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Impact of ibrutinib dose intensity on patient outcomes in previously treated Waldenstr öm macroglobulinemia.
Impact of ibrutinib dose intensity on patient outcomes in previously treated Waldenström macroglobulinemia. Haematologica. 2018 May 17;: Authors: Castillo JJ, Gustine JN, Meid K, Dubeau TE, Xu L, Yang G, Hunter ZR, Advani R, Palomba L, Treon SP PMID: 29773590 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - May 17, 2018 Category: Hematology Authors: Castillo JJ, Gustine JN, Meid K, Dubeau TE, Xu L, Yang G, Hunter ZR, Advani R, Palomba L, Treon SP Tags: Haematologica Source Type: research

t(11;14)-positive mantel cell Lymphomas lacking cyclin D1 (CCND1) immunostaining because of a CCND1 mutation or exclusive expression of the CCND1b isoform.
apper W PMID: 29773591 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - May 17, 2018 Category: Hematology Authors: Iaccarino I, Afify L, Aukema SM, Reddemann K, Schütt P, Flür M, Klapper W Tags: Haematologica Source Type: research

Investigating chemoresistance to improve sensitivity of childhood T cell acute lymphoblastic leukemia to parthenolide.
This study examined mechanisms for this resistance, including protective effects conferred by bone marrow stromal components. T cell acute leukemia cells co-cultured with mesenchymal stem cells demonstrated significantly enhanced survival against parthenolide (73+/-11%) compared to cells treated without mesenchymal stem cell support (11+/-9%). Direct cell contact between mesenchymal cells and leukemia cells was not required to afford protection from parthenolide. Mesenchymal stem cells released thiols and protected leukemia cells from reactive oxygen species stress, which is associated with parthenolide cytotoxicity. Block...
Source: Haematologica - May 17, 2018 Category: Hematology Authors: Ede BC, Asmaro RR, Moppett JP, Diamanti P, Blair A Tags: Haematologica Source Type: research

Safety and efficacy of second-line bosutinib for chronic phase chronic myeloid leukemia over a five-year period: final results of a phase 1/2 study.
We report the final results of a phase 1/2 study of second-line bosutinib in chronic phase chronic myeloid leukemia patients after imatinib failure (n=284). Median follow-up and treatment durations were 54.8 (range, 0.6-96.3) and 25.6 (0.2&-96.3) months, respectively. At years 2 and 5, 54% and 40% of patients, respectively, remained on bosutinib. Cumulative major cytogenetic response and complete cytogenetic response rates (newly-attained or maintained from baseline) were 58% and 46%, respectively, by year 2 and 60% and 50% by year 5. Kaplan-Meier probability of maintaining major and complete cytogenetic response was 7...
Source: Haematologica - May 17, 2018 Category: Hematology Authors: Gambacorti-Passerini C, Cortes JE, Lipton JH, Kantarjian HM, Kim DW, Schafhausen P, Crescenzo R, Bardy-Bouxin N, Shapiro M, Noonan K, Leip E, DeAnnuntis L, Brümmendorf TH, Khoury HJ Tags: Haematologica Source Type: research

Interleukin-22 levels are increased in gastrointestinal graft versus host disease in children.
PMID: 29773594 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - May 17, 2018 Category: Hematology Authors: Lounder DT, Khandelwal P, Gloude NJ, Dandoy CE, Jodele S, Medvedovic M, Denson LA, Lane A, Lake K, Litts B, Wilkey A, Davies SM Tags: Haematologica Source Type: research

Therapeutic effects of the novel subtype-selective histone deacetylase (HDAC) inhibitor chidamide on myeloma-associated bone disease.
Abstract Histone deacetylases are promising therapeutic targets in hematological malignancies. In the present work, we investigated the effect of chidamide, a new subtype-selective histone deacetylase inhibitor that was independently produced in China, on multiple myeloma and its associated bone diseases using different models. The cytotoxicity of chidamide toward myeloma is due to its induction of cell apoptosis and cell cycle arrest by increasing the levels of caspase family proteins, including p21 and p27, among others. Furthermore, chidamide exhibited significant cytotoxicity against myeloma cells co-cultured ...
Source: Haematologica - May 17, 2018 Category: Hematology Authors: He J, Chen Q, Gu H, Chen J, Zhang E, Guo X, Huang X, Yan H, He D, Yang Y, Zhao Y, Wang G, He H, Yi Q, Cai Z Tags: Haematologica Source Type: research

