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Injection site reactions after long-term subcutaneous delivery of drisapersen : a retrospective study

(2019) EUROPEAN JOURNAL OF PEDIATRICS. 178(2). p.253-258
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Abstract
A retrospective study in which we reviewed the hospital files of a subset of 7 patients with Duchenne muscular dystrophy participating in the open-label phase I/II PRO051-02 study in Leuven. The objective of this study was to describe in detail the injection site reactions in these children treated with drisapersen (PRO-051), a 2′-O-methyl phosphorothioate RNA antisense oligonucleotide, that induces exon 51 skipping in Duchenne muscular dystrophy. Antisense oligonucleotides, restoring the reading frame by skipping of exons, have become a potential treatment of Duchenne muscular dystrophy and other monogenetic diseases. Erythema followed by hyperpigmentation, fibrosis, and calcification were seen at the injection sites in all children. Ulcerations, which were difficult to heal, occurred in 5 of 7 children. Progression still occurred after switching to intravenous administration of drisapersen or even after stopping therapy. Systemic reactions included a reversible proteinuria and α1-microglobulinuria. Moreover, hypotrichosis was a common feature. Conclusion: Subcutaneous administration of drisapersen causes severe and progressive injection site effects.
Keywords
Duchenne muscular dystrophy, Antisense oligonucleotides, Drisapersen (PRO-051), Injection site reactions, THERAPY, RNA

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MLA
Hilhorst, Niels, et al. “Injection Site Reactions after Long-Term Subcutaneous Delivery of Drisapersen : A Retrospective Study.” EUROPEAN JOURNAL OF PEDIATRICS, vol. 178, no. 2, 2019, pp. 253–58.
APA
Hilhorst, N., Spanoudi-Kitrimi, I., Goemans, N., & Morren, M.-A. (2019). Injection site reactions after long-term subcutaneous delivery of drisapersen : a retrospective study. EUROPEAN JOURNAL OF PEDIATRICS, 178(2), 253–258.
Chicago author-date
Hilhorst, Niels, Ifigenia Spanoudi-Kitrimi, Nathalie Goemans, and Marie-Anne Morren. 2019. “Injection Site Reactions after Long-Term Subcutaneous Delivery of Drisapersen : A Retrospective Study.” EUROPEAN JOURNAL OF PEDIATRICS 178 (2): 253–58.
Chicago author-date (all authors)
Hilhorst, Niels, Ifigenia Spanoudi-Kitrimi, Nathalie Goemans, and Marie-Anne Morren. 2019. “Injection Site Reactions after Long-Term Subcutaneous Delivery of Drisapersen : A Retrospective Study.” EUROPEAN JOURNAL OF PEDIATRICS 178 (2): 253–258.
Vancouver
1.
Hilhorst N, Spanoudi-Kitrimi I, Goemans N, Morren M-A. Injection site reactions after long-term subcutaneous delivery of drisapersen : a retrospective study. EUROPEAN JOURNAL OF PEDIATRICS. 2019;178(2):253–8.
IEEE
[1]
N. Hilhorst, I. Spanoudi-Kitrimi, N. Goemans, and M.-A. Morren, “Injection site reactions after long-term subcutaneous delivery of drisapersen : a retrospective study,” EUROPEAN JOURNAL OF PEDIATRICS, vol. 178, no. 2, pp. 253–258, 2019.
@article{8659706,
  abstract     = {A retrospective study in which we reviewed the hospital files of a subset of 7 patients with Duchenne muscular dystrophy participating in the open-label phase I/II PRO051-02 study in Leuven. The objective of this study was to describe in detail the injection site reactions in these children treated with drisapersen (PRO-051), a 2′-O-methyl phosphorothioate RNA antisense oligonucleotide, that induces exon 51 skipping in Duchenne muscular dystrophy. Antisense oligonucleotides, restoring the reading frame by skipping of exons, have become a potential treatment of Duchenne muscular dystrophy and other monogenetic diseases. Erythema followed by hyperpigmentation, fibrosis, and calcification were seen at the injection sites in all children. Ulcerations, which were difficult to heal, occurred in 5 of 7 children. Progression still occurred after switching to intravenous administration of drisapersen or even after stopping therapy. Systemic reactions included a reversible proteinuria and α1-microglobulinuria. Moreover, hypotrichosis was a common feature.

Conclusion: Subcutaneous administration of drisapersen causes severe and progressive injection site effects.},
  author       = {Hilhorst, Niels and Spanoudi-Kitrimi, Ifigenia and Goemans, Nathalie and Morren, Marie-Anne},
  issn         = {0340-6199},
  journal      = {EUROPEAN JOURNAL OF PEDIATRICS},
  keywords     = {Duchenne muscular dystrophy,Antisense oligonucleotides,Drisapersen (PRO-051),Injection site reactions,THERAPY,RNA},
  language     = {eng},
  number       = {2},
  pages        = {253--258},
  title        = {Injection site reactions after long-term subcutaneous delivery of drisapersen : a retrospective study},
  url          = {http://dx.doi.org/10.1007/s00431-018-3272-1},
  volume       = {178},
  year         = {2019},
}

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