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haematopoietic stem cell transplant

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https://www.readbyqxmd.com/read/28635677/t-cell-manipulation-strategies-to-prevent-graft-versus-host-disease-in-haploidentical-stem-cell-transplantation
#1
REVIEW
Jayakumar Vadakekolathu, Sergio Rutella
Allogeneic haematopoietic stem cell transplantation (HSCT) from an human leukocyte antigen (HLA)-identical donor can be curative for eligible patients with non-malignant and malignant haematological disorders. HSCT from alternative donor sources, such as HLA-mismatched haploidentical donors, is increasingly considered as a viable therapeutic option for patients lacking HLA-matched donors. Initial attempts at haploidentical HSCT were associated with vigorous bidirectional alloreactivity, leading to unacceptably high rates of graft rejection and graft-versus-host disease (GVHD)...
June 21, 2017: Biomedicines
https://www.readbyqxmd.com/read/28634034/absence-of-%C3%AE-chain-in-keratinocytes-alters-chemokine-secretion-resulting-in-reduced-immune-cell-recruitment
#2
Karolin Nowak, Daniela Linzner, Adrian J Thrasher, Paul F Lambert, Wei Li Di, Siobhan O Burns
Loss of function mutations in the common gamma (γc) chain cytokine receptor subunit give rise to severe combined immunodeficiency (SCID) characterised by lack of T and natural killer cells and infant death from infection. Haematopoietic stem cell transplantation or gene therapy offer cure but despite successful replacement of lymphoid immune lineages a long-term risk of severe cutaneous human papilloma virus (HPV) infections persists, possibly related to persistent γc-deficiency in other cell types. Here we demonstrate that keratinocytes, the only cell type directly infected by HPV, express functional γc and its co-receptors...
June 17, 2017: Journal of Investigative Dermatology
https://www.readbyqxmd.com/read/28631609/autologous-haematopoietic-stem-cell-transplantation-for-systemic-lupus-erythematosus-time-ready-for-a-paradigm-shift
#3
EDITORIAL
Tobias Alexander, Falk Hiepe
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May 2017: Clinical and Experimental Rheumatology
https://www.readbyqxmd.com/read/28628276/acute-myeloid-leukaemia-relapsing-after-allogeneic-haematopoietic-stem-cell-transplantation-prognostic-factors-and-impact-of-initial-therapy-of-relapse
#4
Andrew B M Lim, Cameron Curley, Chun Yew Fong, Ian Bilmon, Ashanka Beligaswatte, Duncan Purtill, Bartlomiej Getta, Anne Maree Johnston, Tasman Armytage, Marnie Collins, Kate Mason, Katherine Fielding, Matthew Greenwood, John Gibson, Mark Hertzberg, Matthew Wright, Ian Lewis, John Moore, David Curtis, Jeff Szer, Glen Kennedy, David Ritchie
AIMS: We sought to determine factors associated with overall survival from relapse (OSR) of acute myeloid leukaemia (AML) after allogeneic haematopoietic stem cell transplantation (alloHSCT), and the effect of first salvage therapy and subsequent GVHD on OSR. METHODS: Data on 386 patients from nine Australian centres with relapsed AML post-alloHSCT were collected retrospectively. OSR was calculated using the Kaplan-Meier method. Univariate and multivariate analyses were conducted using the logrank test and proportional hazards modelling respectively, and a prognostic index for OSR was derived from multivariate modelling...
June 19, 2017: Internal Medicine Journal
https://www.readbyqxmd.com/read/28628088/an-individualised-risk-adapted-protocol-of-pre-and-post-transplant-zoledronic-acid-reduces-bone-loss-after-allogeneic-stem-cell-transplantation-results-of-a-phase-ii-prospective-trial
#5
A Grigg, B Butcher, B Khodr, A Bajel, M Hertzberg, S Patil, A B D'Souza, P Ganly, P Ebeling, E Wong
Bone loss occurs frequently following allogeneic haematopoietic stem cell transplantation (alloSCT). The Australasian Leukaemia and Lymphoma Group conducted a prospective phase II study of pretransplant zoledronic acid (ZA) and individualised post-transplant ZA to prevent bone loss in alloSCT recipients. Patients received ZA 4 mg before conditioning. Administration of post-transplant ZA from days 100 to 365 post alloSCT was determined by a risk-adapted algorithm based on serial bone density assessments and glucocorticoid exposure...
June 19, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28621766/autologous-haematopoietic-stem-cell-transplantation-for-treatment-of-multiple-sclerosis
#6
REVIEW
Paolo A Muraro, Roland Martin, Giovanni Luigi Mancardi, Richard Nicholas, Maria Pia Sormani, Riccardo Saccardi
Autologous haematopoietic stem cell transplantation (AHSCT) is a multistep procedure that enables destruction of the immune system and its reconstitution from haematopoietic stem cells. Originally developed for the treatment of haematological malignancies, the procedure has been adapted for the treatment of severe immune-mediated disorders. Results from ∼20 years of research make a compelling case for selective use of AHSCT in patients with highly active multiple sclerosis (MS), and for controlled trials...
