Active clinical trials for "Thrombocythemia, Essential"

This study involves screening for portal vein thrombosis and portal hypertension in patients with Philadelphia negative myeloproliferative neoplasms (MPNs). These include polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis. Portal vein thrombosis and portal hypertension are serious complications that are often seen in myeloproliferative patients. These complications are usually diagnosed when patients become symptomatic, and are often already at an advanced stage. They can further progress to cause non-reversible damage to the liver, also called cirrhosis of the liver. As a result of this, patients often accumulate fluid in the abdomen which is ascites; and can develop swelling of veins in the lining of the esophagus known as varices. If untreated, varices have the risk of rupturing resulting in life-threatening bleeding. When diagnosed at an advanced stage, the treatment is usually supportive therapy and there are no treatments available at present which can reverse these conditions. This study is looking at screening for these two conditions using Doppler ultrasound and upper gastrointestinal endoscopy.

This is an observational safety study being conducted in Europe comparing patients taking Xagrid to patients taking other cytoreductive treatments. The plan is to enrol at least 750 subjects taking Xagrid with up to 3000 subjects taking other cytoreductive therapies. The study will collect follow up data for 5 years for each patient enrolled that will focus on collecting data related to pre-defined events (PDEs) and Suspected Serious Adverse Reactions (SSARs).

Researchers will use abnormal blood and/or bone marrow cells, or materials derived from these abnormal cells, like DNA, RNA, protein or plasma, in laboratory studies. Toenail clippings will provide normal material like DNA for comparison with the abnormal material derived from the blood and/or bone marrow. The results of these studies will be correlated with subjects' disease symptoms and response to their experimental treatment. The MPD-RC researchers are interested in studying molecules from the blood and bone marrow, the exact molecules changing over time with the investigators choosing only the most promising for investigation. The investigators are attempting to better understand the causes of MPD and to develop improved methods for the diagnosis and treatment of these diseases. These syndromes carry a high risk of developing leukemia. It is important to continue to learn more about these blood cancers and to learn more about the effectiveness and potential side effects of various treatments. It is believed that further basic knowledge about these cancer cells as well as the effects of treatment will lead to the improvement of current therapies and the development of entirely new treatments for these diseases. The MPD-RC is hoping to determine if a number of laboratory tests (biomarkers) will allow for the prediction of response in future patients to the treatment they would receive.

The three main chronic myeloproliferative disorders are polycythemia vera (PV), essential thrombocythemia (ET) and idiopathic myelofibrosis (IMF). These are clonal neoplastic diseases characterized by proliferation of one or more hematopoietic lineages. Recently a mutation of the Janus Kinase 2 (JAK2) gene that leads to the substitution of phenylalanine for valine at position 617 of the JAK2 protein, JAK2 V617F, has been found in 76% to 97% of patients with PV, 29% to 57% of patients with ET and 50% of patients with IMF. This mutation confers constitutive activity on to the JAK2 protein and appears to play an important role in the pathobiology of these conditions. However, not all patients with myeloproliferative disorders have this mutation and it may not be the primary cause of these diseases. The primary goal of this prospective natural history study is to investigate the molecular basis of these diseases in groups of patients who have JAK2 V617F and in those who do not. A second goal is to identify biomarkers for PV and the other myeloproliferative disorders that are easier to measure than JAK2 V617F. Approximately, 150 patients with myeloproliferative disorders will be studied over 3 years. The studies will involve the collection of 40 mL to 50 mL of peripheral blood from each subject. The blood will be used to assess neutrophil gene and protein expression, gene polymorphisms, and plasma protein levels.

The aim of this study is to evaluate the progression of ET in children (aged 6-17years inclusive) over a period of 5 years maximum. The study will also assess how children are diagnosed, treatment options for those children with symptoms and events related to their ET and the outcomes of those treatments.

The purpose of this prospective, longitudinal, noninterventional study is to describe clinical characteristics, evolution of disease burden, and treatment patterns in patients with select subcategories of essential thrombocythemia (ET) or myelofibrosis (MF).

Patients treated with oral or sub-cutaneous drugs for polycythemia vera or essential thrombocythemia completed an unique paper questionnaire in order to describe their observance, the side effects and their own appreciation of the taken drug.

This study that will allow for the preservation and/or storage of a small portion one or more of the following tissues: Peripheral blood Bone marrow Bone marrow biopsy A phlebotomized unit of blood Spleen cells Toenail clippings This material will be used for the study of Myeloproliferative Disorders (MPD) by researchers. The goals of this research study are to understand the causes of MPDs, how to diagnose them more easily and how to treat them better. MPD is a disease affecting hematopoietic stem cells. Hematopoietic stem cells are cells that make blood cells. These stem cells grow in the center portion of the bones called bone marrow. Under some conditions, these cells are also found in blood. There are several diseases, which are classified as MPD. These include polycythemia vera (too many red blood cells), essential thrombocythemia (too many platelets), and idiopathic myelofibrosis (abnormal blood cells and fibers build up in the bone marrow). These syndromes carry a high risk of developing leukemia. It is important to continue to learn more about these blood cancers and to learn more about the effectiveness and potential side effects of various treatments.

The purpose of the study is to isolate and characterize stem cells of patients with Polycythemia Vera (PV) and Essential Thrombocythemia (ET) to find out why these cells are not working the way they should be and why they seem to be sensitive to regulatory factors in the blood, such as clotting.

The main purpose of this project is to study the uptake pattern of FLT-PET in cases, and it is value in assessing the malignant hematopoiesis in cases of Pre-PMF and ET, regarding diagnosis, staging and monitoring response to therapy. Identifying different patterns of uptake in patients with Pre-PMF and ET in various clinical settings. Evaluating FLT-PET as a novel non-invasive technique in cases with Pre-PMF and ET, in comparison to the standard bone marrow biopsy about disease diagnosis, assessment of disease activity, detection of transformation, monitoring of treatment response and grading of fibrosis.Study the ability of FLT-PET to differentiate between Pre-PMF and ET. the investigators also aim to examine the association of FLT-PET uptake patterns with different genetic makeup (JAK2 (Janus kinase 2), CALR (Calreticulin), MPL (myeloproliferative leukemia protein), or Triple negative disease) or allele burden in cases of Pre-PMF and ET.