CARD10, a CEBPE target involved in granulocytic differentiation.
In this study, we have identified Card10 as a novel target of CEBPE. We show that CEBPE binds to regulatory elements upstream of the murine Card10 locus, and expression of CARD10 is significantly reduced in Cebpe knock-out mice. Silencing Card10 in a human cell line and in murine primary cells impaired granulopoiesis, affecting expression of genes involved in myeloid cell development and function. Taken together, our data demonstrate for the first time that Card10 is expressed in granulocytes and is a direct target of CEBPE with functions extending to myeloid differentiation. PMID: 29773596 [PubMed - as supplied by pu...
Source: Haematologica - May 17, 2018 Category: Hematology Authors: Shyamsunder P, Sankar H, Mayakonda A, Han L, Nordin HBM, Woon TW, Shanmugasundaram M, Dakle P, Madan V, Koeffler HP Tags: Haematologica Source Type: research

Hematopoietic stem cell loss and hematopoietic failure in severe aplastic anemia is driven by macrophages and aberrant podoplanin expression.
Abstract Severe aplastic anemia results from profound hematopoietic stem cell loss. T cells and interferon gamma have long been associated with severe aplastic anemia, yet the underlying mechanisms driving hematopoietic stem cell loss remain unknown. Using a mouse model of severe aplastic anemia, we demonstrate that interferon gamma-dependent hematopoietic stem cell loss required macrophages. Interferon gamma was necessary for bone marrow macrophage persistence, despite loss of other myeloid cells and hematopoietic stem cells. Depleting macrophages or abrogating interferon gamma signaling specifically in macrophag...
Source: Haematologica - May 17, 2018 Category: Hematology Authors: McCabe A, Smith JNP, Costello A, Maloney J, Katikaneni D, MacNamara KC Tags: Haematologica Source Type: research

Concomitant WT1 mutations predict poor prognosis in acute myeloid leukemia patients with double mutant CEBPA.
PMID: 29773598 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - May 17, 2018 Category: Hematology Authors: Tien FM, Hou HA, Tang JL, Kuo YY, Chen CY, Tsai CH, Yao M, Lin CT, Li CC, Huang SY, Ko BS, Hsu SC, Wu SJ, Liu JH, Chou SC, Tsay W, Tseng MH, Liu MC, Liu CW, Lin LI, Chou WC, Tien HF Tags: Haematologica Source Type: research

TGF β1-mediated functional inhibition of mesenchymal stromal cells in myelodysplastic syndromes and acute myeloid leukemia.
TGFβ1-mediated functional inhibition of mesenchymal stromal cells in myelodysplastic syndromes and acute myeloid leukemia. Haematologica. 2018 May 17;: Authors: Geyh S, Rodríguez-Paredes M, Jäger P, Koch A, Bormann F, Gutekunst J, Zilkens C, Germing U, Kobbe G, Lyko F, Haas R, Schroeder T Abstract Mesenchymal stromal cells are involved in the pathogenesis of myelodysplastic syndromes and acute myeloid leukemia, but the underlying mechanisms are incompletely understood. To further characterize the pathological phenotype we performed RNA sequencing of mesenchymal stromal cells from pati...
Source: Haematologica - May 17, 2018 Category: Hematology Authors: Geyh S, Rodríguez-Paredes M, Jäger P, Koch A, Bormann F, Gutekunst J, Zilkens C, Germing U, Kobbe G, Lyko F, Haas R, Schroeder T Tags: Haematologica Source Type: research

The interleukin-3 receptor CD123 targeted SL-401 mediates potent cytotoxic activity against CD34+CD123+ cells from acute myeloid leukemia / myelodysplastic syndrome patients and healthy donors.
We report here indiscriminate killing of CD123+ normal and acute myeloid leukemia / myelodysplastic syndrome cells by SL-401, a diphtheria toxin interleukin-3 fusion protein. SL-401 induced cytotoxicity of CD123+ primary cells/blasts from acute myeloid leukemia and myelodysplastic syndrome patients but not CD123- lymphoid cells. Importantly, SL-401 was highly active even in cells expressing low levels of CD123, with minimal effect on modulation of the CD123 target in acute myeloid leukemia. SL-401 significantly prolonged survival of leukemic mice in acute myeloid leukemia patient-derived xenograft mouse models. In addition...
Source: Haematologica - May 17, 2018 Category: Hematology Authors: Mani R, Goswami S, Gopalakrishnan B, Ramaswamy R, Wasmuth R, Tranh M, Mo X, Gordon A, Bucci D, Lucas DM, Mims A, Brooks C, Dorrance A, Walker A, Blum W, Byrd JC, Lozanski G, Vasu S, Muthusamy N Tags: Haematologica Source Type: research

