-
How complete must an AML remission be? Blood (IF 21.0) Pub Date : 2024-07-11 Christopher S. Hourigan
-
-
-
In cancer, not all platelets are created equal Blood (IF 21.0) Pub Date : 2024-07-11 Elisabeth M. Battinelli
-
-
Paving the way to factor X deficiency therapies Blood (IF 21.0) Pub Date : 2024-07-11 Jeremy P. Wood
-
-
Chronic eosinophilic leukemia with a novel JAK1 mutation responds well to the JAK1/2 inhibitor ruxolitinib Blood (IF 21.0) Pub Date : 2024-07-11 Qing Wei, Jie Xu
-
Mantle cell lymphoma with IRTA1+ reactive expansion of the marginal zone Blood (IF 21.0) Pub Date : 2024-07-11 Brunangelo Falini, Stefano Lazzi
-
Eltrombopag plus diacerein vs eltrombopag in patients with ITP: a multicenter, randomized, open-label phase 2 trial Blood (IF 21.0) Pub Date : 2024-07-06 Lu Sun, Xiaoyang Huang, Juan Wang, Chenglu Yuan, Hongyu Zhao, Daqi Li, Ruirong Xu, Yan Wang, Ping Qin, Yan Shi, Jun Peng, Ming Hou, Yu Hou
This study aimed to compare the efficacy and safety of eltrombopag plus diacerein vs eltrombopag alone in patients with primary immune thrombocytopenia (ITP) who were previously unresponsive to 14 days of eltrombopag treatment at the full dose. Recruited patients were randomly assigned 1:1 to receive either eltrombopag plus diacerein (n = 50) or eltrombopag monotherapy (n = 52). Overall response rate
-
Momelotinib for the treatment of myelofibrosis Blood (IF 21.0) Pub Date : 2024-07-05 Prithviraj Bose
In September 2023, the US Food and Drug Administration approved momelotinib for the treatment of myelofibrosis (MF) with anemia, marking the fourth US regulatory approval of a Janus kinase inhibitor for MF. A positive opinion from the European Medicines Agency followed in November 2023. Momelotinib’s ability to address splenomegaly, symptoms, and anemia, including in patients with thrombocytopenia
-
-
Platelet TGF-β triggers immunosuppression in ITP Blood (IF 21.0) Pub Date : 2024-07-04 Rick Kapur, John W. Semple
-
Polycythemia revealing PIEZO1 hereditary xerocytosis Blood (IF 21.0) Pub Date : 2024-07-04 Stephanie Cordeil, Laurent Jallades
-
Transferrin: the iron transporter takes control Blood (IF 21.0) Pub Date : 2024-07-04 Thomas Weichhart
-
-
GADD45A: a key tumor suppressor in AML subtypes Blood (IF 21.0) Pub Date : 2024-07-04 Zhijian Qian, Fang Yu
-
When InO says no: understanding escape Blood (IF 21.0) Pub Date : 2024-07-04 Judith M. Boer, C. Michel Zwaan
-
A 1-year-old child with essential thrombocythemia Blood (IF 21.0) Pub Date : 2024-07-04 Ian A. Gelarden, Aida I. Richardson
-
Optimizing drug combinations for T-PLL: restoring DNA damage and P53-mediated apoptotic responses Blood (IF 21.0) Pub Date : 2024-07-01 Jana von Jan, Sanna Timonen, Till Braun, Qu Jiang, Aleksandr Ianevski, Yayi Peng, Kathleen McConnell, Paola Sindaco, Tony Andreas Müller, Sabine Pützer, Hanna Klepzig, Dennis Jungherz, Annika Dechow, Linus Wahnschaffe, Anil K Giri, Matti Kankainen, Heikki Kuusanmäki, Heidi A. Neubauer, Richard Moriggl, Paolo Mazzeo, Nicole Schmidt, Raphael Koch, Michael Hallek, Amel Chebel, David Armisen, Laurent Genestier
T-prolymphocytic leukemia (T-PLL) is a mature T-cell neoplasm associated with marked chemotherapy resistance and continued poor clinical outcomes. Current treatments, that is, the CD52-antibody alemtuzumab, offer transient responses, with relapses being almost inevitable without consolidating allogeneic transplantation. Recent more detailed concepts of T-PLL’s pathobiology fostered the identification
-
IL-9 secreted by leukemia stem cells induces Th1-skewed CD4+ T cells, which promote their expansion Blood (IF 21.0) Pub Date : 2024-06-29 Ramin Radpour, Cedric Simillion, Bofei Wang, Hussein A. Abbas, Carsten Riether, Adrian F. Ochsenbein
In acute myeloid leukemia (AML), leukemia stem cells (LSCs) and leukemia progenitor cells (LPCs) interact with various cell types in the bone marrow (BM) microenvironment, regulating their expansion and differentiation. To study the interaction of CD4 and CD8 T cells in the BM with LSCs and LPCs, we analyzed their transcriptome and predicted cell-cell interactions by unbiased high-throughput correlation
