DARWIN EU® - Real-World Overall Survival in Patients with Acute Gut GvHD Following Failure of Corticosteroids and Ruxolitinib

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

• Study drug International non-proprietary name (INN) or common name:
  RUXOLITINIB
• Anatomical Therapeutic Chemical (ATC) code:
  (L01EJ01) ruxolitinib

  ruxolitinib
• Medical condition to be studied:
  Acute graft versus host disease in intestine

Population studied

• Short description of the study population:
  The phenotype of aGvHD with intestinal involvement will consist of patients with a disease code for Graft versus host disease of intestine. ; If source data are not sufficiently granular, the phenotype will be defined as the presence of Acute graft versus host disease in combination with intestinal symptoms recorded during the same visit occurrence or within -14/+14 days of the index date. ; Intestinal symptoms include gastrointestinal haemorrhage, abdominal pain and tenderness, abdominal distension/bloating, diarrhoea, haematochezia, nausea, vomiting, anorectal tenesmus, malabsorption syndrome, loss or decrease of appetite, intestinal disorders and inflammatory digestive tract disorders, enterocolitis-related conditions, paralytic ileus, nutritional complications (e.g. protein-calorie malnutrition), dyspepsia, changes in stool output, and evidence of gastrointestinal endoscopy or biopsy
• Age groups:
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)

Study design details

• Study design:
  A retrospective cohort study will be conducted using routinely collected health data from five data sources.
• Main study objective:
  1. To characterise the baseline demographic and clinical characteristics of patients with aGvHD with intestinal involvement, including age, sex, transplant type, and indication for transplant.; 2. To describe treatment duration of individual treatments, progression to subsequent treatment lines, and the time from aGvHD diagnosis to initiation of second- and third-line therapy among patients with aGvHD with intestinal involvement.; 3. To evaluate real-world Progression-Free Survival (rwPFS) in patients who initiate third-line therapy after failure of corticosteroids and ruxolitinib.; 4. To evaluate overall survival up to one year in patients who initiate third-line therapy after failure of corticosteroids and ruxolitinib.
• Outcomes:
  Objective 2 ; • Progression to a subsequent line of therapy. ; ; Objective 3 ; • rwPFS, defined as the time from initiation of third-line therapy to the earliest occurrence of initiation of a subsequent treatment line, death from any cause, or documented disease progression (where available).; • rwPFS probabilities at 56, 180, and 365 days following initiation of third-line therapy. ; ; Objective 4 ; • Overall survival, defined as the time from initiation of third-line therapy to death from any cause. ; • Survival probability at 56, 180, and 365 days following initiation of third-line therapy.