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Engraftment syndrome

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https://www.readbyqxmd.com/read/28345002/cxcl12-promotes-stem-cell-recruitment-and-uterine-repair-after-injury-in-asherman-s-syndrome
#1
Gulcin Sahin Ersoy, Masoumeh Majidi Zolbin, Emine Cosar, Irene Moridi, Ramanaiah Mamillapalli, Hugh S Taylor
Asherman's syndrome is an acquired condition of uterine fibrosis and adhesions in response to injury that adversely affects fertility and pregnancy. We have previously demonstrated that bone marrow-derived mesenchymal stem cells (BMDSCs) contribute to uterine repair after injury and that stem cells supplementation improves fertility. Here, we demonstrate that CXCL12 is the chemokine that mediates stem cell engraftment and functional improvement using a murine model of Asherman's syndrome. After uterine injury, we demonstrate that CXCL12 augmentation increased BMDSC engraftment and that the CXCL12 receptor (CXCR4) antagonist, ADM3100, blocked stem cell recruitment...
March 17, 2017: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/28344987/safe-and-effective-gene-therapy-for-murine-wiskott-aldrich-syndrome-using-an-insulated-lentiviral-vector
#2
Swati Singh, Iram Khan, Socheath Khim, Brenda Seymour, Karen Sommer, Matthew Wielgosz, Zachary Norgaard, Hans-Peter Kiem, Jennifer Adair, Denny Liggitt, Arthur Nienhuis, David J Rawlings
Wiskott-Aldrich syndrome (WAS) is a life-threatening immunodeficiency caused by mutations within the WAS gene. Viral gene therapy to restore WAS protein (WASp) expression in hematopoietic cells of patients with WAS has the potential to improve outcomes relative to the current standard of care, allogeneic bone marrow transplantation. However, the development of viral vectors that are both safe and effective has been problematic. While use of viral transcriptional promoters may increase the risk of insertional mutagenesis, cellular promoters may not achieve WASp expression levels necessary for optimal therapeutic effect...
March 17, 2017: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/28331226/the-histone-deacetylase-inhibitor-givinostat-itf2357-exhibits-potent-anti-tumor-activity-against-crlf2-rearranged-bcp-all
#3
A M Savino, J Sarno, L Trentin, M Vieri, G Fazio, M Bardini, C Bugarin, G Fossati, K Davis, G Gaipa, S Izraeli, L H Meyer, G P Nolan, A Biondi, G Te Kronnie, C Palmi, G Cazzaniga
Leukemias bearing CRLF2 and JAK2 gene alterations are characterized by aberrant JAK/STAT signaling and poor prognosis. The HDAC inhibitor givinostat/ITF2357 has been shown to exert antineoplastic activity against both systemic juvenile idiopathic arthritis and myeloproliferative neoplasms through inhibition of the JAK/STAT pathway. These findings led us to hypothesize that givinostat might also act against CRLF2-rearranged BCP-ALL, which lack effective therapies. Here, we found that givinostat inhibited proliferation and induced apoptosis of BCP-ALL CRLF2-rearranged cell lines, positive for exon 16 JAK2 mutations...
March 23, 2017: Leukemia: Official Journal of the Leukemia Society of America, Leukemia Research Fund, U.K
https://www.readbyqxmd.com/read/28321091/azacitidine-therapy-for-low-risk-myelodysplastic-syndrome-developing-after-solid-organ-transplantation
#4
Hiroaki Inoue, Yasuyoshi Morita, Shinya Rai, Hiroaki Kakutani, Yasuyo Ohyama, Yasuhiro Taniguchi, Hirokazu Tanaka, Takahiro Shimada, Yoichi Tatsumi, Takashi Ashida, Itaru Matsumura
Immunosuppressive therapy after solid organ transplantation is known to be a risk factor for the development of myelodysplastic syndromes (MDS). Herein, we report 2 patients, both of whom developed low-risk MDS after solid organ transplantation and were successfully treated with azacitidine (AZA). The 1st case was a 74-year-old man who had received liver transplantation. The initial immunosuppressive therapy consisted of cyclosporine and prednisolone. Nine years after transplantation, he was diagnosed as having MDS (RCMD)...
