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https://www.readbyqxmd.com/read/28331055/reticular-dysgenesis-international-survey-on-clinical-presentation-transplantation-and-outcome
#1
Manfred Hoenig, Chantal Lagresle-Peyrou, Ulrich Pannicke, Luigi D Notarangelo, Fulvio Porta, Andrew R Gennery, Mary Slatter, Morton J Cowan, Polina Stepensky, Hamoud Al-Mousa, Daifulah Al-Zahrani, Sung-Yun Pai, Waleed Al Herz, Hubert B Gaspar, Paul Veys, Koichi Oshima, Kohsuke Imai, Hiromasa Yabe, Lenora M Noroski, Nico M Wulffraat, Karl-Walter Sykora, Pere Soler-Palacin, Hideki Muramatsu, Mariam Al Hilali, Despina Moshous, Klaus-Michael Debatin, Catharina Schuetz, Eva-Maria Jacobsen, Ansgar S Schulz, Klaus Schwarz, Alain Fischer, Wilhelm Friedrich, Marina Cavazzana
Reticular Dysgenesis (RD) is a rare congenital disorder defined clinically by the combination of severe combined immunodeficiency (SCID), agranulocytosis and sensorineural deafness. Mutations in the gene encoding Adenylate Kinase 2 (AK2) were identified to cause the disorder. Hematopoietic stem cell transplantation (HSCT) is the only option to cure this otherwise fatal disease. Retrospective data on clinical presentation, genetics and outcome of HSCT were collected from centers in Europe, Asia and North America for a total of 32 patients born between 1982 and 2011...
March 22, 2017: Blood
https://www.readbyqxmd.com/read/28330501/intraarterial-transplantation-of-human-umbilical-cord-blood-mononuclear-cells-in-hyperacute-stroke-improves-vascular-function
#2
Lei Huang, Yichu Liu, Jianfei Lu, Bianca Cerqueira, Vivek Misra, Timothy Q Duong
BACKGROUND: Human umbilical cord blood (hUCB) cell therapy is a promising treatment for ischemic stroke. The effects of hyperacute stem cell transplantation on cerebrovascular function in ischemic stroke are, however, not well understood. This study evaluated the effects of hyperacute intraarterial transplantation of hUCB mononuclear cells (MNCs) on cerebrovascular function in stroke rats using serial magnetic resonance imaging (MRI). METHODS: HUCB MNCs or vehicle were administered to stroke rats via the internal carotid artery immediately after reperfusion at 60 min following ischemia onset...
March 22, 2017: Stem Cell Research & Therapy
https://www.readbyqxmd.com/read/28321921/insights-into-cell-free-therapeutic-approach-role-of-stem-cell-soup-ernatant
#3
REVIEW
Shalini Raik, Ajay Kumar, Shalmoli Bhattacharyya
Current advances in medicine have revolutionized the field of regenerative medicine dramatically with newly evolved therapies for repair or replacement of degenerating or injured tissues. Stem cells can be harvested from different sources for clinical therapeutics which include fetal tissues, umbilical cord blood, embryos and adult tissues. Stem cells can be isolated and differentiated into desired lineages for tissue regeneration and cell replacement therapy. However, several loopholes need to be addressed properly before this can be extended for large scale therapeutic application...
March 21, 2017: Biotechnology and Applied Biochemistry
https://www.readbyqxmd.com/read/28321092/refractory-double-hit-lymphoma-leukemia-in-childhood-mimicking-b-precursor-acute-lymphoblastic-leukemia-at-initial-presentation
#4
Suguru Uemura, Daiichiro Hasegawa, Takehito Yokoi, Nanako Nino, Teppei Tahara, Akihiro Tamura, Atsuro Saito, Aiko Kozaki, Kenji Kishimoto, Toshiaki Ishida, Keiichiro Kawasaki, Nobuyuki Yamamoto, Takeshi Mori, Noriyuki Nishimura, Yoshiyuki Kosaka
A 10-year-old girl was referred to our hospital with left preauricular adenopathy and gingival swelling. She was diagnosed with B-cell precursor acute lymphoblastic leukemia (BCP-ALL) based on being positive for expressions of CD10, CD19, TdT and HLA-DR. She showed no CD20 expression at the time of diagnosis. Based on the initial diagnosis of BCP-ALL, induction chemotherapy for BCP-ALL was initiated. However, the blasts did not disappear from her peripheral blood. Bone marrow examination on day 33 identified 81...
