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haematopoietic stem cell transplant

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https://www.readbyqxmd.com/read/27893415/pentraxin-3-plasma-levels-at-graft-versus-host-disease-onset-predict-disease-severity-and-response-to-therapy-in-children-given-haematopoietic-stem-cell-transplantation
#1
Erica Dander, Paola De Lorenzo, Barbara Bottazzi, Paola Quarello, Paola Vinci, Adriana Balduzzi, Francesca Masciocchi, Sonia Bonanomi, Claudia Cappuzzello, Giulia Prunotto, Fabio Pavan, Fabio Pasqualini, Marina Sironi, Ivan Cuccovillo, Roberto Leone, Giovanni Salvatori, Matteo Parma, Elisabetta Terruzzi, Fabio Pagni, Franco Locatelli, Alberto Mantovani, Franca Fagioli, Andrea Biondi, Cecilia Garlanda, Maria Grazia Valsecchi, Attilio Rovelli, Giovanna D'Amico
Acute Graft-versus-Host Disease (GvHD) remains a major complication of allogeneic haematopoietic stem cell transplantation, with a significant proportion of patients failing to respond to first-line systemic corticosteroids. Reliable biomarkers predicting disease severity and response to treatment are warranted to improve its management. Thus, we sought to determine whether pentraxin 3 (PTX3), an acute-phase protein produced locally at the site of inflammation, could represent a novel acute GvHD biomarker. Using a murine model of the disease, we found increased PTX3 plasma levels after irradiation and at GvHD onset...
November 21, 2016: Oncotarget
https://www.readbyqxmd.com/read/27892949/the-ebmt-eln-working-group-recommendations-on-the-prophylaxis-and-treatment-of-gvhd-a-change-control-analysis
#2
T Ruutu, A Gratwohl, D Niederwieser, T de Witte, S van der Werf, A van Biezen, M Mohty, N Kröger, A Rambaldi, E McGrath, A Sureda, G Basak, H Greinix, R F Duarte
In 2013, recommendations for a standardized practice in the prophylaxis and treatment of GvHD were adopted and published by the European Society for Blood and Marrow Transplantation and the European LeukemiaNet. One year later, all 341 European Society for Blood and Marrow Transplantation centres performing allogeneic haematopoietic stem cell transplantation were contacted for a change-control analysis and asked to fill in a questionnaire; 111 centres (33%) responded. Of these, 83% had been aware of the recommendations...
November 28, 2016: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/27892944/veno-occlusive-disease-sinusoidal-obstruction-syndrome-after-haematopoietic-stem-cell-transplantation-middle-east-north-africa-regional-consensus-on-prevention-diagnosis-and-management
#3
A H Al Jefri, H Abujazar, A Al-Ahmari, A Al Rawas, Z Al Zahrani, A Alhejazi, M A Bekadja, A Ibrahim, M Lahoucine, S Ousia, A Bazarbachi
Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) of the liver is a serious, early complication of haematopoietic stem cell transplantation (HSCT), severe and very severe forms of which are associated with a high mortality rate. A wide variety of patient, disease and treatment-related risk factors for VOD/SOS have been identified. Several bodies have published recommendations for the diagnosis, prevention and management of VOD/SOS following HSCT. A group of regional experts have developed a consensus statement on the diagnosis, prevention and management of VOD/SOS in the Middle East and North Africa region to help in the management of HSCT patients in the region...
