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51. Use of azacitidine and its safety and efficacy in daily clinical practice in The Netherlands: the OCEAN study

Author

Cruijsen, M., primary, van der Velden, W.J.F.M, additional, de Haan, A.F.J., additional, Klein, S. K., additional, Hoogendoorn, M., additional, Tromp, Y., additional, de Valk, B., additional, van Rees, B., additional, de Boer, F., additional, van der Spek, E., additional, Pruijt, J., additional, Verdonck, L.F., additional, Vellenga, E., additional, Blijlevens, N., additional, van de Loosdrecht, A. A., additional, and Huls, G., additional

Published
2020
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53. Association of vitamin B12, methylmalonic acid, and functional parameters

Author

Wolffenbuttel, B. H. R., Wouters, H. J. C. M., de Jong, W. H. A., Huls, G., van der Klauw, M. M., Lifestyle Medicine (LM), Stem Cell Aging Leukemia and Lymphoma (SALL), Life Course Epidemiology (LCE), Center for Liver, Digestive and Metabolic Diseases (CLDM), and Guided Treatment in Optimal Selected Cancer Patients (GUTS)

Subjects
methylmalonic acid, Epidemiology, COBALAMIN, DISORDERS, DEMENTIA, HOMOCYSTEINE, vitamin B12, COGNITIVE PERFORMANCE, DEFICIENCY, SERUM VITAMIN-B-12, MARKERS, muscle strength, NHANES, FOLATE, HOLOTRANSCOBALAMIN
Abstract

Introduction: Diagnosis of vitamin B12 deficiency is difficult, as there is no conclusive single test for this disorder. We evaluated the association of serum B12 and methylmalonic acid (MMA) with haematologic parameters and physical and cognitive functioning in an effort to use such clinical parameters to improve the interpretation of serum values. Methods: We used data of participants > 19 years of age from NHANES 2011-2012 and 2013-2014, a cross-sectional survey in the United States. Functional status was assessed with questionnaires on current health condition, disability, hospital utilisation, cognitive functioning, mental health and depression, and physical functioning. Muscle strength assessed with a handgrip dynamometer was used as a performance parameter. Results were evaluated both for the entire population and participants of Western European descent. Because renal function influences MMA concentrations and is a proxy for both frailty and comorbidity, all results were additionally stratified for individuals with normal vs impaired renal function (eGFR < 60 ml/min). Results: In total, data of 9645 participants (mean age 49 (SD 17) years, 49.3% males) were included. Out of all participants with serum B12 < 140, 140-300, and 301-1000 pmol/l, 56.2%, 13.5%, and 4.1%, respectively had elevated MMA. MMA concentrations were more strongly associated with poor functional status and physical performance than serum B12. We identified a significant and independent association of MMA concentrations, as well as haemoglobin and co-morbidity with muscle strength. Conclusions/interpretations: A large proportion of individuals with a decreased serum B12 concentration still has normal MMA concentrations. Elevated MMA concentrations were more strongly associated with poor functional performance than serum B12.

Published
2020

56. Characterization of post-transplant lymphoproliferative disorder with semi-quantitative FDG-PET/CT

Author

Montes De Jesus, F., Noordzij, W., Kahle, X., Nijland, M., Verschuuren, E., Dierckx, R., Van Der Meerten, T., Van Der Bij, W., Huls, G., Kwee, T., Glaudemans, A., Molecular Neuroscience and Ageing Research (MOLAR), Stem Cell Aging Leukemia and Lymphoma (SALL), Guided Treatment in Optimal Selected Cancer Patients (GUTS), Translational Immunology Groningen (TRIGR), ​Basic and Translational Research and Imaging Methodology Development in Groningen (BRIDGE), and Groningen Institute for Organ Transplantation (GIOT)

Subjects
cancer patient, clinical article, posttransplant lymphoproliferative disease, conference abstract, quantitative analysis, software, adult, human cell, practice guideline, immunosuppressive treatment, lymph node, PET-CT scanner, surgical procedures, operative, classical Hodgkin lymphoma, female, peak standardized uptake value, male, hemic and lymphatic diseases, histopathology, positron emission tomography-computed tomography, controlled study, maximum standardized uptake value, human, mean standardized uptake value
Abstract

Aim/Introduction: One of the most dire complications of hematopoietic stem cell (HSCT) and solid organ transplantation (SOT) is the development of post-transplant lymphoproliferative disorder (PTLD). PTLD compromises a broad spectrum of disorders classified by the 2017 World Health Organization (WHO) in non-destructive, polymorphic, monomorphic and classic Hodgkin lymphoma. Distinct morphologies are associated with a more favorable clinical course and better response to initial treatment. Reduction of immunosuppression, commonly used as first-line treatment, has been associated with higher response rates in non-destructive and polymorphic PTLD, while a more aggressive therapy is advised for monomorphic PTLD. Biopsy is the reference standard for PTLD diagnosis and classification, but may not always be safely possible. Therefore, there is a need for non-invasive imaging-based tools. Materials and Methods: All patients with histopathologically proven PTLD at the UMC Groningen were included in this study between January 2010 to March 2019. FDG-PET/CT scans were performed on a Siemens Biograph mCT camera, according to EANM procedure guidelines for tumor imaging and reconstruction parameters compliant with EARL recommendations. Semi-quantitative measurements (SUVmax, SUVpeak and SUVmean) were performed using dedicated Hermes Hybrid 3D software with the ?Tumor Finder? application. Semi-quantitative measurements were obtained from the biopsy site or in cases in which a biopsy was performed before the scan from the nearest lymph node in the same lymph node region Results: In total 41 patients were included. From those, 27 were monomorphic PTLD and 14 were ?other PTLD morphologies?, including non-destructive (n=4), polymorphic (n=9) and Hodgkin-like PTLD (n=1). Median SUVmax, SUVpeak, SUVmean values were statistically significantly higher in monomorphic PTLD than in ?other PTLD morphologies (p

Published
2019

57. Use of azacitidine and its safety and efficacy in daily clinical practice in The Netherlands: the OCEAN study

Author

Cruijsen, M.J., Velden, W.J.F.M. van der, Haan, A.F.J. de, Klein, S.K., Hoogendoorn, M., Tromp, Y., Valk, B. de, Rees, B. van, Boer, F. de, Spek, E. van der, Pruijt, J., Verdonck, L.F., Vellenga, E., Blijlevens, N.M.A., Loosdrecht, A.A. van de, Huls, G., Cruijsen, M.J., Velden, W.J.F.M. van der, Haan, A.F.J. de, Klein, S.K., Hoogendoorn, M., Tromp, Y., Valk, B. de, Rees, B. van, Boer, F. de, Spek, E. van der, Pruijt, J., Verdonck, L.F., Vellenga, E., Blijlevens, N.M.A., Loosdrecht, A.A. van de, and Huls, G.

Abstract

Contains fulltext : 229349.pdf (Publisher’s version ) (Closed access)

Published
2020

58. Efficacy of MSC for steroid-refractory acute GVHD associates with MSC donor age and a defined molecular profile

Author

Wagen, L.E. van der, Miranda-Bedate, A., Janssen, A., Fernando, F., Appukudige, N., Dooremalen, S. van, Westinga, K., Admiraal, R., Lorenowicz, M.J., Huls, G., Janssen, J, Broers, A.E., Velden, W.J.F.M. van der, Kooy, R. van, Hazenberg, M.D., Haar, C. De, Lindemans, C., Boelens, J. Jan, Kuball, J., Wagen, L.E. van der, Miranda-Bedate, A., Janssen, A., Fernando, F., Appukudige, N., Dooremalen, S. van, Westinga, K., Admiraal, R., Lorenowicz, M.J., Huls, G., Janssen, J, Broers, A.E., Velden, W.J.F.M. van der, Kooy, R. van, Hazenberg, M.D., Haar, C. De, Lindemans, C., Boelens, J. Jan, and Kuball, J.

Abstract

Contains fulltext : 229413.pdf (Publisher’s version ) (Closed access)

Published
2020

59. Ibrutinib added to 10-day decitabine for older patients with AML and higher risk MDS

Author

Huls, G. (Gerwin), Chitu, D.A., Pabst, T. (Thomas), Klein, SK, Stussi, G. (Georg), Griskevicius, L., Valk, P.J.M. (Peter), Cloos, J. (Jacqueline), de Loosdrecht, AAV, Breems, D.A. (Dimitri), van Lammeren - Venema, D. (Danielle), van Zeventer, I., Boersma, R., Jongen-Lavrencic, M. (Mojca), Fehr, M., Hoogendoorn, M. (Martine), Manz, MG, Söhne, M. (Maaike), Kooy, RV, Deeren, D., van der Poel, M.W.M., Legdeur, MC, Tick, L.W. (Lidwine), Chalandon, Y. (Yves), Ammatuna, E., Blum, S., Löwenberg, B. (Bob), Ossenkoppele, G.J. (Gert), Huls, G. (Gerwin), Chitu, D.A., Pabst, T. (Thomas), Klein, SK, Stussi, G. (Georg), Griskevicius, L., Valk, P.J.M. (Peter), Cloos, J. (Jacqueline), de Loosdrecht, AAV, Breems, D.A. (Dimitri), van Lammeren - Venema, D. (Danielle), van Zeventer, I., Boersma, R., Jongen-Lavrencic, M. (Mojca), Fehr, M., Hoogendoorn, M. (Martine), Manz, MG, Söhne, M. (Maaike), Kooy, RV, Deeren, D., van der Poel, M.W.M., Legdeur, MC, Tick, L.W. (Lidwine), Chalandon, Y. (Yves), Ammatuna, E., Blum, S., Löwenberg, B. (Bob), and Ossenkoppele, G.J. (Gert)

Abstract

The treatment of older, unfit patients with acute myeloid leukemia (AML) is challenging. Based on preclinical data of Bruton tyrosine kinase expression/phosphorylation and ibrutinib cytotoxicity in AML blasts, we conducted a randomized phase 2 multicenter study to assess the tolerability and efficacy of the addition of ibrutinib to 10-day decitabine in unfit (ie, Hematopoietic Cell Transplantation Comorbidity Index ≥3) AML patients and higher risk myelodysplasia patients (HOVON135/SAKK30/15 trial). In total, 144 eligible patients were randomly (1:1) assigned to either 10-day decitabine combined with ibrutinib (560 mg; sequentially given, starting the day after the last dose of decitabine) (n = 72) or to 10-day decitabine (n = 72). The addition of ibrutinib was well tolerated, and the number of adverse events was comparable for both arms. In the decitabine plus ibrutinib arm, 41% reached complete remission/complete remission with incomplete hematologic recovery (CR/CRi), the median overall survival (OS) was 11 months, and 2-year OS was 27%; these findings compared with 50% CR/CRi, median OS of 11.5 months, and 2-year OS of 21% for the decitabine group (not significant). Extensive molecular profiling at diagnosis revealed that patients with STAG2, IDH2, and ASXL1 mutations had significantly lower CR/CRi rates, whereas patients with mutations in TP53 had significantly higher CR/CRi rates. Furthermore, multicolor flow cytometry revealed that after 3 cycles of treatment, 28 (49%) of 57 patients with available bone marrow samples had no measurable residual disease. In this limited number of cases, measurable residual disease revealed no apparent impact on event-free survival and OS. In conclusion, the addition of ibrutinib does not improve the therapeutic efficacy of decitabine. This trial was registered at the Netherlands Trial Register (NL5751 [NTR6017]) and has EudraCT number 2015-002855-85.

