Neurological effects of recombinant human erythropoietin in Friedreich's ataxia: A clinical pilot trial
Identifieur interne : 002739 ( Main/Exploration ); précédent : 002738; suivant : 002740Neurological effects of recombinant human erythropoietin in Friedreich's ataxia: A clinical pilot trial
Auteurs : Sylvia Boesch [Autriche] ; Brigitte Sturm [Autriche] ; Sascha Hering [Autriche] ; Barbara Scheiber-Mojdehkar [Autriche] ; Hannes Steinkellner [Autriche] ; Hans Goldenberg [Autriche] ; Werner Poewe [Autriche]Source :
- Movement Disorders [ 0885-3185 ] ; 2008-10-15.
Descripteurs français
- Pascal (Inist)
- Wicri :
- topic : Homme.
English descriptors
- KwdEn :
- Adult, Clinical trial, Deoxyguanosine (analogs & derivatives), Deoxyguanosine (urine), Enzyme-Linked Immunosorbent Assay (methods), Erythropoietin, Erythropoietin (therapeutic use), Female, Follow-Up Studies, Friedreich Ataxia (drug therapy), Friedreich Ataxia (physiopathology), Friedreich Ataxia (urine), Friedreich ataxia, Friedreich's ataxia, Human, Humans, Iron-Binding Proteins (metabolism), Lymphocytes (metabolism), Male, Middle Aged, Nervous system diseases, Oxidative stress, Pilot Projects, Recombinant Proteins, Severity of Illness Index, Treatment Outcome, erythropoietin, frataxin, oxidative stress.
- MESH :
- chemical , analogs & derivatives : Deoxyguanosine.
- chemical , metabolism : Iron-Binding Proteins.
- chemical , therapeutic use : Erythropoietin.
- chemical , urine : Deoxyguanosine.
- drug therapy : Friedreich Ataxia.
- metabolism : Lymphocytes.
- methods : Enzyme-Linked Immunosorbent Assay.
- physiopathology : Friedreich Ataxia.
- urine : Friedreich Ataxia.
- Adult, Female, Follow-Up Studies, Humans, Male, Middle Aged, Pilot Projects, Recombinant Proteins, Severity of Illness Index, Treatment Outcome.
Abstract
In a “proof‐of‐concept” study, we demonstrated that recombinant human erythropoietin (rhuEPO) increases frataxin levels in Friedreich's ataxia (FRDA) patients. We now report a 6‐month open‐label clinical pilot study of safety and efficacy of rhuEPO treatment in FRDA. Eight adult FRDA patients received 2.000 IU rhuEPO thrice a week subcutaneously. Clinical outcome measures included Ataxia Rating Scales. Frataxin levels and indicators for oxidative stress were assessed. Hematological parameters were monitored biweekly. Scores in Ataxia Rating Scales such as FARS (P = 0.0063) and SARA (P = 0.0045) improved significantly. Frataxin levels increased (P = 0.017) while indicators of oxidative stress such as urine 8‐OHdG (P = 0.012) and peroxide levels decreased (P = 0.028). Increases in hematocrit requiring phlebotomies occurred in 4 of 8 patients. In this explorative open‐label clinical pilot study, we found an evidence for clinical improvement together with a persistent increase of frataxin levels and a reduction of oxidative stress parameters in patients with FRDA receiving chronic treatment with rhuEPO. Safety monitoring with regular blood cell counts and parameters of iron metabolism is a potential limitation of this approach. © 2008 Movement Disorder Society
Url:
DOI: 10.1002/mds.22294
Affiliations:
- Autriche
- Tyrol (Land), Vienne (Autriche)
- Innsbruck, Vienne (Autriche)
- Université de médecine d'Innsbruck
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Le document en format XML
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Disord.</title><idno type="ISSN">0885-3185</idno><idno type="eISSN">1531-8257</idno><imprint><publisher>Wiley Subscription Services, Inc., A Wiley Company</publisher><pubPlace>Hoboken</pubPlace><date type="published" when="2008-10-15">2008-10-15</date><biblScope unit="vol">23</biblScope><biblScope unit="issue">13</biblScope><biblScope unit="page" from="1940">1940</biblScope><biblScope unit="page" to="1944">1944</biblScope></imprint><idno type="ISSN">0885-3185</idno></series><idno type="istex">4C7B32F700236278AE6021ACB34F6AD02C74DE2A</idno><idno type="DOI">10.1002/mds.22294</idno><idno type="ArticleID">MDS22294</idno></biblStruct></sourceDesc><seriesStmt><idno type="ISSN">0885-3185</idno></seriesStmt></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Adult</term><term>Clinical trial</term><term>Deoxyguanosine (analogs & derivatives)</term><term>Deoxyguanosine (urine)</term><term>Enzyme-Linked Immunosorbent Assay (methods)</term><term>Erythropoietin</term><term>Erythropoietin (therapeutic use)</term><term>Female</term><term>Follow-Up Studies</term><term>Friedreich Ataxia (drug therapy)</term><term>Friedreich Ataxia (physiopathology)</term><term>Friedreich Ataxia (urine)</term><term>Friedreich ataxia</term><term>Friedreich's ataxia</term><term>Human</term><term>Humans</term><term>Iron-Binding Proteins (metabolism)</term><term>Lymphocytes (metabolism)</term><term>Male</term><term>Middle Aged</term><term>Nervous system diseases</term><term>Oxidative stress</term><term>Pilot Projects</term><term>Recombinant Proteins</term><term>Severity of Illness Index</term><term>Treatment Outcome</term><term>erythropoietin</term><term>frataxin</term><term>oxidative stress</term></keywords><keywords scheme="MESH" type="chemical" qualifier="analogs & derivatives" xml:lang="en"><term>Deoxyguanosine</term></keywords><keywords scheme="MESH" type="chemical" qualifier="metabolism" xml:lang="en"><term>Iron-Binding Proteins</term></keywords><keywords scheme="MESH" type="chemical" qualifier="therapeutic use" xml:lang="en"><term>Erythropoietin</term></keywords><keywords scheme="MESH" type="chemical" qualifier="urine" xml:lang="en"><term>Deoxyguanosine</term></keywords><keywords scheme="MESH" qualifier="drug therapy" xml:lang="en"><term>Friedreich Ataxia</term></keywords><keywords scheme="MESH" qualifier="metabolism" xml:lang="en"><term>Lymphocytes</term></keywords><keywords scheme="MESH" qualifier="methods" xml:lang="en"><term>Enzyme-Linked Immunosorbent Assay</term></keywords><keywords scheme="MESH" qualifier="physiopathology" xml:lang="en"><term>Friedreich Ataxia</term></keywords><keywords scheme="MESH" qualifier="urine" xml:lang="en"><term>Friedreich Ataxia</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Adult</term><term>Female</term><term>Follow-Up Studies</term><term>Humans</term><term>Male</term><term>Middle Aged</term><term>Pilot Projects</term><term>Recombinant Proteins</term><term>Severity of Illness Index</term><term>Treatment Outcome</term></keywords><keywords scheme="Pascal" xml:lang="fr"><term>Erythropoïétine</term><term>Essai clinique</term><term>Homme</term><term>Hérédodégénérescence spinocérébelleuse de Friedreich</term><term>Pathologie du système nerveux</term><term>Stress oxydatif</term></keywords><keywords scheme="Wicri" type="topic" xml:lang="fr"><term>Homme</term></keywords></textClass><langUsage><language ident="en">en</language></langUsage></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">In a “proof‐of‐concept” study, we demonstrated that recombinant human erythropoietin (rhuEPO) increases frataxin levels in Friedreich's ataxia (FRDA) patients. We now report a 6‐month open‐label clinical pilot study of safety and efficacy of rhuEPO treatment in FRDA. Eight adult FRDA patients received 2.000 IU rhuEPO thrice a week subcutaneously. Clinical outcome measures included Ataxia Rating Scales. Frataxin levels and indicators for oxidative stress were assessed. Hematological parameters were monitored biweekly. Scores in Ataxia Rating Scales such as FARS (P = 0.0063) and SARA (P = 0.0045) improved significantly. Frataxin levels increased (P = 0.017) while indicators of oxidative stress such as urine 8‐OHdG (P = 0.012) and peroxide levels decreased (P = 0.028). Increases in hematocrit requiring phlebotomies occurred in 4 of 8 patients. In this explorative open‐label clinical pilot study, we found an evidence for clinical improvement together with a persistent increase of frataxin levels and a reduction of oxidative stress parameters in patients with FRDA receiving chronic treatment with rhuEPO. Safety monitoring with regular blood cell counts and parameters of iron metabolism is a potential limitation of this approach. © 2008 Movement Disorder Society</div></front></TEI><affiliations><list><country><li>Autriche</li></country><region><li>Tyrol (Land)</li><li>Vienne (Autriche)</li></region><settlement><li>Innsbruck</li><li>Vienne (Autriche)</li></settlement><orgName><li>Université de médecine d'Innsbruck</li></orgName></list><tree><country name="Autriche"><noRegion><name sortKey="Boesch, Sylvia" sort="Boesch, Sylvia" uniqKey="Boesch S" first="Sylvia" last="Boesch">Sylvia Boesch</name></noRegion><name sortKey="Goldenberg, Hans" sort="Goldenberg, Hans" uniqKey="Goldenberg H" first="Hans" last="Goldenberg">Hans Goldenberg</name><name sortKey="Hering, Sascha" sort="Hering, Sascha" uniqKey="Hering S" first="Sascha" last="Hering">Sascha Hering</name><name sortKey="Poewe, Werner" sort="Poewe, Werner" uniqKey="Poewe W" first="Werner" last="Poewe">Werner Poewe</name><name sortKey="Scheiber Ojdehkar, Barbara" sort="Scheiber Ojdehkar, Barbara" uniqKey="Scheiber Ojdehkar B" first="Barbara" last="Scheiber-Mojdehkar">Barbara Scheiber-Mojdehkar</name><name sortKey="Steinkellner, Hannes" sort="Steinkellner, Hannes" uniqKey="Steinkellner H" first="Hannes" last="Steinkellner">Hannes Steinkellner</name><name sortKey="Sturm, Brigitte" sort="Sturm, Brigitte" uniqKey="Sturm B" first="Brigitte" last="Sturm">Brigitte Sturm</name></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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