November 28, 2016: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/27879990/sequential-chemotherapy-followed-by-reduced-intensity-conditioning-and-allogeneic-haematopoietic-stem-cell-transplantation-in-adult-patients-with-relapse-or-refractory-acute-myeloid-leukaemia-a-survey-from-the-acute-leukaemia-working-party-of-ebmt
#4
Olle Ringdén, Myriam Labopin, Christoph Schmid, Behnam Sadeghi, Emmanuelle Polge, Johanna Tischer, Arnold Ganser, Mauricette Michallet, Lothar Kanz, Rainer Schwerdtfeger, Arnon Nagler, Mohamad Mohty
This study analysed the outcome of 267 patients with relapse/refractory acute myeloid leukaemia (AML) who received sequential chemotherapy including fludarabine, cytarabine and amsacrine followed by reduced-intensity conditioning (RIC) and allogeneic haematopoietic stem cell transplantation (HSCT). The transplants in 77 patients were from matched sibling donors (MSDs) and those in 190 patients were from matched unrelated donors. Most patients (94·3%) were given anti-T-cell antibodies. The incidence of acute graft-versus-host disease (GVHD) of grades II-IV was 32·1% and that of chronic GVHD was 30·2%...
November 23, 2016: British Journal of Haematology
https://www.readbyqxmd.com/read/27870830/clonal-fate-mapping-quantifies-the-number-of%C3%A2-haematopoietic-stem-cells-that-arise-during%C3%A2-development
#5
Jonathan Henninger, Buyung Santoso, Stefan Hans, Ellen Durand, Jessica Moore, Christian Mosimann, Michael Brand, David Traver, Leonard Zon
Haematopoietic stem cells (HSCs) arise in the developing aorta during embryogenesis. The number of HSC clones born has been estimated through transplantation, but experimental approaches to assess the absolute number of forming HSCs in a native setting have remained challenging. Here, we applied single-cell and clonal analysis of HSCs in zebrafish to quantify developing HSCs. Targeting creER(T2) in developing cd41:eGFP(+) HSCs enabled long-term assessment of their blood contribution. We also applied the Brainbow-based multicolour Zebrabow system with drl:creER(T2) that is active in early haematopoiesis to induce heritable colour barcoding unique to each HSC and its progeny...
November 21, 2016: Nature Cell Biology
https://www.readbyqxmd.com/read/27870355/impact-of-genomic-risk-factors-on-survival-after-haematopoietic-stem-cell-transplantation-for-patients-with-acute-leukaemia
#6
K F Pearce, Y Balavarca, J Norden, G Jackson, E Holler, R Dressel, H Greinix, A Toubert, E Gluckman, I Hromadnikova, P Sedlacek, D Wolff, U Holtick, H Bickeböller, A M Dickinson
The EBMT risk score is an established tool successfully used in the prognosis of survival post-HSCT and is applicable for a range of haematological disorders. One of its main advantages is that score generation involves summation of clinical parameters that are available pretransplant. However, the EBMT risk score is recognized as not being optimal. Previous analyses, involving patients with various diagnoses, have shown that non-HLA gene polymorphisms influence outcome after allogeneic HSCT. This study is novel as it focuses only on patients having acute leukaemia (N = 458) and attempts to demonstrate how non-HLA gene polymorphisms can be added to the EBMT risk score in a Cox regression model to improve prognostic ability for overall survival...
December 2016: International Journal of Immunogenetics
https://www.readbyqxmd.com/read/27863874/successful-autologous-haematopoietic-stem-cell-transplantation-for-refractory-myasthenia-gravis-a-case-report
#7
Irene Håkansson, Anna Sandstedt, Fredrik Lundin, Håkan Askmark, Ritva Pirskanen, Kristina Carlson, Fredrik Piehl, Hans Hägglund
Myasthenia gravis (MG) is an autoimmune disease, with immune reactivity against the post-synaptic endplate of the neuromuscular junction. Apart from symptomatic treatment with choline esterase blockers, many patients also require immunomodulatory treatment. Despite existing treatment options, some patients are treatment refractory. We describe a patient with severe MG refractory to corticosteroids, four oral immunosuppressants, cyclophosphamide, rituximab and bortezomib who was treated with autologous haematopoietic stem cell transplantation...
