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Erythropoietin in Spinocerebellar Ataxia Type 2: Feasibility and Proof‐of‐Principle Issues from a Randomized Controlled Study
Background Several pieces of evidence have shown the neurotrophic effect of erythropoietin (EPO) and its introduction in the therapeutic practice of neurological diseases. However, its usefulness in the treatment of spinocerebellar ataxia type 2 (SCA2) has not been proven despite the fact that it is...
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| Published in: | Movement disorders 2022-07, Vol.37 (7), p.1516-1525 |
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| container_title | Movement disorders |
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| creator | Rodriguez‐Labrada, Roberto Ortega‐Sanchez, Ricardo Hernández Casaña, Patricia Santos Morales, Orestes Padrón‐Estupiñan, Maria del Carmen Batista‐Nuñez, Maricela Jiménez Rodríguez, Daise Canales‐Ochoa, Nalia Peña Acosta, Arnoy Medrano Montero, Jacqueline Labrada Aguilera, Pedro Enrique Estupiñán Rodriguez, Annelie Vazquez‐Mojena, Yaimee Almaguer Gotay, Dennis Aymed‐García, Judey García‐García, Idrian Torres Vega, Reydenis Viada González, Carmen Valenzuela Silva, Carmen M. Silva Ricardo, Yanelis Columbié Ximelis, Jorge Tribin Rivero, Kenia Valle Cabrera, Roselin García‐Rodriguez, Julio Cesar Crombet Ramos, Tania Amaro‐González, Daniel Rodriguez‐Obaya, Teresita Velázquez‐Pérez, Luis |
| description | Background
Several pieces of evidence have shown the neurotrophic effect of erythropoietin (EPO) and its introduction in the therapeutic practice of neurological diseases. However, its usefulness in the treatment of spinocerebellar ataxia type 2 (SCA2) has not been proven despite the fact that it is endogenously reduced in these patients.
Objective
The study aims to investigate the safety, tolerability, and clinical effects of a nasally administered recombinant EPO in SCA2 patients.
Methods
Thirty‐four patients were enrolled in this double‐blind, randomized, placebo‐controlled, phase I–II clinical trial of the nasally administered human‐recombinant EPO (NeuroEPO) for 6 months. The primary outcome was the change in the spinocerebellar ataxia functional index (SCAFI), while other motor, neuropsychological, and oculomotor measures were assessed.
Results
The 6‐month changes in SCAFI score were slightly higher in the patients allocated to NeuroEPO treatment than placebo in spite of the important placebo effect observed for this parameter. However, saccade latency was significantly decreased in the NeuroEPO group but not in placebo. The frequency and severity of adverse events were similar between both groups, without evidences of hematopoietic activity of the drug.
Conclusions
This study demonstrated the safety and tolerability of NeuroEPO in SCA2 patients after 6 months of treatments and suggested a small clinical effect of this drug on motor and cognitive abnormalities, but confirmatory studies are warranted. © 2022 International Parkinson and Movement Disorder Society. |
| doi_str_mv | 10.1002/mds.29045 |
| format | article |
