DARWIN EU® Treatment patterns of drugs used in adult and paediatric population with systemic lupus erythematosus

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Drug utilisation
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medicinal product name, other:
  - Cyclosporine; - Fluocortolone; - Paramethasone ; - Prednisone; - Triamcinolone ; - Cortisone; - Prednylidene; - Rimexolone ; - Deflazacort; - Cloprednol ; - Meprednisone ; - Cortivazol
• Study drug International non-proprietary name (INN) or common name:
  AZATHIOPRINE

  BELIMUMAB

  BETAMETHASONE

  CYCLOPHOSPHAMIDE

  DEXAMETHASONE

  HYDROCORTISONE

  HYDROXYCHLOROQUINE

  METHOTREXATE

  METHYLPREDNISOLONE

  MYCOPHENOLATE MOFETIL

  PREDNISOLONE

  RITUXIMAB

  TACROLIMUS

  VOCLOSPORIN
• Anatomical Therapeutic Chemical (ATC) code:
  (H02AB01) betamethasone

  betamethasone

  (H02AB02) dexamethasone

  dexamethasone

  (H02AB03) fluocortolone

  fluocortolone

  (H02AB04) methylprednisolone

  methylprednisolone

  (H02AB05) paramethasone

  paramethasone

  (H02AB06) prednisolone

  prednisolone

  (H02AB07) prednisone

  prednisone

  (H02AB08) triamcinolone

  triamcinolone

  (H02AB09) hydrocortisone

  hydrocortisone

  (H02AB10) cortisone

  cortisone

  (H02AB11) prednylidene

  prednylidene

  (H02AB12) rimexolone

  rimexolone

  (H02AB13) deflazacort

  deflazacort

  (H02AB14) cloprednol

  cloprednol

  (H02AB15) meprednisone

  meprednisone

  (H02AB17) cortivazol

  cortivazol

  (L01AA01) cyclophosphamide

  cyclophosphamide

  (L01BA01) methotrexate

  methotrexate

  (L01FA01) rituximab

  rituximab

  (L04AA06) mycophenolic acid

  mycophenolic acid

  (L04AA26) belimumab

  belimumab

  (L04AD01) ciclosporin

  ciclosporin

  (L04AD02) tacrolimus

  tacrolimus

  (L04AD03) voclosporin

  voclosporin

  (L04AX01) azathioprine

  azathioprine

  (L04AX03) methotrexate

  methotrexate

  (P01BA02) hydroxychloroquine

  hydroxychloroquine
• Medical condition to be studied:
  Systemic lupus erythematosus

Population studied

• Short description of the study population:
  The study population will include all individuals with a first diagnosis of SLE identified in the database during the patient selection period, which is between 01/01/2013 and 180 days prior to the end of available data in each database.
• Age groups:
  • Infants and toddlers (28 days – 23 months)
  • 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)
• Estimated number of subjects:
  19900000

Study design details

• Study design:
  A retrospective cohort study of all patients newly diagnosed with SLE will be conducted. For the description of each treatment objective, a new drug user cohort will be used to characterise patient-level SLE drug utilisation.
• Main study objective:
  To characterise paediatric and adult patients with SLE.
• Setting:
  Inpatient and outpatient setting from 5 databases in 4 European countries.
• Data analysis plan:
  Large scale patient level characterisation will be conducted. Medical condition and medication use history will be reported at any time and 365 days prior to index date, respectively. The number and percentage of patients receiving each of a pre specified list of SLE treatments and treatment combinations will be described per calendar year. Additionally, sunburst plots and Sankey diagrams will be used to describe treatment patterns and sequences over time. For the new user cohort, the index date is the initiation of SLE treatment after SLE diagnosis. Treatment duration, initial dose strength, cumulative dose, number of prescriptions will be estimated for new users of each SLE treatments at the ingredient level. For all continuous variables, mean with standard deviation and median with interquartile range will be reported. For all categorical analyses, number and percentages will be reported. A minimum cell count of 5 will be used when reporting results, smaller counts reported as 5.
• Summary results:
  CONCLUSION ; The most frequent treatment for SLE was similar in paediatric and adult patients, being hydroxychloroquine and glucocorticoids, with a higher proportion of use in paediatric patients, with adults exposed to a wider range of treatment such as methotrexate. The duration and dose of SLE treatments varied between databases, particularly between primary care and hospital settings. Worryingly, children with SLE were exposed to longer and higher cumulative doses of systemic glucocorticoids compared to adults, suggesting the need for further guidance and for alternative therapeutic options in JSLE.