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Año 2023
Autores Ji J , Wan Z , Ruan J , Yang Y , Hu Q , Chen Z - Más
Revista Blood cancer journal
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This trial compared eltrombopag (EPAG)+tacrolimus and EPAG monotherapy in patients with refractory/relapsed acquired aplastic anaemia (AA). Patients with refractory/relapsed AA were randomly assigned to receive either EPAG+tacrolimus or EPAG monotherapy at a ratio of 2:1. Patient response, safety, clonal evolution and survival were compared. In total, 114 patients were included in the analysis, with 76 patients receiving EPAG+tacrolimus and 38 receiving EPAG only. With a median follow-up of 18 (6–24) months, the overall response rate (ORR) for patients treated with EPAG+tacrolimus and EPAG alone was 38.2% vs. 31.6% (P = 0.490) at the 3rd month, 61.8% vs. 39.5% (P = 0.024) at the 6th month, 64.5% vs. 47.1% (P = 0.097) at the 12th month, and 60.5% vs. 34.2% (P = 0.008) at the last follow-up. The rate of each adverse event, overall survival curves (P = 0.635) and clonal evolution rate (P = 1.000) were comparable between the groups. A post hoc subgroup analysis showed that EPAG+tacrolimus could have advantage over EPAG monotherapy in terms of the ORR at the 6th month (P = 0.030)/last follow-up (P = 0.013) and the cumulative relapse-free survival (RFS) curves (P = 0.048) in patients <60 years old. © 2023, Springer Nature Limited.

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Año 2011
Autores Novartis
Registro de estudios ClinicalTrials.gov

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This was a worldwide, three-part (Part 1: open-label, Part 2: randomized, double-blind, Part 3: extension), multi-center study to evaluate the effect of eltrombopag in subjects with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) who have thrombocytopenia due to bone marrow insufficiency from their underlying disease or prior chemotherapy. This objective was assessed by a composite primary endpoint that consists of the following: the proportion of ≥Grade 3 hemorrhagic adverse events, or platelet counts \<10 Gi/L, or platelet transfusions. Patients with MDS or AML and Grade 4 thrombocytopenia due to bone marrow insufficiency from their underlying disease or prior chemotherapy were enrolled in the study. No low or intermediate-1 risk MDS subjects were enrolled in the study. Subjects must have had at least one of the following during the 4 weeks prior to enrolment: platelet count \<10 Gi/L, platelet transfusion, or symptomatic hemorrhagic event. Supportive standard of care (SOC), including hydroxyurea, was allowed as indicated by local practice throughout the study. The study had 3 sequential parts. Subjects who were enrolled in Part 1 (open-label) cannot be enrolled in Part 2 of the study (randomized, double-blind); however, subjects who completed the treatment period for Part 1 or Part 2 (8 and 12 weeks, respectively) continued in Part 3 (extension) if the investigator determined that the subject was receiving clinical benefit on treatment.

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Año 2017
Autores LLC Novartis Pharma
Registro de estudios EU Clinical Trials Register

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Estudio primario

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Año 2023
Autores Hu XR
Revista Chinese Academy of Medical Science & Peking Union Medical College
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Estudio primario

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Año 2017
Autores CHU Toulouse
Registro de estudios EU Clinical Trials Register

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INTERVENTION:

Trade Name: Revolade Product Name: Eltrombopag Pharmaceutical Form: Film‐coated tablet

CONDITION:

Inherited thrombocytopenias ; MedDRA version: 20.0 Level: HLT Classification code 10043555 Term: Thrombocytopenias System Organ Class: 100000004851 Therapeutic area: Diseases [C] ‐ Blood and lymphatic diseases [C15]

PRIMARY OUTCOME:

Main Objective: The primary objective of the study is to estimate the response to eltrombopag based on platelet count increase and lack of requirement for PC transfusion for performing invasive acts at mild or high bleeding risk Primary end point(s): Full response (“success”) is defined as an increase in platelet count above the safety level and a procedure performed without PC transfusion, Secondary Objective: The secondary objective is to document in this particular setting the safety profile of eltrombopag, the kinetics of platelet recovery and the predictive value Inherited thrombocytopenia (IT) type, serum thrombopoietin (TPO) level and baseline platelet count on the response to eltrombopag. Timepoint(s) of evaluation of this end point: 4 weeks

SECONDARY OUTCOME:

Secondary end point(s): Adverse effects recorded during treatment and the 4‐week follow‐up after discontinuation, including excessive bleeding or any complication related to bleeding or thrombotic events.; Serial platelet counts and change in platelet size; Timepoint(s) of evaluation of this end point: 1/week during 8 weeks

INCLUSION CRITERIA:

Related to patients: Symptomatic inherited thrombocytopenia (IT) with family history and/or molecular identification with an average platelet count in the previous year below the safety level required for the procedure. Written informed consent of the patient or relatives. Related to the procedure: scheduled (=4 weeks) invasive procedure with anticipated risk and limited possibility of mechanical control of bleeding, which would have systematically required prophylactic and therapeutic PC transfusions, according to current international recommendations (standard care). Are the trial subjects under 18? yes Number of subjects for this age range: 25 F.1.2 Adults (18‐64 years) yes F.1.2.1 Number of subjects for this age range 25 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 25

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Año 2018
Autores Novartis
Registro de estudios clinicaltrials.gov

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The purpose of this trial was to assess the ability of eltrombopag to induce sustained treatment-free remission in immune thrombocytopenia purpura (ITP) subjects who relapsed or failed to respond to an initial treatment with steroids.

