journal
https://read.qxmd.com/read/38625997/decision-theoretical-foundation-of-clinical-practice-guidelines-an-extension-of-the-ash-thrombophilia-guidelines
#1
JOURNAL ARTICLE
Benjamin Djulbegovic, Iztok Hozo, Gordon H Guyatt
Decision analysis can play an essential role in informing practice guidelines. The American Society of Hematology (ASH) thrombophilia guidelines have made a significant step forward in demonstrating how decision modeling integrated within GRADE (Grading of Recommendations Assessment, Developing, and Evaluation) methodology can advance the field of guideline development. Although the ASH model was transparent and understandable, it does, however, suffer from the certain limitations that may have generated potentially wrong recommendations...
April 16, 2024: Blood Advances
https://read.qxmd.com/read/38625984/tabelecleucel-for-ebv-ptld-following-allogeneic-hct-or-sot-in-a-multicenter-expanded-access-protocol
#2
JOURNAL ARTICLE
Sarah Nikiforow, Jennifer S Whangbo, Ran Reshef, Donald E Tsai, Nancy J Bunin, Rolla F Abu-Arja, Kris Michael Mahadeo, Wen-Kai Weng, Koen Van Besien, David Loeb, Sunita D Nasta, Eneida R Nemecek, Weizhi Zhao, Yan Sun, Faith C Galderisi, Justin Wahlstrom, Aditi Mehta, Laurence Isabelle Gamelin, Rajani Dinavahi, Susan E Prockop
Patients with Epstein-Barr virus (EBV)-positive post-transplant lymphoproliferative disease (EBV+ PTLD) in whom initial treatment fails have few options and historically low median overall survival (OS) of 0.7 months following allogeneic hematopoietic cell transplant (HCT) and 4.1 months following solid organ transplant (SOT). Tabelecleucel is an off-the-shelf, allogeneic EBV-specific cytotoxic T-lymphocyte immunotherapy for EBV+ PTLD. Previous single-center experience showed responses in patients with EBV+ PTLD following HCT or SOT...
April 16, 2024: Blood Advances
https://read.qxmd.com/read/38625983/infections-in-lymphoma-patients-treated-with-bispecific-antibodies-a-systematic-review-and-meta-analysis
#3
JOURNAL ARTICLE
Gemma Reynolds, Marsali Maclean, Edward Robert Scheffer Cliff, Benjamin W Teh, Karin A Thursky, Monica A Slavin, Mary Ann Anderson, Eliza A Hawkes
No abstract text is available yet for this article.
April 16, 2024: Blood Advances
https://read.qxmd.com/read/38621200/optimized-cytogenetic-risk-group-stratification-of-kmt2a-rearranged-pediatric-acute-myeloid-leukemia
#4
JOURNAL ARTICLE
Romy Elise van Weelderen, Christine J Harrison, Kim Klein, Yilin Jiang, Jonas Abrahamsson, Todd Alonzo, Richard Aplenc, Nira Arad-Cohen, Emmanuelle Bart-Delabesse, Barbara Buldini, Barbara De Moerloose, Michael N Dworzak, Sarah Elitzur, José M Fernández Navarro, Alan S Gamis, Robert B Gerbing, Bianca F Goemans, Hester A de Groot-Kruseman, Erin M Guest, Shau Yin Ha, Henrik Hasle, Charikleia Kelaidi, Helene Lapillonne, Guy Leverger, Franco Locatelli, Takako Miyamura, Ulrika Noren-Nystrom, Sophia Polychronopoulou, Mareike Rasche, Jeffrey E Rubnitz, Jan Stary, Anne Tierens, Daisuke Tomizawa, Michel C Zwaan, Gertjan J L Kaspers
Comprehensive international consensus on cytogenetic risk-group stratification of KMT2A-rearranged (KMT2A-r) pediatric acute myeloid leukemia (AML) is lacking. This retrospective (2005-2016) International Berlin-Frankfurt-Münster Study Group study on 1,256 children with KMT2A-r AML aimed to validate the prognostic value of established recurring KMT2A fusions and additional cytogenetic aberrations (ACAs), and secondly, to define additional, recurring KMT2A fusions and ACAs, evaluating their prognostic relevance...
