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haematopoietic stem cell transplant

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https://www.readbyqxmd.com/read/29155317/treosulfan-fludarabine-conditioning-for-hsct-in-children-with-primary-immunodeficiency-uk-experience
#1
Mary A Slatter, Kanchan Rao, Intan Juliana Abd Hamid, Zohreh Nademi, Robert Chiesa, Reem Elfeky, Mark S Pearce, Persis Amrolia, Austen Worth, Terence Flood, Mario Abinun, Sophie Hambleton, Waseem Qasim, Hubert B Gaspar, Andrew J Cant, Andrew R Gennery, Paul Veys
We previously published results of 70 children who received treosulfan with cyclophosphamide (30) or fludarabine (40) before haematopoietic stem cell transplantation (HSCT) for Primary Immunodeficiency (PID). Toxicity was lower and T cell chimerism better in those receiving fludarabine, but numbers were relatively small and follow-up short. We now report outcome of 160 children who received homogeneous conditioning with treosulfan, fludarabine mostly with alemtuzumab (n=124). Median age at transplant was 1...
November 16, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29143315/fetal-haemoglobin-induction-in-sickle-cell-disease
#2
REVIEW
Alireza Paikari, Vivien A Sheehan
Fetal haemoglobin (HbF, α2γ2) induction has long been an area of investigation, as it is known to ameliorate the clinical complications of sickle cell disease (SCD). Progress in identifying novel HbF-inducing strategies has been stymied by limited understanding of gamma (γ)-globin regulation. Genome-wide association studies (GWAS) have identified variants in BCL11A and HBS1L-MYB that are associated with HbF levels. Functional studies have established the roles of BCL11A, MYB, and KLF1 in γ-globin regulation, but this information has not yielded new pharmacological agents...
November 16, 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/29143281/long-term-outcome-in-patients-treated-at-home-during-the-pancytopenic-phase-after-allogeneic-haematopoietic-stem-cell-transplantation
#3
Olle Ringdén, Behnam Sadeghi, Gianluca Moretti, Sigrun Finnbogadottir, Brita Eriksson, Jonas Mattsson, Britt-Marie Svahn, Mats Remberger
Patients undergoing allogeneic haematopoietic stem cell transplantation (HSCT) were given the option to be treated at home during the pancytopenic phase. Daily visits by a nurse and phone calls from a physician from the unit were part of the protocol. During almost two decades, 252 patients with haematological malignancies and non-malignant disorders were included. Median age was 47 (range 0-72) years. Myeloablative conditioning was given to 102 patients and reduced intensity to 150. Donors were matched unrelated (n = 160), HLA-identical siblings (n = 71), or HLA-mismatched (n = 21)...
November 15, 2017: International Journal of Hematology
https://www.readbyqxmd.com/read/29138099/effect-of-an-antimicrobial-stewardship-programme-on-antimicrobial-utilisation-and-costs-in-patients-with-leukaemia-a-retrospective-interventional-controlled-study
#4
Miranda So, Muhammad Mamdani, Andrew Morris, Tim Lau, Raewyn Broady, Uday Deotare, Jennifer Grant, Dennis Dong Hwan Kim, Aaron D Schimmer, Andre Schuh, Salomeh Shajari, Marilyn Steinberg, Chaim M Bell, Shahid Husain
OBJECTIVES: To examine the effectiveness of an antimicrobial stewardship programme (ASPs) on utilisation and cost of antimicrobials in leukaemia patients in Canada. METHODS: We conducted a multisite, retrospective, observational time-series study from 2005 to 2013. We implemented academic detailing as the intervention of an ASP in leukaemia units at a hospital, piloted February-July 2010, then fully implemented December 2010-March 2013, with no intervention in August-November 2010...