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Año 2013
Autores Shandong University
Registro de estudios clinicaltrials.gov

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This project was undertaken by Qilu Hospital of Shandong University and other well-known hospitals in China. In order to report the alteration of Different Immune Parameters in ITP Patients Receiving Eltrombopag Treatment.

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Año 2010
Autores Chouhan JD , Herrington JD
Revista Pharmacotherapy

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Idiopathic thrombocytopenic purpura (ITP) is a platelet disorder that affects approximately 1 in 10,000 people. In adults, the rate of spontaneous remission is only 5%, and generally, it is a chronic disease persisting for more than 6 months. Chronic refractory ITP may be defined as the failure of any modality to keep the platelet count above 20 x 103/mm3 for an appreciable time without unacceptable toxicity. Many pharmacologic treatments have been used to manage chronic refractory ITP by attempting to increase platelet counts by decreasing the rate of destruction of these cells. They include, but are not limited to, azathioprine, danazol, dapsone, combination chemotherapy, cyclosporine, and rituximab. However, these therapies offer modest response rates and can cause adverse events that necessitate drug discontinuation. The recent United States Food and Drug Administration approval of the thrombopoietin mimetics, romiplostim and eltrombopag, has provided clinicians with a novel approach for treating chronic refractory ITP. By stimulating platelet production, these drugs offer patients with this disease an alternative to the other agents. The preapproval phase III study with subcutaneous romiplostim showed significantly higher overall response rates versus placebo in both splenectomized and nonsplenectomized patients (83% for romiplostim vs 7% for placebo, p<0.0001). Twenty-five percent of patients receiving romiplostim achieved a platelet count greater than 50 x 10 3/mm3 after 1 week, and 50% achieved this platelet count within 2-3 weeks. The preapproval phase III study with oral eltrombopag demonstrated that 70% of patients receiving 50 mg/day and 81% of patients receiving 75 mg/day achieved a platelet count of at least 50 x 10 3/mm3 by day 43 (p<0.001 vs placebo for both 50 and 75 mg). Forty-four percent and 62% of patients achieved a platelet count of at least 50 x 103/mm3 by day 8 with eltrombopag 50 and 75 mg/day, respectively. When deciding which of these agents to prescribe, considerations include oral versus injectable dosage form, adverse-event profiles, and patient adherence with both taking the drug and keeping clinic appointments for monitoring of platelet counts. Several studies are under way to evaluate these drugs in chronic refractory ITP as well as other disease states. Long-term data will also be needed to assess the safety and efficacy of these agents.

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Año 2011
Revista Clinics and practice

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HIV-associated thrombocytopenia is a disease which can be recurrent to standard therapy which includes highly active antiretroviral therapy (HAART) therapy, steroids and immunoglobulin. We report a patient with HIV and hepatitis C who presented with resistant thrombocytopenia. Treatment with Eltrombopag - a thrombopoeitin receptor agonist showed initial good response with recurrence of thrombocytopenia. This novel agent could be considered as a treatment option prior to splenectomy and may be useful as a temporizing measure.

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Año 2025
Autores Zhang B , Yang W , Kang R , Shi Y , Hu X , Zhang L - Más
Revista European journal of haematology

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The addition of thrombopoietin receptor agonists (TPO-RAs) to immunosuppressive therapy (IST) improves the hematologic response rate and quality in patients with severe aplastic anemia (SAA). However, no studies have yet reported on whether there are differences in the efficacy of TPO-RAs. Here, we retrospectively analyzed the clinical data of SAA patients who received hetrombopag (HPAG) or eltrombopag (EPAG) as part of first-line standard IST to compare the efficacy. Sixty-seven patients were enrolled in the HPAG group and 42 patients in the EPAG group. The overall hematologic response (OR) rates of the HPAG group at 3 and 6 months after IST were 50.7% and 65.6%, respectively, close to that of the EPAG group (50%, p = 0.973; 73.8%, p = 0.494). The rates of complete response (CR) at 3 and 6 months were 13.4% and 31.3% in the HPAG group, respectively, which were like those in the EPAG group (11.9% and 28.6%), with no statistical difference (p = 1.00 and 0.59). The median time to first response (3.0 vs. 3.2 months, p = 0.79) was similar in HPAG and EPAG. The overall survival (OS) rates were 91.0% and 92.8% in the HPAG group and EPAG group (p = 0.53), respectively. Monosomy 7 was detected in one patient in the EPAG group and her disease transformed into acute myelocytic leukemia (AML) at 25 months after ATG treatment. One patient in HPAG had trisomy 8 at 9 months of ATG treatment, and bone marrow examination showed no disease progression.

CONCLUSION:

The addition of HPAG to standard IST in SAA patients showed similar response rates and response quality to that of EPAG. HPAG could be an alternative to EPAG for the first-line treatment of SAA patients.

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