Romiplostim in treatment-naïve acquired aplastic anemia
- Conditions
- Acquired aplastic anemia.Idiopathic aplastic anemiaD61.3
- Registration Number
- IRCT20221129056655N2
- Lead Sponsor
- Shiraz University of Medical Sciences
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 23
Children 1 month up to the age of 18 years with severe and very severe acquired aplastic anemia who are eligible to be treated with IST (ATG plus cyclosporine)
1.Patients with inherited aplastic anemia
2.Previously treated with ATG, cyclosporine
3.Diagnosed as having acute myeloid leukemia (AM)L or myelodysplastic syndrome (MDS)
4.Concurrent active infection not adequately responding to appropriate therapy
5.Having active malignancies, or having a history of the treatment of malignancies within 5 years prior to informed consent
6.Concurrent paroxysmal nocturnal hemoglobinuria
7.History of chromosome aberrations discovered in bone marrow cells
8.Bone marrow fibrosis based on reticulin stain
9.Active or latent HIV infection
10.Active or latent HBV infection
11.Active or latent HCV infection
12.Active or latent tuberculous infection
13.Active or latent visceral leishmaniasis
14.Planned hematopoietic stem cell transplantation during the study
Study & Design
- Study Type
- interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Complete or partial hematologic response at the end of study. Timepoint: At week 27 of the study. Method of measurement: Check CBC.
- Secondary Outcome Measures
Name Time Method The time to achieve complete or partial hematologic response. Timepoint: weekly. Method of measurement: CBC.;The need to platelet or blood transfusion. Timepoint: Week 27. Method of measurement: The proportion of patients with transfusion independence or reduction in transfusion needs among patients who had received a transfusion within 8 weeks prior to the first romiplostim administration.