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https://www.readbyqxmd.com/read/28914546/defibrotide-an-oligonucleotide-for-sinusoidal-obstruction-syndrome
#1
May T Aziz, Payal P Kakadiya, Samantha M Kush, Kylie Weigel, Denise K Lowe
OBJECTIVE: To review the efficacy and safety of defibrotide as well as its pharmacology, mechanism of action, pharmacokinetics (PK), drug-drug interactions, dosing, cost considerations, and place in therapy. DATA SOURCES: A PubMed search was performed through August 2017 using the terms defibrotide, oligonucleotide, hepatic veno-occlusive disease (VOD), sinusoidal obstruction syndrome (SOS), and hematopoietic cell transplantation (HCT). Other data sources were from references of identified studies, review articles, and conference abstracts plus manufacturer product labeling and website, the Food and Drug Administration website, and clinicaltrials...
September 1, 2017: Annals of Pharmacotherapy
https://www.readbyqxmd.com/read/28877973/heritable-pulmonary-hypertension-from-bench-to-bedside
#2
REVIEW
Barbara Girerd, Jason Weatherald, David Montani, Marc Humbert
Mutations in the BMPR2 gene, and more rarely in ACVRL1, endoglin, caveolin-1, KCNK3 and TBX4 genes predispose to heritable pulmonary arterial hypertension, an autosomal dominant disease with incomplete penetrance. Bi-allelic mutations in the EIF2AK4 gene predispose to heritable pulmonary veno-occlusive disease/pulmonary capillary haemangiomatosis, an autosomal recessive disease with an unknown penetrance.In France, the national pulmonary hypertension referral centre offers genetic counselling and testing to adults and children...
September 30, 2017: European Respiratory Review: An Official Journal of the European Respiratory Society
https://www.readbyqxmd.com/read/28870776/consensus-report-by-palisi-and-pbmtc-joint-working-committees-supportive-care-guidelines-for-management-of-vod-in-children-and-adolescents-part-3-focus-on-cardio-respiratory-dysfunction-infections-liver-dysfunction-and-delirium
#3
Nadia Ovchinsky, Warren Frazier, Jeffery J Auletta, Christopher C Dvorak, Monica Ardura, Enkyung Song, Jennifer McArthur, Asumthia Jeyapalan, Robert Tamburro, Kris M Mahadeo, Chani Traube, Christine N Duncan, Rajinder P S Bajwa
Some patients with veno-occlusive disease (VOD) have multi-organ dysfunction (MOD) and multiple teams are involved in their daily care in the pediatric intensive care units (PICU). Cardio-respiratory dysfunction is critical in these patients as it needs immediate action. Whether to use non-invasive or invasive ventilation strategy may be a difficult decision in the setting of mucositis or other comorbidities in patients with VOD. Similarly monitoring of their organ functions could be very challenging; they may have fulminant hepatic failure with or without hepatic encephalopathy complicated by delirium and/or infections...
September 1, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28754544/consensus-report-by-pediatric-acute-lung-injury-and-sepsis-investigators-and-pediatric-blood-and-marrow-transplantation-consortium-joint-working-committees-supportive-care-guidelines-for-management-of-veno-occlusive-disease-in-children-and-adolescents-part
#4
REVIEW
Rajinder P S Bajwa, Kris M Mahadeo, Benjamin H Taragin, Christopher C Dvorak, Jennifer McArthur, Asumthia Jeyapalan, Christine N Duncan, Robert Tamburro, Alison Gehred, Leslie Lehman, Paul Richardson, Jeffery J Auletta, Ann E Woolfrey
Veno-occlusive disease (VOD) is a common and potentially fatal complication in children undergoing hematopoietic cell transplantation (HCT). It occurs in about one-third of all patients undergoing transplantation and is fatal in 50% of patients with severe disease. Early intervention and specific treatment with defibrotide are associated with improved outcomes. However, there is a lack of supportive care guidelines for management of the multiorgan dysfunction seen in most cases. There is high variability in the management of VOD, which may contribute to the increased morbidity and mortality...
July 25, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28661905/genetics-of-pulmonary-hypertension-in-the-clinic
#5
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...