Combinatorial targeting of XPO1 and FLT3 exerts synergistic anti-leukemia effects through induction of differentiation and apoptosis in FLT3-mutated acute myeloid leukemias: from concept to clinical trial.
Abstract Targeted therapies against FLT3-mutated acute myeloid leukemia have shown limited clinical efficacy primarily because of the acquisition of secondary mutations in FLT3 and persistent activation of downstream pro-survival pathways such as MEK/ERK, PI3K/AKT, and STAT5. Activation of these additional kinases may also result in phosphorylation of tumor suppressor proteins promoting their nuclear export. Thus, co-targeting nuclear export proteins (i.e., XPO1) and FLT3 concomitantly may be therapeutically effective. Here we report on the combinatorial inhibition of XPO1 using selinexor and FLT3 using sorafenib....
Source: Haematologica - May 17, 2018 Category: Hematology Authors: Zhang W, Ly C, Ishizawa J, Mu H, Ruvolo V, Shacham S, Daver N, Andreeff M Tags: Haematologica Source Type: research

Clofarabine, high-dose cytarabine and liposomal daunorubicin in pediatric relapsed/refractory acute myeloid leukemia: a phase IB study.
In conclusion, clofarabine was well tolerated and showed high response rates in relapsed/refractory pediatric acute myeloid leukemia. Patients with (sub)clinical fungal infections should be treated with caution. Clofarabine has been taken forward in the Berlin-Frankfurt-Munster study for newly diagnosed acute myeloid leukemia. The Study ITCC-020 was registered as EUDRA-CT 2009-009457-13; Dutch Trial Registry number 1880. PMID: 29773602 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - May 17, 2018 Category: Hematology Authors: van Eijkelenburg NKA, Rasche M, Ghazaly E, Dworzak MN, Klingebiel T, Rossig C, Leverger G, Stary J, de Bont ESJM, Chitu DA, Bertrand Y, Brethon B, Strahm B, van der Sluis IM, Kaspers GJL, Reinhardt D, Zwaan CM Tags: Haematologica Source Type: research

Clinical efficacy of ruxolitinib and chemotherapy in a child with Philadelphia chromosome-like acute lymphoblastic leukemia with GOLGA5-JAK2 fusion and induction failure.
PMID: 29773603 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - May 17, 2018 Category: Hematology Authors: Ding YY, Stern JW, Jubelirer TF, Wertheim GB, Li F, Chang F, Gu Z, Mullighan CG, Li Y, Harvey RC, Chen IM, Willman CL, Hunger SP, Li MM, Tasian SK Tags: Haematologica Source Type: research

Transcriptional activities of DUX4 fusions in B-cell acute lymphoblastic leukemia.
PMID: 29773604 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - May 17, 2018 Category: Hematology Authors: Tanaka Y, Kawazu M, Yasuda T, Tamura M, Hayakawa F, Kojima S, Ueno T, Kiyoi H, Naoe T, Mano H Tags: Haematologica Source Type: research

End-of-treatment and serial PET imaging in primary mediastinal B-cell lymphoma following dose-adjusted-EPOCH-R: A paradigm shift in clinical decision making.
Abstract Dose-adjusted-EPOCH-R obviates the need for radiotherapy in most patients with primary mediastinal B-cell lymphoma. End-of-treatment PET, however, does not accurately identify patients at risk of treatment failure, thereby confounding clinical decision making. To define the role of PET in primary mediastinal B-cell lymphoma following dose-adjusted-EPOCH-R, we extended enrollment and follow-up on our published phase II trial and independent series. Ninety-three patients received dose-adjusted-EPOCH-R without radiotherapy. End-of-treatment PET was performed in 80 patients, of whom 57 received 144 serial sca...
Source: Haematologica - May 10, 2018 Category: Hematology Authors: Melani C, Advani R, Roschewski M, Walters KM, Chen CC, Baratto L, Ahlman MA, Miljkovic MD, Steinberg SM, Lam J, Shovlin M, Dunleavy K, Pittaluga S, Jaffe ES, Wilson WH Tags: Haematologica Source Type: research

Genomic characterization of spleens in patients with myelofibrosis.
PMID: 29748436 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - May 10, 2018 Category: Hematology Authors: Zimran E, Tripodi J, Rampal R, Rapaport F, Zirkiev S, Hoffman R, Najfeld V Tags: Haematologica Source Type: research

