Acquired aplastic anemia. Idiopathic aplastic anemia
Conditions
Interventions
Intervention group: Romiplostim is administered subcutaneously at a fixed dose of 10 mcg/kg weekly for 4 weeks (weeks 1-4) followed by titrated dose steps of 10, 15, and 20 mcg/kg once weekly up to 2
Sponsors
Shiraz University of Medical Sciences
Eligibility
Sex/Gender
All
Age
1 Months to 18 Years
Inclusion criteria
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
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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. | — |
Countries
Iran (Islamic Republic of)
Contacts
Public ContactMohammadreza Bordbar
Shiraz University of Medical Sciences
Outcome results
None listed