journal
https://read.qxmd.com/read/36951168/molecular-targeting-of-the-udp-glucuronosyltransferase-enzymes-in-high-eukaryotic-translation-initiation-factor-4e-refractory-relapsed-acute-myeloid-leukemia-patients-a-randomized-phase-ii-trial-of-vismodegib-ribavirin-with-or-without-decitabine
#1
JOURNAL ARTICLE
Sarit Assouline, Jadwiga Gasiorek, Julie Bergeron, Caroline Lambert, Biljana Culjkovic-Kraljacic, Eftihia Cocolakis, Chadi Zakaria, David Szlachtycz, Karen Yee, Katherine L B Borden
Drug resistance underpins poor outcomes in many malignancies including refractory and relapsed acute myeloid leukemia (R/R AML). Glucuronidation is a common mechanism of drug inactivation impacting many AML therapies e.g. cytarabine, decitabine, azacytidine and venetoclax. In AML cells, the capacity for glucuronidation arises from increased production of the UDP-glucuronosyltransferase 1A (UGT1A) enzymes. UGT1A elevation was first observed in AML patients who relapsed after response to ribavirin, a drug used to target the eukaryotic translation initiation factor eIF4E, and subsequently in patients who relapsed on cytarabine...
March 23, 2023: Haematologica
https://read.qxmd.com/read/36951166/high-risk-additional-cytogenetic-aberrations-in-a-dutch-chronic-phase-chronic-myeloid-leukemia-patient-population
#2
JOURNAL ARTICLE
Camille C B Kockerols, Inge G P Geelen, Mark-David Levin, Jeroen J W M Janssen, Berna H Beveloo, Avinash G Dinmohamed, Mels Hoogendoorn, Jan J Cornelissen, Peter E Westerweel
Not available.
March 23, 2023: Haematologica
https://read.qxmd.com/read/36951165/graft-versus-host-disease-and-relapse-rejection-free-survival-after-allogeneic-transplantation-for-idiopathic-severe-aplastic-anemia-a-comprehensive-analysis-from-the-saawp-of-the-ebmt
#3
JOURNAL ARTICLE
Raynier Devillier, Dirk-Jan Eikema, Carlo Dufour, Mahmoud Aljurf, Depei Wu, Alexei Maschan, Alexander Kulagin, Constantijn J M Halkes, Matthew Collin, John Snowden, Cécile Renard, Arnold Ganser, Karl-Walter Sykora, Brenda E Gibson, Johan Maertens, Maija Itäla-Remes, Paola Corti, Jan Cornelissen, Martin Bornhäuser, Mercedes Colorado Araujo, Hakan Ozdogu, Antonio Risitano, Gerard Socie, Regis Peffault De Latour
Survival after Allo-HSCT for severe idiopathic aplastic anemia (SAA) has improved in recent years, approaching 75% at 5 years. However, an SAA-adapted composite endpoint, GVHD and relapse/rejection-free survival (GRFS), may more accurately assess patient outcomes beyond survival. We analyzed GRFS to identify risk factors and specific causes of GRFS failure. Our retrospective analysis from the SAAWP of the EBMT included 479 patients with idiopathic SAA who underwent Allo-HSCT in 2 conventional situations: i) upfront Allo-HSCT from a matched related donor (MRD) (upfront cohort), and ii) Allo-HSCT for relapsed or refractory SAA (rel/ref cohort)...
