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Stem cell transplantation

Deepthi Alapati, Edward E Morrisey
While our understanding of the genetics and pathology of congenital lung diseases such as surfactant protein deficiency, cystic fibrosis and alpha 1 antitrypsin deficiency is extensive, treatment options are lacking. Since the lung is a barrier organ in direct communication with the external environment, targeted delivery of gene corrective technologies to the respiratory system via intra-tracheal or intranasal routes is an attractive option for therapy. CRISPR/Cas9 gene editing technology is a promising approach to repair or inactivate disease causing mutations...
October 25, 2016: American Journal of Respiratory Cell and Molecular Biology
Darshana Kadekar, Sonal Rangole, Vaijayanti Kale, Lalita Limaye
BACKGROUND: The limited cell dose in umbilical cord blood (UCB) necessitates ex vivo expansion of UCB. Further, the effective cryopreservation of these expanded cells is important in widening their use in the clinics. During cryopreservation, cells experience oxidative stress due to the generation of reactive oxygen species (ROS). Conditioned medium from mesenchymal stem cells (MSCs-CM) has been shown to alleviate the oxidative stress during wound healing, Alzheimer's disease and ischemic disease...
2016: PloS One
P Huezo-Diaz Curtis, C R S Uppugunduri, J Muthukumaran, M A Rezgui, C Peters, P Bader, M Duval, H Bittencourt, Maja Krajinovic, Marc Ansari
Sinusoidal obstruction syndrome (SOS) is a severe complication of hematopoietic stem cell transplantation (HSCT) that can be fatal, often attributed to the conditioning regimen prior to HSCT. We evaluated the association of SOS risk with gene variants in cystathionase (CTH), an enzyme involved in glutathione synthesis, in 76 children receiving intravenous busulfan (Bu) before HSCT. Our results indicated an association with CTHc.1364 G>T (ORTT=10.6, 95% confidence interval (CI)=2.16, 51.54) and SOS risk, which was sex dependent (female patients, ORTT=21...
October 25, 2016: Pharmacogenomics Journal
Stefania Oliva, Manuela Gambella, Milena Gilestro, Vittorio Emanuele Muccio, Francesca Gay, Daniela Drandi, Simone Ferrero, Roberto Passera, Chiara Pautasso, Annalisa Bernardini, Mariella Genuardi, Francesca Patriarca, Elona Saraci, Maria Teresa Petrucci, Norbert Pescosta, Anna Marina Liberati, Tommaso Caravita, Concetta Conticello, Alberto Rocci, Pellegrino Musto, Mario Boccadoro, Antonio Palumbo, Paola Omedè
We analyzed 50 patients who achieved at least a very good partial response in the RV-MM-EMN-441 study. Patients received consolidation with autologous stem-cell transplantation (ASCT) or cyclophosphamide-lenalidomide-dexamethasone (CRD), followed by Lenalidomide-based maintenance. We assessed minimal residual disease (MRD) by multi-parameter flow cytometry (MFC) and allelic-specific oligonucleotide real-time quantitative polymerase chain reaction (ASO-RQ-PCR) after consolidation, after 3 and 6 courses of maintenance, and thereafter every 6 months until progression...
October 13, 2016: Oncotarget
David R Raleigh, Bryan Tomlin, Benedict Del Buono, Erika Roddy, Katherine Sear, Lennox Byer, Erin Felton, Anu Banerjee, Joseph Torkildson, David Samuel, Biljana Horn, Steve E Braunstein, Daphne A Haas-Kogan, Sabine Mueller
Pediatric embryonal brain tumor patients treated with craniospinal irradiation (CSI) are at risk for adverse effects, with greater severity in younger patients. Here we compare outcomes of CSI vs. high-dose chemotherapy (HD), stem cell transplant (SCT) and delayed CSI in newly diagnosed patients. Two hundred one consecutive patients treated for medulloblastoma (72 %), supratentorial primitive neuroectodermal tumor (sPNET; 18 %) or pineoblastoma (10 %) at two institutions between 1988 and 2014 were retrospectively identified...
October 24, 2016: Journal of Neuro-oncology
Maximilian Fleischmann, Ulf Schnetzke, Karin G Schrenk, Volker Schmidt, Herbert G Sayer, Inken Hilgendorf, Andreas Hochhaus, Sebastian Scholl
BACKGROUND: Activating mutations of the receptor tyrosine kinase FLT3 (fms-related tyrosine kinase 3) reflect the most frequent molecular aberration in acute myeloid leukemia (AML). In particular, FLT3 internal tandem duplications (FLT3-ITD) are characterized by an unfavorable prognosis and allogeneic stem cell transplantation (allogeneic SCT) in first complete remission is recommended. In case of imminent or frank relapse following allogeneic SCT, treatment with FLT3 tyrosine kinase inhibitors (TKI) constitutes a promising clinical approach to induce hematologic remission without conventional chemotherapy...
