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JOURNAL ARTICLE
Stéphanie Roullet, Norman Luc, Julie Rayes, Jean Solarz, Dante Disharoon, Andrew J Ditto, Emily Gahagan, Christa Pawlowski, Thibaud Sefiane, Frédéric Adam, Caterina Casari, Olivier D Christophe, Michael A Bruckman, Peter J Lenting, Anirban Sen Gupta, Cecile V Denis
The lack of innovation in Von Willebrand disease (VWD) originates from many factors including the complexity and heterogeneity of the disease but also from a lack of recognition of the impact of the bleeding symptoms experienced by VWD patients. Recently, a few research initiatives aiming to move past replacement therapies using plasma-derived or recombinant Von Willebrand factor (VWF) concentrates have started to emerge. Here we report an original approach using synthetic platelet (SP) nanoparticles for treatment of VWD type 2B (VWD-2B) and severe VWD (type 3 VWD)...
March 17, 2023: Blood
#2
JOURNAL ARTICLE
Sarah Anne Bird, Charlotte Pawlyn
Immunomodulatory agents (IMiDs) are a cornerstone of treatment for patients with multiple myeloma. IMiDs are used in therapeutic combinations at all stages of disease and are approved as a single agent maintenance treatment after autologous stem cell transplantation. However patients will become resistant to ongoing therapy over time and inevitably relapse. It is only in the last decade that the mechanism of IMiD action has been elucidated; through binding to the cereblon component of the CRL4CRBN E3 ubiquitin ligase a set of neosubstrates is designated for degradation by the proteosome...
March 16, 2023: Blood
#3
JOURNAL ARTICLE
Cavan Bennett, Victoria E Jackson, Anne Pettikiriarachchi, Thomas Hayman, Ute Schaeper, Gemma L Moir-Meyer, Katherine Louise Fielding, Ricardo Ataide, Danielle Clucas, Andrew James Baldi, Alexandra L Garnham, Connie Sn Li-Wai-Suen, Stephen John Loughran, E Joanna Baxter, Anthony R Green, Warren S Alexander, Melanie Bahlo, Kate Burbury, Ashley P Ng, Sant-Rayn Pasricha
Polycythemia Vera (PV) is a myeloproliferative neoplasm driven by activating mutations in JAK2 that result in unrestrained erythrocyte production, increasing patients' hematocrit and hemoglobin concentration, placing them at risk of life-threatening thrombotic events. Our GWAS of 440 PV cases and 403,351 controls utilizing UK Biobank data found that SNPs in HFE known to cause hemochromatosis are highly associated with PV diagnosis, linking iron regulation to PV. Analysis of the FinnGen dataset independently confirmed over-representation of homozygous HFE variants in PV patients...
March 16, 2023: Blood
#4
JOURNAL ARTICLE
Hao Zhao, Miao-Yan Zhang, Kai-Ni Shen, Jun Feng, Xin-Xin Cao, Ming-Hui Duan, Dao-Bin Zhou, Lu Zhang, Jian Li
No abstract text is available yet for this article.
March 16, 2023: Blood
#5
JOURNAL ARTICLE
Helen E Heslop
No abstract text is available yet for this article.
March 16, 2023: Blood
#6
JOURNAL ARTICLE
Ji-Liang Gao, Albert Owusu-Ansah, Alexander Yang, Erin Yim, David H McDermott, Paejonette Jacobs, Shamik Majumdar, Uimook Choi, Colin L Sweeney, Harry L Malech, Philip M Murphy
WHIM syndrome is an autosomal dominant immunodeficiency disorder caused by gain-of-function mutations in chemokine receptor CXCR4 that promote severe panleukopenia due to bone marrow (BM) retention of mature leukocytes. We previously reported that Cxcr4-haploinsufficient (Cxcr4+/o) hematopoietic stem cells (HSCs) have a strong selective advantage for durable hematopoietic reconstitution over wildtype (Cxcr4+/+) and WHIM (Cxcr4+/w) HSCs, and that a WHIM patient was spontaneously cured by chromothriptic deletion of the disease allele in an HSC, suggesting that WHIM allele inactivation through gene editing may be a safe genetic cure strategy for the disease...
