Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich’sAtaxia: Retrospective Study

GULTEKIN, MURAT; Yetkin, Mehmet Fatih

doi:https://doi.org/10.29399/npa.25047

Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich’sAtaxia: Retrospective Study

Mehmet Fatih YETKİN, (Erciyes Üniversitesi, Tıp Fakültesi, Nöroloji Anabilim Dalı, Kayseri, Türkiye)

Murat Gültekin (Erciyes Üniversitesi, Tıp Fakültesi, Nöroloji Anabilim Dalı, Kayseri, Türkiye)

Nöropsikiyatri Arşivi

6 0

Yıl: 2020 Cilt: 57 Sayı: 4 Sayfa Aralığı: 270 - 273 Metin Dili: İngilizce DOI: https://doi.org/10.29399/npa.25047 İndeks Tarihi: 03-05-2021

Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich’sAtaxia: Retrospective Study

Öz:

Friedreich’s Ataxia (FRDA) is the most common form of autosomal recessive ataxia. The disease primarily results from a GAA trinucleotide repeat expansion within the FXN gene in up to 97% of patients. The clinical presentation begins approximately between the ages of 5 and 15. The major clinical findings of FRDA are progressive extremity and gait ataxia. Although it is known that the disease is caused by low levels of functional protein in the target tissues, there is no effective treatment available for this pathology. However, significant improvements have been achieved in the research into pharmacological treatments for FRDA in recent years. Interferon-gamma (IFN-γ) has been shown to induce frataxin production in many cell types. In this study, the clinical features, tolerability, and the prognosis of individuals with FRDA to whom IFN-γwas administered in a university hospital were evaluated retrospectively and the results were discussed. To the best of our knowledge, this is the first study conducted in our country to evaluate the effect of IFN gamma on this patient group.

Anahtar Kelime:

Friedreich Ataksisi Tedavisinde İnterferon Gama Etkinliği ve Tolerabilitesi: Retrospektif Çalışma

Öz:

Friedreich Ataksisi (FRDA) en yaygın otozomal resesif ataksidir. Hastalık,hastaların %97’sine yakınında FXN geninde GAA trinükleotid tekrarartışından kaynaklanır. Klinik prezentasyon yaklaşık 5 ile 15 yaşları arasındabaşlar. FRDA’nın başlıca klinik bulguları progresif ekstremite ve yürüyüşataksisidir. Hastalığın hedef dokulardaki düşük fonksiyonel proteindüzeylerinden kaynaklandığı bilinmesine rağmen, bu patoloji için etkilibir tedavi yoktur. Bununla birlikte, son yıllarda FRDA için farmakolojiktedavilerle ilgili araştırmalarda önemli gelişmeler sağlanmıştır. İnterferongama (IFN-γ)’nın birçok hücre tipinde frataksin üretimini indüklediğigösterilmiştir. Bu çalışmada bir üniversite hastanesinde IFN-γ uygulananFRDA’lı bireylerin klinik özellikleri, toleransı ve prognozu retrospektifolarak değerlendirilmiş ve sonuçlar tartışılmıştır. Bildiğimiz kadarıylabu çalışma ülkemizde IFN-γ’nın bu hasta grubu üzerindeki etkisinideğerlendirmek için yapılan ilk çalışmadır

Anahtar Kelime:

Belge Türü: Makale Makale Türü: Araştırma Makalesi Erişim Türü: Erişime Açık

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APA	Yetkin M, GULTEKIN M (2020). Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich’sAtaxia: Retrospective Study. , 270 - 273. https://doi.org/10.29399/npa.25047
Chicago	Yetkin Mehmet Fatih,GULTEKIN MURAT Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich’sAtaxia: Retrospective Study. (2020): 270 - 273. https://doi.org/10.29399/npa.25047
MLA	Yetkin Mehmet Fatih,GULTEKIN MURAT Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich’sAtaxia: Retrospective Study. , 2020, ss.270 - 273. https://doi.org/10.29399/npa.25047
AMA	Yetkin M,GULTEKIN M Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich’sAtaxia: Retrospective Study. . 2020; 270 - 273. https://doi.org/10.29399/npa.25047
Vancouver	Yetkin M,GULTEKIN M Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich’sAtaxia: Retrospective Study. . 2020; 270 - 273. https://doi.org/10.29399/npa.25047
IEEE	Yetkin M,GULTEKIN M "Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich’sAtaxia: Retrospective Study." , ss.270 - 273, 2020. https://doi.org/10.29399/npa.25047
ISNAD	Yetkin, Mehmet Fatih - GULTEKIN, MURAT. "Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich’sAtaxia: Retrospective Study". (2020), 270-273. https://doi.org/https://doi.org/10.29399/npa.25047

APA	Yetkin M, GULTEKIN M (2020). Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich’sAtaxia: Retrospective Study. Nöropsikiyatri Arşivi, 57(4), 270 - 273. https://doi.org/10.29399/npa.25047
Chicago	Yetkin Mehmet Fatih,GULTEKIN MURAT Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich’sAtaxia: Retrospective Study. Nöropsikiyatri Arşivi 57, no.4 (2020): 270 - 273. https://doi.org/10.29399/npa.25047
MLA	Yetkin Mehmet Fatih,GULTEKIN MURAT Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich’sAtaxia: Retrospective Study. Nöropsikiyatri Arşivi, vol.57, no.4, 2020, ss.270 - 273. https://doi.org/10.29399/npa.25047
AMA	Yetkin M,GULTEKIN M Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich’sAtaxia: Retrospective Study. Nöropsikiyatri Arşivi. 2020; 57(4): 270 - 273. https://doi.org/10.29399/npa.25047
Vancouver	Yetkin M,GULTEKIN M Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich’sAtaxia: Retrospective Study. Nöropsikiyatri Arşivi. 2020; 57(4): 270 - 273. https://doi.org/10.29399/npa.25047
IEEE	Yetkin M,GULTEKIN M "Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich’sAtaxia: Retrospective Study." Nöropsikiyatri Arşivi, 57, ss.270 - 273, 2020. https://doi.org/10.29399/npa.25047
ISNAD	Yetkin, Mehmet Fatih - GULTEKIN, MURAT. "Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich’sAtaxia: Retrospective Study". Nöropsikiyatri Arşivi 57/4 (2020), 270-273. https://doi.org/https://doi.org/10.29399/npa.25047