| fullrecord | <record><control><sourceid>proquest_cross</sourceid><recordid>TN_cdi_proquest_miscellaneous_2668912513</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>2691769988</sourcerecordid><originalsourceid>FETCH-LOGICAL-c3535-fa109394f9668140769a5c67f641c9ee18512942592c11336646241bd192962e3</originalsourceid><addsrcrecordid>eNp1kcFO3DAQhi3UCpaFAy9QWeoFDgGPHTtxb2iBFokKxMI58iYT1SiJUzsRhBO8Ac_YJ6lhaQ-VKln2HD5_M6OfkD1gh8AYP2qrcMg1S-UGmYEUkORcZh_IjOW5TATkcotsh3DHGIAEtUm2hFQsyzI1I8-nfhp-eNc7i4PtaDzL3nauRI8rbBrj6fFgHqyhN1OPlH-hZ2iCXdnGDhM1XUWvvHP1r6eXt-vK2660fYP0PIQRA629a6mh15F0rX3Eii5cN3jXNLFcDmM17ZCPtWkC7r6_c3J7dnqz-JZcXH49XxxfJKWQQia1AaaFTmutVA4py5Q2slRZrVIoNWLcErhOudS8BBBCqVTxFFYVaK4VRzEn-2tv793PONpQtDaUryt26MZQ8OjVwGX8Oyef_0Hv3Oi7OF2kNMTWOs8jdbCmSu9C8FgXvbet8VMBrHjNpYi5FG-5RPbTu3FctVj9Jf8EEYGjNXBvG5z-byq-nyzXyt-iG5fg</addsrcrecordid><sourcetype>Aggregation Database</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>2691769988</pqid></control><display><type>article</type><title>Erythropoietin in Spinocerebellar Ataxia Type 2: Feasibility and Proof‐of‐Principle Issues from a Randomized Controlled Study</title><source>Wiley-Blackwell Journals</source><creator>Rodriguez‐Labrada, Roberto ; Ortega‐Sanchez, Ricardo ; Hernández Casaña, Patricia ; Santos Morales, Orestes ; Padrón‐Estupiñan, Maria del Carmen ; Batista‐Nuñez, Maricela ; Jiménez Rodríguez, Daise ; Canales‐Ochoa, Nalia ; Peña Acosta, Arnoy ; Medrano Montero, Jacqueline ; Labrada Aguilera, Pedro Enrique ; Estupiñán Rodriguez, Annelie ; Vazquez‐Mojena, Yaimee ; Almaguer Gotay, Dennis ; Aymed‐García, Judey ; García‐García, Idrian ; Torres Vega, Reydenis ; Viada González, Carmen ; Valenzuela Silva, Carmen M. ; Silva Ricardo, Yanelis ; Columbié Ximelis, Jorge ; Tribin Rivero, Kenia ; Valle Cabrera, Roselin ; García‐Rodriguez, Julio Cesar ; Crombet Ramos, Tania ; Amaro‐González, Daniel ; Rodriguez‐Obaya, Teresita ; Velázquez‐Pérez, Luis</creator><creatorcontrib>Rodriguez‐Labrada, Roberto ; Ortega‐Sanchez, Ricardo ; Hernández Casaña, Patricia ; Santos Morales, Orestes ; Padrón‐Estupiñan, Maria del Carmen ; Batista‐Nuñez, Maricela ; Jiménez Rodríguez, Daise ; Canales‐Ochoa, Nalia ; Peña Acosta, Arnoy ; Medrano Montero, Jacqueline ; Labrada Aguilera, Pedro Enrique ; Estupiñán Rodriguez, Annelie ; Vazquez‐Mojena, Yaimee ; Almaguer Gotay, Dennis ; Aymed‐García, Judey ; García‐García, Idrian ; Torres Vega, Reydenis ; Viada González, Carmen ; Valenzuela Silva, Carmen M. ; Silva Ricardo, Yanelis ; Columbié Ximelis, Jorge ; Tribin Rivero, Kenia ; Valle Cabrera, Roselin ; García‐Rodriguez, Julio Cesar ; Crombet Ramos, Tania ; Amaro‐González, Daniel ; Rodriguez‐Obaya, Teresita ; Velázquez‐Pérez, Luis</creatorcontrib><description>Background
Several pieces of evidence have shown the neurotrophic effect of erythropoietin (EPO) and its introduction in the therapeutic practice of neurological diseases. However, its usefulness in the treatment of spinocerebellar ataxia type 2 (SCA2) has not been proven despite the fact that it is endogenously reduced in these patients.
Objective
The study aims to investigate the safety, tolerability, and clinical effects of a nasally administered recombinant EPO in SCA2 patients.
Methods
Thirty‐four patients were enrolled in this double‐blind, randomized, placebo‐controlled, phase I–II clinical trial of the nasally administered human‐recombinant EPO (NeuroEPO) for 6 months. The primary outcome was the change in the spinocerebellar ataxia functional index (SCAFI), while other motor, neuropsychological, and oculomotor measures were assessed.
Results
The 6‐month changes in SCAFI score were slightly higher in the patients allocated to NeuroEPO treatment than placebo in spite of the important placebo effect observed for this parameter. However, saccade latency was significantly decreased in the NeuroEPO group but not in placebo. The frequency and severity of adverse events were similar between both groups, without evidences of hematopoietic activity of the drug.