March 16, 2023: Blood
#7
JOURNAL ARTICLE
Ryan C Lynch, Chaitra S Ujjani, Christina Poh, Edus H Warren, Stephen D Smith, Mazyar Shadman, Brian G Till, Vikram Raghunathan, Stefan Alig, Ash A Alizadeh, Avanti Gulhane, Delphine Chen, Yolanda Tseng, Hilary Coye, Megan Shelby, Susan Ottemiller, Sarith Keo, Kaitlin Verni, Hongyan Du, Jacquelin Vandermeer, Ashley Gaston, Heather Rasmussen, Paul Martin, Edmond Marzbani, Jenna M Voutsinas, Ajay K Gopal
Concurrent administration pembrolizumab with chemotherapy in untreated classical Hodgkin lymphoma (CHL) has not previously been studied. To investigate this combination, we conducted a single arm study of concurrent pembrolizumab with AVD (APVD) for untreated CHL. We enrolled 30 patients (6 early favorable, 6 early unfavorable, and 18 advanced stage, median age 33 years (range 18-69 years)) and met the primary safety endpoint with no observed significant treatment delays in the first two cycles. Twelve patients experienced grade 3-4 non-hematologic adverse events (AEs) most commonly febrile neutropenia (5, 17%) and infection/sepsis (3, 10%)...
March 13, 2023: Blood
#8
JOURNAL ARTICLE
Lok Lam Ngai, Diana Hanekamp, Fleur Janssen, Jannemieke Carbaat-Ham, Maaike A M Hofland, Mona M H E Fayed, Angèle Kelder, Laura Oudshoorn-van Marsbergen, Willemijn J Scholten, Alexander N Snel, Costa Bachas, Jesse M Tettero, Dimitri A Breems, Thomas Fischer, Bjorn T Gjertsen, Laimonas Griskevicius, Gunnar Juliusson, Arjan A van de Loosdrecht, Johan A Maertens, Markus G Manz, Thomas Pabst, Jakob R Passweg, Kimmo Porkka, Peter J M Valk, Patrycja Gradowska, Bob Löwenberg, David C de Leeuw, Jeroen J W M Janssen, Gert J Ossenkoppele, Jacqueline Cloos
No abstract text is available yet for this article.
March 10, 2023: Blood
#9
JOURNAL ARTICLE
Antonino Carbone, Annunziata Gloghini, Carmelo Carlo Stella
Classic Hodgkin lymphoma (cHL) is characterized by a tumor microenvironment (TME) containing inflammatory/immune cells. Follicular lymphoma, mediastinal grey zone lymphoma and diffuse large B-cell lymphomas, may show a TME containing inflammatory/immune cells, but the TMEs are quite different. In these B-cell lymphomas and cHL, PD-1/PD-L1 blockade drugs differ in their effectiveness in patients with refractory/relapsed disease. Research should still explore innovative assays that could reveal which molecules are influencing the sensitivity or the resistance to therapy in an individual patient...
March 10, 2023: Blood
#10
JOURNAL ARTICLE
Armando Santoro, Alison J Moskowitz, Silvia Ferrari, Carmelo Carlo-Stella, Julie M Lisano, Stephen Francis, Rachael Wen, Alev Akyol, Kerry J Savage
No abstract text is available yet for this article.
