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Hematopoietic cell transplant

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https://www.readbyqxmd.com/read/28822832/combined-immunodeficiency-with-ebv-positive-b-cell-lymphoma-and-epidermodysplasia-verruciformis-due-to-a-novel-homozygous-mutation-in-rasgrp1
#1
Craig D Platt, Ari J Fried, Rodrigo Hoyos-Bachiloglu, G Naheed Usmani, Birgitta Schmidt, Jennifer Whangbo, Roberto Chiarle, Janet Chou, Raif S Geha
RASGRP1 is a guanine-nucleotide-exchange factor essential for MAP-kinase mediated signaling in lymphocytes. We report the second case of RASGRP1 deficiency in a patient with a homozygous nonsense mutation in the catalytic domain of the protein. The patient had epidermodysplasia verruciformis, suggesting a clinically important intrinsic T cell function defect. Like the previously described patient, our proband also presented with CD4(+) T cell lymphopenia, impaired T cell proliferation to mitogens and antigens, reduced NK cell function, and EBV-associated lymphoma...
August 16, 2017: Clinical Immunology: the Official Journal of the Clinical Immunology Society
https://www.readbyqxmd.com/read/28822830/chronic-kidney-disease-in-long-term-survivors-after-allogeneic-hematopoietic-stem-cell-transplantation-retrospective-analysis-at-a-single-institute
#2
Tomoyasu Jo, Yasuyuki Arai, Tadakazu Kondo, Toshiyuki Kitano, Masakatsu Hishizawa, Kouhei Yamashita, Akifumi Takaori-Kondo
The number of patients eligible for allogeneic stem cell transplantation (allo-HSCT) has increased because of improvements in transplantation procedures. Among long-term survivors of allo-HSCT, chronic kidney disease (CKD) is a major cause of morbidity. We retrospectively analyzed the clinical data of 106 consecutive patients with a median age of 43 (17-73) years who had undergone allo-HSCT at our institution between January 2001 and September 2009. Patients who died within 5 years after transplantation or had CKD at the time of transplantation were excluded in this study...
August 16, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28822119/five-questions-answered-a-review-of-autologous-hematopoietic-stem-cell-transplantation-for-the-treatment-of-multiple-sclerosis
#3
REVIEW
Harold L Atkins, Mark S Freedman
Multiple sclerosis (MS) is thought to be an autoimmune disease targeting the central nervous system leading to demyelination, and axonal and neuronal damage, resulting in progressive disability. More intensive therapies such as immunodepletion with hematopoietic stem-cell rescue are being used at a time prior to patients becoming irreversibly disabled. Over the last 15 years, there has been a shift away from using autologous hematopoietic stem-cell transplants (aHSCT) to treat patients with progressive MS, towards treating those with active inflammation and relapses...
August 18, 2017: Neurotherapeutics: the Journal of the American Society for Experimental NeuroTherapeutics
https://www.readbyqxmd.com/read/28822107/successful-treatment-and-fdg-pet-ct-monitoring-of-hhv-6-encephalitis-in-a-non-neutropenic-patient-case-report-and-literature-review
#4
Elda Righi, Alessia Carnelutti, Daniele Muser, Francesco Zaja, Elisa Lucchini, Federico Pea, Fernando Di Gregorio, Abass Alavi, Matteo Bassetti
Human herpesvirus (HHV)-6 reactivation is associated with severe forms of encephalitis among patients undergoing hematopoietic stem cell transplantation. Cases in non-neutropenic patients are uncommon. The efficacy of ganciclovir and other compounds that are used for the treatment of HHV-6 encephalitis remains suboptimal and linked to toxicity. Valganciclovir, the oral prodrug of ganciclovir, could be practical to treat outpatients, but it is not commonly used for severe cases. We report a case of HHV-6 encephalitis in a non-neutropenic patient successfully treated with valganciclovir and undergoing therapeutic drug monitoring in plasma and in the cerebrospinal fluid...
