Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy

Figures

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  Figure 1. Age distribution of normalized titers at first test for antibodies that bind AAV9 (N = 882) Prevalence greatest at ≤3 months old (18.2%) and decreased after 9 months of age (0%). CTL, Cellular Technology Limited; E+1, 1:25 titer concentration on Athena test or 1:100 titer concentration on CTL test; E+2, 1:50 titer concentration on Athena test or 1:200 titer concentration on CTL test; E+3, 1:100 titer concentration on Athena test or 1:400 titer concentration on CTL test; E+4, ≥1:200 titer concentration on Athena test or ≥1:800 titer concentration on CTL test; NE, not elevated. Note: For the Athena test, a titer concentration ≥1:25 was considered elevated, and for the CTL test, >1:50 was considered elevated.

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  Figure 2. Potential regional variations in AAV9-Ab seroprevalencea (A) Patients with elevated titers by state (n = 794). (B) Patients <1 month old with elevated titers by state (n = 383). AAV9-Ab, adeno-associated virus serotype 9 antibody. aStates with a small sample size (n ≤ 3) are not included.

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  Figure 3. Sequence of AAV9-Ab titers for patients younger than 6 weeks old with elevated first test for AAV9-Ab who underwent repeat testing (n = 65)

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  Figure 4. Time to retest for patients younger than 6 weeks old with elevated first test for AAV9-Ab who underwent repeat testing (n = 125)

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  Figure 5. Testing sequence of newborns (≤4 weeks old) with repeat tests for AAV9-Ab performed at a single lab and with elevated first test result (A) Testing sequence of newborns with an elevated first test (n = 30). (B) Testing sequence of newborns with first test result at E+4 titer concentration (n = 18) (CTL: n = 3; Athena: n = 15). (C) Testing sequence of newborns with first test result at E+3 titer concentration (n = 2) (Athena: n = 2). (D) Testing sequence of newborns with first test result at E+2 titer concentration (n = 5) (CTL: n = 1; Athena: n = 4). (E) Testing sequence of newborns with first test result at E+1 titer concentration (n = 5) (CTL: n = 1; Athena: n = 4). Note: Two patients had identical results and are represented by a single line. For the Athena test, a titer concentration ≥1:25 was considered elevated, and for the CTL test, >1:50 was considered elevated. AAV9-Ab, adeno-associated virus serotype 9 antibody; CTL, Cellular Technology Limited; E+1, 1:25 titer concentration on Athena test or 1:100 titer concentration on CTL test; E+2, 1:50 titer concentration on Athena test or 1:200 titer concentration on CTL test; E+3, 1:100 titer concentration on Athena test or 1:400…