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https://www.readbyqxmd.com/read/28516949/paroxysmal-nocturnal-haemoglobinuria
#1
REVIEW
Anita Hill, Amy E DeZern, Taroh Kinoshita, Robert A Brodsky
Paroxysmal nocturnal haemoglobinuria (PNH) is a clonal haematopoietic stem cell (HSC) disease that presents with haemolytic anaemia, thrombosis and smooth muscle dystonias, as well as bone marrow failure in some cases. PNH is caused by somatic mutations in PIGA (which encodes phosphatidylinositol N-acetylglucosaminyltransferase subunit A) in one or more HSC clones. The gene product of PIGA is required for the biosynthesis of glycosylphosphatidylinositol (GPI) anchors; thus, PIGA mutations lead to a deficiency of GPI-anchored proteins, such as complement decay-accelerating factor (also known as CD55) and CD59 glycoprotein (CD59), which are both complement inhibitors...
May 18, 2017: Nature Reviews. Disease Primers
https://www.readbyqxmd.com/read/28512561/bone-marrow-homing-and-engraftment-defects-of-human-hematopoietic-stem-and-progenitor-cells
#2
REVIEW
Giovanni Caocci, Marianna Greco, Giorgio La Nasa
Homing of hematopoietic stem cells (HSC) to their microenvironment niches in the bone marrow is a complex process with a critical role in repopulation of the bone marrow after transplantation. This active process allows for migration of HSC from peripheral blood and their successful anchoring in bone marrow before proliferation. The process of engraftment starts with the onset of proliferation and must, therefore, be functionally dissociated from the former process. In this overview, we analyze the characteristics of stem cells (SCs) with particular emphasis on their plasticity and ability to find their way home to the bone marrow...
2017: Mediterranean Journal of Hematology and Infectious Diseases
https://www.readbyqxmd.com/read/28512445/the-therapeutic-value-of-bone-marrow-derived-endothelial-progenitor-cell-transplantation-after-intracerebral-hemorrhage-in-rats
#3
Rui Zhang, Jing Yang, Jingjing Yuan, Bo Song, Yan Wang, Yuming Xu
AIMS: To study the effect of endothelial progenitor cell (EPC) treatment on intracerebral hemorrhage (ICH) in rats and elucidate possible mechanisms. METHODS: The rats were randomly divided into three groups: (1) EPC group: ICH + EPC, (2) phosphate-buffered saline group: ICH + PBS, and (3) sham group. EPCs were transplanted intravenously 6 h after ICH. Modified neurological severity score was used to evaluate neurological function. Blood-brain barrier (BBB) integrity was evaluated...
2017: Frontiers in Neurology
https://www.readbyqxmd.com/read/28510616/salvage-high-dose-chemotherapy-in-female-patients-with-relapsed-refractory-germ-cell-tumors-a-retrospective-analysis-of-the-european-group-for-blood-and-marrow-transplantation-ebmt
#4
U De Giorgi, S Richard, M Badoglio, E Kanfer, J H Bourrhis, E Nicolas-Virelizier, K Vettenranta, B Lioure, S Martin, P Dreger, M K Schuler, K Thomson, E Scarpi, G Rosti, F Selle, G Mangili, F Lanza, M Bregni
Background: High-dose chemotherapy (HDC) with hematopoietic progenitor cell transplantation is a standard option for relapsed/refractory testicular germ cell tumor (GCT), but only few data have been reported in female patients with GCT. We conducted a retrospective analysis of female patients with GCT treated with HDC and registered with the European Society for Blood and Marrow Transplantation (EBMT). Patients and methods: Between 1985 and 2013, 60 registered female patients with GCT, median age 27 years (range 15-48), were treated with salvage HDC...