June 16, 2017: Nature Reviews. Neurology
https://www.readbyqxmd.com/read/28620928/the-importance-of-the-number-of-transplanted-cells-with-dipeptidyl-peptidase-4-expression-on-the-haematopoietic-recovery-and-lymphocyte-reconstitution-in-patients-with-multiple-myeloma-after-autologous-haematopoietic-stem-cell-transplantation
#7
Anna Kopinska, Małgorzata Krawczyk-Kulis, Joanna Dziaczkowska-Suszek, Katarzyna Bieszczad, Krystyna Jagoda, Slawomira Kyrcz-Krzemien
Autologous haematopoietic stem cell transplantation (AHSCT) remains recommended treatment in the first remission in multiple myeloma (MM). In earlier research it has been suggested that there is an influence of the expression of dipeptidyl peptidase-4 (CD26) on both the homing and lymphocyte reconstitution after AHSCT. The aim of the study is to investigate the influence of transplanted cells CD26+ on the haematopoietic recovery and lymphocyte reconstitution after AHSCT in MM. Forty eight patients with MM underwent AHSCT in our centre...
June 2017: Hematological Oncology
https://www.readbyqxmd.com/read/28593997/severe-congenital-neutropenias
#8
REVIEW
Julia Skokowa, David C Dale, Ivo P Touw, Cornelia Zeidler, Karl Welte
Severe congenital neutropenias are a heterogeneous group of rare haematological diseases characterized by impaired maturation of neutrophil granulocytes. Patients with severe congenital neutropenia are prone to recurrent, often life-threatening infections beginning in their first months of life. The most frequent pathogenic defects are autosomal dominant mutations in ELANE, which encodes neutrophil elastase, and autosomal recessive mutations in HAX1, whose product contributes to the activation of the granulocyte colony-stimulating factor (G-CSF) signalling pathway...
June 8, 2017: Nature Reviews. Disease Primers
https://www.readbyqxmd.com/read/28593990/loss-of-asxl2-leads-to-myeloid-malignancies-in-mice
#9
Jianping Li, Fuhong He, Peng Zhang, Shi Chen, Hui Shi, Yanling Sun, Ying Guo, Hui Yang, Na Man, Sarah Greenblatt, Zhaomin Li, Zhengyu Guo, Yuan Zhou, Lan Wang, Lluis Morey, Sion Williams, Xi Chen, Qun-Tian Wang, Stephen D Nimer, Peng Yu, Qian-Fei Wang, Mingjiang Xu, Feng-Chun Yang
ASXL2 is frequently mutated in acute myeloid leukaemia patients with t(8;21). However, the roles of ASXL2 in normal haematopoiesis and the pathogenesis of myeloid malignancies remain unknown. Here we show that deletion of Asxl2 in mice leads to the development of myelodysplastic syndrome (MDS)-like disease. Asxl2(-/-) mice have an increased bone marrow (BM) long-term haematopoietic stem cells (HSCs) and granulocyte-macrophage progenitors compared with wild-type controls. Recipients transplanted with Asxl2(-/-) and Asxl2(+/-) BM cells have shortened lifespan due to the development of MDS-like disease or myeloid leukaemia...
June 8, 2017: Nature Communications
https://www.readbyqxmd.com/read/28589551/centre-characteristics-and-procedure-related-factors-have-an-impact-on-outcomes-of-allogeneic-transplantation-for-patients-with-cll-a-retrospective-analysis-from-the-european-society-for-blood-and-marrow-transplantation-ebmt
#10
Johannes Schetelig, Liesbeth C de Wreede, Niels S Andersen, Carol Moreno, Michel van Gelder, Antonin Vitek, Michal Karas, Mauricette Michallet, Maciej Machaczka, Martin Gramatzki, Dietrich Beelen, Jürgen Finke, Julio Delgado, Liisa Volin, Jakob Passweg, Peter Dreger, Nicolaas Schaap, Eva Wagner, Anja Henseler, Anja van Biezen, Martin Bornhäuser, Simona Iacobelli, Hein Putter, Stefan O Schönland, Nicolaus Kröger
The best approach for allogeneic haematopoietic stem cell transplantations (alloHCT) in patients with chronic lymphocytic leukaemia (CLL) is unknown. We therefore analysed the impact of procedure- and centre-related factors on 5-year event-free survival (EFS) in a large retrospective study. Data of 684 CLL patients who received a first alloHCT between 2000 and 2011 were analysed by multivariable Cox proportional hazards models with a frailty component to investigate unexplained centre heterogeneity. Five-year EFS of the whole cohort was 37% (95% confidence interval [CI], 34-42%)...
