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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/28513891/haematopoietic-stem-cell-transplantation-for-severe-combined-immunodeficiency-long-term-health-outcomes-and-patient-perspectives
#2
Adrian Ys Lee, Katie Frith, Lilian Schneider, John B Ziegler
AIM: To examine the long-term follow-up and health outcomes of patients who have undergone haematopoietic stem cell transplant (HSCT) for severe combined immunodeficiency (SCID). METHODS: Through a structured questionnaire, we examined follow-up arrangements and long-term health outcomes in 22 children who have had a successful HSCT for SCID during the period of 1984-2012 at the Sydney Children's Hospital, Sydney, Australia. RESULTS: Most children considered themselves healthy and 'cured' from SCID...
May 17, 2017: Journal of Paediatrics and Child Health
https://www.readbyqxmd.com/read/28504663/a-new-time-dependent-approach-for-assessment-of-the-impact-of-invasive-aspergillosis-shows-effect-on-short-but-not-on-long-term-survival-of-patients-with-aml-or-high-risk-mds
#3
R J van de Peppel, P A von dem Borne, S le Cessie, M G J de Boer
Invasive aspergillosis (IA) has been reported to yield high mortality rates. Patients with an unfavourable prognostic haematological disease not only have a higher probability of developing IA but are also more likely to die due to causes directly related to the underlying disease. This complexity of risk mechanisms confounds the causal interpretation of IA occurrence and mortality. Full consideration of the changing patient characteristics over time is necessary to obtain reliable estimates of the correlation of IA with mortality...
May 15, 2017: Bone 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
#4
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/28494265/intramyocardial-fate-and-effect-of-iron-nanoparticles-co-injected-with-macs-%C3%A2-purified-stem-cell-products
#5
Paula Müller, Ralf Gaebel, Heiko Lemcke, Frank Wiekhorst, Frauke Hausburg, Cajetan Lang, Nicole Zarniko, Bernd Westphal, Gustav Steinhoff, Robert David
BACKGROUND: Magnetic activated cell sorting (MACS(®)) is routinely used to isolate stem cell subpopulations intended for the treatment of cardiovascular diseases. In strong contrast, studies examining the amount, effect and intramyocardial distribution of iron nanoparticles used for magnetic cell labelling are missing, although iron excess can cause functional disorders in the heart. METHODS AND RESULTS: CD133(+) haematopoietic and CD271(+) mesenchymal stem cells were purified from bone marrow using automatically and manually MACS(®) based systems...
May 4, 2017: Biomaterials
https://www.readbyqxmd.com/read/28490733/pharmacometabolomics-for-predicting-variable-busulfan-exposure-in-paediatric-haematopoietic-stem-cell-transplantation-patients
#6
Bora Kim, Ji Won Lee, Kyung Taek Hong, Kyung-Sang Yu, In-Jin Jang, Kyung Duk Park, Hee Young Shin, Hyo Seop Ahn, Joo-Youn Cho, Hyoung Jin Kang
Owing to its narrow therapeutic range and high pharmacokinetic variability, optimal dosing for busulfan is important to minimise overexposure-related systemic toxicity and underexposure-related graft failure. Using global metabolomics, we investigated biomarkers for predicting busulfan exposure. We analysed urine samples obtained before busulfan administration from 59 paediatric patients divided into 3 groups classified by area under the busulfan concentration-time curve (AUC), i.e., low-, medium-, and high-AUC groups...
May 10, 2017: Scientific Reports
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
#7
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/28485401/foxp3-regulatory-t-cells-maintain-the-bone-marrow-microenvironment-for-b-cell-lymphopoiesis
#8
Antonio Pierini, Hidekazu Nishikii, Jeanette Baker, Takaharu Kimura, Hye-Sook Kwon, Yuqiong Pan, Yan Chen, Maite Alvarez, William Strober, Andrea Velardi, Judith A Shizuru, Joy Y Wu, Shigeru Chiba, Robert S Negrin
Foxp3(+) regulatory T cells (Treg cells) modulate the immune system and maintain self-tolerance, but whether they affect haematopoiesis or haematopoietic stem cell (HSC)-mediated reconstitution after transplantation is unclear. Here we show that B-cell lymphopoiesis is impaired in Treg-depleted mice, yet this reduced B-cell lymphopoiesis is rescued by adoptive transfer of affected HSCs or bone marrow cells into Treg-competent recipients. B-cell reconstitution is abrogated in both syngeneic and allogeneic transplantation using Treg-depleted mice as recipients...