Published
2020
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60. Correction: Lenalidomide added to standard intensive treatment for older patients with AML and high-risk MDS

Author

UCL - (SLuc) Centre du cancer, UCL - SSS/IREC/SLUC - Pôle St.-Luc, UCL - (SLuc) Service d'hématologie, UCL - SSS/DDUV/MEXP - Médecine expérimentale, UCL - SSS/IREC/MONT - Pôle Mont Godinne, UCL - (MGD) Service d'hématologie, Ossenkoppele, G. J., Breems, D. A., Stuessi, G., van Norden, Y., Bargetzi, M., Biemond, B. J., A von dem Borne, P., Chalandon, Y., Cloos, J., Deeren, D., Fehr, M., Gjertsen, B., Graux, Carlos, Huls, G., Janssen, J. J. J. W., Jaspers, A., Jongen-Lavrencic, M., de Jongh, E., Klein, S. K., van der Klift, M., van Marwijk Kooy, M., Maertens, J., Michaux, L., van der Poel, M. W. M., van Rhenen, A., Tick, L., Valk, P., Vekemans, Marie-Christiane, van der Velden, W. J. F. M., de Weerdt, O., Pabst, T., Manz, M., Löwenberg, B., Havelange, Violaine, Sonet, Anne, UCL - (SLuc) Centre du cancer, UCL - SSS/IREC/SLUC - Pôle St.-Luc, UCL - (SLuc) Service d'hématologie, UCL - SSS/DDUV/MEXP - Médecine expérimentale, UCL - SSS/IREC/MONT - Pôle Mont Godinne, UCL - (MGD) Service d'hématologie, Ossenkoppele, G. J., Breems, D. A., Stuessi, G., van Norden, Y., Bargetzi, M., Biemond, B. J., A von dem Borne, P., Chalandon, Y., Cloos, J., Deeren, D., Fehr, M., Gjertsen, B., Graux, Carlos, Huls, G., Janssen, J. J. J. W., Jaspers, A., Jongen-Lavrencic, M., de Jongh, E., Klein, S. K., van der Klift, M., van Marwijk Kooy, M., Maertens, J., Michaux, L., van der Poel, M. W. M., van Rhenen, A., Tick, L., Valk, P., Vekemans, Marie-Christiane, van der Velden, W. J. F. M., de Weerdt, O., Pabst, T., Manz, M., Löwenberg, B., Havelange, Violaine, and Sonet, Anne

Abstract

An amendment to this paper has been published and can be accessed via a link at the top of the paper.

Published
2020

61. Ibrutinib added to 10-day decitabine for older patients with AML and higher risk MDS

Author

Huls, G, Chitu, Dana, Pabst, T, Klein, SK, Stussi, G, Griskevicius, L, Valk, Peter, Cloos, J, de Loosdrecht, AAV, Breems, D, van Lammeren-Venema, D, van Zeventer, I, Boersma, R, Jongen - Lavrencic, Mojca, Fehr, M, Hoogendoorn - Lips, EJI, Manz, MG, Sohne, M, Kooy, RV, Deeren, D, van der Poel, MWM, Legdeur, MC, Tick, L, Chalandon, Y, Ammatuna, E, Blum, S, Löwenberg, Bob, Ossenkoppele, GJ, Huls, G, Chitu, Dana, Pabst, T, Klein, SK, Stussi, G, Griskevicius, L, Valk, Peter, Cloos, J, de Loosdrecht, AAV, Breems, D, van Lammeren-Venema, D, van Zeventer, I, Boersma, R, Jongen - Lavrencic, Mojca, Fehr, M, Hoogendoorn - Lips, EJI, Manz, MG, Sohne, M, Kooy, RV, Deeren, D, van der Poel, MWM, Legdeur, MC, Tick, L, Chalandon, Y, Ammatuna, E, Blum, S, Löwenberg, Bob, and Ossenkoppele, GJ

Published
2020

63. Richtlijn Acute promyelocytenleukemie (APL): richtlijnen voor diagnostiek en behandeling

Author

Huls, G, Löwenberg, Bob, van de Loosdrecht, AA, Janssen, JJWM, Jongen - Lavrencic, Mojca, Raaijmakers, Marc, Wouters, Bas, van den Borne, P, Kuball, J, Biemond, B, van Gelder, M, van der Velden, WJFM, de Weerdt, O, Marwijk Kooy, R, Klein, S, Ammatuna, Emanuele, Poel, M, van Rhenen, A, Jong, JJM, Valk, Peter, van der Reijden, B, Cornelissen, Jan, Vellenga, E, Ossenkoppele, G, and Hematology

Published
2019

64. Diagnostic performance of FDG-PET/CT of post-transplant lymphoproliferative disorder and factors affecting diagnostic yield

Author

Montes de Jesus, F. M., primary, Kwee, T. C., additional, Kahle, X. U., additional, Nijland, M., additional, van Meerten, T., additional, Huls, G., additional, Dierckx, R. A. J. O., additional, Rosati, S., additional, Diepstra, A., additional, van der Bij, W., additional, Verschuuren, E. A. M., additional, Glaudemans, A. W. J. M., additional, and Noordzij, W., additional

Published
2019
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71. Labile plasma iron levels predict survival in patients with lower-risk myelodysplastic syndromes

Author

de Swart, L. Reiniers, C. Bagguley, T. van Marrewijk, C. Bowen, D. Hellström-Lindberg, E. Tatic, A. Symeonidis, A. Huls, G. Cermak, J. van de Loosdrecht, A.A. Garelius, H. Culligan, D. Macheta, M. Spanoudakis, M. Panagiotidis, P. Krejci, M. Blijlevens, N. Langemeijer, S. Droste, J. Swinkels, D.W. Smith, A. de Witte, T. EUMDS Steering Committee

Abstract

Red blood cell transfusions remain one of the cornerstones in supportive care of lower-risk patients with myelodysplastic syndromes. We hypothesized that patients develop oxidant-mediated tissue injury through the formation of toxic iron species, caused either by red blood cell transfusions or by ineffective erythropoiesis. We analyzed serum samples from 100 lower-risk patients with myelodysplastic syndromes at six-month intervals for transferrin saturation, hepcidin-25, growth differentiation factor 15, soluble transferrin receptor, non-transferrin bound iron and labile plasma iron in order to evaluate temporal changes in iron metabolism and the presence of potentially toxic iron species and their impact on survival. Hepcidin levels were low in 34 patients with ringed sideroblasts compared to 66 patients without. Increases of hepcidin and non-transferrin bound iron levels were visible early in follow-up of all transfusion-dependent patient groups. Hepcidin levels significantly decreased over time in transfusion-independent patients with ringed sideroblasts. Increased soluble transferrin receptor levels in transfusion-independent patients with ringed sideroblasts confirmed the presence of ineffective erythropoiesis and suppression of hepcidin production in these patients. Detectable labile plasma iron levels in combination with high transferrin saturation levels occurred almost exclusively in patients with ringed sideroblasts and all transfusiondependent patient groups. Detectable labile plasma iron levels in transfusion-dependent patients without ringed sideroblasts were associated with decreased survival. In conclusion, toxic iron species occurred in all transfusion-dependent patients and in transfusion-independent patients with ringed sideroblasts. Labile plasma iron appeared to be a clinically relevant measure for potential iron toxicity and a prognostic factor for survival in transfusion-dependent patients. © 2018 Ferrata Storti Foundation.

Published
2018

73. Characterization of acute myeloid leukemia based on levels of global hydroxymethylation

Author

Kroeze, L.I., Aslanyan, M.G., Rooij, A. van, Koorenhof-Scheele, T.N., Massop, M., Carell, T., Boezeman, J.B., Marie, J.P., Halkes, C.J.M., Witte, T. de, Huls, G., Suciu, S., Wevers, R.A., Reijden, B.A. van der, Jansen, J.H., EORTC Leukemia Grp, and GIMEMA

Subjects
Adult, Oncology, medicine.medical_specialty, IDH1, Adolescent, Cancer development and immune defence Radboud Institute for Molecular Life Sciences [Radboudumc 2], DNA Mutational Analysis, Immunology, Biology, Biochemistry, IDH2, DNA Methyltransferase 3A, Dioxygenases, Cytosine, Young Adult, Proto-Oncogene Proteins, Internal medicine, medicine, Humans, Disorders of movement Radboud Institute for Molecular Life Sciences [Radboudumc 3], DNA (Cytosine-5-)-Methyltransferases, Young adult, Promoter Regions, Genetic, Survival analysis, Aged, Hazard ratio, Cancer, Myeloid leukemia, Cell Biology, Hematology, DNA Methylation, Middle Aged, Prognosis, medicine.disease, Survival Analysis, Isocitrate Dehydrogenase, DNA-Binding Proteins, Leukemia, Leukemia, Myeloid, Acute Disease, Mutation, 5-Methylcytosine, Inflammatory diseases Radboud Institute for Molecular Life Sciences [Radboudumc 5]
Abstract

Contains fulltext : 137617.pdf (Publisher’s version ) (Closed access) Patients with acute myeloid leukemia (AML) frequently harbor mutations in genes involved in the DNA (hydroxy)methylation pathway (DNMT3A, TET2, IDH1, and IDH2). In this study, we measured 5-hydroxymethylcytosine (5hmC) levels in 206 clinically and molecularly well-characterized younger adult AML patients (

Published
2014
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74. GFI1 is required for RUNX1/ETO positive acute myeloid leukemia

Author

Marneth, A.E., Botezatu, L., Hönes, J.M., Israël, J.C.L., Schütte, J., Vassen, L., Lams, R.F., Bergevoet, S.M., Groothuis, L., Mandoli, A., Martens, J.H.A., Huls, G., Jansen, J.H., Dührsen, U., Berg, T. van den, Möröy, T., Wichmann, C., Lo, M.C., Zhang, D.E., Reijden, B.A. van der, Khandanpour, C., Marneth, A.E., Botezatu, L., Hönes, J.M., Israël, J.C.L., Schütte, J., Vassen, L., Lams, R.F., Bergevoet, S.M., Groothuis, L., Mandoli, A., Martens, J.H.A., Huls, G., Jansen, J.H., Dührsen, U., Berg, T. van den, Möröy, T., Wichmann, C., Lo, M.C., Zhang, D.E., Reijden, B.A. van der, and Khandanpour, C.

