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JOURNAL ARTICLE
Sanjeet Singh Dadwal, Rajat Bansal, Michael Schuster, Jean A Yared, Gary Douglas Myers, Michelle Elizabeth Matzko, Sama Adnan, David McNeel, Julie Ma, Sarah A Gilmore, Spyridoula Vasileiou, Ann M Leen, Joshua A Hill, Jo-Anne Young
Allogeneic hematopoietic cell transplantation (allo-HCT) recipients are susceptible to viral infections. We conducted a phase 2 trial evaluating the safety and rate of clinically significant infections (CSIs; viremia requiring treatment or end-organ disease) following infusion of posoleucel, a partially HLA-matched, allogeneic, off-the-shelf, multivirus-specific T cell investigational product for preventing CSIs with adenovirus, BK virus, cytomegalovirus, Epstein-Barr virus, human herpesvirus-6, or JC virus...
April 9, 2024: Blood Advances
#22
JOURNAL ARTICLE
Jun Wan, Sophia Dhrolia, Rohan R Kasthuri, Yuriy Prokopenko, Anton Ilich, Prakash Saha, Mark Roest, Alisa S Wolberg, Nigel S Key, Rafal Pawlinski, Pavan K Bendapudi, Nigel Mackman, Steven P Grover
Plasma kallikrein (PKa) is an important activator of factor (F)XII of the contact pathway of coagulation. Several studies have shown that PKa also possesses procoagulant activity independent of FXII, likely through its ability to directly activate FIX. We evaluated the procoagulant activity of PKa using a mouse whole blood (WB) thrombin generation (TG) assay. TG was measured in WB from PKa-deficient mice using contact pathway or extrinsic pathway triggers. PKa-deficient WB had significantly reduced contact pathway-initiated TG compared to wild-type controls and was comparable to that observed in FXII-deficient WB...
April 9, 2024: Blood Advances
#23
JOURNAL ARTICLE
Ying Liu, Rohan Sardana, David Nemirovsky, Denise Frosina, Achim Jungbluth, William T Johnson, Santosha A Vardhana, Maria E Arcila, Steven M Horwitz, Andriy Derkach, Ahmet Dogan, Wenbin Xiao
While significant progress has been made in understanding the genetic basis of primary hemophagocytic lymphohistiocytosis (HLH), the pathogenesis of secondary HLH, the more prevalent form, remains unclear. Among the various conditions giving rise to secondary HLH, HLH in lymphoma patients (HLH-L) accounts for a substantial proportion. In this study, we investigated the role of somatic mutations in the pathogenesis of HLH-L in a cohort of patients with T- and/or NK-cell lymphoma. We identified a 3-time higher frequency of mutations in FAS pathway in patients with HLH-L...
April 9, 2024: Blood Advances
#24
JOURNAL ARTICLE
Shermarke Hassan, Guido Baselli, Luca Mollica, Riccardo L Rossi, Himani Chand, Amal Mohamed El-Beshlawy, Mohsen Saleh Elalfy, Vijay Ramanan, Peyman Eshghi, Mehran Karimi, Roberta Palla, Frits R Rosendaal, Flora Peyvandi
Inhibitor development is the most severe complication of hemophilia A care, and is associated with increased morbidity and mortality. The aim of this study was to use a novel IgG epitope mapping method to explore the factor VIII (FVIII)-specific epitope profile in the SIPPET cohort population and to develop an epitope-mapping based inhibitor prediction model. The population consisted of 122 previously untreated patients with severe hemophilia A that were followed-up for 50 days of exposure to FVIII or 3 years, whichever occurred first...
April 9, 2024: Blood Advances
#25
JOURNAL ARTICLE
V Koneti Rao, Elaine M Kulm, Jennifer K Grossman, David K Buchbinder, Hey Chong, Jason Bradt, Sharon Webster, Anna Šedivá, Virgil A Dalm, Gulbu Uzel
Activated phosphoinositide 3-kinase delta syndrome (APDS) is an inborn error of immunity that manifests as immune deficiency and dysregulation; symptoms include frequent infections and lymphoproliferation. In our dose-finding and phase 3 placebo-controlled trials, treatment with the selective PI3Kδ inhibitor leniolisib reduced lymphoproliferation and normalized lymphocyte subsets. Here, we present 6 years of follow-up from the 6 adult patients in the original dose-finding trial receiving leniolisib. We used data from the ongoing open-label extension study, which was supplemented at later time points by investigators, including health-related quality of life assessed through a clinician-reported questionnaire...
