abstract：:Systemic AL amyloidosis, a disease with improving outcomes using novel therapies, is increasingly recognized in the elderly but treatment and outcomes have not been systematically studied in this group of patients in whom comorbidities and frailty may compound morbidity and mortality. We report the outcomes of 295 pat...

journal_title：Haematologica

authors： Sachchithanantham S,Offer M,Venner C,Mahmood SA,Foard D,Rannigan L,Lane T,Gillmore JD,Lachmann HJ,Hawkins PN,Wechalekar AD

更新日期：2015-11-01 00:00:00

abstract：:This phase Ib/II trial combined the pan-deacetylase inhibitor panobinostat with chemotherapy followed by panobinostat maintenance in elderly patients with newly diagnosed acute myeloid leukemia. Patients with prior history of myelodysplastic syndrome were excluded and 38 evaluable patients were included in the study (...

journal_title：Haematologica

authors： Ocio EM,Herrera P,Olave MT,Castro N,Pérez-Simón JA,Brunet S,Oriol A,Mateo M,Sanz MÁ,López J,Montesinos P,Chillón MC,Prieto-Conde MI,Díez-Campelo M,González M,Vidriales MB,Mateos MV,San Miguel JF,PETHEMA Group.

更新日期：2015-10-01 00:00:00

abstract：:Patients with refractory or relapsed and refractory multiple myeloma who no longer receive benefit from novel agents have limited treatment options and short expected survival. del(17p) and t(4;14) are correlated with shortened survival. The phase 3 MM-003 trial demonstrated significant progression-free and overall su...

journal_title：Haematologica

authors： Dimopoulos MA,Weisel KC,Song KW,Delforge M,Karlin L,Goldschmidt H,Moreau P,Banos A,Oriol A,Garderet L,Cavo M,Ivanova V,Alegre A,Martinez-Lopez J,Chen C,Spencer A,Knop S,Bahlis NJ,Renner C,Yu X,Hong K,Sternas L,

更新日期：2015-10-01 00:00:00

abstract：:Shwachman-Diamond syndrome is a congenital bone marrow failure disorder characterized by debilitating neutropenia. The disease is associated with loss-of-function mutations in the SBDS gene, implicated in ribosome biogenesis, but the cellular and molecular events driving cell specific phenotypes in ribosomopathies rem...

journal_title：Haematologica

authors： Zambetti NA,Bindels EM,Van Strien PM,Valkhof MG,Adisty MN,Hoogenboezem RM,Sanders MA,Rommens JM,Touw IP,Raaijmakers MH

更新日期：2015-10-01 00:00:00

abstract：:Ruxolitinib, a potent Janus kinase 1/2 inhibitor, resulted in rapid and durable improvements in splenomegaly and disease-related symptoms in the 2 phase III COMFORT studies. In addition, ruxolitinib was associated with prolonged survival compared with placebo (COMFORT-I) and best available therapy (COMFORT-II). We pre...

journal_title：Haematologica

authors： Vannucchi AM,Kantarjian HM,Kiladjian JJ,Gotlib J,Cervantes F,Mesa RA,Sarlis NJ,Peng W,Sandor V,Gopalakrishna P,Hmissi A,Stalbovskaya V,Gupta V,Harrison C,Verstovsek S,COMFORT Investigators.

更新日期：2015-09-01 00:00:00

abstract：:Multiple myeloma is preceded by an asymptomatic phase, comprising monoclonal gammopathy of uncertain significance and smoldering myeloma. Compared to the former, smoldering myeloma has a higher and non-uniform rate of progression to clinical myeloma, reflecting a subset of patients with higher risk. We evaluated the g...

journal_title：Haematologica

authors： Khan R,Dhodapkar M,Rosenthal A,Heuck C,Papanikolaou X,Qu P,van Rhee F,Zangari M,Jethava Y,Epstein J,Yaccoby S,Hoering A,Crowley J,Petty N,Bailey C,Morgan G,Barlogie B

