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Table 1 Examples of rare diseases which could make use of the augmented binary method

From: Improving the analysis of composite endpoints in rare disease trials

Disease Example responder endpoint
Primary biliary cholongitis (PBC) ∙ ALP <1.67×ULN
  ∙ Total bilirubin < ULN
  ∙ ALP decrease ≥ 15%
Behçets disease ∙ Length of principal intestinal
  ulcer compared to size at baseline (%)
  ∙ No new lesions
Lupus Nephritis ∙ eGFR no more than 10% below
  preflare value or normal
  ∙ Proteinuria UPC ratio < 0.5
  ∙Urine sediment: Inactive
  ∙ No rescue therapy
Neuroblastoma ∙ < 10mm residual soft tissue at
  primary site
  ∙ Complete resolution of MIBG of
  FDG-PET uptake (for MIBG non avid
  tumours) at primary site
Advanced hepatocellular carcinoma ∙ < 20% increase in the sum of the
  longest diameters of target lesions
  ∙ No new lesions
  1. ALP alkaline phosphatase, ULN upper limits of normal, eGdFR estimated glomerular filtration rate, UPC urinary protein to creatinine, MIBG metaiodobenzylguanidine, FDG-PET 18-fluorodeoxyglucose positron emission tomography