Romiplostim in treatment-refractory acquired aplastic anemia
Phase 2
Recruiting
- Conditions
- Acquired aplastic anemia.Idiopathic aplastic anemiaD61.3
- Registration Number
- IRCT20221129056655N1
- Lead Sponsor
- Shiraz University of Medical Sciences
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 24
Inclusion Criteria
Children up to the age of 18 years with non-severe transfusion-dependent AA, severe and very severe acquired aplastic anemia who are ineligible to be treated with IST (ATG plus cyclosporine) or are refractory to IST are enrolled.
Exclusion Criteria
1.Patients with inherited aplastic anemia
2.Diagnosed as having acute myeloid leukemia (AM)L or myelodysplastic syndrome (MDS)
3.Having active malignancies, or having a history of the treatment of malignancies within 5 years prior to informed consent.
4.Concurrent paroxysmal nocturnal hemoglobinuria
5.History of chromosome aberrations discovered in bone marrow cells.
6.Bone marrow fibrosis based on reticulin stain
7.Planned hematopoietic stem cell transplantation during the study
Study & Design
- Study Type
- interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method The percentage of patients who achieved complete or partial hematologic response at the end of study. Timepoint: Week 27. Method of measurement: To divide the number of patients with complete or partial hematologic response over total number of patients.
- Secondary Outcome Measures
Name Time Method The time to achieve complete or partial hematologic response. Timepoint: weekly. Method of measurement: CBC.;Reduction in the need to platelet or blood transfusion. Timepoint: Week 27. Method of measurement: The proportion of patients who achieved transfusion independence or reduction in transfusion needs among patients who had received a transfusion within 8 weeks prior to the first romiplostim administration.