-
ATM germ line pathogenic variants affect outcomes in children with ataxia-telangiectasia and hematological malignancies Blood (IF 21.0) Pub Date : 2024-06-27 Sarah Elitzur, Ruth Shiloh, Jan L. C. Loeffen, Agata Pastorczak, Masatoshi Takagi, Simon Bomken, Andre Baruchel, Thomas Lehrnbecher, Sarah K. Tasian, Oussama Abla, Nira Arad-Cohen, Itziar Astigarraga, Miriam Ben-Harosh, Nicole Bodmer, Triantafyllia Brozou, Francesco Ceppi, Liliia Chugaeva, Luciano Dalla Pozza, Stephane Ducassou, Gabriele Escherich, Roula Farah, Amber Gibson, Henrik Hasle, Julieta Hoveyan
Ataxia telangiectasia (AT) is an autosomal-recessive disorder caused by pathogenic variants (PVs) of the gene. Children with AT are predisposed to hematological malignancies. We aimed to investigate their characteristics and outcomes in order to generate data-based treatment recommendations. In this multinational, observational study we report 202 patients aged ≤25 years with AT and hematological malignancies
-
How I treat infant acute lymphoblastic leukemia Blood (IF 21.0) Pub Date : 2024-06-25 Jack Bartram, Phil Ancliff, Ajay Vora
Infant acute lymphoblastic leukemia (ALL) is an aggressive malignancy that has historically been associated with a very poor prognosis. Despite large cooperative international trials and incremental increases in intensity of therapy, there has been no significant improvement in outcome over the last 3 decades. Using representative cases, we highlight the key differences between -rearranged and –germ
-
Quadriparesis and paraparesis following chimeric antigen receptor T-cell therapy in children and adolescents Blood (IF 21.0) Pub Date : 2024-06-25 Caroline Diorio, Laura Hernandez-Miyares, Diego A. Espinoza, Brenda L. Banwell, Amit Bar-Or, Amanda M. DiNofia, Allison Barz Leahy, Zachary Martinez, Regina M. Myers, Sarah E. Hopkins, Susan R. Rheingold, David T. Teachey, Angela N. Viaene, Lisa M. Wray, Shannon L. Maude, Stephan A. Grupp, Jennifer L. McGuire
Immune effector cell–associated neurotoxicity syndrome (ICANS) is a common but potentially severe adverse event associated with chimeric antigen receptor T-cell (CART) therapy, characterized by the development of acute neurologic symptoms following CART infusion. ICANS encompasses a wide clinical spectrum typified by mild to severe encephalopathy, seizures, and/or cerebral edema. As more patients have
-
Preclinical efficacy of potent and selective menin-KMT2A inhibitor JNJ-75276617 in KMT2A- and NPM1-altered leukemias Blood (IF 21.0) Pub Date : 2024-06-25 Min Chul Kwon, Jan Willem Thuring, Olivier Querolle, Xuedong Dai, Tinne Verhulst, Vineet Pande, Ann Marien, Dries Goffin, Daniela V. Wenge, Hong Yue, Jevon A. Cutler, Cyrus Jin, Florian Perner, Shanna M. Hogeling, Paul L. Shaffer, Frank Jacobs, Petra Vinken, Wei Cai, Vikki Keersmaekers, Filmon Eyassu, Balpreet Bhogal, Karin Verstraeten, Sara El Ashkar, Jennifer A. Perry, Prathiba Jayaguru, Laura Barreyro
The interaction between menin and histone-lysine N-methyltransferase 2A () is a critical dependency for - or nucleophosmin 1 ()–altered leukemias and an emerging opportunity for therapeutic development. JNJ-75276617 is a novel, orally bioavailable, potent, and selective protein-protein interaction inhibitor of the binding between menin and KMT2A. In -rearranged (-r) and -mutant (c) acute myeloid leukemia
-
Emerging genetic technologies informing personalized medicine in SDS and other inherited bone marrow failure disorders Blood (IF 21.0) Pub Date : 2024-06-24 Alyssa H. Cull, David G. Kent, Alan J. Warren
Ribosomopathy Shwachman-Diamond syndrome (SDS) is a rare autosomal recessive inherited bone marrow failure syndrome (IBMFS) caused by mutations in the Shwachman-Bodian-Diamond syndrome gene, which is associated with an increased risk of myeloid malignancy. Tracking how hematopoietic stem cell (HSC) clonal dynamics change over time, assessing whether somatic genetic rescue mechanisms affect these dynamics
-