Abstract

Contains fulltext : 196389.pdf (publisher's version ) (Open Access)

Published
2018

75. The phenotypic spectrum of germline YARS2 variants: from isolated sideroblastic anemia to mitochondrial myopathy, lactic acidosis and sideroblastic anemia 2

Author

Riley, LG, Heeney, MM, Rudinger-Thirion, J, Frugier, M, Campagna, DR, Zhou, R, Hale, GA, Hilliard, LM, Kaplan, JA, Kwiatkowski, JL, Sieff, CA, Steensma, DP, Rennings, AJ, Simons, A, Schaap, N, Roodenburg, RJ, Kleefstra, T, Arenillas, L, Fita-Torro, J, Ahmed, R, Abboud, M, Bechara, E, Farah, R, Tamminga, RYJ, Bottomley, SS, Sanchez, M, Huls, G, Swinkels, DW, Christodoulou, J, Fleming, MD, Riley, LG, Heeney, MM, Rudinger-Thirion, J, Frugier, M, Campagna, DR, Zhou, R, Hale, GA, Hilliard, LM, Kaplan, JA, Kwiatkowski, JL, Sieff, CA, Steensma, DP, Rennings, AJ, Simons, A, Schaap, N, Roodenburg, RJ, Kleefstra, T, Arenillas, L, Fita-Torro, J, Ahmed, R, Abboud, M, Bechara, E, Farah, R, Tamminga, RYJ, Bottomley, SS, Sanchez, M, Huls, G, Swinkels, DW, Christodoulou, J, and Fleming, MD

Abstract

YARS2 variants have previously been described in patients with myopathy, lactic acidosis and sideroblastic anemia 2 (MLASA2). YARS2 encodes the mitochondrial tyrosyl-tRNA synthetase, which is responsible for conjugating tyrosine to its cognate mt-tRNA for mitochondrial protein synthesis. Here we describe 14 individuals from 11 families presenting with sideroblastic anemia and YARS2 variants that we identified using a sideroblastic anemia gene panel or exome sequencing. The phenotype of these patients ranged from MLASA to isolated congenital sideroblastic anemia. As in previous cases, inter- and intra-familial phenotypic variability was observed, however, this report includes the first cases with isolated sideroblastic anemia and patients with biallelic YARS2 variants that have no clinically ascertainable phenotype. We identified ten novel YARS2 variants and three previously reported variants. In vitro amino-acylation assays of five novel missense variants showed that three had less effect on the catalytic activity of YARS2 than the most commonly reported variant, p.(Phe52Leu), associated with MLASA2, which may explain the milder phenotypes in patients with these variants. However, the other two missense variants had a more severe effect on YARS2 catalytic efficiency. Several patients carried the common YARS2 c.572 G>T, p.(Gly191Val) variant (minor allele frequency =0.1259) in trans with a rare deleterious YARS2 variant. We have previously shown that the p.(Gly191Val) variant reduces YARS2 catalytic activity. Consequently, we suggest that biallelic YARS2 variants, including severe loss-of-function alleles in trans of the common p.(Gly191Val) variant, should be considered as a cause of isolated congenital sideroblastic anemia, as well as the MLASA syndromic phenotype.

Published
2018

76. The european hematology association roadmap for european hematology research: A consensus document

Author

Engert, A. Balduini, C. Brand, A. Coiffier, B. Cordonnier, C. Döhner, H. De Wit, T.D. Eichinger, S. Fibbe, W. Green, T. De Haas, F. Iolascon, A. Jaffredo, T. Rodeghiero, F. Sall Es, G. Schuringa, J.J. André, M. Andre-Schmutz, I. Bacigalupo, A. Bochud, P.-Y. Den Boer, M. Bonini, C. Camaschella, C. Cant, A. Cappellini, M.D. Cazzola, M. Celso, C.L. Dimopoulos, M. Douay, L. Dzierzak, E. Einsele, H. Ferreri, A. De Franceschi, L. Gaulard, P. Gottgens, B. Greinacher, A. Gresele, P. Gribben, J. De Haan, G. Hansen, J.-B. Hochhaus, A. Kadir, R. Kaveri, S. Kouskoff, V. Kühne, T. Kyrle, P. Ljungman, P. Maschmeyer, G. Méndez-Ferrer, S. Milsom, M. Mummery, C. Ossenkoppele, G. Pecci, A. Peyvandi, F. Philipsen, S. Reitsma, P. Ribera, J.M. Risitano, A. Rivella, S. Ruf, W. Schroeder, T. Scully, M. Socie, G. Staal, F. Stanworth, S. Stauder, R. Stilgenbauer, S. Tamary, H. Theilgaard-Mönch, K. Thein, S.L. Tilly, H. Trneny, M. Vainchenker, W. Vannucchi, A.M. Viscoli, C. Vrielink, H. Zaaijer, H. Zanella, A. Zolla, L. Zwaginga, J.J. Martinez, P.A. Van Den Akker, E. Allard, S. Anagnou, N. Andolfo, I. Andrau, J.-C. Angelucci, E. Anstee, D. Aurer, I. Avet-Loiseau, H. Aydinok, Y. Bakchoul, T. Balduini, A. Barcellini, W. Baruch, D. Baruchel, A. Bayry, J. Bento, C. Van Den Berg, A. Bernardi, R. Bianchi, P. Bigas, A. Biondi, A. Bohonek, M. Bonnet, D. Borchmann, P. Borregaard, N. Brækkan, S. Van Den Brink, M. Brodin, E. Bullinger, L. Buske, C. Butzeck, B. Cammenga, J. Campo, E. Carbone, A. Cervantes, F. Cesaro, S. Charbord, P. Claas, F. Cohen, H. Conard, J. Coppo, P. Vives Corron, J.-L. Da Costa, L. Davi, F. Delwel, R. Dianzani, I. Domanović, D. Donnelly, P. Drnovšek, T.D. Dreyling, M. Du, M.-Q. Dufour, C. Durand, C. Efremov, D. Eleftheriou, A. Elion, J. Emonts, M. Engelhardt, M. Ezine, S. Falkenburg, F. Favier, R. Federico, M. Fenaux, P. Fitzgibbon, J. Flygare, J. Foà, R. Forrester, L. Galacteros, F. Garagiola, I. Gardiner, C. Garraud, O. Van Geet, C. Geiger, H. Geissler, J. Germing, U. Ghevaert, C. Girelli, D. Godeau, B. Gökbuget, N. Goldschmidt, H. Goodeve, A. Graf, T. Graziadei, G. Griesshammer, M. Gruel, Y. Guilhot, F. Von Gunten, S. Gyssens, I. Halter, J. Harrison, C. Harteveld, C. Hellström-Lindberg, E. Hermine, O. Higgs, D. Hillmen, P. Hirsch, H. Hoskin, P. Huls, G. Inati, A. Johnson, P. Kattamis, A. Kiefel, V. Kleanthous, M. Klump, H. Krause, D. Hovinga, J.K. Lacaud, G. Lacroix-Desmazes, S. Landman-Parker, J. Legouill, S. Lenz, G. Von Lilienfeld-Toal, M. Von Lindern, M. Lopez-Guillermo, A. Lopriore, E. Lozano, M. Macintyre, E. Makris, M. Mannhalter, C. Martens, J. Mathas, S. Matzdorff, A. Medvinsky, A. Menendez, P. Migliaccio, A.R. Miharada, K. Mikulska, M. Minard, V. Montalbán, C. De Montalembert, M. Montserrat, E. Morange, P.-E. Mountford, J. Muckenthaler, M. Müller-Tidow, C. Mumford, A. Nadel, B. Navarro, J.-T. El Nemer, W. Noizat-Pirenne, F. O’Mahony, B. Oldenburg, J. Olsson, M. Oostendorp, R. Palumbo, A. Passamonti, F. Patient, R. De Latour, R.P. Pflumio, F. Pierelli, L. Piga, A. Pollard, D. Raaijmakers, M. Radford, J. Rambach, R. Koneti Rao, A. Raslova, H. Rebulla, P. Rees, D. Ribrag, V. Rijneveld, A. Rinalducci, S. Robak, T. Roberts, I. Rodrigues, C. Rosendaal, F. Rosenwald, A. Rule, S. Russo, R. Saglio, G. Sanchez, M. Scharf, R.E. Schlenke, P. Semple, J. Sierra, J. So-Osman, C. Soria, J.M. Stamatopoulos, K. Stegmayr, B. Stunnenberg, H. Swinkels, D. Barata, J.P.T. Taghon, T. Taher, A. Terpos, E. Thachil, J. Tissot, J.D. Touw, I. Toye, A. Trappe, R. Traverse-Glehen, A. Unal, S. Vaulont, S. Viprakasit, V. Vitolo, U. Van Wijk, R. Wójtowicz, A. Zeerleder, S. Zieger, B. EHA Roadmap for European Hematology Research

Abstract

The European Hematology Association (EHA) Roadmap for European Hematology Research highlights major achievements in diagnosis and treatment of blood disorders and identifies the greatest unmet clinical and scientific needs in those areas to enable better funded, more focused European hematology research. Initiated by the EHA, around 300 experts contributed to the consensus document, which will help European policy makers, research funders, research organizations, researchers, and patient groups make better informed decisions on hematology research. It also aims to raise public awareness of the burden of blood disorders on European society, which purely in economic terms is estimated at ∈ European Hematology Association (EHA) Roadmap for European Hematology Research highlights major achievements in diagnosis and treatment of blood disorders and identifies the greatest unmet clinical and scientific needs in those areas to enable better fu treatments, sometimes in revolutionary ways. This progress highlights the potential of focused basic research programs such as this EHA Roadmap. The EHA Roadmap identifies nine ‘sections’ in hematology: normal hematopoiesis, malignant lymphoid and myeloid diseases, anemias and related diseases, platelet disorders, blood coagulation and hemostatic disorders, transfusion medicine, infections in hematology, and hematopoietic stem cell transplantation. These sections span 60 smaller groups of diseases or disorders. The EHA Roadmap identifies priorities and needs across the field of hematology, including those to develop targeted therapies based on genomic profiling and chemical biology, to eradicate minimal residual malignant disease, and to develop cellular immunotherapies, combination treatments, gene therapies, hematopoietic stem cell treatments, and treatments that are better tolerated by elderly patients. © 2016 Ferrata Storti Foundation.