November 11, 2017: Clinical Microbiology and Infection
https://www.readbyqxmd.com/read/29136088/abcd1-dysfunction-alters-white-matter-microvascular-perfusion
#5
Arne Lauer, Xiao Da, Mikkel Bo Hansen, Gregoire Boulouis, Yangming Ou, Xuezhu Cai, Afonso Liberato Celso Pedrotti, Jayashree Kalpathy-Cramer, Paul Caruso, Douglas L Hayden, Natalia Rost, Kim Mouridsen, Florian S Eichler, Bruce Rosen, Patricia L Musolino
Cerebral X-linked adrenoleukodystrophy is a devastating neurodegenerative disorder caused by mutations in the ABCD1 gene, which lead to a rapidly progressive cerebral inflammatory demyelination in up to 60% of affected males. Selective brain endothelial dysfunction and increased permeability of the blood-brain barrier suggest that white matter microvascular dysfunction contributes to the conversion to cerebral disease. Applying a vascular model to conventional dynamic susceptibility contrast magnetic resonance perfusion imaging, we demonstrate that lack of ABCD1 function causes increased capillary flow heterogeneity in asymptomatic hemizygotes predominantly in the white matter regions and developmental stages with the highest probability for conversion to cerebral disease...
November 9, 2017: Brain: a Journal of Neurology
https://www.readbyqxmd.com/read/29131156/impact-of-choice-timing-sequence-and-combination-of-broad-spectrum-antibiotics-on-the-outcome-of-allogeneic-haematopoietic-stem-cell-transplantation
#6
F Farowski, V Bücker, J J Vehreschild, L Biehl, R Cruz-Aguilar, C Scheid, U Holtick, N Jazmati, H Wisplinghoff, O A Cornely, M J G T Vehreschild
Recent data link the incidence of intestinal GvHD (iGvHD) after allogeneic haematopoietic stem cell transplantation (aSCT) to exposure with piperacillin-tazobactam or imipenem-cilastatin. To assess relevance of timing, duration, sequence and combination of antibiotic treatment in this setting, we applied a time-dependent model to our aSCT cohort. Patients from the prospective Cologne Cohort of Neutropenic Patients (CoCoNut) undergoing aSCT from January 2007 to April 2013 were included into a time-dependent multivariate Cox proportional hazards regression model with backward-stepwise selection...
November 13, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/29128933/proven-invasive-pulmonary-aspergillosis-in-stem-cell-transplant-recipient-due-to-aspergillus-sublatus-a-cryptic-species-of-a-nidulans
#7
Vanda Chrenkova, Vit Hubka, Petr Cetkovsky, Michal Kouba, Barbora Weinbergerova, Pavlina Lyskova, Ludmila Hornofova, Petr Hubacek
Invasive fungal disease represents one of the severe complications in haematopoietic stem cell transplant recipients. We describe a case of a patient treated for relapse of chronic lymphoblastic leukaemia 6 years after HSCT. The patient was treated for invasive pulmonary aspergillosis but died 3 months later from multiple organ failures consisting of haemorrhagic necrotizing fungal pneumonia, refractory chronic hepatic graft versus host disease and cytomegalovirus hepatitis. Autopsy samples revealed histopathological evidence of fungal hyphae and an unusual Aspergillus nidulans-like species was isolated in pure culture...
November 11, 2017: Mycopathologia
https://www.readbyqxmd.com/read/29125439/intense-immunosuppression-followed-by-autologous-haematopoietic-stem-cell-transplantation-as-a-therapeutic-strategy-in-aggressive-forms-of-multiple-sclerosis
#8
Gianluigi Mancardi, Maria Pia Sormani, Paolo A Muraro, Giacomo Boffa, Riccardo Saccardi
In the majority of relapsing multiple sclerosis patients, the disease can be quite easily controlled by already available, approved therapies. There are, however, some aggressive cases who continue to have clinical and magnetic resonance imaging (MRI) activity in spite of the treatment. These are the cases who may now receive benefit from intense immunosuppression followed by autologous haematopoietic stem cell transplantation (aHSCT). In this review, we describe the method and the rationale of aHSCT, the more recently published studies that demonstrate its efficacy in selected multiple sclerosis cases, the problems related to safety and the transplant-related mortality risk of the procedure...
