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https://www.readbyqxmd.com/read/29767845/final-results-from-a-defibrotide-treatment-ind-study-for-patients-with-hepatic-veno-occlusive-disease-sinusoidal-obstruction-syndrome
#1
Nancy A Kernan, Stephan Grupp, Angela R Smith, Sally Arai, Brandon Triplett, Joseph H Antin, Leslie Lehmann, Tsiporah Shore, Vincent T Ho, Nancy Bunin, Massimo Iacobelli, Wei Liang, Robin Hume, William Tappe, Robert Soiffer, Paul Richardson
Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life-threatening complication of haematopoietic stem cell transplant (HSCT) conditioning and chemotherapy. Defibrotide is approved for treatment of hepatic VOD/SOS with pulmonary or renal dysfunction [i.e., multi-organ dysfunction (MOD)] after HSCT in the United States and severe VOD/SOS after HSCT in patients aged older than 1 month in the European Union. Defibrotide was available as an investigational drug by an expanded-access treatment programme (T-IND; NCT00628498)...
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29715079/sinusoidal-obstruction-syndrome-veno-occlusive-disease-following-hematopoietic-stem-cell-transplant-insights-and-therapeutic-advances
#2
REVIEW
Samah Nassereddine, Saud Alsubait, Imad Tabbara
Hepatic sinusoidal obstruction syndrome (SOS) is a rare fatal clinical entity seen following hematopoietic stem cell transplant (HSCT). It is more commonly reported to occur following allogeneic HSCT compared to autologous HSCT. Historically, it is known as hepatitis following HSCT. It is thought that endothelial damage to the hepatic venules leading to occlusion of the terminal hepatic venules and hepatic sinusoids is the trigger for the development of SOS. Several risk factors have been associated with this condition...
May 2018: Anticancer Research
https://www.readbyqxmd.com/read/29673920/-sinusoidal-obstruction-syndrome-after-beam-conditioning-regiment-for-autologous-stem-cell-transplantation-imputability-of-bendamustine-report-of-two-cases-and-literature-review
#3
A Meyer, L-M Fornecker, A Guffroy, A-S Korganow, M Martin
INTRODUCTION: Sinusoidal obstruction syndrome is a rare complication of autologous hematopoietic stem cell transplantation. This syndrome is mainly described following conditioning regiment with busulfan, cyclophosphamide and/or total body irradiation. CASE REPORTS: We report for the first time two cases of sinusoidal obstruction syndrome occurring lately after BeAM conditioning regiment (bendamustine, etoposide, aracytine, melphalan) for autologous stem cell transplantation in patients treated for malignant lymphoma...
April 16, 2018: La Revue de Médecine Interne
https://www.readbyqxmd.com/read/29579286/involvement-of-heparanase-in-the-pathogenesis-of-mesothelioma-basic-aspects-and-clinical-applications
#4
Uri Barash, Moshe Lapidot, Yaniv Zohar, Cynthia Loomis, Andre Moreira, Sari Feld, Chandra Goparaju, Haining Yang, Edward Hammond, Ganlin Zhang, Jin-Ping Li, Neta Ilan, Arnon Nagler, Harvey I Pass, Israel Vlodavsky
Background: Mammalian cells express a single functional heparanase, an endoglycosidase that cleaves heparan sulfate and thereby promotes tumor metastasis, angiogenesis, and inflammation. Malignant mesothelioma is highly aggressive and has a poor prognosis because of the lack of markers for early diagnosis and resistance to conventional therapies. The purpose of this study was to elucidate the mode of action and biological significance of heparanase in mesothelioma and test the efficacy of heparanase inhibitors in the treatment of this malignancy...
March 21, 2018: Journal of the National Cancer Institute
https://www.readbyqxmd.com/read/29537138/-pre-emptive-strike-the-case-for-early-treatment-of-hepatic-sinusoidal-obstruction-syndrome-with-defibrotide
#5
Revathi Rajagopal, Marianne Phillips, Nicholas G Gottardo
The initial signs of hepatic sinusoidal obstruction syndrome (HSOS) can be challenging to recognize in children, especially outside the hematopoietic stem cell transplantation setting. To assist clinicians to promptly identify HSOS, the European Society for Blood and Marrow Transplantation has proposed pediatric HSOS diagnostic criteria which emphasize unexplained consumptive and transfusion-refractory thrombocytopenia. To highlight the importance of these "bellwether" early signs of HSOS and the efficacy of pre-emptive treatment with defibrotide, we describe the case of a child with a right 11th rib primitive neuroectodermal tumor who developed HSOS following focal radiotherapy and actinomycin-D treatment...