April 9, 2024: Blood Advances
#26
JOURNAL ARTICLE
Johannes Lübke, Alicia Schmid, Deborah Christen, Hanneke N G Oude Elberink, Lambert F R Span, Marek Niedoszytko, Aleksandra Górska, Magdalena Lange, Karoline V Gleixner, Emir Hadzijusufovic, Alex Stefan, Irena Angelova-Fischer, Roberta Zanotti, Massimiliano Bonifacio, Patrizia Bonadonna, Khalid Shoumariyeh, Nikolas von Bubnoff, Sabine Müller, Cecelia Perkins, Chiara Elena, Luca Malcovati, Hans G Hagglund, Mattias Mattsson, Roberta Parente, Judit Varkonyi, Anna Belloni Fortina, Francesca Caroppo, Knut Brockow, Alexander Zink, Christine Breynaert, Toon Ieven, Akif Selim Yavuz, Michael Doubek, Vito Sabato, Tanja Schug, Karin Hartmann, Massimo Triggiani, Jason Gotlib, Olivier Hermine, Michel Arock, Hanneke C Kluin-Nelemans, Jens Peter Panse, Wolfgang R Sperr, Peter Valent, Andreas Reiter, Juliana Schwaab
Certain laboratory abnormalities correlate with subvariants of systemic mastocytosis (SM) and are often prognostically relevant. To assess the diagnostic and prognostic value of individual serum chemistry parameters in SM, 2607 patients enrolled within the European Competence Network on Mastocytosis (ECNM) and 575 patients enrolled within the German Registry on Eosinophils and Mast Cells (GREM) were analyzed. For screening and diagnosis of SM, tryptase was identified as the most specific serum parameter. For differentiation between indolent and advanced SM (AdvSM), the following serum parameters were most relevant: tryptase, alkaline phosphatase (AP), ß2-microglobulin, lactate dehydrogenase (LDH), albumin, vitamin B12, and C-reactive protein (P<0...
April 9, 2024: Blood Advances
#27
JOURNAL ARTICLE
Henning Nilius, Hind Hamzeh-Cognasse, Janna Hastings, Jan-Dirk Studt, Dimitrios A Tsakiris, Andreas Greinacher, Adriana Mendez, Adrian Emanuel Schmidt, Walter A Wuillemin, Bernhard Gerber, Prakash Vishnu, Lukas Graf, Johanna A Kremer Hovinga, Tamam Bakchoul, Fabrice Cognasse, Michael Nagler
New analytical techniques can assess hundreds of proteins simultaneously with high sensitivity, facilitating the observation of their complex interplay and role in disease mechanisms. We hypothesized that proteomic profiling targeting proteins involved in thrombus formation, inflammation, and the immune response would identify potentially new biomarkers for heparin-induced thrombocytopenia (HIT). Four existing panels of the Olink proximity extension assay covering 356 proteins involved in thrombus formation, inflammation, and immune response were applied to randomly selected patients with suspected HIT (confirmed HIT, n=32; HIT ruled-out, n=38; positive heparin/PF4 [H/PF4] antibodies, n=28)...
April 8, 2024: Blood Advances
#28
JOURNAL ARTICLE
Deniz Yildirim Dogan, Eugen Ioan Urzica, Isabelle Hornung, Philipp Kastl, David Oguama, Franca Melanie Fette, Lien Huong Nguyen, Frank Rosenbauer, Kai Zacharowski, Ursula Klingmüller, Elise Gradhand, Andreas von Knethen, Rüdiger Popp, Ingrid Fleming, Lisa Schrader, Andrea Ulrike Steinbicker
Hemojuvelin (HJV) is a GPI-anchored protein of the repulsive guidance molecule (RGM) family acting as a bone morphogenetic protein (BMP) co-receptor to induce the hepatic iron regulatory protein hepcidin. Hepcidin causes ubiquitination and degradation of the sole known iron exporter ferroportin thereby limiting iron availability. The detailed signaling mechanism of HJV in vivo has yet to be investigated. In the current manuscript, we used an established model of adeno-associated virus (AAV) mediated liver-specific overexpression of HJV in murine models of hepatocyte-specific deficiency of the BMP type I receptors Alk2 or Alk3...
