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. 2023 Nov 21;101(21):e2103–e2113. doi: 10.1212/WNL.0000000000207878

Figure 3. Recreated Kaplan-Meier Curves for the Probability of Losing Ambulation.

Figure 3

*The pooled curve for the 7 individual studies (i.e., Bladen et al., 2014, Chung et al., 2004, Kaneko et al., 2017, Lusakowska et al., 2021, Russman et al., 1996, Wadman et al., 2017, and Zerres et al., 1997) is presented (along with the corresponding 95% CI). The pooled curve across all 22 study arms (i.e., Bladen et al., 2014 [Argentina, Germany and Austria, Hungary, Serbia, Switzerland, the United Kingdom, and Ukraine], Chung et al., 2004 [type 3a and type 3b], Kaneko et al., 2017 [type 3a onset younger than 3 years and type 3b onset 3 years or older], Lusakowska et al., 2021 [type 3a 3 copies, type 3a 4 copies, type 3b 3 copies, and type 3b 4 copies], Russman et al., 1996 [type 3b younger than 2 years, type 3b 2 years or older], Wadman et al., 2017 [type 3a, type 3b onset younger than 12 years, and type 3b onset younger than 12 years], and Zerres et al., 1997 [type 3a and type 3b]), which is based on a total of 1,327 patients, is presented. †The pooled curve across all 7 study arms (i.e., Chung et al., 2004 [type 3a] and Kaneko et al., 2017 [type 3a onset younger than 3 years], Lusakowska et al., 2021 [type 3a 3 copies and type 3a 4 copies], Russman et al., 1996 [type 3b onset younger than 2 years], Wadman et al., 2017 [type 3a], and Zerres et al., 1997 [type 3a]), which is based on a total of 413 patients, is presented (along with the corresponding 95% CI). The median time to loss of walking/ambulation based on pooled study arms was estimated to be 13.4 years (95% CI 12.5–14.5). In the sensitivity analysis (which excluded Russman et al., 1996 [type 3b onset younger than 2 years]), the median time to loss of walking/ambulation across pooled study arms is estimated to be 14.0 years (95% CI 12.5–14.9). Compared with the base-case analysis, in the sensitivity analysis, the median increased by 7 months. ‡The pooled curve across all 8 study arms (i.e., Chung et al., 2004 [type 3b], Kaneko et al., 2017 [type 3b onset 3 years or older], Lusakowska et al., 2021 [type 3b 3 copies and type 3b 4 copies], Russman et al., 1996 [type 3b onset 2 years or older], Wadman et al., 2017 [type 3b onset younger than 12 years and type 3b onset older than 12 years], and Zerres et al., 1997 [type 3b]), which is based on a total of 331 patients, is presented (along with the corresponding 95% CI). The median to loss of walking/ambulation based on pooled study arms was estimated to be 44.2 years (95% CI 43.0–49.4). In the sensitivity analysis (which excluded Russman et al., 1996 type 3b 2 years or older), the median time to loss of walking/ambulation across pooled study arms is estimated to be 46.1 years (95% CI 43.0–58.4). Compared with the base-case analysis, the median increased by 1.9 years. SMA = spinal muscular atrophy.