of the EBMT SAAWP and PDWP, aims to report the current outcomes of haplo-SCT in I-BMFs, comparing the different in vivo and ex vivo T-cell depletion approaches. One hundred and sixty-two
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Genetically determined telomere length and risk for haematologic diseases: results from large prospective cohorts and Mendelian Randomization analysis Blood Cancer J. (IF 12.8) Pub Date : 2024-03-18 Yang Li, Jia Chen, Ting Sun, Yunfei Chen, Rongfeng Fu, Xiaofan Liu, Feng Xue, Wei Liu, Mankai Ju, Xinyue Dai, Huan Dong, Huiyuan Li, Wentian Wang, Ying Chi, Lei Zhang
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Progression free survival of myeloma patients who become IFE-negative correlates with the detection of residual monoclonal free light chain (FLC) by mass spectrometry Blood Cancer J. (IF 12.8) Pub Date : 2024-03-18 H. V. Giles, M. T. Drayson, B. Kishore, C. Pawlyn, M. Kaiser, G. Cook, R. de Tute, R. G. Owen, D. Cairns, T. Menzies, F. E. Davies, G. J. Morgan, G. Pratt, G. H. Jackson
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Age-specific survival in acute myeloid leukemia in the Nordic countries through a half century Blood Cancer J. (IF 12.8) Pub Date : 2024-03-14 Kari Hemminki, Frantisek Zitricky, Asta Försti, Mika Kontro, Bjorn T. Gjertsen, Marianne Tang Severinsen, Gunnar Juliusson
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Beyond the marrow: insights from comprehensive next-generation sequencing of extramedullary multiple myeloma tumors Leukemia (IF 11.4) Pub Date : 2024-03-16 T. Jelinek, D. Zihala, T. Sevcikova, A. Anilkumar Sithara, V. Kapustova, H. Sahinbegovic, O. Venglar, L. Muronova, L. Broskevicova, S. Nenarokov, D. Bilek, T. Popkova, H. Plonkova, J. Vrana, V. Zidlik, P. Hurnik, M. Havel, M. Hrdinka, Z. Chyra, G. Stracquadanio, M. Simicek, R. Hajek
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CD22 CAR T cells demonstrate high response rates and safety in pediatric and adult B-ALL: Phase 1b results Leukemia (IF 11.4) Pub Date : 2024-03-15 Liora M. Schultz, Nikeshan Jeyakumar, Anne Marijn Kramer, Bita Sahaf, Hrishi Srinagesh, Parveen Shiraz, Neha Agarwal, Mark Hamilton, Courtney Erickson, Ashley Jacobs, Jennifer Moon, Christina Baggott, Sally Arai, Sushma Bharadwaj, Laura J. Johnston, Michaela Liedtke, Robert Lowsky, Everett Meyer, Robert Negrin, Andrew Rezvani, Judy Shizuru, Surbhi Sidana, Emily Egeler, Sharon Mavroukakis, Ramya Tunuguntla
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Differential in vivo roles of Mpl cytoplasmic tyrosine residues in murine hematopoiesis and myeloproliferative disease Leukemia (IF 11.4) Pub Date : 2024-03-15 Kira Behrens, Maria Kauppi, Elizabeth M. Viney, Andrew J. Kueh, Craig D. Hyland, Tracy A. Willson, Liam Salleh, Carolyn A. de Graaf, Jeffrey J. Babon, Marco J. Herold, Nicos A. Nicola, Warren S. Alexander
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NFKBIE mutations are selected by the tumor microenvironment and contribute to immune escape in chronic lymphocytic leukemia Leukemia (IF 11.4) Pub Date : 2024-03-15 Alice Bonato, Supriya Chakraborty, Riccardo Bomben, Giulia Canarutto, Giulia Felician, Claudio Martines, Antonella Zucchetto, Federico Pozzo, Marija Vujovikj, Jerry Polesel, Annalisa Chiarenza, Maria Ilaria Del Principe, Giovanni Del Poeta, Giovanni D’Arena, Roberto Marasca, Agostino Tafuri, Luca Laurenti, Silvano Piazza, Aleksandar J. Dimovski, Valter Gattei, Dimitar G. Efremov
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Favorable outcome of non‐myeloablative allogeneic transplantation in adult patients with severe sickle cell disease: A single center experience of 200 patients Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-16 Moussab Damlaj, Bader Alahmari, Ahmed Alaskar, Ayman Alhejazi, Husam Alsadi, Mazin Ahmed, Tahani Alanazi, Rasha Ahmed, Amani Alharbi, Inaam Shehabeddine, Afnan Alzaidi, Suha Alkhuraisat, Isam Mahassnah, Hamza Alquraan, Maybelle Ballili, Mohsen Alzahrani
Allogeneic hematopoietic stem cell transplant (HSCT) for adults with severe sickle cell disease (SCD) is potentially curative but not commonly utilized therapy due to complications such as graft failure (GF) and organ toxicity. Herein, we are reporting our long‐term outcome data of non‐myeloablative (NMA) HSCT in adults with severe SCD with emphasis on factors predicting event free survival (EFS).