Quantitative competitive allele-specific TaqMan duplex PCR (qCAST-Duplex PCR) assay: a refined method for highly sensitive and specific detection of JAK2V617F mutant allele burdens.
PMID: 29748437 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - May 10, 2018 Category: Hematology Authors: Hsu CC, Huang CE, Wu YY, Chen YY, Lung J, Leu YW, Li CP, Tsou HY, Chuang WH, Lu CH, Chen CC Tags: Haematologica Source Type: research

Outcomes of hematopoietic stem cell transplantation from unmanipulated haploidentical versus matched sibling donor in patients with acute myeloid leukemia in first complete remission with intermediate or high-risk cytogenetics: a study from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation.
ler A Abstract Allogeneic hematopoietic stem cell transplantation is the optimal care for patients with high-risk or intermediate acute myeloid leukemia. In patients lacking matched sibling donor, haploidentical donors are an option. We compared outcomes of unmanipulated haploidentical to matched sibling donor transplant in acute myeloid leukemia patients in first complete remission. Included were int- and high-risk acute myeloid leukemia in first complete remission undergoing haploidentical and matched sibling donor transplant from 2007-2015 and reported to the ALWP of the EBMT. A propensity score technique was u...
Source: Haematologica - May 10, 2018 Category: Hematology Authors: Salvatore D, Labopin M, Ruggeri A, Battipaglia G, Ghavamzadeh A, Ciceri F, Blaise D, Arcese W, Socié G, Bourhis JH, Van Lint M, Bruno B, Huynh A, Santarone S, Deconinck E, Mohty M, Nagler A Tags: Haematologica Source Type: research

Autologous T cell activation fosters ABT-199 resistance in chronic lymphocytic leukemia: rationale for a combined therapy with SYK inhibitors and anti-CD20 MoAbs.
M, Borge M, Giordano M, Gamberale R PMID: 29748439 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - May 10, 2018 Category: Hematology Authors: Elías EE, Almejún MB, Colado A, Cordini G, Vergara-Rubio M, Podaza E, Risnik D, Cabrejo M, Fernández-Grecco H, Bezares RF, Custidiano MDR, Sánchez-Ávalos JC, Vicente Á, Garate GM, Borge M, Giordano M, Gamberale R Tags: Haematologica Source Type: research

Meaningful changes in end-of-life care among patients with myeloma.
Abstract Patients with advanced myeloma experience a high symptom burden particularly near the end of life, making timely hospice use crucial. Little is known about the quality and determinants of end-of-life care for this population, including if potential increases in hospice use are also accompanied by "late" enrollment (≤ 3 days before death). Using the Surveillance, Epidemiology, and End-Results-Medicare database, we identified patients ≥ 65 years diagnosed with myeloma between 2000 and 2013 that died by December 31, 2013. We assessed prevalence and trends in hospice, including late enrollment...
Source: Haematologica - May 10, 2018 Category: Hematology Authors: Odejide OO, Li L, Cronin AM, Murillo A, Richardson PG, Anderson KC, Abel GA Tags: Haematologica Source Type: research

Maternal embryonic leucine zipper kinase inhibitor OTSSP167 has preclinical activity on multiple myeloma bone disease.
In conclusion, we show that OTSSP167 has a direct effect on myeloma-induced bone disease in addition to its anti-multiple myeloma effect, which warrants further clinical development of MELK inhibition in multiple myeloma. PMID: 29748441 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - May 10, 2018 Category: Hematology Authors: Muller J, Bolomsky A, Dubois S, Duray E, Stangelberger K, Plougonven E, Lejeune M, Léonard A, Marty C, Hempel U, Baron F, Beguin Y, Cohen-Solal M, Ludwig H, Heusschen R, Caers J Tags: Haematologica Source Type: research

NPM1 mutation is not associated with prolonged complete remission in acute myeloid leukemia patients treated with hypomethylating agents.
e;s L PMID: 29748442 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - May 10, 2018 Category: Hematology Authors: Prata PH, Bally C, Prebet T, Recher C, Venton G, Thomas X, Raffoux E, Pigneux A, Cluzeau T, Desoutter J, Gay J, Preudhomme C, Fenaux P, Adès L Tags: Haematologica Source Type: research