March 23, 2023: Haematologica
https://read.qxmd.com/read/36951163/characteristics-and-clinical-outcomes-of-patients-with-acute-myeloid-leukemia-with-inv-3-q21q26-2-or-t-3-3-q21-q26-2
#4
JOURNAL ARTICLE
Guillaume Richard-Carpentier, Caitlin R Rausch, Koji Sasaki, Danielle Hammond, Kiyomi Morita, Koichi Takahashi, Guilin Tang, Rashmi Kanagal-Shamanna, Kapil Bhalla, Courtney D Dinardo, Gautam Borthakur, Naveen Pemmaraju, Elizabeth J Shpall, Amin Alousi, Naval G Daver, Guillermo Garcia-Manero, Marina Y Konopleva, Farhad Ravandi, Hagop M Kantarjian, Tapan M Kadia
Acute myeloid leukemia (AML) with inv(3)(q21q26.2)/t(3;3)(q21;q26.2) has a very poor prognosis. Determinants of clinical outcomes and optimal treatment remain uncertain. We retrospectively reviewed 108 cases of AML with inv(3)/t(3;3) and evaluated clinicopathological characteristics and clinical outcomes: 53 newly diagnosed (ND) AML and 55 relapsed/refractory (R/R) AML. Median age was 55 years. White blood cell (WBC) count ≥ 20 x 109/L and platelet count ≥ 140 x 109/L was observed in 25% and 32% of ND patients, respectively...
March 23, 2023: Haematologica
https://read.qxmd.com/read/36951162/chemotherapy-in-solitary-bone-plasmacytoma-to-prevent-evolution-to-multiple-myeloma
#5
JOURNAL ARTICLE
Sophia Ascione, Stéphanie Harel, Florent L Besson, Rakiba Belkhir, Julien Henry, Bruno Royer, Bertrand Arnulf, Xavier Mariette, Raphaèle Seror
Not available.
March 23, 2023: Haematologica
https://read.qxmd.com/read/36951160/impact-of-additional-genetic-abnormalities-at-diagnosis-of-chronic-myeloid-leukemia-for-first-line-imatinib-treated-patients-receiving-proactive-treatment-intervention
#6
JOURNAL ARTICLE
Naranie Shanmuganathan, Carol Wadham, NurHezrin Shahrin, Jinghua Feng, Daniel Thomson, Paul Wang, Verity Saunders, Chung Hoow Kok, Rob M King, Rosalie R Kenyon, Ming Lin, Ilaria S Pagani, David M Ross, Agnes S M Yong, Andrew P Grigg, Anthony K Mills, Anthony P Schwarer, Jodi Braley, Haley Altamura, David T Yeung, Hamish S Scott, Andreas W Schreiber, Timothy P Hughes, Susan Branford
The BCR::ABL1 gene fusion initiates chronic myeloid leukemia (CML), however evidence has accumulated from studies of highly selected cohorts that variants in other cancer-related genes are associated with treatment failure. Nevertheless, the true incidence and impact of additional genetic abnormalities (AGAs) at diagnosis of chronic phase (CP)-CML is unknown. We sought to determine whether AGAs at diagnosis in a consecutive imatinib-treated cohort of 210 patients enrolled in the TIDEL-II trial influenced outcome despite a highly proactive treatment intervention strategy...
March 23, 2023: Haematologica
https://read.qxmd.com/read/36951158/risk-of-relapse-after-sars-cov-2-vaccine-in-the-milan-cohort-of-thrombotic-thrombocytopenic-purpura-patients
#7
JOURNAL ARTICLE
Marco Capecchi, Pasqualina De Leo, Maria Abbattista, Ilaria Mancini, Pasquale Agosti, Marina Biganzoli, Chiara Suffritti, Barbara Ferrari, Anna Lecchi, Silvia La Marca, Lidia Padovan, Erica Scalambrino, Marigrazia Clerici, Armando Tripodi, Andrea Artoni, Roberta Gualtierotti, Flora Peyvandi
Not available.
March 23, 2023: Haematologica
https://read.qxmd.com/read/36951156/survival-outcomes-with-oral-azacitidine-maintenance-in-patients-with-acute-myeloid-leukemia-in-remission-by-receipt-of-initial-chemotherapy-subgroup-analyses-from-the-phase-3-quazar-aml-001-trial
#8
JOURNAL ARTICLE
Andrew H Wei, Gail J Roboz, Herve Dombret, Hartmut Dohner, Andre C Schuh, Pau Montesinos, Dominik Selleslag, Sergey N Bondarenko, Thomas Prebet, Yinzhi Lai, Barry Skikne, C L Beach, Farhad Ravandi
Not available.
