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European Journal of Haematology

Lene Kongsgaard Nielsen, Tobias Wirenfeldt Klausen, Mary Jarden, Henrik Frederiksen, Annette Juul Vangsted, Trung Do, Ida Bruun Kristensen, Ulf Christian Frølund, Christen Lykkegaard Andersen, Niels Abildgaard, Henrik Gregersen
OBJECTIVES: The Danish Myeloma Study Group initiated a randomized, placebo-controlled, double-blinded phase II study to investigate the efficacy of adding clarithromycin to cyclophosphamide-bortezomib-dexamethasone (VCD) induction therapy in transplant eligible, newly diagnosed multiple myeloma patients. The study was prematurely terminated due to severe complications, and no effect of adding clarithromycin was found. The aim of this study was to compare health-related quality of life (HRQoL) between the two groups and to explore the coherence hereof with adverse event (AE) registration by clinicians...
September 19, 2018: European Journal of Haematology
Nadege Wesner, Louis Drevon, Alexis Guedon, Jean Baptiste Fraison, Salim Trad, Jean Emmanuel Kahn, Achille Aouba, Jerome Gillard, Matthieu Ponsoye, Thomas Hanslik, Clement Gourguechon, Eric Liozon, Kamel Laribi, Julien Rossignol, Olivier Hermine, Lionel Adès, Fabrice Carrat, Pierre Fenaux, Arsene Mekinian, Olivier Fain
OBJECTIVE: We report cases of myelodysplastic syndrome/myeloproliferative neoplasms (MDS/MPN) with trisomy 8 associated with inflammatory and autoimmune diseases (IADs). METHOD: Data for 21 patients with trisomy 8-MDS/MPN and IADs were analyzed and compared to 103 patients with trisomy 8-MDS/MPN without IADs. RESULTS: The median age of MDS/MPN patients with IADs was 67 [59-80]. The IADs were Behçet's-like disease in 11 (52%) patients, inflammatory arthritis in 4 (19%) and Sjögren's syndrome, autoimmune hemolytic anemia, aseptic abscess, periarteritis nodosa, Sweet's syndrome and unclassified vasculitis in one patient each...
September 15, 2018: European Journal of Haematology
Samir K Ballas, Philippe Connes
OBJECTIVE: Determine the effect of foetal haemoglobin (HbF) and α-thalassaemia on red blood cell (RBC) deformability of patients with sickle cell anaemia (SCA) with and without hydroxyurea (HU). METHODS: Adult patients were enrolled in the Sickle Cell Program of the Cardeza Foundation (Thomas Jefferson University) and were followed-up prospectively during the period in which the Multicenter Study of Hydroxyurea (MSH) in patients with SCA was conducted. Ninety-one patients did not receive HU, 20 patients were enrolled in MSH and 10 patients were enrolled in an open label study of HU in SCA...
September 11, 2018: European Journal of Haematology
Sabine Kayser, Mark J Levis
The recently updated World Health Organization Classification (WHO) of myeloid neoplasms and leukemia reflects the fact that research in the underlying pathogenic mechanisms of acute myeloid leukemia (AML) has led to remarkable advances in our understanding of the disease. Gene mutations now allow us to explore the enormous diversity among cytogenetically defined subsets of AML, particularly the large subset of cytogenetically normal AML. Despite the progress in unravelling the tumor genome, only a small number of recurrent mutations have been incorporated into risk-stratification schemes and have been proven to be clinically relevant, targetable lesions...
September 10, 2018: European Journal of Haematology
Elise Larson, David M German, Joseph Shatzel, Thomas G DeLoughery
Anticoagulation has multiple roles in the treatment of cardiovascular disease, including in management of acute myocardial infarction, during percutaneous coronary intervention, as stroke prophylaxis in patients with atrial arrhythmias, and in patients with mechanical heart valves. Clinical anticoagulation choices in the aforementioned diseases vary widely, due to conflicting data to support established agents and the rapid evolution of evidence-based practice that parallels more widespread use of novel oral anticoagulants...
September 10, 2018: European Journal of Haematology
Katharina Kriegsmann, Mark Kriegsmann, Michael von Bergwelt-Baildon, Martin Cremer, Mathias Witzens-Harig
Low levels of peripheral blood natural killer T (NKT) cells in cancer patients and a favorable outcome associated with a high number of tumor-infiltrating NKT cells demonstrated in several studies indicated the important role of these immune cells in the antitumor response. With effective antitumor immunity via direct tumor lysis, cytokine modulation of effector cells and regulation of immunosuppressive cells, type I NKT cells display interesting features/properties for the rapidly developing chimeric antigen receptor (CAR) technology...