March 10, 2023: Blood
#11
JOURNAL ARTICLE
Rebecca Karp Leaf, Amy K Dickey
Erythropoietic protoporphyria (EPP) is an inherited cutaneous porphyria caused by reduced expression of ferrochelatase, the enzyme that catalyzes the final step in heme biosynthesis. The resultant accumulation of protoporphyrin IX leads to severe painful cutaneous photosensitivity, as well as potentially life-threatening liver disease in a small percentage of patients. X-linked protoporphyria (XLP) is similar to EPP clinically, but results from increased activity of δ-aminolevulinic acid synthase 2 (ALAS2), the first step in heme biosynthesis in the bone marrow, and also causes protoporphyrin accumulation...
March 10, 2023: Blood
#12
JOURNAL ARTICLE
Kunhua Qin, Xianjiang Lan, Peng Huang, Megan S Saari, Eugene Khandros, Cheryl A Keller, Belinda M Giardine, Osheiza Abdulmalik, Junwei Shi, Ross C Hardison, Gerd A Blobel
The fetal (HbF)-to-adult (HbA) hemoglobin switch is a paradigm for developmental gene expression control with relevance to sickle cell disease and b-thalassemia. Polycomb repressive complex (PRC) proteins regulate this switch, and an inhibitor of PRC2 has entered a clinical trial for fetal hemoglobin activation. Yet, how PRC complexes function in this process, their target genes, and relevant subunit composition are unknown. Here, we identified the PRC1 subunit BMI1 as a novel HbF repressor. We uncovered the RNA binding proteins LIN28B, IGF2BP1, and IGF2BP3 as direct BMI1 targets and demonstrate that they account for the entirety of BMI1's effects on HbF regulation...
March 9, 2023: Blood
#13
JOURNAL ARTICLE
Stephanie Guillet, Etienne Crickx, Imane Azzaoui, Pascal Chappert, Emmanuelle Boutin, Jean-François Viallard, Etienne Riviere, Delphine Gobert, Lionel Galicier, Marion Malphettes, Stéphane Cheze, Francois Lefrere, Sylvain Audia, Bernard Bonnotte, Olivier Lambotte, Nicolas Noel, Olivier Fain, Guillaume Moulis, Mohamed Hamidou, Mathieu Gerfaud-Valentin, Jean-Pierre Marolleau, Louis Terriou, Nihal Martis, Anne-Sophie Morin, Antoinette Perlat, Thomas Le Gallou, Frédérique Roy-Peaud, Ailsa Robbins, Jean-Christophe Lega, Mathieu Puyade, Thibault Comont, Nicolas Limal, Laetitia Languille, Anissa Zarour, Marine Luka, Mickaël Mathieu Ménager, Thibaut Belmondo, Sophie Hue, Florence Canoui-Poitrine, Marc Michel, Bertrand Godeau, Matthieu Mahevas
Sustained response off-treatment (SROT) after thrombopoietin receptor agonist (TPO-RA) discontinuation has been reported in ITP. This prospective multicenter interventional study enrolled adults with persistent or chronic primary ITP and complete response on TPO-RAs. The primary endpoint was the proportion of patients achieving SROT (platelet count > 30x109/L and no bleeding) at W24 with no other ITP-specific medications. Secondary endpoints included the proportion of sustained complete response off-treatment (SCROT, platelet count > 100x109/L and no bleeding) and SROT at W52, bleeding events, and pattern of response to a new course of TPO-RAs...
March 9, 2023: Blood
#14
JOURNAL ARTICLE
Aisha Jibril, Charlotte Hellmich, Edyta Wojtowicz, Katherine Hampton, Rebecca S Maynard, Ravindu De Silva, Dominic J Fowler-Shorten, Jayna J Mistry, Jamie A Moore, Kristian M Bowles, Stuart A Rushworth
Mitochondrial damage-associated molecular patterns (mtDAMPs) include proteins, lipids, metabolites and DNA and have various context specific immunoregulatory functions. Cell-free mitochondrial DNA (mtDNA) is recognised via pattern recognition receptors and is a potent activator of the innate immune system. Cell-free mtDNA is elevated in the circulation of trauma and cancer patients, however the functional consequences of elevated mtDNA are largely undefined. Multiple myeloma (MM) relies upon cellular interactions within the bone marrow (BM) microenvironment for survival and progression...