April 15, 2024: Blood Advances
https://read.qxmd.com/read/38621198/converting-improve-bleeding-and-vte-risk-assessment-models-into-a-fast-and-frugal-decision-tree-for-vte-prophylaxis
#5
JOURNAL ARTICLE
Benjamin Djulbegovic, Alice Boylan, Shelby J Kolo, Danielle Bowen Scheurer, Sydney Anuskiewicz, Flora Khaledi, Kimberley Youkhana, Sarah Madgwick, Neema Maharjan, Iztok Hozo
Current hospital VTE (venous thromboembolism) prophylaxis for medical patients has been characterized by both underuse and, increasingly, overuse. The American Society of Hematology (ASH) has endorsed the use of risk assessment models (RAM) as an approach to individualize VTE prophylaxis as a way of balancing overuse (excessive risk of bleeding) and underuse (risk of avoidable VTE). ASH has endorsed IMPROVE (International Medical Prevention Registry on Venous Thromboembolism) risk assessment models - the only RAM to assess short-term bleeding and VTE risk in acutely ill medical inpatients...
April 15, 2024: Blood Advances
https://read.qxmd.com/read/38621250/cutaneous-clonal-mature-plasmacytoid-dendritic-cell-dermatosis-in-patients-with-myeloid-neoplasms
#6
JOURNAL ARTICLE
Thibault Mahévas, Amélie Osio, Lise Larcher, Emmanuelle Clappier, Werner Kempf, Lionel Adès, Pierre Fenaux, Marie Sébert, Jérémie Delaleu, Marie Jachiet, Florence Cordoliani, Estelle Charvet, Olivier Carpentier, Raphaël A Itzykson, Marie Weinborn, Nicoleta Mardare, Joséfina Marco-Bonnet, Adèle de Masson, Nicolas Duployez, Tony Huynh, Jean-David Bouaziz, Marie-Dominique Vignon-Pennamen, Maxime Battistella
No abstract text is available yet for this article.
April 12, 2024: Blood Advances
https://read.qxmd.com/read/38621239/unscheduled-healthcare-interactions-in-multiple-myeloma-patients-receiving-t-cell-redirection-therapies
#7
JOURNAL ARTICLE
Anna J Howard, Isabel Concepcion, Alice X Wang, Issam S Hamadeh, Malin L Hultcrantz, Sham Mailankody, Carlyn Rose Tan, Neha Korde, Alexander M Lesokhin, Hani Hassoun, Urvi A Shah, Kylee H Maclachlan, Sridevi Rajeeve, Heather J Landau, Michael Scordo, Gunjan L Shah, Oscar B Lahoud, David J Chung, Sergio A Giralt, Saad Z Usmani, Ross S Firestone
Outcomes for relapsed/refractory multiple myeloma (RRMM) patients have dramatically improved following the development and now growing utilization of B cell maturation antigen targeted chimeric antigen receptor (CAR) T cell therapy and bispecific antibody (BsAb) therapy. However, healthcare utilization as a quality-of-life metric in these growing populations has not been thoroughly evaluated. We performed a retrospective cohort study evaluating the frequency and cause of unscheduled healthcare interactions (UHIs) among RRMM patients responding to B-cell maturation antigen targeted BsAbs and CAR T cell therapies (N = 46)...
April 12, 2024: Blood Advances
https://read.qxmd.com/read/38608258/2022-review-of-the-2019-american-society-of-hematology-guidelines-on-immune-thrombocytopenia
#8
JOURNAL ARTICLE
Cindy E Neunert, Donald M Arnold, Rachael F Grace, Thomas Kühne, Keith R McCrae, Deirdra R Terrell
The 2019 ASH guidelines for immune thrombocytopenia (ITP) included recommendations on management of adults (recommendations 1-9) and children (recommendations 10-21) with primary ITP (1). We describe here results of a review of the 2019 guidelines by a working group of experts requested by ASH to inform decision-making about the need for and timing of a guideline revision. An updated Medline and Embase search applied the same search terms as in the 2019 ASH guidelines, limited to systematic reviews and clinical trials, from May 2017 to July 2022...
