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British Journal of Haematology

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https://www.readbyqxmd.com/read/29774521/patient-reported-outcomes-of-multiple-myeloma-patients-treated-with-panobinostat-after-%C3%A2-2-lines-of-therapy-based-on-the-international-phase-3-randomized-double-blind-placebo-controlled-panorama-1-trial
#1
Paul G Richardson, Robert L Schlossman, Anuja N Roy, Ashok Panneerselvam, Suddhasatta Acharyya, Monika Sopala, Sagar Lonial
The phase 3 PANORAMA-1 trial led to regulatory approvals of panobinostat (PAN) in combination with bortezomib (BTZ) and dexamethasone (DEX) for the treatment of multiple myeloma after ≥2 prior regimens, including BTZ and an immunomodulatory drug. Patient-reported outcomes (PROs) were assessed in PANORAMA-1, with data available for 73 patients in the PAN + BTZ + DEX arm and 74 patients in the placebo (PBO) + BTZ + DEX arm. Per the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30), global health status/quality of life (QoL) scores initially declined with PAN + BTZ + DEX during the first 24 weeks before approaching baseline scores and remaining steady during the next 24 weeks, with no difference between arms at Week 48...
May 17, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767851/risk-factors-for-vitamin-d-deficiency-in-sickle-cell-disease
#2
Jin Han, Xu Zhang, Santosh L Saraf, Michel Gowhari, Robert E Molokie, Johara Hassan, Shivi Jain, Binal N Shah, Taimur Abbasi, Roberto F Machado, Victor R Gordeuk
Vitamin D deficiency (VDD), 25-OHD levels <20 ng/ml, is prevalent among patients with sickle cell disease (SCD) and is linked to acute and chronic pain and bone fracture in this population. There is limited literature regarding VDD-associated risk factors for SCD. We examined potential clinical and genomic parameters associated with VDD in 335 adults with SCD in a cross-sectional study. VDD was present in 65% of adult SCD patients, and 25-OHD levels independently and positively correlated with older age (P < 0·001) and vitamin D supplementation (P < 0·001)...
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767848/difficulty-distinguishing-essential-thrombocythaemia-from-polycythaemia-vera-in-children-with-jak2-v617f-positive-myeloproliferative-neoplasms
#3
LETTER
Nicole Kucine, Mustafa Al-Kawaaz, Daher Hajje, James Bussel, Attilio Orazi
No abstract text is available yet for this article.
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767845/final-results-from-a-defibrotide-treatment-ind-study-for-patients-with-hepatic-veno-occlusive-disease-sinusoidal-obstruction-syndrome
#4
Nancy A Kernan, Stephan Grupp, Angela R Smith, Sally Arai, Brandon Triplett, Joseph H Antin, Leslie Lehmann, Tsiporah Shore, Vincent T Ho, Nancy Bunin, Massimo Iacobelli, Wei Liang, Robin Hume, William Tappe, Robert Soiffer, Paul Richardson
Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life-threatening complication of haematopoietic stem cell transplant (HSCT) conditioning and chemotherapy. Defibrotide is approved for treatment of hepatic VOD/SOS with pulmonary or renal dysfunction [i.e., multi-organ dysfunction (MOD)] after HSCT in the United States and severe VOD/SOS after HSCT in patients aged older than 1 month in the European Union. Defibrotide was available as an investigational drug by an expanded-access treatment programme (T-IND; NCT00628498)...
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767844/comorbidities-associated-with-higher-von-willebrand-factor-vwf-levels-may-explain-the-age-related-increase-of-vwf-in-von-willebrand-disease
#5
Ferdows Atiq, Karina Meijer, Jeroen Eikenboom, Karin Fijnvandraat, Eveline P Mauser-Bunschoten, Karin P M van Galen, Marten R Nijziel, Paula F Ypma, Joke de Meris, Britta A P Laros-van Gorkom, Johanna G van der Bom, Moniek P de Maat, Marjon H Cnossen, Frank W G Leebeek
Some comorbidities, such as hypertension, are associated with higher von Willebrand factor (VWF) levels in the general population. No studies have been conducted to assess this association in patients with von Willebrand disease (VWD). Therefore, we studied this association in patients with type 1 (n = 333) and type 2 (n = 203) VWD from the 'WiN" study. VWF antigen (VWF:Ag) was higher in type 1 VWD patients with hypertension [difference: 0·23 iu/ml, 95% confidence interval (CI): 0·11-0·35], diabetes mellitus (0·11 iu/ml, 95% CI: -0·02 to 0·23), cancer (0·14 iu/ml, 95% CI: 0·03-0·25) and thyroid dysfunction (0·14 iu/ml, 95% CI: 0·03-0·26) than in patients without these comorbidities (all corrected for age, sex and blood group)...
