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European Journal of Haematology

Marta González-Vicent, Marta Verna, Cécile Pochon, Aastha Chandak, Enrikas Vainorius, Tom Brundage, Essy Mozaffari, Garrett Nichols, Kanchan Rao
OBJECTIVE: Adenovirus (AdV) infections are potentially life-threatening for allogeneic hematopoietic cell transplant (allo-HCT) recipients. The AdVance study aimed to evaluate the incidence management and outcomes of AdV infections in European allo-HCT recipients. METHODS: As part of the study physician surveys were conducted to determine current AdV screening and treatment practices at their center. RESULTS: All of the 28 respondents who treat pediatric patients reported routine AdV screening practices with 93% screening all allo-HCT recipients and others screening those with transplant-related risk factors...
November 12, 2018: European Journal of Haematology
Rolf Ljung, Guenter Auerswald, Gary Benson, Gerry Dolan, Anne Duffy, Cedric Hermans, Victor Jiménez-Yuste, Thierry Lambert, Massimo Morfini, Silva Zupančić-Šalek, Elena Santagostino
The standard therapy for patients with haemophilia is prophylactic treatment with replacement factor VIII (FVIII) or factor IX (FIX). Patients who develop inhibitors against FVIII/FIX face an increased risk of bleeding, and the likelihood of early development of progressive arthropathy, alongside higher treatment-related costs. Bypassing agents can be used to prevent and control bleeding, as well as the recently-licensed prophylaxis, emicizumab, but their efficacy is less predictable than that of factor replacement therapy...
November 8, 2018: European Journal of Haematology
Elisabetta Abruzzese, Paolo de Fabritiis, Malgorzata M Trawinska, Pasquale Niscola, Jane F Apperley, Michael J Mauro
tyrosine kinase inhibitors (TKIs) for the treatment of chronic myeloid leukemia (CML) have revamped entirely the approach to this diagnosis. What once was a predictably fatal disease (with the exception of rare patients with long term success from interferon and those who could undergo and survive allogeneic stem cell transplant) is now a highly treatable condition, with 'functional cure' (sustained remission after TKI cessation) a possibility for many. For the patient with CML diagnosed in the chronic phase in the current era, initial discussions typically focus on risk stratification and optimized choice among several approved TKI options, with stem cell transplantation evaluation deferred and reserved for cases of advanced phase disease and sequential or highly selective TKI resistance...
November 7, 2018: European Journal of Haematology
Ichiro Yonese, Hiroshi Takase, Mayumi Yoshimori, Erika Onozawa, Akiho Tsuzura, Tohru Miki, Manabu Mochizuki, Osamu Miura, Ayako Arai
OBJECTIVE: Primary vitreoretinal lymphoma (PVRL) is a rare type of lymphoma wherein the lesions are limited to the eyes. PVRL is difficult to diagnose because of the challenges related to obtaining sufficient samples for biopsy. Moreover, PVRL has poor outcomes and often leads to the development of central nervous system (CNS) lesions during its course. Two studies recently reported that approximately 70%-80% of patients with vitreoretinal lymphoma have MYD88L265P , which is frequently mutated in primary CNS lymphoma (PCNSL)...
November 2, 2018: European Journal of Haematology
Prajwal Chaitanya Boddu, Tapan Mahendra Kadia
The application of next generation sequencing (NGS) has enhanced our understanding of the genetic landscape in acquired aplastic anemia (AA). Parallel progress has been in addressing aspects underlying immune dysregulation in disease pathogenesis. Novel insights into the molecular and biologic mechanisms have led to a shift in the paradigm of AA, from a solely autoimmune pathogenic concept toward its recognition as a multifaceted pathophysiology characterized by cytogenetic abnormalities, recurrent somatic mutations, telomere attrition, and immune dysregulation...
October 31, 2018: European Journal of Haematology
Georges E Tanios, Peter B Doley, Reinhold Munker
BACKGROUND: Immune checkpoint inhibitors (CPI) are widely used in modern oncology and have improved the prognosis of lung cancer, malignant melanoma and other malignancies. Unlike cytotoxic chemotherapy, drugs like nivolumab, pembrolizumab and ipilimumab are associated with immune-related adverse effects. We recently observed a patient with lung cancer who developed a fulminant warm-antibody autoimmune hemolytic anemia (AIHA). OBJECTIVES: Investigate the frequency and prognosis of AIHA secondary to CPI in a public database and review the literature...
