Health Condition 1: D618- Other specified aplastic anemias and other bone marrow failure syndromes
Conditions
Interventions
Intervention1: Antithymocyte globulin (ATG)+Cyclosporine A(CSA)+Romiplostim: Immunosuppression ATG 40mg/kg/day for 4 days, Cyclosporine 5 mg/kg/day for 6 to 12 months and Romiplostim 10 µgm/kg/week fo
Sponsors
Indian Council of Medical Research- National Institute of Immunohaematology
Eligibility
Inclusion criteria
Inclusion criteria: Newly diagnosed acquired AA patients (Patients with hypocellular marrow i.e Less than 25% marrow cellularity; peripheral blood pancytopenia) of any gender aged 18 years to 45 years, willing for consent and follow up will be recruited for the study. Negative for chromosomal breakages in MMC induced peripheral blood culture (negative for Fanconi’s anemia). Willing to follow the study protocol. No history of infections, chemical exposure or radiation therapy. Treatment naive AA. ECOG performance status 1-2
Exclusion criteria
Exclusion criteria: Aplastic anemia patients positive for chromosomal breakages in PHA stimulated, MMC induced peripheral blood cultures and other inherited bone marrow failure syndromes. Secondary marrow aplasia (secondary to HSCT, drug or chemical exposure, radiotherapy, hepatitis), Patients with MDS Aplastic anemia Patients planning for HSCT. History of allergy to the drug ingredients. Participating in other drug clinical trials in the past 1 month. Known case of Human Immunodeficiency Virus, active Hepatitis C infection or Hepatitis B infection. Women who are pregnant or breast feeding or women of childbearing potential not willing to follow double contraceptive measures. Any other reason that in the opinion of the investigator is likely to cause harm to the participant or will adversely affect the results of the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The study will establish safety of combine therapy of ROM and IST vs ELT and IST as first line of therapy in Indian AA. Proportion of patients with complete response (CR) (hematological CR defined by hemoglobin level more than 10 g/dL, absolute neutrophil count more than 1000 and platelets more than 100 X109 cells/L) Proportion of patients with partial response (PR) (PR defined as no longer meeting the criteria for SAA and transfusion independency with hemoglobin level more than 8 g/dL, absolute neutrophil count than 500, and platelet count more than20 X 109/L). Proportion of Non-responder (NR)(NR defined as any patient not meeting any of the response criteria defined above). Timepoint: Time from recruitment to the primary outcome will be 6 months followed by 3 months follow up. | — |
Secondary
| Measure | Time frame |
|---|---|
| The study will identify a differential gene expression pattern in AA patients treated with TPO-R agonist (ELT or ROM) in combination with IST.Timepoint: 3 Years;Identification of prime biomarkers in responders and non-responders at the time of diagnosis.Timepoint: 3 Years;Correlation of genomic changes and expression profile of AA patients with response and non-response to the therapy.Timepoint: 3 Years;To correlate response-based reticulocyte count, PNH clone, lineage specific differential gene expression pattern identified through ScRNA seq.Timepoint: 3 Years;Hematological profile at 3, 6 and 12 months.Timepoint: 3,6 and 12 months;Volume of blood and platelets transfused at the end of 3, 6 and 12 months (in case of transfusion dependence).Timepoint: 3,6 and 12 months;To correlate the trough concentrations of ROM+IST with safety and efficacy parametersTimepoint: 3 Years;Change in quality of life at 6 months as assessed by Transfusion-dependent Quality of Life (TranQoL) questionnaire and the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ) scoresTimepoint: 6 months;Incidence of treatment emergent adverse events [TEAEs] of grade 3 or more severity at 12 months [As per Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 dated 27 November 2017]Timepoint: 12 months | — |
Countries
India
Contacts
Public ContactDr Chandrakala Shanmukhaiah
ICMR National Institute of Immunohaematology
Outcome results
None listed