November 1, 2017: Multiple Sclerosis: Clinical and Laboratory Research
https://www.readbyqxmd.com/read/29116023/caffeic-acid-phenethyl-ester-promotes-haematopoietic-stem-progenitor-cell-homing-and-engraftment
#9
Xiaofang Chen, Yi Han, Bowen Zhang, Yiming Liu, Sihan Wang, Tuling Liao, Ziliang Deng, Zeng Fan, Jing Zhang, Lijuan He, Wen Yue, Yanhua Li, Xuetao Pei
BACKGROUND: Several studies have suggested that caffeic acid phenethyl ester (CAPE) can induce the expression of hypoxia inducible factor-1α (HIF-1α) protein. We determined whether CAPE has a novel function in improving the homing and engraftment of haematopoietic stem/progenitor cells (HSPCs) by regulating HIF-1α gene expression in the bone marrow (BM) niche. METHODS: For survival experiments, lethally irradiated C57BL/6 mice were injected with a low number of BM mononuclear cells (MNCs) and CAPE according to the indicated schedule...
November 7, 2017: Stem Cell Research & Therapy
https://www.readbyqxmd.com/read/29105206/risk-factors-for-psychological-and-psychosomatic-symptoms-among-children-with-malignancies
#10
Kenichi Maeda, Daisuke Hasegawa, Kevin Y Urayama, Shinichi Tsujimoto, Yuriko Azami, Miwa Ozawa, Atsushi Manabe
AIM: With increasing survival rates in paediatric malignancies, the quality-of-life of children during hospitalisation should be given more attention. We aimed to identify factors associated with psychological and psychosomatic symptoms (PPS) that required medication among children hospitalised for treatment of malignancies. METHODS: We retrospectively analysed data of 190 patients aged 2-18 years old. They were diagnosed with malignant diseases and admitted for treatment at St...
November 3, 2017: Journal of Paediatrics and Child Health
https://www.readbyqxmd.com/read/29090451/treatment-strategies-for-lysosomal-storage-disorders
#11
REVIEW
Michael Beck
Over the past several years the number of treatments available for patients with lysosomal storage disorders has rapidly increased. Haematopoietic stem cell transplantation, enzyme replacement therapy, substrate reduction, and chaperone therapies are currently available, and gene therapies and other treatments are rapidly advancing. Despite remarkable advances, the efficacy of most of these therapies is limited, particularly because the treatments are usually initiated when organ damage has already occurred...
November 1, 2017: Developmental Medicine and Child Neurology
https://www.readbyqxmd.com/read/29080718/sex-specific-survival-difference-in-association-with-hla-drb1%C3%A2-04-following-allogeneic-haematopoietic-stem-cell-transplantation-for-lymphoid-malignancies
#12
Katalin Balassa, Hajnalka Andrikovics, Peter Remenyi, Arpad Batai, Aniko Szilvasi, Andras Bors, Katalin Piroska Kiss, Katalin Rajczy, Dora Inotai, Eva Torbagyi, Lilla Lengyel, Aniko Barta, Laszlo Gopcsa, Attila Tordai, Tamas Masszi
The role of HLA system in allogeneic haematopoietic stem cell transplantation (allo-HSCT) outcome is unarguable. In this study we investigated association of HLA-A,-B and-DRB1 alleles with overall survival (OS) in 186 patients undergoing allo-HSCT for lymphoid malignancies. Analyses confirmed significantly better OS for HLA-DRB1∗04 carriers compared with non-carriers (p=0.01). Survival benefit was confined to male patients (in multivariate analyses p=0.034, hazard ratio 0.35, 95% confidence interval 0.13-0...
