Severe Aplastic Anemia
Conditions
Brief summary
After exiting the RACE trial (NCT02099747) patients will be invited to participate in this long term follow-up study
Detailed description
Patients will be followed up annually, according to standard of care. All diagnostic and therapeutic intervention will be performed according to standard of care, at discretion of the treating physician. In particular, during the study no extra Peripheral blood or Bone Marrow sampling will be performed, in addition to routine sampling for morphology and karyotype surveillance. Molecular analysis by Next Generation Sequencing (NGS) will also be collected if the centre is doing this on a routine basis. No Investigational Medicinal Product (IMP) or Non-Investigational Medicinal Product (NIMP) will be given to the patients.
Interventions
DRUGATGAM plus CsA with or without Eltrombopag
Standard treatment with or without Eltrombopag
Sponsors
European Society for Blood and Marrow Transplantation
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Inclusion criteria
1. Subject participated in the RACE trial (NCT02099747, EudraCT number: 2014-000363-40) during which patient received ATGAM, Cyclosporine A with or without Eltrombopag. 2. Subject has provided informed consent to participate in long-term data collection
Exclusion criteria
None
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Failure Free Survival | 15 years | Failure Free Survival, where treatment failure is defined as one or more of the following: death, relapse, malignant clonal evolution, need for further (e.g. transplant) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Overall Survival | 15 years | Overall Survival |
| Cumulative incidence of relapse after response | 15 years | Cumulative incidence of relapse after initial hematological response (complete or partial) |
| Cumulative incidence of clonal evolution | 15 years | Cumulative incidence of clonal evolution: Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS) or karyotypic abnormalities qualifying for the diagnosis of MDS (see World Health Organization (WHO) 2016) |
| Cumulative incidence of clinical Paroxysmal nocturnal hemoglobinuria (PNH) | 15 years | Cumulative incidence of clinical PNH (hemolysis and/or thromboembolism), and of need of anti-complement treatment |
| Cumulative incidence of Solid Tumors | 15 years | Cumulative incidence of solid tumours |
| Response Rate: number of patients who reach a hematological response | 15 years | Response Rate |
| Number of patients who need additional IST | 15 years | Need for additional intensive Imune Suppressive Therapy (IST) (e.g. ATG, alemtuzumab or cyclophosphamide-based, or any other lymphocyte-depleting agent) |
| Number of patients who need Maintenance IST (e.g. CsA beyond 2 years) | 15 years | Need for maintenance intensive IST (e.g. CsA beyond 2 years) |
| Number of patients who need additional Eltrombopag (EPAG) | 15 years | Need for additional EPAG |
| Number of patients who need any other approved Aplastic Anemia (AA) treatment | 15 years | Need for any other approved Aplastic Anemia (AA) treatment |
| Monitoring of Clonal Hematopoiesis of Indetermined Potential (CHIP) | 15 years | As tracked by somatic mutations in genes associated with myeloid disorders |
| Number of patients who need a Human Stem Cell Transplantation (HSCT) | 15 years | Need for HSCT |
Countries
France
Outcome results
None listed