Acquired aplastic anemia. Idiopathic aplastic anemia
Conditions
Interventions
Intervention group: Romiplostim (N-Plate, Amgen company) is administered subcutaneously at a fixed dose of 10 mcg/kg weekly for 4 weeks (weeks 1-4). The drug is administered on day 1 of IST (horse ATG
Sponsors
Shiraz University of Medical Sciences
Eligibility
Sex/Gender
All
Age
1 Months to 18 Years
Inclusion criteria
Inclusion criteria: 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)
Exclusion criteria
Exclusion criteria: 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Complete or partial hematologic response at the end of study. Timepoint: At week 27 of the study. Method of measurement: Check CBC. | — |
Secondary
| Measure | Time frame |
|---|---|
| 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. | — |
Countries
Iran (Islamic Republic of)
Contacts
Public ContactMohammadreza Bordbar
Shiraz University of Medical Sciences
Outcome results
None listed