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Thromboembolic risk of carfilzomib or bortezomib in combination with lenalidomide and dexamethasone for newly diagnosed multiple myeloma: A comparative systematic review and meta‐analysis Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-16 Bruno Almeida Costa, Thomaz Alexandre Costa, Sara Diaz Saravia, Nicole Felix, Carlyn Rose Tan, Neha Korde, Joshua Richter
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Flow cytometric analysis of peripheral blood neutrophil myeloperoxidase expression in myelodysplastic neoplasms Leukemia (IF 11.4) Pub Date : 2024-03-14 Tatiana Raskovalova, Marie-Christine Jacob, Sophie Park
In a letter to the editor, Oelschlaegel et al. [1] describes the new flow cytometry score (MDS-PB13) for the diagnosis of myelodysplastic syndromes (MDS) that relies on aberrant antigen expression of thirteen parameters in different cell populations of peripheral blood with 79–81% specificity and 61–70% sensibility. Suspicion of MDS is the commonest reason for bone marrow aspirate and biopsy in older
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Mutation order in acute myeloid leukemia identifies uncommon patterns of evolution and illuminates phenotypic heterogeneity Leukemia (IF 11.4) Pub Date : 2024-03-11 Matthew Schwede, Katharina Jahn, Jack Kuipers, Linde A. Miles, Robert L. Bowman, Troy Robinson, Ken Furudate, Hidetaka Uryu, Tomoyuki Tanaka, Yuya Sasaki, Asiri Ediriwickrema, Brooks Benard, Andrew J. Gentles, Ross Levine, Niko Beerenwinkel, Koichi Takahashi, Ravindra Majeti
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Proteomic characterization of esophageal squamous cell carcinoma response to immunotherapy reveals potential therapeutic strategy and predictive biomarkers J. Hematol. Oncol. (IF 28.5) Pub Date : 2024-03-15 Fahan Ma, Yan Li, Chan Xiang, Bing Wang, Jie Lv, Jinzhi Wei, Zhaoyu Qin, Yan Pu, Kai Li, Haohua Teng, Subei Tan, Jinwen Feng, Zhanxian Shang, Yunzhi Wang, Sha Tian, Changsheng Du, Yuchen Han, Chen Ding
Immunotherapy is the first-line therapy for esophageal squamous cell carcinoma (ESCC), yet many patients do not respond due to drug resistance and the lack of reliable predictive markers. We collected 73 ESCC patients (including discovery cohort and validation cohort) without immune thrombocytopenia and undergoing anti-PD1 immunotherapy. Proteomic and phosphoproteomic analysis of 73 ESCC treatment-naive
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PTCy versus ATG as graft-versus-host disease prophylaxis in mismatched unrelated stem cell transplantation Blood Cancer J. (IF 12.8) Pub Date : 2024-03-15 Olaf Penack, Mouad Abouqateb, Christophe Peczynski, William Boreland, Zafer Gülbas, Tobias Gedde-Dahl, Cristina Castilla-Llorente, Nicolaus Kröger, Mathias Eder, Alessandro Rambaldi, Francesca Bonifazi, Igor Wolfgang Blau, Matthias Stelljes, Peter Dreger, Ivan Moiseev, Hélène Schoemans, Christian Koenecke, Zinaida Peric
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In vivo expression of anti-CD19/CD3 BiTE by liver-targeted AAV for the treatment of B cell malignancies Blood Cancer J. (IF 12.8) Pub Date : 2024-03-15 Zhiqiang Song, Ping Liu, Dongliang Zhang, Tao Wang, Wenqin Yue, Yuke Geng, Na Liu, Yang Wang, Jianmin Yang
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Optical nanomaterial-based detection of biomarkers in liquid biopsy J. Hematol. Oncol. (IF 28.5) Pub Date : 2024-03-14 Young Jun Kim, Won-Yeop Rho, Seung-min Park, Bong-Hyun Jun
Liquid biopsy, which is a minimally invasive procedure as an alternative to tissue biopsy, has been introduced as a new diagnostic/prognostic measure. By screening disease-related markers from the blood or other biofluids, it promises early diagnosis, timely prognostication, and effective treatment of the diseases. However, there will be a long way until its realization due to its conceptual and practical