Genotype-phenotype correlations in chronic granulomatous disease: insights from a large national cohort Blood (IF 21.0) Pub Date : 2024-06-24 Baruch Wolach, Ronit Gavrieli, Ofir Wolach, Pazit Salamon, Martin de Boer, Karin van Leeuwen, Omar Abuzaitoun, Arnon Broides, Giora Gottesman, Galia Grisaru-Soen, David Hagin, Nufar Marcus, Menachem Rottem, Yechiel Schlesinger, Tali Stauber, Polina Stepensky, Yael Dinur-Schejter, Tal Zeeli, Suheir Hanna, Amos Etzioni, Shirly Frizinsky, Raz Somech, Dirk Roos, Idit Lachover Roth
Neutrophils are the first line of defense against invading pathogens. Neutrophils execute and modulate immune responses by generating reactive oxygen species (ROS). Chronic granulomatous disease (CGD) is a primary immune deficiency disorder of phagocytes, caused by inherited mutations in the genes of the nicotinamide adenine dinucleotide phosphate reduced oxidase enzyme. These mutations lead to failure
-
GVHD targets organoid-forming bile duct stem cells in a TGF-β–dependent manner Blood (IF 21.0) Pub Date : 2024-06-24 Yuta Hasegawa, Daigo Hashimoto, Zixuan Zhang, Toru Miyajima, Yumika Saito, Wenyu Li, Ryo Kikuchi, Hajime Senjo, Tomoko Sekiguchi, Takahiro Tateno, Xuanzhong Chen, Emi Yokoyama, Shuichiro Takahashi, Hiroyuki Ohigashi, Takahide Ara, Eiko Hayase, Isao Yokota, Takanori Teshima
Graft-versus-host disease (GVHD) is a major life-threatening complication that occurs after allogeneic hematopoietic cell transplantation (HCT). Although adult tissue stem cells have been identified as targets of GVHD in the skin and gut, their role in hepatic GVHD is yet to be clarified. In the current study, we explored the fate of bile duct stem cells (BDSCs), capable of generating liver organoids
-
Murine models of erythroid 5ALA synthesis disorders and their conditional synthetic lethal dependency on pyridoxine Blood (IF 21.0) Pub Date : 2024-06-24 Sarah Ducamp, Anoop K. Sendamarai, Dean R. Campagna, Wai Loon Desmond Chin, Yuko Fujiwara, Paul J. Schmidt, Mark D. Fleming
X-linked sideroblastic anemia (XLSA) and X-linked protoporphyria (XLPP) are uncommon diseases caused by loss-of-function and gain-of-function mutations, respectively, in the erythroid form of 5-aminolevulinic acid synthetase (ALAS), ALAS2, which encodes the first enzyme in heme biosynthesis. A related sideroblastic anemia is due to mutations in SLC25A38 (solute carrier family 25 member A38), which
-
NK cells with adhesion defects and reduced cytotoxic functions are associated with a poor prognosis in multiple myeloma Blood (IF 21.0) Pub Date : 2024-06-19 Eve Blanquart, Rüçhan Ekren, Bineta Rigaud, Marie-Véronique Joubert, Virginie Baylot, Hélène Daunes, Marine Cuisinier, Marine Villard, Nadège Carrié, Céline Mazzotti, Liliana E. Lucca, Aurore Perrot, Jill Corre, Thierry Walzer, Hervé Avet-Loiseau, Pierre-Paul Axisa, Ludovic Martinet
The promising results obtained with immunotherapeutic approaches for multiple myeloma (MM) call for a better stratification of patients based on immune components. The most pressing being cytotoxic lymphocytes such as natural killer (NK) cells that are mandatory for MM surveillance and therapy. Here, we performed a single-cell RNA sequencing analysis of NK cells from 10 patients with MM and 10 age/sex–matched
-
Early-life infection depletes preleukemic cells in a mouse model of hyperdiploid B-cell acute lymphoblastic leukemia Blood (IF 21.0) Pub Date : 2024-06-18 Ali Farrokhi, Tanmaya Atre, Samuel Salitra, Maryam Aletaha, Ana Citlali Márquez, Matthew Gynn, Mario Fidanza, Sumin Jo, Nina Rolf, Karen Simmons, Jesus Duque-Afonso, Michael L. Cleary, Alix E. Seif, Tobias Kollmann, Soren Gantt, Gregor S. D. Reid
Epidemiological studies report opposing influences of infection on childhood B-cell acute lymphoblastic leukemia (B-ALL). Although infections in the first year of life appear to exert the largest impact on leukemia risk, the effect of early pathogen exposure on the fetal preleukemia cells (PLC) that lead to B-ALL has yet to be reported. Using cytomegalovirus infection as a model early-life infection
-
How I treat bleeding in hereditary hemorrhagic telangiectasia Blood (IF 21.0) Pub Date : 2024-06-14 Hanny Al-Samkari
