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https://www.readbyqxmd.com/read/29240299/the-effect-of-haemopoietic-stem-cell-transplantation-on-the-ocular-phenotype-in-mucopolysaccharidosis-type-i-hurler
#1
Ahmed Javed, Tariq Aslam, Simon A Jones, Jean Mercer, Karen Tyler, Heather Church, Arunabha Ghosh, Robert Wynn, Krishanthy Sornalingam, Jane Ashworth
PURPOSE: To determine whether the ocular phenotype in patients with mucopolysaccharidosis type I (MPSI) Hurler is affected by the efficacy of previous haemopoietic stem cell transplantation (HSCT). DESIGN: A retrospective cohort study of patients with MPSI who had undergone treatment with HSCT. METHODS: Ocular phenotype was documented for each patient and compared to levels of biomarkers representing efficacy of previous transplantation. MAIN OUTCOME MEASURES: Assessment of visual acuity (VA), severity of corneal clouding and the presence of optic neuropathy or retinopathy...
December 14, 2017: Acta Ophthalmologica
https://www.readbyqxmd.com/read/29239497/clinical-outcomes-of-unrelated-cord-blood-transplantation-in-children-with-malignant-and-non-malignant-diseases-multicenter-experience-in-china
#2
Xiangfeng Tang, Jianpei Fang, Jie Yu, Zhiguang Li, Jing Chen, Xin Sun, Yiping Zhu, Shaoyan Hu, Maoquan Qin, Yongmin Tang, Zuo Luan
This multicenter retrospective study included 184 children with malignant and non-malignant diseases who underwent UCBT between January 1998 and August 2012. The malignant disease group included 101 children with ALL, AML, CML, JMML, and MDS, and the non-malignant disease group included 83 children with PID, β-thalassemia, IMD BMF, and HLH. The median duration to neutrophil and platelet engraftment was 16 and 35 days in the malignant disease group vs 15 and 38 days in the non-malignant disease group. The cumulative incidence of grade II-IV aGVHD and cGVHD was 25...
December 14, 2017: Pediatric Transplantation
https://www.readbyqxmd.com/read/29239076/allogeneic-hematopoietic-stem-cell-transplantation-is-associated-with-cure-and-durable-remission-of-late-onset-primary-isolated-central-nervous-system-hemophagocytic-lymphohistiocytosis
#3
Sajad Khazal, Veronika Polishchuk, Gary Soffer, Samantha Prinzing, Jonathan Gill, Kris M Mahadeo
Primary isolated CNS presentation of HLH is exceedingly rare and typically associated with significant morbidity and mortality. We describe an adolescent patient with late-onset, primary isolated CNS HLH and a compound heterozygous PRF1 mutation (c50delT (p.L17 fs); c.1229G>C (p.R410P)), not previously reported with this phenotype. He was successfully treated with allogeneic HSCT following a reduced-intensity conditioning regimen, despite a high pre-HSCT comorbidity index. Two years after transplant, he is alive and in disease remission...
December 13, 2017: Pediatric Transplantation
https://www.readbyqxmd.com/read/29237963/analysis-of-factors-affecting-initial-cyclosporine-level-and-its-impact-on-post-transplant-outcomes-in-acute-leukemia
#4
Alok Gupta, Sachin Punatar, Jayant Gawande, Libin Mathew, Sadhana Kannan, Navin Khattry
BACKGROUND: Trough cyclosporine (CsA) blood level can influence incidence of graft-versus-host disease (GVHD) and relapse in patients with acute leukemia undergoing allogeneic hematopoietic stem cell transplant (HSCT). We sought to determine factors affecting initial trough CsA level (CsA-1) and its impact on transplant outcome in acute leukemia. MATERIALS AND METHODS: Seventy-seven patients underwent HSCT for acute leukemia between January 2008 and March 2013 and were included...