May 16, 2017: Annals of Oncology: Official Journal of the European Society for Medical Oncology
https://www.readbyqxmd.com/read/28505619/bone-marrow-mesenchymal-stem-cell-transplantation-increases-gap-43-expression-via-erk1-2-and-pi3k-akt-pathways-in-intracerebral-hemorrhage
#5
Jianzhong Cui, Changmeng Cui, Ying Cui, Ran Li, Huaxin Sheng, Xiaohua Jiang, Yanxia Tian, Kaijie Wang, Junling Gao
BACKGROUND/AIMS: Intracerebral hemorrhage (ICH) occurs in hypertensive patients and results in high rates of mortality and disability. This study determined whether bone marrow mesenchymal stem cell (BMSC) transplantation affects axonal regeneration and examined the underlying mechanisms after the administration of PD98059 (p-ERK1/2 inhibitor) or/ and LY294002 (PI3K inhibitor). The hypothesis that was intended to be tested was that BMSC transplantation regulates the expression of growth-associated protein-43 (GAP-43) via the ERK1/2 and PI3K/Akt signaling pathways...
May 12, 2017: Cellular Physiology and Biochemistry
https://www.readbyqxmd.com/read/28502825/treatment-of-severe-forms-of-lps-responsive-beige-like-anchor-protein-lrba-deficiency-by-allogeneic-hematopoietic-stem-cell-transplantation
#6
Markus G Seidel, Katrin Böhm, Figen Dogu, Austen Worth, Adrian Thrasher, Benoit Florkin, Aydan İkincioğulları, Anke Peters, Shahrzad Bakhtiar, Marie Meeths, Polina Stepensky, Isabelle Meyts, Svetlana O Sharapova, Laura Gámez-Díaz, Lennart Hammarström, Stephan Ehl, Bodo Grimbacher, Andrew R Gennery
LRBA deficiency, a life-threatening immunodeficiency/autoimmunity disorder, was treated by HSCT in 12 patients, four of whom died from transplant-related causes. Six patients were cured without further need of immunosuppression, and two are in partial remission.
May 10, 2017: Journal of Allergy and Clinical Immunology
https://www.readbyqxmd.com/read/28499937/haploidentical-allogeneic-hematopoietic-cell-transplantation-for-multiple-myeloma-using-post-transplant-cyclophosphamide-gvhd-prophylaxis
#7
Luca Castagna, Alberto Mussetti, Raynier Devillier, Alida Dominietto, Magda Marcatti, Giuseppe Milone, Francesco Maura, Chiara de Philippis, Benedetto Bruno, Sabine Furst, Didier Blaise, Paolo Corradini, Vittorio Montefusco
Allogeneic Hematopoietic Cell Transplantation (allo-HCT) currently represents the only potentially curative therapy for patients affected by multiple myeloma (MM). Up to 30% of patients in western countries do not have a matched donor. Haploidentical HCT (haplo-HCT) may be an option, but currently, there is are available data regarding this treatment. We analyzed survival outcomes of 30 heavily pretreated MM patients who received haplo-HCT with post-transplant cyclophosphamide (PT-Cy) as graft-versus-host-disease (GVHD) prophylaxis...
May 9, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28497755/autologous-stem-cell-transplantation-in-treatment-refractory-crohn-s-disease-an-analysis-of-pooled-data-from-the-astic-trial
#8
James O Lindsay, Mathieu Allez, Miranda Clark, Myriam Labopin, Elenor Ricart, Gerhard Rogler, Montserrat Rovira, Jack Satsangi, Dominique Farge, Christopher J Hawkey
BACKGROUND: The randomised controlled ASTIC trial showed no benefit of mobilisation and autologous haematopoietic stem-cell transplantation (HSCT) compared with mobilisation followed by conventional therapy using a stringent primary endpoint (steroid-free clinical remission for 3 months with no endoscopic or radiological evidence of intestinal inflammation) in patients with treatment-refractory Crohn's disease. We now assess HSCT in patients enrolled in the ASTIC trial using endpoints that are traditional for clinical trials in Crohn's disease, and identify factors that predict benefit or harm...
