Estudio primario

No clasificado

Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia

Año 2022
Revista The New England journal of medicine
Enlaces DOI , Pubmed

Este artículo está incluido en 3 Revisiones sistemáticas Revisiones sistemáticas (3 referencias) 1 Síntesis amplia Síntesis amplias (1 referencia)

Cargando información sobre las referencias
Mostrar resumen

BACKGROUND:

A single-group, phase 1-2 study indicated that eltrombopag improved the efficacy of standard immunosuppressive therapy that entailed horse antithymocyte globulin (ATG) plus cyclosporine in patients with severe aplastic anemia.

METHODS:

In this prospective, investigator-led, open-label, multicenter, randomized, phase 3 trial, we compared the efficacy and safety of horse ATG plus cyclosporine with or without eltrombopag as front-line therapy in previously untreated patients with severe aplastic anemia. The primary end point was a hematologic complete response at 3 months.

RESULTS:

Patients were assigned to receive immunosuppressive therapy (Group A, 101 patients) or immunosuppressive therapy plus eltrombopag (Group B, 96 patients). The percentage of patients who had a complete response at 3 months was 10% in Group A and 22% in Group B (odds ratio, 3.2; 95% confidence interval [CI], 1.3 to 7.8; P = 0.01). At 6 months, the overall response rate (the percentage of patients who had a complete or partial response) was 41% in Group A and 68% in Group B. The median times to the first response were 8.8 months (Group A) and 3.0 months (Group B). The incidence of severe adverse events was similar in the two groups. With a median follow-up of 24 months, a karyotypic abnormality that was classified as myelodysplastic syndrome developed in 1 patient (Group A) and 2 patients (Group B); event-free survival was 34% and 46%, respectively. Somatic mutations were detected in 29% (Group A) and 31% (Group Β) of the patients at baseline; these percentages increased to 66% and 55%, respectively, at 6 months, without affecting the hematologic response and 2-year outcome.

CONCLUSIONS:

The addition of eltrombopag to standard immunosuppressive therapy improved the rate, rapidity, and strength of hematologic response among previously untreated patients with severe aplastic anemia, without additional toxic effects. (Funded by Novartis and others; RACE ClinicalTrials.gov number, NCT02099747; EudraCT number, 2014-000363-40.).

Mostrar resumen

Estudio primario

No clasificado

Eltrombopag and Early Refractory Immune Thrombocytopenia (ITP)

Año 2013
Autores Alfred Health
Registro de estudios Australian New Zealand Clinical Trials Register
Enlaces ANZCTR

Este artículo no está incluido en ninguna revisión sistemática

Cargando información sobre las referencias
Mostrar resumen

Este artículo no tiene resumen

Mostrar resumen

Estudio primario

No clasificado

Eltrombopag for Peripheral Blood Stem Cell Harvest

Año 2023
Registro de estudios clinicaltrials.gov
Enlaces ClinicalTrials.gov

Este artículo no está incluido en ninguna revisión sistemática

Cargando información sobre las referencias
Mostrar resumen

The goal of this clinical trial is to explore the activity of eltrombopag in lymphoma patients receiving autologous hematopoietic stem cell harvest. The main questions it aims to answer are:

* Determine the efficacy of adding eltrombopag during autologous hematopoietic stem cell mobilization and harvest.
* Determine the pharmacokinetics and pharmacodynamics of serum eltrombopag concentration, circulating CD34+ cells during autologous hematopoietic stem cell mobilization.

Participants will receiving additional eltrombopag during stem cell harvest procedure. The amount of harvested stem cells will be compared with historical group to see if eltrombopag could increase the amount of harvested stem cells.

Mostrar resumen

Estudio primario

No clasificado

Severe Thrombotic Complication of Eltrombopag in a Cirrhotic Patient.

Año 2016
Autores Baumann AJ , Wheeler DS , Varadi G , Feyssa E
Revista ACG case reports journal
Enlaces PMC , DOI , Pubmed

Este artículo no está incluido en ninguna revisión sistemática

Cargando información sobre las referencias
Mostrar resumen

We present a patient with hepatitis C virus (HCV) and cirrhosis who was treated with eltrombopag for idiopathic thrombocytopenic purpura and was incidentally found to have a right atrial thrombus with extension into the left internal jugular vein. Eltrombopag was discontinued and the patient was treated with thrombectomy and anticoagulation. Given the proposed use of eltrombopag in HCV-associated thrombocytopenia, we advise caution when treating cirrhotics who are at higher intrinsic risk of thrombosis.