Conclusions
This study demonstrated the safety and tolerability of NeuroEPO in SCA2 patients after 6 months of treatments and suggested a small clinical effect of this drug on motor and cognitive abnormalities, but confirmatory studies are warranted. © 2022 International Parkinson and Movement Disorder Society.</description><identifier>ISSN: 0885-3185</identifier><identifier>EISSN: 1531-8257</identifier><identifier>DOI: 10.1002/mds.29045</identifier><identifier>PMID: 35607776</identifier><language>eng</language><publisher>Hoboken, USA: John Wiley & Sons, Inc</publisher><subject>Ataxia ; clinical trial ; Clinical trials ; Cognitive ability ; Erythropoietin ; human‐recombinant erythropoietin ; Intranasal administration ; Latency ; Movement disorders ; Neurological diseases ; Patients ; Placebos ; Saccadic eye movements ; Spinocerebellar ataxia ; spinocerebellar ataxia type 2</subject><ispartof>Movement disorders, 2022-07, Vol.37 (7), p.1516-1525</ispartof><rights>2022 International Parkinson and Movement Disorder Society.</rights><rights>2022 International Parkinson and Movement Disorder Society</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c3535-fa109394f9668140769a5c67f641c9ee18512942592c11336646241bd192962e3</citedby><cites>FETCH-LOGICAL-c3535-fa109394f9668140769a5c67f641c9ee18512942592c11336646241bd192962e3</cites><orcidid>0000-0003-2479-3941 ; 0000-0003-3193-7683 ; 0000-0003-1628-2703</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://onlinelibrary.wiley.com/doi/pdf/10.1002%2Fmds.29045$$EPDF$$P50$$Gwiley$$H</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1002%2Fmds.29045$$EHTML$$P50$$Gwiley$$H</linktohtml><link.rule.ids>315,786,790,27957,27958,50923,51032</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/35607776$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Rodriguez‐Labrada, Roberto</creatorcontrib><creatorcontrib>Ortega‐Sanchez, Ricardo</creatorcontrib><creatorcontrib>Hernández Casaña, Patricia</creatorcontrib><creatorcontrib>Santos Morales, Orestes</creatorcontrib><creatorcontrib>Padrón‐Estupiñan, Maria del Carmen</creatorcontrib><creatorcontrib>Batista‐Nuñez, Maricela</creatorcontrib><creatorcontrib>Jiménez Rodríguez, Daise</creatorcontrib><creatorcontrib>Canales‐Ochoa, Nalia</creatorcontrib><creatorcontrib>Peña Acosta, Arnoy</creatorcontrib><creatorcontrib>Medrano Montero, Jacqueline</creatorcontrib><creatorcontrib>Labrada Aguilera, Pedro Enrique</creatorcontrib><creatorcontrib>Estupiñán Rodriguez, Annelie</creatorcontrib><creatorcontrib>Vazquez‐Mojena, Yaimee</creatorcontrib><creatorcontrib>Almaguer Gotay, Dennis</creatorcontrib><creatorcontrib>Aymed‐García, Judey</creatorcontrib><creatorcontrib>García‐García, Idrian</creatorcontrib><creatorcontrib>Torres Vega, Reydenis</creatorcontrib><creatorcontrib>Viada González, Carmen</creatorcontrib><creatorcontrib>Valenzuela Silva, Carmen M.</creatorcontrib><creatorcontrib>Silva Ricardo, Yanelis</creatorcontrib><creatorcontrib>Columbié Ximelis, Jorge</creatorcontrib><creatorcontrib>Tribin Rivero, Kenia</creatorcontrib><creatorcontrib>Valle Cabrera, Roselin</creatorcontrib><creatorcontrib>García‐Rodriguez, Julio Cesar</creatorcontrib><creatorcontrib>Crombet Ramos, Tania</creatorcontrib><creatorcontrib>Amaro‐González, Daniel</creatorcontrib><creatorcontrib>Rodriguez‐Obaya, Teresita</creatorcontrib><creatorcontrib>Velázquez‐Pérez, Luis</creatorcontrib><title>Erythropoietin in Spinocerebellar Ataxia Type 2: Feasibility and Proof‐of‐Principle Issues from a Randomized Controlled Study</title><title>Movement disorders</title><addtitle>Mov Disord</addtitle><description>Background
Several pieces of evidence have shown the neurotrophic effect of erythropoietin (EPO) and its introduction in the therapeutic practice of neurological diseases. However, its usefulness in the treatment of spinocerebellar ataxia type 2 (SCA2) has not been proven despite the fact that it is endogenously reduced in these patients.
