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

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