Retrospective observational study of the conditions of use, safety and efficacy of MYLOTARG® (Gemtuzumab Ozogamicin, GO) in the treatment of patients with newly diagnosed CD33-positive acute myeloid leukaemia (AML). (MYLobs)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Drug utilisation

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Multicentre retrospective observational study

Study drug and medical condition

• Medical condition to be studied:
  Acute myeloid leukaemia
• Additional medical condition(s):
  untreated CD33-positive acute myeloid leukaemia (AML) patients aged 15 years and older, with the exception of acute promyelocytic leukaemia (APL)

Population studied

• Age groups:
  • Adolescents (12 to < 18 years)
  • 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:
  Hepatic impaired

  Immunocompromised

  Pregnant women

  Renal impaired
• Estimated number of subjects:
  130

Study design details

• Main study objective:
  The purpose of this study is to describe the use, efficacy and safety of MYLOTARG® in real-world conditions.
• Data analysis plan:
  For continuous variables, the usual statistics (n, missing n, mean, standard deviation (SD), median, first and third quartile (Q1 and Q3), minimum and maximum) are presented. For discrete variables, the usual statistics (n, missing n, frequency and percentage) will be given. Unless stated otherwise in the SAP, percentages are only calculated for items completed and missing data will be excluded. The EFS endpoint criterion will be presented using the Kaplan-Meier method with the associated survival curve. Standard statistics will be used to summarise the response endpoint, tolerance endpoints, patient and disease characteristics at initiation of treatment and subsequent treatments. An exploratory analysis will also be performed of patients treated under the ATU and those treated post-ATU. The percentage of VOD cases will be provided with its two2-sided 95% confidence interval (Clopper-Pearson exact method). Methodology for summary and statistical analyses of data collected in SAP.