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Veno-occlusive disease

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https://www.readbyqxmd.com/read/28733263/consolidation-therapy-for-newly-diagnosed-pediatric-high-risk-neuroblastoma-patients-using-busulfan-melphalan-autologous-hematopoietic-cell-transplant-anti-gd2-antibody-gm-csf-il-2-and-haploidentical-nk-cells
#1
Aimee C Talleur, Brandon M Triplett, Sara Federico, Ewelina Mamcarz, William Janssen, Jianrong Wu, David Shook, Wing Leung, Wayne L Furman
The treatment of pediatric high-risk neuroblastoma is intensive and multimodal. Despite the introduction of immunotherapy for minimal residual disease, survival rates remain suboptimal and new therapies are needed. As part of a phase 2 trial, we are using a consolidation therapy regimen which combines a busulfan/melphalan conditioning schema, autologous hematopoietic cell transplant (AHCT), and experimental immunotherapy with hu14.18K322A (a humanized anti-GD2 monoclonal antibody), GM-CSF, and IL-2, with or without the adoptive transfer of haploidentical natural killer cells (NKs)...
July 18, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28729640/radiation-induced-liver-disease-current-understanding-and-future-perspectives
#2
REVIEW
Jieun Kim, Youngmi Jung
Although radiotherapy (RT) is used for the treatment of cancers, including liver cancer, radiation-induced liver disease (RILD) has emerged as a major limitation of RT. Radiation-induced toxicities in nontumorous liver tissues are associated with the development of numerous symptoms that may limit the course of therapy or have serious chronic side effects, including late fibrosis. Although the clinical characteristics of RILD patients have been relatively well described, the understanding of RILD pathogenesis has been hampered by a lack of reliable animal models for RILD...
July 21, 2017: Experimental & Molecular Medicine
https://www.readbyqxmd.com/read/28697925/variable-expressivity-of-a-founder-mutation-in-the-eif2ak4-gene-in-hereditary-pulmonary-veno-occlusive-disease-and-its-impact-on-survival
#3
Paula Navas Tejedor, Julián Palomino Doza, Jair Antonio Tenorio Castaño, Ana Belén Enguita Valls, José Julián Rodríguez Reguero, Amaya Martínez Meñaca, Ignacio Hernández González, Héctor Bueno Zamora, Pablo Daniel Lapunzina Badía, Pilar Escribano Subías
INTRODUCTION AND OBJECTIVES: Hereditary pulmonary veno-occlusive disease (PVOD) has been associated with biallelic mutations in EIF2AK4 with the recent discovery of a founder mutation in Iberian Romani patients with familial PVOD. The aims of this study were phenotypical characterization and survival analysis of Iberian Romani patients with familial PVOD carrying the founder p.Pro1115Leu mutation in EIF2AK4, according to their tolerance to pulmonary vasodilators (PVD). Familial genetic screening was conducted, as well as assessment of sociocultural determinants with a potential influence on disease course...
July 8, 2017: Revista Española de Cardiología
https://www.readbyqxmd.com/read/28687420/hepatic-adverse-event-profile-of-inotuzumab-ozogamicin-in-adult-patients-with-relapsed-or-refractory-acute-lymphoblastic-leukaemia-results-from-the-open-label-randomised-phase-3-ino-vate-study
#4
Hagop M Kantarjian, Daniel J DeAngelo, Anjali S Advani, Matthias Stelljes, Partow Kebriaei, Ryan D Cassaday, Akil A Merchant, Naohito Fujishima, Toshiki Uchida, Maria Calbacho, Anna A Ejduk, Susan M O'Brien, Elias J Jabbour, Hui Zhang, Barbara J Sleight, Erik R Vandendries, David I Marks
BACKGROUND: The INO-VATE study demonstrated efficacy and safety of inotuzumab ozogamicin versus standard care in adults with relapsed or refractory B-cell acute lymphoblastic leukaemia. Here, we report the frequency of, and potential risk factors for, hepatotoxicity in patients in this trial and after treatment and subsequent haemopoietic stem-cell transplantation (HSCT). METHODS: In this open-label, phase 3, multicentre, international study, adults with relapsed or refractory, CD22-positive, Philadelphia chromosome (Ph)-positive or Ph-negative B-cell acute lymphoblastic leukaemia who were due to receive first or second salvage treatment were randomly assigned (1:1) via an interactive voice response system to receive inotuzumab ozogamicin (starting dose 1·8 mg/m(2) per cycle [0·8 mg/m(2) on day 1; 0·5 mg/m(2) on days 8 and 15 of a 21-28 day cycle for ≤6 cycles]) or standard care (either fludarabine plus cytarabine plus granulocyte colony-stimulating factor, mitoxantrone plus cytarabine, or high-dose cytarabine)...