October 24, 2016: Journal of Cancer Research and Clinical Oncology
Takeru Asano, Ken-Ichi Matsuoka, Satoshi Iyama, Kazuteru Ohashi, Yoshihiro Inamoto, Chikako Ohwada, Makoto Murata, Atsushi Satake, Chikamasa Yoshida, Koichi Nakase, Yasuo Mori, Mitsune Yamamoto
Chronic graft versus host disease (cGVHD) remains a major problem for long survivors after allogeneic hematopoietic stem cell transplantation (HSCT). Currently, corticosteroid therapy is effective for cGVHD as the first line therapy. However, prolonged therapy with corticosteroids causes various severe adverse events. To develop the new therapeutic strategy of cGVHD, we have launched a multicenter phase I/IIa clinical trial of low dose subcutaneous interleukin-2 (IL-2) for treatment of steroid refractory cGVHD, which is constituted of 2 sequential phases (induction phase and maitanance phase)...
October 2016: Acta Medica Okayama
W Li, L Huang, J Zeng, W Lin, K Li, J Sun, W Huang, J Chen, G Wang, Q Ke, J Duan, X Lai, R Chen, M Liu, Y Liu, T Wang, X Yang, Y Chen, H Xia, A P Xiang
The enteric nervous system (ENS) is recognized as a second brain because of its complexity and its largely autonomic control of bowel function. Recent progress in studying the interactions between the ENS and the central nervous system (CNS) has implicated alterations of the gut/brain axis as a possible mechanism in the pathophysiology of autism spectrum disorders (ASDs), Parkinson's disease (PD) and other human CNS disorders, whereas the underlying mechanisms are largely unknown because of the lack of good model systems...
October 25, 2016: Molecular Psychiatry
Yngvar Fløisand, Knut E A Lundin, Vladimir Lazarevic, Jørn Dehli Kristiansen, Liv T N Osnes, Geir E Tjønnfjord, Henrik Mikael Reims, Tobias Gedde-Dahl
Steroid refractory acute graft-versus-host-disease of the gut is a serious complication after allogeneic stem cell transplantation associated with high mortality. Treatment options are limited and not predictably effective. We describe the treatment of steroid refractory acute graft-versus-host-disease with vedolizumab, an antibody directed against integrin α4β7, in six patients. All patients responded, and four out of six patients are alive with a median follow-up of 10 months.
October 21, 2016: Biology of Blood and Marrow Transplantation
Ioannis Politikos, Haesook Kim, Theodoros Karantanos, Julia Brown, Sean McDonough, Lequn Li, Corey Cutler, Joseph H Antin, Karen K Ballen, Jerome Ritz, Vassiliki A Boussiotis
Umbilical cord blood (UCB) is a valuable graft source for allogeneic hematopoietic stem cell transplantation (HSCT) in patients who lack adult donors. UCB transplantation (UCBT) in adults results in delayed immune reconstitution leading to high infection-related morbidity and mortality. Angiogenic factors and markers of endothelial dysfunction have biologic and prognostic significance in conventional HSCT, but their role in UCBT has not been investigated. Furthermore, the interplay between angiogenesis and immune reconstitution has not been studied...
October 21, 2016: Biology of Blood and Marrow Transplantation
Annalisa Ruggeri, Fernanda Volt, Franco Locatelli, Gerard Michel, Cristina Diaz de Heredia, Manuel Abecasis, Marco Zecca, Ajay Vora, Karima Yakouben, Tracey A O'Brien, Stefano Giardino, Jacqueline Cornish, Vanderson Rocha, Christina Peters, Peter Bader, Eliane Gluckman, Jean Hugues Dalle
Infant acute leukemia still has poor prognosis and allogeneic hematopoietic stem cell transplantation is indicated in selected patients. Umbilical cord blood (UCB) is an attractive cell source for this population due to the low risk of chronic graft-versus-host disease (GVHD), the strong graft-versus-leukemia (GVL) effect and prompt donor availability. This is a retrospective, registry-based study reporting umbilical cord blood transplantation (UCBT) outcomes in 252 children with ALL (n=157) or AML (n=95) diagnosed before 1 year of age who received a single-unit UCBT after myeloablative conditioning between 1996 and 2012 in EBMT centers...
October 21, 2016: Biology of Blood and Marrow Transplantation
Takaaki Konuma, Yasushi Miyazaki, Naoyuki Uchida, Kazuteru Ohashi, Tadakazu Kondo, Hirohisa Nakamae, Satoshi Takahashi, Takehiko Mori, Yukiyasu Ozawa, Chiaki Kato, Koji Iwato, Takahiro Fukuda, Tatsuo Ichinohe, Yoshiko Atsuta, Ken Ishiyama
Trisomy 8 (+8) is one of the most common cytogenetic abnormalities in adult patients with myelodysplastic syndrome (MDS). However, the outcome of allogeneic hematopoietic stem cell transplantation (HSCT) in adult patients with MDS harboring +8 remains unclear. To evaluate the outcome and prognostic factors in patients with MDS harboring +8 as the sole cytogenetic abnormality or in association with other abnormalities, we retrospectively analyzed the Japanese registration data of 381 adult patients with MDS harboring +8 treated with allogeneic HSCT between 1990 and 2013...