May 9, 2017: Nature Communications
https://www.readbyqxmd.com/read/28476244/imaging-of-hepatic-toxicity-of-systemic-therapy-in-a-tertiary-cancer-centre-chemotherapy-haematopoietic-stem-cell-transplantation-molecular-targeted-therapies-and-immune-checkpoint-inhibitors
#9
REVIEW
F Alessandrino, S H Tirumani, K M Krajewski, A B Shinagare, J P Jagannathan, N H Ramaiya, D N Di Salvo
The purpose of this review is to familiarise radiologists with the spectrum of hepatic toxicity seen in the oncology setting, in view of the different systemic therapies used in cancer patients. Drug-induced liver injury can manifest in various forms, and anti-neoplastic agents are associated with different types of hepatotoxicity. Although chemotherapy-induced liver injury can present as hepatitis, steatosis, sinusoidal obstruction syndrome, and chronic parenchymal damages, molecular targeted therapy-associated liver toxicity ranges from mild liver function test elevation to fulminant life-threatening acute liver failure...
May 2, 2017: Clinical Radiology
https://www.readbyqxmd.com/read/28450387/composite-biomarker-panel-for-prediction-of-severity-and-diagnosis-of-acute-gvhd-with-t-cell-depleted-allogeneic-stem-cell-transplants-single-centre-pilot-study
#10
San San Min, Varun Mehra, Jennifer Clay, Gemma F Cross, Abdel Douiri, Tracy Dew, Tanya N Basu, Victoria Potter, M Mansour Ceesay, Antonio Pagliuca, Roy A Sherwood, Royce P Vincent
AIMS: Acute graft-versus-host disease (aGVHD) is a leading cause of morbidity and mortality following allogeneic haematopoietic stem cell transplantation (HSCT). The aim of this study was to evaluate the clinical utility of a composite biomarker panel to help identify individuals at risk of developing aGVHD, and to help predict and differentiate between severity of aGVHD following T-cell-depleted allogeneic HSCT. METHODS: We retrospectively analysed our cohort of biopsy confirmed patients with aGVHD, who underwent T-cell-depleted HSCT and matched them with negative controls without any evidence of aGVHD...
April 27, 2017: Journal of Clinical Pathology
https://www.readbyqxmd.com/read/28444784/earlier-defibrotide-initiation-post-diagnosis-of-veno-occlusive-disease-sinusoidal-obstruction-syndrome-improves-day-100-survival-following-haematopoietic-stem-cell-transplantation
#11
Paul G Richardson, Angela R Smith, Brandon M Triplett, Nancy A Kernan, Stephan A Grupp, Joseph H Antin, Leslie Lehmann, Maja Miloslavsky, Robin Hume, Alison L Hannah, Bijan Nejadnik, Robert J Soiffer
Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a progressive, potentially fatal complication of conditioning for haematopoietic stem cell transplant (HSCT). The VOD/SOS pathophysiological cascade involves endothelial-cell activation and damage, and a prothrombotic-hypofibrinolytic state. Severe VOD/SOS (typically characterized by multi-organ dysfunction) may be associated with >80% mortality. Defibrotide is approved for treating severe hepatic VOD/SOS post-HSCT in the European Union, and for hepatic VOD/SOS with renal or pulmonary dysfunction post-HSCT in the United States...