Mostrar resumen

Estudio primario

No clasificado

Eltrombopag for Thrombocytopenia in Patients With Relapsed Multiple Myeloma

Año 2012
Registro de estudios clinicaltrials.gov
Enlaces ClinicalTrials.gov

Este artículo no está incluido en ninguna revisión sistemática

Cargando información sobre las referencias
Mostrar resumen

Eltrombopag is a compound that may help stimulate the production of platelets. This drug has been used in treatment of low platelet counts caused by a disorder called idiopathic thrombocytopenic purpura and information from those other research studies suggests that Eltrombopag may help to maintain platelet counts in patients with relapsed multiple myeloma in this research study.

In this research study,the investigators are trying to determine if Eltrombopag is effective in maintaining platelet counts in patients who are being treated for relapsed multiple myeloma.

Mostrar resumen

Estudio primario

No clasificado

Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia.

Año 2017
Revista The New England journal of medicine
Enlaces PMC , DOI , Pubmed

Este artículo está incluido en 2 Revisiones sistemáticas Revisiones sistemáticas (2 referencias)

Cargando información sobre las referencias
Mostrar resumen

BACKGROUND:

Acquired aplastic anemia results from immune-mediated destruction of bone marrow. Immunosuppressive therapies are effective, but reduced numbers of residual stem cells may limit their efficacy. In patients with aplastic anemia that was refractory to immunosuppression, eltrombopag, a synthetic thrombopoietin-receptor agonist, led to clinically significant increases in blood counts in almost half the patients. We combined standard immunosuppressive therapy with eltrombopag in previously untreated patients with severe aplastic anemia.

METHODS:

We enrolled 92 consecutive patients in a prospective phase 1-2 study of immunosuppressive therapy plus eltrombopag. The three consecutively enrolled cohorts differed with regard to the timing of initiation and the duration of the eltrombopag regimen (cohort 1 received eltrombopag from day 14 to 6 months, cohort 2 from day 14 to 3 months, and cohort 3 from day 1 to 6 months). The cohorts were analyzed separately. The primary outcome was complete hematologic response at 6 months. Secondary end points included overall response, survival, relapse, and clonal evolution to myeloid cancer.

RESULTS:

The rate of complete response at 6 months was 33% in cohort 1, 26% in cohort 2, and 58% in cohort 3. The overall response rates at 6 months were 80%, 87%, and 94%, respectively. The complete and overall response rates in the combined cohorts were higher than in our historical cohort, in which the rate of complete response was 10% and the overall response rate was 66%. At a median follow-up of 2 years, the survival rate was 97%; one patient died during the study from a nonhematologic cause. Marked increases in bone marrow cellularity, CD34+ cell number, and frequency of early hematopoietic progenitors were noted. Rates of relapse and clonal evolution were similar to our historical experience. Severe rashes occurred in two patients, resulting in the early discontinuation of eltrombopag.

CONCLUSIONS:

The addition of eltrombopag to immunosuppressive therapy was associated with markedly higher rates of hematologic response among patients with severe aplastic anemia than in a historical cohort. (Funded by the National Heart, Lung, and Blood Institute; ClinicalTrials.gov number, NCT01623167 .).

Mostrar resumen

Estudio primario

No clasificado

Eltrombopag: How secure in triple therapy of HCV?

Año 2015
Revista Acta gastro-enterologica Belgica
Enlaces Pubmed

Este artículo no está incluido en ninguna revisión sistemática

Cargando información sobre las referencias
Mostrar resumen

Triple therapy of hepatitis C usually leads to some hematological and dermatological side effects. Thrombocytopenia is one of the most common side effects that are encountered during triple therapy. Eltrombopag was approved for the treatment of patients with chronic hepatitis C and thrombocytopenia to allow the initiation and maintenance of interferon based therapies. During eltrombopag therapy, some side effects like headache, abdominal pain, and some complications such as portal vein thrombosis, deep vein thrombosis and arterial thrombosis were observed more frequently than placebo. We described here a patient who developing thrombosis secondary to eltrombopag in receiving triple therapy.