2017: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://www.readbyqxmd.com/read/28318633/capillary-leak-syndrome-etiologies-pathophysiology-and-management
#5
REVIEW
Eric Siddall, Minesh Khatri, Jai Radhakrishnan
In various human diseases, an increase in capillary permeability to proteins leads to the loss of protein-rich fluid from the intravascular to the interstitial space. Although sepsis is the disease most commonly associated with this phenomenon, many other diseases can lead to a "sepsis-like" syndrome with manifestations of diffuse pitting edema, exudative serous cavity effusions, noncardiogenic pulmonary edema, hypotension, and, in some cases, hypovolemic shock with multiple-organ failure. The term capillary leak syndrome has been used to describe this constellation of disease manifestations associated with an increased capillary permeability to proteins...
March 16, 2017: Kidney International
https://www.readbyqxmd.com/read/28314577/nanotubes-the-fast-track-to-treatment-of-dent-disease
#6
Thomas E Willnow
Studies in mice have suggested bone marrow transplantation as a strategy to correct the endocytic dysfunction of the proximal tubules in renal Fanconi syndrome, yet the mode of action has remained mysterious. Using a mouse model of Dent disease, Gabriel et al. now show that rescue of the resorptive capacity in the diseased kidney involves cell-to-cell contact between engrafted and host cells via nanotubes, cellular projections that enable transfer of wild-type activity into mutant cells of the proximal tubule...
April 2017: Kidney International
https://www.readbyqxmd.com/read/28283844/long-term-survival-and-cardiopulmonary-outcome-in-children-with-hurler-syndrome-after-haematopoietic-stem-cell-transplantation
#7
Su Han Lum, Karolina M Stepien, Arunabha Ghosh, Alexander Broomfield, Heather Church, Jean Mercer, Simon Jones, Robert Wynn
Premature death in untreated children with Hurler syndrome (HS) in the first decade of life is largely due to life-threatening cardiopulmonary complications. We examined the long-term survival and cardiopulmonary outcome in 54 children undergoing haematopoietic stem cell transplantation (HSCT) at the Royal Manchester Children's Hospital from 1985 to 2008. The median age at first HSCT was 15.1 months. Eighteen had graft failure and nine died after first HSCT. Of 18 patients with graft failure, 17 underwent second HSCT and the remaining one was lost to follow-up (LOF)...
March 10, 2017: Journal of Inherited Metabolic Disease
https://www.readbyqxmd.com/read/28282510/cxcr4-cd184-expression-on-stem-cell-harvest-and-cd34-cells-post-transplant
#8
Inas Asfour, Hanaa Afify, Shaza Elkourashy, Maryse Ayoub, Gihan Kamal, Mary Gamal, Ghada Elgohary
OBJECTIVES/BACKGROUND: CXCR4 is a receptor for stromal-derived factor-1 (SDF-1), a molecule that has a chemotactic activity for lymphocytes and is important in homing of hematopoietic stem cells to their adult marrow. We evaluated the CXCR4 (CD184) expression in the harvest cells and in the post-transplant bone marrow (BM) and its relation to engraftment, as determined by the consensus criteria and chimerism. METHODS: This is a prospective study which included 30 patients undergoing hematopoietic stem cell transplantation; 15 patients received autograft and 15 patients received allograft on dates between January 2012 and May 2014...
March 2, 2017: Hematology/oncology and Stem Cell Therapy
https://www.readbyqxmd.com/read/28266766/early-mixed-t-cell-chimerism-is-predictive-of-pediatric-aml-or-mds-relapse-after-hematopoietic-stem-cell-transplant
#9
Larisa Broglie, Irene Helenowski, Lawrence J Jennings, Kristian Schafernak, Reggie Duerst, Jennifer Schneiderman, William Tse, Morris Kletzel, Sonali Chaudhury
Patients with acute myeloid leukemia (AML) who relapse after hematopoietic stem cell transplantation (HCT) have dismal outcomes. Our ability to predict those at risk for relapse is limited. We examined chimerism trends post-HCT in 63 children who underwent HCT for AML or myelodysplastic syndrome (MDS). Mixed T-cell chimerism at engraftment and absence of chronic graft versus host disease (cGVHD) were associated with relapse (P = 0.04 and P = 0.02, respectively). Mixed T-cell chimerism at engraftment was predictive in patients without cGVHD (P = 0...