April 26, 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/28439877/increased-non-relapse-mortality-due-to-high-dose-cytarabine-plus-cy-tbi-in-bmt-pbsct-for-acute-lymphoblastic-leukaemia-in-adults
#12
Yasuyuki Arai, Tadakazu Kondo, Akio Shigematsu, Junji Tanaka, Kazuteru Ohashi, Takahiro Fukuda, Toshiro Kawakita, Takehiko Mori, Takumi Hoshino, Makoto Onizuka, Yukiyasu Ozawa, Shuro Yoshida, Yasunori Ueda, Ishikazu Mizuno, Yoshiko Atsuta, Shuichi Mizuta
The efficacy of high-dose cytarabine (HDCA) plus cyclophosphamide/total-body irradiation (CY/TBI) has been proved in cord blood transplantation (CBT) for acute lymphoblastic leukaemia (ALL), but not in bone marrow or peripheral blood stem cell transplantation (BMT/PBSCT). In this cohort study, we compared the prognosis of CY/TBI (N = 1244) and HDCA/CY/TBI (N = 316) regimens in BMT/PBSCT for ALL. The addition of HDCA decreased post-transplant relapse, while significantly increasing non-relapse mortality (risk ratio, 1·33), and overall survival was not improved...
April 25, 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/28436977/provision-of-long-term-monitoring-and-late-effects-services-following-adult-allogeneic-haematopoietic-stem-cell-transplant-a-survey-of-uk-nhs-based-programmes
#13
A Hamblin, D M Greenfield, M Gilleece, N Salooja, M Kenyon, E Morris, N Glover, P Miller, H Braund, A Peniket, B E Shaw, J A Snowden
Despite international guidelines, optimal delivery models of late effects (LE) services for HSCT patients are unclear from the clinical, organizational and economic viewpoints. To scope current LE service delivery models within the UK NHS (National Health Service), in 2014, we surveyed the 27 adult allogeneic HSCT centres using a 30-question online tool, achieving a 100% response rate. Most LE services were led and delivered by senior physicians (>80% centres). Follow-up was usually provided in a dedicated allograft or LE clinic for the first year (>90% centres), but thereafter attrition meant that only ~50% of patients were followed after 5 years...
April 24, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28436969/associations-between-levels-of-insulin-like-growth-factor-1-and-sinusoidal-obstruction-syndrome-after-allogeneic-haematopoietic-stem-cell-transplantation
#14
S Weischendorff, K Kielsen, H Sengeløv, K Jordan, C H Nielsen, A E Pedersen, L P Ryder, A Juul, K G Müller
Allogeneic myeloablative haematopoietic stem cell transplantation (HSCT) is challenged by severe adverse events, as cytotoxic effects of the conditioning may result in systemic inflammation, leaky epithelial barriers and organ toxicities, contributing to treatment-related morbidity and mortality. We hypothesised that insulin-like growth factor-1 (IGF-1), a mediator of growth and proliferation of various tissues, may attenuate chemotherapy-induced tissue damage after HSCT. We prospectively measured plasma levels of IGF-1 and its binding protein 3 (IGFBP-3) in 41 patients undergoing myeloablative HSCT...
April 24, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28429862/epidemiology-of-invasive-fungal-diseases-in-children-with-solid-tumours-undergoing-autologous-haematopoietic-stem-cell-transplantation-a-10-year-experience-in-a-tertiary-italian-centre
#15
Paola Tatarelli, Maura Faraci, Ilaria Caviglia, Roberto Bandettini, Giuliana Cangemi, Gian Michele Magnano, Alberto Garaventa, Claudio Viscoli, Elio Castagnola
The objective of the study was to determine the incidence of invasive fungal disease (IFD) in children undergoing autologous haematopoietic stem cell transplantation (auHSCT) for solid tumours (ST). Retrospective study on auHSCT was performed in children with ST (January 2006-December 2015). Data on the number of patient-days at risk (pdr) during the first 30 and 90 days after auHSCT and cases of proven/probable IFDs were collected. Infection rate (IR, episodes/1000 pdr) and proportions and cumulative risk (CR) of IFD were evaluated...
