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After exiting the RACE trial (NCT02099747) patients will be invited to participate in this long term follow-up study
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| RACE 1 patients | After exiting the RACE trial (NCT02099747) patient will be invited to participate in this study |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ATGAM plus CsA with or without Eltrombopag | Drug | Standard treatment with or without Eltrombopag |
|
| Measure | Description | Time Frame |
|---|---|---|
| Failure Free Survival | 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) | 15 years |
| Measure | Description | Time Frame |
|---|---|---|
| Response Rate: number of patients who reach a hematological response | Response Rate | 15 years |
| Overall Survival | Overall Survival | 15 years |
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Inclusion Criteria:
Exclusion Criteria:
None
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Patients who participated in the initial RACE 1 trial
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| Name | Affiliation | Role |
|---|---|---|
| Regis Peffault de Latour, Prof, MD | Hospital St. Louis, Paris, France | Principal Investigator |
| Antonio M Risitano, MD, PhD | Hospital Avellino, Napels, Italy | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hospital St. Louis | Paris | France |
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| ID | Term |
|---|---|
| D000741 | Anemia, Aplastic |
| ID | Term |
|---|---|
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000080983 | Bone Marrow Failure Disorders |
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| ID | Term |
|---|---|
| D000961 | Antilymphocyte Serum |
| C520809 | eltrombopag |
| ID | Term |
|---|---|
| D007106 | Immune Sera |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
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| Cumulative incidence of relapse after response | Cumulative incidence of relapse after initial hematological response (complete or partial) | 15 years |
| Cumulative incidence of clonal evolution | 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) | 15 years |
| Cumulative incidence of clinical Paroxysmal nocturnal hemoglobinuria (PNH) | Cumulative incidence of clinical PNH (hemolysis and/or thromboembolism), and of need of anti-complement treatment | 15 years |
| Cumulative incidence of Solid Tumors | Cumulative incidence of solid tumours | 15 years |
| Number of patients who need a Human Stem Cell Transplantation (HSCT) | Need for HSCT | 15 years |
| Number of patients who need additional IST | Need for additional intensive Imune Suppressive Therapy (IST) (e.g. ATG, alemtuzumab or cyclophosphamide-based, or any other lymphocyte-depleting agent) | 15 years |
| Number of patients who need Maintenance IST (e.g. CsA beyond 2 years) | Need for maintenance intensive IST (e.g. CsA beyond 2 years) | 15 years |
| Number of patients who need additional Eltrombopag (EPAG) | Need for additional EPAG | 15 years |
| Number of patients who need any other approved Aplastic Anemia (AA) treatment | Need for any other approved Aplastic Anemia (AA) treatment | 15 years |
| Monitoring of Clonal Hematopoiesis of Indetermined Potential (CHIP) | As tracked by somatic mutations in genes associated with myeloid disorders | 15 years |
| D001855 | Bone Marrow Diseases |
| D001798 |
| Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D001688 | Biological Products |
| D045424 | Complex Mixtures |