July 4, 2017: Lancet Haematology
https://www.readbyqxmd.com/read/28668489/pulmonary-clinicopathological-correlation-following-allogeneic-hematopoietic-stem-cell-transplantation-an-autopsy-series
#5
Lee Gazourian, Laura Spring, Emily Meserve, David Hwang, Alejandro A Diaz, Samuel Y Ash, Vincent T Ho, Lynette M Sholl, George R Washko
Pulmonary complications are a significant cause of morbidity, mortality and resource utilization following Hematopoietic Stem Cell Transplantation (HSCT). The objective of this study was to compare ante mortem clinical suspicion of pulmonary complications and postmortem findings in a modern HSCT cohort. All patients who underwent allogeneic HSCT at our institution (n=1854) between January 1, 2000 and June 30th 2010 were reviewed and patients who died of any cause greater than one year following HSCT and had an unrestricted autopsy available for analysis...
June 28, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28661905/genetics-of-pulmonary-hypertension-in-the-clinic
#6
Barbara Girerd, Edmund Lau, David Montani, Marc Humbert
PURPOSE OF REVIEW: Heritable pulmonary arterial hypertension (PAH) is an autosomal dominant disease with incomplete penetrance because of mutations in bone morphogenetic protein receptor-II (BMPR2), activin A receptor type II-like kinase 1, endoglin, caveolin-1, potassium channel subfamily K, member 3, and T-box gene 4 genes. Heritable pulmonary veno-occlusive disease and/or pulmonary capillary hemangiomatosis (PVOD/PCH) is an autosomal recessive disease because of biallelic mutations in the eukaryotic translation initiation factor 2 alpha kinase 4 gene...
June 28, 2017: Current Opinion in Pulmonary Medicine
https://www.readbyqxmd.com/read/28644135/evaluation-of-thromboelastometry-parameters-as-predictive-markers-for-sinusoidal-obstruction-syndrome-in-patients-undergoing-allogeneic-stem-cell-transplantation-for-acute-leukaemia
#7
Joanna Rupa-Matysek, Lidia Gil, Ewelina Wojtasińska, Zuzanna Kanduła, Adam Nowicki, Magdalena Matuszak, Mieczysław Komarnicki
Hepatic sinusoidal obstruction syndrome (previously named veno-occlusive disease, SOS/VOD) is a serious complication of allogeneic stem cell transplantation (HSCT). Early identification of patients at risk of SOS/VOD may possibly improve the outcome and reduce mortality. Rotation thromboelastometry (ROTEM) is global assay allowing for the precise assessment of both bleeding and thrombotic conditions, however, its usefulness in patients undergoing HSCT for acute leukaemia has not been studied.We evaluated the thromboelastometry parameters in patients undergoing allogeneic HSCT for acute leukaemia to identify candidate biomarkers of SOS/VOD occurrence...
June 15, 2017: Oncotarget
https://www.readbyqxmd.com/read/28592769/thrombotic-complications-after-hematopoietic-stem-cell-transplantation
#8
Shosaku Nomura
Hematopoietic stem cell transplantation (HSCT) involves some serious transplant-associated complications (TACs) or vascular disorders, such as veno-occlusive disease (VOD), thrombotic microangiopathy (TMA), and graft-versus-host disease (GVHD). VOD is related to a clinical syndrome characterized by tender hepatomegaly, jaundice, fluid retention, and unexplained weight gain. When TMA is described in patients who have undergone HSCT, it is often implied that the clinical diagnosis of TMA is similar to that of thrombotic thrombocytopenic purpura...
2017: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://www.readbyqxmd.com/read/28562132/burden-of-illness-associated-with-sinusoidal-obstruction-syndrome-veno-occlusive-disease-in-patients-with-hematopoietic-stem-cell-transplantation
#9
Zhun Cao, Kathleen F Villa, Craig B Lipkin, Scott B Robinson, Bijan Nejadnik, Christopher C Dvorak
AIMS: Sinusoidal obstruction syndrome (SOS) is a life-threatening complication of hematopoietic stem cell transplantation (HSCT) associated with significant morbidity and mortality. Healthcare utilization, costs, and mortality were assessed in HSCT patients diagnosed with SOS, with and without multi-organ dysfunction (MOD). MATERIALS AND METHODS: This retrospective observational study identified real-world patients undergoing HSCT between January 1, 2009 and May 31, 2014 using the Premier Healthcare Database...