March 14, 2018: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/29510235/treatment-of-antiphospholipid-syndrome-beyond-anticoagulation
#6
Chrisanna Dobrowolski, Doruk Erkan
Antiphospholipid syndrome (APS) is a systemic autoimmune disorder marked by thrombosis and/or pregnancy morbidity in the presence of antiphospholipid antibodies (aPL). At the present time, treatment is primarily focused on anticoagulation. However, there is increasing awareness of the mechanisms involved in APS pathogenesis, which has led to the trial of novel therapies targeting those mechanisms. Following a brief review of the etiopathogenesis of and current management strategies in APS, this paper focuses on the evidence for these potential, targeted APS treatments, e...
March 3, 2018: Clinical Immunology: the Official Journal of the Clinical Immunology Society
https://www.readbyqxmd.com/read/29477779/defibrotide-for-sinusoidal-obstruction-syndrome-veno-occlusive-disease-prophylaxis-in-high-risk-adult-patients-a-single-center-experience-study
#7
Adrien Picod, Agnès Bonnin, Giorgia Battipaglia, Federica Giannotti, Annalisa Ruggeri, Eolia Brissot, Florent Malard, Clémence Médiavilla, Ramdane Belhocine, Anne Vekhoff, Mor Sény Gueye, Simona Lapusan, Rosa Adaeva, Françoise Isnard, Ollivier Legrand, Minh-Tam Baylatry, Anne-Christine Joly, Myriam Labopin, Rémy Duléry, Mohamad Mohty
Sinusoidal obstruction syndrome (SOS), also known as hepatic veno-occlusive disease (VOD), is a serious complication after hematopoietic stem cell transplantation (HSCT). SOS/VOD usually occurs within 3 weeks of HSCT, but the 2016 European Society for Blood and Marrow Transplantation diagnosis criteria have been revised to include late forms. Prophylactic use of defibrotide is recommended in the pediatric setting, but its value remains uncertain in the adult population. We report here a single-center series of 63 adult patients considered at high risk for SOS/VOD who received defibrotide prophylaxis in combination with ursodeoxycholic acid between May 2012 and August 2016...
March 1, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29388370/the-role-of-continuous-renal-replacement-therapy-in-the-management-of-acute-kidney-injury-associated-with-sinusoidal-obstruction-syndrome-following-hematopoietic-cell-transplantation
#8
Rupesh Raina, Ghada A Abusin, Prashant Vijayaraghavan, Jeffery J Auletta, Linda Cabral, Hasan Hashem, Beth A Vogt, Kenneth R Cooke, Rolla F Abu-Arja
Maintaining fluid balance, pre- and post-MA-HCT is essential and usually requires frequent administration of diuretics. Hepatic sinusoidal obstructive syndrome is potentially life-threatening, especially when associated with AKI and MOF. This study describes six patients who developed AKI-associated SOS and diuretic-resistant FO who subsequently underwent CRRT using standardized management guidelines for fluid balance post-HCT. Retrospective chart review was done for HCT patients between September 2011 and October 2013 at a tertiary care children's hospital...
March 2018: Pediatric Transplantation
https://www.readbyqxmd.com/read/29350496/venoocclusive-disease-due-to-chemotherapy-for-pediatric-acute-lymphoblastic-leukemia-is-associated-with-increased-levels-of-plasminogen-activator-inhibitor-1
#9
Margherita Mauro, Graziella Saggiorato, Maria Teresa Sartori, Giuseppe Gallo, Massimiliano De Bortoli, Elisa Bonetti, Ada Zaccaron, Virginia Vitale, Rita Balter, Matteo Chinello, Simone Cesaro
We describe three cases of sinusoidal obstruction syndrome/venoocclusive disease (SOS) in pediatric patients with acute lymphoblastic leukemia (ALL). All three episodes occurred during or just after the induction or reinduction phase of treatment based on prednisone/dexamethasone, vincristine, daunorubicin, and pegylated-l-asparaginase. SOS episodes were categorized as mild/moderate and resolved in 7, 10, and 16 days using supportive measures or defibrotide therapy. In all three episodes, the clinical diagnosis of SOS was associated with a significant increase in plasminogen-activator inhibitor-1 (PAI-1) that reduced with patient clinical improvement...