A Phase 2 multicenter study of the anti-CD19 antibody drug conjugate coltuximab ravtansine (SAR3419) in patients with relapsed or refractory diffuse large B-cell lymphoma previously treated with rituximab-based immunotherapy.
ni AM Abstract This phase 2, single-arm, multicenter study examined the efficacy and safety of coltuximab ravtansine (an anti-CD19 antibody drug conjugate) in 61 patients with histologically documented (de novo or transformed) relapsed or refractory diffuse large B-cell lymphoma who had previously received rituximab containing immuno-chemotherapy. Patients had received a median of 2.0 (range 0-9) prior treatment regimens for diffuse large B-cell lymphoma and almost half (45.9%) had bulky disease (≥ lesion>5 cm) at trial entry. Patients received coltuximab ravtansine (55 mg/m2) in 4 weekly and 4 biweekly admi...
Source: Haematologica - May 10, 2018 Category: Hematology Authors: Trněný M, Verhoef G, Dyer MJ, Yehuda DB, Patti C, Canales M, Lopez A, Awan FT, Montgomery PG, Janikova A, Barbui AM, Sulek K, Terol MJ, Radford J, Guidetti A, Di Nicola M, Siraudin L, Hatteville L, Schwab S, Oprea C, Gianni AM Tags: Haematologica Source Type: research

Rituximab plus Bendamustine as front line treatment in frail elderly ( > 70 years) patients with diffuse large B-cell non-Hodgkin's lymphoma: a phase II multicenter study of the Fondazione Italiana Linfomi.
Rituximab plus Bendamustine as front line treatment in frail elderly (>70 years) patients with diffuse large B-cell non-Hodgkin's lymphoma: a phase II multicenter study of the Fondazione Italiana Linfomi. Haematologica. 2018 May 10;: Authors: Storti S, Spina M, Pesce EA, Salvi F, Merli M, Ruffini A, Cabras G, Chiappella A, Angelucci E, Fabbri A, Liberati AM, Tani M, Musuraca G, Molinari A, Petrilli MP, Palladino C, Ciancia R, Ferrario A, Gasbarrino C, Monaco F, Fraticelli V, De Vellis A, Merli F, Luminari S Abstract We conducted a phase II study to assess activity and safety profile of bendamustine...
Source: Haematologica - May 10, 2018 Category: Hematology Authors: Storti S, Spina M, Pesce EA, Salvi F, Merli M, Ruffini A, Cabras G, Chiappella A, Angelucci E, Fabbri A, Liberati AM, Tani M, Musuraca G, Molinari A, Petrilli MP, Palladino C, Ciancia R, Ferrario A, Gasbarrino C, Monaco F, Fraticelli V, De Vellis A, Merli Tags: Haematologica Source Type: research

GRP94 rewires and buffers the FLT3-ITD signaling network and promotes survival of acute myeloid leukemic stem cells.
t McCormack Group Abstract Internal tandem duplications in the tyrosine kinase receptor FLT3 (FLT3-ITD) are among the most common lesions in acute myeloid leukemia and there exists a need for new forms of treatment. Using ex vivo drug sensitivity screening, we found that FLT3-ITD+ patient cells are particularly sensitive to HSP90 inhibitors. While it is well known that HSP90 is important for FLT3-ITD stability, we found that HSP90 family members play a much more complex role in FLT3-ITD signaling than previously appreciated. First, we found that FLT3-ITD activates the unfolded protein response, leading to increase...
Source: Haematologica - May 10, 2018 Category: Hematology Authors: Zhang B, Durán PA, Piechaczyk L, Fløisand Y, Safont MMS, Karlsen IT, Fandalyuk Z, Tadele D, Mierlo PV, Rowe AD, Robertson JM, Gjertsen BT, McCormack E, Enserink JM, Bjørn Tore Gjertsen Group, Emmet McCormack Group Tags: Haematologica Source Type: research

BRAF V600E mutation detected in a case of Rosai-Dorfman disease.
le JF PMID: 29748446 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - May 10, 2018 Category: Hematology Authors: Fatobene G, Haroche J, Hélias-Rodzwicz Z, Charlotte F, Taly V, Ferreira AM, Abdo ANR, Rocha V, Emile JF Tags: Haematologica Source Type: research

Morphologic, immunophenotypic, and genetic features of chronic lymphocytic leukemia with trisomy 12: a comprehensive review.
Abstract Chronic lymphocytic leukemia is an extremely heterogeneous disease and prognostic factors such as chromosomal abnormalities are important predictors of time to first treatment and survival. Trisomy 12 is the second most frequent aberration detected by fluorescence in situ hybridization at the time of diagnosis (10% to 25%), and it confers an intermediate prognostic risk, with a median time to first treatment of 33 months and a median overall survival of 114 months. Here, we review the unique morphologic, immunophenotypic, and genetic characteristics of patients with chronic lymphocytic leukemia and trisom...
Source: Haematologica - May 10, 2018 Category: Hematology Authors: Autore F, Strati P, Laurenti L, Ferrajoli A Tags: Haematologica Source Type: research

