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Veno-occlusive disease

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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
#1
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
https://www.readbyqxmd.com/read/28315488/sorafenib-as-a-potential-strategy-for-refractory-pulmonary-arterial-hypertension
#2
Gou Kimura, Masaharu Kataoka, Takumi Inami, Keiichi Fukuda, Hideaki Yoshino, Toru Satoh
Sorafenib is an inhibitor of multi-kinases including tyrosine and serine/threonine kinases. We investigated the efficacy and safety of sorafenib for the treatment of patients with refractory pulmonary arterial hypertension (PAH). Sorafenib was started in 9 patients (7 with idiopathic PAH, 2 with pulmonary veno-occlusive disease) who had severe PAH and right heart failure, in spite of treatment with vasodilators specific for PAH. Sorafenib was started as an add-on therapy at a dose of 50 or 100 mg/day, and increased to 100-400 mg/day...
March 14, 2017: Pulmonary Pharmacology & Therapeutics
https://www.readbyqxmd.com/read/28288949/evaluation-of-the-pharmacokinetics-and-efficacy-of-a-busulfan-test-dose-in-adult-patients-undergoing-myeloablative-hematopoietic-cell-transplant
#3
Elizabeth Weil, Felicia Zook, Carolyn Oxencis, Angela Canadeo, Angela Urmanski, Mindy Waggoner, Daniel Eastwood, Marcelo Pasquini, Mehdi Hamadani, Parameswaran Hari
Due to inter-patient variability in busulfan exposure, therapeutic monitoring of busulfan is often employed in myeloablative allogeneic transplantation to ensure that patients are near the optimal steady state goal of 900 ng/mL. One challenge with therapeutic monitoring of busulfan is the brief course of treatment busulfan is administered over, so prompt analysis and dose adjustments are necessary. Pharmacokinetic evaluation of a busulfan test dose prior to beginning the conditioning regimen would allow for all conditioning regimen doses to be given at the calculated optimized dose...
March 10, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28285079/defibrotide-for-patients-with-hepatic-veno-occlusive-disease-sinusoidal-obstruction-syndrome-vod-sos-interim-results-from-a-treatment-ind-study
#4
Paul G Richardson, Angela R Smith, Brandon M Triplett, Nancy A Kernan, Stephan A Grupp, Joseph H Antin, Leslie Lehmann, Tsiporah Shore, Massimo Iacobelli, Maja Miloslavsky, Robin Hume, Alison L Hannah, Bijan Nejadnik, Robert J Soiffer
Hepatic veno-occlusive disease, or sinusoidal obstruction syndrome (VOD/SOS), is a serious and potentially fatal complication of conditioning for hematopoietic stem cell transplantation (HSCT) or of chemotherapy regimens alone. Defibrotide is a complex mixture of single-stranded polydeoxyribonucleotides that is approved in the United States for treatment of hepatic VOD/SOS with renal or pulmonary dysfunction post-HSCT and in the European Union, Israel, and South Korea for treatment of severe hepatic VOD/SOS post-HSCT...
March 8, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28259608/busulfan-and-melphalan-versus-carboplatin-etoposide-and-melphalan-as-high-dose-chemotherapy-for-high-risk-neuroblastoma-hr-nbl1-siopen-an-international-randomised-multi-arm-open-label-phase-3-trial
#5
Ruth Ladenstein, Ulrike Pötschger, Andrew D J Pearson, Penelope Brock, Roberto Luksch, Victoria Castel, Isaac Yaniv, Vassilios Papadakis, Geneviève Laureys, Josef Malis, Walentyna Balwierz, Ellen Ruud, Per Kogner, Henrik Schroeder, Ana Forjaz de Lacerda, Maja Beck-Popovic, Pavel Bician, Miklós Garami, Toby Trahair, Adela Canete, Peter F Ambros, Keith Holmes, Mark Gaze, Günter Schreier, Alberto Garaventa, Gilles Vassal, Jean Michon, Dominique Valteau-Couanet
BACKGROUND: High-dose chemotherapy with haemopoietic stem-cell rescue improves event-free survival in patients with high-risk neuroblastoma; however, which regimen has the greatest patient benefit has not been established. We aimed to assess event-free survival after high-dose chemotherapy with busulfan and melphalan compared with carboplatin, etoposide, and melphalan. METHODS: We did an international, randomised, multi-arm, open-label, phase 3 cooperative group clinical trial of patients with high-risk neuroblastoma at 128 institutions in 18 countries that included an open-label randomised arm in which high-dose chemotherapy regimens were compared...
