European Journal of Haematology

Most patients affected by Myelodysplastic syndromes (MDS) develop transfusion dependence, which can be correlated with poorer outcomes partially caused by transfusional iron overload. Oral iron chelator deferasirox was approved in 2005 for the treatment of chronic iron overload due to blood transfusions, inclusive of transfusion-dependent lower IPSS risk (1-2). A prospective, open-label, single arm, Italian multicenter study (GIMEMA MDS0306) has been proposed to establish the safety and efficacy of deferasirox in a real-world population of transfusion-dependent lower IPSS MDS patients(3)...

BACKGROUND: Romiplostim is a thrombopoietin-mimetic peptibody for adult refractory chronic immune thrombocytopenia (ITP). We aimed to describe ITP patients receiving romiplostim, platelet counts, and romiplostim usage in UK clinical practice. METHODS: This was a retrospective cohort study of patients in the UKITP Registry who received romiplostim between October 2009 and January 2015, including data up to 6 months before romiplostim initiation through follow-up...

OBJECTIVE: This prospective, observational, postmarketing surveillance was conducted to evaluate the safety and effectiveness of mogamulizumab, an anti-CC chemokine receptor 4 (CCR4) monoclonal antibody, in patients with CCR4-positive, relapsed or refractory (r/r) adult T-cell leukemia-lymphoma (ATL) in Japan. METHOD: All patients were scheduled to receive intravenous infusions of mogamulizumab 1.0 mg/kg once weekly for 8 weeks, alone or in combination with other modalities...

OBJECTIVE: In patients with Large B-cell Lymphoma (LBCL) according to WHO the prognostic significance of MYC translocation is still not sufficiently clarified. We therefore aimed to investigate whether prognostication could be improved in patients with MYC translocation positive LBCL by additional stratification according to MYC and BCL2 protein expression levels or MYC translocation partner gene as well as concurrent BCL2 and/or BCL6 translocation (DH). METHODS: From an unselected consecutive cohort of >600 patients with LBCL investigated with fluorescent in situ hybridization (FISH) 64 patients were diagnosed with MYC translocation positive LBCL and included in the study...

Secondary acute myeloid leukemia (sAML) evolving from an antecedent hematological disorder and therapy-related sAML represent high-risk subsets of AML and are associated with poor clinical outcome. The hypomethylating agents (HMA), azacitidine and decitabine represent treatment options for elderly AML patients including sAML patients unfit for intensive chemotherapy. This article is protected by copyright. All rights reserved.

OBJECTIVE: The presence of plasmacytomas (Ps) in patients with multiple myeloma (MM) is associated with a poor outcome, both in patients treated conventionally and in patients treated with novel agents. Two types of plasmacytomas have being recognized: paraskeletal plasmacytomas (PPs) and extramedulary plasmacytomas (EMPs), being the incidence of EMPs lower but with worse prognosis. Our aim has been to analyze the efficacy of the pomalidomide-dexamethasone combination in this patient profile...

OBJECTIVE: In individuals with cytogenetically normal (CN) AML, disease risk is estimated using molecular features such as the status of NPM1 and FLT3-ITD genes. However, data regarding the impact of NPM1 and FLT3-ITD status on hematopoietic stem cell transplant (HCT) outcomes are limited. We examined the effect of NPM1 and FLT3-ITD status on transplant outcomes in 131 CN AML patients transplanted at Princess Margaret Hospital between 2006 and 2017. METHODS: Overall survival (OS) was calculated using Kaplan-Meier analysis and multivariable Cox proportional hazards regression...

Weighting the risks and benefits for patients with chronic myeloid leukemia (CML) and pregnancy is a challenge. Two main risks have to be evaluated: the risk of tyrosine kinase inhibitors (TKI) exposure during pregnancy and the risk of disease relapse without TKI when treatment is discontinued. A balanced decision considering leukemic burden and gestational age has to be found in accordance with the new data about TKI and pregnancy. This article is protected by copyright. All rights reserved.

OBJECTIVES: T cell-replete Haploidentical stem transplantation (Haplo-SCT) with post-transplant cyclophosphamide (PT-Cy) is at high risk of invasive fungal infections (IFI) and anti-mold active drug is required for primary antifungal prophylaxis (PAP) according to international guidelines. No data are available on the efficacy of caspofungin as PAP in this setting. METHODS: Here we report our retrospective experience with 103 consecutive patients treated with caspofungin as PAP after Haplo-SCT...

The management of Severe Aplastic Anaemia is particularly challenging when it occurs in the context of recent liver transplantation. Rapid identification of a suitable donor followed by allogeneic haematopoietic stem cell transplantation is the only curative option. This scenario is often complicated by potentially life threatening infections that develop as a consequence of immunosuppression. Alternative donor transplantation using suitably matched unrelated donors can be potentially lifesaving when suitably matched sibling donors are unavailable...

