Read by QxMD icon Read


Myrna R Nahas, Robert J Soiffer, Haesook T Kim, Edwin Alyea, Jon Arnason, Robin Joyce, Joseph H Antin, Vincent Ho, Dina Stroopinsky, Shuli Li, James D Levine, Malgorzata McMasters, Salvia Jain, Ayad Hamdan, Dimitrios Tzachanis, Mary Paty Bryant, Emma Logan, Josie Bazemore, Jeremy Stewart, Amy Joyce, Susan Stephenson, Abigail Washington, Leandra Cole, Athalia Pyzer, Rebecca Karp Leaf, David Avigan, Jacalyn Rosenblatt
Steroid-refractory chronic graft-versus-host disease (SR-cGVHD) remains a major cause of morbidity and mortality following allogeneic transplantation. Innovative immunotherapeutic strategies are urgently needed for the treatment of SR-cGVHD. We conducted a phase I clinical trial to evaluate the safety, efficacy, and immune effects of abatacept, a novel immunomodulatory drug that acts as an inhibitor of T cell activation via co-stimulatory blockade, in the treatment of SR-cGVHD. The study followed a 3+3 design with two escalating abatacept doses: 3 mg/kg and 10 mg/kg, with an expansion cohort treated at 10 mg/kg...
March 16, 2018: Blood
Hirofumi Shibata, Takahiro Yasumi, Saeko Shimodera, Eitaro Hiejima, Kazushi Izawa, Tomoki Kawai, Ryutaro Shirakawa, Taizo Wada, Ryuta Nishikomori, Hisanori Horiuchi, Osamu Ohara, Eiichi Ishii, Toshio Heike
Familial hemophagocytic lymphohistiocytosis (FHL) is the major form of hereditary hemophagocytic lymphohistiocytosis (HLH); as such, it requires prompt and accurate diagnosis. We previously reported that FHL type 3 (FHL3) can be rapidly screened by detecting munc13-4 expression in platelets using flow cytometry; however, the reliability of the munc13-4 expression assay for FHL3 diagnosis is unclear. Regardless of the type of UNC13D mutation, all reported FHL3 cases examined for munc13-4 protein showed significantly reduced expression...
March 16, 2018: Blood
Rachael F Grace, Paola Bianchi, Eduard J van Beers, Stefan W Eber, Bertil Glader, Hassan M Yaish, Jenny M Despotovic, Jennifer A Rothman, Mukta Sharma, Melissa M McNaull, Elisa Fermo, Kimberly Lezon-Geyda, D Holmes Morton, Ellis J Neufeld, Satheesh Chonat, Nina Kollmar, Christine M Knoll, Kevin Kuo, Janet L Kwiatkowski, Dagmar Pospíŝilová, Yves D Pastore, Alexis A Thompson, Peter E Newburger, Yaddanapudi Ravindranath, Winfred C Wang, Marcin W Wlodarski, Heng Wang, Susanne Holzhauer, Vicky R Breakey, Joachim Kunz, Sujit Sheth, Melissa J Rose, Heather A Bradeen, Nolan Neu, Dongjing Guo, Hasan Al-Sayegh, Wendy B London, Patrick G Gallagher, Alberto Zanella, Wilma Barcellini
An international, multicenter registry was established to collect retrospective and prospective clinical data on patients with pyruvate kinase (PK) deficiency, the most common glycolytic defect causing congenital non-spherocytic hemolytic anemia. Medical history and laboratory and radiologic data were retrospectively collected at enrollment in 254 patients with molecularly confirmed PK deficiency. Perinatal complications were common, including anemia requiring transfusions, hyperbilirubinemia, hydrops, and prematurity...
March 16, 2018: Blood
Sabrina Giammarco, Régis Peffault de Latour, Simona Sica, Carlo Dufour, Gerard Socie, Jakob Passweg, Nicolaus Kröger, Eefke Petersen, Maria Teresa Van Lint, Rosi Oneto, Alessio Signori, Andrea Bacigalupo
No abstract text is available yet for this article.