March 23, 2023: Haematologica
https://read.qxmd.com/read/36951152/tagraxofusp-for-blastic-plasmacytoid-dendritic-cell-neoplasm
#9
JOURNAL ARTICLE
Marlise R Luskin, Andrew A Lane
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic malignancy that presents with characteristic dark purple skin papules, plaques, and tumors, but may also involve the bone marrow, blood, lymph nodes, and central nervous system. The disease, which commonly affects older men but can also present in children, is associated with a distinct immunophenotype including universal expression of CD123, the alpha chain of the interleukin 3 receptor. Recently, tagraxofusp, a CD123-targeting drug consisting of the ligand for CD123, interleukin 3, conjugated to a truncated diphtheria toxin payload was approved for treatment of BPDCN...
March 23, 2023: Haematologica
https://read.qxmd.com/read/36951151/allogeneic-transplantation-in-acute-myelogenous-leukemia-a-comprehensive-single-institution-s-experience
#10
JOURNAL ARTICLE
Gerard Socie, Jacques-Emmanuel Galimard, Emmanuel Raffoux, Raphael Itzykson, Pierre Edouard Debureaux, David Michonneau, Etienne Lengliné, Marie Robin, Flore Sicre De Fontbrune, Marie Sébert, Aliénor Xhaard, Rathana Kim, Anne Couprie, Nathalie Dhedin, Matteo Dragani, Pierre Lemaire, Lise Larcher, Emmanuelle Clappier, Nicolas Boissel, Jean Soulier, Hervé Dombret, Pierre Fenaux, Régis Peffault De Latour, Lionel Adès
Since decades, debates on the role and timing of allogeneic transplantation HSCT in acute myelogenous leukemia (AML) persist. Time to transplant introduces an immortal time and current treatment algorithm mainly relies on the ELN's disease risk classification. Previous studies are also limited to age groups, remission status and other ill-defined parameters. We studied all patients at diagnosis irrespective of age and comorbidities to estimate the cumulative incidence and potential benefit or disadvantage of HSCT in a single center...
March 23, 2023: Haematologica
https://read.qxmd.com/read/36951150/evidence-of-cure-for-extranodal-nasal-type-natural-killer-t-cell-lymphoma-with-current-treatment-an-analysis-of-the-clcg-database
#11
JOURNAL ARTICLE
Xin Liu, Li-Ling Zhang, Bao-Lin Qu, Qiu-Zi Zhong, Li-Ting Qian, Yong Yang, Xiao-Rong Hou, Xue-Ying Qiao, Hua Wang, Yuan Zhu, Jian-Zhong Cao, Jun-Xin Wu, Tao Wu, Su-Yu Zhu, Mei Shi, Hui-Lai Zhang, Xi-Mei Zhang, Hang Su, Yu-Qin Song, Jun Zhu, Yu-Jing Zhang, Hui-Qiang Huang, Ying Wang, Fan Chen, Lin Yin, Xia He, Shang Cai, Ye-Xiong Li, Shu-Nan Qi
Survival from extranodal nasal-type NK/T-cell lymphoma (ENKTCL) has substantially improved over the last decade. However, there is little consensus as to whether a population of patients with ENKTCL can be considered "cured" of the disease. We aimed to evaluate the statistical "cure" of ENKTCL in the modern treatment era. This retrospective multicentric study reviewed the clinical data of 1955 patients with ENKTCL treated with non-anthracycline-based chemotherapy and/or radiotherapy between 2008 and 2016 in China Lymphoma Collaborative Group multicenter database...
March 23, 2023: Haematologica
https://read.qxmd.com/read/36924323/the-lymphoma-microenvironment-comes-of-age-in-the-r-chop-era
#12
JOURNAL ARTICLE
Alan Cooper, Justin Kline
Not available.
