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Defibrotide

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https://www.readbyqxmd.com/read/28939456/combination-of-high-dose-methylprednisolone-and-defibrotide-for-veno-occlusive-disease-in-pediatric-hematopoietic-stem-cell-transplant-recipients
#1
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 following HSCT. Our group previously published on the use of high-dose methylprednisolone (500 mg/m(2)/dose every 12 hours for 6 doses) in patients with VOD showing good success...
September 19, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28914546/defibrotide-an-oligonucleotide-for-sinusoidal-obstruction-syndrome
#2
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/28860002/thrombotic-microangiopathy-after-allogeneic-stem-cell-transplantation-a-comparison-of-eculizumab-therapy-and-conventional-therapy
#3
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 two subgroups of patients: 24 patients treated between 1997 and 2014 who received conventional therapy and 15 patients who were treated with the complement-inhibiting monoclonal antibody eculizumab between 2014 and 2016. The conventional therapy group was predominantly treated with defibrotide alone or in combination with plasmapheresis or rituximab...
August 28, 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
#4
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...
August 30, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28842141/defibrotide-use-in-vincristine-induced-hepatic-sinusoidal-obstruction-syndrome
#5
Catherine Tang, Julian Lindsay, Anthony Gill, Ian Kerridge
No abstract text is available yet for this article.
August 2017: Clinical Lymphoma, Myeloma & Leukemia
https://www.readbyqxmd.com/read/28825848/defibrotide-for-children-and-adults-with-hepatic-veno-occlusive-disease-post-hematopoietic-cell-transplantation
#6
Selim Corbacioglu, Paul G Richardson
Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a complication that is typically associated with conditioning for hematopoietic stem cell transplantation (HSCT). In patients with concomitant multi-organ dysfunction, mortality may be >80%. Recently, the European Society for Blood and Marrow Transplantation established separate criteria for diagnosis and severity of VOD/SOS for adults and children, to better reflect current understanding of the disease. Areas covered: This review provides an overview of post-HSCT hepatic VOD/SOS and defibrotide, including its pharmacological, clinical, and regulatory profile...
September 4, 2017: Expert Review of Gastroenterology & Hepatology
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
#7
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/28715896/use-of-abdominal-ultrasound-in-the-diagnosis-of-hepatic-sinusoidal-obstruction-syndrome-sos-in-a-patient-receiving-a-hematopoietic-cell-transplant
#8
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
#9
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
#10
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
#11
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
#12
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...
July 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
#13
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/28285079/defibrotide-for-patients-with-hepatic-veno-occlusive-disease-sinusoidal-obstruction-syndrome-interim-results-from-a-treatment-ind-study
#14
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...
June 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
#15
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
#16
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
#17
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
#18
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
#19
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
#20
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...
May 2017: Journal of Medical Economics
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