Severe aplastic anaemia Haematological Disorders Other aplastic anaemias
Conditions
Interventions
Combined immunosupressive therapy with cyclosporin A and either horse ATG (ATGAM, Apjohn) - standard arm or rabbit ATG (ATG-Fresenius, Fresenius) - study arm.
Horse ATG (ATGAM) was used in a standard
Sponsors
Federal Clinical Research Center of Pediatric Hematology, Oncology and Immunology (Russia)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Aged from 1 - 18 years, either sex 2. Diagnosis of severe aquiered aplastic anaemia
Exclusion criteria
Exclusion criteria: 1. Previous immune supressive therapy with ATG and/or cyclosporin A 2. Previous corticosteroid therapy over 2 weeks 3. Inherited bone marrow failure syndrome 4. Uncontrolled invasive fungal infection
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Minimal haematologic response rate at day 180 from therapy start 2. Complete haematologic response rate, assessed at last follow-up (5 years from time of enrolment of the last patient) 3. Overall survival, assessed at last follow-up (5 years from time of enrolment of the last patient) | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Relapse probability in patients who achieved haematologic response, assessed at last follow-up (5 years from time of enrolment of the last patient) 2. Event-free survival, assessed at last follow-up (5 years from time of enrolment of the last patient) 3. Toxicity, assessed at day 30 from therapy start | — |
Countries
Russian Federation
Outcome results
None listed