Published
2016

80. Comparative value of post-remission treatment in cytogenetically normal AML subclassified by NPM1 and FLT3-ITD allelic ratio

Author

MS Hematologie, Infection & Immunity, Regenerative Medicine and Stem Cells, CTI Kuball, Cancer, Versluis, J, In 't Hout, F E M, Devillier, R, van Putten, W L J, Manz, M G, Vekemans, M-C, Passweg, J R, Maertens, J, Kuball, J, Biemond, B J, Valk, P J M, van der Reijden, B A, Meloni, G, Schouten, H C, Vellenga, E, Pabst, T, Willemze, R, Löwenberg, B, Ossenkoppele, G, Baron, F, Huls, G, Cornelissen, J J, MS Hematologie, Infection & Immunity, Regenerative Medicine and Stem Cells, CTI Kuball, Cancer, Versluis, J, In 't Hout, F E M, Devillier, R, van Putten, W L J, Manz, M G, Vekemans, M-C, Passweg, J R, Maertens, J, Kuball, J, Biemond, B J, Valk, P J M, van der Reijden, B A, Meloni, G, Schouten, H C, Vellenga, E, Pabst, T, Willemze, R, Löwenberg, B, Ossenkoppele, G, Baron, F, Huls, G, and Cornelissen, J J

Published
2017

82. CHARACTERIZATION OF GLOBAL HYDROXYMETHYLATION LEVELS IN AML

Author

Kroeze, L.I., Aslanyan, M.G., Rooij, A. van, Koorenhof-Scheele, T.N., Massop, M., Carell, T., Boezeman, J.B., Marie, J.P., Halkes, C.J., Witte, T. de, Huls, G., Suciu, S., Wevers, R.A., Reijden, B.A. van der, and Jansen, J.H.

Published
2014

83. Enteropathy associated T‐cell lymphoma: a population‐based cohort study on incidence, treatment and outcome in The Netherlands.

Author

Meeuwes, F. O., Brink, M., Plattel, W. J., Vermaat, J. S. P., Kersten, M. J., Wondergem, M., Visser, O., van der Poel, M. W. M., Oostvogels, R., Woei‐A‐Jin, F. J. S. H., Böhmer, L. H., Huls, G. A., and Nijland, M.

Subjects
T-cell lymphoma, INTESTINAL diseases, COHORT analysis, OVERALL survival, STEM cell transplantation
Abstract

B Conclusion: b The prognosis of patients with EATL is dismal with a high mortality rate early after diagnosis and a lack of a survival plateau up to 5 years after diagnosis. B Introduction: b Enteropathy associated T-cell lymphoma (EATL) is a rare peripheral T-cell lymphoma (PTCL) associated with celiac disease that has a poor prognosis. This nationwide Dutch study describes the incidence, treatment and outcome of all reported EATL cases. [Extracted from the article]

Published
2023
Full Text
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84. Comparative value of post-remission treatment in cytogenetically normal AML subclassified by NPM1 and FLT3-ITD allelic ratio

Author

UCL - (SLuc) Service d'hématologie, Versluis, J, in ‘t Hout, F E M, Devillier, R, van Putten, W L J, Manz, M G, Vekemans, M -C, Legdeur, M -C, Passweg, J R, Maertens, J, Kuball, J, Biemond, B J, Valk, P J M, van der Reijden, B A, Meloni, G, Schouten, H C, Vellenga, E, Pabst, T, Willemze, R, Löwenberg, B, Ossenkoppele, G, Baron, F, Huls, G, Cornelissen, J J, UCL - (SLuc) Service d'hématologie, Versluis, J, in ‘t Hout, F E M, Devillier, R, van Putten, W L J, Manz, M G, Vekemans, M -C, Legdeur, M -C, Passweg, J R, Maertens, J, Kuball, J, Biemond, B J, Valk, P J M, van der Reijden, B A, Meloni, G, Schouten, H C, Vellenga, E, Pabst, T, Willemze, R, Löwenberg, B, Ossenkoppele, G, Baron, F, Huls, G, and Cornelissen, J J

Abstract

Post-remission treatment (PRT) in patients with cytogenetically normal (CN) acute myeloid leukemia (AML) in first complete remission (CR1) is debated. We studied 521 patients with CN-AML in CR1, for whom mutational status of NPM1 and FLT3-ITD was available, including the FLT3-ITD allelic ratio. PRT consisted of reduced intensity conditioning (RIC) allogeneic hematopoietic stem cell transplantation (alloHSCT) (n=68), myeloablative conditioning (MAC) alloHSCT (n=137), autologous hematopoietic stem cell transplantation (autoHSCT) (n=168) or chemotherapy (n=148). Favorable overall survival (OS) was found for patients with mutated NPM1 without FLT3-ITD (71±4%). Outcome in patients with a high FLT3-ITD allelic ratio appeared to be very poor with OS and relapse-free survival (RFS) of 23±8% and 12±6%, respectively. Patients with wild-type NPM1 without FLT3-ITD or with a low allelic burden of FLT3-ITD were considered as intermediate-risk group because of similar OS and RFS at 5 years, in which PRT by RIC alloHSCT resulted in better OS and RFS as compared with chemotherapy (hazard ratio (HR) 0.56, P=0.022 and HR 0.50, P=0.004, respectively) or autoHSCT (HR 0.60, P=0.046 and HR 0.60, P=0.043, respectively). The lowest cumulative incidence of relapse (23±4%) was observed following MAC alloHSCT. These results suggest that alloHSCT may be preferred in patients with molecularly intermediate-risk CN-AML, while the choice of conditioning type may be personalized according to risk for non-relapse mortality.