March 7, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28255902/imaging-spectrum-of-central-nervous-system-complications-of-hematopoietic-stem-cell-and-solid-organ-transplantation
#10
REVIEW
Andrés Server, Nuria Bargalló, Yngvar Fløisand, Jon Sponheim, Francesc Graus, John K Hald
Neurologic complications are common after hematopoietic stem cell transplantation (HSCT) and solid organ transplantation (SOT) and affect 30-60% of transplant recipients. The aim of this article is to provide a practical imaging approach based on the timeline and etiology of CNS abnormalities, and neurologic complications related to transplantation of specific organs. The lesions will be classified based upon the interval from HSCT procedure: pre-engraftment period <30 days, early post-engraftment period 30-100 days, late post-engraftment period >100 days, and the interval from SOT procedure: postoperative phase 1-4 weeks, early posttransplant syndromes 1-6 months, late posttransplant syndromes >6 months...
March 2, 2017: Neuroradiology
https://www.readbyqxmd.com/read/28255019/a-risk-factor-analysis-of-outcomes-after-unrelated-cord-blood-transplantation-for-children-with-wiskott-aldrich-syndrome
#11
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/28245775/allogeneic-hematopoietic-stem-cell-transplant-for-severe-aplastic-anemia-current-state-and-future-directions
#12
Qixin Sun, Bingyi Wu, Zhigang Zhu, Can Sun, Jingxia Xu, Hui Long, Yuxian Huang, Jianhui Xu, Chaoyang Song
Acquired severe aplastic anemia (SAA) is a rare and life-threatening bone marrow failure syndrome characterized by cytotoxic T-cells excessive activity, hematopoietic precursors decrease and peripheral blood (PB) pancytopenia. Patients with severe aplastic anemia (SAA) died 1 to 2 years after diagnosis due to fatal infections and/or hemorrhagic complications if they went without any effective treatment. Nowadays, Immunosuppressive therapy (IST) and allogeneic hematopoietic stem cell transplantation (HSCT) are still the standard treatment for SAA...
February 27, 2017: Current Stem Cell Research & Therapy
https://www.readbyqxmd.com/read/28218755/changes-in-the-incidence-patterns-and-outcomes-of-graft-failure-following-hematopoietic-stem-cell-transplantation-for-hurler-syndrome
#13
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
https://www.readbyqxmd.com/read/28207973/risk-factors-of-human-herpesvirus-6-encephalitis-myelitis-after-allogeneic-hematopoietic-stem-cell-transplantation
#14
Naohiro Miyashita, Tomoyuki Endo, Masahiro Onozawa, Daigo Hashimoto, Takeshi Kondo, Katsuya Fujimoto, Kaoru Kahata, Junichi Sugita, Hideki Goto, Toshihiro Matsukawa, Satoshi Hashino, Takanori Teshima
BACKGROUND: Human herpesvirus 6 (HHV-6) encephalitis/myelitis is now a well-known complication after allogeneic stem cell transplantation (allo-HSCT), particularly after cord blood transplantation (CBT). In this study, we evaluated the risk factors of HHV-6 encephalitis/myelitis. METHODS: We evaluated 253 patients who received allo-HSCT from 2007 to 2015 at our institute. HHV-6 encephalitis/myelitis was defined as HHV-6 DNA detection in the cerebrospinal fluid or peripheral blood by polymerase chain reaction in the presence of typical manifestations without other concurrent condition that led to the manifestations...
February 16, 2017: Transplant Infectious Disease: An Official Journal of the Transplantation Society
https://www.readbyqxmd.com/read/28195180/successful-resolution-of-recurrent-clostridium-difficile-infection-using-freeze-dried-encapsulated-fecal-microbiota-pragmatic-cohort-study
#15
Christopher Staley, Matthew J Hamilton, Byron P Vaughn, Carolyn T Graiziger, Krista M Newman, Amanda J Kabage, Michael J Sadowsky, Alexander Khoruts
OBJECTIVES: Fecal microbiota transplantation (FMT) is increasingly being used for treatment of recurrent Clostridium difficile infection (R-CDI) that cannot be cured with antibiotics alone. In addition, FMT is being investigated for a variety of indications where restoration or restructuring of the gut microbial community is hypothesized to be beneficial. We sought to develop a stable, freeze-dried encapsulated preparation of standardized fecal microbiota that can be used for FMT with ease and convenience in clinical practice and research...
