Evaluate the Real-World Safety Outcomes and Clinical Efficacy of Ponatinib and Other Tyrosine Kinase Inhibitors among Chronic Myeloid Leukemia Patients

Study type

• Study topic:
  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Effectiveness study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medical condition to be studied:
  Chronic myeloid leukaemia

Population studied

• Short description of the study population:
  The study population included patients with chronic myeloid leukemia received treatment with ponatinib and other tyrosine kinase inhibitors identified from Humedica EMR data between October 1, 2012 to September 30, 2017.
• Age groups:
  • Adults (18 to < 46 years)
  • Adults (46 to < 65 years)
  • Adults (65 to < 75 years)
  • Adults (75 to < 85 years)
  • Adults (85 years and over)
• Special population of interest:
  Other
• Special population of interest, other:
  Patients with chronic myeloid leukemia
• Estimated number of subjects:
  1769

Study design details

• Main study objective:
  The main objective of this study is using Humedica Electronic Health Records (EHR) data from October 1, 2012—September 30, 2017 (or the most recent 5 years of data), STATinMED Research proposes to evaluate the real-world treatment patterns and clinical outcomes of ponatinib and other Tyrosine Kinase Inhibitors (TKIs) among patients with prior TKI uses among CP-CML participants.
• Outcomes:
  Participants Categorized by Socio-demographic Variables, Clinical Characteristics of Disease,Comorbidities Severity,Concomitant Medication,Bone Marrow Stem Cell Transplant,Major Adverse Cardiac Event(MACE),Arterial Occlusive Event(AOE), and Venous Thrombotic Events(VTE),Quan-Charlson Comorbidity Index Score,Number of Previous Treatments of TKI Drugs,Duration From Last TKI Run-out to Index Date. Participants With BCR-ABL,Bone Marrow Testing, Disease Severity as per Medstat Disease Staging Clinical Criteria Version 5.21, Treatment-Free Gap of the Index Treatment,Treatment Patterns Based on Duration of Index Treatment,Mean Starting Daily Dose and Average Daily Dose,Number of Participants With CML on Concomitant Medication. Disease Progression, Progression Free Survival(PFS).
• Data analysis plan:
  All variables will first be analyzed descriptively and compared across ponatinib, bosutinib, and other TKIs (imatinib, dasatinib, or nilotinib) for patients with prior TKI use. Numbers and percentages will be provided for dichotomous and polychotomous variables. Means and standard deviations will be provided for continuous variables. For dichotomous and polychotomous variables, p-values will be calculated according to the chi-square test, for continuous variables, t-tests will be used to calculate p-values. Ponatinib will be considered as the reference group for the comparisons.