October 21, 2016: Biology of Blood and Marrow Transplantation
Tatsanee Phermthai, Kittima Tungprasertpol, Suphakde Julavijitphong, Puttachart Pokathikorn, Sasiprapa Thongbopit, Suparat Wichitwiengrat
Transplantation of mesenchymal stem cells (MSC) can effectively repair endometrial deficiencies, including infertile patients with a problem of inadequate endometrium thickness. Although, MSC derived from different organ sources have a similarity of MSC specific characteristics, endometrial stem cells (EMSC) are temporally regulated throughout the menstrual cycle in a micro-environmental niche found only in endometrial tissue. Given the micro-environment niche, developing treatments for endometrial disorders with EMSC should be a top priority...
October 21, 2016: Reproductive Biology
Eva de Berranger, Sandie Balcaen, Malika Ainaoui, Caroline Bompoint, Cécile Borel, Nathalie Chevallier, Natacha de Bentzmann, Virginie Denis, Karine Kerautret, Sandrine Godin, Marie-Laure Labat, Maïna Letort-Bertrand, Sophie Porcheron, Leonardo Magro, Ibrahim Yakoub-Agha, Myriam Guiraud
In an attempt to harmonize clinical practices among French hematopoietic stem cell transplantation centers, the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) held its sixth annual workshop series in September 2015 in Lille. This event brought together practitioners from across the country. Our article discusses the updates and modifications for the 2016 version of the national patient follow-up care logbook.
October 21, 2016: Bulletin du Cancer
Hanaa Alkharobi, Aishah Alhodhodi, Yousef Hawsawi, Hasanain Alkafaji, Deirdre Devine, Reem El-Gendy, James Beattie
Human dental pulp cells (DPCs), which are known to contain a subset of stem cells capable of reforming a dentin and pulp-like complex upon in vivo transplantation, were isolated from third molars of three healthy donors and differentiated to a matrix mineralisation phenotype using by culture in dexamethasone and l-ascorbic acid. qRT-PCR analysis of insulin-like growth factor ( IGF) axis gene expression indicated that all genes, except insulin-like growth factor 1 (IGF1) and insulin-like growth factor binding protein-1 ( IGFBP-1), were expressed in DPCs...
September 24, 2016: Stem Cell Research
Aliyah Baluch, Yanina Pasikhova, Matthew Snyder
Nontuberculous mycobacterial infections can often occur in individuals with adequate immune function. Such infections typically have cutaneous involvement and are caused by rapidly growing mycobacterium. Other nontuberculous mycobacteria species, like Mycobacterium haemophilum, almost always present as opportunistic infections occurring in severely immunocompromised hosts. Here, we present a complicated and protracted course of diagnosing M. haemophilum lower extremity cutaneous infection in a matched-unrelated donor stem cell transplant recipient...
October 24, 2016: Transplant Infectious Disease: An Official Journal of the Transplantation Society
Z Li, Y Wang, J Wang, J Zhang, Z Wang
No abstract text is available yet for this article.
October 24, 2016: Bone Marrow Transplantation
N Ben Abdejlil, D Belloumi, M Mâammar, R El Fatimi, L Torjman, A Lakhal, F Jenhani, S Hmida, T Ben Othman, S Ladeb
This study compared retrospectively the effectiveness, toxicity and hematopoietic recovery after autologous peripheral blood stem cell transplantation (ASCT) of two consecutive peripheral blood stem cell mobilization regimens in newly diagnosed MM patients. Patients in group 1 (n=178) were treated with 4 g/m(2) of cyclophosphamide (CY) plus G-CSF (5 μg/kg/day). Patients in group 2 (n=117) with 750 mg/m(2) of VP16 plus G-CSF (10 μg/kg/day). Optimal mobilization, defined by a target number of 8 × 10(6) CD34+ cells/kg collected, was achieved in 62...
October 24, 2016: Bone Marrow Transplantation
J Gauthier, L Holmberg, D Wu, W Bensinger, A K Gopal, O Press, D Maloney, D J Green, B G Till, D Byelykh, A Shustov
No abstract text is available yet for this article.
October 24, 2016: Bone Marrow Transplantation
Philip Egan, Stephen Drain, Caroline Conway, Anthony J Bjourson, H Denis Alexander
Plasma cell myeloma is a clinically heterogeneous malignancy accounting for approximately one to 2% of newly diagnosed cases of cancer worldwide. Treatment options, in addition to long-established cytotoxic drugs, include autologous stem cell transplant, immune modulators, proteasome inhibitors and monoclonal antibodies, plus further targeted therapies currently in clinical trials. Whilst treatment decisions are mostly based on a patient's age, fitness, including the presence of co-morbidities, and tumour burden, significant scope exists for better risk stratification, sub-classification of disease, and predictors of response to specific therapies...
October 21, 2016: International Journal of Molecular Sciences
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