April 8, 2024: Blood Advances
#29
JOURNAL ARTICLE
Guy Ledergor, Zenghua Fan, Kai Wu, Elizabeth McCarthy, Axel Hyrenius-Wittsten, Alec Starzinski, Hewitt Chang, Mark Bridge, Serena S Kwek, Alexander Cheung, Sophia Anna Bylsma, Erik Hansen, Jeffrey Lee Wolf, Sandy W Wong, Nina Shah, Kole T Roybal, Thomas G Martin, Chun Jimmie Ye, Lawrence Fong
Multiple myeloma is characterized by frequent clinical relapses following conventional therapy. Recently, chimeric antigen receptor T (CAR-T) cells targeting B-cell maturation antigen (BCMA) has been established as a treatment option for patients with relapsed or refractory disease. However, while >70% of patients initially respond to this treatment, clinical relapse and disease progression occur in most cases. Recent studies showed persistent expression of BCMA at the time of relapse, indicating that immune intrinsic mechanisms may contribute to this resistance...
April 4, 2024: Blood Advances
#30
JOURNAL ARTICLE
Jeremy Jacobs, Garrett S Booth, Laura D Stephens, Brian D Adkins, Amarilis A Martin, George D Lundberg, Raeshun Trey Glover, Shazia S Khan, Julie K Silver, Jennifer S Woo
No abstract text is available yet for this article.
April 3, 2024: Blood Advances
#31
JOURNAL ARTICLE
Shanshan Guo, Wen Lei, Xueli Jin, Hui Liu, James Q Wang, Wenhai Deng, Wenbin Qian
Chimeric antigen receptor (CAR)-NK cells can eliminate tumors not only through the ability of the CAR molecule to recognize antigen expressed cancer cells but also through NK cell receptors themselves. This overcomes some of the limitations of CAR-T cells, paving CAR-NK cells for safer and more effective off-the-shelf cellular therapy. In this study, CD70, a pan-target of lymphoma, specific fourth-generation CAR with 4-1BB co-stimulatory domain and IL-15 was constructed and transduced into cord blood-derived NK cells by Baboon envelope pseudotyped lenti-vector...
April 2, 2024: Blood Advances
#32
JOURNAL ARTICLE
Madhav V Dhodapkar
Newer immune-based approaches based on recruitment and redirection of endogenous and/or synthetic immunity such as chimeric antigen-receptor-T (CAR-T) cells or bispecific antibodies are transforming the clinical management of multiple myeloma (MM). Contributions of the immune system to the anti-tumor effects of myeloma therapies are also increasingly appreciated. Clinical malignancy in MM originates in the setting of systemic immune alterations that begin early in myelomagenesis and regional changes in immunity impacted by spatial contexture...
April 2, 2024: Blood Advances
#33
JOURNAL ARTICLE
Marissa M Li, Anmol Baranwal, Mark Gurney, Syed Naseem Shah, Aref Al-Kali, Hassan B Alkhateeb, James M Foran, Cecilia Arana-Yi, Laura Ongie, Dong Chen, Abhishek A Mangaonkar, Kristen McCullough, Ayalew Tefferi, Terra L Lasho, Christy M Finke, Mrinal M Patnaik, Mithun Vinod Shah
Clonal cytopenia of undetermined significance (CCUS) is defined by a myeloid driver mutation in the context of otherwise unexplained cytopenia. CCUS has an inherent risk of progressing to myeloid neoplasm. However, it is unknown how exposure to previous cytotoxic therapy may impact the risk of progression and survival. We stratified CCUS patients by prior exposure to DNA-damaging therapy. Of 151 patients, 46 (30%) had received cytotoxic therapy and were classified as therapy-related CCUS (t-CCUS), whereas 105 (70%) had de novo CCUS...
April 2, 2024: Blood Advances
#34
JOURNAL ARTICLE
Frank M Horling, Birgit M Reipert, Peter Allacher, Werner Engl, Luying Pan, Srilatha D Tangada
Rurioctocog alfa pegol is an extended half-life full-length recombinant factor VIII (FVIII) bound to 20 kDa polyethylene glycol (PEG) that has been shown to be well tolerated and efficacious in the treatment and prevention of bleeding events in previously treated patients with severe hemophilia A. Here, we present a comprehensive analysis of immunogenicity data collected during 6 clinical studies of rurioctocog alfa pegol including a total of 360 unique previously treated patients with severe hemophilia A. The analysis included treatment-emerging FVIII neutralizing antibodies (FVIII inhibitors), pre-existing and treatment-emerging antibodies binding to FVIII, PEG-FVIII, or PEG, and treatment-emerging antibodies binding to Chinese hamster ovary host cell proteins...