January 19, 2018: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/29335623/sinusoidal-obstruction-syndrome-veno-occlusive-disease-after-high-dose-intravenous-busulfan-melphalan-conditioning-therapy-in-high-risk-ewing-sarcoma
#10
Massimo Eraldo Abate, Anna Paioli, Sivlia Cammelli, Marilena Cesari, Alessandra Longhi, Emanuela Palmerini, Stefano Ferrari, Elisa Carretta, Piero Picci, Fabio Piscaglia
This mono-institutional observational study was conducted to determine incidence, severity, risk factors, and outcome of sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) in high-risk Ewing sarcoma (ES) patients treated with intravenous busulfan and melphalan (BU-MEL) followed by autologous stem cell transplantation (ASCT). During the past 10 years, 75 consecutive ES patients resulted evaluable for the analysis. After diagnosis of SOS/VOD, defibrotide therapy was started as soon as the medication was available...
January 15, 2018: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/29333010/sinusoidal-obstruction-syndrome-during-treatment-for-wilms-tumor-a-life-threatening-complication
#11
Sidharth Totadri, Amita Trehan, Deepak Bansal, Richa Jain
Context: Survival rates exceed 90% in Wilms' tumor (WT). Actinomycin-D (ACT-D) which is indispensable in the management of WT is associated with the development of sinusoidal obstruction syndrome (SOS), a potentially fatal complication. Aims: The aim is to study the presentation, management, and outcome of SOS complicating ACT-D administration in WT. Settings and Design: Retrospective file review conducted in a Pediatric Hematology-Oncology unit...
October 2017: Indian Journal of Medical and Paediatric Oncology
https://www.readbyqxmd.com/read/29326798/late-onset-hepatic-veno-occlusive-disease-after-allografting-report-of-two-cases-with-atypical-clinical-features-successfully-treated-with-defibrotide
#12
Alessia Castellino, Stefano Guidi, Chiara Maria Dellacasa, Antonella Gozzini, Irene Donnini, Chiara Nozzoli, Sara Manetta, Semra Aydin, Luisa Giaccone, Moreno Festuccia, Lucia Brunello, Enrico Maffini, Benedetto Bruno, Ezio David, Alessandro Busca
Hepatic Veno-Occlusive Disease (VOD) is a potentially severe complication of hematopoietic stem cell transplantation (HSCT). Here we report two patients receiving an allogeneic HSCT who developed late onset VOD with atypical clinical features. The two patients presented with only few risk factors, namely, advanced acute leukemia, a myeloablative busulphan-containing regimen and received grafts from an unrelated donor. The first patient did not experience painful hepatomegaly and weight gain and both patients showed only a mild elevation in total serum bilirubin level...
2018: Mediterranean Journal of Hematology and Infectious Diseases
https://www.readbyqxmd.com/read/29304536/defibrotide-stimulates-angiogenesis-and-protects-endothelial-cells-from-calcineurin-inhibitor-induced-apoptosis-via-upregulation-of-akt-bcl-xl
#13
Xiangmin Wang, Bin Pan, Yuko Hashimoto, Hiroshi Ohkawara, Kailin Xu, Lingyu Zeng, Takayuki Ikezoe
Sinusoidal obstruction syndrome is a life-threatening complication that can occur after haematopoietic stem cell transplantation. Defibrotide (DF) has been approved for the treatment of individuals with severe sinusoidal obstruction syndrome following haematopoietic stem cell transplantation in the European Union and the United States. However, the precise mechanisms by which DF protects endothelial cells remain to be elucidated. In this study, we found that DF stimulated angiogenesis in vitro and in vivo as assessed by vascular tube formation, scratch-wound repair and Matrigel plug assays...
January 2018: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/29301447/defibrotide-sodium-for-the-treatment-of-hepatic-veno-occlusive-disease-sinusoidal-obstruction-syndrome
#14
REVIEW
Paul G Richardson, Brandon M Triplett, Vincent T Ho, Nelson Chao, Fiona L Dignan, Michelle Maglio, Mohamad Mohty
Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is an unpredictable condition associated with endothelial-cell damage due to conditioning for hematopoietic stem-cell transplantation (HSCT) or chemotherapy without HSCT. Mortality in patients with VOD/SOS and multi-organ dysfunction (MOD) may be >80%. Areas covered: Defibrotide is the only approved drug for the treatment of severe hepatic VOD/SOS after HSCT in the European Union and hepatic VOD/SOS with renal or pulmonary dysfunction in the United States...