Hereditary hemorrhagic telangiectasia (HHT; Osler-Weber-Rendu disease) affects 1 in 5000 persons, making it the second most common inherited bleeding disorder worldwide. Telangiectatic bleeding, primarily causing recurrent epistaxis and chronic gastrointestinal bleeding, is the most common and most important manifestation of this multisystem vascular disorder. HHT-associated bleeding results in substantial
-
How I diagnose and treat organizing pneumonia in hematopoietic cell transplant recipients Blood (IF 21.0) Pub Date : 2024-06-13 Yu Kuang Lai, Husham Sharifi, Joe L. Hsu
Organizing pneumonia (OP) is a known noninfectious pulmonary complication following allogeneic hematopoietic cell transplant (HCT) and represents a significant risk factor for nonrelapse mortality in HCT recipients. Unlike bronchiolitis obliterans syndrome, it is not universally acknowledged as a distinctive pulmonary manifestation of chronic graft-versus-host disease (cGVHD) and, therefore, its diagnostic
-
Fixed-duration pirtobrutinib plus venetoclax with or without rituximab in relapsed/refractory CLL: the phase 1b BRUIN trial Blood (IF 21.0) Pub Date : 2024-06-13 Lindsey E. Roeker, Jennifer A. Woyach, Chan Y. Cheah, Catherine C. Coombs, Nirav N. Shah, William G. Wierda, Manish R. Patel, Nicole Lamanna, Donald E. Tsai, Binoj Nair, Chunxiao Wang, Xiang Zhao, Dan Liu, David Radtke, Sonya Chapman, Narasimha Marella, Samuel C. McNeely, Jennifer R. Brown
Pirtobrutinib is a highly selective, noncovalent (reversible) Bruton tyrosine kinase inhibitor (BTKi). Patients with relapsed or refractory (R/R) chronic lymphocytic leukemia (CLL) were treated with fixed-duration pirtobrutinib plus venetoclax (PV) or pirtobrutinib plus venetoclax and rituximab (PVR) in this phase 1b trial. Prior covalent BTKi therapy was allowed, but not prior treatment with venetoclax
-
In vivo CAR T-cell generation in nonhuman primates using lentiviral vectors displaying a multidomain fusion ligand Blood (IF 21.0) Pub Date : 2024-06-13 Christopher J. Nicolai, Maura Parker, Jim Qin, Weiliang Tang, Justin Ulrich-Lewis, Rebecca J. Gottschalk, Sara Cooper, Susana Hernandez, Don Parrilla, Richard Mangio, Nolan Ericson, Alissa Brandes, Saluwa Umuhoza, Kathryn Michels, Mollie McDonnell, Lisa Park, Seungjin Shin, Wai-Hang Leung, Andrew M. Scharenberg, Hans-Peter Kiem, Ryan P. Larson, Laurie O. Beitz, Byoung Y. Ryu
Chimeric antigen receptor (CAR) T-cell therapies have demonstrated transformative efficacy in treating B-cell malignancies. However, high costs and manufacturing complexities hinder their widespread use. To overcome these hurdles, we have developed the VivoVec platform, a lentiviral vector capable of generating CAR T cells in vivo. Here, we describe the incorporation of T-cell activation and costimulatory
-
Targeting PKCα alleviates iron overload in diabetes and hemochromatosis through the inhibition of ferroportin Blood (IF 21.0) Pub Date : 2024-06-13 Somesh Banerjee, Shaolei Lu, Anand Jain, Irene Wang, Hui Tao, Shanthi Srinivasan, Elizabeta Nemeth, Peijian He
Ferroportin (Fpn) is the only iron exporter, playing a crucial role in systemic iron homeostasis. Fpn is negatively regulated by its ligand hepcidin, but other potential regulators in physiological and disease conditions remain poorly understood. Diabetes is a metabolic disorder that develops body iron loading with unknown mechanisms. By using diabetic mouse models and human duodenal specimens, we
-
Updated cutaneous T-cell lymphoma TNMB staging criteria fail to identify patients with Sézary syndrome with low blood burden Blood (IF 21.0) Pub Date : 2024-06-11 Lauren P. Chrisman, Payton Fors Trimark, Yanzhen Pang, David Randall Pease, Maria Estela Martinez-Escala, William Q. Nguyen, Rony Fernandez, Teresa L. Griffin, Lindsey Ayanruoh, Madeline J. Hooper, Xiaolong A. Zhou, Lucy Fu, Kristy L. Wolniak, Joan Guitart
Comparison of the 2007 EORTC/ISCL and the 2022 EORTC/ISCL/USCLC blood staging guidelines for cutaneous T-cell lymphoma at a single institution reveals the newer guidelines fail to detect a subset of patients with Sézary syndrome with low blood burden.