June 2017: Lancet. Gastroenterology & Hepatology
https://www.readbyqxmd.com/read/28495640/sources-of-hematopoietic-stem-and-progenitor-cells-and-methods-to-optimize-yields-for-clinical-cell-therapy
#9
REVIEW
Sandhya R Panch, James Szymanski, Bipin N Savani, David F Stroncek
Bone marrow (BM) aspirates, mobilized peripheral blood (PB) and umbilical cord blood (UCB) have developed as graft sources of hematopoietic stem and progenitor cells (HSPC) for stem cell transplantation and other cellular therapeutics. Individualized techniques are necessary to enhance graft HSPC yields and cell quality from each graft source. BM aspirates yield adequate CD34+cells but can result in relative delays in engraftment. Granulocyte-colony stimulating factor (G-CSF) primed bone marrow HSPC may facilitate faster engraftment while minimizing graft-versus-host disease (GVHD) in certain patient subsets...
May 8, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28491138/clinicohematological-and-cytogenetic-profile-of-myelodysplastic-syndromes-in-pakistan-compare-and-contrast
#10
Nida Anwar, Aisha Arshad, Muhammad Nadeem, Sana Khurram, Naveena Fatima, Sumaira Sharif, Saira Shan, Tahir Shamsi
BACKGROUND: Myelodysplastic syndromes (MDS) are clonal stem cell disorders exhibiting cytopenias, ineffective hematopoiesis and morphological dysplasia. Bone marrow cytogenetics, inspite of being incorporated as mandatory tool in diagnosis are done less frequently due to limited availability of this technique in Pakistan. The aim of the study was to study baseline clinicohematological and cytogenetic characteristics of patients presenting with de novo MDS. RESULTS: A retrospective cross sectional study was done at National Institute of Blood Diseases and Bone Marrow Transplantation, Karachi, Pakistan from 2010 to 2016...
2017: Molecular Cytogenetics
https://www.readbyqxmd.com/read/28490935/characteristics-predicting-outcomes-of-allogeneic-stem-cell-transplantation-in-relapsed-acute-myelogenous-leukemia
#11
J Frazer, S Couban, S Doucette, S Shivakumar
BACKGROUND: Allogeneic hematopoietic stem-cell transplantation (ahsct) is associated with significant morbidity and mortality, but it can cure carefully selected patients with acute myeloid leukemia (aml) in second remission (cr2). In a cohort of patients with aml who underwent ahsct in cr2, we determined the pre-transplant factors that predicted for overall survival (os), relapse, and non-relapse mortality. We also sought to validate the prognostic risk groups derived by Michelis and colleagues in this independent population...
April 2017: Current Oncology
https://www.readbyqxmd.com/read/28488966/haemolysis-pure-red-cell-aplasia-and-red-cell-antibody-formation-associated-with-major-and-bidirectional-abo-incompatible-haematopoietic-stem-cell-transplantation
#12
Gordana Tomac, Ines Bojanić, Sanja Mazić, Ivana Vidović, Mirela Raos, Branka Golubić Ćepulić, Ranka Serventi Seiwerth, Jadranka Kelečić, Boris Labar
BACKGROUND: Acute and delayed haemolysis, alloimmunisation and pure red cell aplasia (PRCA) are potential complications after ABO incompatible haematopoietic stem cell transplantation (HSCT). The aims of this study were to investigate acute and delayed red blood cell (RBC) antibody-associated complications, including haemolysis, PRCA and alloimmunisation in major and bidirectional ABO incompatible HSCT. MATERIALS AND METHODS: We retrospectively examined the transplant courses of 36 recipients of bone marrow or peripheral blood stem cells from ABO incompatible donors and evaluated the current practice of performing plasmapheresis in patients with higher isoagglutinin titres...