Objective
The study aims to investigate the safety, tolerability, and clinical effects of a nasally administered recombinant EPO in SCA2 patients.
Methods
Thirty‐four patients were enrolled in this double‐blind, randomized, placebo‐controlled, phase I–II clinical trial of the nasally administered human‐recombinant EPO (NeuroEPO) for 6 months. The primary outcome was the change in the spinocerebellar ataxia functional index (SCAFI), while other motor, neuropsychological, and oculomotor measures were assessed.
Results
The 6‐month changes in SCAFI score were slightly higher in the patients allocated to NeuroEPO treatment than placebo in spite of the important placebo effect observed for this parameter. However, saccade latency was significantly decreased in the NeuroEPO group but not in placebo. The frequency and severity of adverse events were similar between both groups, without evidences of hematopoietic activity of the drug.
Conclusions
This study demonstrated the safety and tolerability of NeuroEPO in SCA2 patients after 6 months of treatments and suggested a small clinical effect of this drug on motor and cognitive abnormalities, but confirmatory studies are warranted. © 2022 International Parkinson and Movement Disorder Society.</description><subject>Ataxia</subject><subject>clinical trial</subject><subject>Clinical trials</subject><subject>Cognitive ability</subject><subject>Erythropoietin</subject><subject>human‐recombinant erythropoietin</subject><subject>Intranasal administration</subject><subject>Latency</subject><subject>Movement disorders</subject><subject>Neurological diseases</subject><subject>Patients</subject><subject>Placebos</subject><subject>Saccadic eye movements</subject><subject>Spinocerebellar ataxia</subject><subject>spinocerebellar ataxia type 2</subject><issn>0885-3185</issn><issn>1531-8257</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2022</creationdate><recordtype>article</recordtype><recordid>eNp1kcFO3DAQhi3UCpaFAy9QWeoFDgGPHTtxb2iBFokKxMI58iYT1SiJUzsRhBO8Ac_YJ6lhaQ-VKln2HD5_M6OfkD1gh8AYP2qrcMg1S-UGmYEUkORcZh_IjOW5TATkcotsh3DHGIAEtUm2hFQsyzI1I8-nfhp-eNc7i4PtaDzL3nauRI8rbBrj6fFgHqyhN1OPlH-hZ2iCXdnGDhM1XUWvvHP1r6eXt-vK2660fYP0PIQRA629a6mh15F0rX3Eii5cN3jXNLFcDmM17ZCPtWkC7r6_c3J7dnqz-JZcXH49XxxfJKWQQia1AaaFTmutVA4py5Q2slRZrVIoNWLcErhOudS8BBBCqVTxFFYVaK4VRzEn-2tv793PONpQtDaUryt26MZQ8OjVwGX8Oyef_0Hv3Oi7OF2kNMTWOs8jdbCmSu9C8FgXvbet8VMBrHjNpYi5FG-5RPbTu3FctVj9Jf8EEYGjNXBvG5z-byq-nyzXyt-iG5fg</recordid><startdate>202207</startdate><enddate>202207</enddate><creator>Rodriguez‐Labrada, Roberto</creator><creator>Ortega‐Sanchez, Ricardo</creator><creator>Hernández Casaña, Patricia</creator><creator>Santos Morales, Orestes</creator><creator>Padrón‐Estupiñan, Maria del Carmen</creator><creator>Batista‐Nuñez, Maricela</creator><creator>Jiménez Rodríguez, Daise</creator><creator>Canales‐Ochoa, Nalia</creator><creator>Peña Acosta, Arnoy</creator><creator>Medrano Montero, Jacqueline</creator><creator>Labrada Aguilera, Pedro Enrique</creator><creator>Estupiñán Rodriguez, Annelie</creator><creator>Vazquez‐Mojena, Yaimee</creator><creator>Almaguer Gotay, Dennis</creator><creator>Aymed‐García, Judey</creator><creator>García‐García, Idrian</creator><creator>Torres Vega, Reydenis</creator><creator>Viada González, Carmen</creator><creator>Valenzuela Silva, Carmen M.