-
How I treat patients with low-risk polycythemia vera who require cytoreduction Blood (IF 21.0) Pub Date : 2024-06-11 Mary Frances McMullin, Claire N. Harrison
Polycythemia vera (PV) was first described by Louis Henri Vaquez in 1892. This is a chronic hematological malignancy that affects both older and young patients. Perhaps because of lack of a curative treatment and the perceived toxicities of prior therapies, our focus in the past was to intensify treatment only for patients at higher risk of thrombosis. Recent triggers to challenge this approach include
-
How I manage major hemorrhage Blood (IF 21.0) Pub Date : 2024-06-11 Jeannie L. Callum, Ronald B. George, Keyvan Karkouti
Acute hemorrhage can be a life-threatening emergency that is complex in its management and affects many patient populations. The past 15 years has seen the introduction of comprehensive massive hemorrhage protocols, wider use of viscoelastic testing, new coagulation factor products, and the publication of robust randomized controlled trials in diverse bleeding patient populations. Although gaps continue
-
NGS-based stratification refines the risk stratification in T-ALL and identifies a very-high-risk subgroup of patients Blood (IF 21.0) Pub Date : 2024-06-11 Mathieu Simonin, Loïc Vasseur, Etienne Lengliné, Ludovic Lhermitte, Aurélie Cabannes-Hamy, Marie Balsat, Aline Schmidt, Marie-Emilie Dourthe, Aurore Touzart, Carlos Graux, Nathalie Grardel, Jean-Michel Cayuela, Isabelle Arnoux, Virginie Gandemer, Françoise Huguet, Stéphane Ducassou, Véronique Lhéritier, Yves Chalandon, Norbert Ifrah, Hervé Dombret, Elizabeth Macintyre, Arnaud Petit, Philippe Rousselot
We previously reported a better outcome in adult and pediatric T-cell acute lymphoblastic leukemia (T-ALL) harboring and/or mutations without alterations of and genes. Availability of high-throughput next-generation sequencing (NGS) strategies led us to refine the outcome prediction in T-ALL. Targeted whole-exome sequencing of 72 T-ALL–related oncogenes was performed in 198 adults with T-ALLs in first
-
A moonlighting job for α-globin in blood vessels Blood (IF 21.0) Pub Date : 2024-06-11 Prabhodh S. Abbineni, Srishti Baid, Mitchell J. Weiss
Red blood cells express high levels of hemoglobin A tetramer (α2β2) to facilitate oxygen transport. Hemoglobin and related proteins are also expressed at lower levels in other tissues across the animal kingdom. Physiological functions for most nonerythroid globins likely derive from their ability to catalyze reduction–oxidation (redox) reactions via electron transfer through heme-associated iron. An
-
Foxo1 is an iron-responsive transcriptional factor regulating systemic iron homeostasis Blood (IF 21.0) Pub Date : 2024-06-11 Teng Xu, Xu Zhang, Wenting Zhao, Jiaxin Shi, Sitong Wan, Yan Zhang, Yanling Hao, Mingyue Sun, Jingjing He, Li Jiang, Hao Wang, Hong Gao, Junjie Luo, Yongting Luo, Peng An
The liver plays a crucial role in maintaining systemic iron homeostasis by secreting hepcidin, which is essential for coordinating iron levels in the body. Imbalances in iron homeostasis are associated with various clinical disorders related to iron deficiency or iron overload. Despite the clinical significance, the mechanisms underlying how hepatocytes sense extracellular iron levels to regulate hepcidin
-
Asciminib is a novel inhibitor of ABL1 and ABL2 gene fusions in ALL but requires the ABL SH3 domain for efficacy Blood (IF 21.0) Pub Date : 2024-06-10 Laura N. Eadie, Elias Lagonik, Elyse C. Page, Caitlin E. Schutz, Susan L. Heatley, Barbara J. McClure, Michelle O. Forgione, David T. Yeung, Timothy P. Hughes, Deborah L. White
-
Management of immune thrombotic thrombocytopenic purpura without therapeutic plasma exchange Blood (IF 21.0) Pub Date : 2024-06-10 Lucas Kühne, Paul Knöbl, Kathrin Eller, Johannes Thaler, Wolfgang R. Sperr, Karoline Gleixner, Thomas Osterholt, Jessica Kaufeld, Jan Menne, Veronika Buxhofer-Ausch, Anja Mühlfeld, Evelyn Seelow, Adrian Schreiber, Polina Todorova, Sadrija Cukoski, Wolfram J. Jabs, Fedai Özcan, Anja Gäckler, Kristina Schönfelder, Felix S. Seibert, Timm Westhoff, Vedat Schwenger, Dennis A. Eichenauer, Linus A. Völker
Immune thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening autoimmune disorder caused by ADAMTS13 deficiency. Caplacizumab, an anti–von Willebrand factor nanobody, is approved for iTTP treatment, reducing the need for therapeutic plasma exchange (TPE) and improving platelet count recovery and survival. We conducted a retrospective study on 42 acute iTTP cases in Austria and Germany
-
A modern view of LGL leukemia Blood (IF 21.0) Pub Date : 2024-06-10 Tony Marchand, Thierry Lamy, Thomas P. Loughran Jr.