Phase I trial of plerixafor combined with decitabine in newly diagnosed older patients with acute myeloid leukemia.
The objective of this study was to investigate the safety and efficacy of decitabine combined with the CXCR4 antagonist plerixafor in newly diagnosed older patients with acute myeloid leukemia and to evaluate the effects of plerixafor on leukemia stem cells. Patients were treated with monthly cycles of decitabine 20 mg/m2 days 1-10 and escalating doses of plerixafor (320-810 mcg/kg) days 1-5. Sixty-nine patients were treated, with overall response rate 43%. Adverse karyotype did not predict response (P=0.31). Prior hypomethylating agent treatment was the strongest independent predictor of adverse overall survival (hazard r...
Source: Haematologica - May 3, 2018 Category: Hematology Authors: Roboz GJ, Ritchie EK, Dault Y, Lam L, Marshall DC, Cruz NM, Hsu HC, Hassane DC, Christos PJ, Ippoliti C, Scandura JM, Guzman ML Tags: Haematologica Source Type: research

Natural history of GATA2 deficiency in a survey of 79 French and Belgian patients.
o B, Terriou L, Francois S, Lioure B, Ahle G, Bachelerie F, Preudhomme C, Delabesse E, Cavé H, Bellanné-Chantelot C, Pasquet M, French GATA2 working party Abstract Heterozygous germline GATA2 mutations strongly predispose to leukaemia, immunodeficiency, and/or lymphoedema. We now describe a series of 79 patients (53 families) diagnosed since 2011, compiling all patients in France and Belgium, with a follow up of 2249 patients/years. Median age at first clinical symptoms was 18.6 years (range, 0-61 years). Severe infectious diseases (mycobacteria, fungus, and human papilloma virus) and haematological ...
Source: Haematologica - May 3, 2018 Category: Hematology Authors: Donadieu J, Lamant M, Fieschi C, Sicre de Fontbrune F, Caye A, Ouachee M, Beaupain B, Bustamante J, Poirel HA, Isidor B, Van Den Neste E, Neel A, Nimubona S, Toutain F, Barlogis V, Schleinitz N, Leblanc T, Rohrlich P, Suarez F, Ranta D, Abou Chahla W, Bru Tags: Haematologica Source Type: research

(Auto-)immune signature in aplastic anemia.
PMID: 29712817 [PubMed - in process] (Source: Haematologica)
Source: Haematologica - May 1, 2018 Category: Hematology Authors: Risitano AM Tags: Haematologica Source Type: research

Hematopoietic stem cell mobilization with plerixafor in sickle cell disease.
PMID: 29712818 [PubMed - in process] (Source: Haematologica)
Source: Haematologica - May 1, 2018 Category: Hematology Authors: Hsieh MM, Tisdale JF Tags: Haematologica Source Type: research

Age-related clonal hematopoiesis and monoclonal B-cell lymphocytosis/chronic lymphocytic leukemia: a new association?
PMID: 29712819 [PubMed - in process] (Source: Haematologica)
Source: Haematologica - May 1, 2018 Category: Hematology Authors: Condoluci A, Rossi D Tags: Haematologica Source Type: research

Denosumab for bone lesions in multiple myeloma - what is its value?
PMID: 29712820 [PubMed - in process] (Source: Haematologica)
Source: Haematologica - May 1, 2018 Category: Hematology Authors: Goldstein DA Tags: Haematologica Source Type: research

Osteoprotective medication in the era of novel agents: a European perspective on values, risks and future solutions.
PMID: 29712821 [PubMed - in process] (Source: Haematologica)
Source: Haematologica - May 1, 2018 Category: Hematology Authors: Engelhardt M, Herget GW, Graziani G, Ihorst G, Reinhardt H, Ajayi S, Knop S, Wasch R Tags: Haematologica Source Type: research

NKG2D-based chimeric antigen receptor therapy induced remission in a relapsed/refractory acute myeloid leukemia patient.
la ML PMID: 29703727 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - April 27, 2018 Category: Hematology Authors: Sallman DA, Brayer J, Sagatys EM, Lonez C, Breman E, Agaugué S, Verma B, Gilham DE, Lehmann FF, Davila ML Tags: Haematologica Source Type: research

Phase I study of the heparanase inhibitor Roneparstat: an innovative approach for multiple myeloma therapy.
PMID: 29700168 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - April 26, 2018 Category: Hematology Authors: Galli M, Chatterjee M, Grasso M, Specchia G, Magen H, Einsele H, Celeghini I, Barbieri P, Paoletti D, Pace S, Sanderson RD, Rambaldi A, Nagler A Tags: Haematologica Source Type: research