Mostrar resumen

Estudio primario

No clasificado

Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag

Año 2020
Registro de estudios clinicaltrials.gov
Enlaces ClinicalTrials.gov

Este artículo no está incluido en ninguna revisión sistemática

Cargando información sobre las referencias
Mostrar resumen

Background:

Diamond-Blackfan anemia (DBA) is treated with steroids. But some people cannot take steroids, or steroids don t work. Other patients must get blood transfusions regularly which are time consuming and can have significant side effects. The drug eltrombopag can increase red blood cells. Researchers want to see if it can help people with DBA and, if so, for how long.

Objective:

To study the safety and efficacy of eltrombopag in people with DBA who have not responded to steroids or could not take them.

Eligibility:

People ages 2 and older with DBA who did not respond to steroids or could not take them, or their disease has returned despite taking them

Design:

Participants will be screened with:

Medical and medicine history

Physical exam

MRI:

Participants will lie in a machine that takes pictures of the liver.

Blood and urine tests

Bone marrow biopsy: A thin needle will remove a marrow sample from the participant\'s hip bone.

Electrocardiogram

Participants will take eltrombopag pills once daily for 24 weeks. They will have blood taken every 2 weeks.

Participants will have visits 6 months. At 6 months, they will repeat all the screening tests and also have:

Quality-of-life questionnaire

Neurodevelopmental test (for participants younger than 18 years)

If participants blood cell counts improve, they may keep taking eltrombopag for up to 3 more years. If so, they will have blood taken every 4 weeks. They will visit NIH every 6 months and repeat the above tests.

Participants will be monitored for up to 3 years after they stop taking eltrombopag. They will visit NIH 6 months after treatment ends. If participants blood counts go down after treatment ends, they may restart the drug....

Mostrar resumen

Estudio primario

No clasificado

Eltrombopag in Elderly Acute Myelogenous Leukemia (AML)

Año 2010
Registro de estudios clinicaltrials.gov
Enlaces ClinicalTrials.gov
Cargando información sobre las referencias
Mostrar resumen

This is a phase I/II open label study being conducted to evaluate the overall safety and initial effectiveness of an investigational drug, Eltrombopag in patients who are 60 years of age and older and who have Acute Myelogenous Leukemia (AML). Eltrombopag is an investigational drug, which means it has not been approved by the U.S. Food and Drug Administration (FDA) for use in this type of disease. Approximately 35 people will be enrolled on this study at the University of Pennsylvania

Mostrar resumen

Estudio primario

No clasificado

Eltrombopag and improved hematopoiesis in refractory aplastic anemia.

Año 2012
Revista The New England journal of medicine
Enlaces PMC , DOI , Pubmed

Este artículo está incluido en 1 Revisión sistemática Revisiones sistemáticas (1 referencia)

Cargando información sobre las referencias
Mostrar resumen

BACKGROUND:

Severe aplastic anemia, which is characterized by immune-mediated bone marrow hypoplasia and pancytopenia, can be treated effectively with immunosuppressive therapy or allogeneic transplantation. One third of patients have disease that is refractory to immunosuppression, with persistent, severe cytopenia and a profound deficit in hematopoietic stem cells and progenitor cells. Thrombopoietin may increase the number of hematopoietic stem cells and progenitor cells.

METHODS:

We conducted a phase 2 study involving patients with aplastic anemia that was refractory to immunosuppression to determine whether the oral thrombopoietin mimetic eltrombopag (Promacta) can improve blood counts. Twenty-five patients received eltrombopag at a dose of 50 mg, which could be increased, as needed, to a maximum dose of 150 mg daily, for a total of 12 weeks. Primary end points were clinically significant changes in blood counts or transfusion independence. Patients with a response continued to receive eltrombopag.

RESULTS:

Eleven of 25 patients (44%) had a hematologic response in at least one lineage at 12 weeks, with minimal toxic effects. Nine patients no longer needed platelet transfusions (median increase in platelet count, 44,000 per cubic millimeter). Six patients had improved hemoglobin levels (median increase, 4.4 g per deciliter); 3 of them were previously dependent on red-cell transfusions and no longer needed transfusions. Nine patients had increased neutrophil counts (median increase, 1350 per cubic millimeter). Serial bone marrow biopsies showed normalization of trilineage hematopoiesis in patients who had a response, without increased fibrosis. Monitoring of immune function revealed no consistent changes.

CONCLUSIONS:

Treatment with eltrombopag was associated with multilineage clinical responses in some patients with refractory severe aplastic anemia. (Funded by the National Heart, Lung, and Blood Institute; ClinicalTrials.gov number, NCT00922883.).

Mostrar resumen