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Systemic mastocytosis: dying or survivin Blood (IF 20.3) Pub Date : 2024-03-14 Joakim S. Dahlin, Gunnar Nilsson
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A road map of relapse in MDS after allo-HSCT Blood (IF 20.3) Pub Date : 2024-03-14 Juan Jose Rodriguez-Sevilla, Simona Colla
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Hemolysis impairs sickle cell erythropoiesis Blood (IF 20.3) Pub Date : 2024-03-14 Constance T. Noguchi
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Monomorphic epitheliotropic T-cell lymphoma with strong CD4 expression Blood (IF 20.3) Pub Date : 2024-03-14 Siba El Hussein
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Detection of signature double‐negative T cells is a predictive marker to identify autoimmune lymphoproliferative syndrome associated with FAS loss of function Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-14 Nina Eisenhauer, Maurizio Miano, Nora Naumann‐Bartsch, Jörg Leyh, Gianluca Dell'Orso, Michael Aigner, Gisela Fecker, Claas Hinze, Helmut Wittkowski, Heiko Bruns, Jakob Zierk, Markus Metzler, Peter D. Arkwright, Frederik Graw, Andreas Mackensen, Simon Völkl
The autoimmune lymphoproliferative syndrome (ALPS) is caused by defects in the FAS-dependent apoptosis leading to chronic lymphoproliferation, autoimmunity, chronic multilineage cytopenia, an increased risk for developing B cell lymphomas, and a characteristic accumulation of CD4/CD8-negative, T-cell receptor (TCR), αβ-positive T-cells (double-negative, DNT-cells).1, 2 The heterogeneous clinical phenotype
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JAK inhibitor treatment‐resistant splenomegaly before transplantation in myelofibrosis: Splenectomy or radiotherapy? Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-14 Maria Chiara Finazzi, Ayalew Tefferi, Alessandro Rambaldi
CONFLICT OF INTEREST STATEMENT The authors declare no conflicts of interest.
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A new chemotherapy‐free regimen of olverembatinib in combination with venetoclax and dexamethasone for newly diagnosed Ph+ acute lymphoblastic leukemia: Preliminary outcomes of a prospective study Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-14 Hailong Tang, Weijing Jia, Shuangshuang Jia, Runan Dong, Shan Gao, Juan Feng, Hongjuan Dong, Hongtao Gu, Tao Zhang, Ruifeng Yuan, Xiangxiang Liu, Lu Cheng, Shuya Zhou, Guangxun Gao
In the era following the development of tyrosine kinase inhibitors (TKIs), the current standard of treatment for clinically fit patients with previously untreated Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph + ALL) is first- or second-generation TKIs combined with chemotherapy or corticosteroids, followed by allogeneic stem cell transplantation (allo-HCT). However, relapse still
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Targeting glycolysis to rescue 2-hydroxyglutarate immunosuppressive effects in dendritic cells and acute myeloid leukemia. Haematologica (IF 10.1) Pub Date : 2024-03-14 Angela Maria Savino, Lucille Stuani
Not available.
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Low-dose non-steroidal anti-inflammatory drugs: a promising approach for the treatment of symptomatic bone marrow failure in Ghosal hematodiaphyseal dysplasia. Haematologica (IF 10.1) Pub Date : 2024-03-14 Jonathan Bordat, Felipe Suarez, Valérie Cormier-Daire, Regis Peffault De Latour, Jean Soulier, Veronique Meignin, Mathilde Doyard, Lise Larcher, Stephane Vanderbecken, Flore Sicre De Fontbrune
Not available.