</creator><creator>Silva Ricardo, Yanelis</creator><creator>Columbié Ximelis, Jorge</creator><creator>Tribin Rivero, Kenia</creator><creator>Valle Cabrera, Roselin</creator><creator>García‐Rodriguez, Julio Cesar</creator><creator>Crombet Ramos, Tania</creator><creator>Amaro‐González, Daniel</creator><creator>Rodriguez‐Obaya, Teresita</creator><creator>Velázquez‐Pérez, Luis</creator><general>John Wiley & Sons, Inc</general><general>Wiley Subscription Services, Inc</general><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7TK</scope><scope>8FD</scope><scope>FR3</scope><scope>K9.</scope><scope>NAPCQ</scope><scope>P64</scope><scope>RC3</scope><scope>7X8</scope><orcidid>https://orcid.org/0000-0003-2479-3941</orcidid><orcidid>https://orcid.org/0000-0003-3193-7683</orcidid><orcidid>https://orcid.org/0000-0003-1628-2703</orcidid></search><sort><creationdate>202207</creationdate><title>Erythropoietin in Spinocerebellar Ataxia Type 2: Feasibility and Proof‐of‐Principle Issues from a Randomized Controlled Study</title><author>Rodriguez‐Labrada, Roberto ; Ortega‐Sanchez, Ricardo ; Hernández Casaña, Patricia ; Santos Morales, Orestes ; Padrón‐Estupiñan, Maria del Carmen ; Batista‐Nuñez, Maricela ; Jiménez Rodríguez, Daise ; Canales‐Ochoa, Nalia ; Peña Acosta, Arnoy ; Medrano Montero, Jacqueline ; Labrada Aguilera, Pedro Enrique ; Estupiñán Rodriguez, Annelie ; Vazquez‐Mojena, Yaimee ; Almaguer Gotay, Dennis ; Aymed‐García, Judey ; García‐García, Idrian ; Torres Vega, Reydenis ; Viada González, Carmen ; Valenzuela Silva, Carmen M. ; Silva Ricardo, Yanelis ; Columbié Ximelis, Jorge ; Tribin Rivero, Kenia ; Valle Cabrera, Roselin ; García‐Rodriguez, Julio Cesar ; Crombet Ramos, Tania ; Amaro‐González, Daniel ; Rodriguez‐Obaya, Teresita ; Velázquez‐Pérez, Luis</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c3535-fa109394f9668140769a5c67f641c9ee18512942592c11336646241bd192962e3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2022</creationdate><topic>Ataxia</topic><topic>clinical trial</topic><topic>Clinical trials</topic><topic>Cognitive ability</topic><topic>Erythropoietin</topic><topic>human‐recombinant erythropoietin</topic><topic>Intranasal administration</topic><topic>Latency</topic><topic>Movement disorders</topic><topic>Neurological diseases</topic><topic>Patients</topic><topic>Placebos</topic><topic>Saccadic eye movements</topic><topic>Spinocerebellar ataxia</topic><topic>spinocerebellar ataxia type 2</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Rodriguez‐Labrada, Roberto</creatorcontrib><creatorcontrib>Ortega‐Sanchez, Ricardo</creatorcontrib><creatorcontrib>Hernández Casaña, Patricia</creatorcontrib><creatorcontrib>Santos Morales, Orestes</creatorcontrib><creatorcontrib>Padrón‐Estupiñan, Maria del Carmen</creatorcontrib><creatorcontrib>Batista‐Nuñez, Maricela</creatorcontrib><creatorcontrib>Jiménez Rodríguez, Daise</creatorcontrib><creatorcontrib>Canales‐Ochoa, Nalia</creatorcontrib><creatorcontrib>Peña Acosta, Arnoy</creatorcontrib><creatorcontrib>Medrano Montero, Jacqueline</creatorcontrib><creatorcontrib>Labrada Aguilera, Pedro Enrique</creatorcontrib><creatorcontrib>Estupiñán Rodriguez, Annelie</creatorcontrib><creatorcontrib>Vazquez‐Mojena, Yaimee</creatorcontrib><creatorcontrib>Almaguer Gotay, Dennis</creatorcontrib><creatorcontrib>Aymed‐García, Judey</creatorcontrib><creatorcontrib>García‐García, Idrian</creatorcontrib><creatorcontrib>Torres Vega, Reydenis</creatorcontrib><creatorcontrib>Viada González, Carmen</creatorcontrib><creatorcontrib>Valenzuela Silva, Carmen M.