Outcomes in Bone Marrow Aplasia.
Long Term Outcomes of Eltrombopag in Patients With Bone Marrow Aplasia, Assiut University Hospital Insight.
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Eltrombopag (E-PAG) is an oral synthetic small-molecule, noncompetitive, TPO agonist that initially was approved by the US Food and Drug Administration (FDA) for the treatment of chronic immune thrombocytopenic purpura. Single-agent activity of E-PAG was demonstrated in at least 1 lineage in 40 to 45% of patients with AA that was refractory to IST, leading to its approval by the FDA in this setting (5).
Eltrombopag evades cytokines blockade of c-MPL signaling and activates the c-MPL receptor by interacting with the transmembrane receptor domain, resulting in a conformational change without competing with TPO. Regarding AA, it is possible that eltrombopag promotes DNA repair in hematopoietic stem cells and progenitor cells. However, AA may appear to evolve to other hematologic diseases, most notably paroxysmal nocturnal hemoglobinuria and myelodysplastic syndrome, even to acute myeloid leukemia, and about 15% of patients evolve to myelodysplastic syndrome, acute myeloid leukemia or both after immunosuppressive therapy. Therefore, it is unclear but alarming that the use of eltrombopag exacerbates the clone evolution (6).
Eltrombopag and cyclosporin was active as front-line treatment of severe aplastic anaemia, with no unexpected safety concerns. This approach might be beneficial where horse-ATG is not available or not tolerated.
Study Type
Enrollment (Estimated)
Phase
- Phase 3
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Adult
- Older Adult
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- Age > 18. Newely diagnosed bone marrow aplasia Eastern Cooperative Oncology Group (ECOG) performance status of 0-2. Patients started CSA plus Eltrombopag therapy Normal cardiac, hepatic & renal functions
Exclusion Criteria:
Hypersensitivity or contraindications to eltrombopag. Cardiovascular, pulmonary, hepatic, or renal diseases. History of malignancy. Pregnant, breastfeeding. Inherited bone marrow aplasia. Secondry bone marrow aplasia Previous thromboembolic events. Previous malignancies either solid or hematologic.
-
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
|---|---|
|
Experimental: Cases of aplastic anemia recieving Eltrombopag
Newely diagnosed bone marrow aplasia starting treatment with Eltrombopag in adose of 50-150mg / day
|
Treatment with eltrombopag in a dose of (50-150mg/d)
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Determine hematological response after 6 months
Time Frame: 6 months to 5 years
|
Change in CBC elements after 6 months of treatment
|
6 months to 5 years
|
|
Determine overall survival rate.
Time Frame: 5 years
|
Number if years estimated to survive after treatment
|
5 years
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
|---|---|
|
Rate of relapse in patients deemed responsers at 6 months.
Time Frame: Around 1 year
|
Around 1 year
|
|
Clonal evolution to myeloid malignancy or new chromosomal abnormality
Time Frame: Around 2 years
|
Around 2 years
|
|
Change in serum iron and ferritin over time of treatment
Time Frame: From 6 months to 5 years
|
From 6 months to 5 years
|
|
Eltrombopag efficacy in increasing platelet count
Time Frame: After6 months
|
After6 months
|
|
Adverse effects raelated to treatment
Time Frame: Within 2 years
|
Within 2 years
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Estimated)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- Noha Mahmoud Muhammed
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
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