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Efficacy of intravenous high-dose methotrexate in preventing relapse to the central nervous system in R-CHOP(-like)-treated, high-risk, diffuse large B-cell lymphoma patients and its effect on mortality: a systematic review and meta-analysis. Haematologica (IF 10.1) Pub Date : 2024-03-14 Elisabeth R Tolley, Christian Lewinter, Lars M Pedersen, Torsten Holm Nielsen
CNS relapse in patients with diffuse large B-cell lymphoma (DLBCL) carries a dismal prognosis with most clinical guidelines recommending CNS prophylaxis to patients deemed at high risk for CNS relapse. However, results from observational studies investigating the effect of CNS prophylaxis have yielded conflicting results.
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Imatinib treatment and longitudinal growth in pediatric patients with chronic myeloid leukemia: Influence of demographic, pharmacological, and genetic factors in the German CML-PAED cohort. Haematologica (IF 10.1) Pub Date : 2024-03-14 Sophie Stiehler, Stephanie Sembill, Oliver Schleicher, Michaela Marx, Manfred Rauh, Manuela Krumbholz, Axel Karow, Meinolf Suttorp, Joachim Woelfle, Carlo Maj, Markus Metzler
In children and adolescents, impaired growth due to tyrosine kinase inhibitor therapy remains an insufficiently studied adverse effect. This study examines demographic, pharmacological, and genetic factors associated with impaired longitudinal growth in a uniform pediatric cohort treated with imatinib. We analyzed 94 pediatric patients with chronic myeloid leukemia (CML) diagnosed in the chronic phase
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Validation of LymphGen classification on a 400-gene clinical next-generation sequencing panel in diffuse large B-cell lymphoma: real-world experience from a cancer center. Haematologica (IF 10.1) Pub Date : 2024-03-14 Meng-Lei Zhu, Esther Drill, Erel Joffe, Gilles Salles, Alfredo Rivas Delgado, Andrew Zelenetz, Maria Lia Palomba, Maria Arcila, Ahmet Dogan
Not available.
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Bendamustine and rituximab as first-line treatment for symptomatic splenic marginal zone lymphoma: long-term outcome and impact of early unmeasurable minimal residual disease attainment from the BRISMA/IELSG36 phase II study. Haematologica (IF 10.1) Pub Date : 2024-03-14 Emilio Iannitto, Simone Ferrero, Côme Bommier, Daniela Drandi, Martina Ferrante, Krimo Bouabdallah, Sylvain Carras, Guido Gini, Vincent Camus, Salvatrice Mancuso, Luigi Marcheselli, Angela Ferrari, Michele Merli, Benoit Tessoulin, Caterina Stelitano, Kheira Beldjord, Giovanni Roti, Fabrice Jardin, Barbara Castagnari, Francesca Palombi, Lucile Baseggio, Alexandra Traverse-Glehen, Claudio Tripodo, Anna
Not available.
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Classical meets malignant hematology: a case of acquired εγδβ-thalassemia in clonal hematopoiesis. Haematologica (IF 10.1) Pub Date : 2024-03-14 Armin P Piehler, Marietta Truger, Jan-Hendrik Kozik, Sandra Weissmann, Martin Schwonzen, Manja Meggendorfer, Wolfgang Kern, Torsten Haferlach, Gregor Hoermann, Claudia Haferlach
Hemoglobinopathies including thalassemias are among the most frequent genetic disorders worldwide. Primarily, these entities result from germline variants in the globin gene clusters and their cis-acting regulatory elements, and thus the WHO classifies thalassemias as inherited diseases. Non-inherited disorders of globin chain synthesis mimicking the phenotype of thalassemias have also been described
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Stroke without cerebral arteriopathy in sickle cell disease children: causes and treatment. Haematologica (IF 10.1) Pub Date : 2024-03-14 Sarah Liane Linguet, Suzanne Verlhac, Florence Missud, Laurent Holvoet-Vermaut, Valentine Brousse, Ghislaine Ithier, Alexandra Ntorkou, Emmanuelle Lesprit, Malika Benkerrou, Manoëlle Kossorotoff, Berengere Koehl