March 16, 2018: Blood
Hannah Major-Monfried, Anne S Renteria, Attaphol Pawarode, Pavan Reddy, Francis Ayuk, Ernst Holler, Yvonne A Efebera, William J Hogan, Matthias Wölfl, Muna Qayed, Elizabeth O Hexner, Kitsada Wudhikarn, Rainer Ordemann, Rachel Young, Jay Shah, Matthew J Hartwell, Mohammed Chaudhry, Mina Aziz, Aaron Etra, Gregory A Yanik, Nicolaus Kröger, Daniela Weber, Yi-Bin Chen, Ryotaro Nakamura, Wolf Rösler, Carrie L Kitko, Andrew C Harris, Michael Pulsipher, Ran Reshef, Steven Kowalyk, George Morales, Ivan Torres, Umut Özbek, James L M Ferrara, John E Levine
Acute graft versus host disease (GVHD) is treated with systemic corticosteroid immunosuppression. Clinical response after one week of therapy often guides further treatment decisions, but long term outcomes vary widely between centers and more accurate predictive tests are urgently needed. We analyzed clinical data and blood samples taken after one week of systemic treatment for GVHD from 507 patients from 17 centers of the Mount Sinai Acute GVHD International Consortium (MAGIC), dividing them into test (n=236) and two validation cohorts separated in time (n = 142 and 129, respectively)...
March 15, 2018: Blood
Weicheng Ren, Xiaofei Ye, Hong Su, Wei Li, Dongbing Liu, Mohammad Pirmoradian, Xianhuo Wang, Bo Zhang, Qiang Zhang, Longyun Chen, Man Nie, Yao Liu, Bin Meng, Huiqiang Huang, Wenqi Jiang, Yixin Zeng, Wenyu Li, Kui Wu, Yong Hou, Klas G Wiman, Zhiming Li, Huilai Zhang, Roujun Peng, Shida Zhu, Qiang Pan-Hammarström
Hepatitis B virus (HBV) infection is endemic in some parts of Asia, Africa and South America and remains to be a significant public health problem in these areas. It is known as a leading risk factor for the development of hepatocellular carcinoma, but epidemiological studies have also shown that the infection may increase the incidence of several types B-cell lymphoma. Here, by characterizing altogether 275 Chinese diffuse large B-cell lymphoma (DLBCL) patients, we showed that patients with concomitant HBV infection (surface antigen positive, HBsAg+ ) are characterized by a younger age, a more advanced disease stage at diagnosis and a reduced overall survival...
March 15, 2018: Blood
Anat Rabinovich, Susan R Kahn
The post-thrombotic syndrome (PTS) is a chronic complication of deep vein thrombosis (DVT) that imposes significant morbidity, reduces quality of life and is costly. After DVT, 20-50% of patients will develop PTS, and up to 5% will develop severe PTS. The principal risk factors for PTS are anatomically extensive DVT, recurrent ipsilateral DVT, obesity and older age. By preventing the initial DVT and DVT recurrence, primary and secondary prophylaxis of DVT will reduce the occurrence of PTS. The effectiveness of elastic compression stockings (ECS) for PTS prevention is controversial...
March 15, 2018: Blood
Michael Hallek, Bruce D Cheson, Daniel Catovsky, Federico Caligaris-Cappio, Guillermo Dighiero, Hartmut Döhner, Peter Hillmen, Michael Keating, Emili Montserrat, Nicholas Chiorazzi, Stephan Stilgenbauer, Kanti R Rai, John C Byrd, Barbara Eichhorst, Susan O'Brien, Tadeusz Robak, John F Seymour, Thomas J Kipps
In 2008, International Workshop on CLL (iwCLL) published consensus guidelines for the design and conduct of clinical trials for patients with chronic lymphocytic leukemia (CLL) that were revised from those previously published by the National Cancer Institute-sponsored Working Group. These guidelines provided definitions intended to standardize the assessment of patients that were adopted by the Food and Drug Administration (FDA) and European Medicines Agency (EMA) for the evaluation of new drugs. Since the publication of these guidelines there have been major advances in the biology and treatment of patients with CLL, prompting the iwCLL to evaluate and revise the 2008 criteria...
