TOPASE = Therapeutic Observatory of Ponatinib About Safety and Efficacy [France]

What is the purpose of the study

This is a non-interventional study. Ponatinib will be prescribed in the usual manner in accordance with the terms of the marketing authorization. All treatment decisions are made at the discretion of the patient’s healthcare provider (HCP) as per indication and are not mandated by the study design or protocol.

The study aims at better qualifying the benefit-risk balance of ponatinib in real life and in relation with the treatment history of CML patients therapeutic history.

The patient’s health status will be evaluated during a 24-month follow-up. Time points for patient evaluation will be at the discretion of the investigator. It is anticipated that these will occur approximately every 3 months the first year, and then every 6 months.

• Effectiveness will be assessed on the basis of hematologic, cytogenetic and molecular response rates and survival duration (overall and progression-free) according to the center practices.
• Tolerance will be assessed by the collection of adverse events.

Key inclusion criteria

This study includes patients who:
– are aged ≥18 years and agree to participate in the study
– are presenting CML in any phase
– have been receiving treatment with ponatinib for less than six months
– have the ability to understand the requirements of the study and to comply with the study data collection procedures

Key exclusion criteria

Where can I find additional information

“Last Interim Results of the Real-Life Study Evaluating the Efficacy and Safety of Ponatinib “Topase” in Patients with TKI-Resistant or Intolerant CML” published in the medical journal “Blood” (2022)

Study centers / principal investigators

France

Paris Sud University Hospitals
Prof. Ali Turhan

Centre Hospitalier Universitaire de Lille
Dr. Valérie Coiteux