September 6, 2018: European Journal of Haematology
Andrea Jarisch, Emilia Salzmann-Manrique, Holger Cario, Regine Grosse, Jan Soerensen, Roland Fischer, Ansgar Schulz, Renate Hammerstingl, Arthur Wunderlich, Peter Bader
OBJECTIVE: Iron overload (IO) in transfusion-dependent anemia persists after hematopoietic stem cell transplantation (HSCT) and can cause long-term organ damage. In many studies, the diagnosis of IO before and after HSCT is based on serum ferritin (SF) levels rather than on assessment of liver iron concentration (LIC) by MRI or SQUID. METHOD: In a multicenter, retrospective study we analyzed the concordance for indication of iron depletion therapy and correlation between LIC and SF of 36 thalassemia patients after HSCT...
September 6, 2018: European Journal of Haematology
Tyler W Buckner, Robert Sidonio, Christine Guelcher, Craig M Kessler, Michelle Witkop, David Clark, Wendy Owens, Moshe Fridman, Neeraj N Iyer, David L Cooper
OBJECTIVE: To assess the reliability and validity of 6 patient-reported outcomes (PRO) instruments for evaluating health-related quality of life in adults with mild-severe hemophilia B and caregivers of children with hemophilia B, including affected women/girls. METHODS: Adults with hemophilia B and caregivers completed separate online surveys containing several PRO instruments, which were administered to adult participants only (EQ-5D-5L, Brief Pain Inventory v2 Short Form, Hemophilia Activities List, and International Physical Activities Questionnaire), both adults and caregivers (Patient Health Questionnaire [PHQ-9]), or caregivers only (Generalized Anxiety Disorder 7-Item [GAD-7] scale)...
September 4, 2018: European Journal of Haematology
Marie Balsat, Madeleine Etienne, Mohamed Elhamri, Sandrine Hayette, Gilles Salles, Xavier Thomas
BACKGROUND: Management of pregnant patients with BCR-ABL-positive leukemia is challenging. Managing a patient who has been diagnosed while pregnant requires a different approach as compared with a patient who plans to become pregnant while on the treatment with tyrosine kinase inhibitor (TKI). Interferon (IFN)-alpha is a useful option in both situations due to teratogenic potential of TKIs. METHODS: We presented a series of 12 successful pregnancies in 11 women with BCR-ABL-positive leukemia, whose leukemia was managed with IFN-alpha throughout their pregnancy...
September 4, 2018: European Journal of Haematology
T Devos, S Meers, N Boeckx, A Gothot, D Deeren, B Chatelain, C Chatelain, B Devalet
Despite its considerable morbidity and mortality, paroxysmal nocturnal haemoglobinuria (PNH) is still underdiagnosed. Patients with PNH can suffer from cardiovascular, gastro-intestinal, neurological or haematological symptoms and refer to several specialists. The aim of this paper is to review the diagnosis and the management of PNH patients, with the primary focus on identifying high-risk groups. Additionally, the implementation and prognostic value of the defined high-risk groups will be commented on and the management of PNH patients is discussed from a Belgian perspective...
September 1, 2018: European Journal of Haematology
Matthew O'Donnell, Joseph J Shatzel, Sven R Olson, Molly M Daughety, Khanh P Nguyen, Justine Hum, Thomas G DeLoughery
While cardiovascular disease is common, occasionally hematologists and other practitioners will encounter patients with arterial thrombosis/infarction in unusual sites, without clear cause or obvious diagnostic and treatment paradigms. Contrary to the more commonly encountered cerebrovascular accident and cardiovascular disorders, the various infarctions outlined in this review have unique presentations, pathophysiology, workup, and treatments that all hematologists should be aware of. This review outlines the current literature on arterial thrombosis, with consideration given to anatomic sources and hypercoagulable associations, while focusing on the epidemiology, pathophysiology, provoking factors, and current recommended treatments for intracardiac thrombus, primary aortic mural thrombus, visceral infarctions, and cryptogenic limb ischemia to provide a useful and practical review for the practitioner...
August 21, 2018: European Journal of Haematology
D Robert Sutherland, Andrea J Illingworth, Liam Whitby, Iuri Marinov
In designing their 'screening assay', the authors have broken several well-established rules of good PNH flow assay design. Such rules require the loss of two GPI-linked structures (per WBC lineage tested) before a PNH phenotype can be unequivocally established. This is an important aspect of robust high-sensitivity flow assay design for PNH detection. It was also strongly recommended that granulocyte and monocyte lineages should be specifically delineated with appropriately-selected CD15 and CD64 conjugates respectively...
August 20, 2018: European Journal of Haematology
Noa Lavi, Doaa Okasha, Edmond Sabo, Ilana Oren, Noam Benyamini, Haggai Bar-Yoseph
Survival of multiple myeloma (MM) patients has improved with introduction of novel anti-myeloma agents. Myeloma has transformed into a chronic condition, accompanied with multiple relapses requiring salvage therapies, which results in cumulative immunosuppression. The impact of novel therapies on the immune system differs from that of traditional chemotherapy, and their application leads to emergence of infections not previously associated with myeloma, cytomegalovirus (CMV) infection being one of them(1). This article is protected by copyright...