2017: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://www.readbyqxmd.com/read/28320105/anti-fibrotic-potential-of-human-umbilical-cord-mononuclear-cells-and-mouse-bone-marrow-cells-in-ccl4-induced-liver-fibrosis-in-mice
#5
Nageh Ahmed Elmahdy, Samia Salem Sokar, Mohamed Labib Salem, Naglaa Ibrahim Sarhan, Sherin Hamed Abou-Elela
Liver fibrosis is the consequence of hepatocyte injury that leads to the activation of hepatic stellate cells (HSC). The treatment of choice is Liver transplantation; however, it has many problems such as surgery-related complications, immunological rejection and high costs associated with the procedure. Stem cell-based therapy would be a potential alternative, so the aim of this study is to investigate the therapeutic potential of human umbilical cord mononuclear cells (MNC) and mouse bone marrow cells (BMC) against carbon tetrachloride (CCl4) induced liver fibrosis in mice and compare it with that of silymarin...
March 18, 2017: Biomedicine & Pharmacotherapy, Biomédecine & Pharmacothérapie
https://www.readbyqxmd.com/read/28319446/good-laboratory-practice-preclinical-safety-studies-for-gsk2696273-mlv-vector-based-ex-vivo-gene-therapy-for-adenosine-deaminase-deficiency-severe-combined-immunodeficiency-in-nsg-mice
#6
Nicola Carriglio, Jan Klapwijk, Raisa Jofra Hernandez, Michela Vezzoli, Franck Chanut, Rhiannon Lowe, Draghici Elena, Melanie Nord, Paola Albertini, Patrizia Cristofori, Jane Richards, Hazel Staton, Jonathan Appleby, Alessandro Aiuti, Aisha V Sauer
GSK2696273 (autologous CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase [ADA] enzyme) is a gamma-retroviral ex vivo gene therapy of bone marrow-derived CD34+ cells for the treatment of adenosine deaminase deficiency severe combined immunodeficiency (ADA-SCID). ADA-SCID is a severe monogenic disease characterized by immunologic and nonimmunologic symptoms. Bone-marrow transplant from a matched related donor is the treatment of choice, but it is available for only a small proportion of patients...
March 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28302713/family-cord-blood-banking-for-sickle-cell-disease-a-twenty-year-experience-in-two-dedicated-public-cord-blood-banks
#7
Hanadi Rafii, Françoise Bernaudin, Helene Rouard, Valérie Vanneaux, Annalisa Ruggeri, Marina Cavazzana, Valerie Gauthereau, Aurélie Stanislas, Malika Benkerrou, Mariane De Montalembert, Christele Ferry, Robert Girot, Cecile Arnaud, Annie Kamdem, Joelle Gour, Claudine Touboul, Audrey Cras, Mathieu Kuentz, Claire Rieux, Fernanda Volt, Barbara Cappelli, Karina T Maio, Annalisa Paviglianiti, Chantal Kenzey, Jerome Larghero, Eliane Gluckman
Efforts to implement family cord blood banking have been developed in the past decades for siblings requiring stem cell transplantation for conditions such as sickle cell disease. However, public banks are faced with challenging decisions about the units to be stored, discarded, or used for other endeavors. We report here 20 years of experience in family cord blood banking for sickle cell disease in two dedicated public banks. Participants were pregnant women who had previous child diagnosed with homozygous sickle cell disease...
March 16, 2017: Haematologica
https://www.readbyqxmd.com/read/28297572/safety-and-efficacy-of-megakaryocytes-induced-from-hematopoietic-stem-cells-in-murine-and-nonhuman-primate-models
#8
Xin Guan, Meng Qin, Yu Zhang, Yanan Wang, Bin Shen, Zhihua Ren, Xinxin Ding, Wei Dai, Yongping Jiang
Because of a lack of platelet supply and a U.S. Food and Drug Administration-approved platelet growth factor, megakaryocytes have emerged as an effective substitute for alleviating thrombocytopenia. Here, we report the development of an efficient two-stage culture system that is free of stroma, animal components, and genetic manipulations for the production of functional megakaryocytes from hematopoietic stem cells. Safety and functional studies were performed in murine and nonhuman primate models. One human cryopreserved cord blood CD34(+) cell could be induced ex vivo to produce up to 1...