October 25, 2017: Human Immunology
https://www.readbyqxmd.com/read/29076145/durable-graft-versus-leukaemia-effects-without-donor-lymphocyte-infusions-results-of-a-phase-ii-study-of-sequential-t-replete-allogeneic-transplantation-for-high-risk-acute-myeloid-leukaemia-and-myelodysplasia
#13
Jeff K Davies, Sandra Hassan, Shah-Jalal Sarker, Caroline Besley, Heather Oakervee, Matthew Smith, David Taussig, John G Gribben, Jamie D Cavenagh
Allogeneic haematopoietic stem-cell transplantation remains the only curative treatment for relapsed/refractory acute myeloid leukaemia (AML) and high-risk myelodysplasia but has previously been limited to patients who achieve remission before transplant. New sequential approaches employing T-cell depleted transplantation directly after chemotherapy show promise but are burdened by viral infection and require donor lymphocyte infusions (DLI) to augment donor chimerism and graft-versus-leukaemia effects. T-replete transplantation in sequential approaches could reduce both viral infection and DLI usage...
October 26, 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/29076142/donor-derived-cd19-targeted-t-cell-infusion-induces-minimal-residual-disease-negative-remission-in-relapsed-b-cell-acute-lymphoblastic-leukaemia-with-no-response-to-donor-lymphocyte-infusions-after-haploidentical-haematopoietic-stem-cell-transplantation
#14
Yuhong Chen, Yifei Cheng, Pan Suo, Chenhua Yan, Yu Wang, Yao Chen, Wei Han, Lanping Xu, Xiaohui Zhang, Kaiyan Liu, Lungji Chang, Lei Xiao, Xiaojun Huang
Relapse is a common cause of failure in patients with B-cell acute lymphoblastic leukaemia (B-ALL) after haploidentical haematopoietic stem cell transplantation (haplo-HSCT), and non-responders to donor lymphoblastic infusion after HSCT have a very poor prognosis. Although donor-derived CD19-directed chimeric antigen receptor-modified (CAR) T cells can potentially cure leukaemia, their effectiveness and safety have not been confirmed in relapsed B-ALL cases after haplo-HSCT. Between January 2015 and January 2017, two and four patients each received one and two infusions of CAR T cells from haplo-HSCT donors...
October 26, 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/29057425/cutaneous-chronic-graft-versus-host-disease-following-allogeneic-haematopoietic-stem-cell-transplantation-in-children-a-retrospective-study
#15
Rony Shreberk-Hassidim, Michal Neumark, Shoshana Greenberger, Gal Goldstein, Ayal Hassidim, Yuval Dukler, Alexander Maly, Polina Stepensky, Vered Molho-Pessach
Chronic graft versus host disease (cGVHD) is a complication of allogeneic haematopoietic stem cell transplantation (HSCT). The aim of this study was to clinically characterize childhood cutaneous cGVHD. A retrospective study of children treated with HSCT at 2 tertiary medical centres in Israel between 2011 and 2014 was performed. A total of 112 children were included. Cutaneous cGVHD developed in 18% of subjects. Risk factors were older age, HSCT from peripheral blood and acute lymphoblastic leukaemia. The eruption was lichenoid in 90% of subjects, of whom one-third progressed to sclerosis...
October 23, 2017: Acta Dermato-venereologica
https://www.readbyqxmd.com/read/29053779/cell-based-therapeutic-strategies-for-multiple-sclerosis
#16
REVIEW
Neil J Scolding, Marcelo Pasquini, Stephen C Reingold, Jeffrey A Cohen
The availability of multiple disease-modifying medications with regulatory approval to treat multiple sclerosis illustrates the substantial progress made in therapy of the disease. However, all are only partially effective in preventing inflammatory tissue damage in the central nervous system and none directly promotes repair. Cell-based therapies, including immunoablation followed by autologous haematopoietic stem cell transplantation, mesenchymal and related stem cell transplantation, pharmacologic manipulation of endogenous stem cells to enhance their reparative capabilities, and transplantation of oligodendrocyte progenitor cells, have generated substantial interest as novel therapeutic strategies for immune modulation, neuroprotection, or repair of the damaged central nervous system in multiple sclerosis...