OBJECTIVES: Although hemoglobin thresholds for red blood cell (RBC) transfusion have decreased, double-unit RBC transfusion practices persist. We studied the effects switching from predominantly double-unit to single-unit RBC transfusions had on utilization and clinical outcomes for malignant hematology patients. METHODS: Retrospective chart review compared malignant hematology patients before and after implementing single-unit RBC transfusion policy. Hemoglobin threshold was 8...

Sickle cell anaemia (SCA) is the consequence of abnormal haemoglobin production due to an inherited point mutation in the β -globin gene. The resulting haemoglobin tetramer is poorly soluble when deoxygenated, and when this is prolonged, intracellular gelation of sickle haemoglobin occurs, followed by haemoglobin polymerization. If many cycles of sickling and unsickling occur, the red cell membrane will be disrupted leading to haemolysis and vaso-occlusive events. Recent studies have also shown that leukocyte adhesion molecules and nitric oxide (NO) depletion are involved in endothelial damage...

No abstract text is available yet for this article.

The Hermansky-Pudlak syndrome (HPS) is a genetically heterogeneous group of autosomal recessive disorders characterized by: oculocutaneous albinism (OCA); bleeding diathesis; and other systemic complications including: chronic granulomatous colitis, and pulmonary fibrosis. Despite HPS being a rare genetic disease worldwide, it is the most common single-gene disorder in the island of Puerto Rico (PR), particularly in the northwestern region, where it occurs with a frequency of 1:1,800 and where carrier frequency is estimated to be 1 out of 21 citizens...

OBJECTIVES: The purpose of this study was to compare the prevalence of common oral diseases between allogeneic haematopoietic stem cell transplantation (HSCT) recipients and healthy controls. MATERIALS AND METHODS: A total of 143 adult allogeneic HSCT recipients who were treated for haematological malignancies between 2008 and 2016 were included in the study. The HSCT recipients were age and sex matched with healthy controls. A dental examination was performed on the HSCT recipients prior to HSCT...

Direct oral anticoagulants (DOACs) have been shown to be superior to vitamin K antagonists (VKAs) in regards to safety and efficacy in numerous clinical trials, and are now the preferred oral anticoagulant by multiple professional societies. However, patients with significant levels of organ dysfunction were excluded from all major clinical trials, leaving the clinical benefit in these subsets uncertain. Patients with chronic kidney disease (CKD) specifically often require anticoagulation for acute or long-term indications such as venous thromboembolism, atrial fibrillation, or mechanical heart valves...

OBJECTIVES: To assess real-world treatment patterns and healthcare resource utilization (HRU) among patients with FLT3-mutated (FLT3mut ) and FLT3-wild type (FLT3wt ) acute myeloid leukemia (AML). METHODS: Data were abstracted from medical charts of patients with AML from 10 countries. Patients were grouped based on their FLT3 mutation status, age (18-64 or ≥65), and whether they were newly diagnosed (ND) or relapsed/refractory (R/R). RESULTS: Charts of 1,027 AML patients were included (183 FLT3mut 18-64 ND; 136 FLT3mut ≥65 ND; 181 FLT3mut R/R; 186 FLT3wt 18-64 ND; 159 FLT3wt ≥65 ND; 182 FLT3wt R/R)...

OBJECTIVE: We evaluated the development of ICU survival of patients with haematopoietic malignancies and discussed changes in admittance policies. METHOD: We compared 166 patients treated between 2009 and 2012 with 138 patients treated between 2013 and 2016. Patient characteristics and outcome were analysed. RESULTS: ICU survival was 45.2% in the first group and 66.7% in the second (P < 0.0005). Infection (P = 0.033), invasive ventilation (IMV) (P = 0...

The molecular pathogenesis of deletion 5q (del(5q)) MDS has recently been realized as a result of major advances in our understanding of the mechanisms responsible for clinical phenotype. Identification of commonly deleted genes such as RPS14, miRNA-145, HSPA9, CD78, and CSNK1a1 have elucidated the precise biological changes responsible for the anemia, leukopenia, and thrombocytosis that characterizes del(5q) MDS, and highlighted the importance of allelic haploinsufficiency in the hematological phenotype. Recent elegant investigations have also highlighted a critical role of innate immune signaling in del(5q) pathogenesis...

OBJECTIVES: To establish the optimal strategy for haploidentical hematopoietic stem cell transplantation (HSCT). METHODS: We performed a prospective study on haploidentical HSCT using low-dose alemtuzumab. Alemtuzumab was added at 0.25 mg/kg for 2 days. The primary outcome measure was the survival rate with the engraftment of donor cells and without grade III-IV acute graft-versus-host disease (GVHD) at 60 days after transplantation. RESULTS: Fourteen adult patients with advanced hematological disease were enrolled...