August 18, 2017: Journal of Neurovirology
https://www.readbyqxmd.com/read/28821477/tgf-%C3%AE-induced-intracellular-pai-1-is-responsible-for-retaining-hematopoietic-stem-cells-in-the-niche
#5
Takashi Yahata, Abd Aziz Ibrahim, Yukari Muguruma, Mesut Eren, Alexander M Shaffer, Nobuo Watanabe, Satoko Kaneko, Tetsuo Nakabayashi, Takashi Dan, Noriaki Hirayama, Douglas E Vaughan, Toshio Miyata, Kiyoshi Ando
Hematopoietic stem and progenitor cells (HSPCs) reside in the supportive stromal niche in bone marrow (BM); when needed, however, they are rapidly mobilized into the circulation, suggesting that HSPCs are intrinsically highly motile but are usually stayed in the niche. We questioned what determines the motility of HSPCs. Here we show that transforming growth factor (TGF)-β-induced intracellular plasminogen activator inhibitor (PAI)-1 activation is responsible for keeping HSPCs in the BM niche. We found that the expression of PAI-1, a downstream target of TGF-β-signaling, was selectively augmented in niche-residing HSPCs...
August 18, 2017: Blood
https://www.readbyqxmd.com/read/28821470/design-of-the-drepagreffe-trial-a-prospective-controlled-multicenter-study-evaluating-the-benefit-of-genoidentical-hematopoietic-stem-cell-transplantation-over-chronic-transfusion-in-sickle-cell-anemia-children-detected-to-be-at-risk-of-stroke-by-transcranial
#6
Sylvie Chevret, Suzanne Verlhac, Elisabeth Ducros-Miralles, Jean-Hugues Dalle, Regis Peffault de Latour, Mariane de Montalembert, Malika Benkerrou, Corinne Pondarré, Isabelle Thuret, Corinne Guitton, Emmanuelle Lesprit, Maryse Etienne-Julan, Gisèle Elana, Jean-Pierre Vannier, Patrick Lutz, Bénédicte Neven, Claire Galambrun, Catherine Paillard, Camille Runel, Charlotte Jubert, Cécile Arnaud, Annie Kamdem, Valentine Brousse, Florence Missud, Marie Petras, Lydia Doumdo-Divialle, Claire Berger, Françoise Fréard, Olivier Taieb, Elise Drain, Monique Elmaleh, Manuela Vasile, Yacine Khelif, Myriam Bernaudin, Philippe Chadebech, France Pirenne, Gérard Socié, Françoise Bernaudin
BACKGROUND: Children with sickle cell anemia (SCA) have an 11% risk of stroke by the age of 18. Chronic transfusion applied in patients detected to be at risk by transcranial Doppler allows a significant reduction of stroke risk. However, chronic transfusion exposes to several adverse events, including alloimmunization and iron overload, and is not curative. Hematopoietic stem cell transplantation allows termination of the transfusion program, but its benefit has not been demonstrated...
August 15, 2017: Contemporary Clinical Trials
https://www.readbyqxmd.com/read/28821454/low-dose-anti-thymocyte-globulin-for-graft-versus-host-disease-prophylaxis-in-matched-unrelated-allogeneic-hematopoietic-stem-cell-transplant
#7
A Bryant, R Mallick, L Huebsch, D Allan, H Atkins, G Anstee, M Sabloff, N Scrivens, D Maze, C Bredeson, N Kekre
Graft-versus-host disease (GVHD) is a leading cause of morbidity and mortality in allogeneic hematopoietic stem cell transplantation (alloHCT). Prophylactic in-vivo T-Cell depletion with antithymocyte globulin (ATG) has been associated with decreased GVHD rates many alloHCT settings. Despite decades of clinical study, optimal ATG dosing has not been established. Understanding that higher rates of GVHD are observed with matched unrelated (MUD) versus matched related donor (MRD) alloHCT, at our institution MUD alloHSCT recipients have historically had low dose thymoglobulin (total dose 2...
August 15, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28820507/hematopoietic-stem-cell-transplantation-recipient-and-caregiver-factors-affecting-length-of-stay-and-readmission
#8
Thiruppavai Sundaramurthi, Leslie Wehrlen, Erika Friedman, Sue Thomas, Margaret Bevans
PURPOSE/OBJECTIVES: To evaluate the contributions of patient and caregiver factors to length of stay (LOS) and 30-day readmission status for recipients of allogeneic hematopoietic stem cell transplantation (HSCT).