更新日期：2015-09-01 00:00:00

abstract：:Next generation sequencing technologies have provided insights into the molecular heterogeneity of various myeloid neoplasms, revealing previously unknown somatic genetic events. In our cohort of 1444 cases analyzed by next generation sequencing, somatic mutations in the gene BRCA1-BRCA2-containing complex 3 (BRCC3) w...

journal_title：Haematologica

authors： Huang D,Nagata Y,Grossmann V,Radivoyevitch T,Okuno Y,Nagae G,Hosono N,Schnittger S,Sanada M,Przychodzen B,Kon A,Polprasert C,Shen W,Clemente MJ,Phillips JG,Alpermann T,Yoshida K,Nadarajah N,Sekeres MA,Oakley K,Ngu

更新日期：2015-08-01 00:00:00

abstract：:Successful expansion of hematopoietic stem cells would benefit the use of hematopoietic stem cell transplants in the clinic. Several angiopoietin-like proteins, including angiopoietin-like 7, can support the activity of hematopoietic stem cells. However, effects of ANGPTL7 on human hematopoietic stem cells and the dow...

journal_title：Haematologica

authors： Xiao Y,Jiang Z,Li Y,Ye W,Jia B,Zhang M,Xu Y,Wu D,Lai L,Chen Y,Chang Y,Huang X,Liu H,Qing G,Liu P,Li Y,Xu B,Zhong M,Yao Y,Pei D,Li P

更新日期：2015-05-01 00:00:00

abstract：:Clinical and hematologic characteristics of beta(β)-thalassemia are determined by several factors resulting in a wide spectrum of severity. Phenotype modulators are: HBB mutations, HBA defects and fetal hemoglobin production modulators (HBG2:g.-158C>T polymorphism, HBS1L-MYB intergenic region and the BCL11A). We chara...

journal_title：Haematologica

authors： Danjou F,Francavilla M,Anni F,Satta S,Demartis FR,Perseu L,Manca M,Sollaino MC,Manunza L,Mereu E,Marceddu G,Pissard S,Joly P,Thuret I,Origa R,Borg J,Forni GL,Piga A,Lai ME,Badens C,Moi P,Galanello R

更新日期：2015-04-01 00:00:00

abstract：:The mammalian target of rapamycin plays an important role in multiple myeloma. The allosteric mammalian target of rapamycin inhibitor everolimus has long been approved for immunosuppression and has shown activity in certain cancers. This investigator-initiated phase I trial explored the use of everolimus in relapsed a...

journal_title：Haematologica

authors： Günther A,Baumann P,Burger R,Kellner C,Klapper W,Schmidmaier R,Gramatzki M

更新日期：2015-04-01 00:00:00

abstract：:Blastic plasmacytoid dendritic cell neoplasm is an aggressive malignancy derived from plasmacytoid dendritic cells. There is currently no accepted standard of care for treating this neoplasm, and therapeutic strategies have never been prospectively evaluated. Since blastic plasmacytoid dendritic cell neoplasm cells ex...

journal_title：Haematologica

authors： Angelot-Delettre F,Roggy A,Frankel AE,Lamarthee B,Seilles E,Biichle S,Royer B,Deconinck E,Rowinsky EK,Brooks C,Bardet V,Benet B,Bennani H,Benseddik Z,Debliquis A,Lusina D,Roussel M,Solly F,Ticchioni M,Saas P,Garna

更新日期：2015-02-01 00:00:00

abstract：:Double umbilical cord blood transplantation is increasingly applied in the treatment of adult patients with high-risk hematological malignancies and has been associated with improved engraftment as compared to that provided by single unit cord blood transplantation. The mechanism of improved engraftment is, however, s...

journal_title：Haematologica

authors： Somers JA,Braakman E,van der Holt B,Petersen EJ,Marijt EW,Huisman C,Sintnicolaas K,Oudshoorn M,Groenendijk-Sijnke ME,Brand A,Cornelissen JJ

更新日期：2014-11-01 00:00:00

abstract：:ALOX5 is implicated in chronic myeloid leukemia development in mouse leukemic stem cells, but its importance in human chronic myeloid leukemia is unknown. Functional ALOX5 was assessed using an LTB4 ELISA and ALOX5, and LTB4R1 mRNA expression was determined via a TaqMan gene expression assay. LTB4R1 and 5-LOX protein ...

