Swiss Soliris® and Ultomiris® Reimbursement Registry SSURR: An observational registry for capturing data of patients suffering from paroxysmal nocturnal hemoglobinuria (PNH) who are treated with Soliris® (eculizumab) or Ultomiris® (ravulizumab)

Study type

• Study type:
  Non-interventional study

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Observational registry

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (L04AA25) eculizumab

  eculizumab

  (L04AA43) ravulizumab

  ravulizumab
• Medical condition to be studied:
  Paroxysmal nocturnal haemoglobinuria

Population studied

• 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)
• Estimated number of subjects:
  69

Study design details

• Main study objective:
  Collection of medical information related to initiation and continuation of therapy for patients diagnosed with paroxysmal nocturnal hemoglobinuria (PNH) and treated with Soliris® or Ultomiris® in Switzerland.
• Outcomes:
  hematological parameters, LDH value, PNH symptoms such as abdominal pain, chest pain, dyspnoea and pain requiring medical intervention, performance on a "quality of life" scale, need for transfusions, thrombotic events, occurrence of renal failure and pulmonary arterial hypertension
• Data analysis plan:
  The statistical evaluation of will be laid out according to the reporting requirements for the FOPH/BAG. Mainly descriptive statistics will be applied.