April 19, 2017: Blood Transfusion, Trasfusione del Sangue
https://www.readbyqxmd.com/read/28485360/monotherapy-versus-combination-therapy-against-carbapenem-resistant-gram-negative-bacteria-a-retrospective-observational-study
#13
A Ghafur, V Devarajan, T Raja, J Easow, M A Raja, S Sreenivas, B Ramakrishnan, S G Raman, D Devaprasad, B Venkatachalam, R Nimmagadda
BACKGROUND: Colistin-based combination therapy (CCT) is extensively used to treat infections due to carbapenem-resistant Gram-negative bacteria (CRGNB). There are no data available from India on the usefulness of combination therapy, especially in the oncology setup. The aim of this study was to analyze the clinical effectiveness of CCT over monotherapy in patients with CRGNB. MATERIALS AND METHODS: We conducted a retrospective, observational study of patients with CRGNB bloodstream infections in our oncology and bone marrow transplant center...
October 2016: Indian Journal of Cancer
https://www.readbyqxmd.com/read/28484721/donor-specific-anti-human-leukocyte-antigen-antibodies-predict-prolonged-isolated-thrombocytopenia-and-inferior-outcomes-of-haploidentical-hematopoietic-stem-cell-transplantation
#14
Xiaosu Zhao, Xiangyu Zhao, Mingrui Huo, Qiaozhen Fan, Xuying Pei, Yu Wang, Xiaohui Zhang, Lanping Xu, Xiaojun Huang, Kaiyan Liu, Yingjun Chang
Prolonged isolated thrombocytopenia (PT) after allogeneic stem cell transplantation (allo-SCT) has a great impact on transplant outcome. In this study, we performed a retrospective analysis to investigate the association of donor-specific anti-human leukocyte antigen (HLA) antibodies (DSAs) with PT in 394 patients who underwent unmanipulated haploidentical blood and marrow transplantation (HBMT). For HLA antibody positive samples with a median fluorescent intensity (MFI) > 500, DSAs were further examined...
2017: Journal of Immunology Research
https://www.readbyqxmd.com/read/28479164/recommendations-for-screening-and-management-of-late-effects-in-scid-patients-after-allogenic-hematopoietic-cell-transplantation-hct-a-consensus-statement-from-the-second-pediatric-blood-and-marrow-transplant-consortium-international-conference-on-late-effects
#15
REVIEW
J Heimall, R H Buckley, J Puck, T A Fleisher, A R Gennery, E Haddad, B Neven, M Slatter, Roderick S, K S Baker, A C Dietz, C Duncan, L M Griffith, L Notarangelo, M A Pulsipher, M J Cowan
Severe Combined Immunodeficiency (SCID) is effectively treated with HCT with overall survival approaching 90% in contemporary reports. However, survivors are at risk for development of late complications due to the variable durability of high quality immune function, underlying genotype of SCID, comorbidities due to infections in the pre- and post- transplant period and use of conditioning pre-transplant. An international group of transplant experts was convened in 2016 to review the current knowledge of late effects seen in SCID patients following HCT, and develop recommendations for screening and monitoring for late effects...
May 4, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28474594/un-manipulated-haploidentical-transplant-in-wiskott-aldrich-syndrome
#16
M Joseph John, Chepsy C Philip, Amrith Mathew, Abhilasha Williams, Naveen Kakkar
BACKGROUND: Allogeneic stem cell transplant is the only curative treatment for Wiskott-Aldrich syndrome. CASE CHARACTERISTICS: 18-months-old boy with no sibling, cord blood or matched unrelated donor transplant options. OUTCOME: Doing well 7 years after haplo-identical stem cell transplantation using unmanipulated bone marrow as the stem cell source. MESSAGE: Father as a haplo-identical donor is a feasible option.