March 1, 2017: Lancet Oncology
https://www.readbyqxmd.com/read/28256569/aggressive-non-alcoholic-steatohepatitis-following-rapid-weight-loss-and-or-malnutrition
#6
Jia-Huei Tsai, Linda D Ferrell, Vivian Tan, Matthew M Yeh, Monika Sarkar, Ryan M Gill
While non-alcoholic steatohepatitis is a slowly progressive disease, patients may rarely present in acute liver failure. We describe six patients who developed severe hepatic dysfunction following rapid weight loss or malnutrition. Rapid weight loss (18 to 91 kg) occurred after Roux-en-Y gastric bypass in four patients and starvation-like dieting or hypoalbuminemia was noted in two patients. Four patients either died or received an urgent liver transplant. Pathologic findings were characterized by advanced alcoholic steatohepatitis-like features, including extensive/circumferential centrizonal pericellular fibrosis, central scar with perivenular sclerosis/veno-occlusion with superimposed hepatocellular dropout, abundant/prominent hepatocellular balloons, and numerous Mallory-Denk bodies, but there was no history of excess alcohol consumption...
March 3, 2017: Modern Pathology: An Official Journal of the United States and Canadian Academy of Pathology, Inc
https://www.readbyqxmd.com/read/28244636/ascitic-fluid-drainage-using-a-peritoneal-dialysis-catheter-to-prevent-and-treat-multi-organ-dysfunction-in-veno-occlusive-disease-in-children-undergoing-hematopoietic-stem-cell-transplantation
#7
Vijal Parmar, Malcolm Lewis, Mohan Shenoy, Denise Bonney, Robert Wynn
Veno-occlusive disease (VOD), or sinusoidal obstruction syndrome, is a well-recognised, serious complication associated with the chemotherapy conditioning therapy used in hematopoietic stem cell transplantation (HSCT). Fluid management is typically challenging in children with this condition. We describe effective early use of peritoneal dialysis catheters to drain extravascular, intra-abdominal fluid in children with VOD, allowing intravascular fluid administration to preserve renal perfusion and function, preventing multi-organ dysfunction...
February 28, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28211168/the-medtronic-micro-vascular-plug%C3%A2-for-vascular-embolization-in-children-with-congenital-heart-diseases
#8
Shyam Sathanandam, Henri Justino, B Rush Waller, Srinath T Gowda, Wolfgang Radtke, Athar M Qureshi
OBJECTIVES: To describe the early multi-center, clinical experience with the Medtronic Micro Vascular Plug™ (MVP) in children with congenital heart disease (CHD) undergoing vascular embolization. BACKGROUND: The MVP is a large diameter vascular occlusion device that can be delivered through a microcatheter for embolization of abnormal blood vessels. METHODS: A retrospective review of embolization procedures using the MVP in children with CHD was performed in 3-centers...
February 16, 2017: Journal of Interventional Cardiology
https://www.readbyqxmd.com/read/28205194/novel-and-expanded-oncology-drug-approvals-of-2016-part-2-new-options-in-the-management-of-hematologic-malignancies
#9
REVIEW
Todd C Knepper, James Saller, Christine M Walko
The recent past has brought pharmacotherapeutic advances that benefit patients with hematologic malignancies. In 2016, two novel hematology drugs were approved and four previously approved hematology drugs were granted expanded use for the treatment of appropriate patient populations by the US Food and Drug Administration. These new approvals and indications represent significant steps forward in patient management: they include the first-in-class B-cell lymphoma 2 inhibitor, venetoclax, the newest targeted therapy available for the treatment of hematologic malignancies; and nivolumab, the first immune checkpoint inhibitor to be approved for treatment of a hematologic malignancy...
February 15, 2017: Oncology (Williston Park, NY)
https://www.readbyqxmd.com/read/28182036/acute-kidney-injury-and-mortality-in-hematopoietic-stem-cell-transplantation-a-single-center-experience
#10
B Sehgal, P George, M J John, C Samuel
Hematopoietic stem cell transplant (HSCT) is a life-saving procedure for patients with several malignant and nonmalignant hematological disorders. Acute kidney injury (AKI) is a common complication after HSCT. The aim of the study was to identify the incidence and outcomes of AKI associated with HSCT in our center. Sixty-six HSCT recipients from October 2008 to March 2014 at Christian Medical College, Ludhiana, were followed up till July 31, 2014. RIFLE criteria utilizing serum creatinine was used to diagnose and stage AKI...