March 16, 2023: Haematologica
https://read.qxmd.com/read/36924255/the-trim31-paradox-an-unexpected-benefit-for-leukemia-stem-cells
#13
JOURNAL ARTICLE
Jasmin Straube, Steven W Lane
Not available.
March 16, 2023: Haematologica
https://read.qxmd.com/read/36924254/myeloid-cells-from-langerhans-cell-histiocytosis-patients-exhibit-increased-vesicle-trafficking-and-an-altered-secretome-capable-of-activating-nk-cells
#14
JOURNAL ARTICLE
Daniel W Hagey, Egle Kvedaraite, Mira Akber, André Görgens, Joman Javadi, Tatiana Von Bahr Greenwood, Caroline Björklund, Selma Olsson Åkefeldt, Tova Hannegård-Hamrin, Henrik Arnell, Katalin Dobra, Nikolas Herold, Mattias Svensson, Samir El Andaloussi, Jan-Inge Henter, Magda Lourda
Langerhans cell histiocytosis (LCH) is a potentially life-threatening inflammatory myeloid neoplasia linked to paediatric neurodegeneration, whereby transformed LCH cells form agglomerated lesions in various organs. Although MAP-kinase pathway mutations have been identified in LCH cells, the functional consequences of these mutations and the mechanisms that cause the pathogenic behaviour of LCH cells are not well understood. In our study, we used an in vitro differentiation system and RNA-sequencing to compare monocyte-derived dendritic cells from LCH patients to those derived from healthy controls or patients with Crohn's disease, a non-histiocytic inflammatory disease...
March 16, 2023: Haematologica
https://read.qxmd.com/read/36924252/pax-transactivation-domain-interacting-protein-is-required-for-preserving-hematopoietic-stem-cell-quiescence-via-regulating-lysosomal-activity
#15
JOURNAL ARTICLE
Tong Zhang, Manman Cui, Yashu Li, Ying Cheng, Zhuying Gao, Jing Wang, Tiantian Zhang, Guoqiang Han, Rong Yin, Peipei Wang, Wen Tian, Weidong Liu, Jin Hu, Yuhua Wang, Zheming Liu, Haojian Zhang
Hematopoietic stem cells (HSCs) maintain lifetime whole blood hematopoiesis through self-renewal and differentiation. To sustain HSC stemness, most HSCs reside in a quiescence state, which is affected by diverse cellular stress and intracellular signal transduction. How HSCs accommodate those challenges to preserve lifetime capacity remains elusive. Here we show that Pax transactivation domain-interacting protein (PTIP) is required for preserving hematopoietic stem cell quiescence via regulating lysosomal activity...
March 16, 2023: Haematologica
https://read.qxmd.com/read/36924249/the-glanzmann-thrombasthenia-registry-safety-of-platelet-therapy-in-patients-with-glanzmann-thrombasthenia-and-changes-in-alloimmunization-status
#16
JOURNAL ARTICLE
Man-Chiu Poon, Roseline D'Oiron, Sibin Baby, Rainer B Zotz, Giovanni Di Minno
Not available.
March 16, 2023: Haematologica
https://read.qxmd.com/read/36924240/artificial-intelligence-to-empower-diagnosis-of-myelodysplastic-syndromes-by-multiparametric-flow-cytometry
#17
JOURNAL ARTICLE
Valentin Clichet, Delphine Lebon, Nicolas Chapuis, Jaja Zhu, Valérie Bardet, Jean-Pierre Marolleau, Loïc Garçon, Alexis Caulier, Thomas Boyer
The diagnosis of myelodysplastic syndromes (MDS) might be challenging and relies on the convergence of cytological, cytogenetic, and molecular arguments. Multiparametric flow cytometry (MFC) helps diagnose MDS, especially when other features are non-contributory, but remains underestimated mostly due to a lack of standardization of cytometers. We present here an innovative model integrating artificial intelligence (AI) with MFC to improve the diagnosis and the classification of MDS. We develop a machine learning model by elasticnet algorithm trained on a cohort of 191 patients and only based on flow cytometry parameters selected by Boruta algorithm, to build a simple but reliable prediction score with 5 parameters...