Large granular lymphocytic (LGL) leukemia is a rare lymphoproliferative chronic disorder characterized by expansion of either T- or natural killer (NK)-cytotoxic cells. Contrary to Epstein-Barr virus–induced aggressive NK-LGL leukemia, chronic T- and NK-LGL leukemia are indolent diseases affecting elderly patients with a median age of 66.5 years. LGL leukemia is frequently associated with autoimmune
-
Fostamatinib effectiveness and safety for immune thrombocytopenia in clinical practice Blood (IF 21.0) Pub Date : 2024-06-10 Tomás José González-López, Nuria Bermejo-Vega, Rocío Cardesa-Cabrera, Violeta Martínez-Robles, Gerardo Aguilar-Monserrate, Gloria Pérez-Segura, Abel Domingo, Josefa Luis-Navarro, Sunil Lakhwani, Natalia Acedo, María Luisa Lozano, Silvia Bernat, Ana Torres-Tienza, Ana Ruano, Isidro Jarque, Pilar Galán, Carmen Benet, Shally Marcellini, Reyes Jimenez-Bárcenas, Daniel Martínez-Carballeira, Dunia De Miguel-Llorente
Fostamatinib, a recently approved Syk inhibitor used in adult primary immune thrombocytopenia (ITP), has been shown to be safe and effective in this disorder. However, clinical trial results may not be similarly reproduced in clinical practice. Here, 138 patients with ITP (both primary and secondary) from 42 Spanish centers who had been treated with fostamatinib were evaluated prospectively and retrospectively
-
Randomized induction with bendamustine-rituximab plus bortezomib and maintenance with rituximab plus lenalidomide for MCL Blood (IF 21.0) Pub Date : 2024-06-04 Mitchell R. Smith, Opeyemi A. Jegede, Peter Martin, Brian G. Till, Samir S. Parekh, David T. Yang, Eric D. Hsi, Thomas Witzig, Sandeep Dave, David Scott, Curtis Hanson, Lale Kostakoglu Shields, Nizar Abdel-Samad, Carla Casulo, Nancy L. Bartlett, Paolo F. Caimi, Tareq Al Baghdadi, Kristie A. Blum, Mark D. Romer, David J. Inwards, Rachel E. Lerner, Lynne I. Wagner, Richard F. Little, Jonathan W. Friedberg
Although initial therapy of mantle cell lymphoma (MCL) is not standardized, bendamustine-rituximab (BR) is commonly used in older patients. Rituximab (R) maintenance after induction is often used. Thus, the open-label, randomized phase 2 Eastern Cooperative Oncology Group and the American College of Radiology Imaging Network Cancer Research Group E1411 trial was designed to test 2 questions: (1) does
-
4D intravital imaging studies identify platelets as the predominant cellular procoagulant surface in a mouse hemostasis model Blood (IF 21.0) Pub Date : 2024-06-03 Abigail Ballard-Kordeliski, Robert H. Lee, Ellen C. O’Shaughnessy, Paul Y. Kim, Summer R. Jones, Rafal Pawlinski, Matthew J. Flick, David S. Paul, Nigel Mackman, David A. Adalsteinsson, Wolfgang Bergmeier
Interplay between platelets, coagulation factors, endothelial cells (ECs), and fibrinolytic factors is necessary for effective hemostatic plug formation. This study describes a 4-dimensional (4D) imaging platform to visualize and quantify hemostatic plug components in mice with high spatiotemporal resolution. Fibrin accumulation after laser-induced vascular injury was observed at the platelet plug–EC
-
Patients with a cardiac complete response in AL amyloidosis have survival rates similar to those of a matched general population Blood (IF 21.0) Pub Date : 2024-06-03 Eli Muchtar, Susan Geyer, Giampaolo Merlini, Morie A. Gertz
The survival of patients achieving a cardiac complete response in light chain amyloidosis, defined as N-terminal pro B-type natriuretic peptide ≤350 pg/mL or B-type natiuretic peptide ≤80 pg/mL, was similar to a matched general population with an estimated 5-year survival rate of 93% vs 95%, respectively.