February 2018: Expert Review of Clinical Pharmacology
https://www.readbyqxmd.com/read/29022099/defibrotide-for-the-treatment-of-sinusoidal-obstruction-syndrome-evaluation-of-response-to-therapy-and-patient-outcomes
#15
John Coutsouvelis, Sharon Avery, Michael Dooley, Carl Kirkpatrick, Andrew Spencer
PURPOSE: Defibrotide is an agent used to treat sinusoidal obstruction syndrome (SOS/VOD) in patients undergoing haemopoietic stem cell transplantation. The aim of this study was to evaluate the effectiveness of defibrotide used within institutional guidelines for the treatment of SOS/VOD in patients undergoing haemopoietic stem cell transplantation (HSCT). METHODS: Data for 23 patients was retrospectively reviewed to evaluate the effectiveness of defibrotide and the utility of response criteria to direct therapy as specified within institution guidelines...
March 2018: Supportive Care in Cancer: Official Journal of the Multinational Association of Supportive Care in Cancer
https://www.readbyqxmd.com/read/28993886/thrombotic-microangiopathy-following-haematopoietic-stem-cell-transplant
#16
Eleanor G Seaby, Rodney D Gilbert
Thrombotic microangiopathy is a potentially lethal complication of haematopoietic stem cell (bone marrow) transplantation. The pathophysiology is incompletely understood, although endothelial damage appears to be central. Platelet activation, neutrophil extracellular traps and complement activation appear to play key roles. Diagnosis may be difficult and universally accepted diagnostic criteria are not available. Treatment remains controversial. In some cases, withdrawal of calcineurin inhibitors is adequate...
October 9, 2017: Pediatric Nephrology: Journal of the International Pediatric Nephrology Association
https://www.readbyqxmd.com/read/28939456/combination-of-high-dose-methylprednisolone-and-defibrotide-for-veno-occlusive-disease-in-pediatric-hematopoietic-stem-cell-transplant-recipients
#17
Nicholas J Gloude, Sonata Jodele, Ashley Teusink-Cross, Michael Grimley, Stella M Davies, Adam Lane, Kasiani C Myers
Veno-occlusive disease (VOD) is a serious complication of hematopoietic stem cell transplant (HSCT), with high mortality in severe cases and until recently very limited therapeutic options consisting largely of supportive care. Defibrotide was recently approved in the United States for the treatment of severe VOD in patients with renal or pulmonary dysfunction after HSCT. Our group previously published on the use of high-dose methylprednisolone (500 mg/m2 per dose every 12 hours for 6 doses) in patients with VOD, showing good success...
January 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28914546/defibrotide-an-oligonucleotide-for-sinusoidal-obstruction-syndrome
#18
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...
February 2018: Annals of Pharmacotherapy
https://www.readbyqxmd.com/read/28860002/thrombotic-microangiopathy-after-allogeneic-stem-cell-transplantation-a-comparison-of-eculizumab-therapy-and-conventional-therapy
#19
Stephan R Bohl, Florian Kuchenbauer, Stefanie von Harsdorf, Nadine Kloevekorn, Stefan S Schönsteiner, Arefeh Rouhi, Phyllis Schwarzwälder, Hartmut Döhner, Donald Bunjes, Martin Bommer
We report the results of a single-center analysis of a cohort of 39 patients treated between 1997 and 2016 for transplantion-associated thrombotic microangiopathy. We evaluated 2 subgroups of patients: 24 patients treated between 1997 and 2014 who received conventional therapy and 15 patients treated with the complement-inhibiting monoclonal antibody eculizumab between 2014 and 2016. The conventional therapy group was treated predominantly with defibrotide alone or in combination with plasmapheresis or rituximab...
December 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28859028/sinusoidal-obstruction-syndrome-during-chemotherapy-of-pediatric-cancers-and-its-successful-management-with-defibrotide
#20
Hande Kizilocak, Gürcan Dikme, Nihal Özdemir, Sebuh Kuruğoğlu, İbrahim Adaletli, Tülay Erkan, Tiraje Celkan
Sinusoidal obstruction syndrome (SOS) is a life-threatening complication generally occurring after hematopoietic stem cell transplantation. SOS after standard dose chemotherapy in malignancies is rare. Between the year 1995 and 2016, 414 patients were diagnosed with acute lymphoblastic leukemia and 113 patients were diagnosed with Wilms tumor in our institution. Among these patients, 4 patients with acute lymphoblastic leukemia (0.96%) and 2 patients with Wilms tumor (1.7%) developed SOS during treatment. SOS behaves like a local disseminated intravascular coagulation...
October 2017: Journal of Pediatric Hematology/oncology
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