. DESIGN: Secondary data analysis from a phase 2 clinical trial.
. SETTING: National Institutes of Health Clinical Center in Bethesda, Maryland.
. SAMPLE: 68 dyads (N = 136) comprised of patients receiving HSCT and their caregivers...
September 1, 2017: Oncology Nursing Forum
https://www.readbyqxmd.com/read/28819653/targeting-cytokines-in-gvhd-therapy
#9
Sandeep Kumar, Hemn Mohammadpour, Xuefang Cao
Transplantation of donor-derived allogeneic hematopoietic cells causes increased survival in patients suffering from various blood cancers and other hematologic and immunologic diseases. However, this health benefit is limited to certain patients. One major complication is graft-versus-host disease (GVHD) that occurs when donor-derived immune cells recognize host cells/tissues as foreign and perpetrate subsequent destruction. Cytokines are a major class of effector molecules that are involved in GVHD pathogenesis...
2017: Journal of Immunology Research and Therapy
https://www.readbyqxmd.com/read/28819574/sequential-kinase-inhibition-idelalisib-ibrutinib-induces-clinical-remission-in-b-cell-prolymphocytic-leukemia-harboring-a-17p-deletion
#10
H Coelho, M Badior, T Melo
B-cell prolymphocytic leukemia (B-PLL) is a rare lymphoid neoplasm with an aggressive clinical course. Treatment strategies for B-PLL remain to be established, and, until recently, alemtuzumab was the only effective therapeutic option in patients harboring 17p deletions. Herein, we describe, for the first time, a case of B-cell prolymphocytic leukemia harboring a 17p deletion in a 48-year-old man that was successfully treated sequentially with idelalisib-rituximab/ibrutinib followed by allogeneic hematopoietic stem cell transplant (allo-HSCT)...
2017: Case Reports in Hematology
https://www.readbyqxmd.com/read/28818869/clinical-risks-and-healthcare-utilization-of-haematopoietic-cell-transplantation-for-sickle-cell-disease-in-the-u-s-using-merged-databases
#11
Staci D Arnold, Ruta Brazauskas, Naya He, Yimei Li, Richard Aplenc, Zhezhen Jin, Matt Hall, Yoshiko Atsuta, Jignesh Dalal, Theresa Hahn, Nandita Khera, Carmem Bonfim, Navneet S Majhail, Miguel Angel Diaz, Cesar O Freytes, William A Wood, Bipin M Savani, Rammurti T Kamble, Susan Parsons, Ibrahim Ahmed, Keith Sullivan, Sara Beattie, Christopher Dandoy, Reinhold Munker, Susana Marino, Menachem Bitan, Hisham Abdel-Azim, Mahmoud Aljurf, Richard F Olsson, Sarita Joshi, Dave Buchbinder, Michael J Eckrich, Shahrukh Hashmi, Hillard Lazarus, David I Marks, Amir Steinberg, Ayman Saad, Usama Gergis, Lakshmanan Krishnamurti, Allistair Abraham, Hemalatha G Rangarajan, Mark Walters, Joseph Lipscomb, Wael Saber, Prakash Satwani
Allogeneic hematopoietic cell transplant advances for sickle cell disease improve outcomes, but limited healthcare utilization analysis exists. We hypothesized that early transplant (age < 10 years) improves outcomes and decreases healthcare utilization compared to late. We performed a retrospective study of US children transplanted for sickle cell during 2000-2013 using two large databases. Univariate and Cox models estimated associations between demographics, sickle cell severity, and transplant-related variables on mortality and chronic graft versus-host-disease; while, Wilcoxon, Kruskal-Wallis, or linear trend tests estimated against healthcare utilization...