September 2017: Current Opinion in Pulmonary Medicine
https://www.readbyqxmd.com/read/28359910/cost-effectiveness-of-defibrotide-in-the-prophylaxis-of-veno-occlusive-disease-after-pediatric-allogeneic-stem-cell-transplantation
#6
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...
July 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/27770853/external-mechanical-devices-and-vascular-surgery-for-erectile-dysfunction
#7
REVIEW
Landon W Trost, Ricardo Munarriz, Run Wang, Allen Morey, Laurence Levine
INTRODUCTION: The field of sexual medicine is continuously advancing, with novel outcomes reported on a regular basis. Given the rapid evolution, updated guidelines are essential to inform practicing clinicians on best practices. AIM: To summarize the current literature and provide clinical guidelines on penile traction therapy, vacuum erection devices, and penile revascularization. METHODS: A consensus panel was held with leading sexual medicine experts during the 2015 International Consultation on Sexual Medicine (ICSM)...
November 2016: Journal of Sexual Medicine
https://www.readbyqxmd.com/read/27684876/eif2ak4-mutation-in-pulmonary-veno-occlusive-disease-a-case-report-and-review-of-the-literature
#8
REVIEW
Li Liang, Guofeng Ma, Kai Chen, Yangxiang Liu, Xiaohong Wu, Kejing Ying, Ruifeng Zhang
BACKGROUND: Pulmonary veno-occlusive disease (PVOD) is a rare and devastating cause of pulmonary arterial hypertension with a non-specific clinical presentation and a relatively specific presentation in high-resolution thoracic CT scan images. Definitive diagnosis is made by histological examination in previous. According to the 2015 ESC/ERS Guidelines, detection of a mutation in the eukaryotic translation initiation factor 2 alpha kinase 4 (EIF2AK4) without histological confirmation is recommended to validate the diagnosis of PVOD...
September 2016: Medicine (Baltimore)
https://www.readbyqxmd.com/read/26431626/hepatic-veno-occlusive-disease-after-hematopoietic-stem-cell-transplantation-risk-factors-and-stratification-prophylaxis-and-treatment
#9
REVIEW
Jean-Hugues Dalle, Sergio A Giralt
Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), can develop in a subset of patients, primarily after myeloablative hematopoietic stem cell transplantation, but it also may occur after reduced-intensity conditioning. Severe VOD/SOS, typically characterized by multiorgan failure, has been associated with a mortality rate greater than 80%. Therefore, an accurate and prompt diagnosis of VOD/SOS is essential for early initiation of appropriate therapy to improve clinical outcomes...
March 2016: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/26297369/protocol-for-updating-a-systematic-review-of-randomised-controlled-trials-on-the-prophylactic-use-of-intravenous-immunoglobulin-for-patients-undergoing-haematopoietic-stem-cell-transplantation
#10
Juthaporn Cowan, D W Cameron, Greg Knoll, Jason Tay
INTRODUCTION: Haematopoietic stem cell transplantation (HSCT) is commonly employed in the management of haematological malignancies. This intervention results in an increased risk of infectious and immune-related complications. Prophylactic immunoglobulin therapy has been used to prevent post-HSCT complications, including infections, with varying efficacy. We sought to update the current evidence supporting the use of immunoglobulins in the modern HSCT era. METHODS/ANALYSIS: Using a structured search strategy, we will perform a systematic review of the literature from MEDLINE, EMBASE and all EBM Reviews databases...
2015: BMJ Open
https://www.readbyqxmd.com/read/25735576/prospects-for-improving-outcomes-in-systemic-sclerosis-related-pulmonary-hypertension
#11
REVIEW
V Thakkar, M Nikpour, W M Stevens, S M Proudman
Pulmonary arterial hypertension (PAH) is a leading cause of morbidity and mortality in patients with systemic sclerosis (SSc). Approximately one in 10 will develop PAH during their lifetime. These patients have a worse prognosis than those with PAH due to other causes. The most common clinical feature of SSc-PAH in the early stages is non-specific exercise intolerance that can be erroneously attributed to other manifestations of SSc. Screening provides an opportunity for early identification of SSc-PAH and prompt initiation of therapies with the potential to improve quality of life and survival...
