DARWIN EU® Natural history of dermatomyositis (DM) and polymyositis (PM) in adults and paediatric populations

Study type

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

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine, other:
  - Prednisone ; - Cyclosporine (ATC code: L04AD01); - Beta interferone -1a; - Beta interferone-1b; - Inmunoglobulines; - Brimionidine
• Study drug International non-proprietary name (INN) or common name:
  ABATACEPT

  ADALIMUMAB

  AZATHIOPRINE

  BASILIXIMAB

  CHLOROQUINE

  CYCLOPHOSPHAMIDE

  ECULIZUMAB

  ETANERCEPT

  HYDROXYCHLOROQUINE

  INFLIXIMAB

  METHOTREXATE

  METHYLPREDNISOLONE

  MYCOPHENOLATE MOFETIL

  RITUXIMAB

  TACROLIMUS
• Anatomical Therapeutic Chemical (ATC) code:
  (A07EA03) prednisone

  prednisone

  (H02AB04) methylprednisolone

  methylprednisolone

  (J06BA) Immunoglobulins, normal human

  Immunoglobulins, normal human

  (L01AA01) cyclophosphamide

  cyclophosphamide

  (L01FA01) rituximab

  rituximab

  (L03AB07) interferon beta-1a

  interferon beta-1a

  (L03AB08) interferon beta-1b

  interferon beta-1b

  (L04AA06) mycophenolic acid

  mycophenolic acid

  (L04AA24) abatacept

  abatacept

  (L04AA25) eculizumab

  eculizumab

  (L04AB01) etanercept

  etanercept

  (L04AB02) infliximab

  infliximab

  (L04AB04) adalimumab

  adalimumab

  (L04AC02) basiliximab

  basiliximab

  (L04AD02) tacrolimus

  tacrolimus

  (L04AX01) azathioprine

  azathioprine

  (L04AX03) methotrexate

  methotrexate

  (P01BA01) chloroquine

  chloroquine

  (P01BA02) hydroxychloroquine

  hydroxychloroquine

  (S01EA05) brimonidine

  brimonidine
• Medical condition to be studied:
  Dermatomyositis

  Polymyositis

  Juvenile polymyositis
• Additional medical condition(s):
  Juvenile dermatomyositis, Neonatal dermatomyositis

Population studied

• Age groups:
  Preterm newborn infants (0 – 27 days)

  Term newborn infants (0 – 27 days)

  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:
  19500000

Study design details

• Main study objective:
  The overall objective of this study is to describe and characterise dermatomyositis (DM), polymyositis (PM) and their juvenile forms (JDM and JPM), in terms of prevalence, natural history of the disease, disease severity, and treatment.
• Data analysis plan:
  Point prevalence of each outcome of interest (DM, PM, JDM, JPM), with every individual deemed to have the diagnosis from first occurrence until end of follow-up calculated on an annual basis as of the 1st January for each year, estimated overall and stratified by age and sex. Age and sex at time of DM, PM, JDM, JPM diagnosis (index date) will be described for each of the generated study cohorts (Objective 2). Large-scale patient-level characterisation will be conducted for objectives 3 to 5. Occurrence of co-morbidities, measurements, clinical manifestations, and severity markers will be assessed for anytime –and up to 365 days before index date, for 364 to 91, for 90 to 31, and for 30 to 1 day before index date, and at index date. We will also report them for 1 to 90, 91 to 180, 181 to 365 days, 366 to 1095, 1096 to 1825 days, and 1826 days to any time post index date.