August 18, 2018: European Journal of Haematology
Carmelina Calitri, Eliana Ruberto, Elio Castagnola
Antibiotics are frequently administered for prophylaxis of fever in neutropenic children with cancer. This strategy is mainly derived from adults' data, and various pediatric studies evidenced the effectiveness of antibiotics (e.g. fluorquinolones) in the prevention of febrile neutropenia. However, only two pediatric randomized, double blind, placebo-controlled trials have been performed, with a total of 262 leukemic children enrolled, and no other one was ever powered for analyzing effectiveness over other infectious complications...
August 14, 2018: European Journal of Haematology
Carina Intke, Sini Korpelainen, Sari Hämäläinen, Matti Vänskä, Irma Koivula, Esa Jantunen, Kari Pulkki, Auni Juutilainen
OBJECTIVE: The study aim was to compare the performance of interleukin-1 receptor antagonist (IL-1Ra) to C-reactive protein (CRP) and procalcitonin (PCT) in early prediction of the clinical course of febrile neutropenia. METHODS: The study population consisted of 86 consecutive patients with febrile neutropenia who received intensive chemotherapy for haematological malignancy between November 2009 and November 2012 at the adult haematology ward of Kuopio University Hospital...
August 12, 2018: European Journal of Haematology
J Wehmeyer, M Zaiss, C Losem, S Schmitz, B Niemeier, J Harde, C V Hannig, H-D Harich, J Müller, M Klausmann, H W Tessen, K Potthoff
OBJECTIVE: Azacitidine (Vidaza® ) is the standard treatment for patients with higher-risk MDS not eligible for allogeneic stem cell transplantation. In the non-interventional study PIAZA we evaluated the effectiveness and safety of azacitidine-treatment in 149 patients with higher-risk MDS, CMML and AML in routine clinical practice. METHOD: Patients were treated according to physician's discretion. Besides evaluation of safety and effectiveness, impact of covariates on PFS was assessed...
August 9, 2018: European Journal of Haematology
Alessandra Casonato, Eva Galletta, Viviana Daidone
Type 2M is a very heterogeneous form of von Willebrand disease (VWD) associated with impaired platelet and von Willebrand factor (VWF) interactions not due to a lack of large VWF multimers. OBJECTIVES: To investigate type 2M heterogeneity and to establish the most appropriate diagnostic flowchart. METHODS: Hemostatic and genetic VWF analyses were performed in 14 type 2M VWD patients carrying the p.G1324S, p.R1374H, p.R1374C, p.A1344_A1350del, or p.F1293L mutations...
August 6, 2018: European Journal of Haematology
Frederick W Thielen, Hedwig M Blommestein, Liesbeth E M Oosten, Friso G Calkoen, Arjan C Lankester, Jaap J Zwaginga, Katarina Le Blanc, Alba Redondo, Fermin Sánchez-Guijo, Mattia Algeri, Franco Locatelli, Wim E Fibbe, Carin A Uyl-de Groot
OBJECTIVE: No standard second-line treatment exists for acute graft-versus-host disease steroid-refractory (SR-aGvHD), and long-term outcomes remain poor. Mesenchymal stromal cells (MSCs) have been evaluated as treatment, but no disease model (DM) exists that integrates and extrapolates currently available evidence. The aim of this study was to develop such a DM to describe the natural history of SR-aGvHD and to predict long-term outcomes. METHOD: The DM was developed in collaboration with experts in haematology-oncology...
August 6, 2018: European Journal of Haematology
Tin Sing Lam, Marian van de Meent, Erik W A Marijt, J H Frederik Falkenburg, Inge Jedema
OBJECTIVES: The importance of autologous T-cell responses in immune surveillance against acute myeloid leukemia (AML) remains unclear. Therefore, we investigated the presence and functional reactivity of autoreactive T-cell responses against autologous AML blasts. METHODS: T cells purified from PB samples harvested from patients during first complete remission were stimulated with autologous AML material harvested at diagnosis. After 12-14 days of coculture, the T cells were restimulated with autologous AML cells, and leukemia-reactive T-cell clones were isolated based on their expression of the activation marker CD137...
August 4, 2018: European Journal of Haematology
Hervé Lobbes, Vincent Grobost, Richard Lemal, Virginie Rieu, Guillaume Le Guenno, Marc Ruivard
Treatment of non-IgM type I cryoglobulinaemic vasculitis (CV) is challenging. Corticosteroids are first-line therapy, but relapses are frequent leading to therapeutic escalation. Bortezomib is a proteasome inhibitor with rapid effect on monoclonal component. However, its use in non-IgM type I CV has been barely reported. OBJECTIVE: to assess the efficacy of bortezomib in non-IgM type I CV. METHOD: single centre case series of 4 patients with non-IgM type I CV treated with bortezomib monotherapy...
July 30, 2018: European Journal of Haematology
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