Health Condition 1: D610- Constitutional aplastic anemia
Conditions
Interventions
Intervention1: Inj Romiplostim: Study Drug Inj Romiplostim, a thrombopoietin Receptor agonist, will be administered subcutaneously on weekly basis starting at the dose of 3mcg per kg and escalated by
Sponsors
INDIAN COUNCIL OF MEDICAL RESEARCH
Eligibility
Inclusion criteria
Inclusion criteria: Patients with IBMFS including Fanconi anemia, dyskeratosis congenita causing cytopenias Hb less than 8 gram per decilitre, ANC less than 500 cells or Platelet count less than 30,000 per microlitre or bone marrow cellularity less than 25 percent and with danazol resistance or intolerance
Exclusion criteria
Exclusion criteria: Known hypersensitivity or past use of Romiplostim, history of thrombosis, patients with IBMF with co-existing myelodysplasia or malignancies and organ dysfunction; presence of FANCD mutation
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| A. Twenty five percent reduction in transfusion requirement compared to baseline B. Achieving platelet count more than 30,000 per microlitre, Hb more than 8gram per decilitre or ANC more than 500 per microlitre compared to baseline C. Transfusion independence for 4 consecutive weeks D. Bone marrow response, defined as an increase in bone marrow cellularity by at least two-fold from baseline. Timepoint: 12 and 24 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| The following known drug related adverse reactions will be monitored 1. Occurrence of clonal evolution 2. Thrombotic events 3. Allergy/ hypersensitivity 4. Pain at injection site 5. Headache 6. Myalgia 7. Fatigue 8. Dizziness 9. Transaminitis 10. Any other symptom reported by the patient up to 4 weeks after study completion. Along with disease related adverse events like 1. Infection 2. Anemia 3. Bleeding Timepoint: weekly basis from week 1 to week 36 | — |
Countries
India
Contacts
Public ContactAruna Rajendran
Madras Medical College
Outcome results
None listed