February 14, 2017: American Journal of Gastroenterology
https://www.readbyqxmd.com/read/28129450/viral-encephalitis-after-haplo-identical-hematopoietic-stem-cell-transplantation-causative-viral-spectrum-characteristics-and-risk-factors
#16
Xiao-Hui Zhang, Jia-Min Zhang, Wei Han, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Jing-Zhi Wang, Yuan-Yuan Zhang, Xiao-Dong Mo, Yao Chen, Yu Wang, Ying-Jun Chang, Lan-Ping Xu, Kai-Yan Liu, Xiao-Jun Huang
OBJECTIVE: To retrospectively identify characteristics and risk factors of viral encephalitis (VE) in patients who underwent a haplo-identical hematopoietic stem cell transplant (HSCT). METHODS: A nested case-control study was designed. Cases with VE and controls were identified from a cohort composed of 1274 patients who underwent a haplo-identical HSCT from 2012 to 2015. RESULTS: VE was identified in 30 patients (2.4%). The median time from HSCT to diagnosis was 144...
January 27, 2017: European Journal of Haematology
https://www.readbyqxmd.com/read/28111964/association-between-c-reactive-protein-levels-in-the-first-1-3-days-post-transplant-and-allogeneic-immune-reactions
#17
Yao Chen, Xiao-Jun Huang, Kai-Yan Liu, Huan Chen, Yu-Hong Chen, Xiao-Hui Zhang, Yu Wang, Feng-Rong Wang, Wei Han, Jing-Zhi Wang, Chen-Hua Yan, Lan-Ping Xu
AIM: The purpose of this study was to determine whether C-reactive protein (CRP) in the first 1-3 days post-transplant could predict allogeneic immune reactions, including engraftment syndrome or acute graft-versus-host disease (GVHD), in pediatric haploidentical stem cell transplantation. PATIENTS & METHODS: The study population comprised 175 consecutive pediatric patients. Receiver operating characteristic analysis was performed to identify the cut-off CRP value...
February 2017: Biomarkers in Medicine
https://www.readbyqxmd.com/read/28067165/gene-therapy-in-fanconi-anemia-a-matter-of-time-safety-and-gene-transfer-tool-efficiency
#18
Els Verhoeyen, Francisco José Román Rodríguez, François-Loïc Cosset, Camille Lévy, Paula Rio
Fanconi anemia (FA) is a rare genetic syndrome characterized by progressive marrow failure. Gene therapy by infusion of FA-corrected autologous hematopoietic stem cells (HSCs) may offer a potential cure since it is a monogenetic disease with mutations in the FANC genes, coding for DNA repair enzymes (See review[1]). However, the collection of hCD34 +-cells in FA patients implies particular challenges because of the reduced numbers of progenitor cells present in their bone marrow (BM)[2] or mobilized peripheral blood[3-5]...
January 9, 2017: Current Gene Therapy
https://www.readbyqxmd.com/read/27989932/allele-level-hla-matching-impacts-key-outcomes-following-umbilical-cord-blood-transplantation-for-inherited-metabolic-disorders
#19
Kanwaldeep K Mallhi, Angela R Smith, Todd E DeFor, Troy C Lund, Paul J Orchard, Weston P Miller
Allogeneic hematopoietic stem cell transplantation has demonstrated efficacy for numerous inherited metabolic disorders (IMDs). Umbilical cord blood transplant (UCBT) is increasingly used as a graft source in IMDs, but little is known of the impact of cord blood unit (CBU)/recipient HLA allelic disparity on key outcomes following UCBT for IMD. We reviewed outcomes of 106 consecutive first, single UCBTs for IMD at the University of Minnesota with regard to CBU/recipient HLA allelic matching (HLA-A, -B, -C, and -DRB1)...
January 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/27932186/late-graft-rejection-in-association-with-t-large-granular-lymphocyte-expansion-of-recipient-origin-after-human-leukocyte-antigen-haploidentical-stem-cell-transplantation-a-case-report
#20
N Nakagawa, H Yamazaki, G Aoki, Y Kondo, S Nakao
BACKGROUND: Large granular lymphocyte (LGL) expansion occasionally occurs after allogeneic stem cell transplantation (allo-SCT), and is thought to be a good prognostic sign that is associated with a lower relapse rate. However, there have been no reports of late graft failure (LGF) due to graft rejection in association with oligoclonal LGL expansion. We herein report a case of LGF associated with the transient expansion of recipient-derived T-LGL after allo-SCT. CASE REPORT: A 65-year-old man underwent peripheral blood stem cell transplantation (PBSCT) from his human leukocyte antigen (HLA)-haploidentical son for the treatment of acute myeloid leukemia, which had evolved from a myelodysplastic syndrome (MDS)...
November 2016: Transplantation Proceedings
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