April 12, 2024: Blood Advances
https://read.qxmd.com/read/38608257/clonal-hematopoiesis-and-inflammation-in-the-vasculature-chive-a-prospective-longitudinal-cohort-and-biorepository
#9
JOURNAL ARTICLE
Morgan L Shannon, Jonathan Brett Heimlich, Sydney Olson, Ariana Debevec, Zachary Copeland, Ashwin Kishtagari, Caitlyn Vlasschaert, Christina A Snider, Alexander J Silver, Donovan Brown, Travis P Spaulding, Manasa Ram Bhatta, Kelly Pugh, Shannon S Stockton, Jessica Ulloa, Yaomin Xu, Muhamed Baljevic, Javid Moslehi, Eiman Jahangir, Paul Brent Ferrell, David Alan Slosky, Alexander G Bick, Michael R Savona
Clonal hematopoiesis (CH) is an age-associated phenomenon leading to increased risk of both hematologic malignancy and non-malignant organ dysfunction. Increasingly available genetic testing has made incidental discovery of CH clinically common, yet evidence-based guidelines and effective management strategies to prevent adverse CH health outcomes are lacking. To address this gap, the prospective CHIVE registry and biorepository was created to identify and monitor individuals at risk, support multidisciplinary CH clinics, and to refine standards of practice for CH risk mitigation...
April 12, 2024: Blood Advances
https://read.qxmd.com/read/38603572/dual-role-of-signaling-pathways-in-myeloma-requires-cell-type-specific-targeting-of-ligand-receptor-interactions
#10
JOURNAL ARTICLE
Pablo Hernandez-Lopez, Tushara Vijaykumar, Praveen Anand, Daniel Auclair, Julia Frede, Birgit Knoechel, Jens G Lohr
Although most patients with multiple myeloma respond to treatment initially, therapy resistance develops almost invariably and only a subset of patients show durable responses to immunomodulatory (IMiD) therapies. While the immune microenvironment has been extensively studied in myeloma patients, its composition is currently not used as prognostic markers in clinical routine. We hypothesized that the outcome of immune signaling pathway engagement can be highly variable, depending on which two cellular populations participate in this interaction...
April 11, 2024: Blood Advances
https://read.qxmd.com/read/38603567/organ-involvement-in-adults-with-bpdcn-is-associated-with-sun-exposure-history-tet2-and-ras-mutations-and-survival
#11
JOURNAL ARTICLE
Shai Shimony, Julia H Keating, Christopher J Fay, Marlise R Luskin, Donna S Neuberg, Nicole R LeBoeuf, Andrew A Lane
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) can involve skin, bone marrow (BM), central nervous system (CNS), and non-CNS extramedullary sites. Preclinical models demonstrated clonal advantage of TET2-mutated plasmacytoid dendritic cells exposed to UV radiation. However, whether sun exposure, disease characteristics, and patient survival are clinically related is unclear. We classified 66 BPDCN patients based on organ involvement at diagnosis as skin only (n=19), systemic plus skin (n=33), or systemic only (n=14)...
April 11, 2024: Blood Advances
https://read.qxmd.com/read/38598754/haploidentical-transplantation-in-primary-refractory-relapsed-secondary-versus-de-novo-aml-from-the-alwp-ebmt
#12
JOURNAL ARTICLE
Arnon Nagler, Myriam Labopin, Johanna Tischer, Anna Maria Raiola, Desiree Kunadt, Jan Vydra, Didier Blaise, Patrizia Chiusolo, Renato Fanin, Julia Winkler, Edouard Forcade, Gwendolyn van Gorkom, Fabio Ciceri, Mohamad Mohty
We compared the outcomes of haploidentical stem cell transplantation (HaploHSCT) with post-transplant cyclophosphamide (PTCy) in 719 patients (pts) with primary refractory (PR) / first relapse (Rel) secondary acute myeloid leukemia (sAML) (n=129) versus those of de novo AML (n=590), transplanted between 2010 and 2022. A higher percentage of pts with sAML versus de novo AML had PR disease (73.6% vs. 58.6%) (p=0.002). In 81.4% of sAML pts, the antecedent hematological disorder was myelodysplastic syndrome. Engraftment was 83...