March 14, 2018: Blood
Genki Yamato, Norio Shiba, Kenichi Yoshida, Yusuke Hara, Yuichi Shiraishi, Kentaro Ohki, Jun Okubo, Myoung-Ja Park, Manabu Sotomatsu, Hirokazu Arakawa, Nobutaka Kiyokawa, Daisuke Tomizawa, Souichi Adachi, Takashi Taga, Keizo Horibe, Satoru Miyano, Seishi Ogawa, Yasuhide Hayashi
No abstract text is available yet for this article.
March 14, 2018: Blood
Yongxia Wu, Steven Schutt, Katelyn Paz, Mengmeng Zhang, Ryan P Flynn, David Bastian, M Hanief Sofi, Hung Nguyen, Min Dai, Chen Liu, Ying-Jun Chang, Bruce R Blazar, Xue-Zhong Yu
Chronic graft-versus-host disease (cGVHD) is characterized as autoimmune-like fibrosis and antibody production mediated by pathogenic T-cells and B-cells. MicroRNA (miR)-17-92 influences the survival, differentiation and function of lymphocytes in cancer, infections and autoimmunity. To determine whether miR-17-92 regulates T- and B-cell responses in cGVHD, we generated mice conditionally deficient for miR-17-92 in T-cells, B-cells, or both. Using murine models of allogeneic bone marrow transplantation (allo-BMT), we demonstrate that expression of miR-17-92 in donor T- and B-cells is essential for the induction of both scleroderma and bronchiolitis obliterans in cGVHD...
March 12, 2018: Blood
Carla Casu, Elizabeta Nemeth, Stefano Rivella
Hepcidin agonists are a new class of compounds that regulate blood iron levels and limit iron absorption, and could improve the treatment of hemochromatosis, β-thalassemia, polycythemia vera, and other disorders where disrupted iron homeostasis causes disease or contributes to it. Hepcidin agonists also have the potential to prevent severe complications of siderophilic infections in patients with iron overload or chronic liver disease. This review highlights the preclinical studies that support the development of hepcidin agonists for the treatment of these disorders...
March 9, 2018: Blood
Chiara Antoniani, Vasco Meneghini, Annalisa Lattanzi, Tristan Felix, Oriana Romano, Elisa Magrin, Leslie Weber, Giulia Pavani, Sara El Hoss, Ryo Kurita, Yukio Nakamura, Thomas J Cradick, Ante S Lundberg, Matthew Porteus, Mario Amendola, Wassim El Nemer, Marina Cavazzana, Fulvio Mavilio, Annarita Miccio
Naturally occurring, large deletions in the β-globin locus result in hereditary persistence of fetal hemoglobin, a condition that mitigates the clinical severity of sickle-cell disease (SCD) and β-thalassemia. We designed a CRISPR/Cas9 strategy to disrupt a 13.6-kb genomic region encompassing the δ- and β-globin genes and a putative γ-δ intergenic fetal hemoglobin (HbF) silencer. Disruption of just the putative HbF silencer results in a mild increase in γ-globin expression, whereas deletion or inversion of a 13...
March 8, 2018: Blood
Xiaohong Ruby Xu, George M Yousef, Heyu Ni
Platelets have long been recognized as key players in hemostasis and thrombosis; however, growing evidence suggests that they are also significantly involved in cancer, the second leading cause of mortality worldwide. Preclinical and clinical studies showed that tumorigenesis and metastasis can be promoted by platelets through a wide variety of crosstalk between platelets and cancer cells. For example, cancer changes platelet behavior by directly inducing tumor-platelet aggregates, triggering platelet granule and extracellular vesicle release, altering platelet phenotype and platelet RNA profiles, and enhancing thrombopoiesis...
March 8, 2018: Blood
Yi Jin, Kenian Chen, Ayla De Paepe, Eva Hellqvist, Aleksandra D Krstic, Lauren Metang, Charlotte Gustafsson, Richard E Davis, Yair M Levy, Rakesh Surapaneni, Ann Wallblom, Hareth Nahi, Robert Mansson, Yin C Lin
Multiple myeloma (MM) is an aggressive cancer that originates from antibody-secreting plasma cells. While genetically and transcriptionally well characterized, the aberrant gene regulatory networks that underpin this disease remain poorly understood. Here, we mapped regulatory elements, open chromatin and transcription factor footprints in primary MM cells. In comparison to normal antibody-secreting cells, MM cells displayed consistent changes in enhancer activity that are connected to super-enhancer (SE)-mediated deregulation of transcription factor (TF) genes...