October 22, 2018: European Journal of Haematology
Bastian von Tresckow, Cyrus Sayehli, Walter E Aulitzky, Maria-Elisabeth Goebeler, Matthias Schwab, Eunice Braz, Babett Krauss, Rolf Krauss, Frank Hermann, René Bartz, Andreas Engert
OBJECTIVES: Domatinostat (4SC-202) is a selective class I histone deacetylase inhibitor (HDACi). This phase I study investigated safety, tolerability, pharmacokinetics (PK), pharmacodynamics and anti-tumor activity in patients with advanced hematological malignancies. METHODS: Domatinostat was administered orally once (QD) or twice daily (BID) on days 1-14 with 7 days off or continuously days 1-21 in a 3+3 design at 7 dose levels from 25 to 400 mg total daily dose (TDD)...
October 22, 2018: European Journal of Haematology
Thomas Wolff, Holger Schulz, Christoph Losem, Dietmar Reichert, Hans-Jürgen Hurtz, Reiner Sandner, Johanna Harde, Sina Grebhardt, Karin Potthoff, Udo Mueller, Thomas Fietz
OBJECTIVE: The prospective non-interventional study (NIS) NADIR was designed to evaluate both effectiveness and safety of prophylactic use of lipegfilgrastim (Lonquex® ), a glycopegylated granulocyte colony stimulating factor, in cancer patients with different tumor entities undergoing chemotherapy in routine clinical practice. The primary objective was incidence of severe neutropenia, febrile neutropenia (FN) and neutropenia-associated complications. METHOD: NADIR was a national, multicenter, prospective NIS...
October 22, 2018: European Journal of Haematology
Reyad Dada, Binyam Usman
OBJECTIVES: Relapsed cHL patients after autologous hematopoietic stem cell transplantation (HSCT) with treatment sensitive disease are potential candidates for curative allogeneic HSCT. However, there are some concerns around performing such procedure after checkpoint inhibitors (CPIs). METHODS: We collected published data of patients undergoing allogeneic HSCT after treatment with CPIs (cohort 1). Abstracts of recent conferences (2015-2017) (ASCO, ASH, EBMT, ASBMT) were also included...
October 20, 2018: European Journal of Haematology
Christine G Kohn, Thomas J Bunz, Jan Beyer-Westendorf, Craig I Coleman
OBJECTIVES: To assess the association between rivaroxaban and warfarin and major bleeding risk in unprovoked venous thromboembolism (VTE) patients. METHODS: Using US MarketScan claims from 1/2012-12/2016, we identified patients who had ≥1 primary hospitalization/emergency department visit diagnosis code for an unprovoked VTE, newly-initiated on rivaroxaban or warfarin within 30-days after the VTE and ≥12-months of insurance coverage prior to the VTE. Differences in baseline covariates were adjusted using inverse probability-of-treatment weights based on propensity scores (residual absolute standardized differences <0...
October 16, 2018: European Journal of Haematology
Jacques Zimmer
The retrospective study by Chang et al. describes the characteristics of a cohort of 375 ITP patients. The first conclusion is that corticosteroids are efficient as a first-line option, which is not really a surprise. The paper then focuses on 2 second-line treatments, splenectomy and azathioprine. Whereas the former has long been the reference for chronic/refractory ITP (2), the latter is used much more rarely and is not even mentioned in a timely review of "old" ITP medications. This article is protected by copyright...
October 16, 2018: European Journal of Haematology
Sarah Bertoli, Etienne Paubelle, Emilie Bérard, Estelle Saland, Xavier Thomas, Suzanne Tavitian, Marie-Virginie Larcher, François Vergez, Audrey Sarry, Françoise Huguet, Clément Larrue, Claudie Bosc, Thomas Farge, Jean Emmanuel Sarry, Mauricette Michallet, Christian Récher
OBJECTIVES: We previously reported the prognostic value of serum ferritin in younger patients with intermediate-risk acute myeloid leukemia (AML). The aims of this study were to confirm this finding in a larger cohort regardless of age and prognostic subgroups, to explore the expression and functional role of ferritin in AML cells as well as the regulation of serum ferritin levels in AML patients. PATIENTS/MATERIALS/METHODS: Serum ferritin levels at diagnosis were collected in a cohort of 527 patients treated by intensive chemotherapy...
October 16, 2018: European Journal of Haematology
Bulent Antmen, Zeynep Karakaş, Mehmet Akif Yeşilipek, Osman Alphan Küpesiz, İlgen Şaşmaz, Vedat Uygun, Erdal Kurtoğlu, Gonul Oktay, Gonul Aydogan, Mehmet Akın, Zafer Salcioglu, Canan Vergin, Elif Güler Kazancı, Selma Ünal, Ümran Çalışkan, Yusuf Ziya Aral, Emine Türkkan, Adalet Meral Güneş, Bahattin Tunç, Fatma Gümrük, Aylin Canbolat Ayhan, Murat Söker, Ahmet Koç, Yeşim Oymak, Mehmet Ertem, Çetin Timur, Yıldız Yıldırmak, Gülersu İrken, Hilmi Apak, Betül Biner, Tuğba Gürleyen Eren, Yasemin Işık Balcı, Ülker Koçak, Gülsün Karasu, Diyar Akkaynak, Türkan Patıroğlu
OBJECTIVES: To evaluate the long-term efficacy and safety of deferasirox therapy in a large observational cohort of children with transfusion-dependent thalassemia (TDT) and sickle cell anemia (SCA) in Turkey. METHODS: This was a multicenter, prospective cohort study including TDT and SCA patients aged 2-18 years with iron overload (≥100 mL/kg of pRBC or a serum ferritin [SF] level >1000 μg/L) receiving deferasirox. Patients were followed for up to 3 years according to standard practice...