-
Interleukin-1 mediated hyperinflammation in XIAP deficiency is associated with defective autophagy Blood (IF 21.0) Pub Date : 2024-06-03 Dilan Dissanayake, Ashkan Firouzabady, Mohammad Massumi, Guillermo A. de Paz Linares, Christian Marshall, Spencer A. Freeman, Ronald M. Laxer, Rae S. M. Yeung
Deficiency of X-linked inhibitor of apoptosis protein (XIAP) is a rare genetic condition that can present with recurrent episodes of hemophagocytic lymphohistiocytosis (HLH), though the exact mechanisms leading to this hyperinflammatory disorder are unclear. Understanding its biology is critical to developing targeted therapies for this potentially fatal disease. Here, we report on a novel multiexonic
-
Novel therapeutic approaches in thalassemias, sickle cell disease, and other red cell disorders Blood (IF 21.0) Pub Date : 2024-06-03 Valeria Maria Pinto, Filippo Mazzi, Lucia De Franceschi
In this last decade, a deeper understanding of the pathophysiology of hereditary red cell disorders and the development of novel classes of pharmacologic agents have provided novel therapeutic approaches to thalassemias, sickle cell disease (SCD), and other red cell disorders. Here, we analyze and discuss the novel therapeutic options according to their targets, taking into consideration the complex
-
Heme induced loss of renovascular endothelial protein C receptor promotes chronic kidney disease in sickle mice Blood (IF 21.0) Pub Date : 2024-06-03 Qiyang Chen, Rimi Hazra, Danielle Crosby, Diane Lenhart, Shane C. Lenhart, Paritosh Mondal, Yingze Zhang, Seyed M. Nouraie, Roderick J. Tan, Charles T. Esmon, L. Vijay Mohan Rao, Kang Kim, Samit Ghosh
Chronic kidney disease (CKD) is a major contributor to morbidity and mortality in sickle cell disease (SCD). Anemia, induced by chronic persistent hemolysis, is associated with the progressive deterioration of renal health, resulting in CKD. Moreover, patients with SCD experience acute kidney injury (AKI), a risk factor for CKD, often during vaso-occlusive crisis associated with acute intravascular
-
Adaptation to ex vivo culture reduces human hematopoietic stem cell activity independently of the cell cycle Blood (IF 21.0) Pub Date : 2024-05-31 Carys S. Johnson, Matthew Williams, Kendig Sham, Serena Belluschi, Wenjuan Ma, Xiaonan Wang, Winnie W. Y. Lau, Kerstin B. Kaufmann, Gabriela Krivdova, Emily F. Calderbank, Nicole Mende, Jessica McLeod, Giovanna Mantica, Juan Li, Charlotte Grey-Wilson, Michael Drakopoulos, Shaaezmeen Basheer, Shubhankar Sinha, Evangelia Diamanti, Christina Basford, Nicola K. Wilson, Steven J. Howe, John E. Dick, Berthold
Loss of long-term hematopoietic stem cell (LT-HSC) function ex vivo hampers the success of clinical protocols that rely on culture. However, the kinetics and mechanisms through which this occurs remain incompletely characterized. In this study, through time-resolved single-cell RNA sequencing, matched in vivo functional analysis, and the use of a reversible in vitro system of early G arrest, we defined
-
Clinical networking results in continuous improvement of the outcome of patients with acute promyelocytic leukemia Blood (IF 21.0) Pub Date : 2024-05-31 Luísa Corrêa de Araújo Koury, Haesook T. Kim, Maria Soledad Undurraga, Juan Ramon Navarro-Cabrera, Victor Salinas, Pablo Muxi, Raul A. M. Melo, Ana Beatriz Glória, Katia Pagnano, Elenaide C. Nunes, Rosane I. Bittencourt, Ninoska Rojas, Shirley Quintana, Ana Ayala-Lugo, Ana Carolina Oliver, Lorena Figueiredo-Pontes, Fabiola Traina, Frederico Moreira, Evandro M. Fagundes, Bruno K. L. Duarte, Analí Pamela
The introduction of all- retinoic acid combined with anthracyclines has significantly improved the outcomes for patients diagnosed with acute promyelocytic leukemia (APL), and this strategy remains the standard of care in countries in which arsenic trioxide is not affordable. However, data from national registries and real-world databases indicate that low- and middle-income countries (LMIC) still
-
