Neurological effects of recombinant human erythropoietin in Friedreich's ataxia: a clinical pilot trial.
Identifieur interne : 002284 ( Ncbi/Curation ); précédent : 002283; suivant : 002285Neurological effects of recombinant human erythropoietin in Friedreich's ataxia: a clinical pilot trial.
Auteurs : Sylvia Boesch [Autriche] ; Brigitte Sturm ; Sascha Hering ; Barbara Scheiber-Mojdehkar ; Hannes Steinkellner ; Hans Goldenberg ; Werner Poewe [Autriche]Source :
- Movement disorders : official journal of the Movement Disorder Society [ 1531-8257 ] ; 2008.
English descriptors
- KwdEn :
- Adult, Deoxyguanosine (analogs & derivatives), Deoxyguanosine (urine), Enzyme-Linked Immunosorbent Assay (methods), Erythropoietin (therapeutic use), Female, Follow-Up Studies, Friedreich Ataxia (drug therapy), Friedreich Ataxia (physiopathology), Friedreich Ataxia (urine), Humans, Iron-Binding Proteins (metabolism), Lymphocytes (metabolism), Male, Middle Aged, Pilot Projects, Recombinant Proteins, Severity of Illness Index, Treatment Outcome.
- 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.
DOI: 10.1002/mds.22294
PubMed: 18759345
Links toward previous steps (curation, corpus...)
- to stream PubMed, to step Corpus: Pour aller vers cette notice dans l'étape Curation :002079
- to stream PubMed, to step Curation: Pour aller vers cette notice dans l'étape Curation :002079
- to stream PubMed, to step Checkpoint: Pour aller vers cette notice dans l'étape Curation :002190
- to stream Ncbi, to step Merge: Pour aller vers cette notice dans l'étape Curation :002284
Links to Exploration step
pubmed:18759345Le document en format XML
<record><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en">Neurological effects of recombinant human erythropoietin in Friedreich's ataxia: a clinical pilot trial.</title><author><name sortKey="Boesch, Sylvia" sort="Boesch, Sylvia" uniqKey="Boesch S" first="Sylvia" last="Boesch">Sylvia Boesch</name><affiliation wicri:level="1"><nlm:affiliation>Department of Neurology, Innsbruck Medical University, Innsbruck, Austria. sylvia.boesch@i-med.ac.at</nlm:affiliation><country xml:lang="fr">Autriche</country><wicri:regionArea>Department of Neurology, Innsbruck Medical University, Innsbruck</wicri:regionArea><wicri:noRegion>Innsbruck</wicri:noRegion></affiliation></author><author><name sortKey="Sturm, Brigitte" sort="Sturm, Brigitte" uniqKey="Sturm B" first="Brigitte" last="Sturm">Brigitte Sturm</name></author><author><name sortKey="Hering, Sascha" sort="Hering, Sascha" uniqKey="Hering S" first="Sascha" last="Hering">Sascha Hering</name></author><author><name sortKey="Scheiber Mojdehkar, Barbara" sort="Scheiber Mojdehkar, Barbara" uniqKey="Scheiber Mojdehkar B" first="Barbara" last="Scheiber-Mojdehkar">Barbara Scheiber-Mojdehkar</name></author><author><name sortKey="Steinkellner, Hannes" sort="Steinkellner, Hannes" uniqKey="Steinkellner H" first="Hannes" last="Steinkellner">Hannes Steinkellner</name></author><author><name sortKey="Goldenberg, Hans" sort="Goldenberg, Hans" uniqKey="Goldenberg H" first="Hans" last="Goldenberg">Hans Goldenberg</name></author><author><name sortKey="Poewe, Werner" sort="Poewe, Werner" uniqKey="Poewe W" first="Werner" last="Poewe">Werner Poewe</name><affiliation><country>Autriche</country><placeName><settlement type="city">Innsbruck</settlement><region nuts="2" type="region">Tyrol (Land)</region></placeName><orgName type="university">Université de médecine d'Innsbruck</orgName></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PubMed</idno><date when="2008">2008</date><idno type="doi">10.1002/mds.22294</idno><idno type="RBID">pubmed:18759345</idno><idno type="pmid">18759345</idno><idno type="wicri:Area/PubMed/Corpus">002079</idno><idno type="wicri:Area/PubMed/Curation">002079</idno><idno type="wicri:Area/PubMed/Checkpoint">002190</idno><idno type="wicri:Area/Ncbi/Merge">002284</idno><idno type="wicri:Area/Ncbi/Curation">002284</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en">Neurological effects of recombinant human erythropoietin in Friedreich's ataxia: a clinical pilot trial.