journal_title：Haematologica

authors： Lucas CM,Harris RJ,Giannoudis A,McDonald E,Clark RE

更新日期：2014-11-01 00:00:00

abstract：:Second-line therapy for corticosteroid-refractory or -dependent acute graft-versus-host disease remains ill-defined, due to limited efficacy of drugs and evolving clinical trial endpoints. Six-month freedom from treatment failure has been proposed as a novel clinical trial endpoint and is defined by the absence of dea...

journal_title：Haematologica

authors： Das-Gupta E,Greinix H,Jacobs R,Zhou L,Savani BN,Engelhardt BG,Kassim A,Worel N,Knobler R,Russell N,Jagasia M

更新日期：2014-11-01 00:00:00

abstract：:The National Institutes of Health global score for chronic graft-versus-host disease was devised by experts but was not based on empirical data. We hypothesized that analysis of prospectively collected data would enable derivation of a more accurate model for estimating mortality risk. We analyzed 574 adult patients w...

journal_title：Haematologica

authors： Inamoto Y,Martin PJ,Storer BE,Palmer J,Weisdorf DJ,Pidala J,Flowers ME,Arora M,Jagasia M,Arai S,Chai X,Pavletic SZ,Vogelsang GB,Lee SJ,Chronic GVHD Consortium.

更新日期：2014-10-01 00:00:00

abstract：:Monitoring minimal residual disease is an important way to identify patients with acute myeloid leukemia at high risk of relapse. In this study we investigated the prognostic potential of minimal residual disease monitoring by quantitative real-time polymerase chain reaction analysis of NPM1 mutations in patients trea...

journal_title：Haematologica

authors： Hubmann M,Köhnke T,Hoster E,Schneider S,Dufour A,Zellmeier E,Fiegl M,Braess J,Bohlander SK,Subklewe M,Sauerland MC,Berdel WE,Büchner T,Wörmann B,Hiddemann W,Spiekermann K

更新日期：2014-08-01 00:00:00

abstract：:The sialic-acid-binding immunoglobulin-like lectin SIGLEC-G is a negative regulator of B-cell receptor-mediated calcium signaling. Its deficiency leads to reduced turnover and increased proliferation and survival of murine B-1a cells. Siglecg(-/-) mice show a premature expansion of polyclonal CD5(+) B cells in the spl...

journal_title：Haematologica

authors： Simonetti G,Bertilaccio MT,Rodriguez TV,Apollonio B,Dagklis A,Rocchi M,Innocenzi A,Casola S,Winkler TH,Nitschke L,Ponzoni M,Caligaris-Cappio F,Ghia P

更新日期：2014-08-01 00:00:00

abstract：:Differentiation of erythroblasts to mature red blood cells involves dynamic changes of the membrane and cytoskeleton networks that are not fully characterized. Using a mouse fetal liver erythroblast culture system and a targeted shRNA functional screening strategy, we identified a critical role of pleckstrin-2 in acti...

journal_title：Haematologica

authors： Zhao B,Keerthivasan G,Mei Y,Yang J,McElherne J,Wong P,Doench JG,Feng G,Root DE,Ji P

更新日期：2014-07-01 00:00:00

abstract：:Immune dysregulation is a mechanism contributing to ineffective hematopoiesis in a subset of myelodysplastic syndrome patients. We report the first US multicenter non-randomized, phase II trial examining the efficacy of rabbit(r)-anti-thymocyte globulin using 2.5 mg/kg/day administered daily for 4 doses. The primary e...

journal_title：Haematologica

authors： Komrokji RS,Mailloux AW,Chen DT,Sekeres MA,Paquette R,Fulp WJ,Sugimori C,Paleveda-Pena J,Maciejewski JP,List AF,Epling-Burnette PK

