Long term treatment with ataluren-the Swedish experience

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Michael, EvaGothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics (författare)

Long term treatment with ataluren-the Swedish experience

Artikel/kapitelEngelska2021

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2021-09-30
Springer Science and Business Media LLC,2021

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LIBRIS-ID:oai:gup.ub.gu.se/310186
https://gup.ub.gu.se/publication/310186URI
https://doi.org/10.1186/s12891-021-04700-zDOI

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Språk:engelska

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Introduction Ataluren is a relatively new treatment for male patients with Duchenne muscular dystrophy (DMD) due to a premature stop codon. Long-term longitudinal data as well as efficacy data on non-ambulant patients are still lacking. Here we present the results from a long-term follow-up study of all DMD patients treated with ataluren and followed at the Queen Silvia Children's Hospital in Gothenburg, Sweden, with focus on the evolution of patients' upper motor and respiratory function over time. Methods This is a retrospective longitudinal case-series study of all male DMD patients treated with ataluren and followed at the Queen Silvia Children's Hospital in Gothenburg, Sweden, since 2008. Results Our eleven patients had a median exposure to ataluren of 2312 days which is almost a fourfold higher than previous studies. Loss of ambulation occurred at a median age of 13.2 years. Patients who lost ambulation prior to 13.2 years of age had received ataluren for 5 years, whereas patients who continued to be ambulatory after 13.2 years of age had received ataluren for 6.5 years until loss of ambulation or last follow-up if still ambulatory. Four of six non ambulatory patients had Performance of the Upper Limb scores above the expected mean values over time. All but one patient maintained a pulmonary decline above the expected over time. All ambulatory patients increased in their predicted forced vital capacity (FVC) with 2.8 to 8.2% annually. Following loss of ambulation, 5 of 6 patients declined in predicted FVC (%), with annual rate of decline varying from 1.8 to 21.1%. The treatment was safe and well tolerated throughout the follow-up period. Conclusions This is the first study to present long-term cumulative treatment outcomes over a median period of 6.3 years on ataluren treatment. Our results indicate a delay in loss of ambulation, as well as a slower decline in FVC and upper limb motor function even after loss of ambulation. We suggest that treatment with ataluren should be initiated as soon as the diagnosis is confirmed, closely monitored and, in case of sustainable benefit, continued even after loss of ambulation.

Ämnesord och genrebeteckningar

MEDICIN OCH HÄLSOVETENSKAP Klinisk medicin hsv//swe
MEDICAL AND HEALTH SCIENCES Clinical Medicine hsv//eng
Duchenne muscular dystrophy
Ataluren treatment
Performance of upper
limbs
Forced vital capacity
Long-term effect
Ambulatory vs
non-ambulatory
duchenne muscular-dystrophy
double-blind
glucocorticoids
performance
prednisone
trial
dmd
Orthopedics
Rheumatology

Biuppslag (personer, institutioner, konferenser, titlar ...)

Sofou, KalliopiGothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics(Swepub:gu)xsofka (författare)
Wahlgren, Lisa,1980Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics(Swepub:gu)xwalis (författare)
Kroksmark, Anna-KarinGothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics(Swepub:gu)xkrann (författare)
Tulinius, Mar,1953Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics(Swepub:gu)xtulim (författare)
Göteborgs universitetInstitutionen för kliniska vetenskaper, Avdelningen för pediatrik (creator_code:org_t)

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Ingår i:Bmc Musculoskeletal Disorders: Springer Science and Business Media LLC22:11471-2474

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Av författaren/redakt...: Michael, Eva; Sofou, Kalliopi; Wahlgren, Lisa, ...; Kroksmark, Anna- ...; Tulinius, Mar, 1 ...

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MEDICIN OCH HÄLSOVETENSKAP: MEDICIN OCH HÄLS ...; och Klinisk medicin

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Av lärosätet: Göteborgs universitet

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