The Experts below are selected from a list of 318 Experts worldwide ranked by ideXlab platform

Paul G. Richardson - One of the best experts on this subject based on the ideXlab platform.

  • Recent developments with defibrotide for the treatment of Hepatic Veno-Occlusive Disease/sinusoidal obstruction syndrome
    Expert Opinion on Orphan Drugs, 2019
    Co-Authors: Christine Duncan, Justine M. Kahn, Stephan A. Grupp, Paul G. Richardson
    Abstract:

    ABSTRACTIntroduction: Hepatic Veno-Occlusive Disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life-threatening condition associated with endothelial cell damage due to hematopoiet...

  • Defibrotide sodium for the treatment of Hepatic Veno-Occlusive Disease/sinusoidal obstruction syndrome.
    Expert review of clinical pharmacology, 2018
    Co-Authors: Paul G. Richardson, Brandon M. Triplett, Nelson J. Chao, Fiona L. Dignan, Michelle E. Maglio, Mohamad Mohty
    Abstract:

    ABSTRACTIntroduction: 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. Its efficacy in patients with VOD/SOS with MOD post-HSCT was demonstrated in a clinical-trial program that included a historically controlled treatment study, a phase 2 trial, and a large T-IND expanded-access program that also included patients without MOD and who received chemotherapy without HSCT.Expert commentary: Defibrotide appears to protect endothelial cells and restore the thrombolytic–fibrinolytic balance. It addresses a significant clinical need and ...

  • Defibrotide for children and adults with Hepatic Veno-Occlusive Disease post hematopoietic cell transplantation.
    Expert review of gastroenterology & hepatology, 2017
    Co-Authors: Selim Corbacioglu, Paul G. Richardson
    Abstract:

    ABSTRACTIntroduction: 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. In children and adults following HSCT, defibrotide is approved for the treatment of Hepatic VOD/SOS with concomitant renal or pulmonary dysfunction in the United States and for the treatment of severe Hepatic VOD/SOS in the European Union. Day +100 survival rates with defibrotide are superior to those of historical controls receiving best supportive care...

  • Defibrotide for the treatment of Hepatic Veno-Occlusive Disease/sinusoidal obstruction syndrome with multiorgan failure.
    International journal of hematologic oncology, 2017
    Co-Authors: Paul G. Richardson, Stephan A. Grupp, Antonio Pagliuca, Amrita Krishnan, Selim Corbacioglu
    Abstract:

    Hepatic Veno-Occlusive Disease, also called sinusoidal obstruction syndrome (VOD/SOS), is a potentially life-threatening and unpredictable complication of hematopoietic stem cell transplantation (HSCT). Characterized by a prothrombotic-hypofibrinolytic state, VOD/SOS typically presents with hyperbilirubinemia, ascites, weight gain and painful hepatomegaly; VOD/SOS with multiorgan failure may be associated with >80% mortality. Treatment has been mainly supportive. However, defibrotide is now approved in the USA for treatment of Hepatic VOD/SOS with renal or pulmonary dysfunction following HSCT and in the European Union for treatment of severe Hepatic VOD/SOS post-HSCT. In vitro evidence suggests defibrotide may restore thrombotic-fibrinolytic balance at the endothelial level and protect endothelial cells. Defibrotide has demonstrated significant reduction in VOD/SOS-related mortality and resolved VOD/SOS-related symptoms, with a manageable safety profile.

  • Safety and efficacy of defibrotide for the treatment of severe Hepatic Veno-Occlusive Disease
    Therapeutic advances in hematology, 2012
    Co-Authors: Paul G. Richardson, Sally Arai, Sergio Giralt, Shin Mineishi, Corey Cutler, Joseph H. Antin, Nicole Stavitzski, Dietger Niederwieser, Ernst Holler
    Abstract:

    Hepatic Veno-Occlusive Disease (VOD), also known as sinusoidal obstruction syndrome, is a potentially life-threatening complication of chemotherapeutic conditioning used in preparation for hematopoietic stem-cell transplantation (SCT). VOD may occur in up to 62% of patients undergoing SCT, with onset generally within the first month after SCT. In severe cases, 100-day mortality is in excess of 80%. Current management consists of best supportive care, with no agents to date approved for treatment in the USA or the EU. Defibrotide, a polydisperse oligonucleotide, has been shown in phase II and III trials to improve complete response and survival in patients undergoing SCT with severe VOD. This article reviews our current understanding of VOD, and examines recent clinical findings on defibrotide for the treatment and prophylaxis of VOD.

Ayalew Tefferi - One of the best experts on this subject based on the ideXlab platform.

  • Hepatic Veno-Occlusive Disease (Sinusoidal Obstruction Syndrome) After Hematopoietic Stem Cell Transplantation
    Mayo Clinic proceedings, 2003
    Co-Authors: Shaji Kumar, Laurie D. Deleve, Patrick S. Kamath, Ayalew Tefferi
    Abstract:

    Hepatic Veno-Occlusive Disease (VOD), increasingly referred to as sinusoidal obstruction syndrome, is a well-recognized complication of hematopoietic stem cell transplantation and contributes to considerable morbidity and mortality. In the Western Hemisphere, VOD, classified as a conditioning-related toxicity, is most commonly caused by stem cell transplantation. VOD has been described after all types of stem cell transplantation, irrespective of the stem cell source, type of conditioning therapy, or underlying Disease. Recognition of this Disease in the posttransplantation setting remains a challenge in the absence of specific diagnostic features because many other more com-mon conditions can mimic it. Limited therapeutic or preventive strategies are currently available for the management of VOD. In this review, we provide a comprehensive account of the pathophysiology of this Disease as we understand it today, risk factors for its development, and the current state of knowledge regarding preventive and therapeutic options.

