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Defibrotide

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https://www.readbyqxmd.com/read/28715896/use-of-abdominal-ultrasound-in-the-diagnosis-of-hepatic-sinusoidal-obstruction-syndrome-sos-in-a-patient-receiving-a-hematopoietic-cell-transplant
#1
Marta Abadía Barnó, Eva Marín Serrano, Gloria Ruiz Fernández
The sinusoidal obstruction syndrome (SOS) is an uncommon form of portal hypertension that occurs in hematopoietic-cell transplant recipients who receive intense conditioning treatments. The diagnosis is clinical and it is usually delayed, because in these patients there are many causes that can damage the liver and can delay its detection. We present the case of a 47-year-old man, whose diagnosis was made with clinical and analytical data supported by ultrasound signs that showed hepatic congestion. After treatment with defibrotide the patient improved clinically and analytically...
July 18, 2017: Revista Española de Enfermedades Digestivas
https://www.readbyqxmd.com/read/28504490/additive-effect-of-mesenchymal-stem-cells-and-defibrotide-in-an-arterial-rat-thrombosis-model
#2
Dilek Dilli, Emine Kılıç, Nihat Yumuşak, Serdar Beken, Duygu Uçkan Çetinkaya, Ramazan Karabulut, Ayşegu L Zenciroğlu
BACKGROUND/AIM: In this study, we aimed to investigate the additive effect of mesenchymal stem cells (MSC) and defibrotide (DFT) in a rat model of femoral arterial thrombosis. METHODS: Thirty Sprague Dawley rats were included. An arterial thrombosis model by ferric chloride (FeCl3) was developed in the left femoral artery. The rats were equally assigned to 5 groups: Group 1-Sham-operated (without arterial injury); Group 2-Phosphate buffered saline (PBS) injected; Group 3-MSC; Group 4-DFT; Group 5-MSC + DFT...
June 1, 2017: Archivos Argentinos de Pediatría
https://www.readbyqxmd.com/read/28491036/pharmacological-activity-and-clinical-use-of-pdrn
#3
REVIEW
Francesco Squadrito, Alessandra Bitto, Natasha Irrera, Gabriele Pizzino, Giovanni Pallio, Letteria Minutoli, Domenica Altavilla
PDRN is a proprietary and registered drug that possesses several activities: tissue repairing, anti-ischemic, and anti-inflammatory. These therapeutic properties suggest its use in regenerative medicine and in diabetic foot ulcers. PDRN holds a mixture of deoxyribonucleotides with molecular weights ranging between 50 and 1,500 KDa, it is derived from a controlled purification and sterilization process of Oncorhynchus mykiss (Salmon Trout) or Oncorhynchus keta (Chum Salmon) sperm DNA. The procedure guarantees the absence of active protein and peptides that may cause immune reactions...
2017: Frontiers in Pharmacology
https://www.readbyqxmd.com/read/28460209/low-fixed-dose-defibrotide-in-management-of-hepatic-veno-occlusive-disease-post-stem-cell-transplantation
#4
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
#5
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/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...
March 27, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28285079/defibrotide-for-patients-with-hepatic-veno-occlusive-disease-sinusoidal-obstruction-syndrome-interim-results-from-a-treatment-ind-study
#7
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 treating hepatic VOD/SOS with renal or pulmonary dysfunction post-HSCT and in the European Union, Israel, and South Korea for treating severe hepatic VOD/SOS post-HSCT...
March 8, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28205194/novel-and-expanded-oncology-drug-approvals-of-2016-part-2-new-options-in-the-management-of-hematologic-malignancies
#8
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/28115278/the-role-of-thromboelastography-in-pediatric-patients-with-sinusoidal-obstructive-syndrome-receiving-defibrotide
#9
Joanna L Gendreau, Christine Knoll, Roberta H Adams, Leon L Su
Sinusoidal obstructive syndrome (SOS) is a potentially fatal form of hepatic injury after hematopoietic stem cell transplantation. Patients can develop liver dysfunction, portal hypertension, ascites, coagulopathies, and multisystem organ failure. The mortality rate of severe SOS has been reported as high as 98% by day 100 after transplantation. Defibrotide, which is now approved for the treatment of SOS, has significantly decreased mortality. Defibrotide is a polynucleotide with profibrinolytic, anti-ischemic, and anti-inflammatory activity...