January 2017: Indian Journal of Nephrology
https://www.readbyqxmd.com/read/28128441/restrictive-versus-liberal-red-blood-cell-transfusion-strategies-for-people-with-haematological-malignancies-treated-with-intensive-chemotherapy-or-radiotherapy-or-both-with-or-without-haematopoietic-stem-cell-support
#11
REVIEW
Lise J Estcourt, Reem Malouf, Marialena Trivella, Dean A Fergusson, Sally Hopewell, Michael F Murphy
BACKGROUND: Many people diagnosed with haematological malignancies experience anaemia, and red blood cell (RBC) transfusion plays an essential supportive role in their management. Different strategies have been developed for RBC transfusions. A restrictive transfusion strategy seeks to maintain a lower haemoglobin level (usually between 70 g/L to 90 g/L) with a trigger for transfusion when the haemoglobin drops below 70 g/L), whereas a liberal transfusion strategy aims to maintain a higher haemoglobin (usually between 100 g/L to 120 g/L, with a threshold for transfusion when haemoglobin drops below 100 g/L)...
January 27, 2017: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/28121342/clinical-applications-of-haploidentical-hematopoietic-stem-cell-transplantation-in-severe-aplastic-anemia
#12
P Zhang, K Feng, Y Xue, C-X Zhang, Y Wang, X-L Li
OBJECTIVE: The purpose of this study was to investigate the efficacy and safety of haploidentical allogeneic hemopoietic stem cell transplantation (allo-HSCT) in severe aplastic anemia (SAA) and prophylaxis of complications involved. PATIENTS AND METHODS: 8 patients with clinically diagnosed SAA (5 cases of SAA-I and 3 cases of SAA-II) were recruited, with the parents as the donors of hemopoietic stem cells. The conditioning regimen before HSCT included cyclophosphamide, fludarabine, pig anti-human lymphocyte immune globulin (p-ALG) and/or total body irradiation (TBI)...
January 2017: European Review for Medical and Pharmacological Sciences
https://www.readbyqxmd.com/read/28118962/pulmonary-veno-occlusive-disease-and-pulmonary-capillary-hemangiomatosis
#13
REVIEW
Carlos Ortiz-Bautista, Ignacio Hernández-González, Pilar Escribano-Subías
Pulmonary veno-occlusive disease is a rare cause of pulmonary hypertension which is part, together with pulmonary capillary hemangiomatosis, of the special designation (subgroup 1') within pulmonary hypertension group 1 in the latest classification of the pulmonary hypertension World Symposium. Recent discovery that gene mutations in eukaryotic translation initiation factor 2 alpha kinase 4 (EIF2AK4) are responsible for inherited forms of pulmonary veno-occlusive disease has changed the role of genetic testing, acquiring relevant importance in the diagnosis of these patients...
January 21, 2017: Medicina Clínica
https://www.readbyqxmd.com/read/28087362/clinical-phenotypes-and-outcomes-of-heritable-and-sporadic-pulmonary-veno-occlusive-disease-a-population-based-study
#14
David Montani, Barbara Girerd, Xavier Jaïs, Marilyne Levy, David Amar, Laurent Savale, Peter Dorfmüller, Andrei Seferian, Edmund M Lau, Mélanie Eyries, Jérôme Le Pavec, Florence Parent, Damien Bonnet, Florent Soubrier, Elie Fadel, Olivier Sitbon, Gérald Simonneau, Marc Humbert
BACKGROUND: Bi-allelic mutations of the EIF2AK4 gene cause heritable pulmonary veno-occlusive disease and/or pulmonary capillary haemangiomatosis (PVOD/PCH). We aimed to assess the effect of EIF2AK4 mutations on the clinical phenotypes and outcomes of PVOD/PCH. METHODS: We did a population-based study using clinical, functional, and haemodynamic data from the registry of the French Pulmonary Hypertension Network. We reviewed the clinical data and outcomes from all patients referred to the French Referral Centre (Pulmonary Department, Hospital Kremlin-Bicêtre, University Paris-Sud) with either confirmed or highly probable PVOD/PCH with DNA available for mutation screening (excluding patients with other risk factors of pulmonary hypertension, such as chronic respiratory diseases)...
February 2017: Lancet Respiratory Medicine
https://www.readbyqxmd.com/read/28087361/pulmonary-veno-occlusive-disease-characterising-a-rare-but-important-disease
#15
Charaka Hadinnapola, Nicholas W Morrell
No abstract text is available yet for this article.