June 14, 2017: Journal of Medical Economics
https://www.readbyqxmd.com/read/28460209/low-fixed-dose-defibrotide-in-management-of-hepatic-veno-occlusive-disease-post-stem-cell-transplantation
#10
Bhausaheb Bagal, Arun Chandrasekharan, Aliya Chougle, Navin Khattry
OBJECTIVE/BACKGROUND: Hepatic veno-occlusive disease (VOD) is well recognized potentially serious regimen-related toxicity seen after stem cell transplantation. Severe VOD is associated with poor long-term outcomes with very high mortality. Besides supportive care, only defibrotide has been found to be effective in the management of VOD. The recommended dose of defibrotide is 25mg/kg/d but there has been no classical dose finding study done for this drug. A higher dose of defibrotide is associated with increased risk of bleeding and this drug is prohibitively expensive...
April 25, 2017: Hematology/oncology and Stem Cell Therapy
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/28430541/a-comprehensive-approach-to-hepatic-vascular-disease
#12
Khaled M Elsayes, Akram M Shaaban, Sarah M Rothan, Sanaz Javadi, Beatrice L Madrazo, Rosa P Castillo, Victor J Casillas, Christine O Menias
The liver has a complex vascular supply, which involves the inflow of oxygenated blood through the hepatic artery (systemic circulation) and deoxygenated blood through the portal vein (portal circulation), as well as the outflow of deoxygenated blood through the hepatic veins to the inferior vena cava. A spectrum of vascular variants can involve the liver. Some of these variants may result in areas of enhancement that can mimic more serious pathologic conditions. In this article, the authors discuss a spectrum of variants and pathologic conditions that may involve the liver vasculature...
May 2017: Radiographics: a Review Publication of the Radiological Society of North America, Inc
https://www.readbyqxmd.com/read/28413774/a-diffuse-lung-emphysema-severe-pulmonary-hypertension-and-lack-of-airflow-limitation
#13
Morgane Faure, Emmanuel Gomez, Peter Dorfmüller, Damien Mandry, Matthieu Canuet, Romain Kessler, François Chabot, Ari Chaouat
Pulmonary veno-occlusive disease is characterized by remodeling of pulmonary arteries, capillaries and venules. We report a case of diffuse lung emphysema and pulmonary veno-occlusive disease with the characteristic of having no airflow limitation. A very low diffusing capacity for carbon monoxide and results of high-resolution computed tomography of the chest suggested pulmonary veno-occlusive disease. The diagnosis was confirmed on histological analysis after lung transplantation. The combination of results of the computed tomography of the chest and the histological analysis suggested a relationship between diffuse lung emphysema and remodeling of pulmonary vessels...
2017: Respiratory Medicine Case Reports
https://www.readbyqxmd.com/read/28398284/manifestations-and-management-of-veno-occlusive-disease-sinusoidal-obstruction-syndrome-in-the-era-of-contemporary-therapies
#14
REVIEW
Priti Tewari, Whitney Wallis, Partow Kebriaei
The concept of veno-occlusive disease (VOD), along with our understanding of it, has historically been and remains an evolving phenomenon. This review presents a broad view of VOD, also known as sinusoidal obstruction syndrome (SOS), including (1) traditional hematopoietic stem cell transplant-associated VOD/SOS, (2) late-onset VOD/SOS, (3) pulmonary VOD, and (4) VOD/SOS associated with chemotherapy only. Several VOD/SOS management modalities exist that include modes for both prophylaxis and treatment. An extensive review of the literature on monoclonal antibodies, both approved and pending approval by the US Food and Drug Administration, reveals that only a few have been associated with an increased risk for VOD/SOS...
February 2017: Clinical Advances in Hematology & Oncology: H&O
https://www.readbyqxmd.com/read/28379594/pyrrolizidine-alkaloids-enhance-alcohol-induced-hepatocytotoxicity-in-vitro-in-normal-human-hepatocytes
#15
M G Neuman, L B Cohen, V Steenkamp
OBJECTIVE: Herbal remedies containing pyrrilidozine alkaloids (PA)s can induce liver damage, including hepato-sinusoidal obstruction syndrome (HSOS) or veno-occlusive liver disease (VOD). Some individuals misusing alcohol consume also teas and/or herbal remedies containing PA. The interaction or additive toxicity of alcohol to PA toxicity needs to be addressed. The objectives of this study are 1) to review the scientific literature on the PA-induced liver toxicity; 2) identify possible mechanism(s) involved in PA-induced hepatocytotoxicity in the presence or absence of ethanol (EtOH) in vitro in normal human hepatocytes (NHH) in primary culture...