April 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28099080/catastrophic-antiphospholipid-syndrome-candidate-therapies-for-a-potentially-lethal-disease
#10
Ozan Unlu, Doruk Erkan
Catastrophic antiphospholipid syndrome (CAPS) is a potentially lethal disease that presents with rapidly progressive multiple organ thromboses. Anticoagulation, corticosteroids, intravenous immunoglobulin, and plasma exchange are the most commonly used treatments for CAPS patients. However, the high mortality despite these medications necessitates new treatment strategies. Following a brief review of current diagnostic and management strategies, we discuss the candidate therapies, i.e., hydroxychloroquine, rituximab, eculizumab, sirolimus, and defibrotide, that can be considered in CAPS patients refractory to traditional treatment...
January 14, 2017: Annual Review of Medicine
https://www.readbyqxmd.com/read/28092357/risk-of-sinusoidal-obstruction-syndrome-in-allogeneic-stem-cell-transplantation-after-prior-gemtuzumab-ozogamicin-treatment-a-retrospective-study-from-the-acute-leukemia-working-party-of-the-ebmt
#11
G Battipaglia, M Labopin, A Candoni, R Fanin, J El Cheikh, D Blaise, M Michallet, A Ruggeri, N Contentin, J M Ribera, M Stadler, J Sierra, P A von dem Borne, A Bloor, G Socié, A Nagler, M Mohty
Gemtuzumab ozogamicin (GO) may increase the risk of sinusoidal obstruction syndrome (SOS) when used prior to allogeneic stem cell transplantation (HSCT). We assessed SOS incidence and outcomes after HSCT of 146 adults, with a median age of 50 years, previously receiving GO. SOS prophylaxis was used in 69 patients (heparin n=57, ursodeoxycholic acid n=8, defibrotide n=4). Cumulative incidence (CI) of SOS was 8% (n=11), with death in 3 patients. Median interval between last GO dose and HSCT was 130 days. Overall survival (OS) and SOS incidence did not differ for patients receiving GO ⩽3...
April 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28092354/use-of-defibrotide-to-treat-transplant-associated-thrombotic-microangiopathy-a-retrospective-study-of-the-paediatric-diseases-and-inborn-errors-working-parties-of-the-european-society-of-blood-and-marrow-transplantation
#12
L Yeates, M A Slatter, S Bonanomi, F L W I Lim, S Y Ong, A Dalissier, W Barberi, A Shulz, M Duval, C Heilmann, A Willekens, W H Y Hwang, C Uderzo, P Bader, A R Gennery
No abstract text is available yet for this article.
January 16, 2017: Bone Marrow Transplantation
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
#13
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/27939616/defibrotide-in-severe-sinusoidal-obstruction-syndrome-medicine-and-economic-issues
#14
Julie Steelandt, François Bocquet, Anne-Laure Cordonnier, Charlotte De Courtivron, Isabelle Fusier, Pascal Paubel
In Europe, Defitelio (defibrotide) has a Market Authorization in curative treatment of severe sinusoidal obstruction syndrome (SOS) but not in prophylaxis (2013). In France, defibrotide has had a compassionate-use program since 2009. Today, the high cost of defibrotide remains a major hurdle for hospital budgets. Medicine and economic issues were evaluated for the 39 hospitals of the French Public Assistance-Hospitals of Paris (AP-HP). We analyzed literature reviews, consumption, and expenditures through AP-HP data in 2014 and patient profiles with defibrotide in the corresponding diagnostic-related groups (DRGs) and consulted a board of hematologists...