March 16, 2023: Haematologica
https://read.qxmd.com/read/36924235/severity-and-burden-of-sickle-cell-disease-in-france-a-nationwide-realworld-study
#18
JOURNAL ARTICLE
Valentine Brousse, Francoise Bernaudin, Asma Melaine, Melanie Goguillot, Meghan Gallagher, Steve Benard, Anoosha Habibi
The burden of sickle cell disease (SCD) in France has been difficult to apprehend due to the paucity of reliable nationwide epidemiological data. We aimed to describe the epidemiology of SCD and evaluate its burden and costs. Patients with SCD and most severely affected patients were identified between 2012 and 2018 from the French National Health Data System database (SNDS, Systeme national des donnees de sante). Outcomes of interest included rates of acute and chronic complications, healthcare resource utilisation and associated costs, and were compared in subpopulations of patients before and after Hematopoietic Stem Cell Transplantation (HSCT), initiating hydroxyurea (HU) or a chronic transfusion program (CTP)...
March 16, 2023: Haematologica
https://read.qxmd.com/read/36891751/impact-of-central-nervous-system-involvement-in-adult-patients-with-philadelphia-negative-acute-lymphoblastic-leukemia-a-graall-2005-study
#19
JOURNAL ARTICLE
Corentin Orvain, Sylvain Chantepie, Xavier Thomas, Martine Escofrre-Barbe, Francoise Huguet, Yohan Desbrosses, Gaelle Guillerm, Madalina Uzunov, Thibaut Leguay, Sarah Barbieux, Norbert Vey, Patrice Chevallier, Jean-Valere Malfuson, Stephane Lepretre, Michael Baumann, Murat Aykut, Abdelaziz Chaib, Magalie Joris, Hacene Zerazhi, Georg Stussi, Jacques Chapiro, Celine Berthon, Caroline Bonmati, Eric Jourdan, Diana Carp, Amb Roise Marcais, Maria-Pilar Gallego-Hernanz, Iona Vaida, Karin Bilger, Alban Villate, Florence Pasquier, Yves Chalandon, Sebastien Maury, Veronique Lheritier, Norbert Ifrah, Herve Dombret, Nicolas Boissel, Mathilde Hunault-Berger
Whereas the prognosis of adult patients with Philadelphia-negative acute lymphoblastic leukemia (ALL) has greatly improved since the advent of pediatric-inspired regimens, the impact of initial central nervous system (CNS) involvement has not been formerly reevaluated. We report herein the outcome of patients with initial CNS involvement included in the pediatric-inspired prospective randomized GRAALL-2005 study. Between 2006 and 2014, 784 adult patients (18-59 years old) with newly diagnosed Philadelphia-negative ALL were included of whom 55 (7%) had CNS involvement...
March 9, 2023: Haematologica
https://read.qxmd.com/read/36891749/post-transplantation-burkitt-lymphoma-a-retrospective-study-of-55-patients
#20
JOURNAL ARTICLE
Pierre Walczak, Sylvain Choquet, Jacques Dantal, David Boutboul, Felipe Suarez, Marine Baron, Veronique Morel, Thomas Cluzeau, Mohamed Touati, Michelle Elias, Emmanuel Bachy, Emmanuelle Nicolas-Virelizier, Roch Houot, Geoffroy Venton, Caroline Jacquet, Marie-Pierre Moles-Moreau, Fabrice Jardin, Eric Durot, Noureddine Balegroune, Laure Ecotiere, Romain Guieze, Nassim Kamar, Loic Ysebaert, Lionel Couzi, Hugo Gonzalez, Louise Roulin, Kevin Ou, Sophie Caillard, Heiner Zimmermann, Ralf Ulrich Trappe, Damien Roos-Weil
Not available.
March 9, 2023: Haematologica
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