MovDisordV3, PascalFrancis, Corpus, bibRecord, 001085

Neurological Effects of Recombinant Human Erythropoietin in Friedreich's Ataxia : A Clinical Pilot Trial

Identifieur interne : 001085 ( PascalFrancis/Corpus ); précédent : 001084; suivant : 001086

Neurological Effects of Recombinant Human Erythropoietin in Friedreich's Ataxia : A Clinical Pilot Trial

Auteurs : Sylvia Boesch ; Brigitte Sturm ; Sascha Hering ; Barbara Scheiber-Mojdehkar ; Hannes Steinkellner ; Hans Goldenberg ; Werner Poewe

Source :

Movement disorders [ 0885-3185 ] ; 2008.

RBID : Pascal:08-0522203

Descripteurs français

Pascal (Inist)
- Hérédodégénérescence spinocérébelleuse de Friedreich, Pathologie du système nerveux, Homme, Erythropoïétine, Essai clinique, Stress oxydatif.

English descriptors

KwdEn :
- Clinical trial, Erythropoietin, Friedreich ataxia, Human, Nervous system diseases, Oxidative stress.

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.

Notice en format standard (ISO 2709)

Pour connaître la documentation sur le format Inist Standard.

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A11	`01`	`1`		`@1 BOESCH (Sylvia)`
A11	`02`	`1`		`@1 STURM (Brigitte)`
A11	`03`	`1`		`@1 HERING (Sascha)`
A11	`04`	`1`		`@1 SCHEIBER-MOJDEHKAR (Barbara)`
A11	`05`	`1`		`@1 STEINKELLNER (Hannes)`
A11	`06`	`1`		`@1 GOLDENBERG (Hans)`
A11	`07`	`1`		`@1 POEWE (Werner)`
A14	`01`			`@1 Department of Neurology, Innsbruck Medical University @2 Innsbruck @3 AUT @Z 1 aut. @Z 3 aut. @Z 7 aut.`
A14	`02`			`@1 Department of Medical Chemistry, Medical University of Vienna @2 Vienna @3 AUT @Z 2 aut. @Z 4 aut. @Z 5 aut. @Z 6 aut.`
A20				`@1 1940-1944`
A21				`@1 2008`
A23	`01`			`@0 ENG`
A43	`01`			`@1 INIST @2 20953 @5 354000184468160250`
A44				`@0 0000 @1 © 2008 INIST-CNRS. All rights reserved.`
A45				`@0 21 ref.`
A47	`01`	`1`		`@0 08-0522203`
A60				`@1 P @3 CC`
A61				`@0 A`
A64	`01`	`1`		`@0 Movement disorders`
A66	`01`			`@0 USA`
C01	`01`		`ENG`	@0 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.
C02	`01`	`X`		`@0 002B17`
C02	`02`	`X`		`@0 002B17G`
C03	`01`	`X`	`FRE`	`@0 Hérédodégénérescence spinocérébelleuse de Friedreich @5 01`
C03	`01`	`X`	`ENG`	`@0 Friedreich ataxia @5 01`
C03	`01`	`X`	`SPA`	`@0 Heredodegeneración espinocerebelosa Friedreich @5 01`
C03	`02`	`X`	`FRE`	`@0 Pathologie du système nerveux @5 02`
C03	`02`	`X`	`ENG`	`@0 Nervous system diseases @5 02`
C03	`02`	`X`	`SPA`	`@0 Sistema nervioso patología @5 02`
C03	`03`	`X`	`FRE`	`@0 Homme @5 09`
C03	`03`	`X`	`ENG`	`@0 Human @5 09`
C03	`03`	`X`	`SPA`	`@0 Hombre @5 09`
C03	`04`	`X`	`FRE`	`@0 Erythropoïétine @5 10`
C03	`04`	`X`	`ENG`	`@0 Erythropoietin @5 10`
C03	`04`	`X`	`SPA`	`@0 Eritropoyetina @5 10`
C03	`05`	`X`	`FRE`	`@0 Essai clinique @5 11`
C03	`05`	`X`	`ENG`	`@0 Clinical trial @5 11`
C03	`05`	`X`	`SPA`	`@0 Ensayo clínico @5 11`
C03	`06`	`X`	`FRE`	`@0 Stress oxydatif @5 12`
C03	`06`	`X`	`ENG`	`@0 Oxidative stress @5 12`
C03	`06`	`X`	`SPA`	`@0 Estrés oxidativo @5 12`
C07	`01`	`X`	`FRE`	`@0 Pathologie de l'encéphale @5 37`
C07	`01`	`X`	`ENG`	`@0 Cerebral disorder @5 37`
C07	`01`	`X`	`SPA`	`@0 Encéfalo patología @5 37`
C07	`02`	`X`	`FRE`	`@0 Maladie dégénérative @5 38`
C07	`02`	`X`	`ENG`	`@0 Degenerative disease @5 38`
C07	`02`	`X`	`SPA`	`@0 Enfermedad degenerativa @5 38`
C07	`03`	`X`	`FRE`	`@0 Maladie héréditaire @5 39`
C07	`03`	`X`	`ENG`	`@0 Genetic disease @5 39`
C07	`03`	`X`	`SPA`	`@0 Enfermedad hereditaria @5 39`
C07	`04`	`X`	`FRE`	`@0 Pathologie de la moelle épinière @5 40`
C07	`04`	`X`	`ENG`	`@0 Spinal cord disease @5 40`
C07	`04`	`X`	`SPA`	`@0 Médula espinal patología @5 40`
C07	`05`	`X`	`FRE`	`@0 Pathologie du système nerveux central @5 41`
C07	`05`	`X`	`ENG`	`@0 Central nervous system disease @5 41`
C07	`05`	`X`	`SPA`	`@0 Sistema nervosio central patología @5 41`
N21				`@1 343`
N44	`01`			`@1 OTO`
N82				`@1 OTO`

