Active clinical trials for "Hepatic Veno-Occlusive Disease"

Sinusoidal Obstruction Syndrome (SOS), also referred to as hepatic veno-occlusive disease (VOD), is rare but serious complication of allogeneic stem cell transplantation (allo-SCT). Defibrotide is the only FDA approved therapy to treat SOS and has significantly improved outcomes. When applied early, SOS symptoms often quickly improve and an abbreviated course can be applied. This study is looking at an abbreviated 5 day course of defibrotide in those patients with a complete response to therapy with the primary outcome being day 100 overall survival as compared to history data.

To evaluate efficacy and safety of AT-III treatment in patients with hepatic veno-occlusive diseases following hematopoietic stem cell transplantation.

Hepatic veno-occlusive disease/sinusoidal obstructive syndrome (VOD/SOS) is a potentially fatal complication of hematopoietic cell transplant (HCT). Historically VOD/SOS has been clinically diagnosed using the modified Seattle criteria or the Baltimore criteria. The modified Seattle Criteria define VOD/SOS diagnosis is made when two of the following three criteria are present in a patient within 21 days of transplantation: hyperbilirubinemia (total serum bilirubin > 2 mg/dL), hepatomegaly or right upper quadrant liver pain, and weight gain (> 2% of baseline) or ascites. Other conditions like graft versus host disease, sepsis syndrome (fever and hypotension), cardiac failure, or tumor infiltration) have to be excluded. This definition was from a well-designed retrospective cohort study on 255 adult and pediatric HCT patients in which the VOD/SOS incidence was 21%. McDonald et al followed up this work with a prospective cohort study of 355 patients noting an incidence of VOD/SOS of 54%. These seminal studies have had a major impact on the field by defining clinical diagnostic criteria. An alternative diagnostic criteria (Baltimore criteria) was proposed by Jones et al as a part of a well-designed retrospective review of 235 HCT patients finding a VOD/SOS incidence of 22%. Jones defined VOD/SOS as the presence of hyperbilirubinemia (total serum bilirubin > 2 mg/dL) along with at least 2 of 3 other findings: hepatomegaly, ascites, and weight gain (> 5% of baseline).

The aim of this trial is to evaluate whether the prophylactic use of Defibrotide (DF) in pediatric patients (age less than 18 years) undergoing stem cell transplantation and who are at high risk of developing hepatic Veno-occlusive Disease (VOD) will have an impact on the incidence and severity of the disease. Patients will be randomly assigned to one of two treatment arms: Those allocated to the Prophylactic Arm will receive the study drug (Defibrotide) from the day of conditioning onwards. Patients allocated to the Control Arm will receive the study drug (Defibrotide) from the day that VOD is diagnosed.

Sinusoidal obstruction syndrome (SOS) is a potentially fatal hepatic veno-occlusive disease-affecting children following bone marrow transplantation (BMT). SOS most likely develops secondary to sinusoidal endothelial damage and subsequent obstruction. The disease can be separated into mild, moderate, and severe forms; almost all patients diagnosed with severe SOS will die from this disease. Children with severe SOS suffer from multi-organ failure with signs and symptoms of portal hypertension (ascites, varices, edema), renal and respiratory failure . Although these children may be few and far between, the mere severity of the disease process and awful prognosis factors are valid reasons for more medical attention. SOS is usually diagnosed via clinical criteria and, despite available prophylaxis and treatment, children continue to die from this devastating disease. Quantitative shear wave ultrasound elastography with acoustic force radiation imaging is an emerging technology that uses ultrasound pressure waves to provide an estimate of tissue stiffness. This technique is promising for pediatric imaging because it is portable, quick to perform, relatively low cost and involves no ionizing radiation. Acoustic force radiation imaging and ultrasound elastography does not have any increased risks over conventional ultrasound imaging.

Sinusoidal obstruction syndrome (SOS) or hepatic veno-occlusive disease (VOD) is a serious complication that occurs, most often occurring in patients undergoing hematopoietic stem cell transplantation (HSCT), especially in its first thirty days. The morbidity and mortality in this syndrome are considerable, since severe SOS is associated with a mortality of more than 90% in the first hundred days of HSCT. Some risk factors are modifiable, especially those related to transplantation, but when non-alterable factors are present, preventive measures are needed that can reduce the incidence and / or severity of SOS.

Following the licencing of a new drug, Defitelio®, indicated for the treatment of severe Veno-Occlusive Disease of the liver (sVOD), a rare but serious complication of haematopoietic stem cell transplantation (HSCT), as a specific obligation (SOB), the manufacture and marketing Authorisation Holder (MHA) (Gentium, a Jazz Pharmaceuticals Company) was required by PRAC (Pharmacovigilance Risk Assessment Committee) to set up a disease registry to collect safety and outcome data, and to assess patterns of utilization of Defitelio® in the post-approval setting. This registry is a Post Authorization Safety Study (PASS), is being coordinated in collaboration with the European Society for Blood and Marrow Transplantation (EBMT). For this study, anonymised clinical data are being collected from patients who develop VOD and and treated with and patients who have been treated with Defitelio® for conditions other than sVOD. The study DOES NOT involve decisions about treatment, which are clinical decisions, but merely collection of data for patients who develop this complication, whether or not they receive treatment and for patients who are treated with Defitelio® for any other reasons.The study DOES NOT involve decisions about treatment, which are clinical decisions, but merely collection of data for patients who develop this complication, whether or not they receive treatment and for patients who are treated with Defitelio® for any other reasons.

Hepatic veno-occlusive diseases (VOD) during cancer treatment in children are serious toxicities that have occurred with interruptions of chemotherapy and risk of relapse. In addition, these toxicities have a negative impact on the patient's quality of life, serious long-term sequelae and are potentially fatal in children. The risk factors associated with the occurrence of these complications are, to date, unknown, at the exception to the exposition to certain treatments (6-thioguanine, busulfan, actinomycin D, radiotherapy, etc.). To understand the effects of this toxicity and those of susceptibility to the disease becomes a major issue in the treatment of these children.

The purpose of the study is to determine whether anticoagulant use (i.e. salicylates, clopidogrel, low-molecular weight heparin, or coumarin derivates) is able to prevent the development of the sinusoidal obstruction syndrome secondary to oxaliplatin-based neoadjuvant chemotherapy in patients suffering from colorectal liver metastases.

We would like conduct a retrospective study in our center to evaluate the early detection of sinusoidal obstruction syndrome with hepatic ultrasound after allogeneic hematopoietic stem cell transplantation.