Figure 2

Relative uGAG levels reported in nine studies of MPSII patients by ERT dose and length of treatment. Relevant information for included studies are summarized below the figure.^{17, 19, 20, 21, 22, 24, 28, 35, 36} The relative changes in uGAG measurements are shown on the vertical axis beginning at the baseline of 100% (initiation of treatment—reduction of 0%). The x axis identifies the individual studies. Open circles (studies 1 and 2) represent patients receiving a placebo (controls). Thick lines indicate treatment with 0.5 mg/kg/week idursulfase, and thin lines represent alternative doses (lower or higher). Patients were treatment-naive in studies 1 to 8, but not 9. Relative uGAG reductions were observed by 3–4 months, ranging from 43–80% at 1 year at 0.5 mg/kg (studies 1–8). Study 9 compared two drugs in patients previously treated for 14 months with Elaprase (0.5 mg/kg idursulfase). After a 2-week “washout” period, subjects were assigned to one of three groups, continuing 0.5 mg/kg Elaprase or changing to 0.5 or 1.0 mg/kg Hunterase (idursulfase beta). ^aPercent of enrolled patients having an attenuated/mild MPSII phenotype; the percent with a severe phenotype can be derived. ^bFU (yrs) = duration of treatment/follow-up in years. ERT, enzyme replacement therapy; uGAG, urinary glycosaminoglycan.