Published
2016

85. The European Hematology Association Roadmap for European Hematology Research: a consensus document.

Author

EHA Roadmap for European Hematology, Research, Engert, A., Balduini, C., Brand, A., Coiffier, B., Cordonnier, C., Döhner, H., de Wit TD., Eichinger, S., Fibbe, W., Green, T., de Haas, F., Iolascon, A., Jaffredo, T., Rodeghiero, F., Salles, G., Schuringa, JJ., André, M., Andre-Schmutz, I., Bacigalupo, A., Bochud, PY., Boer, Md., Bonini, C., Camaschella, C., Cant, A., Cappellini, MD., Cazzola, M., Celso, CL., Dimopoulos, M., Douay, L., Dzierzak, E., Einsele, H., Ferreri, A., De Franceschi, L., Gaulard, P., Gottgens, B., Greinacher, A., Gresele, P., Gribben, J., de Haan, G., Hansen, JB., Hochhaus, A., Kadir, R., Kaveri, S., Kouskoff, V., Kühne, T., Kyrle, P., Ljungman, P., Maschmeyer, G., Méndez-Ferrer£££Simón£££ S., Milsom, M., Mummery, C., Ossenkoppele, G., Pecci, A., Peyvandi, F., Philipsen, S., Reitsma, P., Ribera, JM., Risitano, A., Rivella, S., Ruf, W., Schroeder, T., Scully, M., Socie, G., Staal, F., Stanworth, S., Stauder, R., Stilgenbauer, S., Tamary, H., Theilgaard-Mönch, K., Thein, SL., Tilly, H., Trneny, M., Vainchenker, W., Vannucchi, AM., Viscoli, C., Vrielink, H., Zaaijer, H., Zanella, A., Zolla, L., Zwaginga, JJ., Martinez, PA., van den Akker, E., Allard, S., Anagnou, N., Andolfo, I., Andrau, JC., Angelucci, E., Anstee, D., Aurer, I., Avet-Loiseau, H., Aydinok, Y., Bakchoul, T., Balduini, A., Barcellini, W., Baruch, D., Baruchel, A., Bayry, J., Bento, C., van den Berg, A., Bernardi, R., Bianchi, P., Bigas, A., Biondi, A., Bohonek, M., Bonnet, D., Borchmann, P., Borregaard, N., Brækkan, S., van den Brink, M., Brodin, E., Bullinger, L., Buske, C., Butzeck, B., Cammenga, J., Campo, E., Carbone, A., Cervantes, F., Cesaro, S., Charbord, P., Claas, F., Cohen, H., Conard, J., Coppo, P., Corrons, JL., Costa, Ld., Davi, F., Delwel, R., Dianzani, I., Domanović, D., Donnelly, P., Drnov?ek£££Tadeja Dovč£££ TD., Dreyling, M., Du, MQ., Dufour, C., Durand, C., Efremov, D., Eleftheriou, A., Elion, J., Emonts, M., Engelhardt, M., Ezine, S., Falkenburg, F., Favier, R., Federico, M., Fenaux, P., Fitzgibbon, J., Flygare, J., Foà, R., Forrester, L., Galacteros, F., Garagiola, I., Gardiner, C., Garraud, O., van Geet, C., Geiger, H., Geissler, J., Germing, U., Ghevaert, C., Girelli, D., Godeau, B., Gökbuget, N., Goldschmidt, H., Goodeve, A., Graf, T., Graziadei, G., Griesshammer, M., Gruel, Y., Guilhot, F., von Gunten, S., Gyssens, I., Halter, J., Harrison, C., Harteveld, C., Hellström-Lindberg, E., Hermine, O., Higgs, D., Hillmen, P., Hirsch, H., Hoskin, P., Huls, G., Inati, A., Johnson, P., Kattamis, A., Kiefel, V., Kleanthous, M., Klump, H., Krause, D., Hovinga, JK., Lacaud, G., Lacroix-Desmazes, S., Landman-Parker, J., LeGouill, S., Lenz, G., von Lilienfeld-Toal, M., von Lindern, M., Lopez-Guillermo, A., Lopriore, E., Lozano, M., MacIntyre, E., Makris, M., Mannhalter, C., Martens, J., Mathas, S., Matzdorff, A., Medvinsky, A., Menendez, P., Migliaccio, AR., Miharada, K., Mikulska, M., Minard, V., Montalbán, C., de Montalembert, M., Montserrat, E., Morange, PE., Mountford, J., Muckenthaler, M., Müller-Tidow, C., Mumford, A., Nadel, B., Navarro, JT., Nemer, We., Noizat-Pirenne, F., O'Mahony, B., Oldenburg, J., Olsson, M., Oostendorp, R., Palumbo, A., Passamonti, F., Patient, R., Peffault, R., Pflumio, F., Pierelli, L., Piga, A., Pollard, D., Raaijmakers, M., Radford, J., Rambach, R., Rao, AK., Raslova, H., Rebulla, P., Rees, D., Ribrag, V., Rijneveld, A., Rinalducci, S., Robak, T., Roberts, I., Rodrigues, C., Rosendaal, F., Rosenwald, A., Rule, S., Russo, R., Saglio, G., Sanchez, M., Scharf, RE., Schlenke, P., Semple, J., Sierra, J., So-Osman, C., Soria, JM., Stamatopoulos, K., Stegmayr, B., Stunnenberg, H., Swinkels, D., Barata£££João Pedro Taborda£££ JP., Taghon, T., Taher, A., Terpos, E., Thachil, J., Tissot, JD., Touw, I., Toye, A., Trappe, R., Traverse-Glehen, A., Unal, S., Vaulont, S., Viprakasit, V., Vitolo, U., van Wijk, R., Wójtowicz, A., Zeerleder, S., Zieger, B., de Wit, T.D., Schuringa, J.J., EHA Roadmap for European Hematology, Research, Engert, A., Balduini, C., Brand, A., Coiffier, B., Cordonnier, C., Döhner, H., de Wit TD., Eichinger, S., Fibbe, W., Green, T., de Haas, F., Iolascon, A., Jaffredo, T., Rodeghiero, F., Salles, G., Schuringa, JJ., André, M., Andre-Schmutz, I., Bacigalupo, A., Bochud, PY., Boer, Md., Bonini, C., Camaschella, C., Cant, A., Cappellini, MD., Cazzola, M., Celso, CL., Dimopoulos, M., Douay, L., Dzierzak, E., Einsele, H., Ferreri, A., De Franceschi, L., Gaulard, P., Gottgens, B., Greinacher, A., Gresele, P., Gribben, J., de Haan, G., Hansen, JB., Hochhaus, A., Kadir, R., Kaveri, S., Kouskoff, V., Kühne, T., Kyrle, P., Ljungman, P., Maschmeyer, G., Méndez-Ferrer£££Simón£££ S., Milsom, M., Mummery, C., Ossenkoppele, G., Pecci, A., Peyvandi, F., Philipsen, S., Reitsma, P., Ribera, JM., Risitano, A., Rivella, S., Ruf, W., Schroeder, T., Scully, M., Socie, G., Staal, F., Stanworth, S., Stauder, R., Stilgenbauer, S., Tamary, H., Theilgaard-Mönch, K., Thein, SL., Tilly, H., Trneny, M., Vainchenker, W., Vannucchi, AM., Viscoli, C., Vrielink, H., Zaaijer, H., Zanella, A., Zolla, L., Zwaginga, JJ., Martinez, PA., van den Akker, E., Allard, S., Anagnou, N., Andolfo, I., Andrau, JC., Angelucci, E., Anstee, D., Aurer, I., Avet-Loiseau, H., Aydinok, Y., Bakchoul, T., Balduini, A., Barcellini, W., Baruch, D., Baruchel, A., Bayry, J., Bento, C., van den Berg, A., Bernardi, R., Bianchi, P., Bigas, A., Biondi, A., Bohonek, M., Bonnet, D., Borchmann, P., Borregaard, N., Brækkan, S., van den Brink, M., Brodin, E., Bullinger, L., Buske, C., Butzeck, B., Cammenga, J., Campo, E., Carbone, A., Cervantes, F., Cesaro, S., Charbord, P., Claas, F., Cohen, H., Conard, J., Coppo, P., Corrons, JL., Costa, Ld., Davi, F., Delwel, R., Dianzani, I., Domanović, D., Donnelly, P., Drnov?ek£££Tadeja Dovč£££ TD., Dreyling, M., Du, MQ., Dufour, C., Durand, C., Efremov, D., Eleftheriou, A., Elion, J., Emonts, M., Engelhardt, M., Ezine, S., Falkenburg, F., Favier, R., Federico, M., Fenaux, P., Fitzgibbon, J., Flygare, J., Foà, R., Forrester, L., Galacteros, F., Garagiola, I., Gardiner, C., Garraud, O., van Geet, C., Geiger, H., Geissler, J., Germing, U., Ghevaert, C., Girelli, D., Godeau, B., Gökbuget, N., Goldschmidt, H., Goodeve, A., Graf, T., Graziadei, G., Griesshammer, M., Gruel, Y., Guilhot, F., von Gunten, S., Gyssens, I., Halter, J., Harrison, C., Harteveld, C., Hellström-Lindberg, E., Hermine, O., Higgs, D., Hillmen, P., Hirsch, H., Hoskin, P., Huls, G., Inati, A., Johnson, P., Kattamis, A., Kiefel, V., Kleanthous, M., Klump, H., Krause, D., Hovinga, JK., Lacaud, G., Lacroix-Desmazes, S., Landman-Parker, J., LeGouill, S., Lenz, G., von Lilienfeld-Toal, M., von Lindern, M., Lopez-Guillermo, A., Lopriore, E., Lozano, M., MacIntyre, E., Makris, M., Mannhalter, C., Martens, J., Mathas, S., Matzdorff, A., Medvinsky, A., Menendez, P., Migliaccio, AR., Miharada, K., Mikulska, M., Minard, V., Montalbán, C., de Montalembert, M., Montserrat, E., Morange, PE., Mountford, J., Muckenthaler, M., Müller-Tidow, C., Mumford, A., Nadel, B., Navarro, JT., Nemer, We., Noizat-Pirenne, F., O'Mahony, B., Oldenburg, J., Olsson, M., Oostendorp, R., Palumbo, A., Passamonti, F., Patient, R., Peffault, R., Pflumio, F., Pierelli, L., Piga, A., Pollard, D., Raaijmakers, M., Radford, J., Rambach, R., Rao, AK., Raslova, H., Rebulla, P., Rees, D., Ribrag, V., Rijneveld, A., Rinalducci, S., Robak, T., Roberts, I., Rodrigues, C., Rosendaal, F., Rosenwald, A., Rule, S., Russo, R., Saglio, G., Sanchez, M., Scharf, RE., Schlenke, P., Semple, J., Sierra, J., So-Osman, C., Soria, JM., Stamatopoulos, K., Stegmayr, B., Stunnenberg, H., Swinkels, D., Barata£££João Pedro Taborda£££ JP., Taghon, T., Taher, A., Terpos, E., Thachil, J., Tissot, JD., Touw, I., Toye, A., Trappe, R., Traverse-Glehen, A., Unal, S., Vaulont, S., Viprakasit, V., Vitolo, U., van Wijk, R., Wójtowicz, A., Zeerleder, S., Zieger, B., de Wit, T.D., and Schuringa, J.J.

Abstract

The European Hematology Association (EHA) Roadmap for European Hematology Research highlights major achievements in diagnosis and treatment of blood disorders and identifies the greatest unmet clinical and scientific needs in those areas to enable better funded, more focused European hematology research. Initiated by the EHA, around 300 experts contributed to the consensus document, which will help European policy makers, research funders, research organizations, researchers, and patient groups make better informed decisions on hematology research. It also aims to raise public awareness of the burden of blood disorders on European society, which purely in economic terms is estimated at euro23 billion per year, a level of cost that is not matched in current European hematology research funding. In recent decades, hematology research has improved our fundamental understanding of the biology of blood disorders, and has improved diagnostics and treatments, sometimes in revolutionary ways. This progress highlights the potential of focused basic research programs such as this EHA Roadmap.The EHA Roadmap identifies nine 'sections' in hematology: normal hematopoiesis, malignant lymphoid and myeloid diseases, anemias and related diseases, platelet disorders, blood coagulation and hemostatic disorders, transfusion medicine, infections in hematology, and hematopoietic stem cell transplantation. These sections span 60 smaller groups of diseases or disorders.The EHA Roadmap identifies priorities and needs across the field of hematology, including those to develop targeted therapies based on genomic profiling and chemical biology, to eradicate minimal residual malignant disease, and to develop cellular immunotherapies, combination treatments, gene therapies, hematopoietic stem cell treatments, and treatments that are better tolerated by elderly patients.

