ESCORT-HU : European Sickle Cell Disease Cohort - Hydroxyurea

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:
  Cohort

Study drug and medical condition

• Name of medicine:
  SIKLOS
• Medical condition to be studied:
  Sickle cell anaemia with crisis

Population studied

• Short description of the study population:
  Patients with Sickle Cell Disease (SCD) treated with Siklos®.; ; Inclusion criteria:; 1. Male or female ambulatory patients, aged 2 years and more (children, adolescents or adults); 2. With symptomatic sickle cell syndrome; 3. Justifying a treatment with Siklos® according to the product indications; 4. Having been informed of the study by the initiating physician and consenting to participate to the cohort.
• Age groups:
  Children (2 to < 12 years)

  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

  Pregnant women

  Renal impaired
• Estimated number of subjects:
  2000

Study design details

• Main study objective:
  To collect information about long-term safety of Siklos® when used in patients with Sickle Cell Disease, assessed on overall mortality and survival time, frequency of malignancies, skin ulceration and impaired postnatal development (growth), myelosuppression and amenorrhea, subgroups (young patients, elderly...)
• Outcomes:
  frequency of malignancies, skin ulceration and impaired postnatal development (growth), myelosuppression, Effects of Siklos® on growth development, outcome of pregnancies, occurrence of adverse events and serious adverse events
• Data analysis plan:
  All statistical analyses will be performed at the 5% significance.Parameters will be summarized using mean, median, standard deviation, range for continuous data and counts or percentages for categorical data.Descriptive statistics will be used to report the prevalence of primary and secondary parameters, describe the global population and the sub-populationsAppropriate multivariate analysis will be used to describe the determinants of the observed prevalence.

Documents

Study results

ADD_12304_ESCORT-HU_Final synopsis_20191216