April 21, 2017: Mycoses
https://www.readbyqxmd.com/read/28418340/risk-adapted-management-of-febrile-neutrepenia-and-early-cessation-of-empirical-antibiotherapy-in-hematopoietic-stem-cell-transplantation-setting
#16
Ali Hakan Kaya, Emre Tekgündüz, Fazilet Duygu, Dicle Koca, Filiz Bekdemir, Hikmetullah Batgi, Bahar Ulu Uncu, Tuğçe Nur Yiğenoğlu, Mehmet Sinan Dal, Merih Çakar Kızıl, Fevzi Altuntaş
BACKGROUND: Haematopoietic stem cell transplantation is a curative treatment option for many haematological disorders. Infection following haematopoietic stem cell transplantation is one of the major causes of mortality. AIMS: To investigate the outcomes of early cessation of empirical antibiotic treatment per protocol in febrile neutropenia patients who have undergone haematopoietic stem cell transplantation at our clinic. STUDY DESIGN: Descriptive study...
April 5, 2017: Balkan Medical Journal
https://www.readbyqxmd.com/read/28410601/the-diagnosis-and-management-of-nk-t-cell-lymphomas
#17
REVIEW
Eric Tse, Yok-Lam Kwong
Extranodal natural killer (NK)/T-cell lymphoma is an aggressive malignancy of putative NK-cell origin, with a minority deriving from the T-cell lineage. Pathologically, the malignancy occurs in two forms, extranodal NK/T-cell lymphoma, nasal type; and aggressive NK-cell leukaemia. Lymphoma occur most commonly (80%) in the nose and upper aerodigestive tract, less commonly (20%) in non-nasal areas (skin, gastrointestinal tract, testis, salivary gland), and rarely as disseminated disease with a leukemic phase...
April 14, 2017: Journal of Hematology & Oncology
https://www.readbyqxmd.com/read/28404538/haematopoietic-stem-cell-transplantation-in-primary-immunodeficiency-patients-in-the-black-sea-region-of-turkey
#18
Alişan Yıldıran, Mehmet Halil Çeliksoy, Stephan Borte, Şükrü Nail Güner, Murat Elli, Tunç Fışgın, Emel Özyürek, Recep Sancak, Gönül Oğur
OBJECTIVE: Haematopoietic stem cell transplantation is a promising curative therapy for many combined primary immunodeficiencies and phagocytic disorders. MATERIALS AND METHODS: We retrospectively reviewed paediatric cases that were diagnosed with primary immunodeficiencies and scheduled for haematopoietic stem cell transplantation. RESULTS: We identified 22 patients (median age, 6 months; age range, 1 month to 10 years) with various diagnoses who received haematopoietic stem cell transplantation...
April 13, 2017: Turkish Journal of Haematology: Official Journal of Turkish Society of Haematology
https://www.readbyqxmd.com/read/28401678/a-and-b-antigen-levels-acquired-by-group-o-donor-derived-erythrocytes-following-abo-non-identical-transfusion-or-minor-abo-incompatible-haematopoietic-stem-cell-transplantation
#19
A K Hult, J H Dykes, J R Storry, M L Olsson
BACKGROUND AND OBJECTIVES: ABO-incompatible haematopoietic stem cell transplantation (HSCT) presents a challenge to blood component transfusion. The aim of this study was to investigate the weak blood group A or B antigen expression by donor-derived group O red blood cells (RBC) observed following transfusion or minor ABO-incompatible HSCT. In addition, in vitro experiments were performed to elucidate possible mechanisms underlying this phenomenon. MATERIALS AND METHODS: A sensitive flow cytometry assay for the semi-quantification of RBC A/B antigen levels was used to assess patient samples and evaluate in vitro experiments...
April 12, 2017: Transfusion Medicine
https://www.readbyqxmd.com/read/28394883/pdgf-signalling-guides-neural-crest-contribution-to-the-haematopoietic-stem-cell-specification-niche
#20
Erich W Damm, Wilson K Clements
Haematopoietic stem cells (HSCs) support maintenance of the haematopoietic and immune systems throughout the life of vertebrates, and are the therapeutic component of bone marrow transplants. Understanding native specification of HSCs, to uncover key signals that might help improve in vitro directed differentiation protocols, has been a long-standing biomedical goal. The current impossibility of specifying true HSCs in vitro suggests that key signals remain unknown. We speculated that such signals might be presented by surrounding 'niche' cells, but no such cells have been defined...
May 2017: Nature Cell Biology
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