February 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/27928191/defibrotide
#15
Danial E Baker, Kendra Demaris
Each month, subscribers to The Formulary Monograph Service receive 5 to 6 well-documented monographs on drugs that are newly released or are in late phase 3 trials. The monographs are targeted to Pharmacy & Therapeutics Committees. Subscribers also receive monthly 1-page summary monographs on agents that are useful for agendas and pharmacy/nursing in-services. A comprehensive target drug utilization evaluation/medication use evaluation (DUE/MUE) is also provided each month. With a subscription, the monographs are are available online to subscribers...
November 2016: Hospital Pharmacy
https://www.readbyqxmd.com/read/27892944/veno-occlusive-disease-sinusoidal-obstruction-syndrome-after-haematopoietic-stem-cell-transplantation-middle-east-north-africa-regional-consensus-on-prevention-diagnosis-and-management
#16
A H Al Jefri, H Abujazar, A Al-Ahmari, A Al Rawas, Z Al Zahrani, A Alhejazi, M A Bekadja, A Ibrahim, M Lahoucine, S Ousia, A Bazarbachi
Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) of the liver is a serious, early complication of haematopoietic stem cell transplantation (HSCT), severe and very severe forms of which are associated with a high mortality rate. A wide variety of patient, disease and treatment-related risk factors for VOD/SOS have been identified. Several bodies have published recommendations for the diagnosis, prevention and management of VOD/SOS following HSCT. A group of regional experts have developed a consensus statement on the diagnosis, prevention and management of VOD/SOS in the Middle East and North Africa region to help in the management of HSCT patients in the region...
April 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/27870480/pharmacokinetics-and-safety-of-defibrotide-in-healthy-japanese-subjects
#17
Kazuo Umemura, Takayuki Iwaki, Toshimi Kimura, Chitose Ogawa, Takahiro Fukuda, Shuichi Taniguchi, Keizo Horibe, Hiroaki Goto, Kenichi Yoshimura, Yasutaka Watanabe, Chika Nitani, Atsushi Kikuta
No abstract text is available yet for this article.
November 2016: Clinical Pharmacology in Drug Development
https://www.readbyqxmd.com/read/27843363/defibrotide-in-the-treatment-of-hepatic-veno-occlusive-disease
#18
REVIEW
Alessandro Fulgenzi, Maria Elena Ferrero
Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), represents the most frequent complication in patients in early phase following hematopoietic stem-cell transplantation (HSCT). In its severe form, VOD/SOS can be associated with multiorgan failure and with a mortality rate >80% by day +100. Defibrotide (DF) (a mixture of 90% single-stranded phosphodiester oligonucleotides and 10% double-stranded phosphodiester oligonucleotides derived from controlled depolarization of porcine intestinal mucosal DNA) has been proposed for the treatment of SOS due to its ability to restore thrombo-fibrinolytic balance and protect endothelial cells...
2016: Hepatic Medicine: Evidence and Research
https://www.readbyqxmd.com/read/27721142/defibrotide-for-the-management-of-sinusoidal-obstruction-syndrome-in-patients-who-undergo-haemopoietic-stem-cell-transplantation
#19
REVIEW
John Coutsouvelis, Sharon Avery, Michael Dooley, Carl Kirkpatrick, Andrew Spencer
Sinusoidal obstruction syndrome, previously known as veno-occlusive disease (VOD/SOS), is a complication in patients undergoing haemopoietic stem cell transplantation (HSCT). Severe VOD/SOS, including progression to multi-organ failure, has resulted in a mortality of greater than 80%. Defibrotide's varying pharmacological actions, particularly on endothelial cells, make it is a useful agent to consider for prophylaxis and treatment of VOD/SOS. Barriers to its routine use include the high acquisition cost and the fact that neither the oral or parenteral formulations are licensed products in many countries at this time...
November 2016: Cancer Treatment Reviews
https://www.readbyqxmd.com/read/27603963/in-brief-defibrotide-defitelio-for-hepatic-veno-occlusive-disease
#20
(no author information available yet)
No abstract text is available yet for this article.
September 12, 2016: Medical Letter on Drugs and Therapeutics
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