November 1, 2017: Brain: a Journal of Neurology
https://www.readbyqxmd.com/read/29048109/evaluation-of-a-disease-risk-index-for-adult-patients-undergoing-umbilical-cord-blood-transplantation-for-haematological-malignancies
#17
Annalisa Paviglianiti, Annalisa Ruggeri, Fernanda Volt, Guillermo Sanz, Noel Milpied, Sabine Furst, Albert Esquirol, William Arcese, Alessandra Picardi, Christelle Ferra, Norbert Ifrah, Jean H Bourhis, Kavita Raj, Peter A von dem Borne, Simona Sica, Anne-Lise Menard, Adrian Bloor, Chantal Kenzey, Eliane Gluckman, Vanderson Rocha
A disease risk index (DRI) has been defined for stratifying heterogeneous cohorts of patients undergoing allogeneic haematopoietic stem cell transplantation (HSCT). This index defines 4 distinct groups with different outcomes, dividing patients by disease type and status and considering cytogenetics for acute myeloid leukaemia and myelodysplastic syndromes (MDS). Recently, the DRI has been refined to include rare diseases and improve MDS stratification by blast percentage and response to prior therapy. Previous reports on DRI include only a small number of UCBT recipients...
October 19, 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/29048102/high-interpatient-variability-of-treosulfan-exposure-is-associated-with-early-toxicity-in-paediatric-hsct-a-prospective-multicentre-study
#18
M Y Eileen C van der Stoep, Alice Bertaina, Marloes H Ten Brink, Robbert G Bredius, Frans J Smiers, Dominique C M Wanders, Dirk Jan A R Moes, Franco Locatelli, Henk-Jan Guchelaar, Juliëtte Zwaveling, Arjan C Lankester
Treosulfan-based conditioning is increasingly employed in paediatric haematopoietic stem cell transplantation (HSCT). Data on treosulfan pharmacokinetics in children are scarce, and the relationship between treosulfan exposure, toxicity and clinical outcome is unresolved. In this multicentre prospective observational study, we studied treosulfan pharmacokinetics and the drug's relationship with regimen-related toxicity and early clinical outcome in 77 paediatric patients. Treosulfan dose was 30 g/m(2) , administered over 3 consecutive days in infants <1 year old (n = 12) and 42 g/m(2) in children ≥1 year old (n = 65)...
October 19, 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/29047185/pluripotent-stem-cell-derived-haematopoietic-progenitors-are-unable-to-downregulate-key-emt-associated-mirnas
#19
Ellie Meader, Tomas Barta, Dario Melguzo-Sanchis, Katarzyna Tilgner, David Montaner, Ashraf A El-Harouni, Lyle Armstrong, Majlinda Lako
Haematopoietic stem cells derived from pluripotent stem cells could be used as an alternative to bone marrow transplants. Deriving these has been a long-term goal for researchers. However, the success of these efforts has been limited, with the cells produced able to engraft in the bone marrow of recipient animals only in very low numbers. There is evidence that defects in the migratory and homing capacity of the cells are due to mis-regulation of miRNA expression, and are responsible for their failure to engraft...
October 19, 2017: Stem Cells
https://www.readbyqxmd.com/read/29033080/reconstitution-of-th17-tc17-and-treg-cells-after-paediatric-haematopoietic-stem-cell-transplantation-impact-of-interleukin-7
#20
Katrine Kielsen, Lars P Ryder, David Lennox-Hvenekilde, Monika Gad, Claus H Nielsen, Carsten Heilmann, Marianne Ifversen, Anders Elm Pedersen, Klaus Müller
Successful reconstitution of T lymphocytes after allogeneic haematopoietic stem cell transplantation (HSCT) is needed to establish the graft-versus-leukaemia effect and an effective anti-microbial defense, but the ratio between functionally different T-cell subsets needs to be balanced to avoid graft-versus-host disease (GVHD). IL-7 is essential for T-cell generation in the thymus and peripheral T-cell homeostasis. High IL-7 levels have been associated with impaired T-cell reconstitution, increased risk of acute GVHD and treatment-related mortality, but the underlying cellular mechanisms behind these associations have not been investigated previously...
October 9, 2017: Immunobiology
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