October 2017: Journal of Cancer Research and Therapeutics
https://www.readbyqxmd.com/read/29237659/long-term-follow-up-of-hepatitis-associated-aplastic-anaemia
#5
Cristina Gonçalves, Sandra Ferreira, Susana Nobre, Isabel Gonçalves
Prognosis of hepatitis-associated aplastic anaemia (HAAA) was improved with haematopoietic stem cell transplantation (HSCT) and immunosuppression, but the long-term outcome remains undefined. Case 1: a girl aged 3 years with acute liver failure (ALF) submitted to orthotopic liver transplantation (OLT) subsequently developed aplastic anaemia and HSCT from a compatible sibling was performed. Post-HSCT, the patient developed post-transplant lymphoproliferative disorder and rituximab was administered with good response...
December 13, 2017: BMJ Case Reports
https://www.readbyqxmd.com/read/29235226/reduced-therapeutic-effect-of-antiviral-drugs-in-patients-with-hepatitis-b-virus-reactivation-after-hematopoietic-stem-cell-transplantation
#6
Masamichi Kimura, Koji Nishikawa, Hisashi Sakamaki, Masashi Mizokami, Kiminori Kimura
BACKGROUND: Patients with resolved Hepatitis B virus (HBV) infection following hematopoietic stem cell transplantation (HSCT) are potentially at high risk of HBV reactivation. Although anti-viral drug therapy is recommended when HBV DNA reappears in the serum, drug efficacy after HBV reactivation remains unclear. METHODS: Host immune response against HBV was investigated via immunological analyses at 12 months after entecavir (ETV) treatment in six HSCT-treated and five non-HSCT-treated patients with HBV reactivation, and 18 patients with chronic hepatitis B (CHB)...
December 13, 2017: Hepatology Research: the Official Journal of the Japan Society of Hepatology
https://www.readbyqxmd.com/read/29234980/low-dose-interleukin-2-as-a-modulator-of-treg-homeostasis-after-hsct-current-understanding-and-future-perspectives
#7
REVIEW
Ken-Ichi Matsuoka
CD4+CD25+Foxp3+ Treg is a functionally distinct subset of mature T cells with broad suppressive activity and has been shown to play an important role in the establishment of immune tolerance after HSCT. Altered cytokine environment in post-HSCT lymphopenia with a relative functional deficiency of IL-2 could hamper the reconstitution of Treg, leading to refractory GVHD. Based on the theory of low-dose IL-2 in which Treg can be selectively stimulated through the high-affinity IL-2 receptor, clinical studies have been conducted and demonstrated that low-dose IL-2 administration can restore Treg homeostasis and promote the expansion of this subset on the polymorphic processes of Treg reconstitution after HSCT...
December 12, 2017: International Journal of Hematology
https://www.readbyqxmd.com/read/29233822/hematopoietic-stem-cell-gene-therapy-for-ifn%C3%AE-r1-deficiency-protects-mice-from-mycobacterial-infections
#8
Miriam Hetzel, Adele Mucci, Patrick Blank, Ariane Hai Ha Nguyen, Jan Schiller, Olga Halle, Mark-Philipp Kühnel, Sandra Billig, Robert Meineke, Daniel Brand, Vanessa Herder, Wolfgang Baumgärtner, Franz-Christoph Bange, Ralph Goethe, Danny Jonigk, Reinhold Förster, Bernhard Gentner, Jean-Laurent Casanova, Jacinta Bustamante, Axel Schambach, Ulrich Kalinke, Nico Lachmann
Mendelian Susceptibility to Mycobacterial Disease (MSMD) is a rare primary immunodeficiency, characterized by severe infections caused by weakly virulent mycobacteria. Bi-allelic null mutations in genes encoding interferon gamma (IFNγ) receptor 1 or -2 (IFNGR1, IFNGR2) result in a life-threatening disease phenotype in early childhood. Recombinant IFNγ therapy is inefficient and hematopoietic stem cell transplantation (HSCT) has a poor prognosis. Thus, we developed a HSC gene therapy approach using lentiviral vectors expressing Ifnγr1 either constitutively or myeloid-specifically...