March 8, 2018: Blood
Jan C Peeken, Jonas S Jutzi, Julius Wehrle, Christoph Koellerer, Felix Staehle, Heiko Becker, Elias Schoenwandt, Thalia S Seeger, Daniel H Schanne, Monika Gothwal, Christopher J Ott, Albert Gründer, Heike L Pahl
The transcription factor "Nuclear Factor Erythroid 2" (NFE2) is overexpressed in the majority of patients with Myeloproliferative Neoplasms (MPN). In murine models, elevated NFE2 levels cause an MPN phenotype with spontaneous leukemic transformation. However, both the molecular mechanisms leading to NFE2 overexpression and its downstream targets remain incompletely understood. Here we show that the histone demethylase JMJD1C constitutes a novel NFE2 target gene. JMJD1C levels are significantly elevated in PV and PMF patients; concomitantly, global H3K9me1 and H3K9me2 levels are significantly decreased...
March 8, 2018: Blood
Anne M R Schrader, Patty M Jansen, Rein Willemze, Maarten H Vermeer, Anne-Marie Cleton-Jansen, Sebastiaan F Somers, J H Hendrik Veelken, Ronald van Eijk, Willem Kraan, Marie José Kersten, Michiel van den Brand, Wendy B C Stevens, Daphne de Jong, Myrurgia Abdul Hamid, Bea C Tanis, Eduardus F M Posthuma, Marcel Nijland, Arjan Diepstra, Steven T Pals, Arjen H G Cleven, Joost S P Vermaat
No abstract text is available yet for this article.
March 7, 2018: Blood
Stephanie C Casey, Virginie Baylot, Dean W Felsher
The MYC proto-oncogene is a gene product that coordinates the transcriptional regulation of a multitude of genes that are essential to cellular programs required for normal as well as neoplastic cellular growth and proliferation, including cell cycle, self-renewal, survival, cell growth, metabolism, protein and ribosomal biogenesis, and differentiation. Here we propose that MYC regulates these programs in a manner that is coordinated with a global influence on the host immune response. MYC had been presumed to contribute to tumorigenesis through tumor cell intrinsic influences...
March 7, 2018: Blood
Jianning Zhang, Fangyi Zhang, Jing-Fei Dong
Traumatic brain injury (TBI)-induced coagulopathy is a common and well-recognized risk for poor clinical outcomes, but its pathogenesis remains poorly understood and treatment options are limited and ineffective. We discuss the recent progress and knowledge gaps in understanding this lethal complication of TBI. We focus on (1) the disruption of the brain-blood barrier to disseminate brain injury systemically by releasing brain-derived molecules into the circulation and (2) TBI-induced hypercoagulable and hyperfibrinolytic states that result in persistent and delayed intracranial hemorrhage and systemic bleeding...
March 5, 2018: Blood
Michelle A Fanale, Steven M Horwitz, Andres Forero-Torres, Nancy L Bartlett, Ranjana H Advani, Barbara Pro, Robert W Chen, Andrew Davies, Tim Illidge, Mayur Uttarwar, Shih-Yuan Lee, Hong Ren, Dana A Kennedy, Andrei R Shustov
This phase 1 study evaluated frontline brentuximab vedotin in combination with cyclophosphamide, doxorubicin, and prednisone (BV+CHP; 6 cycles, then up to 10 cycles of brentuximab vedotin monotherapy) in 26 patients with CD30-positive peripheral T-cell lymphoma, including 19 with systemic anaplastic large cell lymphoma. All patients (100%) achieved an objective response, with a complete remission rate of 92%, and none received a consolidative stem cell transplant. After a median observation period of 59.6 months (range, 4...
March 5, 2018: Blood
Anja Mottok, Christian Steidl
Hodgkin lymphoma is considered a prime example of treatment success with cure rates exceeding 80% using modern combined modality therapies. However, especially in adolescents and young adults, treatment-related toxicity and long-term morbidity still represent persistent challenges. Moreover, outcomes in patients with relapsed or refractory disease remain unfavorable in the era of high-dose chemotherapy and stem cell transplantation. Hence, there is a high demand for novel and innovative alternative treatment approaches...
March 2, 2018: Blood
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"