</title><author><name sortKey="Boesch, Sylvia" sort="Boesch, Sylvia" uniqKey="Boesch S" first="Sylvia" last="Boesch">Sylvia Boesch</name><affiliation wicri:level="1"><nlm:affiliation>Department of Neurology, Innsbruck Medical University, Innsbruck, Austria. sylvia.boesch@i-med.ac.at</nlm:affiliation><country xml:lang="fr">Autriche</country><wicri:regionArea>Department of Neurology, Innsbruck Medical University, Innsbruck</wicri:regionArea><wicri:noRegion>Innsbruck</wicri:noRegion></affiliation></author><author><name sortKey="Sturm, Brigitte" sort="Sturm, Brigitte" uniqKey="Sturm B" first="Brigitte" last="Sturm">Brigitte Sturm</name></author><author><name sortKey="Hering, Sascha" sort="Hering, Sascha" uniqKey="Hering S" first="Sascha" last="Hering">Sascha Hering</name></author><author><name sortKey="Scheiber Mojdehkar, Barbara" sort="Scheiber Mojdehkar, Barbara" uniqKey="Scheiber Mojdehkar B" first="Barbara" last="Scheiber-Mojdehkar">Barbara Scheiber-Mojdehkar</name></author><author><name sortKey="Steinkellner, Hannes" sort="Steinkellner, Hannes" uniqKey="Steinkellner H" first="Hannes" last="Steinkellner">Hannes Steinkellner</name></author><author><name sortKey="Goldenberg, Hans" sort="Goldenberg, Hans" uniqKey="Goldenberg H" first="Hans" last="Goldenberg">Hans Goldenberg</name></author><author><name sortKey="Poewe, Werner" sort="Poewe, Werner" uniqKey="Poewe W" first="Werner" last="Poewe">Werner Poewe</name><affiliation><country>Autriche</country><placeName><settlement type="city">Innsbruck</settlement><region nuts="2" type="region">Tyrol (Land)</region></placeName><orgName type="university">Université de médecine d'Innsbruck</orgName></affiliation></author></analytic><series><title level="j">Movement disorders : official journal of the Movement Disorder Society</title><idno type="eISSN">1531-8257</idno><imprint><date when="2008" type="published">2008</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Adult</term><term>Deoxyguanosine (analogs & derivatives)</term><term>Deoxyguanosine (urine)</term><term>Enzyme-Linked Immunosorbent Assay (methods)</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>Humans</term><term>Iron-Binding Proteins (metabolism)</term><term>Lymphocytes (metabolism)</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="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></textClass></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.</div></front></TEI></record>Pour manipuler ce document sous Unix (Dilib)
EXPLOR_STEP=$WICRI_ROOT/Wicri/Santé/explor/MovDisordV3/Data/Ncbi/Curation
HfdSelect -h $EXPLOR_STEP/biblio.hfd -nk 002284 | SxmlIndent | more
Ou
HfdSelect -h $EXPLOR_AREA/Data/Ncbi/Curation/biblio.hfd -nk 002284 | SxmlIndent | more
Pour mettre un lien sur cette page dans le réseau Wicri
{{Explor lien
|wiki= Wicri/Santé
|area= MovDisordV3
|flux= Ncbi
|étape= Curation
|type= RBID
|clé= pubmed:18759345
|texte= Neurological effects of recombinant human erythropoietin in Friedreich's ataxia: a clinical pilot trial.
}}
Pour générer des pages wiki
HfdIndexSelect -h $EXPLOR_AREA/Data/Ncbi/Curation/RBID.i -Sk "pubmed:18759345" \
| HfdSelect -Kh $EXPLOR_AREA/Data/Ncbi/Curation/biblio.hfd \
| NlmPubMed2Wicri -a MovDisordV3
| This area was generated with Dilib version V0.6.23. |  |