Cerebral arteriopathy (CA) in children with sickle cell disease (SCD) is classically described as chronic stenosis of arteries in the anterior brain circulation, leading to ischemic stroke. Some studies have however reported strokes in children with SCD but without CA. In order to better understand the etiology and risk factors of these strokes, we retrospectively analyzed ischemic strokes occurring
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Why are not all eligible chronic myeloid leukemia patients willing to attempt tyrosine kinase inhibitor discontinuation? A Czech nationwide analysis related to the TKI stopping trial HALF Leukemia (IF 11.4) Pub Date : 2024-03-12 Daniela Žáčková, Lukáš Semerád, Edgar Faber, Hana Klamová, Lukáš Stejskal, Petra Bělohlávková, Michal Karas, Eduard Cmunt, Olga Černá, Jiřina Procházková, Petra Čičátková, Anežka Kvetková, Tomáš Horňák, Ivana Skoumalová, Dana Srbová, Cyril Šálek, David Buffa, Jaroslava Voglová, Tomáš Jurček, Adam Folta, Ivana Ježíšková, Hana Žižková, Kateřina Machová Poláková, Tomáš Papajík, Pavel Žák, Pavel Jindra
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Allogeneic haematopoietic cell transplantation for advanced systemic mastocytosis: Best practice recommendations on behalf of the EBMT Practice Harmonisation and Guidelines Committee Leukemia (IF 11.4) Pub Date : 2024-03-12 Donal P. McLornan, Tomasz Czerw, Gandhi Damaj, Mark Ethell, Carmelo Gurnari, Juan Carlos Hernández-Boluda, Nicola Polverelli, Juliana Schwaab, Katja Sockel, Greco Raffaella, Francesco Onida, Isabel Sánchez-Ortega, Giorgia Battipaglia, Chiara Elena, Jason Gotlib, Andreas Reiter, Julien Rossignol, Celalettin Ustun, Peter Valent, Ibrahim Yakoub-Agha, Deepti H. Radia
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STAT3 protects hematopoietic stem cells by preventing activation of a deleterious autocrine type-I interferon response Leukemia (IF 11.4) Pub Date : 2024-03-11 Bhakti Patel, Yifan Zhou, Rachel L. Babcock, Feiyang Ma, M. Anna Zal, Dhiraj Kumar, Yusra B. Medik, Laura M. Kahn, Josué E. Pineda, Elizabeth M. Park, Sarah M. Schneider, Ximing Tang, Maria Gabriela Raso, Collene R. Jeter, Tomasz Zal, Karen Clise-Dwyer, Khandan Keyomarsi, Filippo G. Giancotti, Simona Colla, Stephanie S. Watowich
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Childbirth rates in women with myeloproliferative neoplasms Leukemia (IF 11.4) Pub Date : 2024-03-09 Anna Ravn Landtblom, Therese M-L Andersson, Anna L. V. Johansson, Frida E. Lundberg, Jan Samuelsson, Magnus Björkholm, Malin Hultcrantz
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Large differencies in age-specific survival in multiple myeloma in the nordic countries Blood Cancer J. (IF 12.8) Pub Date : 2024-03-11 Kari Hemminki, Frantisek Zitricky, Asta Försti, Raija Silvennoinen, Annette Vangsted, Markus Hansson
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MRD at the End of Induction and EFS in T-cell Lymphoblastic Lymphoma: Children’s Oncology Group Trial AALL1231. Blood (IF 20.3) Pub Date : 2024-03-13 Robert J. Hayashi, Michelle L. Hermiston, Brent L. Wood, David T. Teachey, Meenakshi Devidas, Zhiguo Chen, Robert D. Annett, Barbara L. Asselin, Keith August, Steve Cho, Kimberly P. Dunsmore, Jason Lawrence Freedman, Paul J. Galardy, Paul Harker-Murray, Terzah M. Horton, Alok I. Jaju, Allison Lam, Yoav H. Messinger, Rodney R. Miles, Maki Okada, Samir Patel, Eric S. Schafer, Tai Schechter, Kristin A
Defining prognostic variables in T-lymphoblastic lymphoma (T-LL) remains a challenge. AALL1231 was a COG phase 3 clinical trial for newly diagnosed with T Acute Lymphoblastic leukemia or T-LL patients randomizing children and young adults to a modified augmented BFM backbone to receive standard therapy (Arm A) or with addition of bortezomib (Arm B). Optional bone marrow (BM) samples to assess minimal
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A randomized double‐blind pilot study to evaluate the efficacy, safety, and tolerability of intravenous iron versus oral iron for the treatment of restless legs syndrome in patients with iron deficiency anemia Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-13 Vanessa Short, Richard Allen, Christopher J. Earley, Huzefa Bahrain, Stella Rineer, Kiumarce Kashi, Jesse Gerb, Michael Auerbach