March 2015: Internal Medicine Journal
https://www.readbyqxmd.com/read/24933082/wilms-tumor-lessons-and-outcomes-a-25-year-single-center-uk-experience
#12
David W Fawkner-Corbett, Lisa Howell, Barry L Pizer, Carlo Dominici, Heather P McDowell, Paul D Losty
Wilms' tumor (WT) is a common childhood renal cancer. A 25-year single center UK experience is reported. During 1985-2010, 97 children underwent immediate nephrectomy or delayed resection of tumor after chemotherapy. Survival, morbidity, and late effects following treatment are described. Tumor distribution was: Stage I, 25.7% (n = 25); Stage II, 24.7% (n = 24); Stage III, 26.8% (n = 26); Stage IV, 17.5% (n = 17); and Stage V, 5.2% (n = 5). Immediate nephrectomy was performed in 39% (n = 38) patients with elective delayed resection in 61% (n = 59) cases...
August 2014: Pediatric Hematology and Oncology
https://www.readbyqxmd.com/read/24102514/bcsh-bsbmt-guideline-diagnosis-and-management-of-veno-occlusive-disease-sinusoidal-obstruction-syndrome-following-haematopoietic-stem-cell-transplantation
#13
Fiona L Dignan, Robert F Wynn, Nedim Hadzic, John Karani, Alberto Quaglia, Antonio Pagliuca, Paul Veys, Michael N Potter
DIAGNOSIS: It is recommended that the diagnosis of veno-occlusive disease (sinusoidal obstruction syndrome) [VOD (SOS)] be based primarily on established clinical criteria (modified Seattle or Baltimore criteria) (1A). Ultrasound imaging may be helpful in the exclusion of other disorders in patients with suspected VOD (SOS) (1C). It is recommended that liver biopsy be reserved for patients in whom the diagnosis of VOD (SOS) is unclear and there is a need to exclude other diagnoses (1C)...
November 2013: British Journal of Haematology
https://www.readbyqxmd.com/read/23171072/standard-operating-procedures-for-vascular-surgery-in-erectile-dysfunction-revascularization-and-venous-procedures
#14
REVIEW
Michael Sohn, Martin Hatzinger, Irwin Goldstein, Sudhakar Krishnamurti
INTRODUCTION: The impact of penile blood supply on erectile function was recognized some 500 years ago. At the turn of the 20th century first results of penile venous ligation were published and in 1973 the first surgical attempts to restore penile arterial inflow were undertaken. Numerous techniques were published in the meantime, but inclusion criteria, patient selection, and success evaluation differed extremely between study groups. AIM: To develop evidence-based standard operating procedures (SOPs) for vascular surgery in erectile dysfunction, based on recent state of the art consensus reports and recently published articles in peer-reviewed journals...
January 2013: Journal of Sexual Medicine
https://www.readbyqxmd.com/read/23100969/ring-chromosome-8-and-trisomy-8-in-a-patient-with-acute-myeloid-leukemia
#15
Bibhas Kar, B Nandhini, R Revathi
We describe a child with Acute Myeloid Leukemia (AML M7) with trisomy 8 and ring chromosome 8. Ring chromosome 8 associated with AML is uncommon and is reported to have a poor outcome. The combination of trisomy 8 and ring chromosome 8 has not been previously reported. This 15-month-old girl had presented with a history of fever, weight loss of 1 kg, gum bleeds and pallor. Clinical examinations revealed no nodes or organomegaly. Investigations revealed pancytopenia and elevated serum LDH. Bone marrow aspirate confirmed the presence of myeloid blasts positive only for CD 41 and CD 61 on flow cytometry...
