Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

Figures

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  Figure 1.. Age at loss of ambulation for propensity-score matched patients from the STRIDE Registry and CINRG DNHS. Propensity-score model covariates include age at first symptoms, age at first corticosteroid use, duration of deflazacort use and duration of use of corticosteroids other than deflazacort. Censor dates for censored patients in the STRIDE Registry were derived from the last assessment date across treatment, physical examination, vital signs, 6-minute walk distance, timed function tests, North Star Ambulatory Assessments, percentage of predicted FVC and upper limb function tests. Corticosteroid duration is calculated from the date at which corticosteroid use was started to the date of loss of ambulation or the date of the last assessment. †‘+’ indicates a censored observation. ‡p-value is from a log-rank test stratified by deflazacort and other corticosteroid usage durations. §HR is from stratified (by durations of deflazacort and other corticosteroid use) Cox regression with study, age at first symptoms and age at first corticosteroid use as covariates. The HR is STRIDE Registry versus CINRG DNHS. CINRG DNHS: Cooperative International Neuromuscular Research Group Duchenne…

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  Figure 2.. Age at time to stand from supine (A) ≥5 s and (B) ≥10 s for propensity-score matched patients from the STRIDE Registry and CINRG DNHS. Propensity-score model covariates include age at first symptoms, age at first corticosteroid use, duration of deflazacort use and duration of use of corticosteroids other than deflazacort. Censor dates for censored patients in the STRIDE Registry were derived from the last assessment date across treatment, physical examination, vital signs, 6-minute walk distance, timed function tests, North Star Ambulatory Assessments, percentage of predicted FVC and upper limb function tests. Corticosteroid duration is calculated from the date at which corticosteroid use was started to the date of loss of ambulation or the date of the last assessment. †Event = time to stand from supine ≥5 s or ≥10 s. ‡‘+’ indicates a censored observation. §p-value is from a log-rank test stratified by deflazacort and other corticosteroid usage durations. ¶HR is from stratified (by durations of deflazacort and other corticosteroid use) Cox regression with study, age at first symptoms and age at first corticosteroid use as covariates. The HR is STRIDE Registry versus CINRG DNHS.…

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  Figure 3.. Age at time to climb four stairs (A) ≥5 s and (B) ≥10 s for propensity score-matched patients from the STRIDE Registry and CINRG DNHS. Propensity-score model covariates include age at first symptoms, age at first corticosteroid use, duration of deflazacort use and duration of use of corticosteroids other than deflazacort. Censor dates for censored patients in the STRIDE Registry were derived from the last assessment date across treatment, physical examination, vital signs, 6-minute walk distance, timed function tests, North Star Ambulatory Assessments, percentage of predicted FVC and upper limb function tests. Corticosteroid duration is calculated from the date at which corticosteroid use was started to the date of loss of ambulation or the date of the last assessment. †Event = time to climb four stairs ≥5 s or ≥10 s. ‡‘+’ indicates a censored observation. §p-value is from a log-rank test stratified by deflazacort and other corticosteroid usage durations. ¶HR is from stratified (by durations of deflazacort and other corticosteroid use) Cox regression with study, age at first symptoms and age at first corticosteroid use as covariates. The HR is STRIDE Registry versus CINRG DNHS.…

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  Figure 4.. Correlation analysis of the effect of nonsense mutation exon location on disease severity (as measured by age at first symptoms) for patients from the STRIDE Registry (n = 181).

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  Figure 5.. Kaplan–Meier analysis estimating the effect of the type of premature stop codon on ataluren treatment benefit as assessed by age at loss of ambulation for patients from the STRIDE Registry (n = 181). A: Adenine; G: Guanine; U: Uracil.