September 28, 2016: Neuromuscular Disorders: NMD
https://www.readbyqxmd.com/read/27859940/family-members-life-situation-and-experiences-of-different-caring-organisations-during-allogeneic-haematopoietic-stem-cells-transplantation-a-qualitative-study
#8
K Bergkvist, J Larsen, U-B Johansson, J Mattsson, B Fossum
The aim of this study was to describe family members' life situation and experiences of care in two different care settings, the patient's home or in hospital during the acute post-transplantation phase after allogeneic haematopoietic stem cell transplantation (HSCT). Data were collected through semi-structured interviews with 14 family members (seven women and seven men). An inductive qualitative content analysis was used to analyse the data. The majority of the family members' (n = 10) had experiences from home care...
November 17, 2016: European Journal of Cancer Care
https://www.readbyqxmd.com/read/27858309/dental-care-approach-in-patients-with-osteopetrosis
#9
V Detailleur, G Vansteenkiste, M Renard, A Verdonck
AIM: To describe dental and dentofacial characteristics observed in patients diagnosed with osteopetrosis and to advise a dental care approach in these patients. METHODS: Four patients were clinically diagnosed with osteopetrosis, characterised by increased bone density, bone marrow failure, blindness and deafness due to compression of cranial nerves. All patients were dentally screened at different ages (2.5-31 years) and three of them were treated with a haematopoietic stem cell transplantation (HSCT) at the age of 6 months, 1 and 3...
November 17, 2016: European Archives of Paediatric Dentistry: Official Journal of the European Academy of Paediatric Dentistry
https://www.readbyqxmd.com/read/27856159/pre-implantation-genetic-diagnosis
#10
REVIEW
Joanne Traeger-Synodinos
The aim of pre-implantation genetic diagnosis (PGD) is to characterize the genetic status of the cells (usually single cells) that have been biopsied from oocytes/zygotes or embryos created in vitro during assisted reproductive treatment. PGD is a multi-step procedure that requires close collaboration between gynaecologists who are experts in assisted reproduction, embryologists who are experts in micromanipulation of germ cells and in embryo biopsy and geneticists who are experts in genetic analysis at the single-cell level...
October 26, 2016: Best Practice & Research. Clinical Obstetrics & Gynaecology
https://www.readbyqxmd.com/read/27842866/reviewing-the-importance-and-evolution-of-fungal-infections-and-potential-antifungal-resistance-in-haematological-patients
#11
REVIEW
Alessandro Busca, Anna Maria Tortorano, Livio Pagano
There is a growing body of evidence supporting the emergence of azole resistance in Aspergillus and Candida spp. and this may be of particular concern due to the potential implications in the management of invasive fungal infections occurring in immunocompromised hosts. The aim of present review was to describe the magnitude of the problem, summarising the epidemiology and potential impact of yeast and mould antifungal resistance in patients with haematological malignancies. The first cases of triazole-resistant Aspergillus fumigatus isolates were reported in 1997 in patients receiving itraconazole...
December 2015: Journal of Global Antimicrobial Resistance
https://www.readbyqxmd.com/read/27833447/immune-recovery-and-the-risk-of-cmv-ebv-reactivation-in-children-post-allogeneic-haematopoietic-stem-cell-transplantation
#12
Małgorzata Janeczko, Monika Mielcarek, Blanka Rybka, Renata Ryczan-Krawczyk, Dorota Noworolska-Sauren, Krzysztof Kałwak
Immune reconstitution was studied prospectively in 86 children who underwent allogeneic haematopoietic stem cell transplantation (HSCT). We analysed the risk of cytomegalovirus (CMV) and Epstein-Barr virus (EBV) reactivation in correlation with the kinetics of immune recovery and in relation to other potential risk factors that may influence the reactivation of these viruses including: diagnosis, type of HSCT, source of stem cells, type of conditioning, or the occurrence of graft-versus-host disease (GvHD)...