April 15, 2017: Indian Pediatrics
https://www.readbyqxmd.com/read/28474513/bone-marrow-endothelial-progenitor-cells-are-the-cellular-mediators-of-pulmonary-hypertension-in-the-murine-monocrotaline-injury-model
#17
Jason M Aliotta, Mandy Pereira, Sicheng Wen, Mark S Dooner, Michael Del Tatto, Elaine Papa, Yan Cheng, Laura Goldberg, Corey E Ventetuolo, Olin Liang, James R Klinger, Peter J Quesenberry
The role of bone marrow (BM) cells in modulating pulmonary hypertensive responses is not well understood. Determine if BM-derived endothelial progenitor cells (EPCs) induce pulmonary hypertension (PH) and if this is attenuated by mesenchymal stem cell (MSC)-derived extracellular vesicles (EVs). Three BM populations were studied: (a) BM from vehicle and monocrotaline (MCT)-treated mice (PH induction), (b) BM from vehicle-, MCT-treated mice that received MSC-EV infusion after vehicle, MCT treatment (PH reversal, in vivo), (c) BM from vehicle-, MCT-treated mice cultured with MSC-EVs (PH reversal, in vitro)...
May 5, 2017: Stem Cells Translational Medicine
https://www.readbyqxmd.com/read/28472539/hispanics-have-the-lowest-stem-cell-transplant-utilization-rate-for-autologous-hematopoietic-cell-transplantation-for-multiple-myeloma-in-the-united-states-a-cibmtr-report
#18
Jeffrey R Schriber, Parameswaran N Hari, Kwang Woo Ahn, Mingwei Fei, Luciano J Costa, Mohamad A Kharfan-Dabaja, Miguel Angel-Diaz, Robert P Gale, Siddharatha Ganguly, Saulius K Girnius, Shahrukh Hashmi, Attaphol Pawarode, David H Vesole, Peter H Wiernik, Baldeep M Wirk, David I Marks, Taiga Nishihori, Richard F Olsson, Saad Z Usmani, Tomer M Mark, Yago L Nieto, Anita D'Souza
BACKGROUND: Race/ethnicity remains an important barrier in clinical care. The authors investigated differences in the receipt of autologous hematopoietic cell transplantation (AHCT) among patients with multiple myeloma (MM) and outcomes based on race/ethnicity in the United States. METHODS: The Center for International Blood and Marrow Transplant Research database was used to identify 28,450 patients who underwent AHCT for MM from 2008 through 2014. By using data from the National Cancer Institute's Surveillance, Epidemiology, and End Results 18 registries, the incidence of MM was calculated, and a stem cell transplantation utilization rate (STUR) was derived...
May 4, 2017: Cancer
https://www.readbyqxmd.com/read/28472335/tools-for-the-precision-medicine-era-how-to-develop-highly-personalized-treatment-recommendations-from-cohort-and-registry-data-using-q-learning
#19
Elizabeth F Krakow, Michael Hemmer, Tao Wang, Brent Logan, Mukta Arora, Stephen Spellman, Daniel Couriel, Amin Alousi, Joseph Pidala, Michael Last, Silvy Lachance, Erica E M Moodie
Q-learning is a method of reinforcement learning that employs backwards stage-wise estimation to identify sequences of actions that maximize some long-term reward. The method can be applied to sequential multiple-assignment randomized trials to develop personalized adaptive treatment strategies (ATSs) - longitudinal practice guidelines highly tailored to time-varying attributes of individual patients. Sometimes, the basis for choosing which ATSs to include in a sequential multiple-assignment randomized trial (or randomized controlled trial) may be inadequate...
May 3, 2017: American Journal of Epidemiology
https://www.readbyqxmd.com/read/28469041/hepatosplenic-candidiasis-without-prior-documented-candidemia-an-underrecognized-diagnosis
#20
Joffrey van Prehn, C Willemien Menke-van der Houven van Oordt, Madelon L de Rooij, Ellen Meijer, Marije K Bomers, Karin van Dijk
INTRODUCTION: Patients with a history of chemotherapy or stem cell transplantation (SCT) and prolonged neutropenia are at risk for hepatic and/or splenic seeding of Candida. In our experience, hepatosplenic candidiasis (HSC) without documented candidemia often remains unrecognized. CASE PRESENTATIONS: We describe three cases of HSC without documented candidemia and the challenges in establishing the diagnosis and adequately treating this condition. The first patient had a history of SCT for treatment of breast cancer and was scheduled for hemihepatectomy for suspected liver metastasis...
May 3, 2017: Oncologist
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