Published
2016

86. The european hematology association roadmap for european hematology research: A consensus document

Author

Engert, A, Balduini, C, Brand, A, Coiffier, B, Cordonnier, C, Döhner, H, De Wit, T, Eichinger, S, Fibbe, W, Green, T, De Haas, F, Iolascon, A, Jaffredo, T, Rodeghiero, F, Sall Es, G, Schuringa, J, André, M, Andre Schmutz, I, Bacigalupo, A, Bochud, P, Den Boer, M, Bonini, C, Camaschella, C, Cant, A, Cappellini, M, Cazzola, M, Celso, C, Dimopoulos, M, Douay, L, Dzierzak, E, Einsele, H, Ferreri, A, De Franceschi, L, Gaulard, P, Gottgens, B, Greinacher, A, Gresele, P, Gribben, J, De Haan, G, Hansen, J, Hochhaus, A, Kadir, R, Kaveri, S, Kouskoff, V, Kühne, T, Kyrle, P, Ljungman, P, Maschmeyer, G, Méndez Ferrer, S, Milsom, M, Mummery, C, Ossenkoppele, G, Pecci, A, Peyvandi, F, Philipsen, S, Reitsma, P, Ribera, J, Risitano, A, Rivella, S, Ruf, W, Schroeder, T, Scully, M, Socie, G, Staal, F, Stanworth, S, Stauder, R, Stilgenbauer, S, Tamary, H, Theilgaard Mönch, K, Thein, S, Tilly, H, Trneny, M, Vainchenker, W, Vannucchi, A, Viscoli, C, Vrielink, H, Zaaijer, H, Zanella, A, Zolla, L, Zwaginga, J, Martinez, P, Van Den Akker, E, Allard, S, Anagnou, N, Andolfo, I, Andrau, J, Angelucci, E, Anstee, D, Aurer, I, Avet Loiseau, H, Aydinok, Y, Bakchoul, T, Balduini, A, Barcellini, W, Baruch, D, Baruchel, A, Bayry, J, Bento, C, Van Den Berg, A, Bernardi, R, Bianchi, P, Bigas, A, Biondi, A, Bohonek, M, Bonnet, D, Borchmann, P, Borregaard, N, Brækkan, S, Van Den Brink, M, Brodin, E, Bullinger, L, Buske, C, Butzeck, B, Cammenga, J, Campo, E, Carbone, A, Cervantes, F, Cesaro, S, Charbord, P, Claas, F, Cohen, H, Conard, J, Coppo, P, Vives Corron, J, Da Costa, L, Davi, F, Delwel, R, Dianzani, I, Domanović, D, Donnelly, P, Drnovšek, T, Dreyling, M, Du, M, Dufour, C, Durand, C, Efremov, D, Eleftheriou, A, Elion, J, Emonts, M, Engelhardt, M, Ezine, S, Falkenburg, F, Favier, R, Federico, M, Fenaux, P, Fitzgibbon, J, Flygare, J, Foà, R, Forrester, L, Galacteros, F, Garagiola, I, Gardiner, C, Garraud, O, Van Geet, C, Geiger, H, Geissler, J, Germing, U, Ghevaert, C, Girelli, D, Godeau, B, Gökbuget, N, Goldschmidt, H, Goodeve, A, Graf, T, Graziadei, G, Griesshammer, M, Gruel, Y, Guilhot, F, Von Gunten, S, Gyssens, I, Halter, J, Harrison, C, Harteveld, C, Hellström Lindberg, E, Hermine, O, Higgs, D, Hillmen, P, Hirsch, H, Hoskin, P, Huls, G, Inati, A, Johnson, P, Kattamis, A, Kiefel, V, Kleanthous, M, Klump, H, Krause, D, Hovinga, J, Lacaud, G, Lacroix Desmazes, S, Landman Parker, J, Legouill, S, Lenz, G, Von Lilienfeld Toal, M, Von Lindern, M, Lopez Guillermo, A, Lopriore, E, Lozano, M, Macintyre, E, Makris, M, Mannhalter, C, Martens, J, Mathas, S, Matzdorff, A, Medvinsky, A, Menendez, P, Migliaccio, A, Miharada, K, Mikulska, M, Minard, V, Montalbán, C, De Montalembert, M, Montserrat, E, Morange, P, Mountford, J, Muckenthaler, M, Müller Tidow, C, Mumford, A, Nadel, B, Navarro, J, El Nemer, W, Noizat Pirenne, F, O’Mahony, B, Oldenburg, J, Olsson, M, Oostendorp, R, Palumbo, A, Passamonti, F, Patient, R, De Latour, R, Pflumio, F, Pierelli, L, Piga, A, Pollard, D, Raaijmakers, M, Radford, J, Rambach, R, Koneti Rao, A, Raslova, H, Rebulla, P, Rees, D, Ribrag, V, Rijneveld, A, Rinalducci, S, Robak, T, Roberts, I, Rodrigues, C, Rosendaal, F, Rosenwald, A, Rule, S, Russo, R, Saglio, G, Sanchez, M, Scharf, R, Schlenke, P, Semple, J, Sierra, J, So Osman, C, Soria, J, Stamatopoulos, K, Stegmayr, B, Stunnenberg, H, Swinkels, D, Barata, J, Taghon, T, Taher, A, Terpos, E, Thachil, J, Tissot, J, Touw, I, Toye, A, Trappe, R, Traverse Glehen, A, Unal, S, Vaulont, S, Viprakasit, V, Vitolo, U, Van Wijk, R, Wójtowicz, A, Zeerleder, S, Zieger, B, Zieger, B., ZANELLA, ALBERTO, BIONDI, ANDREA, Engert, A, Balduini, C, Brand, A, Coiffier, B, Cordonnier, C, Döhner, H, De Wit, T, Eichinger, S, Fibbe, W, Green, T, De Haas, F, Iolascon, A, Jaffredo, T, Rodeghiero, F, Sall Es, G, Schuringa, J, André, M, Andre Schmutz, I, Bacigalupo, A, Bochud, P, Den Boer, M, Bonini, C, Camaschella, C, Cant, A, Cappellini, M, Cazzola, M, Celso, C, Dimopoulos, M, Douay, L, Dzierzak, E, Einsele, H, Ferreri, A, De Franceschi, L, Gaulard, P, Gottgens, B, Greinacher, A, Gresele, P, Gribben, J, De Haan, G, Hansen, J, Hochhaus, A, Kadir, R, Kaveri, S, Kouskoff, V, Kühne, T, Kyrle, P, Ljungman, P, Maschmeyer, G, Méndez Ferrer, S, Milsom, M, Mummery, C, Ossenkoppele, G, Pecci, A, Peyvandi, F, Philipsen, S, Reitsma, P, Ribera, J, Risitano, A, Rivella, S, Ruf, W, Schroeder, T, Scully, M, Socie, G, Staal, F, Stanworth, S, Stauder, R, Stilgenbauer, S, Tamary, H, Theilgaard Mönch, K, Thein, S, Tilly, H, Trneny, M, Vainchenker, W, Vannucchi, A, Viscoli, C, Vrielink, H, Zaaijer, H, Zanella, A, Zolla, L, Zwaginga, J, Martinez, P, Van Den Akker, E, Allard, S, Anagnou, N, Andolfo, I, Andrau, J, Angelucci, E, Anstee, D, Aurer, I, Avet Loiseau, H, Aydinok, Y, Bakchoul, T, Balduini, A, Barcellini, W, Baruch, D, Baruchel, A, Bayry, J, Bento, C, Van Den Berg, A, Bernardi, R, Bianchi, P, Bigas, A, Biondi, A, Bohonek, M, Bonnet, D, Borchmann, P, Borregaard, N, Brækkan, S, Van Den Brink, M, Brodin, E, Bullinger, L, Buske, C, Butzeck, B, Cammenga, J, Campo, E, Carbone, A, Cervantes, F, Cesaro, S, Charbord, P, Claas, F, Cohen, H, Conard, J, Coppo, P, Vives Corron, J, Da Costa, L, Davi, F, Delwel, R, Dianzani, I, Domanović, D, Donnelly, P, Drnovšek, T, Dreyling, M, Du, M, Dufour, C, Durand, C, Efremov, D, Eleftheriou, A, Elion, J, Emonts, M, Engelhardt, M, Ezine, S, Falkenburg, F, Favier, R, Federico, M, Fenaux, P, Fitzgibbon, J, Flygare, J, Foà, R, Forrester, L, Galacteros, F, Garagiola, I, Gardiner, C, Garraud, O, Van Geet, C, Geiger, H, Geissler, J, Germing, U, Ghevaert, C, Girelli, D, Godeau, B, Gökbuget, N, Goldschmidt, H, Goodeve, A, Graf, T, Graziadei, G, Griesshammer, M, Gruel, Y, Guilhot, F, Von Gunten, S, Gyssens, I, Halter, J, Harrison, C, Harteveld, C, Hellström Lindberg, E, Hermine, O, Higgs, D, Hillmen, P, Hirsch, H, Hoskin, P, Huls, G, Inati, A, Johnson, P, Kattamis, A, Kiefel, V, Kleanthous, M, Klump, H, Krause, D, Hovinga, J, Lacaud, G, Lacroix Desmazes, S, Landman Parker, J, Legouill, S, Lenz, G, Von Lilienfeld Toal, M, Von Lindern, M, Lopez Guillermo, A, Lopriore, E, Lozano, M, Macintyre, E, Makris, M, Mannhalter, C, Martens, J, Mathas, S, Matzdorff, A, Medvinsky, A, Menendez, P, Migliaccio, A, Miharada, K, Mikulska, M, Minard, V, Montalbán, C, De Montalembert, M, Montserrat, E, Morange, P, Mountford, J, Muckenthaler, M, Müller Tidow, C, Mumford, A, Nadel, B, Navarro, J, El Nemer, W, Noizat Pirenne, F, O’Mahony, B, Oldenburg, J, Olsson, M, Oostendorp, R, Palumbo, A, Passamonti, F, Patient, R, De Latour, R, Pflumio, F, Pierelli, L, Piga, A, Pollard, D, Raaijmakers, M, Radford, J, Rambach, R, Koneti Rao, A, Raslova, H, Rebulla, P, Rees, D, Ribrag, V, Rijneveld, A, Rinalducci, S, Robak, T, Roberts, I, Rodrigues, C, Rosendaal, F, Rosenwald, A, Rule, S, Russo, R, Saglio, G, Sanchez, M, Scharf, R, Schlenke, P, Semple, J, Sierra, J, So Osman, C, Soria, J, Stamatopoulos, K, Stegmayr, B, Stunnenberg, H, Swinkels, D, Barata, J, Taghon, T, Taher, A, Terpos, E, Thachil, J, Tissot, J, Touw, I, Toye, A, Trappe, R, Traverse Glehen, A, Unal, S, Vaulont, S, Viprakasit, V, Vitolo, U, Van Wijk, R, Wójtowicz, A, Zeerleder, S, Zieger, B, Zieger, B., ZANELLA, ALBERTO, and BIONDI, ANDREA

Abstract

The European Hematology Association (EHA) Roadmap for European Hematology Research highlights major achievements in diagnosis and treatment of blood disorders and identifies the greatest unmet clinical and scientific needs in those areas to enable better funded, more focused European hematology research. Initiated by the EHA, around 300 experts contributed to the consensus document, which will help European policy makers, research funders, research organizations, researchers, and patient groups make better informed decisions on hematology research. It also aims to raise public awareness of the burden of blood disorders on European society, which purely in economic terms is estimated at ∈ European Hematology Association (EHA) Roadmap for European Hematology Research highlights major achievements in diagnosis and treatment of blood disorders and identifies the greatest unmet clinical and scientific needs in those areas to enable better fu treatments, sometimes in revolutionary ways. This progress highlights the potential of focused basic research programs such as this EHA Roadmap. The EHA Roadmap identifies nine ‘sections’ in hematology: normal hematopoiesis, malignant lymphoid and myeloid diseases, anemias and related diseases, platelet disorders, blood coagulation and hemostatic disorders, transfusion medicine, infections in hematology, and hematopoietic stem cell transplantation. These sections span 60 smaller groups of diseases or disorders. The EHA Roadmap identifies priorities and needs across the field of hematology, including those to develop targeted therapies based on genomic profiling and chemical biology, to eradicate minimal residual malignant disease, and to develop cellular immunotherapies, combination treatments, gene therapies, hematopoietic stem cell treatments, and treatments that are better tolerated by elderly patients.

