Active clinical trials for "Purpura, Thrombocytopenic, Idiopathic"

Immune thrombocytopenia (ITP) is a rare autoimmune disease (annual incidence: 3-4/105 inhabitants) leading to an increased risk of spontaneous bleeding. ITP is said "primary" when not associated to other systemic disease (lymphoma, systemic autoimmune disease, chronic infectious disease…). First-line treatment is based on corticosteroids. Intravenous immunoglobulin (IVIg) is added in case of serious bleeding. In about 70% of adult cases, ITP becomes persistent or chronic (lasting >3 months and >12 months, respectively). Second-line treatments are then indicated. Among them, thrombopoietin-receptor agonists (TPO-RAs), romiplostim and eltrombopag are increasingly used. Splenectomy is used as ultimate treatment. Paradoxically, the risk of thrombosis is higher in ITP patients in comparison with the general population, due to the release of young hyperactive platelets from bone marrow. The incidence of thrombosis in ITP patients has been estimated between 0.5 and 3/100 patients-years. However, risk factors for thrombosis in ITP are not known, except splenectomy that is used in very few patients now. The role of other ITP treatments in thrombosis occurrence has been evoked, particularly for corticosteroids and IVIg. TPO-RAs have been associated with a risk of thrombosis in clinical trials and pharmacovigilance studies, even in case of low or normal platelet count. However, this risk has not been measured in the real-life practice, adjusted for other risk factors for thrombosis.

This is a retrospective analysis of children (≤21years old) who received clinical treatment with either Romiplostim or Eltrombopag at 2 medical centers from 2009-2013. The children included in this study were from Weill Medical College of Cornell University, New York, New York and Children's Hospital Orange County, Orange, California

Chronic immune thrombocytopenic purpura (ITP) is characterized by low platelet counts and the risk of severe bleeding complications. The two recently introduced TPO-RA drugs, namely, eltrombopag and romiplostim, have shown efficacious sustained response with continuous administration. Both drugs are indicated for the treatment of thrombocytopia in patients with chronic ITP who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. While these trials address important clinical questions they were not intended to evaluate what happens in the real-world settings with actual patient living daily lives. The purpose of this health outcomes study is to understand how the two TPO receptor agonists (TPO-RA) currently available in the US are being used in clinical practice and how their use impacts chronic ITP patients' daily lives. The study hypothesis is that patients who switched to eltrombopag report a better health-related quality of life than those who switched to romiplostim. This study utilized a hybrid design of retrospective chart review study and cross-sectional patient survey. A customized Patient Case Report Form (CRF) will be used to retrospectively collect clinical data from patient medical charts where the primary cohorts consist of patients who have switched from other ITP medication to eltrombopag or romiplostim. A cross-sectional survey will be employed to collect patient reported outcomes (PRO) data, including health-related quality of life and treatment satisfaction, using a compository questionnaire. Analyses of cross-sectional survey data and retrospective medical chart review data in patients who switch to either eltrombopag or romiplostim from their prior primary therapy will be conducted.

The investigators are trying to investigate the coagulation status of idiopathic (immune) thrombocytopenic purpura patients by measuring the platelet count, coagulation battery, von Willebrand factor level, thromboelastography.

Cross-sectional study, observation made by healthcare professionals of subjects or caregivers, administering romiplostim at their first standard-of-care visit 4 weeks after training with the home administration training pack. Further observations can also be recorded in the study if made within 16 weeks of enrolment. Data will be collected from the subjects' dose diary at their first standard of care visit to ensure there were no problems with administration while not at the clinic.

To utilise extended platelet parameters in order to individuate Immune Thrombocytopenia (ITP) from hypo-proliferative causes of thrombocytopenia. To develop the clinical potential of the extended platelet parameters as they pertain to distinguishing different causes of thrombocytopenia from one another. To test the hypothesis that mean platelet component (MPC) and mean platelet mass (MPM) might distinguish between thrombocytopenia related to bone marrow dysfunction and immune mediated destruction of platelets.

Idiopathic thrombocytopenic purpura (ITP) is the most frequent auto-immune cytopenia. There is no specific biological marker and the diagnosis often results from the exclusion of other differential diagnoses, notably inherited thrombocytopenia. Recent studies have reported an original platelet destruction mechanism in ITP, by antibody-mediated desialylation of membrane proteins. The detection of platelet sialylation can be readily achieved using flow cytometry. This could provide a new biomarker of ITP, useful to ascertain a diagnosis of ITP and guide towards proper patient management.

Symptoms of Primary Immune Thrombocytopenic Purpura (pITP), such as spontaneous bruising, menorrhagia, mucosal bleeding and other symptoms might significantly affect the HRQOL of pITP patients. However, very little evidence exists on HRQOL outcomes in patients diagnosed with pITP. The investigators project will aim at providing scientific community solid evidence based data on the extent to which HRQOL is compromised and in which specific area. The purpose of this study is thus to compare generic Health-related Quality of Life (HRQOL) profiles of adult patients with primary Immune Thrombocytopenic Purpura (pITP) with that of a matched Italian population control Group.

This is a multi-center prospective observational descriptive study complemented by a retrospective chart review. Patients diagnosed with ITP and currently treated for ITP by a hematologist or hematologist-oncologist will be recruited from community-based clinics and academic/referral centers. They will be followed prospectively for a period of 12 months. At inception, participants' charts will also be reviewed from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever is less.

The aim of this study is to determine histological immunological parameters, sought on splenectomy pieces that may explain the failure or success of splenectomy in patients with ITP who had a splenectomy to treat their ITP(Immune thrombocytopenic purpura).