更新日期：2014-07-01 00:00:00

abstract：:Age is a strong prognostic factor in multiple myeloma. The overall survival is shorter in patients older than 66 years, and even shorter in those older than 75 years. Whether age is also a prognostic parameter in patients younger than 66 years treated homogeneously with intensive approaches is unknown. To address this...

journal_title：Haematologica

authors： Chretien ML,Hebraud B,Cances-Lauwers V,Hulin C,Marit G,Leleu X,Karlin L,Roussel M,Stoppa AM,Guilhot F,Lamy T,Garderet L,Pegourie B,Dib M,Sebban C,Lenain P,Brechignac S,Royer B,Wetterwald M,Legros L,Orsini-Piocelle

更新日期：2014-07-01 00:00:00

abstract：:A retrospective, international, multicenter study was undertaken to assess: (i) the prognostic role of 'interim' positron emission tomography performed during treatment with doxorubicin, bleomycin, vinblastine and dacarbazine in patients with Hodgkin lymphoma; and (ii) the reproducibility of the Deauville five-point s...

journal_title：Haematologica

authors： Gallamini A,Barrington SF,Biggi A,Chauvie S,Kostakoglu L,Gregianin M,Meignan M,Mikhaeel GN,Loft A,Zaucha JM,Seymour JF,Hofman MS,Rigacci L,Pulsoni A,Coleman M,Dann EJ,Trentin L,Casasnovas O,Rusconi C,Brice P,Bolis

更新日期：2014-06-01 00:00:00

abstract：:The control of mRNA stability plays a central role in orchestrating gene-regulatory networks in hematopoietic cell growth, differentiation and tumorigenesis. HNRNPA0, which encodes an RNA-binding protein shown to regulate transcript stability via binding to the AU-rich elements of mRNAs, is located within the commonly...

journal_title：Haematologica

authors： Young DJ,Stoddart A,Nakitandwe J,Chen SC,Qian Z,Downing JR,Le Beau MM

更新日期：2014-06-01 00:00:00

abstract：:Even though alterations in platelet counts are presumed to be detrimental, their impact on the survival of patients has not been studied in large cohorts. The prevalence of thrombocytopenia and thrombocytosis was examined in a large inner city outpatient population of 36,262 individuals aged ≥65 years old. A significa...

journal_title：Haematologica

authors： Msaouel P,Lam AP,Gundabolu K,Chrysofakis G,Yu Y,Mantzaris I,Friedman E,Verma A

更新日期：2014-05-01 00:00:00

abstract：:Mastocytosis is a rare and chronic disease with phenotypes ranging from indolent to severe. Prognosis for this disease is variable and very few biomarkers to predict disease evolution or outcome are currently known. We have performed comprehensive screening in our large cohort of mastocytosis patients for mutations pr...

journal_title：Haematologica

authors： Hanssens K,Brenet F,Agopian J,Georgin-Lavialle S,Damaj G,Cabaret L,Chandesris MO,de Sepulveda P,Hermine O,Dubreuil P,Soucie E

更新日期：2014-05-01 00:00:00

abstract：:Myeloproliferative neoplasms are a varied group of disorders that can have prolonged chronic phases, but eventually accelerate and can transform into a secondary acute myeloid leukemia that is ultimately fatal. Triapine is a novel inhibitor of the M2 subunit of ribonucleotide reductase. Sequential inhibition of ribonu...

journal_title：Haematologica

authors： Zeidner JF,Karp JE,Blackford AL,Smith BD,Gojo I,Gore SD,Levis MJ,Carraway HE,Greer JM,Ivy SP,Pratz KW,McDevitt MA

更新日期：2014-04-01 00:00:00

abstract：:Some prospective studies showed that rabbit antithymocyte globulin was inferior to horse antithymocyte globulin as first-line therapy for patients with severe aplastic anemia. We retrospectively analyzed the clinical outcome of 455 children with severe aplastic anemia who received horse antithymocyte globulin (n=297) ...

journal_title：Haematologica

authors： Jeong DC,Chung NG,Cho B,Zou Y,Ruan M,Takahashi Y,Muramatsu H,Ohara A,Kosaka Y,Yang W,Kim HK,Zhu X,Kojima S