December 12, 2017: Blood
https://www.readbyqxmd.com/read/29232422/atypical-disseminated-herpes-zoster-management-guidelines-in-immunocompromised-patients
#9
Daniel J Lewis, Megan J Schlichte, Harry Dao
Reactivation of the varicella-zoster virus (VZV) causes dermatomal herpes zoster (HZ) and more rarely severe disseminated HZ including diffuse rash, encephalitis, hepatitis, and pneumonitis. An atypical form of VZV infection, disseminated HZ has been described primarily in immunocompromised hosts. We report 2 cases of atypical disseminated HZ in immunocompromised patients presenting with diffuse, nondermatomal, vesicular eruptions. We also provide a review of the literature and summarize the current guidelines for the treatment and prophylaxis of HZ in patients with human immunodeficiency virus (HIV) infection, solid organ transplantation (SOT), and hematopoietic stem cell transplantation (HSCT)...
November 2017: Cutis; Cutaneous Medicine for the Practitioner
https://www.readbyqxmd.com/read/29230958/treatment-related-mortality-in-relapsed-childhood-acute-lymphoblastic-leukemia
#10
Trausti Oskarsson, Stefan Söderhäll, Johan Arvidson, Erik Forestier, Thomas Leth Frandsen, Marit Hellebostad, Päivi Lähteenmäki, Ólafur G Jónsson, Ida Hed Myrberg, Mats Heyman
BACKGROUND: Treatment of relapsed childhood acute lymphoblastic leukemia (ALL) is particularly challenging due to the high treatment intensity needed to induce and sustain a second remission. To improve results, it is important to understand how treatment-related toxicity impacts survival. PROCEDURE: In this retrospective population-based study, we described the causes of death and estimated the risk for treatment-related mortality in patients with first relapse of childhood ALL in the Nordic Society of Paediatric Haematology and Oncology ALL-92 and ALL-2000 trials...
December 12, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/29226393/analysis-of-abo-chimera-from-peripheral-red-cells-and-reticulocytes-by-flow-cytometry-and-micro-gel-column-technique-in-patients-post-abo-incompatible-hsct
#11
Jianhua Chen, Feng Liu
BACKGROUND: How to choose an appropriate method to monitoring dynamic ABO chimera post ABO incompatible HSCT is crucial to not only assess the status of erythroid engraftment but achieve personalized safety transfusion. METHODS: We evaluated the efficacy of micro gel column technique(MGCT) and flow cytometry(FCM) by series of artificial ABO chimera mixtures from 0.5% to 50% and by investigated 15 cases of ABO incompatible HSCT patients with longitudinally ABO blood grouping and ABO chimera from RBCs and reticulocytes...
December 11, 2017: Clinical Transplantation
https://www.readbyqxmd.com/read/29226172/enteric-microbiome-markers-as-early-predictors-of-clinical-outcome-in-allogeneic-hematopoietic-stem-cell-transplant-results-of-a-prospective-study-in-adult-patients
#12
Nicasio Mancini, Raffaella Greco, Renée Pasciuta, Maria Chiara Barbanti, Giacomo Pini, Olivia Beatrice Morrow, Mara Morelli, Luca Vago, Nicola Clementi, Fabio Giglio, Maria Teresa Lupo Stanghellini, Alessandra Forcina, Laura Infurnari, Sarah Marktel, Andrea Assanelli, Matteo Carrabba, Massimo Bernardi, Consuelo Corti, Roberto Burioni, Jacopo Peccatori, Maria Pia Sormani, Giuseppe Banfi, Fabio Ciceri, Massimo Clementi
Background: Infections and graft-vs-host disease (GvHD) still represent major, not easily predictable complications in allogeneic hematopoietic stem cell transplant (allo-HSCT). Both conditions have been correlated to altered enteric microbiome profiles during the peritransplant period. The main objective of this study was to identify possible early microbiome-based markers useful in pretransplant risk stratification. Methods: Stool samples were collected from 96 consecutive patients at the beginning of the pretransplant conditioning regimen (T0) and at 10 (T1) and 30 (T2) days following transplant...