2016: Central-European Journal of Immunology
https://www.readbyqxmd.com/read/27832748/invasive-mucormycosis-in-children-an-epidemiologic-study-in-european-and-non-european-countries-based-on-two-registries
#13
Zoi Dorothea Pana, Danila Seidel, Anna Skiada, Andreas H Groll, Georgios Petrikkos, Oliver A Cornely, Emmanuel Roilides
BACKGROUND: Mucormycosis has emerged as a rare but frequently fatal invasive fungal disease. Current knowledge on paediatric mucormycosis is based on case reports and small series reported over several decades. Contemporary data on a large cohort of patients is lacking. METHODS: Two large international registries (Zygomyco.net and FungiScope™) were searched for mucormycosis cases in ≤19 year-old patients. Cases enrolled between 2005 and 2014 were extracted, and dual entries in the two databases merged...
November 10, 2016: BMC Infectious Diseases
https://www.readbyqxmd.com/read/27820943/crispr-cas9-%C3%AE-globin-gene-targeting-in-human-haematopoietic-stem-cells
#14
Daniel P Dever, Rasmus O Bak, Andreas Reinisch, Joab Camarena, Gabriel Washington, Carmencita E Nicolas, Mara Pavel-Dinu, Nivi Saxena, Alec B Wilkens, Sruthi Mantri, Nobuko Uchida, Ayal Hendel, Anupama Narla, Ravindra Majeti, Kenneth I Weinberg, Matthew H Porteus
The β-haemoglobinopathies, such as sickle cell disease and β-thalassaemia, are caused by mutations in the β-globin (HBB) gene and affect millions of people worldwide. Ex vivo gene correction in patient-derived haematopoietic stem cells followed by autologous transplantation could be used to cure β-haemoglobinopathies. Here we present a CRISPR/Cas9 gene-editing system that combines Cas9 ribonucleoproteins and adeno-associated viral vector delivery of a homologous donor to achieve homologous recombination at the HBB gene in haematopoietic stem cells...
November 7, 2016: Nature
https://www.readbyqxmd.com/read/27819688/allogeneic-haematopoietic-stem-cell-transplant-in-patients-with-lower-risk-myelodysplastic-syndrome-a-retrospective-analysis-on-behalf-of-the-chronic-malignancy-working-party-of-the-ebmt
#15
M Robin, R Porcher, W Zinke-Cerwenka, A van Biezen, L Volin, G Mufti, C Craddock, J Finke, C Richard, J Passweg, A Peniket, J Maertens, G Sucak, T Gedde-Dahl, A Vitek, A Nagler, D Blaise, D Beelen, N Maillard, R Schwerdtfeger, T de Witte, N Kroger
We report a retrospective analysis of 246 myelodysplastic syndrome (MDS) patients in the EBMT (The European Society for Blood and Marrow Transplantation) database who were transplanted for International Prognostic Scoring System (IPSS) low or intermediate-1 disease. The majority of these patients (76%) were reclassified as intermediate or higher risk according to R-IPSS. The 3-year overall survival (OS) and PFS were 58% and 54%, respectively. In a multivariate analysis, adverse risk factors for PFS were marrow blast percentage (hazard ratio (HR): 1...
November 7, 2016: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/27798034/rationale-and-design-of-platelet-transfusions-in-haematopoietic-stem-cell-transplantation-the-path-pilot-study
#16
Jason Tay, David Allan, Sara Beattie, Christopher Bredeson, Dean Fergusson, Dawn Maze, Mitchell Sabloff, Kednapa Thavorn, Alan Tinmouth
INTRODUCTION: In patients with transient thrombocytopenia being treated with high-dose chemotherapy followed by stem cell rescue-haematopoietic stem cell transplantation (HSCT), prophylactic transfusions are standard therapy to prevent bleeding. However, a recent multicentre trial suggests that prophylactic platelet transfusions in HSCT may not be necessary. Additionally, the potential overuse of platelet products places a burden on a scarce healthcare resource. Moreover, the benefit of prophylactic platelet transfusions to prevent clinically relevant haemorrhage is debatable...