April 10, 2024: Blood Advances
https://read.qxmd.com/read/38598745/an-indirect-comparison-of-acalabrutinib-with-and-without-obinutuzumab-versus-zanubrutinib-in-treatment-naive-cll
#13
JOURNAL ARTICLE
Adam S Kittai, John N Allan, Dan James, Helen Bridge, Miguel Miranda, Alan Sm Yong, Fady Fam, Jack Roos, Vikram Shetty, Alan P Skarbnik, Matthew S Davids
The efficacy and safety of acalabrutinib plus obinutuzumab and acalabrutinib monotherapy versus zanubrutinib in patients with treatment-naive chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) without del(17p) were compared using an unanchored matching-adjusted indirect comparison. Individual patient-level data (IPD) from ELEVATE-TN (acalabrutinib plus obinutuzumab, n = 162; acalabrutinib monotherapy, n = 163) were weighted to match published aggregate baseline data from SEQUOIA cohort 1, which excluded patients with del(17p) (zanubrutinib, n = 241), using variables that were prognostic/predictive of investigator-assessed progression-free survival (INV-PFS) in an exploratory Cox regression analysis of ELEVATE-TN...
April 10, 2024: Blood Advances
https://read.qxmd.com/read/38598725/development-of-combination-therapies-with-btk-inhibitors-and-dasatinib-to-treat-cns-infiltrating-e2a-pbx1-prebcr-all
#14
JOURNAL ARTICLE
Gaia Gentile, Teresa Poggio, Antonella Catalano, Minna Voutilainen, Mari Lahnalampi, Marta Andrade-Martinez, Tobias Ma, Roman Sankowski, Lina Goncharenko, Stefan Tholen, Kyuho Han, David W Morgens, Marco Prinz, Michael Lübbert, Sophia Engel, Tanja N Hartmann, Gunnar Cario, Martin Schrappe, Lennart Lenk, Martin Stanulla, Justus Duyster, Peter Bronsert, Michael Bassik, Michael L Cleary, Oliver Schilling, Merja Heinäniemi, Jesus Duque-Afonso
The t(1;19) translocation, which codes for the oncogenic fusion protein E2A (TCF3)-PBX1, is involved in acute lymphoblastic leukemia (ALL) and associated with a pre-B cell receptor (preBCR+) phenotype. Relapse in E2A-PBX1+ ALL patients frequently occurs in the central nervous system (CNS). Therefore, there is a medical need for the identification of CNS active regimens for the treatment of E2A-PBX1+/preBCR+ ALL. Using unbiased shRNA library screening approaches, we identified Bruton's tyrosine kinase (BTK) as a key gene involved in both proliferation and dasatinib sensitivity of E2A-PBX1+/preBCR+ ALL...
April 10, 2024: Blood Advances
https://read.qxmd.com/read/38598713/patterns-of-crs-with-teclistamab-in-relapsed-refractory-multiple-myeloma-patients-with-prior-t-cell-redirection-therapy
#15
JOURNAL ARTICLE
Issam S Hamadeh, Tala Shekarkhand, Colin Joseph Rueda, Ross S Firestone, Alice X Wang, Neha Korde, Malin L Hultcrantz, Alexander M Lesokhin, Sham Mailankody, Hani Hassoun, Urvi A Shah, Kylee H Maclachlan, Sridevi Rajeeve, Dhwani Patel, Gunjan L Shah, Michael Scordo, Oscar B Lahoud, David J Chung, Heather J Landau, Sergio A Giralt, Saad Z Usmani, Carlyn Rose Tan
Teclistamab (Tec) is a first-in-class BCMA X CD3 bispecific T-cell engager antibody approved for treating multiple myeloma progressing after at least 4 lines of therapy. The objective of this study was to evaluate the rate of cytokine release syndrome (CRS) in patients who were treated with commercial Tec and had prior exposure to other T-cell redirection therapies. A retrospective chart review was performed to identify patients who completed the Tec step-up dosing phase between November 2022 and November 2023...
