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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.
Shohet, Simon; Hummel, Noemi; Fu, Shuai; Keyzor, Ian; MacCulloch, Alasdair; Johnson, Neil; Castelli, Jeff; Czarny-Ozga, Ilona; Mozaffar, Tahseen; Thom, Howard.
Afiliación
  • Shohet S; Amicus Therapeutics UK Ltd, One Globeside, Fieldhouse Lane, Marlow, SL7 1HZ, UK.
  • Hummel N; Certara GmbH, Chester Platz 1, 79539, Lörrach, Germany.
  • Fu S; Certara, Office 610, South Tower, Hong Kong Plaza, No. 283 Huaihai Road Middle, Huangpu District, Shanghai, China.
  • Keyzor I; Amicus Therapeutics UK Ltd, One Globeside, Fieldhouse Lane, Marlow, SL7 1HZ, UK.
  • MacCulloch A; Amicus Therapeutics UK Ltd, One Globeside, Fieldhouse Lane, Marlow, SL7 1HZ, UK.
  • Johnson N; Amicus Therapeutics UK Ltd, One Globeside, Fieldhouse Lane, Marlow, SL7 1HZ, UK.
  • Castelli J; Amicus Therapeutics, Inc., 47 Hulfish St., Princeton, NJ, 08542, USA.
  • Czarny-Ozga I; Certara, Instytut Arcana sp. z o.o. 17, Kuklinskiego Street, 30-720, Krakow, Poland.
  • Mozaffar T; Department of Neurology, University of California, 1001 Health Sciences Road, Irvine, CA 92697-3950, USA.
  • Thom H; Population Health Sciences, Bristol Medical School, University of Bristol, 1-5 Whiteladies Rd, Bristol, BS8 1NU, UK.
J Comp Eff Res ; : e240045, 2024 Sep 17.
Article en En | MEDLINE | ID: mdl-39287071
ABSTRACT

Aim:

Late-onset Pompe disease is characterized by progressive loss of muscular and respiratory function. Until recently, standard of care was enzyme replacement therapy (ERT) with alglucosidase alfa. Second-generation ERTs avalglucosidase alfa (aval) and cipaglucosidase alfa with miglustat (cipa+mig) are now available. Without head-to-head trials comparing aval with cipa+mig, an indirect treatment comparison is informative and timely for understanding potential clinical differentiation. Materials &

methods:

A systematic literature review was performed to identify relevant studies on cipa+mig and aval. Using patient-level and aggregate published data from randomized controlled trials (RCTs) and phase I/II and open-label extension (OLE) trials, a multi-level network meta-regression was conducted, adjusting for various baseline covariates, including previous ERT duration, to obtain relative effect estimates on 6-minute walk distance (6MWD, meters [m]) and forced vital capacity (FVC, % predicted [pp]). Analyses of two networks were conducted Network A, including only RCTs, and network B, additionally including single-arm OLE and phase I/II studies.

Results:

Network B (full evidence analysis) showed that cipa+mig was associated with a relative increase in 6MWD (mean difference 28.93 m, 95% credible interval [8.26-50.11 m]; Bayesian probability 99.7%) and FVC (2.88 pp [1.07-4.71 pp]; >99.9%) compared with aval. The comparison between cipa+mig and aval became more favorable for cipa+mig with increasing previous ERT duration for both end points. Analysis of network A showed that cipa+mig was associated with a relative decrease in 6MWD (-10.02 m [-23.62 to 4.00 m]; 91.8%) and FVC (-1.45 pp [-3.01 to 0.07 pp]; 96.8%) compared with aval.

Conclusion:

Cipa+mig showed a favorable effect versus aval when all available evidence was used in the analysis.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: J Comp Eff Res Año: 2024 Tipo del documento: Article País de afiliación: Reino Unido Pais de publicación: Reino Unido
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: J Comp Eff Res Año: 2024 Tipo del documento: Article País de afiliación: Reino Unido Pais de publicación: Reino Unido