March 2017: Stem Cells Translational Medicine
https://www.readbyqxmd.com/read/28295190/phase-i-study-of-cord-blood-derived-natural-killer-cells-combined-with-autologous-stem-cell-transplantation-in-multiple-myeloma
#9
Nina Shah, Li Li, Jessica McCarty, Indreshpal Kaur, Eric Yvon, Hila Shaim, Muharrem Muftuoglu, Enli Liu, Robert Z Orlowski, Laurence Cooper, Dean Lee, Simrit Parmar, Kai Cao, Catherine Sobieiski, Rima Saliba, Chitra Hosing, Sairah Ahmed, Yago Nieto, Qaiser Bashir, Krina Patel, Catherine Bollard, Muzaffar Qazilabsh, Richard Champlin, Katy Rezvani, Elizabeth J Shpall
Multiple myeloma (MM) is a disease with known immune dysregulation. Natural killer (NK) cells have shown preclinical activity in MM. We conducted a first-in-human study of umbilical cord blood-derived (CB) NK cells for MM patients undergoing high dose chemotherapy and autologous haematopoietic stem cell transplantation (auto-HCT). Patients received lenalidomide (10 mg) on days -8 to -2, melphalan 200 mg/m(2) on day -7, CB-NK cells on day -5 and auto-HCT on day 0. Twelve patients were enrolled, three on each of four CB-NK cell dose levels: 5 × 10(6) , 1 × 10(7) , 5 × 10(7) and 1 × 10(8) CB-NK cells/kg...
March 14, 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/28293049/microrna-modulation-during-the-in-vitro-culture-of-hematopoietic-stem-cells-prior-to-transplantation
#10
Saeid Shahrabi, Saeid Kaviani, Masoud Soleimani, Ali Akbar Pourfathollah, Behnaz Bakhshandeh, Saeideh Hajizamani, Najmaldin Saki
BACKGROUND: Human umbilical cord blood (HUCB) is an acceptable and readily accessible source of stem cells. There is an ongoing interest in cord blood stem cell therapies; however, little is known about the possible unfavorable effects of laboratory modifications on the isolated HUCB cells. The involvement of miRNAs in several biological processes has been shown. The aim of this study was to evaluate the possible changes in miRNA expression profiles in CD133(+) hematopoietic cells after in vitro culture...
January 2017: Iranian Journal of Medical Sciences
https://www.readbyqxmd.com/read/28292847/human-umbilical-cord-blood-borne-fibroblasts-contain-marrow-niche-precursors-that-form-a-bone-marrow-organoid-in-vivo
#11
Alice Pievani, Benedetto Sacchetti, Alessandro Corsi, Benedetta Rambaldi, Samantha Donsante, Valeria Scagliotti, Patrizia Vergani, Cristina Remoli, Andrea Biondi, Pamela G Robey, Mara Riminucci, Marta Serafini
Human umbilical cord blood (CB) has attracted much attention as a reservoir for functional hematopoietic stem and progenitor cells, and, recently, as a source of blood-borne fibroblasts (CB-BFs). Previously, we demonstrated that bone marrow stromal cell (BMSC) and CB-BF pellet cultures make cartilage in vitro Furthermore, upon in vivo transplantation, BMSC pellets remodelled into miniature bone/marrow organoids. Using this in vivo model, we asked whether CB-BF populations that express characteristics of the hematopoietic stem cell (HSC) niche contain precursors that reform the niche...
March 15, 2017: Development
https://www.readbyqxmd.com/read/28288952/outcomes-after-umbilical-cord-blood-transplantation-for-myelodysplastic-syndromes
#12
Aaron T Gerds, Kwang Woo Ahn, Zhen-Huan Hu, Hisham Abdel-Azim, Gorgun Akpek, Mahmoud Aljurf, Karen K Ballen, Amer Beitinjaneh, Ulrike Bacher, Jean-Yves Cahn, Saurabh Chhabra, Corey Cutler, Andrew Daly, Zachariah DeFilipp, Robert Peter Gale, Usama Gergis, Michael R Grunwald, Gregory A Hale, Betty Ky Hamilton, Madan Jagasia, Rammurti T Kamble, Tamila Kindwall-Keller, Taiga Nishihori, Richard F Olsson, Muthalagu Ramanathan, Ayman A Saad, Melhem Solh, Celalettin Ustun, David Valcárcel, Erica Warlick, Baldeep M Wirk, Matt Kalaycio, Edwin Alyea, Uday Popat, Ronald Sobecks, Wael Saber
For patients with hematologic malignancies undergoing allogeneic hematopoietic cell transplantation, umbilical cord blood transplantation (UCBT) has become an acceptable alternative donor source in the absence of a matched sibling or unrelated donor. However, there have been few published series dedicated solely to describing the outcomes of adult patients with myelodysplastic syndrome (MDS) who have undergone UCBT. From 2004 to 2013, 176 adult MDS patients underwent UCBT as reported to the Center for International Blood and Marrow Transplant Research...