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767843/how-i-treat-primary-haemophagocytic-lymphohistiocytosis
#6
REVIEW
Rebecca A Marsh, Elie Haddad
Primary haemophagocytic lymphohistiocytosis (HLH) diseases are a collection of inherited genetic disorders that cause the syndrome of HLH. Great advances have been made in the last 20 years with regard to the discovery of many of the genetic aetiologies of disease. Several advances have also been made on the clinical stage. Accurate screening diagnostics for primary HLH diseases that are superior to traditional Natural Killer cell function testing have been developed and are now available in many countries...
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767839/jak2-v617f-positive-acute-myeloid-leukaemia-aml-a-comparison-between-de-novo-aml-and-secondary-aml-transformed-from-an-underlying-myeloproliferative-neoplasm-a-study-from-the-bone-marrow-pathology-group
#7
Jason Aynardi, Rashmi Manur, Paul R Hess, Seble Chekol, Jennifer J D Morrissette, Daria Babushok, Elizabeth Hexner, Heesun J Rogers, Eric D Hsi, Elizabeth Margolskee, Attilio Orazi, Robert Hasserjian, Adam Bagg
The JAK2 V617F mutation is characteristic of most Philadelphia chromosome-negative myeloproliferative neoplasms (MPNs) and occurs rarely in de novo acute myeloid leukaemia (AML). We sought to characterize AMLs that harbour this mutation and distinguish those that arise de novo (AML-DN) from those that reflect transformation of an underlying MPN (AML-MPN). Forty-five patients with JAK2 V617F-mutated AML were identified; 15 were AML-DN and 30 were AML-MPN. AML-MPN cases were more likely to have splenomegaly (P = 0·02), MPN-like megakaryocytes and higher mean JAK2 V617F VAF at diagnosis (P = 0·04)...
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767837/romiplostim-for-the-management-of-perioperative-thrombocytopenia
#8
Hanny Al-Samkari, Ariela L Marshall, Katayoon Goodarzi, David J Kuter
Thrombocytopenic patients requiring invasive surgery have few options to improve their platelet count preoperatively. This is a single-centre, retrospective review of thrombocytopenic patients receiving the thrombopoietin receptor agonist romiplostim perioperatively to allow for surgical interventions. Patient characteristics, romiplostim use, and surgical and safety outcomes (bleeding, thrombosis, transfusions) were collected and analysed. Forty-seven patients underwent 51 surgical procedures (ranging from total hip arthroplasty to open cardiac surgery) with romiplostim support...
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767836/how-do-we-manage-iron-deficiency-after-blood-donation
#9
REVIEW
Joseph E Kiss, Ralph R Vassallo
Blood donors and the RBCs and other components they willingly provide are essential in the delivery of healthcare in all parts of the world. Nearly 70% of donated blood comes from repeat or committed donors. The amount of iron removed in the 10 min or so it takes to withdraw a unit of blood (500 ml, plus 25 ml for testing) requires over 24 weeks to replace on a "standard" diet, i.e., without added iron in the form of supplements The cumulative effect of repeat blood donations without adequate iron replacement or a longer wait between donations results in iron deficiency (ID) in many donors, low haemoglobin deferral (~8% of donation attempts), and frank anaemia in some...
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767474/mesenchymal-stromal-cells-from-shwachman-diamond-syndrome-patients-fail-to-recreate-a-bone-marrow-niche-in-vivo-and-exhibit-impaired-angiogenesis
#10
Donatella Bardelli, Erica Dander, Cristina Bugarin, Claudia Cappuzzello, Alice Pievani, Grazia Fazio, Paolo Pierani, Paola Corti, Piero Farruggia, Carlo Dufour, Simone Cesaro, Marco Cipolli, Andrea Biondi, Giovanna D'Amico
Shwachman-Diamond syndrome (SDS) is a rare multi-organ recessive disease mainly characterised by pancreatic insufficiency, skeletal defects, short stature and bone marrow failure (BMF). As in many other BMF syndromes, SDS patients are predisposed to develop a number of haematopoietic malignancies, particularly myelodysplastic syndrome and acute myeloid leukaemia. However, the mechanism of cancer predisposition in SDS patients is only partially understood. In light of the emerging role of mesenchymal stromal cells (MSCs) in the regulation of bone marrow homeostasis, we assessed the ability of MSCs derived from SDS patients (SDS-MSCs) to recreate a functional bone marrow niche, taking advantage of a murine heterotopic MSC transplant model...
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767454/guideline-for-the-management-of-mantle-cell-lymphoma
#11
LETTER
Pamela McKay, Mike Leach, Bob Jackson, Stephen Robinson, Simon Rule
No abstract text is available yet for this article.
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767447/expression-of-ribosomal-and-actin-network-proteins-and-immunochemotherapy-resistance-in-diffuse-large-b-cell-lymphoma-patients
#12
Susanne Bram Ednersson, Martin Stenson, Mimmie Stern, Gunilla Enblad, Henrik Fagman, Herman Nilsson-Ehle, Sverker Hasselblom, Per-Ola Andersson
Diffuse large B cell lymphoma (DLBCL) patients with early relapse or refractory disease have a very poor outcome. Immunochemotherapy resistance will probably, also in the era of targeted drugs, remain the major cause of treatment failure. We used proteomic mass spectrometry to analyse the global protein expression of micro-dissected formalin-fixed paraffin-embedded tumour tissues from 97 DLBCL patients: 44 with primary refractory disease or relapse within 1 year from diagnosis (REF/REL), and 53 who were progression-free more than 5 years after diagnosis (CURED)...