Stability of fetal hemoglobin levels in patients receiving metformin therapy.
PMID: 29700169 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - April 26, 2018 Category: Hematology Authors: Boulassel MR, El-Hussain AI, Hassan MM, Toumi ML, Merzoug S, Al-Qarni Z, Khan H, Gaifer Z, Al-Farsi K, Al-Khabori M, Al Huneini M, Al-Kindi S Tags: Haematologica Source Type: research

Real-world treatment and outcomes among older adults with chronic lymphocytic leukemia before the novel agents era.
PMID: 29700170 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - April 26, 2018 Category: Hematology Authors: Mato A, Jahnke J, Li P, Mehra M, Ladage VP, Mahler M, Huntington S, Doshi JA Tags: Haematologica Source Type: research

Short-hairpin RNA against aberrant HBB[IVSI-110(G > A)] mRNA restores β-globin levels in a novel cell model and acts as mono- and combination therapy for β-thalassemia in primary hematopoietic stem cells.
Short-hairpin RNA against aberrant HBB[IVSI-110(G>A)] mRNA restores β-globin levels in a novel cell model and acts as mono- and combination therapy for β-thalassemia in primary hematopoietic stem cells. Haematologica. 2018 Apr 26;: Authors: Patsali P, Papasavva P, Stephanou C, Christou S, Sitarou M, Antoniou MN, Lederer CW, Kleanthous M PMID: 29700171 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - April 26, 2018 Category: Hematology Authors: Patsali P, Papasavva P, Stephanou C, Christou S, Sitarou M, Antoniou MN, Lederer CW, Kleanthous M Tags: Haematologica Source Type: research

The early expansion of anergic NKG2Apos/CD56dim/CD16neg natural killer cells represents a therapeutic target in haploidentical haematopoietic stem cell transplantation.
Abstract Natural Killer cells are the first lymphocyte population to reconstitute early after non myelo-ablative and T cell-replete haploidentical hematopoietic stem cell transplantation with post-transplant infusion of cyclophosphamide. The present study characterizes the transient and predominant expansion starting from the 2nd week after haploidentical hematopoietic stem cell transplantation of a donor-derived unconventional subset of NKp46neg-low/CD56dim/CD16neg natural killer cells expressing remarkable high levels of CD94/NKG2A. Both transcription and phenotypic profiles indicated that unconventional NKp46ne...
Source: Haematologica - April 26, 2018 Category: Hematology Authors: Roberto A, Di Vito C, Zaghi E, Mazza EMC, Capucetti A, Calvi M, Tentorio P, Zanon V, Sarina B, Mariotti J, Bramanti S, Tenedini E, Tagliafico E, Bicciato S, Santoro A, Roederer M, Marcenaro E, Castagna L, Lugli E, Mavilio D Tags: Haematologica Source Type: research

The mutational landscape of 18 investigated genes clearly separates four subtypes of myelodysplastic/myeloproliferative neoplasms.
Abstract not required. PMID: 29700173 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - April 26, 2018 Category: Hematology Authors: Meggendorfer M, Jeromin S, Haferlach C, Kern W, Haferlach T Tags: Haematologica Source Type: research

Safety and efficacy of vorinostat, bortezomib, doxorubicin and dexamethasone in a phase I/II study for relapsed or refractory multiple myeloma (VERUMM study: vorinostat in elderly, relapsed and unfit multiple myeloma).
hardt M Abstract This phase I/II trial was conducted to investigate the safety, efficacy, and pharmacodynamics of the pan-HDAC-inhibitor (HDACi) vorinostat combined with bortezomib, doxorubicin, and dexamethasone (VBDD) in patients suffering from relapsed/refractory multiple myeloma (RRMM). In the phase I part of this study, 9/33 patients received dose-escalated vorinostat (100, 200, 300mg), using a 4-day-on and 4-day-off schedule, and a standard 3+3 design. In the phase II part of the study, 24/33 patients were included to further assess VBDD's safety and efficacy. Complementary analyses were performed throughout...
Source: Haematologica - April 19, 2018 Category: Hematology Authors: Waldschmidt JM, Keller A, Ihorst G, Grishina O, Müller S, Wider D, Frey AV, King K, Simon R, May A, Tassone P, Duyster J, Jung M, Raje N, Wäsch R, Engelhardt M Tags: Haematologica Source Type: research