March 2017: European Review for Medical and Pharmacological Sciences
https://www.readbyqxmd.com/read/28368376/predictors-of-overall-survival-among-patients-treated-with-sirolimus-tacrolimus-vs-methotrexate-tacrolimus-for-gvhd-prevention
#16
F Khimani, J Kim, L Chen, E Dean, V Rizk, B Betts, T Nishihori, F Locke, A Mishra, L Perez, E Ayala, M Kharfan-Dabaja, M Nieder, H Fernandez, C Anasetti, J Pidala
Sirolimus (SIR)/tacrolimus (TAC) is an alternative to methotrexate (MTX)/TAC. However, rational selection among these GvHD prophylaxis approaches to optimize survival of individual patients is not possible based on current evidence. We compared SIR/TAC (n=293) to MTX/TAC (n=414). The primary objective was to identify unique predictors of overall survival (OS). Secondary objective was to compare acute and chronic GvHD, relapse, non-relapse mortality, thrombotic microangiopathy (TMA), hepatic veno-occlusive disease (VOD/SOS), and acute kidney injury...
April 3, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28359910/cost-effectiveness-of-defibrotide-in-the-prophylaxis-of-veno-occlusive-disease-after-pediatric-allogeneic-stem-cell-transplantation
#17
Herbert Pichler, Karolina Horner, Gernot Engstler, Ulrike Poetschger, Evgenia Glogova, Susanne Karlhuber, Manuel Martin, Werner Eibler, Volker Witt, Wolfgang Holter, Susanne Matthes-Martin
Veno-occlusive disease (VOD) remains a serious complication after allogeneic hematopoietic stem cell transplantation (HSCT). Prophylactic use of defibrotide (DF) might further reduce VOD rates but has no impact on the incidence of severe VOD or VOD-associated mortality. We investigated the cost-effectiveness of prophylactic DF according to the British Committee for Standards in Haematology/British Society for Blood and Marrow Transplantation guidelines in 348 children who underwent transplantation between 2001 and 2014 in our hospital, 138 of whom were at risk for VOD...
March 27, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28355983/mesenteric-inflammatory-veno-occlusive-disease-of-the-spleen-metasynchronous-with-two-arterial-thrombotic-events-in-systemic-lupus-erythematosus
#18
D Allali, G Puppa, C Chizzolini
Vasculitides, particularly those affecting small vessels, are known to complicate systemic lupus erythematosus (SLE); however, isolated venulitis of the mesenteric bed has rarely been reported. Here we relate the case of a 46-year-old woman with SLE who presented with acute abdominal pain due to artery thrombosis and extended splenic ischemia requiring splenectomy. The histological examination revealed diffuse venulitis in the absence of arterial vasculitis consistent with the definition of mesenteric inflammatory veno-occlusive disease (MIVOD)...
January 1, 2017: Lupus
https://www.readbyqxmd.com/read/28353604/a-case-report-of-pvod-patient-combined-with-pulmonary-embolism-anticoagulation-or-not
#19
Xiaoling Yuan, Xianghe Hou, Weihong Guo, Haiming Jiang, Junmeng Zheng, Stuti Paudyal, Yanhua Lyu
RATIONALE: Pulmonary veno-occlusive disease (PVOD) is a rare form of pulmonary arterial hypertension (PAH). Oral anticoagulation is confined to patients with idiopathic PAH (IPAH), but no oral anticoagulation has been recommended for PVOD, because occult pulmonary hemorrhage was a common finding in PVOD. PATIENT CONCERNS: We report a case of PVOD, who was misdiagnosed as IPAH for 5 years with worsening dyspnea and two episodes of pulmonary embolism (PE). DIAGNOSES: He was confirmed as PVOD combined with PE by biopsy of the explanted lung specimen...
March 2017: Medicine (Baltimore)
https://www.readbyqxmd.com/read/28329410/long-term-outcome-after-a-treosulfan-based-conditioning-regimen-for-patients-with-acute-myeloid-leukemia-a-report-from-the-acute-leukemia-working-party-of-the-european-society-for-blood-and-marrow-transplantation
#20
Arnon Nagler, Myriam Labopin, Dietrich Beelen, Fabio Ciceri, Liisa Volin, Avichai Shimoni, Roberto Foá, Noel Milpied, Jacopo Peccatori, Emmanuelle Polge, Audrey Mailhol, Mohamad Mohty, Bipin N Savani
BACKGROUND: Allogeneic hematopoietic cell transplantation (HCT) is a curative therapy for patients with acute myeloid leukemia (AML). However, post-HCT relapse and regimen-related toxicity remain significant barriers to long-term survival. In recent years, new conditioning regimens have been explored to improve transplantation outcomes in patients with AML. Treosulfan combines a potent immunosuppressive and antileukemic effect with a low toxicity profile. METHODS: To investigate the role of treosulfan-based conditioning, the European Society for Blood and Marrow Transplantation Acute Leukemia Working Party performed a registry analysis of 520 adult patients with AML who received treosulfan-based conditioning and underwent HCT between 2000 and 2012, including 225 patients in first complete remission, 107 in second or later complete remission, and 188 with active/advanced disease 188 (88 with primary refractory disease)...
March 22, 2017: Cancer
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