Restless legs syndrome (RLS) is a neurological disorder that can have a profound effect on sleep and quality of life. Idiopathic RLS is associated with brain iron insufficiency despite normal peripheral iron stores. There is, however, a five‐ to six‐fold increase in prevalence of RLS in patients with iron deficiency anemia (IDA). Several open‐label trials have demonstrated symptomatic improvement in
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Redefining high risk multiple myeloma with an APOBEC/Inflammation-based classifier Leukemia (IF 11.4) Pub Date : 2024-03-09 Sarah Grasedieck, Afsaneh Panahi, Matthew C. Jarvis, Faezeh Borzooee, Reuben S. Harris, Mani Larijani, Hervé Avet-Loiseau, Mehmet Samur, Nikhil Munshi, Kevin Song, Arefeh Rouhi, Florian Kuchenbauer
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In vivo ablation of NFκB cascade effectors alleviates disease burden in myeloproliferative neoplasms Blood (IF 20.3) Pub Date : 2024-03-12 Angelo B. A. Laranjeira, Tim Kong, Steven C. Snyder, Mary C. Fulbright, Daniel A. C. Fisher, Daniel T. Starczynowski, Stephen T. Oh
Hyperactivation of the NFκB cascade propagates oncogenic signaling and pro-inflammation, which together augments disease burden in myeloproliferative neoplasms (MPNs). Here, we systematically ablate NFκB signaling effectors to identify core dependencies using a series of primary samples and syngeneic and patient-derived xenograft (PDX) mouse models. Conditional knockout of attenuated and -driven onset
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DNA damage response defects in hematologic malignancies: mechanistic insights and therapeutic strategies Blood (IF 20.3) Pub Date : 2024-03-12 Marwan Kwok, Angelo Agathanggelou, Tatjana Stankovic
The DNA damage response (DDR) encompasses the detection and repair of DNA lesions and is fundamental to the maintenance of genome integrity. Germline DDR alterations underlie hereditary chromosome instability syndromes by promoting the acquisition of pathogenic structural variants in hematopoietic cells, resulting in increased predisposition to hematologic malignancies. Also frequent in hematologic
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Nilotinib with or without cytarabine for Philadelphia positive acute lymphoblastic leukemia. Blood (IF 20.3) Pub Date : 2024-03-12 Yves Chalandon, Philippe Rousselot, Sylvie Chevret, Jean-Michel Cayuela, Rathana Kim, Françoise Huguet, Patrice Chevallier, Carlos Graux, Anne Thiebaut-Bertrand, Sylvain Chantepie, Xavier Thomas, Laure Vincent, Céline Berthon, Yosr Hicheri, Emmanuel Raffoux, Martine Escoffre-Barbe, Isabelle Plantier, Magalie Joris, Pascal Turlure, Florence Pasquier, Amine Belhabri, Gabrielle Roth Guepin, Sabine Blum
We previously demonstrated that a reduced-intensity chemotherapy schedule can safely replace Hyper-CVAD cycle 1 when combined with imatinib in adults with Philadelphia-positive (Ph+) acute lymphoblastic leukemia (ALL). In the present randomized GRAAPH-2014 trial, we used nilotinib and addressed the omission of cytarabine (Ara-C) in consolidation. The primary objective was the major molecular response
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Epigenetic control over cell-intrinsic immune response antagonizes self-renewal in acute myeloid leukemia Blood (IF 20.3) Pub Date : 2024-03-11 Eloísa Felipe Fumero, Carolin Walter, Joris Maximillian Frenz, Franca Seifert, Vijay Alla, Thorben Henning, Linus Angenendt, Wolfgang Hartmann, Sebastian Wolf, Hubert Serve, Thomas Oellerich, Georg Lenz, Carsten Müller-Tidow, Christoph Schliemann, Otmar Huber, Martin Dugas, Matthias Mann, Ashok Kumar Jayavelu, Jan-Henrik Mikesch, Maria Francisca Arteaga
The histone demethylase PHF8 is a master regulator of cell-intrinsic immune response in acute myeloid leukemia.