CD20-bispecific antibodies improve response to CD19-CAR T cells in lymphoma in vitro and CLL in vivo models Blood (IF 21.0) Pub Date : 2024-05-29 Berit J. Brinkmann, Alessia Floerchinger, Christina Schniederjohann, Tobias Roider, Mariana Coelho, Norman Mack, Peter-Martin Bruch, Nora Liebers, Sarah Dötsch, Dirk H. Busch, Michael Schmitt, Frank Neumann, Philipp M. Roessner, Martina Seiffert, Sascha Dietrich
Relapse after anti-CD19 chimeric antigen receptor (CD19-CAR) occurs in a substantial proportion of patients with lymphoid malignancies. We assessed the potential benefits of co-administering CD20-targeting bispecific antibodies (CD20-BsAbs) with CD19-CAR T cells with the aim of enhancing immunotherapeutic efficacy. Addition of CD20-BsAbs to cocultures of CD19-CARs and primary samples of B-cell malignancies
-
IL-1 in aging and pathologies of hematopoietic stem cells Blood (IF 21.0) Pub Date : 2024-05-27 Francisco Caiado, Markus G. Manz
Defense-oriented inflammatory reactivity supports survival at younger age but might contribute to health impairments in modern, aging societies. The interleukin-1 (IL-1) cytokines are highly conserved and regulated, pleiotropic mediators of inflammation, essential to respond adequately to infection and tissue damage but also with potential host damaging effects when left unresolved. In this review
-
Phase 1 study of CAR-37 T cells in patients with relapsed or refractory CD37+ lymphoid malignancies Blood (IF 21.0) Pub Date : 2024-05-27 Matthew J. Frigault, Charlotte E. Graham, Trisha R. Berger, Julie Ritchey, Nora K. Horick, Areej El-Jawahri, Irene Scarfò, Andrea Schmidts, Nicholas J. Haradhvala, Marc Wehrli, Won-Ho Lee, Aiyana L. Parker, Hadley R. Wiggin, Amanda Bouffard, Aonkon Dey, Mark B. Leick, Katelin Katsis, Eva L. Elder, Maria A. Dolaher, Daniella T. Cook, Alena A. Chekmasova, Lu Huang, Sarah Nikiforow, Heather Daley, Jerome
We report a first-in-human clinical trial using chimeric antigen receptor (CAR) T cells targeting CD37, an antigen highly expressed in B- and T-cell malignancies. Five patients with relapsed or refractory CD37 lymphoid malignancies were enrolled and infused with autologous CAR-37 T cells. CAR-37 T cells expanded in the peripheral blood of all patients and, at peak, comprised >94% of the total lymphocytes
-
CD8+ T-cell differentiation and dysfunction inform treatment response in acute myeloid leukemia Blood (IF 21.0) Pub Date : 2024-05-24 Francesco Mazziotta, Luca Biavati, Joseph Rimando, Sergio Rutella, Nicholas Borcherding, Sonali Parbhoo, Rupkatha Mukhopadhyay, Hanna A. Knaus, Peter Valent, Hubert Hackl, Ivan M. Borrello, Bruce R. Blazar, Katerina Hatzi, Ivana Gojo, Leo Luznik
The interplay between T-cell states of differentiation, dysfunction, and treatment response in acute myeloid leukemia (AML) remains unclear. Here, we leveraged a multimodal approach encompassing high-dimensional flow cytometry and single-cell transcriptomics and found that early memory CD8 T cells are associated with therapy response and exhibit a bifurcation into 2 distinct terminal end states. One
-
Targeting the tumor microenvironment for treating double-refractory chronic lymphocytic leukemia Blood (IF 21.0) Pub Date : 2024-05-24 Richard I. Lewis, Alexander F. vom Stein, Michael Hallek
-
Rapid tumor DNA analysis of cerebrospinal fluid accelerates treatment of central nervous system lymphoma Blood (IF 21.0) Pub Date : 2024-05-24 Mihir Gupta, Joseph D. Bradley, Elie Massaad, Evan J. Burns, N. Zeke Georgantas, Garrett E. Maron, Julie M. Batten, Aidan Gallagher, Julia Thierauf, Naema Nayyar, Amanda Gordon, SooAe S. Jones, Michelle Pisapia, Ying Sun, Pamela S. Jones, Fred G. Barker II, William T. Curry, Rajiv Gupta, Javier M. Romero, Nancy Wang, Priscilla K. Brastianos, Maria Martinez-Lage, Kensuke Tateishi, Deborah A. Forst,
Delays and risks associated with neurosurgical biopsies preclude timely diagnosis and treatment of central nervous system (CNS) lymphoma and other CNS neoplasms. We prospectively integrated targeted rapid genotyping of cerebrospinal fluid (CSF) into the evaluation of 70 patients with CNS lesions of unknown cause. Participants underwent genotyping of CSF-derived DNA using a quantitative polymerase chain