August 17, 2017: Haematologica
https://www.readbyqxmd.com/read/28817977/a-short-and-efficient-transduction-protocol-of-mouse-hematopoietic-stem-cells-with-lentiviral-vectors
#12
Maria Fernandez-Garcia, Cristina Mesa-Nuñez, Elena Almarza, Juan Bueren, Rosa Yañez
Transduction of hematopoietic stem and progenitor cells (HSPC) with therapeutic lentiviral vectors (LVs) constitutes a new therapeutic option for the treatment of different monogenic diseases affecting the lympho-hematopoietic system. The development of detailed preclinical studies of gene therapy in animal disease models constitutes an essential step in expanding the application of gene therapy in a wide variety of inherited and acquired diseases. Here we describe an efficient protocol to transduce HSPCs from wild-type and Fanconi anemia mice with either gene marking or therapeutic LVs...
August 17, 2017: Human Gene Therapy Methods
https://www.readbyqxmd.com/read/28817727/pilot-study-of-lithium-to-restore-intestinal-barrier-function-in-severe-graft-versus-host-disease
#13
Gideon Steinbach, David M Hockenbery, Gerwin Huls, Terry Furlong, David Myerson, Keith R Loeb, Jesse R Fann, Christina Castilla-Llorente, George B McDonald, Paul J Martin
Severe intestinal graft-vs-host disease (GVHD) after allogeneic hematopoietic cell transplantation (HCT) causes mucosal ulceration and induces innate and adaptive immune responses that amplify and perpetuate GVHD and the associated barrier dysfunction. Pharmacological agents to target mucosal barrier dysfunction in GVHD are needed. We hypothesized that induction of Wnt signaling by lithium, an inhibitor of glycogen synthase kinase (GSK3), would potentiate intestinal crypt proliferation and mucosal repair and that inhibition of GSK3 in inflammatory cells would attenuate the deregulated inflammatory response to mucosal injury...
2017: PloS One
https://www.readbyqxmd.com/read/28817385/novel-molecular-defects-associated-with-very-early-onset-inflammatory-bowel
#14
Sara Ciullini Mannurita, Eleonora Gambineri
PURPOSE OF REVIEW: Immune dysregulation disorders present with common clinical features of multiorgan autoimmunity. Gastrointestinal involvement is the hallmark of an impaired immune homeostasis. This review will give an overview on the novel phenotypes, highlighting the major points that will help to enable early diagnosis and treatment. RECENT FINDINGS: The rapid progress on DNA sequencing technologies have led to the identification of monogenic defects that adversely impact the control of immune homeostasis...
August 16, 2017: Current Opinion in Allergy and Clinical Immunology
https://www.readbyqxmd.com/read/28817182/centralized-patient-reported-outcome-data-collection-in-transplantation-is-feasible-and-clinically-meaningful
#15
Bronwen E Shaw, Ruta Brazauskas, Heather R Millard, Rachel Fonstad, Kathryn E Flynn, Amy Abernethy, Jenny Vogel, Charney Petroske, Deborah Mattila, Rebecca Drexler, Stephanie J Lee, Mary M Horowitz, J Douglas Rizzo
BACKGROUND: Allogeneic hematopoietic cell transplantation (HCT) cures many patients, but often with the risk of late effects and impaired quality of life. The value of quantifying patient-reported outcomes (PROs) is increasingly being recognized, but the routine collection of PROs is uncommon. This study evaluated the feasibility of prospective PRO collection by an outcome registry at multiple time points from unselected HCT patients undergoing transplantation at centers contributing clinical data to the Center for International Blood and Marrow Transplant Research (CIBMTR), and then it correlated the PRO data with clinical and demographic data...
August 17, 2017: Cancer
https://www.readbyqxmd.com/read/28816799/successful-extracorporeal-life-support-in-a-pediatric-hematopoietic-stem-cell-transplant-recipient-with-periengraftment-respiratory-failure
#16
Jenny Potratz, Martina Ahlmann, Claudia Rössig, Heymut Omran, Katja Masjosthusmann
The use of extracorporeal life support (ECLS) as ultimate salvage therapy for hematopoietic stem cell transplant recipients remains controversial among oncologists and critical care specialists. Prognosis is poor, particularly after allogeneic transplantation, and literature to guide clinical decision-making is scarce. Our report describes successful ECLS in a pediatric patient undergoing allogeneic hematopoietic stem cell transplantation, who developed acute respiratory failure during severe neutropenia, followed by immediate neutrophil engraftment...
