Figure 3

Modeled clinical outcomes for a hypothetical cohort of 100 male MPS II patients (5 to 10 years old) receiving ERT for two years. Potential outcomes are stratified by phenotype (severe versus attenuated). When possible, sources of estimates are identified from the Results and Supplementary Materials as indicated. (1) Supplementary Figure S1; MPSII patient survival by phenotype. (2) Supplementary Figure S2; absolute uGAG levels. (3) Supplementary Figure S3; relative liver volume change (%). (4) and (5) Supplementary Figure S4; 6-minute walk test (6-MWT) distance (meters). (6) Supplementary Table S8; Growth (Height). (7) Joint range of motion (JROM). (8) Supplementary Figure S5; Pulmonary function: forced vital capacity (FVC) and %FVC. (9) Cardiac function. (10) Sleep apnea. (11) Physical disability/Quality of life. (12) Supplementary Table S7, assumes an average SAE patient-specific rate of about 10%. (13) Supplementary Table S6; assumes IgG antibodies in about half of treated patients, with at least 41–100% of those patients developing neutralizing antibodies. (14) 2012 Health Technology Assessment²³ estimate of $1.06 million per patient for 2 years of weekly infusions of 0.5 mg/kg idursulfase (converted to 2014 US dollars).