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    Home > Active Ingredient News > Blood System > 2021 EHA Eltropapax shows considerable efficacy and safety in the treatment of aplastic anemia

    2021 EHA Eltropapax shows considerable efficacy and safety in the treatment of aplastic anemia

    • Last Update: 2021-06-22
    • Source: Internet
    • Author: User
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    The 26th European Annual Meeting of Hematology (2021 EHA) will be held in the near future, and a number of research progress on aplastic anemia (AA) will be announced at the meeting
    .

    At present, the main treatments for AA are hematopoietic stem cell transplantation and immunosuppressive therapy (IST)
    .

    The results of a number of studies at this conference showed that for patients who have failed IST treatment and are not suitable for hematopoietic stem cell transplantation, the small-molecule thrombopoietin receptor agonist etroipopag is a viable treatment option
    .

    The editor now organizes the main content of the research as follows for the reference of readers
    .

    Abstract S172: First-line treatment of adult SAA patients with Eltrombopag combined with cyclosporine: Results of Phase II SOAR Study Background Severe Aplastic Anemia (SAA) is a life-threatening disease of bone marrow failure characterized by pancytopenia and Decreased bone marrow cells
    .

    For SAA patients who are not suitable for hematopoietic stem cell transplantation, the standard treatment is immunosuppressive therapy (IST), including antithymocyte globulin (ATG) and cyclosporin A (CsA), in which equine ATG is better than rabbit ATG.
    Effective
    .

    However, this therapy has drawbacks such as low remission rate, recurrence, and clonal evolution.
    The therapy also has problems such as high treatment costs, some patients cannot tolerate it, and the limited supply of equine ATG, which limits the treatment options for many SAA patients
    .

    Eltrobopar is suitable for SAA patients with poor efficacy of IST or refractory to IST.
    Recently, the FDA has also approved Eltrobopar combined with IST as the first-line treatment for SAA patients
    .

    Research method SOAR (NCT02998645) is a 2-stage, single-arm, multi-center, open-label study
    .

    The purpose of the study was to explore the effectiveness and safety of the ATC-free regimen (eltrobopar plus CsA) in the treatment of SAA
    .

    The study included adult SAA patients who received first-line treatment with etrobopar plus CsA for 6 months, and patients who achieved remission continued to receive CsA treatment for 24 months (reduced to 18 months later)
    .

    Blood transfusion was restricted in the study
    .

    In the study, patients will be evaluated for efficacy twice, with an interval of ≥7 days
    .

    The primary efficacy endpoint of the study is the overall response rate (ORR) after 6 months of treatment
    .

    According to historical data, ORR≥30% is considered clinically significant
    .

    Study results The study included 54 patients with a median age of 55.
    0 years (IQR: 40.
    0-67.
    0), of which 63.
    0% were male, and most of the patients were white (40.
    7%) or yellow (40.
    7%)
    .

    The median duration of patients' exposure to Eltrombopag and CsA was 5.
    7 months (IQR: 2.
    5-5.
    8) and 5.
    7 months (IQR: 2.
    4-8.
    1), respectively
    .

    The median dose of Eltrombopag is 150.
    0 mg/day (IQR: 100.
    0-150.
    0)
    .

    The study reached the primary endpoint, with a 6-month ORR of 46.
    3% (95% CI: 32.
    6%-60.
    4%)
    .

    Among 25 patients who achieved disease remission, 2 patients (6.
    3%) achieved complete remission (CR) at 6 months of treatment
    .

    The secondary endpoints of the study included ORR for 3 months of treatment (40.
    7%; 95%CI: 27.
    6%-55.
    0%) and ORR for 6 months of treatment (patients with confirmed remission) (37.
    0%; 95%CI: 24.
    3%) -51.
    3%)
    .

     Fifty-two patients had adverse events (AE), 45 patients (83.
    3%) had treatment-related AEs (TAE), and 23 patients (42.
    6%) had TAE ≥3
    .

    The most common AEs were increased blood bilirubin (40.
    7%), nausea (29.
    6%), increased alanine aminotransferase (22.
    2%), and diarrhea (22.
    2%)
    .

    Seven patients (13.
    0%) discontinued treatment due to AE
    .