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T cell help in the tumor microenvironment enhances rituximab-mediated NK cell ADCC Blood (IF 20.3) Pub Date : 2024-03-11 Jyoti Arora, Sabarish Ayyappan, Chaobo Yin, Brian J. Smith, Caitlin D. Lemke-Miltner, Zhaoming Wang, Umar Farooq, George J. Weiner
Rituximab (RTX) and other monoclonal antibodies (mAbs) that bind directly to malignant cells are of great clinical value but are not effective for all patients. A major mechanism of action of RTX is antibody dependent cellular cytotoxicity (ADCC) mediated by NK cells. Prior in vitro studies in our laboratory demonstrated that T cells contribute to maintaining the viability and cytotoxic potential of
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IDH Mutant Myeloid Neoplasms are Associated with Seronegative Rheumatoid Arthritis and Innate Immune Activation Blood (IF 20.3) Pub Date : 2024-03-11 Lih En Hong, Mihir D Wechalekar, Monika Kutyna, Annabelle Small, Kelly Lim, Chloe Thompson-Peach, Joule J Li, Rakchha Chhetri, Hamish S Scott, Anna Brown, Christopher N Hahn, David T Yeung, Salvia Sajid, Nirmal Robinson, Ranjeny Thomas, Susan Branford, Richard J D’Andrea, Saumya E Samaraweera, Mrinal Patnaik, Susanna Proudman, Daniel Thomas, Chung Hoow Kok, Mithun V Shah, Devendra K Hiwase
High prevalence of -mutations in seronegative rheumatoid arthritis (RA) with myeloid neoplasm, elevated 2-hydroxyglutarate, dysregulated innate immunity and pro-inflammatory microenvironment, suggests causative association between -mutations and seronegative RA. Our findings merit investigation of IDH-inhibitors as therapeutics for seronegative IDH-mutated RA.
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Hemoglobin Bart’s Hydrops Fetalis: Charting the Past and Envisioning the Future Blood (IF 20.3) Pub Date : 2024-03-11 Ali Amid, Siyu Liu, Christian Babbs, Douglas R. Higgs
Hemoglobin Bart’s hydrops fetalis syndrome (BHFS) represents the most severe form of α-thalassemia, arising from deletion of the duplicated α-globin genes from both alleles. The absence of α-globin leads to the formation of non-functional hemoglobin Bart’s (γ) or hemoglobin H (HbH: β) resulting in severe anemia, tissue hypoxia, and, in some cases, variable congenital or neurocognitive abnormalities
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Fitusiran prophylaxis in people with hemophilia A or B who switched from prior BPA/CFC prophylaxis (ATLAS-PPX) Blood (IF 20.3) Pub Date : 2024-03-11 Gili Kenet, Beatrice Nolan, Bulent Zulfikar, Bulent Antmen, Peter Kampmann, Tadashi Matsushita, Chur-Woo You, Kateryna Vilchevska, Catherine N. Bagot, Azizan Sharif, Flora Peyvandi, Guy Young, Claude Negrier, Jiarui Chi, Barbara Kittner, Christian Sussebach, Fadi Shammas, Baisong Mei, Shauna Andersson, Kaan Kavakli.
Fitusiran, a subcutaneous (SC) investigational siRNA therapeutic, targets antithrombin to rebalance hemostasis in people with hemophilia A or B (PwHA/B), irrespective of inhibitor status. This Phase 3, open-label study (NCT03549871) evaluated the efficacy and safety of fitusiran prophylaxis in males aged ≥ 12 years with hemophilia A or B, with or without inhibitors, who received prior bypassing agent
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Vector integration and fate in the hemophilia dog liver multi-years following AAV-FVIII gene transfer Blood (IF 20.3) Pub Date : 2024-03-11 Paul Batty, Sylvia Fong, Matteo Franco, Choong-Ryoul Sihn, Laura L. Swystun, Saira Afzal, Lorianne Harpell, David Hurlbut, Abbey Pender, Cheng Su, Hauke Thomsen, Christopher Wilson, Loubna Youssar, Andrew Winterborn, Irene Gil-Farina, David Lillicrap
Gene therapy using adeno-associated viral (AAV) vectors is a promising approach for the treatment of monogenic disorders. Long-term multi-year transgene expression has been demonstrated in animal models and clinical studies. Nevertheless, uncertainties remain concerning the nature of AAV vector persistence and whether there is a potential for genotoxicity. Here, we describe the mechanisms of AAV vector