Format Inist (serveur)

NO :	PASCAL 08-0522203 INIST
ET :	Neurological Effects of Recombinant Human Erythropoietin in Friedreich's Ataxia : A Clinical Pilot Trial
AU :	BOESCH (Sylvia); STURM (Brigitte); HERING (Sascha); SCHEIBER-MOJDEHKAR (Barbara); STEINKELLNER (Hannes); GOLDENBERG (Hans); POEWE (Werner)
AF :	Department of Neurology, Innsbruck Medical University/Innsbruck/Autriche (1 aut., 3 aut., 7 aut.); Department of Medical Chemistry, Medical University of Vienna/Vienna/Autriche (2 aut., 4 aut., 5 aut., 6 aut.)
DT :	Publication en série; Courte communication, note brève; Niveau analytique
SO :	Movement disorders; ISSN 0885-3185; Etats-Unis; Da. 2008; Vol. 23; No. 13; Pp. 1940-1944; Bibl. 21 ref.
LA :	Anglais
EA :	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.
CC :	002B17; 002B17G
FD :	Hérédodégénérescence spinocérébelleuse de Friedreich; Pathologie du système nerveux; Homme; Erythropoïétine; Essai clinique; Stress oxydatif
FG :	Pathologie de l'encéphale; Maladie dégénérative; Maladie héréditaire; Pathologie de la moelle épinière; Pathologie du système nerveux central
ED :	Friedreich ataxia; Nervous system diseases; Human; Erythropoietin; Clinical trial; Oxidative stress
EG :	Cerebral disorder; Degenerative disease; Genetic disease; Spinal cord disease; Central nervous system disease
SD :	Heredodegeneración espinocerebelosa Friedreich; Sistema nervioso patología; Hombre; Eritropoyetina; Ensayo clínico; Estrés oxidativo
LO :	INIST-20953.354000184468160250
ID :	08-0522203

Links to Exploration step

Pascal:08-0522203

Le document en format XML

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<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>
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<server><NO>PASCAL 08-0522203 INIST</NO>
<ET>Neurological Effects of Recombinant Human Erythropoietin in Friedreich's Ataxia : A Clinical Pilot Trial</ET>
<AU>BOESCH (Sylvia); STURM (Brigitte); HERING (Sascha); SCHEIBER-MOJDEHKAR (Barbara); STEINKELLNER (Hannes); GOLDENBERG (Hans); POEWE (Werner)</AU>
<AF>Department of Neurology, Innsbruck Medical University/Innsbruck/Autriche (1 aut., 3 aut., 7 aut.); Department of Medical Chemistry, Medical University of Vienna/Vienna/Autriche (2 aut., 4 aut., 5 aut., 6 aut.)</AF>
<DT>Publication en série; Courte communication, note brève; Niveau analytique</DT>
<SO>Movement disorders; ISSN 0885-3185; Etats-Unis; Da. 2008; Vol. 23; No. 13; Pp. 1940-1944; Bibl. 21 ref.</SO>
<LA>Anglais</LA>
<EA>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.</EA>
<CC>002B17; 002B17G</CC>
<FD>Hérédodégénérescence spinocérébelleuse de Friedreich; Pathologie du système nerveux; Homme; Erythropoïétine; Essai clinique; Stress oxydatif</FD>
<FG>Pathologie de l'encéphale; Maladie dégénérative; Maladie héréditaire; Pathologie de la moelle épinière; Pathologie du système nerveux central</FG>
<ED>Friedreich ataxia; Nervous system diseases; Human; Erythropoietin; Clinical trial; Oxidative stress</ED>
<EG>Cerebral disorder; Degenerative disease; Genetic disease; Spinal cord disease; Central nervous system disease</EG>
<SD>Heredodegeneración espinocerebelosa Friedreich; Sistema nervioso patología; Hombre; Eritropoyetina; Ensayo clínico; Estrés oxidativo</SD>
<LO>INIST-20953.354000184468160250</LO>
<ID>08-0522203</ID>
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	Movement Disorders (revue)
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Movement Disorders (revue)

Neurological Effects of Recombinant Human Erythropoietin in Friedreich's Ataxia : A Clinical Pilot Trial

Neurological Effects of Recombinant Human Erythropoietin in Friedreich's Ataxia : A Clinical Pilot Trial

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