2017: Open Forum Infectious Diseases
https://www.readbyqxmd.com/read/29224324/-clinical-analysis-of-lung-transplantation-in-eight-patients-with-obstructive-bronchiolitis-syndrome-after-hematopoietic-stem-cell-transplantation
#13
L L Wang, H Y Wang, Y F Shen, M Z Lu, J Y Chen, B Wu
Objective: To investigate the efficacy and prognosis of lung transplantation (LT) for end-stage bronchiolitis obliterans syndrome (BOS) after allogeneic hematopoietic stem cell transplantation (allo-HSCT) . Methods: The clinical data of eight cases with end-stage BOS after allo-HSCT who were treated by LT in our hospital were retrospectively analyzed. Results: Eight patients with hematological malignancy underwent allo-HSCT, and the median age was 23 (12-40) years. The donors are parents or siblings. Severe BOS occurred in 8 patients after allo-HSCT, the median age for LT was 27...
November 14, 2017: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/29224314/-clinical-value-of-pcr-for-viral-detection-of-bronchoalveolar-lavage-fluid-in-the-diagnosis-and-treatment-of-pneumonia-after-allogeneic-hematopoietic-stem-cell-transplantation
#14
Y Y Chen, X Y Luo, X S Zhao, Z H Jiang, Y Chen, H Chen, X D Mo, W Han, F R Wang, J Z Wang, C H Yan, Y Q Sun, Y Y Zhang, T T Han, F F Tang, H X Fu, S Zhang, Y Wang, L P Xu, X H Zhang, K Y Liu, X J Huang
Objective: To analyze the clinical value of real-time PCR for virus detection in the diagnosis and treatment of patients after allo-HSCT who had no infection evidence of pneumonia using routine pathogen detection panel. Methods: The clinical data of 71 episodes with acute lung injury from May 2015 to March 2017 after allo-HSCT in hematology department of Peking University People's Hospital (PKUPH) were retrospectively analyzed. PCR for virus detection and other routine pathogen detection tests were performed on bronchoalveolar lavage fluid (BALF) samples...
November 14, 2017: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/29224313/-incidence-of-blood-stream-infections-of-1265-patients-with-hematopoietic-stem-cell-transplantation-and-analysis-of-pathogenic-bacteria
#15
Q Z Han, Y Chen, H Yang, X F Zhang, J Chen, D P Wu, S N Chen, H Y Qiu
Objective: To analyze the incidence and microbiological features of blood stream infections (BSI) of patients with hematopoietic stem cell transplantation (HSCT) and to provide laboratory data for empirical use of antibiotic for the HSCT patients with BSI. Methods: The incidence of bloodstream infection, the positive rate of blood culture, bacterial spectrum and drug resistance were analyzed in 1 265 HSCT recipients during 2013 and 2015 were retrospectively studied. Results: Of 1265 patients undergoing HSCT, 1 422 cases of suspected BSI occurred in 784 patients (61...
November 14, 2017: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/29223406/chronic-granulomatous-disease-in-children-a-single-center-experience
#16
Alessandra Beghin, Marta Comini, Annarosa Soresina, Luisa Imberti, Michela Zucchi, Alessandro Plebani, Alessandro Montanelli, Fulvio Porta, Arnalda Lanfranchi
Chronic Granulomatous Disease (CGD) is caused by the failure of the phagocytes to kill pathogens. We carried out a retrospective analysis of cellular, molecular and clinical features of 14 young patients (mean age at the onset of symptoms and diagnosis: 10 and 25months, respectively), 7 with autosomal recessive and 7 X-linked form, referred to the Children's Hospital of Brescia between 1999 and 2016. Two new mutations were found, one localized in the CYBB and one in the NCF1 genes. Twelve patients were followed in our institution; the average length of their follow-up after diagnosis was 66months in X-linked patients and 126months in autosomal recessive inheritance...