February 2017: Lancet Respiratory Medicine
https://www.readbyqxmd.com/read/28070482/pulmonary-veno-occlusive-disease-two-children-with-gradual-disease-progression
#16
Ronald W Day, Parker W Clement, Aimee O Hersh, Susan M Connors, Kelli L Sumner, D Hunter Best, Mouied Alashari
Pulmonary veno-occlusive disease and pulmonary capillary hemangiomatosis are rare forms of pulmonary vascular disease. We report two cases of affected children who had evidence of pulmonary hypertension 3-5 years before developing radiographic findings of pulmonary veno-occlusive disease or pulmonary capillary hemangiomatosis. Both patients experienced a moderate decrease in pulmonary arterial pressure during acute vasodilator testing. Both patients experienced an improvement in six-minute walk performance without an increase in pulmonary edema when treated with targeted therapy for pulmonary hypertension...
2017: Respiratory Medicine Case Reports
https://www.readbyqxmd.com/read/28050475/role-of-hrct-chest-in-post-stem-cell-transplant-recipients-suspected-of-pulmonary-complications
#17
Abhishek Dwivedi, R Ravi Kumar, Ajay Sharma, S K Pannu
INTRODUCTION: Stem cell transplantation is today's procedure of choice for management of various hematopoietic malignant and severe immunogenic disorders. High Resolution Computed Tomography (HRCT) is a common technique for the diagnosis of pulmonary complications in stem cell transplant recipients. There are a large number of complications which can complicate the post-transplant period. AIM: To study the role of HRCT chest in stem cell transplant patients developing pulmonary complications, detect any evidence of infection, detect clinical signs of lung infections, Graft versus Host Disease (GvHD) or other regimen related toxicities outlined earlier, detect any evidence of GvHD and correlate these clinical signs with radiological changes in the lungs...
November 2016: Journal of Clinical and Diagnostic Research: JCDR
https://www.readbyqxmd.com/read/28049229/tandem-high-dose-chemotherapy-and-autologous-stem-cell-transplantation-for-high-grade-gliomas-in-children-and-adolescents
#18
Ji Won Lee, Do Hoon Lim, Ki Woong Sung, Hyeong Jin Lee, Eun Sang Yi, Keon Hee Yoo, Hong Hoe Koo, Yeon Lim Suh, Hyung Jin Shin
With the aim to investigate the outcome of tandem high-dose chemotherapy and autologous stem cell transplantation (HDCT/auto-SCT) for high-grade gliomas (HGGs), we retrospectively reviewed the medical records of 30 patients with HGGs (16 glioblastomas, 7 anaplastic astrocytomas, and 7 other HGGs) between 2006 and 2015. Gross or near total resection was possible in 11 patients. Front-line treatment after surgery was radiotherapy (RT) in 14 patients and chemotherapy in the remaining 16 patients including 3 patients less than 3 years of age...
February 2017: Journal of Korean Medical Science
https://www.readbyqxmd.com/read/28008770/the-budget-impact-and-cost-effectiveness-of-defibrotide-for-treatment-of-veno-occlusive-disease-with-multi-organ-dysfunction-in-patients-post-hematopoietic-stem-cell-transplant
#19
David L Veenstra, Gregory F Guzauskas, Kathleen F Villa, Denise M Boudreau
BACKGROUND: A Phase-3 study of defibrotide compared with historical controls demonstrated a 23% improvement in 100-day survival post-hematopoietic stem cell transplantation (HSCT) among patients with veno-occlusive disease with multi-organ dysfunction (VOD with MOD). AIM: To estimate the budget impact and cost-effectiveness of introducing defibrotide to a transplant center. METHODS: The authors developed a budget impact model from the perspective of a bone-marrow transplant center...
January 7, 2017: Journal of Medical Economics
https://www.readbyqxmd.com/read/27992075/management-of-veno-occlusive-disease-the-multidisciplinary-approach-to-care
#20
REVIEW
Elisabeth Wallhult, Michelle Kenyon, Sarah Liptrott, Arno Mank, Mairéad Ní Chonghaile, Aleksandra Babic, Jacobine Bijkerk, Caroline Bompoint, Selim Corbacioglu, Roel de Weijer, Claudia Fink, Sarah Marktel, Vivek Soni, Sarah Sprenger, Eugenia Trigoso Arjona, Mohamad Mohty
Although it is considered a relatively rare disorder, veno-occlusive disease (VOD) is one of the main causes of overall, non-relapse mortality associated with haematopoietic stem cell transplantation (HSCT). This article, based on the consensus opinion of haemato-oncology nurses, haemato-oncologists and pharmacists from both adult and paediatric services at the VOD International Multi-Disciplinary Advisory Board at the European Society for Blood and Marrow Transplantation (EBMT) meeting, Istanbul, 2015, aims to explore the multidisciplinary approach to care for the management of VOD, with an emphasis on current challenges in this area...
December 19, 2016: European Journal of Haematology
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