October 24, 2016: BMJ Open
https://www.readbyqxmd.com/read/27788917/-nutritional-management-for-patients-hospitalized-during-allogeneic-stem-cell-transplantation-guidelines-from-the-francophone-society-of-bone-marrow-transplantation-and-cellular-therapy-sfgm-tc
#17
Jacques-Olivier Bay, Caroline Dendoncker, Marie Angeli, Thomas Biot, Marinette Chikhi, Cécile Combal, Line Jouannic, Guénola Kermeur, Lisa Lopvet, Tony Marchand, Stéphanie Schmitt, Sophie Servais, Ibrahim Yakoub-Agha, David Seguy
Allogeneic haematopoietic stem-cell transplantation is usually applied with success for patients with diseases involving bone marrow and associated with frequent and severe malnutrition. Denutrition is an independent survival factor and contribute to transplant-related mortality. Due to the heaviness of the treatment, this event is frequent. Before allogeneic transplantation, the nutritional statute should be evaluated. The adult or pediatric patient's evaluation modalities and nutritional needs are detailed in this paper...
November 2016: Bulletin du Cancer
https://www.readbyqxmd.com/read/27784228/a-perspective-on-stem-cells-as-biological-systems-that-produce-differentiated-osteoblasts-and-odontoblasts
#18
Shahrul Hisham Zainal Ariffin, Thanaletchumi Manogaran, Intan Zarina Zainol Abidin, Rohaya Megat Abdul Wahab, Sahidan Senafi
Stem cells (SCs) are capable of self-renewal and multilineage differentiation. Human mesenchymal stem cells (MSCs) and haematopoietic stem cells (HSCs) which can be obtained from multiple sources, are suitable for application in regenerative medicine and transplant therapy. The aim of this review is to evaluate the potential of genomic and proteomic profiling analysis to identify the differentiation of MSCs and HSCs towards osteoblast and odontoblast lineages. In vitro differentiation towards both of these lineages can be induced using similar differentiation factors...
October 26, 2016: Current Stem Cell Research & Therapy
https://www.readbyqxmd.com/read/27783593/leukaemogenic-effects-of-ptpn11-activating-mutations-in-the-stem-cell-microenvironment
#19
Lei Dong, Wen-Mei Yu, Hong Zheng, Mignon L Loh, Silvia T Bunting, Melinda Pauly, Gang Huang, Muxiang Zhou, Hal E Broxmeyer, David T Scadden, Cheng-Kui Qu
Germline activating mutations of the protein tyrosine phosphatase SHP2 (encoded by PTPN11), a positive regulator of the RAS signalling pathway, are found in 50% of patients with Noonan syndrome. These patients have an increased risk of developing leukaemia, especially juvenile myelomonocytic leukaemia (JMML), a childhood myeloproliferative neoplasm (MPN). Previous studies have demonstrated that mutations in Ptpn11 induce a JMML-like MPN through cell-autonomous mechanisms that are dependent on Shp2 catalytic activity...
October 26, 2016: Nature
https://www.readbyqxmd.com/read/27781311/phellinus-species-an-emerging-cause-of-refractory-fungal-infections-in-patients-with-x-linked-chronic-granulomatous-disease
#20
REVIEW
Ghady Haidar, Christa S Zerbe, Michelle Cheng, Adrian M Zelazny, Steven M Holland, Kathleen R Sheridan
Aspergillus spp. are a leading cause of mortality in chronic granulomatous disease (CGD), but other fungi have emerged in the era of mould prophylaxis. Of these, Phellinus spp. are an under-recognised cause of invasive fungal infections (IFIs) in CGD, and data on their presentation and management are scarce. We present a patient with CGD who developed disseminated IFI involving the lungs and brain. Surgical specimens grew a basidiomycete which was disregarded as a contaminant. After three months of progressive disease despite antifungals, he was diagnosed with Phellinus tropicalis by internal transcribed spacer (ITS) sequencing...
October 26, 2016: Mycoses
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