March 8, 2023: Blood
#15
JOURNAL ARTICLE
Bianca D Santomasso, Juliane Gust, Fabiana Perna
With growing indications for Chimeric Antigen Receptor (CAR) T-cell therapy, toxicity profiles are evolving. There is an urgent and unmet need for approaches to optimally manage emerging adverse events that extend beyond the standard paradigm of cytokine release syndrome (CRS) and immune effector cell associated neurotoxicity syndrome (ICANS). While management guidelines exist for ICANS, there is little guidance on how to approach patients with neurologic comorbidities, and how to manage rare neurotoxicity presentations such as CAR T-cell therapy related cerebral edema, severe motor complications or late onset neurotoxicity...
March 6, 2023: Blood
#16
JOURNAL ARTICLE
Olga K Weinberg, Daniel A Arber, Hartmut Döhner, Charles G Mullighan, Etan Orgel, Anna Porwit, Richard M Stone, Michael J Borowitz
No abstract text is available yet for this article.
March 6, 2023: Blood
#17
JOURNAL ARTICLE
Chrissie K Lim, Youjia Zhong, Richard Hopkins, Wei-Xiang Sin, Bijin Veonice Au, Sriram Narayanan, Chiung-Hui Huang, Colin Yc Lee, Ming Liang Oon, Avisha Chowdhury, Benjamin Wong, Frances Yeap, Mariflor Villegas, Julien Pompon, Patricia Pl Ng, Siok-Bian Ng, Thuan Chong T Quah, Poh-Lin Tan, Keh-Chuang Chin, John E Connolly
Severe mosquito bite allergy (SMBA) is a manifestation of chronic active Epstein-Barr virus (CAEBV) infection defined by necrotic ulceration of the stings. CAEBV with SMBA has a high mortality rate as most patients eventually develop fulminant and refractory hemophagocytic lymphohistiocytosis (HLH). However, how self-resolving SMBA escalates to systemic lethal HLH remains unclear. Through comprehensive immune profiling of a SMBA patient with CAEBV and her healthy monozygotic twin, we found that both twins were seropositive for EBV but showed high discordance in their circulating natural killer (NK) cells...
March 6, 2023: Blood
#18
JOURNAL ARTICLE
Massimo Cugno, Ilaria Mancini, Dario Consonni, Valentina De Zan, Gianluigi Ardissino, Samantha Griffini, Elena Grovetti, Luigi Porcaro, Barbara Ferrari, Andrea Artoni, Flora Peyvandi
No abstract text is available yet for this article.
March 6, 2023: Blood
#19
JOURNAL ARTICLE
Yao Yao, Jessica Fong Ng, Woojun Daniel Park, Mehmet K Samur, Eugenio Morelli, Jessica Encinas, Zuzana Chyra, Yan Xu, Sanika Derebail, Charles B Epstein, Behnam Nabet, Marta Chesi, Nathanael S Gray, Richard Young, Nicholas Kwiatkowski, Constantine S Mitsiades, Kenneth C Anderson, Charles Y Lin, Nikhil C Munshi, Mariateresa Fulciniti
Therapeutic targeting of CDK7 has proven beneficial in pre-clinical studies, yet the off-target effects of currently available CDK7 inhibitors make it difficult to pinpoint the exact mechanisms behind MM cell death mediated by CDK7 inhibition. Here, we show that CDK7 expression positively correlates with E2F and MYC transcriptional programs in multiple myeloma (MM) patient cells; and its selective targeting counteracts E2F activity via perturbation of the CDKs/Rb axis and impairs MYC-regulated metabolic gene signatures translating into defects in glycolysis and reduced levels of lactate production in MM cells...
March 6, 2023: Blood
#20
JOURNAL ARTICLE
Mario Cazzola
No abstract text is available yet for this article.
March 3, 2023: Blood
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