June 7, 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/28579852/treatment-of-myelofibrosis-old-and-new-strategies
#11
REVIEW
Alessandra Iurlo, Daniele Cattaneo
Myelofibrosis (MF) is a BCR-ABL1-negative myeloproliferative neoplasm that is mainly characterised by reactive bone marrow fibrosis, extramedullary haematopoiesis, anaemia, hepatosplenomegaly, constitutional symptoms, leukaemic progression, and shortened survival. As such, this malignancy is still orphan of curative treatments; indeed, the only treatment that has a clearly demonstrated impact on disease progression is allogeneic haematopoietic stem cell transplantation, but only a minority of patients are eligible for such intensive therapy...
2017: Clinical Medicine Insights. Blood Disorders
https://www.readbyqxmd.com/read/28560469/gene-therapy-for-lysosomal-storage-disorders-recent-advances-for-metachromatic-leukodystrophy-and-mucopolysaccaridosis-i
#12
REVIEW
Rachele Penati, Francesca Fumagalli, Valeria Calbi, Maria Ester Bernardo, Alessandro Aiuti
Lysosomal storage diseases (LSDs) are rare inherited metabolic disorders characterized by a dysfunction in lysosomes, leading to waste material accumulation and severe organ damage. Enzyme replacement therapy (ERT) and haematopoietic stem cell transplant (HSCT) have been exploited as potential treatments for LSDs but pre-clinical and clinical studies have shown in some cases limited efficacy. Intravenous ERT is able to control the damage of visceral organs but cannot prevent nervous impairment. Depending on the disease type, HSCT has important limitations when performed for early variants, unless treatment occurs before disease onset...
May 30, 2017: Journal of Inherited Metabolic Disease
https://www.readbyqxmd.com/read/28559537/high-dose-chemotherapy-with-autologous-haematopoietic-stem-cell-support-for-relapsed-or-refractory-primary-cns-lymphoma-a-prospective-multicentre-trial-by-the-german-cooperative-pcnsl-study-group
#13
B Kasenda, G Ihorst, R Schroers, A Korfel, I Schmidt-Wolf, G Egerer, L von Baumgarten, A Röth, J Bloehdorn, R Möhle, M Binder, U Keller, M Lamprecht, M Pfreundschuh, E Valk, H Fricker, E Schorb, K Fritsch, J Finke, G Illerhaus
To investigate safety and efficacy of high-dose chemotherapy followed by autologous stem cell transplantation (HCT-ASCT) in relapsed/refractory (r/r) primary CNS lymphoma (PCNSL). We conducted a single-arm multicentre study for immunocompetent patients (<66 years) with PCNSL failing high-dose methotrexate (HD-MTX) based chemotherapy. Induction consisted of 2 courses of rituximab (375 mg/m(2)), high-dose cytarabine (2 × 3 g/m(2)) and thiotepa (40 mg/m(2)) with collection of stem cells in between. Conditioning for HCT-ASCT consisted of rituximab 375 mg/m(2), carmustine 400 mg/m(2) and thiotepa (4 × 5 mg/kg)...
May 31, 2017: Leukemia: Official Journal of the Leukemia Society of America, Leukemia Research Fund, U.K
https://www.readbyqxmd.com/read/28551082/gynaecological-care-after-stem-cell-transplant-an-overview
#14
REVIEW
Annabelle Brennan, Martha Hickey
Haematopoietic stem cell transplants are an important treatment for haematological malignancies. With increasing efficacy of treatment, it has become important to provide comprehensive long-term follow-up by experienced specialists in a multidisciplinary setting. This article gives an overview of the gynaecological implications of haematopoietic stem cell transplants, including appropriate pre-treatment counselling, the development of menopausal symptoms, bone loss, genital graft-versus-host disease and secondary genital malignancies...
May 17, 2017: Maturitas
https://www.readbyqxmd.com/read/28542711/preventing-relapse-after-haematopoietic-stem-cell-transplantation-for-acute-leukaemia-the-role-of-post-transplantation-minimal-residual-disease-mrd-monitoring-and-mrd-directed-intervention
#15
REVIEW
Xiao-Dong Mo, Meng Lv, Xiao-Jun Huang
Relapse is the main cause of treatment failure after allogeneic haematopoietic stem cell transplantation (allo-HSCT) for acute leukaemia (AL). Post-transplantation minimal residual disease (MRD) monitoring enables risk stratification and identifies AL patients at higher risk of relapse. MRD assessment primarily involves the determination of leukaemia-associated immunophenotypic patterns using multiparameter flow cytometry, and the polymerase chain reaction (PCR)-based evaluation of expression levels of leukaemia-related genes (specific reciprocal gene rearrangements and other mutation types)...