April 10, 2024: Blood Advances
https://read.qxmd.com/read/38598710/myeloablative-vs-non-myeloablative-consolidation-for-primary-central-nervous-system-lymphoma-results-of-alliance-51101
#16
JOURNAL ARTICLE
Tracy T Batchelor, Sharmila Giri, Amy S Ruppert, Susan Geyer, Scott E Smith, Nimish Mohile, Lode J Swinnen, Jonathan W Friedberg, Brad S Kahl, Nancy L Bartlett, Eric D Hsi, Bruce David Cheson, Nina D Wagner-Johnston, Lakshmi Nayak, John P Leonard, James Louis Rubenstein
While it is evident that standard dose whole brain radiotherapy as consolidation is associated with significant neurotoxicity, the optimal consolidative strategy for primary central nervous system lymphoma (PCNSL) is not defined. We performed a randomized phase 2 clinical trial via the U.S. Alliance cancer cooperative group to compare myeloablative consolidation supported by autologous stem cell transplantation with non-myeloablative consolidation after induction therapy for PCNSL. This is the first randomized trial to be initiated that eliminates whole brain radiotherapy as a consolidative approach in newly-diagnosed PCNSL...
April 10, 2024: Blood Advances
https://read.qxmd.com/read/38593461/distinguishing-ash-clinical-practice-guidelines-from-other-forms-of-ash-clinical-advice
#17
JOURNAL ARTICLE
Adam Cuker, Robert Kunkle, Rachel Sara Bercovitz, Michael T Byrne, Benjamin Djulbegovic, Sandra L Haberichter, Jennifer Holter-Chakrabarty, Richard Lottenberg, Menaka Pai, Suely Meireles Rezende, Matthew D Seftel, Roy L Silverstein, Deirdra R Terrell, Matthew C Cheung
The American Society of Hematology (ASH) develops a variety of resources that provide guidance to clinicians on the diagnosis and management of blood diseases. These resources include clinical practice guidelines (CPGs) and other forms of clinical advice. While both ASH CPGs and other forms of clinical advice provide recommendations, they differ with respect to the methods underpinning their development, the principal type of recommendations they offer, their transparency and concordance with published evidence, and the time and resources required for their development...
April 9, 2024: Blood Advances
https://read.qxmd.com/read/38593449/in-reply-fine-and-gray-or-cox-model
#18
JOURNAL ARTICLE
Yu Akahoshi
No abstract text is available yet for this article.
April 9, 2024: Blood Advances
https://read.qxmd.com/read/38593443/hereditary-hemorrhagic-telangiectasia-may-be-the-most-morbid-inherited-bleeding-disorder-of-women
#19
JOURNAL ARTICLE
Ellen Zhang, Zain M Virk, Josanna Rodriguez-Lopez, Hanny Al-Samkari
Hereditary hemorrhagic telangiectasia (HHT) is the second-most-common inherited bleeding disorder worldwide and remains without approved therapies. HHT causes serious mucosal bleeding resulting in severe iron deficiency anemia, major psychosocial complications, and visceral arteriovenous malformations in brain, lung, and liver that can cause life-threatening hemorrhagic complications. No study has examined the relative morbidity of HHT and von Willebrand disease (VWD), the most common inherited bleeding disorders in women...
April 9, 2024: Blood Advances
https://read.qxmd.com/read/38593241/hemolysis-events-in-the-phase-3-pegasus-study-of-pegcetacoplan-in-patients-with-paroxysmal-nocturnal-hemoglobinuria
#20
JOURNAL ARTICLE
Régis Peffault de Latour, Morag Griffin, Richard J Kelly, Jeff Szer, Carlos de Castro, Regina Horneff, Lisa Tan, Michael M Yeh, Jens Peter Panse
Patients with paroxysmal nocturnal hemoglobinuria (PNH) experience complement-mediated intravascular hemolysis leading to anemia, fatigue, and potentially life-threatening thrombotic complications. Pegcetacoplan, a C3 inhibitor, demonstrated sustained improvements in hematological and clinical parameters in the Phase 3 PEGASUS trial in patients with PNH who remained anemic despite C5 inhibitor therapy. The current post-hoc analysis describes 26 hemolysis adverse events (AEs) experienced in 19 patients during pegcetacoplan therapy in PEGASUS and baseline patient characteristics potentially associated with increased hemolysis risk...
April 9, 2024: Blood Advances
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