Aplastic Anemia
Conditions
Keywords
NIS, Pediatric Aplastic Anemia, AA, Revolade Tablets, eltrombopag, Drug-use Survey
Brief summary
This is a multicenter, single-arm, non-interventional study (NIS) to confirm the safety and efficacy of eltrombopag in Anti-Thymocyte Globulin (ATG) treatment naive pediatric patients with aplastic anemia (AA).
Detailed description
The objective of this survey is to confirm the safety and efficacy of eltrombopag in ATG treatment naive pediatric patients with AA. Eltrombopag should be administered according to the dosage and administration specified in the latest version of the package insert. The observation period is 1 year (364 days) from the start of treatment with this product, regardless of whether treatment with eltrombopag is continued or not. However, if hematopoietic stem cell transplantation is performed within 1 year after the start of treatment with eltrombopag, the observation period shall be until the date of hematopoietic stem cell transplantation.
Interventions
OTHEReltrombopag
This is an observational study. There is no treatment allocation. After confirming that patients are fulfilling the eligibility criteria, patients will be registered in this survey.
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
6 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
* Patients whose legally acceptable representative has given written consent for cooperation in this survey prior to enrollment in this survey * Patients aged ≥ 6 years and \< 18 years at the start of treatment with eltrombopag * Pediatric patients with AA who receive eltrombopag for the first time in combination with ATG after the approval of additional dosage and administration for "ATG-naïve pediatric patients with AA"
Exclusion criteria
* Patients who have received ATG without concomitant use of eltrombopag * Patients with congenital AA * Patients with suspected or confirmed diagnosis of myelodysplastic syndrome (MDS) at the start of treatment with eltrombopag * Patients who have received any drug products containing the same ingredient as eltrombopag (including investigational products)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Occurrence of serious adverse events | Up to 1 year | Occurrence of serious adverse events to be provided |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Occurrence of adverse events and adverse drug reactions leading to treatment discontinuation | Up to 1 Year | Occurrence of adverse events and adverse drug reactions leading to treatment discontinuation to be provided |
| Occurrence of adverse events and adverse drug reactions applicable to safety specifications (hepatic dysfunction and hematopoietic malignancies) | Up to 1 year | Occurrence of adverse events and adverse drug reactions applicable to safety specifications (hepatic dysfunction and hematopoietic malignancies) |
| Occurrence of clonal evolution | Up to 1 year | Occurrence of clonal evolution \[cytogenetic abnormalities, myelodysplastic syndrome (MDS), Acute Myeloid Leukemia (AML), and paroxysmal nocturnal hemoglobinuria (PNH)\] |
| Hematologic response status | Up to 1 year | Hematologic response status to be provided |
| Change over time from baseline in platelet count | Baseline, 1 year | Change over time from baseline in platelet count to be provided |
| Change over time from baseline in hemoglobin | Baseline, 1 year | Change over time from baseline in hemoglobin to be provided |
| Change over time from baseline in neutrophil count | Baseline, 1 year | Change over time from baseline in neutrophil count to be provided |
| Change over time from baseline in reticulocyte count | Baseline, 1 year | Change over time from baseline in reticulocyte count to be provided |
| Change over time from baseline in transfusion dependence | Baseline, 1 year | Change over time from baseline in transfusion dependence to be provided |
| Change over time from baseline in transfusion volume | Baseline, 1 year | Change over time from baseline in transfusion volume to be provided |
Countries
Japan
Contacts
CONTACTNovartis Pharmaceuticals
STUDY_DIRECTORNovartis Pharmaceuticals
Novartis Pharmaceuticals
Outcome results
None listed