Cross-sectional study to assess the effectiveness of the patients’ alert card to inform on the risk of differentiation syndrome in AML patients treated with TIBSOVO® (Ivosidenib) (IMPACTA)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cross-sectional

Study drug and medical condition

• Medicinal product name:
  TIBSOVO
• Study drug International non-proprietary name (INN) or common name:
  IVOSIDENIB
• Anatomical Therapeutic Chemical (ATC) code:
  (L01XM02) ivosidenib

  ivosidenib
• Medical condition to be studied:
  Acute myeloid leukaemia

Population studied

• Short description of the study population:
  Patients will be identified through their Healthcare Professionals (HCPs) – (physicians and nurses) and will be included in the study if they are aged ≥18 years at recruitment, have taken Ivosidenib for newly diagnosed AML in the previous 6 months, and are able to understand and complete the consent form and patient survey.; Participant selection will be based on systematic sampling, i.e., all consecutive eligible participants are expected to be included in the study, with no selection other than the eligibility criteria and the study period defined. ; Physicians will be identified using the lists of AML specialists provided by the Marketing Authorisation Holder. A pilot step will be conducted to determine the feasibility of recruitment and the participants/Healthcare professionals (HCPs) ratio to be used in the study.
• Estimated number of subjects:
  66

Study design details

• Study design:
  This study will be a multi-national, observational cross-sectional survey among adult patients who have recently received TIBSOVO® (Ivosidenib) for treatment of AML, that will be conducted in at least three countries in the European Union (EU). ;
• Main study objective:
  The study will evaluate the effectiveness of the PACs to inform on the risk of differentiation syndrome in AML patients treated with TIBSOVO® (Ivosidenib), using process dimensions for patients’ awareness, receipt of the material, reading, utility of the PAC, self-reported behaviour, and knowledge. ; ; Primary Objective; To assess the effectiveness of the PAC on DS in AML patients treated by TIBSOVO®, i.e., to assess the awareness of the PAC and the knowledge about DS.; ; Secondary Objective; To assess receipt, reading, utility, and self-reported behaviour related to the PAC by AML patients treated with TIBSOVO® (ivosidenib).; ; Exploratory Objective; To assess the global informative value of the PAC regarding DS in AML patients treated by TIBSOVO®, i.e., to assess collectively all dimensions of the PAC: awareness, knowledge, receipt, reading, utility and self-reported behaviour.;
• Setting:
  The survey will collect data resulting from the PAC of TIBSOVO®, referring in particular to the patient’s receipt, the understanding of DS risk, the correct identification of these symptoms onset and the knowledge of the right actions to be taken. Data for this study will be collected exclusively through a participant self-administered paper ; survey. No additional clinical data will be collected, and no medical record review, or ; interventional procedures will be performed as part of this study.; ; Inclusion criteria; Patients will be included if they meet all the following criteria:; 1. Adult patients (female and male) aged ≥18 years at the time of recruitment.; 2. Who started TIBSOVO® for newly diagnosed AML within the last 6 months at their physician discretion, and have been treated with TIBSOVO® prior to enrolment in the study, whether or not still receiving TIBSOVO® at the time of recruitment.; 3. Able to understand and provide written informed consent, including permission to share their responses in aggregate with the EMA or national competent authorities (CA), if requested.; 4. Able to read and understand in native language of each of the participating countries, and willing to complete the patient survey.; ; Exclusion criteria; 1. Patients who declared having participated in user testing for this study.;
• Outcomes:
  Primary outcome ; • Number of participants having at least 80% of understanding rate from pooled questions comprising awareness and knowledge items, for primary respondents and completers. ; ; Secondary outcome ; • Number of participants having at least 80% of understanding rate from pooled questions comprising receipt, reading, utility and self-behaviour dimensions, for secondary respondents and completers. ; ; Exploratory outcome ; • Number of participants (completers) having at least 80% understanding rate in the pooled primary and secondary scores.
• Data analysis plan:
  Data collected from the survey will be reported as descriptive statistics. Frequency distributions will be calculated for items that address the survey objective (excluding demographic questions).