    In the study, 8 patients died (3 cases of aplastic anemia, 1 case each of new coronary pneumonia, bleeding, multiple organ dysfunction syndrome, fever, and thrombosis), and no treatment-related deaths occurred
    .

    Research conclusions SOAR research data show that for SAA patients who are not suitable for ATG treatment, the first-line treatment of Itropopag combined with CsA can bring benefits
    .

    In view of the relatively large median age (55.
    0 years) of the patients in this study, the higher ORR obtained by this treatment program is worthy of attention
    .

    No new safety signals were observed in this study
    .

    Abstract S157: Based on the data from the French RIME Observatory to evaluate the effect of etrobopar on the treatment of AA in the real world.
    Research background AA is characterized by bone marrow failure, and 70% of patients with acquired AA have bone marrow failure
    .

    For adult patients with acquired AA who have been ineffective in previous IST treatment and are not suitable for hematopoietic stem cell transplantation, either alone or combined with IST are viable treatment options
    .

    However, there are few real-world data related to the treatment of AA with Eltrombopag
    .

    Research methods The data in this study comes from the RIME Observatory in France, which incorporates clinical information related to the management of patients with bone marrow failure in France
    .

    The study population consisted of all second-line or later AA patients who were treated with Eltroipopag in the observation station from February 27, 2017 to December 24, 2019
    .

    The purpose of this study is to observe the AA patients treated with itrobopar in the real world, and to evaluate the impact of the treatment with itrobopar, including the impact on survival rate, clinical benefit, hospitalization, quality of life, tolerability, and clonal evolution
    .

    Study Results The study included 93 AA patients, with a median age of 54 years and 58% of the patients were women
    .

    The median follow-up time from the diagnosis of the patients in the study was 37.
    2 months, and the median time from the diagnosis of AA to the treatment of Eltroipopag was 9.
    4 months
    .

    46% of patients had SAA at the time of diagnosis, and 25% of patients had SAA at the time of treatment with Eltroipopag
    .

    Sixty-five patients received the combination regimen of Eltrodopag, and half of the patients received cyclosporine combined with Eltradipal
    .

    Twenty-nine patients received adjuvant therapy, and most of them received iron chelator therapy
    .

     In the study, 76 patients (82%) discontinued Eltropopag at least once, and 71 (76%) patients permanently discontinued Eltroipopag
    .

    Among them, 10 patients (14%) were permanently discontinued with Eltrodopag due to safety issues, and 46 patients (65%) were permanently discontinued with Eltrodopag due to treatment failure
    .

    During the follow-up, 17 patients (18%) died, and the main cause of death was infection or bleeding
    .

    The median overall survival (OS) of the patients in the study after receiving etrobopar treatment was 74.
    4 months, and the 20-month OS rate was approximately 90%
    .

    Sixty-two patients were exposed to etrobopar for at least 6 months, of which 41 patients (66%) achieved disease remission, 26 patients (42%) achieved complete remission (CR), and the median duration of remission was 27.
    5 months
    .

    During the study period, 79 patients received blood transfusion, of which 47 (59%) patients were free from blood transfusion dependence
    .

    Forty patients (43%) were hospitalized after receiving treatment with Eltrombopag.
    The main reasons for the hospitalization were planned hospitalization (30%) and complications (43%)
    .

    The study collected data on the quality of life of 6 patients, but it is temporarily impossible to interpret this part of the data
    .

    59 patients (63%) had at least one AE during treatment.
    The most common AEs were infection, hepatobiliary disease, pain, weakness, and bleeding
    .

    Ten patients (11%) developed clonal evolution (9 cases of myelodysplastic syndrome, 1 case of acute myeloid leukemia)
    .

    Research conclusions The study evaluated the real-world effects of itrobopal in the treatment of AA, and more than two-thirds of patients achieved remission after receiving itrobopal
    .

    Forty-two percent of patients received CR after 2 years of treatment with Eltrombopag, more than 50% of patients were free of blood transfusion dependence, and 11% of patients showed clonal evolution, which is close to the data in the relevant literature
    .

    The safety data in the study is also close to the relevant clinical research data
    .

    The results of this study confirmed the clinical benefits of Eltrombopag in these patients
    .

    Reference source: 1.
    Efreen Montaño-Figueroa, et al.
    2021 EHA.
    S172.
    2.
    Flore Sicre de Fontbrune, et al.
    2021 EHA.
    S157.
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