December 6, 2017: Clinical Immunology: the Official Journal of the Clinical Immunology Society
https://www.readbyqxmd.com/read/29223388/transplantation-in-the-treatment-of-primary-cutaneous-aggressive-epidermotropic-cytotoxic-cd8-positive-t-cell-lymphoma
#17
Benoit M Cyrenne, Juliet Fraser Gibson, Antonio Subtil, Michael Girardi, Iris Isufi, Stuart Seropian, Francine Foss
BACKGROUND: Primary cutaneous aggressive epidermotropic cytotoxic CD8 positive T-cell lymphoma (CD8+ PCAETL) is a rare subtype of peripheral T-cell lymphoma with poor outcomes and without a standardized treatment strategy. Allogeneic hematopoietic stem cell transplantation (HSCT) has been suggested as a potential curative therapy. PATIENTS AND METHODS: We conducted a retrospective case series. We identified 8 patients with the diagnosis of CD8+ PCAETL, 4 of whom also underwent allogeneic HSCT...
December 6, 2017: Clinical Lymphoma, Myeloma & Leukemia
https://www.readbyqxmd.com/read/29222325/cells-to-prevent-treat-relapse-following-allogeneic-stem-cell-transplantation
#18
REVIEW
Andrew C Dietz, Alan S Wayne
Relapse of cancer remains one of the primary causes of treatment failure and mortality after allogeneic hematopoietic stem cell transplantation (HSCT). A multitude of approaches have been used in the management of posttransplant relapse. This review focuses on recent data with cellular therapies designed to treat or prevent posttransplant relapse of hematologic malignancies, although many of these therapeutic approaches also have applications to solid tumors and in the nontransplant setting. Currently available cell therapies include second transplant, natural killer cells, monocyte-derived dendritic cell vaccines, and lymphocytes via donor lymphocyte infusion, antigen-primed cytotoxic T lymphocytes, cytokine-induced killer cells, marrow-infiltrating lymphocytes, and chimeric antigen receptor T cells...
December 8, 2017: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/29222315/extracorporeal-photopheresis-cellular-therapy-for-the-treatment-of-acute-and-chronic-graft-versus-host-disease
#19
REVIEW
Jennifer Schneiderman
Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative option for many disease states. Despite significant improvements in strategies used to prevent and treat acute and chronic graft-versus-host disease (a/cGVHD), they continue to negatively affect outcomes of HSCT significantly. Standard, first-line treatment consists of corticosteroids; beyond this, there is little consistency in therapeutic regimens. Current options include the addition of various immunosuppressive agents, the use of which puts patients at even higher risks for infection and other morbidities...
December 8, 2017: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/29221143/impact-of-human-mesenchymal-stromal-cells-on-antifungal-host-response-against-aspergillus-fumigatus
#20
Stanislaw Schmidt, Lars Tramsen, Andreas Schneider, Ralf Schubert, Ada Balan, Özer Degistirici, Roland Meisel, Thomas Lehrnbecher
Mesenchymal stromal cells (MSCs) are increasingly given as immunotherapy to hematopoietic stem cell transplant (HSCT) recipients with refractory graft-versus-host disease (GvHD). Whereas the immunosuppressive properties of MSCs seem to be beneficial in GvHD, there is, at the same time, major concern that MSCs increase the risk for infection. We therefore investigated the interplay of human MSCs with Aspergillus fumigatus and the impact of MSCs on different arms of the anti-Aspergillus host response in vitro...
November 10, 2017: Oncotarget
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