Published
2016

87. Comparative value of post-remission treatment in cytogenetically normal AML subclassified by NPM1 and FLT3-ITD allelic ratio

Author

Versluis, J, primary, in ‘t Hout, F E M, additional, Devillier, R, additional, van Putten, W L J, additional, Manz, M G, additional, Vekemans, M -C, additional, Legdeur, M -C, additional, Passweg, J R, additional, Maertens, J, additional, Kuball, J, additional, Biemond, B J, additional, Valk, P J M, additional, van der Reijden, B A, additional, Meloni, G, additional, Schouten, H C, additional, Vellenga, E, additional, Pabst, T, additional, Willemze, R, additional, Löwenberg, B, additional, Ossenkoppele, G, additional, Baron, F, additional, Huls, G, additional, and Cornelissen, J J, additional

Published
2016
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88. Severe Ankyrin-R deficiency results in impaired surface retention and lysosomal degradation of RhAG in human erythroblasts

Author

Satchwell, T. J., primary, Bell, A. J., additional, Hawley, B. R., additional, Pellegrin, S., additional, Mordue, K. E., additional, van Deursen, C. T. B. M., additional, Braak, N. H.-t., additional, Huls, G., additional, Leers, M. P. G., additional, Overwater, E., additional, Tamminga, R. Y. J., additional, van der Zwaag, B., additional, Fermo, E., additional, Bianchi, P., additional, van Wijk, R., additional, and Toye, A. M., additional

Published
2016
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89. Het myelodysplastisch syndroom: adviezen voor ijzerchelatie bij secundaire hemochromatose

Author

Cremers, E.M.P., de Swart, L., Huls, G., Wijermans, P., Lowenberg, B., Jongen-Lavrencic, M., de Greef, G.E., Muus, P., van Marwijk Kooy, R., Schaafsma, R., van Maanen, T., Deenik, W., Beeker, A., Brouwer, R.E., Hoogendoorn, M., Breems, D.A., Raaijmakers, H.G.P., Verhoef, GE, Schouten, H.C., von dem Borne, P., Kuball, J., Biemond, B.J., Vellenga, E., Ossenkoppele, G.J., de Witte, T.J.M., van de Loosdrecht, A.A., Hematology laboratory, Hematology, and CCA - Innovative therapy

Published
2014

91. Comparative therapeutic value of post-remission approaches in patients with acute myeloid leukemia aged 40-60 years

Author

Cornelissen, J. J., Versluis, J., Passweg, J. R., Van Putten, W. L J, Manz, M. G., Maertens, J., Beverloo, H. B., Valk, P. J M, Van Marwijk Kooy, M., Wijermans, P. W., Schaafsma, M. R., Biemond, B. J., Vekemans, M. C., Breems, D. A., Verdonck, L. F., Fey, M. F., Jongen-Lavrencic, M., Janssen, J. J W M, Huls, G., Kuball, J., Pabst, T., Graux, C., Schouten, H. C., Gratwohl, A., Vellenga, E., Ossenkoppele, G., Löwenberg, B., Cornelissen, J. J., Versluis, J., Passweg, J. R., Van Putten, W. L J, Manz, M. G., Maertens, J., Beverloo, H. B., Valk, P. J M, Van Marwijk Kooy, M., Wijermans, P. W., Schaafsma, M. R., Biemond, B. J., Vekemans, M. C., Breems, D. A., Verdonck, L. F., Fey, M. F., Jongen-Lavrencic, M., Janssen, J. J W M, Huls, G., Kuball, J., Pabst, T., Graux, C., Schouten, H. C., Gratwohl, A., Vellenga, E., Ossenkoppele, G., and Löwenberg, B.

Published
2015

92. Biomarker profiling of steroid-resistant acute GVHD in patients after infusion of mesenchymal stromal cells

Author

Te Boome, L. C J, Mansilla, C., Van Der Wagen, L. E., Lindemans, C. A., Petersen, E. J., Spierings, E., Thus, K. A., Westinga, K., Plantinga, M., Bierings, M., Broers, A. E C, Cuijpers, M. L H, Van Imhoff, G. W., Janssen, J. J., Huisman, C., Zeerleder, S., Huls, G., Boelens, J. J., Wulffraat, N. M., Slaper-Cortenbach, I. C M, Kuball, J., Te Boome, L. C J, Mansilla, C., Van Der Wagen, L. E., Lindemans, C. A., Petersen, E. J., Spierings, E., Thus, K. A., Westinga, K., Plantinga, M., Bierings, M., Broers, A. E C, Cuijpers, M. L H, Van Imhoff, G. W., Janssen, J. J., Huisman, C., Zeerleder, S., Huls, G., Boelens, J. J., Wulffraat, N. M., Slaper-Cortenbach, I. C M, and Kuball, J.

Published
2015

93. Comparative therapeutic value of post-remission approaches in patients with acute myeloid leukemia aged 40-60 years

Author

UCL - SSS/IREC/MONT - Pôle Mont Godinne, UCL - (MGD) Service d'hématologie, UCL - (SLuc) Service d'hématologie, Cornelissen, J J, Versluis, J, Passweg, J R, van Putten, W L J, Manz, M G, Maertens, J, Beverloo, H B, Valk, P J M, van Marwijk Kooy, M, Wijermans, P W, Schaafsma, M R, Biemond, B J, Vekemans, Marie-Christiane, Breems, D A, Verdonck, L F, Fey, M F, Jongen-Lavrencic, M, Janssen, J J W M, Huls, G, Kuball, J, Pabst, T, Graux, Carlos, Schouten, H C, Gratwohl, A, Vellenga, E, Ossenkoppele, G, Löwenberg, B, UCL - SSS/IREC/MONT - Pôle Mont Godinne, UCL - (MGD) Service d'hématologie, UCL - (SLuc) Service d'hématologie, Cornelissen, J J, Versluis, J, Passweg, J R, van Putten, W L J, Manz, M G, Maertens, J, Beverloo, H B, Valk, P J M, van Marwijk Kooy, M, Wijermans, P W, Schaafsma, M R, Biemond, B J, Vekemans, Marie-Christiane, Breems, D A, Verdonck, L F, Fey, M F, Jongen-Lavrencic, M, Janssen, J J W M, Huls, G, Kuball, J, Pabst, T, Graux, Carlos, Schouten, H C, Gratwohl, A, Vellenga, E, Ossenkoppele, G, and Löwenberg, B

Abstract

The preferred type of post-remission therapy (PRT) in patients with acute myeloid leukemia (AML) in first complete remission (CR1) is a subject of continued debate, especially in patients at higher risk of nonrelapse mortality (NRM), including patients >40 years of age. We report results of a time-dependent multivariable analysis of allogenic hematopoietic stem cell transplantation (alloHSCT) (n=337) versus chemotherapy (n=271) or autologous HSCT (autoHSCT) (n=152) in 760 patients aged 40-60 years with AML in CR1. Patients receiving alloHSCT showed improved overall survival (OS) as compared with chemotherapy (respectively, 57±3% vs 40±3% at 5 years, P<0.001). Comparable OS was observed following alloHSCT and autoHSCT in patients with intermediate-risk AML (60±4 vs 54±5%). However, alloHSCT was associated with less relapse (hazard ratio (HR) 0.51, P<0.001) and better relapse-free survival (RFS) (HR 0.74, P=0.029) as compared with autoHSCT in intermediate-risk AMLs. AlloHSCT was applied following myeloablative conditioning (n=157) or reduced intensity conditioning (n=180), resulting in less NRM, but comparable outcome with respect to OS, RFS and relapse. Collectively, these results show that alloHSCT is to be preferred over chemotherapy as PRT in patients with intermediate- and poor-risk AML aged 40-60 years, whereas autoHSCT remains a treatment option to be considered in patients with intermediate-risk AML.Leukemia advance online publication, 23 December 2014; doi:10.1038/leu.2014.332.

Published
2015

94. Allelic mutations of KITLG, encoding KIT ligand, cause asymmetric and unilateral hearing loss and Waardenburg syndrome type 2

Author

Zazo Seco, C. (Celia), Serrão De Castro, L. (Luciana), Nierop, J.W.I. van, Morín, M. (Matías), Jhangiani, S.N. (Shalini N.), Verver, E.J.J. (Eva J. J.), Schraders, M. (Margit), Maiwald, N. (Nadine), Wesdorp, M. (Mieke), Venselaar, H. (Hanka), Spruijt, L. (Liesbeth), Oostrik, J. (Jaap), Schoots, J. (Jeroen), Reeuwijk, J. (Jeroen) van, Lelieveld, S.H. (Stefan H.), Huygen, P.L.M. (Patrick), Insenser, M. (María), Admiraal, R.J. (Ronald), Pennings, R.J.E. (Ronald J.E.), Hoefsloot, E.H. (Lies), Arias-Vásquez, A. (Alejandro), Ligt, J. (Joep) de, Yntema, H.G., Jansen, J.H. (Joop H.), Muzny, D. (Donna), Huls, G. (Gerwin), Rossum, M.M. (Michelle) van, Lupski, J.R. (James R.), Moreno-Pelayo, M.A. (Miguel Angel), Kunst, H.P.M. (Henricus P.M.), Kremer, H. (Hannie), Zazo Seco, C. (Celia), Serrão De Castro, L. (Luciana), Nierop, J.W.I. van, Morín, M. (Matías), Jhangiani, S.N. (Shalini N.), Verver, E.J.J. (Eva J. J.), Schraders, M. (Margit), Maiwald, N. (Nadine), Wesdorp, M. (Mieke), Venselaar, H. (Hanka), Spruijt, L. (Liesbeth), Oostrik, J. (Jaap), Schoots, J. (Jeroen), Reeuwijk, J. (Jeroen) van, Lelieveld, S.H. (Stefan H.), Huygen, P.L.M. (Patrick), Insenser, M. (María), Admiraal, R.J. (Ronald), Pennings, R.J.E. (Ronald J.E.), Hoefsloot, E.H. (Lies), Arias-Vásquez, A. (Alejandro), Ligt, J. (Joep) de, Yntema, H.G., Jansen, J.H. (Joop H.), Muzny, D. (Donna), Huls, G. (Gerwin), Rossum, M.M. (Michelle) van, Lupski, J.R. (James R.), Moreno-Pelayo, M.A. (Miguel Angel), Kunst, H.P.M. (Henricus P.M.), and Kremer, H. (Hannie)

Abstract

Linkage analysis combined with whole-exome sequencing in a large family with congenital and stable non-syndromic unilateral and asymmetric hearing loss (NS-UHL/AHL) revealed a heterozygous truncating mutation, c.286-303delinsT (p.Ser96Ter), in KITLG. This mutation co-segregated with NS-UHL/AHL as a dominant trait with reduced penetrance. By screening a panel of probands with NS-UHL/AHL, we found an additional mutation, c.200-202del (p.His67-Cys68delinsArg). In vitro studies revealed that the p.His67-Cys68delinsArg transmembrane isoform of KITLG is not detectable at the cell membrane, supporting pathogenicity. KITLG encodes a ligand for the KIT receptor. Also, KITLG-KIT signaling and MITF are suggested to mutually interact in melanocyte development. Because mutations in MITF are causative of Waardenburg syndrome type 2 (WS2), we screened KITLG in suspected WS2-affected probands. A heterozygous missense mutation, c.310C>G (p.Leu104Val), that segregated with WS2 was identified in a small family. In vitro studies revealed that the p.Leu104Val transmembrane isoform of KITLG is located at the cell membrane, as is wild-type KITLG. However, in culture media of transfected cells, the p.Leu104Val soluble isoform of KITLG was reduced, and no soluble p.His67-Cys68delinsArg and p.Ser96Ter KITLG could be detected. These data suggest that mutations in KITLG associated with NS-UHL/AHL have a loss-of-function effect. We speculate that the mechanism of the mutation underlying WS2 and leading to membrane incorporation and reduced secretion of KITLG occurs via a dominant-negative or gain-of-function effect. Our study unveils different phenotypes associated with KITLG, previously associated with pigmentation abnormalities, and will thereby improve the genetic counseling given to individuals with KITLG variants.