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Thrombin receptor activating peptide-6 decreases acute graft-versus-host disease through activating GPR15 Leukemia (IF 11.4) Pub Date : 2024-03-08 Cong Liu, Qiu Lan, Shuo Cao, Fei Zheng, Yiwen Liang, Jingyi Shen, Ying Wang, Takayuki Ikezoe, Kailin Xu, Bin Pan
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Myelodysplastic neoplasms dissected into indolent, leukaemic and unfavourable subtypes by computational clustering of haematopoietic stem and progenitor cells Leukemia (IF 11.4) Pub Date : 2024-03-08 Margot F. van Spronsen, Sofie Van Gassen, Carolien Duetz, Theresia M. Westers, Yvan Saeys, Arjan A. van de Loosdrecht
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The latest insights into rare blood disorders: Diagnosis and treatment strategies Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-09 David J. Kuter, Spero R. Cataland, Catherine M. Broome, Cindy Neunert
Because immune‐mediated rare blood disorders are uncommon, healthcare providers often lack the knowledge and experience necessary to identify, diagnose, and treat them in accordance with best practices. As a result, there are significant gaps in care, including delays in diagnosis and suboptimal treatment. To ensure that more patients with these rare disorders are offered quality, evidence‐based care
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Targeting the innate immune system in pediatric and adult AML Leukemia (IF 11.4) Pub Date : 2024-03-08 Alicia Perzolli, Joost B. Koedijk, C. Michel Zwaan, Olaf Heidenreich
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Axicabtagene ciloleucel treatment is more effective in primary mediastinal large B-cell lymphomas than in diffuse large B-cell lymphomas: the Italian CART-SIE study Leukemia (IF 11.4) Pub Date : 2024-03-08 Annalisa Chiappella, Beatrice Casadei, Patrizia Chiusolo, Alice Di Rocco, Silva Ljevar, Martina Magni, Piera Angelillo, Anna Maria Barbui, Ilaria Cutini, Anna Dodero, Francesca Bonifazi, Maria Chiara Tisi, Stefania Bramanti, Maurizio Musso, Mirko Farina, Massimo Martino, Mattia Novo, Giovanni Grillo, Francesca Patriarca, Giulia Zacchi, Mauro Krampera, Martina Pennisi, Eugenio Galli, Maurizio Martelli
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The dual HCK/BTK inhibitor KIN-8194 impairs growth and integrin-mediated adhesion of BTKi-resistant mantle cell lymphoma Leukemia (IF 11.4) Pub Date : 2024-03-07 Hildo C. Lantermans, Fangxue Ma, Annemieke Kuil, Sanne van Kesteren, Sevtap Yasinoglu, Guang Yang, Sara J. Buhrlage, Jinhua Wang, Nathanael S. Gray, Marie José Kersten, Steven P. Treon, Steven T. Pals, Marcel Spaargaren
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Functional characterization of cooperating MGA mutations in RUNX1::RUNX1T1 acute myeloid leukemia Leukemia (IF 11.4) Pub Date : 2024-03-07 Melvin E. Thomas, Wenqing Qi, Michael P. Walsh, Jing Ma, Tamara Westover, Sherif Abdelhamed, Lauren J. Ezzell, Chandra Rolle, Emily Xiong, Wojciech Rosikiewicz, Beisi Xu, Allister J. Loughran, Shondra M. Pruett-Miller, Laura J. Janke, Jeffery M. Klco
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Reducing and controlling metabolic active tumor volume prior to CAR T-cell infusion can improve survival outcomes in patients with large B-cell lymphoma Blood Cancer J. (IF 12.8) Pub Date : 2024-03-07 Kylie Keijzer, Janneke W. de Boer, Jaap A. van Doesum, Walter Noordzij, Gerwin A. Huls, Lisanne V. van Dijk, Tom van Meerten, Anne G. H. Niezink
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Clinical outcomes of patients with lymphoid blastic phase of chronic myeloid leukemia treated with CAR T-cell therapy Blood Cancer J. (IF 12.8) Pub Date : 2024-03-06 Yujie Liu, Yuqing Tu, Jinyan Xiao, Yifan Shen, Biqi Zhou, Qiannan Yang, Lei Yu, Lijuan Qi, Jia Chen, Tianhui Liu, Depei Wu, Yang Xu
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Enhancing prognostic power in multiple myeloma using a plasma cell signature derived from single-cell RNA sequencing Blood Cancer J. (IF 12.8) Pub Date : 2024-03-06 Jian-rong Li, Shahram Arsang-Jang, Yan Cheng, Fumou Sun, Anita D’Souza, Binod Dhakal, Parameswaran Hari, Quillan Huang, Paul Auer, Yong Li, Raul Urrutia, Fenghuang Zhan, John D. Shaughnessy, Siegfried Janz, Jing Dong, Chao Cheng