August 14, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28816796/intrathecal-infusion-of-haploidentical-nondonor-lymphocytes-for-central-nervous-system-leukemic-relapse-after-haploidentical-hematopoietic-stem-cell-transplantation
#17
Azusa Mayumi, Akihisa Sawada, Aya Ioi, Kohei Higuchi, Mariko Shimizu, Maho Sato, Masahiro Yasui, Masami Inoue
Leukemic relapse in the central nervous system (CNS) after conventional treatment is associated with a poor prognosis. The effectiveness and safety of IV infusion of human leukocyte antigen (HLA)-mismatched lymphocytes for leukemia, and intrathecal (IT) infusion of HLA-mismatched lymphocytes for cerebrospinal fluid (CSF) dissemination of medulloblastoma have been reported. A 13-year-old girl (HLA-A31) was diagnosed as relapsing from Philadelphia chromosome-positive acute leukemia in the CNS after receiving chemotherapy, tyrosine kinase inhibitors, haploidentical hematopoietic stem cell transplantation (HSCT) from her father (HLA-A31), and craniospinal irradiation...
August 14, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28816170/role-of-consolidative-radiation-therapy-after-autologous-hematopoietic-cell-transplantation-for-the-treatment-of-relapsed-or-refractory-hodgkin-lymphoma
#18
Christopher Wilke, Qing Cao, Kathryn E Dusenbery, Veronika Bachanova, Aleksandr Lazaryan, Chung K Lee, Jianling Yuan
PURPOSE: To evaluate the role of the addition of consolidative radiation therapy after high-dose chemotherapy and autologous hematopoietic cell transplantation (AHCT) for relapsed or refractory Hodgkin lymphoma (HL). METHODS AND MATERIALS: Medical records were reviewed from a total of 80 consecutive patients who underwent high-dose chemotherapy with AHCT treated under a single protocol at University of Minnesota between November 2005 and January 2014. Of these, 32 patients received radiation therapy after AHCT as planned consolidation...
September 1, 2017: International Journal of Radiation Oncology, Biology, Physics
https://www.readbyqxmd.com/read/28815897/multidrug-resistant-enterobacteriaceae-pseudomonas-aeruginosa-and-vancomycin-resistant-enterococci-three-major-threats-to-hematopoietic-stem-cell-transplant-recipients
#19
REVIEW
Michael J Satlin, Thomas J Walsh
Hematopoietic stem cell transplant (HSCT) recipients are uniquely threatened by the emergence of multidrug-resistant (MDR) bacteria because these patients rely on immediate active antimicrobial therapy to combat bacterial infections. This review describes the epidemiology and treatment considerations for three challenging MDR bacterial pathogens in HSCT recipients: MDR Enterobacteriaceae, including extended-spectrum β-lactamase (ESBL)-producing and carbapenem-resistant Enterobacteriaceae (CRE), Pseudomonas aeruginosa, and vancomycin-resistant Enterococcus (VRE)...
August 16, 2017: Transplant Infectious Disease: An Official Journal of the Transplantation Society
https://www.readbyqxmd.com/read/28815419/effect-of-sertraline-on-complications-and-survival-after-hematopoietic-stem-cell-transplantation-a-double-blind-placebo-controlled-clinical-study
#20
Maria Tavakoli-Ardakani, Raziyeh Kheshti, Mehrpooya Maryam
Previous studies have found a connection between psychiatric problems and post-hematopoietic stem-cell transplantation (HSCT) complications. We sought to evaluate the effect of sertraline on engraftment time, hospitalization period, mortality, and post-transplantation complications in HSCT recipients with depression and/or anxiety. We recruited adults aged 18-60, who were candidates for autologous or allogeneic HSCT with major depression and/or anxiety disorder. They were administered 50 mg of sertraline or placebo daily for the first week, and then 100 mg for the following seven weeks...
August 16, 2017: International Journal of Hematology
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