Fig. 3.
Changes in organomegaly and diffusing capacity of the lung over time with olipudase alfa treatment. A Individual patient responses for spleen volumes in multiples of normal (MN). Means for the overall population are indicated by the solid black line. Spleen absolute volumes were calculated as MN assuming normal volumes of 0.2% of body weight [17]. Severe and moderate splenomegaly were defined as > 15 and > 5 to ≤ 15 MN, respectively [17]. Cutoffs of MN for severity of splenomegaly are indicated by horizontal lines. B Mean spleen volumes in MN ± standard deviations over time stratified by baseline age group. P values from the ANCOVA test used to examine mean percent difference from baseline to month 24 are: P = 0.0014 for the adolescent group (n = 4) and P < 0.0001 for both the child (n = 7) and the infant/early child (n = 7) groups. Level of significance at each timepoint is indicated in the figure with asterisks (*p < 0.05; **p < 0.01; ***p < 0.001). Summary data at month 24 are provided in Additional file 1: Table S2. C Individual patient responses for liver volumes in MN. Means for the overall population are indicated by the solid black line. Liver absolute volumes were calculated as MN assuming normal liver volumes of 2.5% of body weight [17]. Severe and moderate hepatomegaly were defined as > 2.5 and > 1.25 to ≤ 2.5MN, respectively [17]. Cutoffs of MN for severity of hepatomegaly are indicated by horizontal lines. D Mean liver volumes in MN ± standard deviations over time stratified by baseline age group. P-values from the ANCOVA test used to examine mean percent difference from baseline to month 24 are: P = 0.0014 for the adolescent group (n = 4), P < 0.0001 for the child (n = 9), and P = 0.0002 for the infant/early child group (n = 6). Level of significance at each timepoint is indicated in the figure with asterisks (*p < 0.05; **p < 0.01; ***p < 0.001). Summary data at month 24 are provided in Additional file 1: Table S2. E Individual patient responses for percent predicted DLCO adjusted for hemoglobin for patients able to perform the assessment at baseline. Cutoffs for gas exchange impairment are indicated (> 80% was considered normal/no impairment, > 60–80% mild impairment, 40–60% moderate impairment, and < 40% severe impairment) [19]. F Mean percent predicted DLCO adjusted for hemoglobin ± standard deviations over time stratified by baseline age group. P-values from the ANCOVA test used to examine mean percent difference from baseline to month 24 are: P = 0.1747 for the adolescent group (n = 3) and P = 0.0002 for the child group (n = 6). Level of significance at each timepoint is indicated in the figure with asterisks (*p < 0.05; **p < 0.01; ***p < 0.001). Summary data at month 24 are provided in Additional file 1: Table S2