October 9, 2018: European Journal of Haematology
Umut Aypar, Stephanie A Smoley, Beth A Pitel, Kathryn E Pearce, Roman M Zenka, George Vasmatzis, Sarah H Johnson, James B Smadbeck, Jess F Peterson, Katherine B Geiersbach, Daniel L Van Dyke, Erik C Thorland, Robert B Jenkins, Rhett P Ketterling, Patricia T Greipp, Hutton M Kearney, Nicole L Hoppman, Linda B Baughn
OBJECTIVE: Acute myeloid leukemia (AML) can be subtyped based on recurrent cytogenetic and molecular genetic abnormalities with diagnostic and prognostic significance. Although cytogenetic characterization classically involves conventional chromosome and/or fluorescence in situ hybridization (FISH) assays, limitations of these techniques include poor resolution and the inability to precisely identify breakpoints. METHOD: We evaluated whether an NGS-based methodology that detects structural abnormalities and copy number changes using mate pair sequencing (MPseq) can enhance the diagnostic yield for patients with AML...
October 1, 2018: European Journal of Haematology
Pere Barba, Mireia Morgades, Pau Montesinos, Cristina Gil, María-Laura Fox, Juana Ciudad, María-José Moreno, José González-Campos, Eulàlia Genescà, Daniel Martínez-Carballeira, Rodrigo Martino, Susana Vives, Ramon Guardia, Santiago Mercadal, María-Teresa Artola, Antonia Cladera, Mar Tormo, Jordi Esteve, Juan Bergua, Ferran Vall-Llovera, Jordi Ribera, Pilar Martínez-Sanchez, María-Luz Amigo, Arantxa Bermúdez, María Calbacho, Jesús-Maria Hernández-Rivas, Evaristo Feliu, Alberto Orfao, Josep-María Ribera
OBJECTIVE AND METHODS: Pediatric-inspired regimens have been adopted by several groups as the treatment strategy for adult patients with acute lymphoblastic leukemia (ALL). Whether subsequent modifications of these protocols have led to an improvement in the outcome of patients is uncertain, especially in T-cell ALL. We analyzed 169 patients with high risk T-cell ALL included in 2 consecutive trials of the PETHEMA Group (HR-ALL03 [n=104] and the more contemporary HR-ALL11 [n=65]). RESULTS: Patients- and disease-characteristics were balanced between both groups...
September 29, 2018: European Journal of Haematology
Joseph J Shatzel, Matthew O'Donnell, Sven R Olson, Matthew R Kearney, Molly M Daughety, Justine Hum, Khanh P Nguyen, Thomas G DeLoughery
Thrombosis of unusual venous sites encompasses a large part of consultative hematology and is encountered routinely by practicing hematologists. Contrary to the more commonly encountered lower extremity venous thrombosis and common cardiovascular disorders, the various thromboses outlined in this review have unique presentations, pathophysiology, workup, and treatments that all hematologists should be aware of. This review attempts to outline the most up to date literature on cerebral, retinal, upper extremity, hepatic, portal, splenic, mesenteric, and renal vein thrombosis, focusing on the incidence, pathophysiology, provoking factors, and current recommended treatments for each type of unusual thrombosis to provide a useful and practical review for the hematologist...
September 28, 2018: European Journal of Haematology
Christopher J Patriquin, Thomas Kiss, Stephen Caplan, Ian Chin-Yee, Kuljit Grewal, Jennifer Grossman, Loree Larratt, Daniele Marceau, Tom Nevill, D Robert Sutherland, Richard A Wells, Brian Leber
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disease characterized by intravascular hemolysis, thrombophilia, and marrow failure. Its phenotype is due to absent or reduced expression of GPI-linked complement regulators and subsequent sensitivity of hematopoietic cells to complement-mediated damage and lysis. Introduction of the terminal complement inhibitor eculizumab drastically improved outcomes in PNH patients; however, despite this improvement, there remain several challenges faced by PNH patients and physicians who care for them...
September 22, 2018: 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
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
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