</creatorcontrib><creatorcontrib>Silva Ricardo, Yanelis</creatorcontrib><creatorcontrib>Columbié Ximelis, Jorge</creatorcontrib><creatorcontrib>Tribin Rivero, Kenia</creatorcontrib><creatorcontrib>Valle Cabrera, Roselin</creatorcontrib><creatorcontrib>García‐Rodriguez, Julio Cesar</creatorcontrib><creatorcontrib>Crombet Ramos, Tania</creatorcontrib><creatorcontrib>Amaro‐González, Daniel</creatorcontrib><creatorcontrib>Rodriguez‐Obaya, Teresita</creatorcontrib><creatorcontrib>Velázquez‐Pérez, Luis</creatorcontrib><collection>PubMed</collection><collection>CrossRef</collection><collection>Neurosciences Abstracts</collection><collection>Technology Research Database</collection><collection>Engineering Research Database</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>Nursing & Allied Health Premium</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>Genetics Abstracts</collection><collection>MEDLINE - Academic</collection><jtitle>Movement disorders</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Rodriguez‐Labrada, Roberto</au><au>Ortega‐Sanchez, Ricardo</au><au>Hernández Casaña, Patricia</au><au>Santos Morales, Orestes</au><au>Padrón‐Estupiñan, Maria del Carmen</au><au>Batista‐Nuñez, Maricela</au><au>Jiménez Rodríguez, Daise</au><au>Canales‐Ochoa, Nalia</au><au>Peña Acosta, Arnoy</au><au>Medrano Montero, Jacqueline</au><au>Labrada Aguilera, Pedro Enrique</au><au>Estupiñán Rodriguez, Annelie</au><au>Vazquez‐Mojena, Yaimee</au><au>Almaguer Gotay, Dennis</au><au>Aymed‐García, Judey</au><au>García‐García, Idrian</au><au>Torres Vega, Reydenis</au><au>Viada González, Carmen</au><au>Valenzuela Silva, Carmen M.</au><au>Silva Ricardo, Yanelis</au><au>Columbié Ximelis, Jorge</au><au>Tribin Rivero, Kenia</au><au>Valle Cabrera, Roselin</au><au>García‐Rodriguez, Julio Cesar</au><au>Crombet Ramos, Tania</au><au>Amaro‐González, Daniel</au><au>Rodriguez‐Obaya, Teresita</au><au>Velázquez‐Pérez, Luis</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Erythropoietin in Spinocerebellar Ataxia Type 2: Feasibility and Proof‐of‐Principle Issues from a Randomized Controlled Study</atitle><jtitle>Movement disorders</jtitle><addtitle>Mov Disord</addtitle><date>2022-07</date><risdate>2022</risdate><volume>37</volume><issue>7</issue><spage>1516</spage><epage>1525</epage><pages>1516-1525</pages><issn>0885-3185</issn><eissn>1531-8257</eissn><notes>Relevant conflicts of interest/financial disclosures</notes><notes>All authors received funding from the Fund for Financing Science and Innovation from the Cuban Ministry of Science, Technology and Environment. None has received any other funding for the past 12 months relevant to or not for this manuscript. The authors declare that they have no conflict of interest.</notes><notes>Funding agencies</notes><notes>This work was supported by the Cuban Ministry of Public Health and the Center of Molecular Immunology, Havana, Cuba.</notes><notes>ObjectType-Article-1</notes><notes>SourceType-Scholarly Journals-1</notes><notes>ObjectType-Feature-2</notes><notes>ObjectType-News-3</notes><notes>content type line 23</notes><abstract>Background
Several pieces of evidence have shown the neurotrophic effect of erythropoietin (EPO) and its introduction in the therapeutic practice of neurological diseases. However, its usefulness in the treatment of spinocerebellar ataxia type 2 (SCA2) has not been proven despite the fact that it is endogenously reduced in these patients.