May 23, 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/28539897/long-term-effects-of-haematopoietic-stem-cell-transplantation-after-pediatric-cancer-a-qualitative-analysis-of-life-experiences-and-adaptation-strategies
#16
Magali Lahaye, Isabelle Aujoulat, Christiane Vermylen, Bénédicte Brichard
Haematopoietic stem cell transplantation (HSCT) improves the survival rate of children and adolescents with malignant and non-malignant conditions; however, the physical, psychological and social burden of such a procedure is considerable both during and after treatment. The present qualitative study investigated the long-term effects of HSCT after pediatric cancer. Thirty adolescent and young adult (AYA) survivors (Mage = 23.61 years, SD = 5.21) participated in individual interviews and were invited to speak about their life experiences following their treatment and strategies they use to deal with their past medical experiences and the long-term sequelae...
2017: Frontiers in Psychology
https://www.readbyqxmd.com/read/28528807/comparison-of-amicus-and-cobe-spectra-for-allogenic-peripheral-blood-stem-cell-harvest-study-from-tertiary-care-centre-in-india
#17
Rasika Dhawan Setia, Satyam Arora, Anil Handoo, Tina Dadu, Dharma Choudhary, Sajeev Kumar Sharma, Gaurav Kharya, Vipin Khandelwal, Prerna Sachdeva, Divya Doval, Anamika Bakliwal, Meenu Kapoor, Shalu Bajaj, Virendra Bachchas, Praveen Singh
INTRODUCTION: Most common source of stem cell graft for both autologous and allogenic haematopoietic transplants are peripheral blood haematopoietic progenitor stem cells. Adequate collection of the CD34+ cells and safety of the allogenic donor during the leukapheresis are of prime importance to an apheresis physician. Our retrospective analysis is a comparison between of two platforms namely, COBE Spectra and Amicus, for CD34+ mononuclear cell collection. MATERIAL AND METHOD: The study included the data of GSCF (Granulocyte-Colony-Stimulating Factor) mobilized allogenic PBSC collections at our centre from January 2015 to June 2016...
April 24, 2017: Transfusion and Apheresis Science
https://www.readbyqxmd.com/read/28516949/paroxysmal-nocturnal-haemoglobinuria
#18
REVIEW
Anita Hill, Amy E DeZern, Taroh Kinoshita, Robert A Brodsky
Paroxysmal nocturnal haemoglobinuria (PNH) is a clonal haematopoietic stem cell (HSC) disease that presents with haemolytic anaemia, thrombosis and smooth muscle dystonias, as well as bone marrow failure in some cases. PNH is caused by somatic mutations in PIGA (which encodes phosphatidylinositol N-acetylglucosaminyltransferase subunit A) in one or more HSC clones. The gene product of PIGA is required for the biosynthesis of glycosylphosphatidylinositol (GPI) anchors; thus, PIGA mutations lead to a deficiency of GPI-anchored proteins, such as complement decay-accelerating factor (also known as CD55) and CD59 glycoprotein (CD59), which are both complement inhibitors...
May 18, 2017: Nature Reviews. Disease Primers
https://www.readbyqxmd.com/read/28513891/haematopoietic-stem-cell-transplantation-for-severe-combined-immunodeficiency-long-term-health-outcomes-and-patient-perspectives
#19
Adrian Ys Lee, Katie Frith, Lilian Schneider, John B Ziegler
AIM: To examine the long-term follow-up and health outcomes of patients who have undergone haematopoietic stem cell transplant (HSCT) for severe combined immunodeficiency (SCID). METHODS: Through a structured questionnaire, we examined follow-up arrangements and long-term health outcomes in 22 children who have had a successful HSCT for SCID during the period of 1984-2012 at the Sydney Children's Hospital, Sydney, Australia. RESULTS: Most children considered themselves healthy and 'cured' from SCID...
May 17, 2017: Journal of Paediatrics and Child Health
https://www.readbyqxmd.com/read/28504663/a-new-time-dependent-approach-for-assessment-of-the-impact-of-invasive-aspergillosis-shows-effect-on-short-but-not-on-long-term-survival-of-patients-with-aml-or-high-risk-mds
#20
R J van de Peppel, P A von dem Borne, S le Cessie, M G J de Boer
Invasive aspergillosis (IA) has been reported to yield high mortality rates. Patients with an unfavourable prognostic haematological disease not only have a higher probability of developing IA but are also more likely to die due to causes directly related to the underlying disease. This complexity of risk mechanisms confounds the causal interpretation of IA occurrence and mortality. Full consideration of the changing patient characteristics over time is necessary to obtain reliable estimates of the correlation of IA with mortality...
June 2017: Bone Marrow Transplantation
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