Stopping Tyrosine Kinase Inhibitors in Affecting Treatment-Free Remission in Patients With Chronic Phase Chronic Myeloid Leukemia

Titre officiel

Stopping Tyrosine Kinase Inhibitors (TKI) to Assess Treatment-Free Remission (TFR) in Pediatric Chronic Myeloid Leukemia - Chronic Phase (CML-CP)

Sommaire:

Cet essai de phase II vise à évaluer les effets de l’arrêt d’un traitement par des inhibiteurs de la tyrosine kinase (ITK) sur la rémission sans traitement chez des patients atteints de leucémie myéloïde chronique en phase chronique. Lorsque le niveau de la maladie est très faible, on parle de rémission moléculaire. Les ITK sont un type de médicaments qui permet de maintenir ce faible niveau. Cependant, si la rémission moléculaire dure depuis un certain temps, il se peut que les inhibiteurs de la tyrosine kinase ne soient pas nécessaires. On ne sait pas encore si l’arrêt des inhibiteurs de la tyrosine kinase aidera les patients atteints de leucémie myéloïde chronique en phase chronique à conserver ou à réobtenir une rémission moléculaire.

Description de l'essai

Primary Outcome:

  • Treatment-free remission (TFR)
  • Major molecular remission (MMR/MR3)
Secondary Outcome:
  • Clinical factors and laboratory correlates affecting persistence of MMR

PRIMARY OBJECTIVES:

I. To determine the 2-year treatment free remission (TFR) rate of children, adolescents, and young adults with chronic myeloid leukemia - chronic phase (CML-CP) following discontinuation tyrosine kinase inhibitor (TKI).

II. To estimate the re-induction rate and maintenance of major molecular remission (MMR/MR3) at 1 year after restarting TKI for children, adolescents, and young adults.

SECONDARY OBJECTIVES:

  • 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).

EXPLORATORY OBJECTIVES:

  • To describe change in height standard deviation score over time in patients who are able to discontinue their TKI.
  • 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.
  • To describe differences in patient-reported health status after stopping TKIs, including those who need to resume TKI after stopping.
  • To describe the incidence and characteristics of TKI withdrawal syndrome in children.
  • 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.

OUTLINE:

Patients stop taking TKI medication within 10 days after enrollment. Patients undergo peripheral blood collection to monitor loss of MMR every 4 weeks in year 1, every 6 weeks in year 2, and every 12 weeks in year 3. Patients who lose their molecular remission may restart TKI medication and are monitored every 4 weeks in year 1, every 6 weeks in year 2, and every 12 weeks in year 3.

After completion of study treatment, patients are followed up annually.

Voir cet essai sur ClinicalTrials.gov

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