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Corrigendum
21 November 2024

Corrigendum

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The Research Article by Simon Shohet, Noemi Hummel, Shuai Fu, Ian Keyzor, Alasdair MacCulloch, Neil Johnson, Jeff Castelli, Ilona Czarny-Ozga, Tahseen Mozaffar and Howard Thom, “Comparing the efficacy of cipaglucosidase alfa plus miglustat with other enzyme replacement therapies for late-onset Pompe disease: a network meta-analysis utilizing patient-level and aggregate data”, which appeared in the October 2024 issue of the Journal of Comparative Effectiveness Research (13[10], e240045 [2024]), was published with errors within Table 1 and the Supplementary Materials file, as detailed below.
Table 1 included incorrect reference numbering, which was unfortunately introduced during the manuscript revision process.
In the Supplementary Materials file, the figure legends in Supplementary Figures 2 and 3 (pages 46 and 47) were inadvertently cut off.
Table 1. Trial and patient baseline characteristics of included trials.
Trial nameNCTTrial designFollow-up time (weeks)PopulationInterventionnAge (years), mean (SD)Male (%)ERT duration in years, mean (SD)6MWD in meters, mean (SD)FVC (predicted [%]), mean (SD)Ref.
NEO-1/-EXTNCT01898364/ NCT02032524Phase I/II, single arm312ERT-NAval1044.8 (20.3)30.00.0 (0.0)449.2 (118.4)69.2 (19.3)[17,55]
    ERT-E 1446.7 (14.1)64.34.0 (2.0)440.4 (141.0)77.3 (16.5) 
COMETNCT02782741Phase III RCT49ERT-NAval5146.0 (14.5)52.90.0 (0.0)399.3 (110.9)62.5 (14.4)[12]
     Alglu4950.3 (13.7)51.00.0 (0.0)378.1 (116.2)61.6 (12.4) 
COMET OLENCT02782741Phase III OLE96ERT-EAval4450.7 (13.9)54.50.9 (0)384.7 (139.6)61.5 (13.5)[49,56]
ATB200-02NCT02675465Phase I/II, single arm208ERT-ECipa+mig1746.4 (11.7)§64.77.0 (1.6)393.5 (119.7)57.1 (17.9)[48]
    ERT-NCipa+mig647.2 (12.0)#16.70.0 (0.0)396.0 (75.2)57.2 (20.8) 
PROPELNCT03729362Phase III RCT52ERT-ECipa+mig6548.1 (12.6)††43.1††7.5 (3.4)††346.9 (110.2)67.9 (19.1)[2]
    ERT-N 2046.0 (15.3)††40.0††0.0 (0.0)393.6 (112.4)80.2 (18.7) 
    ERT-EAlglu‡‡3045.9 (13.5)††46.7††7.1 (3.6)††334.6 (114.0)67.5 (21.0) 
    ERT-N 743.0 (13.5)††71.4††0.0 (0.0)420.9 (135.7)79.1 (22.6) 
PROPEL OLENCT04138277Phase III OLE52ERT-ECipa+mig3746.0 (12.7)§§51.46.7 (4.3)††359.7 (137.4)††62.8 (20.5)††[50,57]
This was derived from the range of 0.9 to 7.9 years provided in Dimachkie et al. 2022 [18], using formulas from Wan et al. 2014 [56] and assuming a median of (7.9–0.9)/2 = 3.5.
Patients who switched from alglu to aval after the double-blinded primary analysis period in COMET were re-baselined at Week 49, thus, those patients were followed for 96 weeks and considered ERT experienced with a previous ERT duration of the length of the double-blinded primary analysis period (i.e., 49 weeks = 0.9 years).
§
This was derived as follows: Median and range of Cohort 1 and 4 provided by Mozaffar et al. 2023 [17] were converted to mean and SD using formulas from Wan et al. 2014 [56]. Then, a weighted mean and pooled SD was calculated to obtain mean and SD of the pooled Cohort 1 and 4.
A weighted mean and pooled SD was calculated from mean and standard deviation values for Cohort 1 and 4 provided by Mozaffar et al. 2023 [17] to obtain mean and SD of the pooled Cohort 1 and 4.
#
This was derived as follows: Median and range provided in Schoser et al. 2022 [57] were converted to mean and SD using formulas from Wan et al. 2014 [56].
††
From individual patient-level data.
‡‡
Excluding one patient who deliberately underperformed on baseline assessments to meet inclusion criteria as described in Schoser et al. 2021 [2].
§§
This was derived as follows: Median and range were converted to mean and SD using formulas from Wan et al. 2014 [56].
6MWD: 6-minute walk distance; Alglu: Alglucosidase alfa; Aval: Avalglucosidase alfa; Cipa+mig: Cipaglucosidase alfa plus miglustat; ERT: Enzyme replacement therapy; ERT-E: ERT experienced; ERT-N: ERT naïve; FVC: Forced vital capacity; OLE: Open-label extension; RCT: Randomized controlled trial; SD: Standard deviation.
These errors have now been corrected. These corrections do not affect the overall conclusions of the article.
The authors and editors of the Journal of Comparative Effectiveness Research would like to sincerely apologize for any inconvenience or confusion this may have caused our readers.

Supplementary Material

File (supplementary materials.docx)