  • Development of Hepatic Veno-Occlusive Disease after Mylotarg infusion for relapsed acute myeloid leukemia.
    Bone marrow transplantation, 2001
    Co-Authors: Dk Tack, Louis Letendre, Ps Kamath, Ayalew Tefferi
    Abstract:

    Mylotarg (gemtuzumab zogamicin) is a conjugated monoclonal antibody that has recently become available for use in patients with relapsing or refractory acute myeloid leukemia. Reversible hepatotoxicity is common after administration. We describe the first report of Hepatic Veno-Occlusive Disease (HVOD) developing after Mylotarg infusion in a patient who underwent hematopoietic stem cell transplantation 8 months earlier. Certain antineoplastic agents have been implicated as a cause of HVOD, but the Disease is most commonly seen within 30 days after hematopoietic stem cell transplantation. The possible association between Mylotarg infusion and HVOD is discussed.

Lori S Muffly - One of the best experts on this subject based on the ideXlab platform.

Atul C. Mehta - One of the best experts on this subject based on the ideXlab platform.

  • Hepatic Veno-Occlusive Disease due to tacrolimus in a single-lung transplant patient
    The European respiratory journal, 2006
    Co-Authors: Sonia S. Shah, M. Budev, Holli Blazey, K. Fairbanks, Atul C. Mehta
    Abstract:

    Hepatic Veno-Occlusive Disease is defined as nonthrombotic fibrous obliterative endophlebitis of small centrilobular Hepatic venules. Clinically, patients present with elevated liver enzymes and a triad of jaundice, hepatomegaly and ascites. Although reported as a complication of other solid organ and stem cell transplantation, there have been no reported cases to date of Veno-Occlusive Disease following lung transplantation. The present authors report a case of Veno-Occlusive Disease following single-lung transplantation in a patient on a triple-drug immunosuppressive regimen composed of tacrolimus, mycophenolate mofetil and prednisone. The diagnosis was established by transjugular liver biopsy and by discontinuing tacrolimus; there was clinical regression of symptoms and serological return to baseline.

Riccardo Riccardi - One of the best experts on this subject based on the ideXlab platform.

  • Hepatic Veno-Occlusive Disease
    Pediatric Drugs, 2010
    Co-Authors: Maria Giuseppina Cefalo, Palma Maurizi, Annalisa Arlotta, Maria Scalzone, Giorgio Attinà, Antonio Ruggiero, Riccardo Riccardi
    Abstract:

    Hepatic Veno-Occlusive Disease (VOD) is a major manifestation of liver toxicity associated with conventional and high-dose chemotherapy in children affected by hematologic malignancies and certain solid tumors. Clinically, patients present with jaundice, painful hepatomegaly, and fluid retention, which may evolve into multi-organ failure, a hallmark of severe Disease. The pathogenesis is complex and not completely understood, but the damage to sinusoidal endothelium, typically caused by toxic metabolites released from antineoplastic drugs, is thought to play a crucial role, together with cytokine activation, immune deregulation, and coagulopathy. Diagnosis is based on clinical criteria supported by characteristic ultrasound findings, with the gold standard investigation being Hepatic-venous pressure gradient measurement and biopsy. Several treatment options have been tested; the most convincing approach to date is the use of defibrotide, a novel oligonucleotide with antithrombotic and antiplatelet aggregating properties, as well as endothelial-stabilizing effects. This agent, together with other specific forms of supportive care, has shown efficacy in the treatment of established VOD and promising results in the prevention of VOD in pediatric patients receiving chemotherapy.

  • Hepatic Veno-Occlusive Disease: a chemotherapy-related toxicity in children with malignancies
    Paediatric drugs, 2010
    Co-Authors: Maria Giuseppina Cefalo, Palma Maurizi, Annalisa Arlotta, Maria Scalzone, Giorgio Attinà, Antonio Ruggiero, Riccardo Riccardi
    Abstract:

    Hepatic Veno-Occlusive Disease (VOD) is a major manifestation of liver toxicity associated with conventional and high-dose chemotherapy in children affected by hematologic malignancies and certain solid tumors. Clinically, patients present with jaundice, painful hepatomegaly, and fluid retention, which may evolve into multi-organ failure, a hallmark of severe Disease. The pathogenesis is complex and not completely understood, but the damage to sinusoidal endothelium, typically caused by toxic metabolites released from antineoplastic drugs, is thought to play a crucial role, together with cytokine activation, immune deregulation, and coagulopathy. Diagnosis is based on clinical criteria supported by characteristic ultrasound findings, with the gold standard investigation being Hepatic-venous pressure gradient measurement and biopsy. Several treatment options have been tested; the most convincing approach to date is the use of defibrotide, a novel oligonucleotide with antithrombotic and antiplatelet aggregating properties, as well as endothelial-stabilizing effects. This agent, together with other specific forms of supportive care, has shown efficacy in the treatment of established VOD and promising results in the prevention of VOD in pediatric patients receiving chemotherapy.