prikaz prve stranice dokumenta Impact of Intravenous Trehalose Administration in Patients with Niemann–Pick Disease Types A and B
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Impact of Intravenous Trehalose Administration in Patients with Niemann–Pick Disease Types A and B
Journal of Clinical Medicine, 11 (2022), 1; 247. https://doi.org/10.3390/jcm11010247
Mobini, Moein; Radbakhsh, Shabnam; Kubaski, Francyne; Eshraghi, Peyman; Vakili, Saba; Vakili, Rahim; Khalili, Manijeh; Varesvazirian, Majid; Jamialahmadi, Tannaz; Alamdaran, Seyed; Sayedi, Seyed; Rajabi, Omid; Emami, Seyed; Reiner, Željko; Sahebkar, Amirhossein Više autora...

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Mobini, M., Radbakhsh, S., Kubaski, F., Eshraghi, P., Vakili, S., Vakili, R. ... Sahebkar, A. (2022). Impact of Intravenous Trehalose Administration in Patients with Niemann–Pick Disease Types A and B. Journal of Clinical Medicine, 11. (1). doi: 10.3390/jcm11010247

Mobini, Moein, et al. "Impact of Intravenous Trehalose Administration in Patients with Niemann–Pick Disease Types A and B." Journal of Clinical Medicine, vol. 11, br. 1, 2022. https://doi.org/10.3390/jcm11010247

Mobini, Moein, Shabnam Radbakhsh, Francyne Kubaski, Peyman Eshraghi, Saba Vakili, Rahim Vakili, Manijeh Khalili, et al. "Impact of Intravenous Trehalose Administration in Patients with Niemann–Pick Disease Types A and B." Journal of Clinical Medicine 11, br. 1 (2022). https://doi.org/10.3390/jcm11010247

Mobini, M., et al. (2022) 'Impact of Intravenous Trehalose Administration in Patients with Niemann–Pick Disease Types A and B', Journal of Clinical Medicine, 11(1). doi: 10.3390/jcm11010247

Mobini M, Radbakhsh S, Kubaski F, Eshraghi P, Vakili S, Vakili R, i sur.. Impact of Intravenous Trehalose Administration in Patients with Niemann–Pick Disease Types A and B. Journal of Clinical Medicine [Internet]. 04.01.2022. [pristupljeno 13.10.2024.];11(1). doi: 10.3390/jcm11010247

M. Mobini, et al., "Impact of Intravenous Trehalose Administration in Patients with Niemann–Pick Disease Types A and B", Journal of Clinical Medicine, vol. 11, br. 1, Siječanj 2022. [Online]. Dostupno na: https://urn.nsk.hr/urn:nbn:hr:105:297647. [Citirano: 13.10.2024.]

Za citiranje koristite ovu mrežnu adresu: https://urn.nsk.hr/urn:nbn:hr:105:297647
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Naslov (engleski)Impact of Intravenous Trehalose Administration in Patients with Niemann–Pick Disease Types A and B
AutorMoein Mobini
AutorShabnam Radbakhsh
AutorFrancyne Kubaski
AutorPeyman Eshraghi
AutorSaba Vakili
AutorRahim Vakili
AutorManijeh Khalili
AutorMajid Varesvazirian
AutorTannaz Jamialahmadi
AutorSeyed Alamdaran
AutorSeyed Sayedi
AutorOmid Rajabi
AutorSeyed Emami
AutorŽeljko Reiner
AutorAmirhossein Sahebkar
Autorova ustanovaSveučilište u Zagrebu
Medicinski fakultet
(KATEDRA ZA INTERNU MEDICINU)
Znanstveno / umjetničko
područje, polje i grana		BIOMEDICINA I ZDRAVSTVO
Kliničke medicinske znanosti
Intenzivna medicina
Sažetak (engleski)
Background and aims: Niemann-Pick disease (NPD) types A (NPA) and B (NPB) are caused by deficiency of the acid sphingomyelinase enzyme, which is encoded by the SMPD1 gene, resulting in progressive pathogenic accumulation of lipids in tissues. Trehalose has been suggested as an autophagy inducer with therapeutic neuroprotective effects. We performed a single-arm, open-label pilot study to assess the potential efficacy of trehalose treatment in patients with NPA and NPB patients. ------ Methods: Five patients with NPD type A and B were enrolled in an open-label, single-arm clinical trial. Trehalose was administrated intravenously (IV) (15 g/week) for three months. The efficacy of trehalose in the management of clinical symptoms was evaluated in patients by assessing the quality of life, serum biomarkers, and high-resolution computed tomography (HRCT) of the lungs at the baseline and end of the interventional trial (day 0 and week 12). ----- Results: The mean of TNO-AZL Preschool children Quality of Life (TAPQOL) scores increased in all patients after intervention at W12 compared to the baseline W0, although the difference was not statistically significant. The serum levels of lyso-SM-509 and lyso-SM were decreased in three and four patients out of five, respectively, compared with baseline. Elevated ALT and AST levels were decreased in all patients after 12 weeks of treatment; however, changes were not statistically significant. Pro-oxidant antioxidant balance (PAB) was also decreased and glutathione peroxidase (GPX) activity was increased in serum of patients at the end of the study. Imaging studies of spleen and lung HRCT showed improvement of symptoms in two patients. ----- Conclusions: Positive trends in health-related quality of life (HRQoL), serum biomarkers, and organomegaly were observed after 3 months of treatment with trehalose in patients with NPA and NPB. Although not statistically significant, due to the small number of patients enrolled, these results are encouraging and should be further explored.
Ključne riječi (engleski)
Jezikengleski
Vrsta publikacijeZnanstveni rad - Izvorni znanstveni rad
Status objaveObjavljen
Vrsta recenzijeRecenziran
Verzija publikacijeObjavljena verzija rada (izdavačev PDF)
Naslov časopisaJournal of Clinical Medicine
Brojčani podacivol. 11, br. 1, 247
e-ISSN2077-0383
DOIhttps://doi.org/10.3390/jcm11010247
URN:NBNurn:nbn:hr:105:297647
Datum objave publikacije2022-01-04
URL dokumentahttps://www.mdpi.com/2077-0383/11/1/247
Vrsta resursaTekst
Prava pristupaOtvoreni pristup
Uvjeti korištenjaIkona licence Imenovanje 4.0 međunarodna (CC BY 4.0)
Datum i vrijeme pohrane2022-07-04 10:45:37