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767446/hydroxycarbamide-adherence-and-cumulative-dose-associated-with-hospital-readmission-in-sickle-cell-disease-a-6-year-population-based-cohort-study
#13
Jifang Zhou, Jin Han, Edith A Nutescu, Victor R Gordeuk, Santosh L Saraf, Gregory S Calip
Sickle cell disease (SCD) is a congenital haemoglobinopathy that causes frequent acute care/emergency room visits and hospital admissions for affected individuals. Evidence from population-based studies demonstrating the role of hydroxycarbamide (HC, also termed hydroxyurea) in reducing hospital readmission rates is limited. Our objective was to describe the use of HC and its association with acute care utilization and readmission rates using a large, nationally-representative US health insurance claims database over a 6-year period between 2009 and 2014...
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767440/successful-treatment-of-systemic-aa-amyloidosis-associated-with-underlying-hodgkin-lymphoma
#14
Richa Manwani, David Wrench, Ashutosh Wechalekar, Helen Lachmann
No abstract text is available yet for this article.
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767433/t-6-9-p23-q34-1-acute-myeloid-leukaemia-with-cup-like-blasts
#15
Claire Bories, Thomas Boyer
No abstract text is available yet for this article.
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767429/spectral-domain-optical-coherence-tomography-interpretation-response-to-blacklaws-et-al
#16
LETTER
Falko Fend, Sarah Coupland
No abstract text is available yet for this article.
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767427/azacitidine-in-the-real-world-an-evaluation-of-1101-higher-risk-myelodysplastic-syndrome-low-blast-count-acute-myeloid-leukaemia-patients-in-ontario-canada
#17
Lee Mozessohn, Matthew C Cheung, Saber Fallahpour, Tripat Gill, Asmaa Maloul, Liying Zhang, Olivia Lau, Rena Buckstein
The outcome of myelodysplastic syndrome (MDS) patients with uniformly higher-risk disease treated with azacitidine (AZA) in the 'real-world' remains largely unknown. We evaluated 1101 consecutive higher-risk MDS patients (International Prognostic Scoring System intermediate-2/high) and low-blast count acute myeloid leukaemia (AML; 21-30% blasts) patients treated in Ontario, Canada. By dosing schedule, 24·7% received AZA for seven consecutive days, 12·4% for six consecutive days and 62·9% by 5-2-2. Overall, median number of cycles was 6 (range 1-67) and 8 (range 6-14) when restricted to the 692 (63%) patients who received at least 4 cycles...
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767411/mek1-2-inhibition-by-binimetinib-is-effective-as-a-single-agent-and-potentiates-the-actions-of-venetoclax-and-abt-737-under-conditions-that-mimic-the-chronic-lymphocytic-leukaemia-cll-tumour-microenvironment
#18
Kyle Crassini, Yandong Shen, William S Stevenson, Richard Christopherson, Chris Ward, Stephen P Mulligan, O Giles Best
The survival and proliferation of chronic lymphocytic leukaemia (CLL) cells is driven by multiple signalling pathways, including those mediated by the B cell, Toll-like and chemokine receptors. Many of these pathways converge on the same signalling molecules, including those involved in the Raf-1/MEK/Erk1/2-MAPK pathway. We investigated the effects of the MEK1/2 (also termed MAP2K1/2) inhibitor, binimetinib, against CLL cells cultured under conditions that mimic aspects of the tumour microenvironment. Binimetinib blocked CLL cell survival induced by stroma-conditioned media and phorbol myristylate (PMA)...
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767410/combination-targeted-therapy-in-chronic-lymphocytic-leukaemia-can-pre-clinical-studies-translate-to-the-clinic
#19
EDITORIAL
Nikolaos Ioannou, Alan G Ramsay
No abstract text is available yet for this article.
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29767409/the-use-of-serum-free-light-chain-dimerization-patterns-assist-in-the-diagnosis-of-al-amyloidosis
#20
Moshe E Gatt, Batia Kaplan, Dean Yogev, Elana Slyusarevsky, Galina Pogrebijski, Sizilia Golderman, Olga Kukuy, Avi Livneh
The discrimination between benign and malignant forms of plasma cell dyscrasia (PCD) is often difficult. Free light chain monomer-dimer pattern analysis (FLC-MDPA) may assist in solving this dilemma and distinguish between AL amyloidosis and benign PCD. Serum samples of patients with AL amyloidosis and benign PCD were analysed in a blinded manner. Quantitative Western blotting was performed to estimate dimerization and clonality indices, and thereby determine the source of the tested samples, as derived either from benign or malignant PCD...
May 16, 2018: British Journal of Haematology
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