更新日期：2014-04-01 00:00:00

abstract：:The clinical heterogeneity among first relapses of childhood ETV6/RUNX1-positive acute lymphoblastic leukemia indicates that further genetic alterations in leukemic cells might affect the course of salvage therapy and be of prognostic relevance. To assess the incidence and prognostic relevance of additional copy numbe...

journal_title：Haematologica

authors： Bokemeyer A,Eckert C,Meyr F,Koerner G,von Stackelberg A,Ullmann R,Türkmen S,Henze G,Seeger K

更新日期：2014-04-01 00:00:00

abstract：:Myeloproliferative neoplasms are uncommon disorders in children, for which we have limited understanding of the pathogenesis and optimal management. JAK2 and MPL mutations, while common drivers of myeloproliferative neoplasms in adult patients, are not clearly linked to pediatric disease. Management and clinical outco...

journal_title：Haematologica

authors： Kucine N,Chastain KM,Mahler MB,Bussel JB

更新日期：2014-04-01 00:00:00

abstract：:Decidual natural killer cells accumulate at the fetal-maternal interface and play a key role in a successful pregnancy. However, their origin is still unknown. Do they derive from peripheral natural killer cells recruited in decidua or do they represent a distinct population that originates in situ? Here, we identifie...

journal_title：Haematologica

authors： Chiossone L,Vacca P,Orecchia P,Croxatto D,Damonte P,Astigiano S,Barbieri O,Bottino C,Moretta L,Mingari MC

更新日期：2014-03-01 00:00:00

abstract：:Double cord blood transplantation extends the use of cord blood to adults for whom a single unit is not available, but the procedure is limited by its cost. To evaluate outcomes and cost-effectiveness of double compared to single cord blood transplantation, we analyzed 134 transplants in adults with acute leukemia in ...

journal_title：Haematologica

authors： Labopin M,Ruggeri A,Gorin NC,Gluckman E,Blaise D,Mannone L,Milpied N,Yakoub-Agha I,Deconinck E,Michallet M,Fegueux N,Socié G,Nguyen S,Cahn JY,de Revel T,Garnier F,Faucher C,Taright N,Kenzey C,Volt F,Bertrand D,M

更新日期：2014-03-01 00:00:00

abstract：:It is well known that allo-reactive T cells play a crucial role in graft-versus-leukemia and graft-versus-host disease after allogeneic hematopoietic stem cell transplantation (alloSCT). Allo-reactive CD4(+) T cells can mediate direct cytolysis, but may also stimulate production of IgG antibodies as helper cells. Immu...

journal_title：Haematologica

authors： Kremer AN,van der Griendt JC,van der Meijden ED,Honders MW,Ayoglu B,Schwenk JM,Nilsson P,Falkenburg JH,Griffioen M

更新日期：2014-02-01 00:00:00

abstract：:Multiple myeloma management has undergone profound changes in the past thanks to advances in our understanding of the disease biology and improvements in treatment and supportive care approaches. This article presents recommendations of the European Myeloma Network for newly diagnosed patients based on the GRADE syste...

journal_title：Haematologica

authors： Engelhardt M,Terpos E,Kleber M,Gay F,Wäsch R,Morgan G,Cavo M,van de Donk N,Beilhack A,Bruno B,Johnsen HE,Hajek R,Driessen C,Ludwig H,Beksac M,Boccadoro M,Straka C,Brighen S,Gramatzki M,Larocca A,Lokhorst H,Magar

更新日期：2014-02-01 00:00:00

abstract：:Multiple myeloma is an incurable hematologic cancer characterized by the clonal proliferation of malignant plasma cells within the bone marrow. Numerous studies suggest that the myeloma plasma cells occupy and alter the stromal tissue of the bone marrow as a means of enhancing their survival and growth. However, the n...

journal_title：Haematologica

authors： Noll JE,Williams SA,Tong CM,Wang H,Quach JM,Purton LE,Pilkington K,To LB,Evdokiou A,Gronthos S,Zannettino AC

