Impact of Intravenous Trehalose Administration in Patients with Niemann-Pick Disease Types A and B
Niemann-Pick disease (NPD) types A (NPA) and B (NPB) are caused by deficiency of the acid sphingomyelinase enzyme, which is encoded by the gene, resulting in progressive pathogenic accumulation of lipids in tissues. Trehalose has been suggested as an autophagy inducer with therapeutic neuroprotectiv...
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| Veröffentlicht in: | Journal of clinical medicine 2022-01, Vol.11 (1), p.247 |
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| creator | Mobini, Moein Radbakhsh, Shabnam Kubaski, Francyne Eshraghi, Peyman Vakili, Saba Vakili, Rahim Khalili, Manijeh Varesvazirian, Majid Jamialahmadi, Tannaz Alamdaran, Seyed Ali Sayedi, Seyed Javad Rajabi, Omid Emami, Seyed Ahmad Reiner, Željko Sebkar, Amirhossein |
| description | Niemann-Pick disease (NPD) types A (NPA) and B (NPB) are caused by deficiency of the acid sphingomyelinase enzyme, which is encoded by the
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.
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).
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.
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. |
| doi_str_mv | 10.3390/jcm11010247 |
| format | Article |
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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.
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).
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.
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.</description><identifier>ISSN: 2077-0383</identifier><identifier>EISSN: 2077-0383</identifier><identifier>DOI: 10.3390/jcm11010247</identifier><identifier>PMID: 35011993</identifier><language>eng</language><publisher>Switzerland: MDPI AG</publisher><subject>Antioxidants ; Autophagy ; Bone marrow ; Brain diseases ; Clinical medicine ; Clinical trials ; Disease ; Enzymes ; FDA approval ; Genetic disorders ; Lipids ; Liver ; Metabolic disorders ; Oxidation ; Oxidative stress ; Pharmaceutical industry ; Preschool children ; Quality of life ; Spleen ; Statistical analysis ; Tumor necrosis factor-TNF</subject><ispartof>Journal of clinical medicine, 2022-01, Vol.11 (1), p.247</ispartof><rights>2022 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.</rights><rights>2022 by the authors. 2022</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c409t-dd96f7561aa97d3a22d36241d9283bac000650e68e9ea6ce8384b1d040be11673</citedby><cites>FETCH-LOGICAL-c409t-dd96f7561aa97d3a22d36241d9283bac000650e68e9ea6ce8384b1d040be11673</cites><orcidid>0000-0002-5966-2856 ; 0000-0002-8656-1444 ; 0000-0002-9330-0154</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC8745869/pdf/$$EPDF$$P50$$Gpubmedcentral$$Hfree_for_read</linktopdf><linktohtml>$$Uhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC8745869/$$EHTML$$P50$$Gpubmedcentral$$Hfree_for_read</linktohtml><link.rule.ids>230,314,724,777,781,882,27905,27906,53772,53774</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/35011993$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Mobini, Moein</creatorcontrib><creatorcontrib>Radbakhsh, Shabnam</creatorcontrib><creatorcontrib>Kubaski, Francyne</creatorcontrib><creatorcontrib>Eshraghi, Peyman</creatorcontrib><creatorcontrib>Vakili, Saba</creatorcontrib><creatorcontrib>Vakili, Rahim</creatorcontrib><creatorcontrib>Khalili, Manijeh</creatorcontrib><creatorcontrib>Varesvazirian, Majid</creatorcontrib><creatorcontrib>Jamialahmadi, Tannaz</creatorcontrib><creatorcontrib>Alamdaran, Seyed Ali</creatorcontrib><creatorcontrib>Sayedi, Seyed Javad</creatorcontrib><creatorcontrib>Rajabi, Omid</creatorcontrib><creatorcontrib>Emami, Seyed Ahmad</creatorcontrib><creatorcontrib>Reiner, Željko</creatorcontrib><creatorcontrib>Sebkar, Amirhossein</creatorcontrib><title>Impact of Intravenous Trehalose Administration in Patients with Niemann-Pick Disease Types A and B</title><title>Journal of clinical medicine</title><addtitle>J Clin Med</addtitle><description>Niemann-Pick disease (NPD) types A (NPA) and B (NPB) are caused by deficiency of the acid sphingomyelinase enzyme, which is encoded by the
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.
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).
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.