Platelet Munc13-4 regulates hemostasis, thrombosis and airway inflammation.
Abstract Platelet degranulation is crucial for hemostasis and may participate in inflammation. Exocytosis in platelets is mediated by SNARE proteins and should be controlled by Munc13 proteins. We found that platelets express Munc13-2 and -4. We assessed platelet granule exocytosis in Munc13-2 and -4 global and conditional KO mice, and observed that deletion of Munc13-4 ablates dense granule release and indirectly impairs alpha granule exocytosis. We found no exocytic role for Munc13-2 in platelets, not even in the absence of Munc13-4. In vitro, Munc13-4-deficient platelets exhibited defective aggregation at low d...
Source: Haematologica - April 19, 2018 Category: Hematology Authors: Cardenas EI, Breaux K, Da Q, Flores JR, Ramos MA, Tuvim MJ, Burns AR, Rumbaut RE, Adachi R Tags: Haematologica Source Type: research

GFI1 is required for RUNX1/ETO positive acute myeloid leukemia.
ijden BA, Khandanpour C PMID: 29674496 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - April 19, 2018 Category: Hematology Authors: Marneth AE, Botezatu L, Hönes JM, Israël JCL, Schütte J, Vassen L, Lams RF, Bergevoet SM, Groothuis L, Mandoli A, Martens JHA, Huls G, Jansen JH, Dührsen U, Berg T, Möröy T, Wichmann C, Lo MC, Zhang DE, van der Reijden BA, Khandanpour C Tags: Haematologica Source Type: research

Plasma cell proliferative index predicts outcome in immunoglobulin light chain amyloidosis treated with stem cell transplantation.
Abstract The plasma cell proliferative index provides an insight into plasma cell biology in plasma cell disorders and is an important prognostic marker in myeloma and smoldering myeloma. We analyzed the prognostic impact of the plasma cell proliferative index in 513 patients with systemic AL Amyloidosis undergoing stem cell transplantation at the Mayo Clinic between January 2003 and 31 August 2016. Two cohorts were identified; Low or Elevated plasma cell proliferative index. Patients with an Elevated plasma cell proliferative index had more cardiac involvement (56% vs 44%; p=0.01), less renal involvement (55% vs ...
Source: Haematologica - April 19, 2018 Category: Hematology Authors: Sidiqi MH, Aljama M, Jevremovic D, Morice WG, Timm M, Buadi FK, Warsame R, Lacy MQ, Dispenzieri A, Dingli D, Gonsalves WI, Kumar S, Kapoor P, Kourelis T, Leung N, Hogan WJ, Gertz M Tags: Haematologica Source Type: research

Microenvironmental M1 tumor-associated macrophage polarization influences cancer-related anemia in advanced ovarian cancer: key role of Interleukin-6.
ve; A PMID: 29674498 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - April 19, 2018 Category: Hematology Authors: Madeddu C, Gramignano G, Kotsonis P, Coghe F, Atzeni V, Scartozzi M, Macciò A Tags: Haematologica Source Type: research

True, true unrelated? Coexistence of Waldenstrom's macroglobulinemia and cardiac transthyretin Amyloidosis.
PMID: 29674499 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - April 19, 2018 Category: Hematology Authors: Singh A, Geller HI, Alexander KM, Padera RF, Mitchell RN, Dorbala S, Castillo JJ, Falk RH Tags: Haematologica Source Type: research

Novel GPR34 and CCR6 mutation and distinct genetic profiles in MALT lymphomas of different sites.
Abstract MALT lymphoma originates from a background of diverse chronic inflammatory disorders at various anatomic sites. The genetics underlying its development, particularly in those associated with autoimmune disorders, is poorly characterised. By whole exome sequencing of 21 cases of MALT lymphomas of the salivary gland and thyroid, we have identified recurrent somatic mutations in 2 G-protein coupled receptors (GPR34 and CCR6) not previously reported in human malignancies, 3 genes (PIK3CD, TET2, TNFRSF14) not previously implicated in MALT lymphoma, and a further 2 genes (TBL1XR1, NOTCH1) recently described in ...
Source: Haematologica - April 19, 2018 Category: Hematology Authors: Moody S, Thompson JS, Chuang SS, Liu H, Raderer M, Vassiliou G, Wlodarska I, Wu F, Cogliatti S, Robson A, Ashton-Key M, Bi Y, Goodlad J, Du MQ Tags: Haematologica Source Type: research

Evaluation of serum markers for improved detection of autologous blood transfusions.
PMID: 29674501 [PubMed - as supplied by publisher] (Source: Haematologica)
Source: Haematologica - April 19, 2018 Category: Hematology Authors: Cox HD, Miller GD, Lai A, Cushman D, Ganz T, Eichner D Tags: Haematologica Source Type: research