March 2009: Indian Journal of Hematology & Blood Transfusion
https://www.readbyqxmd.com/read/22088969/ventilation-perfusion-lung-scan-in-pulmonary-veno-occlusive-disease
#16
Andrei Seferian, Badia Helal, Xavier Jaïs, Barbara Girerd, Laura C Price, Sven Günther, Laurent Savale, Peter Dorfmüller, Florence Parent, Olivier Sitbon, Marc Humbert, Gérald Simonneau, David Montani
Pulmonary veno-occlusive disease (PVOD), a rare form of pulmonary arterial hypertension (PAH), requires histological proof for definitive diagnosis; however, lung biopsy is not recommended in PAH. Recent conjoint European Respiratory Society/European Society of Cardiology guidelines suggest that nonmatched perfusion defects on ventilation/perfusion (V'/Q') lung scanning in PAH patients may suggest PVOD. The aim of our study was to evaluate V'/Q' lung scans in a large cohort of PVOD and idiopathic or heritable PAH patients...
July 2012: European Respiratory Journal: Official Journal of the European Society for Clinical Respiratory Physiology
https://www.readbyqxmd.com/read/22015568/2009-esc-ers-pulmonary-hypertension-guidelines-and-connective-tissue-disease
#17
REVIEW
Norifumi Nakanishi
Pulmonary hypertension was defined as mean pulmonary artery pressure ≥ 25 mmHg at the 4th World Symposium on Pulmonary Hypertension. In 2009, the European Society of Cardiology and European Respiratory Society jointly created guidelines for practical pulmonary hypertension classifications and treatments based on the discussions at the 4th World Symposium. This classification is characterized by division into five groups: Pulmonary arterial hypertension (PAH); Pulmonary hypertension due to left heart disease; Pulmonary hypertension due to lung disease and/or hypoxia; Chronic thromboembolic pulmonary hypertension; and Pulmonary hypertension with unclear and/or multifactorial mechanisms...
December 2011: Allergology International: Official Journal of the Japanese Society of Allergology
https://www.readbyqxmd.com/read/21699659/priapism-and-sickle-cell-anemia-diagnosis-and-nonsurgical-therapy
#18
REVIEW
Gregory A Broderick
INTRODUCTION: Priapism describes a persistent erection lasting longer than 4 hours. Ischemic priapism and stuttering priapism are phenotypic manifestations of sickle-cell disease (SCD). AIMS: To define the types of priapism associated with SCD, to address pathogenesis, and to recommend best practices. SOURCES: Literature review and published clinical guidelines. SUMMARY OF FINDINGS: Priapism is a full or partial erection that persists more than 4 hours...
January 2012: Journal of Sexual Medicine
https://www.readbyqxmd.com/read/21376432/-pulmonary-arterial-hypertension-in-systemic-lupus-erythematosus
#19
REVIEW
L Arnaud, C Agard, J Haroche, P Cacoub, J-C Piette, Z Amoura
Pulmonary hypertension (PH) is a serious complication of connective tissue diseases. The prevalence of PH in systemic lupus erythematosus (SLE) ranges from 0.5 to 17.5%, depending on whether echocardiography or right heart catheterization is used as the gold standard for diagnosis. The recent guidelines for the diagnosis and treatment of pulmonary hypertension include several potential causes of PH in SLE, including: a primary vasculopathy similar to idiopathic pulmonary arterial hypertension (PAH); left heart diseases; post-thromboembolic disease; hypoxia and fibrosis resulting from interstitial lung disease; and the infrequent SLE-associated pulmonary veno-occlusive disease...
November 2011: La Revue de Médecine Interne
https://www.readbyqxmd.com/read/21185794/vascular-disorders-of-the-liver-recommendations-from-the-italian-association-for-the-study-of-the-liver-aisf-ad-hoc-committee
#20
REVIEW
Marco Senzolo, Oliviero Riggio, Massimo Primignani
This review summarizes the document elaborated by the Italian Association for the Study of the Liver (AISF) ad hoc committee "Vascular disorders of the liver" on the primary circulatory liver diseases, which include Budd-Chiari syndrome, obstruction of the hepatic portion of the inferior vena cava, portal vein thrombosis, sinusoidal obstruction syndrome (veno-occlusive disease) and hereditary hemorrhagic telangiectasia. A characteristic of the primary circulatory liver diseases is that portal hypertension usually precedes liver dysfunction...
July 2011: Digestive and Liver Disease
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