Grants and Contracts Details
Stopping Tyrosine Kinase Inhibitors (TKI) to Assess Treatment-Free Remission (TFR) in Pediatric Chronic Myeloid Leukemia - Chronic Phase (CML-CP) 1.0 GOALS AND OBJECTIVES (SCIENTIFIC AIMS) 1.1 Primary Aims 1.1.1 To determine the 2-year treatment free remission (TFR) rate of children, adolescents, and young adults with CML-CP following discontinuation TKI. 1.1.2 To estimate the re-induction rate and maintenance of molecular remission (BCR-ABL1 ≤ 0.1%) at 1 year after restarting TKI for children, adolescents, and young adults. 1.2 Secondary Aim 1.2.1 To describe clinical factors and laboratory correlates affecting the persistence of MMR and re-initiation of treatment after stopping TKI (e.g. patient demographics, duration and level of prior molecular remission, duration and type of TKI, clinical presentation at diagnosis and immune studies). 1.3 Exploratory Aims 1.3.1 To describe change in height standard deviation score over time in patients who are able to discontinue their TKI. 1.3.2 To describe the long-term health outcomes including but not limited to gonadal function, endocrine function, and bone metabolism in patients who are able to discontinue TKI as well as those that need to restart TKIs. 1.3.3 To describe differences in patient-reported health status after stopping TKIs, including those who need to resume TKI after stopping. 1.3.4 To describe the incidence and characteristics of TKI withdrawal syndrome in children. 1.3.5 To evaluate changes in neurocognitive outcomes of patients enrolled on this study using a patient-completed, performance-based, computerized measure of neuropsychological functioning and a parent-report/self-report questionnaire.
|Effective start/end date||4/5/21 → 9/9/21|
- Childrens Hospital of Philadelphia: $2.00
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