更新日期：2014-01-01 00:00:00

abstract：:Systemic anaplastic large cell lymphoma is a category of T-cell non-Hodgkin's lymphoma which can be further subdivided into two distinct entities (ALK(+) and ALK(-)) based on the presence or absence of ALK gene rearrangements. Among several pathways triggered by ALK signaling, constitutive activation of STAT3 is stric...

journal_title：Haematologica

authors： Spaccarotella E,Pellegrino E,Ferracin M,Ferreri C,Cuccuru G,Liu C,Iqbal J,Cantarella D,Taulli R,Provero P,Di Cunto F,Medico E,Negrini M,Chan WC,Inghirami G,Piva R

更新日期：2014-01-01 00:00:00

abstract：:β-thalassemia and sickle cell disease are widespread fatal genetic diseases. None of the existing clinical treatments provides a solution for all patients. Two main strategies for treatment are currently being investigated: (i) gene transfer of a normal β-globin gene; (ii) reactivation of the endogenous γ-globin gene....

journal_title：Haematologica

authors： Manchinu MF,Marongiu MF,Poddie D,Casu C,Latini V,Simbula M,Galanello R,Moi P,Cao A,Porcu S,Ristaldi MS

更新日期：2014-01-01 00:00:00

abstract：:In hematopoietic stem cell transplantation for hemophagocytic lymphohistiocytosis, high transplant-related mortality after busulfan-based myeloablative regimens has been observed. Conditioning regimens with reduced toxicity based on melphalan or treosulfan are promising alternatives. We retrospectively analyzed hemato...

journal_title：Haematologica

authors： Lehmberg K,Albert MH,Beier R,Beutel K,Gruhn B,Kröger N,Meisel R,Schulz A,Stachel D,Woessmann W,Janka G,Müller I

更新日期：2014-01-01 00:00:00

abstract：:Chronic lymphocytic leukemia is an incurable B-cell malignancy that is associated with tumor cell-mediated T-cell dysfunction. It therefore represents a challenging disease for T-cell immunotherapeutics. The CD19/CD3 bi-specific antibody construct blinatumomab (AMG103 or MT103) has been tested clinically in non-Hodgki...

journal_title：Haematologica

authors： Wong R,Pepper C,Brennan P,Nagorsen D,Man S,Fegan C

更新日期：2013-12-01 00:00:00

abstract：:The discovery that the Ten-Eleven Translocation (TET) hydroxylases cause DNA demethylation has fundamentally changed the notion of how DNA methylation is regulated. Clonal analysis of the hematopoetic stem cell compartment suggests that TET2 mutations can be early events in hematologic cancers and recent investigation...

journal_title：Haematologica

authors： Asmar F,Punj V,Christensen J,Pedersen MT,Pedersen A,Nielsen AB,Hother C,Ralfkiaer U,Brown P,Ralfkiaer E,Helin K,Grønbæk K

更新日期：2013-12-01 00:00:00

abstract：UNLABELLED:Ruxolitinib, a JAK1/JAK2 inhibitor, is currently the only pharmacological agent approved for the treatment of myelofibrosis. Approval was based on findings from two phase 3 trials comparing ruxolitinib with placebo (COMFORT-I) and with best available therapy (COMFORT-II) for the treatment of primary or secon...

journal_title：Haematologica

authors： Wilkins BS,Radia D,Woodley C,Farhi SE,Keohane C,Harrison CN

更新日期：2013-12-01 00:00:00

abstract：:Aplastic anemia is usually treated with immunosuppression or allogeneic transplant, depending on patient and disease characteristics. Syngeneic transplant offers a rare treatment opportunity with minimal transplant-related mortality, and offers an insight into disease mechanisms. We present here a retrospective analys...

journal_title：Haematologica

authors： Gerull S,Stern M,Apperley J,Beelen D,Brinch L,Bunjes D,Butler A,Ganser A,Ghavamzadeh A,Koh MB,Komarnicki M,Kröger N,Maertens J,Maschan A,Peters C,Rovira M,Sengeløv H,Socié G,Tischer J,Oneto R,Passweg J,Marsh J

更新日期：2013-11-01 00:00:00