March 10, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28281429/single-centre-experience-of-allogeneic-haemopoietic-stem-cell-transplant-in-paediatric-patients-in-cape-town-south-africa
#13
A Van Eyssen, N Novitsky, P De Wit, T Schlaphoff, V Thomas, D Pillay, M Hendricks, A Davidson
BACKGROUND: Allogeneic haemopoietic stem cell transplant (Allo-HSCT) is a specialised and costly intervention, associated with significant morbidity and mortality. It is used to treat a broad range of paediatric conditions. South Africa (SA) is an upper middle-income country with limitations on healthcare spending. The role of paediatric Allo-HSCT in this setting is reviewed. OBJECTIVES: To review paediatric patients who underwent Allo-HSCT at the Groote Schuur Hospital/University of Cape Town Private Academic Hospital transplant unit in Cape Town, South Africa, and received post-transplant care at Red Cross War Memorial Children's Hospital, over the period January 2006 - December 2014 in respect of indications for the transplant, donor sources, conditioning regimens, treatment-related morbidity and overall survival (OS)...
February 27, 2017: South African Medical Journal, Suid-Afrikaanse Tydskrif Vir Geneeskunde
https://www.readbyqxmd.com/read/28280408/effect-of-subcutaneous-treatment-with-human-umbilical-cord-blood-derived-multipotent-stem-cells-on-peripheral-neuropathic-pain-in-rats
#14
Min Ju Lee, Tae Gyoon Yoon, Moonkyu Kang, Hyun Jeong Kim, Kyung Sun Kang
In this study, we aim to determine the in vivo effect of human umbilical cord blood-derived multipotent stem cells (hUCB-MSCs) on neuropathic pain, using three, principal peripheral neuropathic pain models. Four weeks after hUCB-MSC transplantation, we observed significant antinociceptive effect in hUCB-MSC-transplanted rats compared to that in the vehicle-treated control. Spinal cord cells positive for c-fos, CGRP, p-ERK, p-p 38, MMP-9 and MMP 2 were significantly decreased in only CCI model of hUCB-MSCs-grafted rats, while spinal cord cells positive for CGRP, p-ERK and MMP-2 significantly decreased in SNL model of hUCB-MSCs-grafted rats and spinal cord cells positive for CGRP and MMP-2 significantly decreased in SNI model of hUCB-MSCs-grafted rats, compared to the control 4 weeks or 8weeks after transplantation (p<0...
March 2017: Korean Journal of Physiology & Pharmacology
https://www.readbyqxmd.com/read/28279825/optimal-practices-in-unrelated-donor-cord-blood-transplantation-for-hematologic-malignancies
#15
REVIEW
Juliet N Barker, Joanne Kurtzberg, Karen Ballen, Michael Boo, Claudio Brunstein, Corey Cutler, Mitchell Horwitz, Filippo Milano, Amanda Olson, Stephen Spellman, John E Wagner, Colleen Delaney, Elizabeth Shpall
Unrelated donor cord blood (CB) transplantation (CBT) results in disease-free survival comparable to that of unrelated adult donor transplantation in patients with hematologic malignancies. Extension of allograft access to racial and ethnic minorities, rapid graft availability, flexibility of transplant date, and low risks of disabling chronic graft-versus-host disease (GVHD) and relapse are significant advantages of CBT, and multiple series have reported a low risk of late transplant-related mortality (TRM) post-transplant...
March 6, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28263918/prospective-evaluation-of-unrelated-donor-cord-blood-and-haploidentical-donor-access-reveals-graft-availability-varies-by-patient-ancestry-practical-implications-for-donor-selection
#16
Satyajit Kosuri, Tara Wolff, Sean M Devlin, Courtney Byam, Christopher M Mazis, Kristine Naputo, Eric Davis, Jennifer Paulson, Melissa Nhaissi, Deborah S Wells, Parastoo Dahi, Sergio A Giralt, Ann Jakubowski, Miguel-Angel Perales, Brian C Shaffer, Andromachi Scaradavou, Doris M Ponce, Juliet N Barker
The availability of cord blood (CB) and haploidentical (haplo) donors in all patient populations is not established. We have investigated the addition of haplo-CD34+ cells to CB grafts (haplo-CBT) to speed myeloid engraftment. Thus, we have prospectively assessed CB and haplo donor availability in adult patients without 8/8 HLA-allele matched unrelated donors (URDs). Analysis of 89 patients eligible for haplo-CBT revealed 4 distinct patient groups. Firstly, 6 patients (7% of total, 33% non-European) underwent CBT only as they had no suitable family members to type...