Published
2015
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95. Ruxolitinib for corticosteroid-refractory graft-versus-host disease: analysis of 95 patients treated at multiple medical centers

Author

Zeiser, R., Burchert, A., Lengerke, C., Verbeek, M., Maas-Bauer, K., Metzelder, S., Spoerl, S., Ditschkowski, M., Ecsedi, M., Sockel, K., Ayuk, F., Ajib, S., de Fontbrune, Sicre F., Na, I. -K, Penter, L., Holtick, U., Wolf, D., Schuler, E., Meyer, E., Apostolova, P., Bertz, H., Marks, R., Luebbert, M., Waesch, R., Scheid, C., Ordemann, R., Bug, G., Kobbe, G., Negrin, R., Brune, M., Spyridonidis, A., Schmitt-Graeff, A., van der Velden, W., Huls, G., Grigoleit, G. U., Kuball, J., Blazar, B. R., Arnold, R., Kroeger, N., Passweg, J., Halter, J., Socie, G., Beelen, D., Peschel, C., Neubauer, A., Finke, J., Duyster, J., von Bubnoff, N., Zeiser, R., Burchert, A., Lengerke, C., Verbeek, M., Maas-Bauer, K., Metzelder, S., Spoerl, S., Ditschkowski, M., Ecsedi, M., Sockel, K., Ayuk, F., Ajib, S., de Fontbrune, Sicre F., Na, I. -K, Penter, L., Holtick, U., Wolf, D., Schuler, E., Meyer, E., Apostolova, P., Bertz, H., Marks, R., Luebbert, M., Waesch, R., Scheid, C., Ordemann, R., Bug, G., Kobbe, G., Negrin, R., Brune, M., Spyridonidis, A., Schmitt-Graeff, A., van der Velden, W., Huls, G., Grigoleit, G. U., Kuball, J., Blazar, B. R., Arnold, R., Kroeger, N., Passweg, J., Halter, J., Socie, G., Beelen, D., Peschel, C., Neubauer, A., Finke, J., Duyster, J., and von Bubnoff, N.

Published
2015

96. Ruxolitinib in corticosteroid-refractory graft-versus-host disease after allogeneic stem cell transplantation: a multicenter survey

Author

Zeiser, R., Burchert, A., Lengerke, C., Verbeek, M., Maas-Bauer, K., Metzelder, S. K., Spoerl, S., Ditschkowski, M., Ecsedi, M., Sockel, K., Ayuk, F., Ajib, S., de Fontbrune, F. S., Na, I-K, Penter, L., Holtick, U., Wolf, D., Schuler, E., Meyer, E., Apostolova, P., Bertz, H., Marks, R., Luebbert, M., Waesch, R., Scheid, C., Stoelzel, F., Ordemann, R., Bug, G., Kobbe, G., Negrin, R., Brune, M., Spyridonidis, A., Schmitt-Graeff, A., van der Velden, W., Huls, G., Mielke, S., Grigoleit, G. U., Kuball, J., Flynn, R., Ihorst, G., Du, J., Blazar, B. R., Arnold, R., Kroeger, N., Passweg, J., Halter, J., Socie, G., Beelen, D., Peschel, C., Neubauer, A., Finke, J., Duyster, J., von Bubnoff, N., Zeiser, R., Burchert, A., Lengerke, C., Verbeek, M., Maas-Bauer, K., Metzelder, S. K., Spoerl, S., Ditschkowski, M., Ecsedi, M., Sockel, K., Ayuk, F., Ajib, S., de Fontbrune, F. S., Na, I-K, Penter, L., Holtick, U., Wolf, D., Schuler, E., Meyer, E., Apostolova, P., Bertz, H., Marks, R., Luebbert, M., Waesch, R., Scheid, C., Stoelzel, F., Ordemann, R., Bug, G., Kobbe, G., Negrin, R., Brune, M., Spyridonidis, A., Schmitt-Graeff, A., van der Velden, W., Huls, G., Mielke, S., Grigoleit, G. U., Kuball, J., Flynn, R., Ihorst, G., Du, J., Blazar, B. R., Arnold, R., Kroeger, N., Passweg, J., Halter, J., Socie, G., Beelen, D., Peschel, C., Neubauer, A., Finke, J., Duyster, J., and von Bubnoff, N.

Abstract

Despite major improvements in allogeneic hematopoietic cell transplantation over the past decades, corticosteroid-refractory (SR) acute (a) and chronic (c) graft-versus-host disease (GVHD) cause high mortality. Preclinical evidence indicates the potent anti-inflammatory properties of the JAK1/2 inhibitor ruxolitinib. In this retrospective survey, 19 stem cell transplant centers in Europe and the United States reported outcome data from 95 patients who had received ruxolitinib as salvage therapy for SR-GVHD. Patients were classified as having SR-aGVHD (n = 54, all grades III or IV) or SR-cGVHD (n = 41, all moderate or severe). The median number of previous GVHD-therapies was 3 for both SR-aGVHD (1-7) and SR-cGVHD (1-10). The overall response rate was 81.5% (44/54) in SR-aGVHD including 25 complete responses (46.3%), while for SR-cGVHD the ORR was 85.4% (35/41). Of those patients responding to ruxolitinib, the rate of GVHD-relapse was 6.8% (3/44) and 5.7% (2/35) for SR-aGVHD and SR-cGVHD, respectively. The 6-month-survival was 79% (67.3-90.7%, 95% confidence interval (CI)) and 97.4% (92.3-100%, 95% CI) for SR-aGVHD and SR-cGVHD, respectively. Cytopenia and cytomegalovirus-reactivation were observed during ruxolitinib treatment in both SR-aGVHD (30/54, 55.6% and 18/54, 33.3%) and SR-cGVHD (7/41, 17.1% and 6/41, 14.6%) patients. Ruxolitinib may constitute a promising new treatment option for SR-aGVHD and SR-cGVHD that should be validated in a prospective trial.

Published
2015

97. Comparative therapeutic value of post-remission approaches in patients with acute myeloid leukemia aged 40-60 years

Author

HAG Diabetes, JC onderzoeksprogramma Cardiovasculaire Epidemiologie, MS Hematologie, Infection & Immunity, Cancer, Cornelissen, J. J., Versluis, J., Passweg, J. R., Van Putten, W. L J, Manz, M. G., Maertens, J., Beverloo, H. B., Valk, P. J M, Van Marwijk Kooy, M., Wijermans, P. W., Schaafsma, M. R., Biemond, B. J., Vekemans, M. C., Breems, D. A., Verdonck, L. F., Fey, M. F., Jongen-Lavrencic, M., Janssen, J. J W M, Huls, G., Kuball, J., Pabst, T., Graux, C., Schouten, H. C., Gratwohl, A., Vellenga, E., Ossenkoppele, G., Löwenberg, B., HAG Diabetes, JC onderzoeksprogramma Cardiovasculaire Epidemiologie, MS Hematologie, Infection & Immunity, Cancer, Cornelissen, J. J., Versluis, J., Passweg, J. R., Van Putten, W. L J, Manz, M. G., Maertens, J., Beverloo, H. B., Valk, P. J M, Van Marwijk Kooy, M., Wijermans, P. W., Schaafsma, M. R., Biemond, B. J., Vekemans, M. C., Breems, D. A., Verdonck, L. F., Fey, M. F., Jongen-Lavrencic, M., Janssen, J. J W M, Huls, G., Kuball, J., Pabst, T., Graux, C., Schouten, H. C., Gratwohl, A., Vellenga, E., Ossenkoppele, G., and Löwenberg, B.

Published
2015

98. Biomarker profiling of steroid-resistant acute GVHD in patients after infusion of mesenchymal stromal cells

Author

CTI Nierkens, Regenerative Medicine and Stem Cells, Child Health, Infection & Immunity, SCT onderzoek, Externen Hematologie, CTI Kuball, SCT patientenzorg, CDL Celdiagnostiek, Haematologie, HAG Diabetes, Cluster B, Apotheek Celtherapie Faciliteit, MS Hematologie, Cancer, JC onderzoeksprogramma Cardiovasculaire Epidemiologie, Te Boome, L. C J, Mansilla, C., Van Der Wagen, L. E., Lindemans, C. A., Petersen, E. J., Spierings, E., Thus, K. A., Westinga, K., Plantinga, M., Bierings, M., Broers, A. E C, Cuijpers, M. L H, Van Imhoff, G. W., Janssen, J. J., Huisman, C., Zeerleder, S., Huls, G., Boelens, J. J., Wulffraat, N. M., Slaper-Cortenbach, I. C M, Kuball, J., CTI Nierkens, Regenerative Medicine and Stem Cells, Child Health, Infection & Immunity, SCT onderzoek, Externen Hematologie, CTI Kuball, SCT patientenzorg, CDL Celdiagnostiek, Haematologie, HAG Diabetes, Cluster B, Apotheek Celtherapie Faciliteit, MS Hematologie, Cancer, JC onderzoeksprogramma Cardiovasculaire Epidemiologie, Te Boome, L. C J, Mansilla, C., Van Der Wagen, L. E., Lindemans, C. A., Petersen, E. J., Spierings, E., Thus, K. A., Westinga, K., Plantinga, M., Bierings, M., Broers, A. E C, Cuijpers, M. L H, Van Imhoff, G. W., Janssen, J. J., Huisman, C., Zeerleder, S., Huls, G., Boelens, J. J., Wulffraat, N. M., Slaper-Cortenbach, I. C M, and Kuball, J.

Published
2015

99. Treatment, trial participation and survival in adult acute myeloid leukemia: a population-based study in the Netherlands, 1989–2012

Author

Dinmohamed, A G, primary, Visser, O, additional, van Norden, Y, additional, Blijlevens, N M A, additional, Cornelissen, J J, additional, Huls, G A, additional, Huijgens, P C, additional, Sonneveld, P, additional, van de Loosdrecht, A A, additional, Ossenkoppele, G J, additional, Löwenberg, B, additional, and Jongen-Lavrencic, M, additional

Published
2015
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100. 212 TRANSFUSIONS AND PRESENCE OF RINGSIDEROBLASTS INFLUENCE HEPCIDIN AND NTBI LEVELS IN PATIENTS WITH LOWER-RISK MYELODYSPLASTIC SYNDROMES (MDS) - A REPORT FROM THE EUROPEAN LEUKEMIANET MDS REGISTRY

Author

de Swart, L., primary, Reiniers, C., additional, Bagguley, T., additional, Van Marrewijk, C., additional, Bowen, D., additional, Cermak, J., additional, Hellström-Lindberg, E., additional, Tatic, A., additional, Symeonidis, A., additional, Huls, G., additional, Panagiotidis, P., additional, Garelius, H., additional, Culligan, D., additional, Krejci, M., additional, Droste, J., additional, Smith, A., additional, Swinkels, D., additional, and de Witte, T., additional

Published
2015
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