April 2, 2024: Blood Advances
#35
JOURNAL ARTICLE
Joelle Abdallah, Robert George Williams, Hussein Awada, Ganesh Raman, Yusuf M Ozcan, Mark David Orland, Mutlu Mete, Weina Chen, Carmelo Gurnari, Jaroslaw P Maciejewski, Taha Bat
No abstract text is available yet for this article.
April 2, 2024: Blood Advances
#36
JOURNAL ARTICLE
Minghao Li, Liping Wang, Aijing Li, Bo Wang, Xiaohong Yang, Yue Zhang, Chaoqiong Chen, Futing Sun, Ziyan Zhu, Luyi Ye
Phenotype D-- is associated with severe hemolytic transfusion reactions and hemolytic disease of the fetus and newborn. It is typically caused by defective RHCE genes. In this study, we identified a D-- phenotype proband and verified RH phenotypes of other six family members. However, inconsistent results between the phenotypic analysis and Sanger sequencing revealed intact RHCE exons with no mutations in the D-- proband, but the protein was not expressed. Subsequent Oxford Nanopore Technologies whole-genome sequencing of the proband revealed an inversion with ambiguous breakpoints in the intron 2 and intron 7 and copy number variation loss in the RHCE gene region...
March 29, 2024: Blood Advances
#37
JOURNAL ARTICLE
Satheesh Chonat, Alexander Kulagin, Alexey Maschan, Marije Bartels, Jochen Buechner, Rowena Punzalan, Michael Richards, Masayo Ogawa, Eden Hicks, Ji Yu, André Baruchel, Austin G Kulasekararaj
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disease of uncontrolled terminal complement activation leading to intravascular hemolysis, thrombotic events, increased morbidity and mortality. This phase 3, open-label, single-arm multicenter study (NCT03406507) evaluated ravulizumab treatment in eculizumab-naive or -experienced pediatric patients (aged <18 years) with PNH over a 26-week primary evaluation period (PEP) and 4-year extension period (EP). Patients included in the study received weight based intravenous ravulizumab dosing...
March 29, 2024: Blood Advances
#38
JOURNAL ARTICLE
Aaron F J Iding, Ghadir Alkarithi, Hugo Ten Cate, Robert A S Ariëns, Arina J Ten Cate-Hoek
Ultrasound-accelerated catheter-directed thrombolysis (UA-CDT) to improve patency after deep vein thrombosis (DVT) has not conclusively been shown to prevent post-thrombotic syndrome (PTS) but might benefit patients who are unlikely to obtain patency with standard treatment. We hypothesized that these patients could be selected based on their fibrin clot properties. To study this, patients with acute iliofemoral DVT from the CAVA trial had blood samples taken at inclusion. Fibrin clot properties in plasma were determined by turbidimetric clotting (lag time and maximal turbidity) and lysis assays (time to 50% lysis and lysis rate), permeation assay (Ks), and confocal microscopy (fiber density), as well as levels of fibrin clot modifiers fibrinogen and C-reactive protein (CRP)...
March 28, 2024: Blood Advances
#39
JOURNAL ARTICLE
Stacey Fedewa, Leonard A Valentino, Andee Koo, Lorraine Cafuir, Duc Quang Tran, Ana G Antun, Christine L Kempton
Racial and ethnic representativeness in clinical trials is crucial to mitigate outcomes disparities, however, diversity among hemophilia trials is unknown. The aim of this study is to examine the reporting and representation of race and ethnicity in trials of people with hemophilia (PwH). In this cross-sectional study, the clinicaltrials.gov database was queried in April 2023 for interventional clinical trials involving PwH between 2007-2022. The distribution of participants (observed) was compared with expected proportions based on United States (US) hemophilia treatment center (HTC) and country-specific census data with observed-to-expected ratios (OER)...
March 28, 2024: Blood Advances
#40
JOURNAL ARTICLE
Jennifer H Han, Brandi Dupervil, Arash Mahajerin, Roshni Kulkarni, Marilyn Jean Manco-Johnson, Courtney Dawn Thornburg
Infants and toddlers (ITs) with hemophilia have unique bleeding features. Factor prophylaxis has been shown to decrease the risk of intracranial hemorrhage (ICH), which supports recommendations to begin at a young age. Clinical and demographic characteristics were analyzed for 883 ITs ≤2 years old with hemophilia A and B, seen at US Hemophilia Treatment Centers and enrolled in the Community Counts Registry, a surveillance program of the Centers for Disease Control and Prevention (CDC). ICH in the first two years of life was seen in 8% of ITs, of whom 8 (12%) were on continuous prophylaxis at the time of ICH...
March 28, 2024: Blood Advances
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