Eltrombopag + Immunosuppression for Severe Aplastic Anemia
Trial Summary
Do I have to stop taking my current medications for this trial?
The trial protocol does not specify if you need to stop taking your current medications. However, if you are on active chemotherapeutic treatment or take drugs with hematological effects, you will not be eligible to participate.
Do I have to stop taking my current medications for this trial?
The trial protocol does not specify if you need to stop taking your current medications. However, if you are on active chemotherapy or drugs with blood-related effects, you may not be eligible to participate.
What data supports the idea that Eltrombopag + Immunosuppression for Severe Aplastic Anemia is an effective treatment?
The available research shows that adding eltrombopag to the standard immunosuppressive therapy, which includes horse antithymocyte globulin and cyclosporine, improves the treatment's effectiveness for severe aplastic anemia. Specifically, eltrombopag increased the rate of overall and complete responses when used as part of the initial treatment. This combination is more effective than using eltrombopag alone, which has a response rate of 40% to 50% in patients who did not respond to initial immunosuppression. In comparison, the standard treatment without eltrombopag has a response rate of 60% to 70%, but adding eltrombopag seems to enhance these outcomes.12345
What data supports the effectiveness of the drug combination Eltrombopag and Immunosuppression for Severe Aplastic Anemia?
Research shows that adding eltrombopag to standard immunosuppressive therapy, which includes horse antithymocyte globulin and cyclosporine, improves the treatment's effectiveness in patients with severe aplastic anemia. Eltrombopag, when used with immunosuppression as a first-line treatment, increases the rate of overall and complete responses.12345
What safety data exists for Eltrombopag and immunosuppression in severe aplastic anemia?
Eltrombopag, when added to standard immunosuppressive therapy (horse ATG plus cyclosporine), has shown improved efficacy in severe aplastic anemia. It has been approved for patients 2 years and older, although safety data in pediatric cohorts is limited. Eltrombopag as a single agent has shown hematologic response rates of 40% to 50% in those refractory to initial immunosuppression. When combined with immunosuppression as frontline therapy, it increased overall and complete response rates, with a favorable comparison in clonal cytogenetic abnormalities to immunosuppression alone. Longer follow-up is needed to better define long-term risks.12467
Is the combination of Eltrombopag and immunosuppression safe for humans?
Eltrombopag, when combined with immunosuppressive therapy like horse antithymocyte globulin and cyclosporine, has been used in severe aplastic anemia and generally shows good safety with improved response rates. However, longer follow-up is needed to fully understand long-term safety, especially in children.12467
Is the drug combination of Cyclosporine and Horse-ATG promising for treating Severe Aplastic Anemia?
Yes, the combination of Cyclosporine and Horse-ATG is promising for treating Severe Aplastic Anemia. It is a standard treatment that shows good response rates, and when combined with the drug Eltrombopag, it can significantly improve patient outcomes, leading to high response rates and better survival.12368
What makes the drug combination of eltrombopag, cyclosporine, and horse-ATG unique for treating severe aplastic anemia?
The combination of eltrombopag with cyclosporine and horse-ATG is unique because eltrombopag, a thrombopoietin receptor agonist, enhances the effectiveness of standard immunosuppressive therapy, leading to higher overall and complete response rates and achieving transfusion independence in many patients.12468
What is the purpose of this trial?
Background:* Severe aplastic anemia is a rare and serious blood disorder. It happens when the immune system starts to attack the bone marrow cells. This causes the bone marrow to stop making red blood cells, platelets, and white blood cells. Standard treatment for this disease is horse-ATG and cyclosporine, which suppress the immune system and stop it from attacking the bone marrow. However, this treatment does not work in all people. Some people still have poor blood cell counts even after treatment.* Eltrombopag is a drug designed to mimic a protein in the body called thrombopoietin. It helps the body to make more platelets. It may also cause the body to make more red and white blood cells. Studies have shown that eltrombopag may be useful when added to standard treatment for severe aplastic anemia. It may help improve poor blood cell counts.Objectives:- To test the safety and effectiveness of adding eltrombopag to standard immunosuppressive therapy for severe aplastic anemia.Eligibility:- Individuals at least 2 years of age who have severe aplastic anemia that has not yet been treated.Design:* Participants will be screened with a physical exam, medical history, and blood tests. Blood and urine samples will be collected.* Participants will start treatment with horse-ATG and cyclosporine. Treatment will be given according to the standard of care for the disease.* Cohort 1: After 14 days, participants will start taking eltrombopag. They will take eltrombopag for up to 6 months.* Cohort 2: After 14 days, participants will start taking eltrombopag. They will take eltrombopag for up to 3 months.* Cohort 3 and Extension Cohort: Participants will start taking eltrombopag on Day 1. They will take eltrombopag for up to 6 months.* Participants may receive other medications to prevent infections during treatment.* Treatment will be monitored with frequent blood tests. Participants will also fill out questionnaires about their symptoms and their quality of life.
Research Team
Neal S Young, M.D.
Principal Investigator
National Heart, Lung, and Blood Institute (NHLBI)
Eligibility Criteria
This trial is for individuals aged 2 years and older with severe aplastic anemia who haven't been treated yet. They must have specific low blood cell counts, weigh over 12 kg, and not have certain liver issues or infections. People with prior immunosuppressive therapy, pregnancy, or severe concurrent diseases can't participate.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive horse-ATG and cyclosporine, with eltrombopag added at different times depending on the cohort
Follow-up
Participants are monitored for safety, effectiveness, and potential clonal evolution after treatment
Extension Cohort
Participants in the extension cohort receive continued treatment with eltrombopag and cyclosporine
Treatment Details
Interventions
- Cyclosporine
- Eltrombopag
- Horse-ATG
Cyclosporine is already approved in European Union, United States, Canada, Japan for the following indications:
- Prevention of organ rejection in kidney, liver, heart, lung, pancreas, and bone marrow transplants
- Treatment of severe psoriasis
- Treatment of nephrotic syndrome
- Prevention of organ rejection in kidney, liver, and heart transplants
- Treatment of severe rheumatoid arthritis
- Treatment of severe psoriasis
- Prevention of organ rejection in kidney, liver, heart, lung, pancreas, and bone marrow transplants
- Treatment of severe psoriasis
- Treatment of nephrotic syndrome
- Prevention of organ rejection in kidney, liver, and heart transplants
- Treatment of severe rheumatoid arthritis
- Treatment of severe psoriasis
Find a Clinic Near You
Who Is Running the Clinical Trial?
National Heart, Lung, and Blood Institute (NHLBI)
Lead Sponsor
Novartis
Industry Sponsor
Vasant Narasimhan
Novartis
Chief Executive Officer since 2018
MD from Harvard Medical School, Bachelor's in Biological Sciences from University of Chicago, Master's in Public Policy from John F. Kennedy School of Government
Shreeram Aradhye
Novartis
Chief Medical Officer since 2022
MD from Yale University, MSc in Clinical Epidemiology from University of Pennsylvania