March 2, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28263313/glucocorticoid-hormone-induced-chromatin-remodeling-enhances-human-hematopoietic-stem-cell-homing-and-engraftment
#17
Bin Guo, Xinxin Huang, Scott Cooper, Hal E Broxmeyer
Efficient hematopoietic stem cell (HSC) homing is important for hematopoietic cell transplantation (HCT), especially when HSC numbers are limited, as in the use of cord blood (CB). In a screen of small-molecule compounds, we identified glucocorticoid (GC) hormone signaling as an activator of CXCR4 expression in human CB HSCs and hematopoietic progenitor cells (HPCs). Short-term GC pretreatment of human CB HSCs and HPCs promoted SDF-1-CXCR4-axis-mediated chemotaxis, homing, and long-term engraftment when these cells were transplanted into primary- and secondary-recipient NSG mice...
March 6, 2017: Nature Medicine
https://www.readbyqxmd.com/read/28255019/a-risk-factor-analysis-of-outcomes-after-unrelated-cord-blood-transplantation-for-children-with-wiskott-aldrich-syndrome
#18
Zhanna Shekhovtsova, Carmem Bonfim, Annalisa Ruggeri, Samantha Nichele, Kristin Page, Amal AlSeraihy, Francisco Barriga, José Sánchez de Toledo Codina, Paul Veys, Jaap Jan Boelens, Karin Mellgren, Henrique Bittencourt, Tracey O' Brien, Peter J Shaw, Alicja Chybicka, Fernanda Volt, Federica Giannotti, Eliane Gluckman, Joanne Kurtzberg, Andrew R Gennery, Vanderson Rocha
Wiskott-Aldrich syndrome is a severe X-linked recessive immune deficiency disorder. A scoring system of Wiskott-Aldrich syndrome severity (0.5-5) distinguishes 2 phenotypes: X-linked thrombocytopenia and classic Wiskott-Aldrich syndrome. Hematopoietic cell transplantation is curative for Wiskott-Aldrich syndrome, however the use of unrelated umbilical cord blood transplantation has seldom been described. We analyzed umbilical cord blood transplantation outcomes for 90 patients. Median age at umbilical cord blood transplantation was 1...
March 2, 2017: Haematologica
https://www.readbyqxmd.com/read/28238202/brain-abnormalities-in-fucosidosis-transplantation-or-supportive-therapy
#19
Minyan Jiang, Sha Liu, Hua Jiang, Yunting Lin, Yongxian Shao, Hao Hu, Xiaoyuan Zhao, Hongsheng Liu, Yonglan Huang, Li Liu
Fucosidosis is a rare lysosomal storage disease caused by α-fucosidase deficiency, which leads to progressive neurological deterioration and death. Hematopoietic stem cell transplantation is the best curative therapy if performed during the early stages of disease. We report two fucosidosis patients with brain abnormalities and the challenge faced in their management. The first patient received supportive therapy and the second one firstly underwent unrelated donor umbilical cord blood transplantation. After a period of follow-up, we found neurological symptoms were worsening day by day on patient1...
April 2017: Metabolic Brain Disease
https://www.readbyqxmd.com/read/28218755/changes-in-the-incidence-patterns-and-outcomes-of-graft-failure-following-hematopoietic-stem-cell-transplantation-for-hurler-syndrome
#20
S H Lum, W P Miller, S Jones, K Poulton, W Ogden, H Lee, A Logan, D Bonney, T C Lund, P J Orchard, R F Wynn
Hematopoietic stem cell transplantation (HSCT) is the standard of care in children with Hurler syndrome (HS) as it is the only therapy that can arrest disease progression. We examined the incidence, patterns and outcomes of graft failure in all HS children undergoing first HSCT at the Royal Manchester Children's Hospital or the University of Minnesota Children's Hospital from 1983 to 2016. Implementation of busulfan pharmacokinetic monitoring started in 2004 in both institutions. Two hundred and forty HS children were included in this analysis (historical era (pre-2004), n=131; current era (post 2004), n=109)...
February 20, 2017: Bone Marrow Transplantation
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