prikaz prve stranice dokumenta Impact of Intravenous Trehalose Administration in Patients with Niemann–Pick Disease Types A and B
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Scientific paper - Original scientific paper
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 More authors...

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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, no. 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, no. 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, and sur.. Impact of Intravenous Trehalose Administration in Patients with Niemann–Pick Disease Types A and B. Journal of Clinical Medicine [Internet]. 2022 January 04 [cited 2025 April 26];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, no. 1, January 2022. [Online]. Available at: https://urn.nsk.hr/urn:nbn:hr:105:297647. [Accessed: 26 April 2025]

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Metadata
Title (english)Impact of Intravenous Trehalose Administration in Patients with Niemann–Pick Disease Types A and B
AuthorMoein Mobini
AuthorShabnam Radbakhsh
AuthorFrancyne Kubaski
AuthorPeyman Eshraghi
AuthorSaba Vakili
AuthorRahim Vakili
AuthorManijeh Khalili
AuthorMajid Varesvazirian
AuthorTannaz Jamialahmadi
AuthorSeyed Alamdaran
AuthorSeyed Sayedi
AuthorOmid Rajabi
AuthorSeyed Emami
AuthorŽeljko Reiner
AuthorAmirhossein Sahebkar
Author's institutionUniversity of Zagreb
School of Medicine
(Department of Internal Medicine)
Scientific / art field,
discipline and subdiscipline		BIOMEDICINE AND HEALTHCARE
Clinical Medical Sciences
Intensive Care
Abstract (english)
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.
Keywords (english)
Languageenglish
Publication typeScientific paper - Original scientific paper
Publication statusPublished
Peer reviewPeer review
Publication versionPublished version
Journal titleJournal of Clinical Medicine
Numberingvol. 11, no. 1, 247
e-ISSN2077-0383
DOIhttps://doi.org/10.3390/jcm11010247
URN:NBNurn:nbn:hr:105:297647
Publication2022-01-04
Document URLhttps://www.mdpi.com/2077-0383/11/1/247
Type of resourceText
Access conditionsOpen access
Terms of useLicence Attribution 4.0 International (CC BY 4.0) icon
Created on2022-07-04 10:45:37