Objective
The study aims to investigate the safety, tolerability, and clinical effects of a nasally administered recombinant EPO in SCA2 patients.
Methods
Thirty‐four patients were enrolled in this double‐blind, randomized, placebo‐controlled, phase I–II clinical trial of the nasally administered human‐recombinant EPO (NeuroEPO) for 6 months. The primary outcome was the change in the spinocerebellar ataxia functional index (SCAFI), while other motor, neuropsychological, and oculomotor measures were assessed.
Results
The 6‐month changes in SCAFI score were slightly higher in the patients allocated to NeuroEPO treatment than placebo in spite of the important placebo effect observed for this parameter. However, saccade latency was significantly decreased in the NeuroEPO group but not in placebo. The frequency and severity of adverse events were similar between both groups, without evidences of hematopoietic activity of the drug.
Conclusions
This study demonstrated the safety and tolerability of NeuroEPO in SCA2 patients after 6 months of treatments and suggested a small clinical effect of this drug on motor and cognitive abnormalities, but confirmatory studies are warranted. © 2022 International Parkinson and Movement Disorder Society.</abstract><cop>Hoboken, USA</cop><pub>John Wiley & Sons, Inc</pub><pmid>35607776</pmid><doi>10.1002/mds.29045</doi><tpages>10</tpages><orcidid>https://orcid.org/0000-0003-2479-3941</orcidid><orcidid>https://orcid.org/0000-0003-3193-7683</orcidid><orcidid>https://orcid.org/0000-0003-1628-2703</orcidid></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0885-3185 |
| ispartof | Movement disorders, 2022-07, Vol.37 (7), p.1516-1525 |
| issn | 0885-3185 1531-8257 |
| language | eng |
| recordid | cdi_proquest_miscellaneous_2668912513 |
| source | Wiley-Blackwell Journals |
| subjects | Ataxia clinical trial Clinical trials Cognitive ability Erythropoietin human‐recombinant erythropoietin Intranasal administration Latency Movement disorders Neurological diseases Patients Placebos Saccadic eye movements Spinocerebellar ataxia spinocerebellar ataxia type 2 |
| title | Erythropoietin in Spinocerebellar Ataxia Type 2: Feasibility and Proof‐of‐Principle Issues from a Randomized Controlled Study |
| url | http://sfxeu10.hosted.exlibrisgroup.com/loughborough?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2024-09-21T17%3A48%3A08IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-proquest_cross&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=Erythropoietin%20in%20Spinocerebellar%20Ataxia%20Type%202:%20Feasibility%20and%20Proof%E2%80%90of%E2%80%90Principle%20Issues%20from%20a%20Randomized%20Controlled%20Study&rft.jtitle=Movement%20disorders&rft.au=Rodriguez%E2%80%90Labrada,%20Roberto&rft.date=2022-07&rft.volume=37&rft.issue=7&rft.spage=1516&rft.epage=1525&rft.pages=1516-1525&rft.issn=0885-3185&rft.eissn=1531-8257&rft_id=info:doi/10.1002/mds.29045&rft_dat=%3Cproquest_cross%3E2691769988%3C/proquest_cross%3E%3Cgrp_id%3Ecdi_FETCH-LOGICAL-c3535-fa109394f9668140769a5c67f641c9ee18512942592c11336646241bd192962e3%3C/grp_id%3E%3Coa%3E%3C/oa%3E%3Curl%3E%3C/url%3E&rft_id=info:oai/&rft_pqid=2691769988&rft_id=info:pmid/35607776&rfr_iscdi=true |