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.</description><subject>Antioxidants</subject><subject>Autophagy</subject><subject>Bone marrow</subject><subject>Brain diseases</subject><subject>Clinical medicine</subject><subject>Clinical trials</subject><subject>Disease</subject><subject>Enzymes</subject><subject>FDA approval</subject><subject>Genetic disorders</subject><subject>Lipids</subject><subject>Liver</subject><subject>Metabolic disorders</subject><subject>Oxidation</subject><subject>Oxidative stress</subject><subject>Pharmaceutical industry</subject><subject>Preschool children</subject><subject>Quality of life</subject><subject>Spleen</subject><subject>Statistical analysis</subject><subject>Tumor necrosis factor-TNF</subject><issn>2077-0383</issn><issn>2077-0383</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2022</creationdate><recordtype>article</recordtype><sourceid>ABUWG</sourceid><sourceid>AFKRA</sourceid><sourceid>AZQEC</sourceid><sourceid>BENPR</sourceid><sourceid>CCPQU</sourceid><sourceid>DWQXO</sourceid><recordid>eNpdkc1PGzEQxS0EIhHk1HtliQtSteCPXa99QQrfkRDlkJ4trz1pnO7a6XpDxX-PKSlKmYtHmt88vfFD6AslZ5wrcr6yHaWEElbWe2jMSF0XhEu-v9OP0CSlFcklZclofYhGvCKUKsXHqJl1a2MHHBd4FobePEOIm4TnPSxNGxPgqet88CmPBh8D9gE_5Q7CkPAfPyzxo4fOhFA8efsLX_sEJi_NX9aQ8BSb4PDlMTpYmDbBZPseoR-3N_Or--Lh-93savpQ2JKooXBOiUVdCWqMqh03jDkuWEmdYpI3xmb_oiIgJCgwwoLksmyoIyVpgFJR8yN08a673jQdOAtv97R63fvO9C86Gq__nwS_1D_js5Z1WUmhssDpVqCPvzeQBt35ZKFtTYD8KZoJKhURsqwyevIJXcVNH_J5fynGq4qzTH17p2wfU-ph8WGGEv0Wn96JL9Nfd_1_sP_C4q-YhJUY</recordid><startdate>20220104</startdate><enddate>20220104</enddate><creator>Mobini, Moein</creator><creator>Radbakhsh, Shabnam</creator><creator>Kubaski, Francyne</creator><creator>Eshraghi, Peyman</creator><creator>Vakili, Saba</creator><creator>Vakili, Rahim</creator><creator>Khalili, Manijeh</creator><creator>Varesvazirian, Majid</creator><creator>Jamialahmadi, Tannaz</creator><creator>Alamdaran, Seyed Ali</creator><creator>Sayedi, Seyed Javad</creator><creator>Rajabi, Omid</creator><creator>Emami, Seyed Ahmad</creator><creator>Reiner, Željko</creator><creator>Sebkar, Amirhossein</creator><general>MDPI AG</general><general>MDPI</general><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>3V.</scope><scope>7X7</scope><scope>7XB</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AZQEC</scope><scope>BENPR</scope><scope>CCPQU</scope><scope>DWQXO</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>K9.</scope><scope>M0S</scope><scope>PIMPY</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>PRINS</scope><scope>7X8</scope><scope>5PM</scope><orcidid>https://orcid.org/0000-0002-5966-2856</orcidid><orcidid>https://orcid.org/0000-0002-8656-1444</orcidid><orcidid>https://orcid.org/0000-0002-9330-0154</orcidid></search><sort><creationdate>20220104</creationdate><title>Impact of Intravenous Trehalose Administration in Patients with Niemann-Pick Disease Types A and B</title><author>Mobini, Moein ; 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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.
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).
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.
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.</abstract><cop>Switzerland</cop><pub>MDPI AG</pub><pmid>35011993</pmid><doi>10.3390/jcm11010247</doi><orcidid>https://orcid.org/0000-0002-5966-2856</orcidid><orcidid>https://orcid.org/0000-0002-8656-1444</orcidid><orcidid>https://orcid.org/0000-0002-9330-0154</orcidid><oa>free_for_read</oa></addata></record> |
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| source | Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals; PubMed Central Open Access; MDPI - Multidisciplinary Digital Publishing Institute; PubMed Central |
| subjects | Antioxidants Autophagy Bone marrow Brain diseases Clinical medicine Clinical trials Disease Enzymes FDA approval Genetic disorders Lipids Liver Metabolic disorders Oxidation Oxidative stress Pharmaceutical industry Preschool children Quality of life Spleen Statistical analysis Tumor necrosis factor-TNF |
| title | Impact of Intravenous Trehalose Administration in Patients with Niemann-Pick Disease Types A and B |
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