TY - JOUR
T1 - High-dose erythropoietin in patients with progressive multiple sclerosis
T2 - A randomized, placebo-controlled, phase 2 trial
AU - Schreiber, Karen
AU - Magyari, Melinda
AU - Sellebjerg, Finn
AU - Iversen, Pernille
AU - Garde, Ellen
AU - Madsen, Camilla Gøbel
AU - Börnsen, Lars
AU - Romme Christensen, Jeppe
AU - Ratzer, Rikke
AU - Siebner, Hartwig Roman
AU - Laursen, Bjarne
AU - Soelberg Sorensen, Per
PY - 2017/4/1
Y1 - 2017/4/1
N2 - BACKGROUND: Erythropoietin (EPO) is a part of an endogenous neuroprotective system in the brain and may address pathophysiological mechanisms in progressive multiple sclerosis (MS).OBJECTIVE: To evaluate a treatment effect of EPO on progressive MS.METHODS: This was a single-center, randomized, double-blind, placebo-controlled phase 2 trial, in which 52 patients with secondary or primary progressive MS were allocated to treatment with recombinant EPO (48,000 IU) or placebo, administered intravenously 17 times during 24 weeks. Patients had an Expanded Disability Status Score (EDSS) from 4 to 6.5 and clinical progression without relapses in the 2 preceding years. The primary outcome was the change in a composite measure of maximum gait distance, hand dexterity, and cognition from baseline to 24 weeks.RESULTS: A total of 50 patients completed the study. Venesection was performed often but no thromboembolic events occurred. We found no difference in the primary outcome between the EPO and the placebo group using the intention-to-treat principle ( p = 0.22). None of the secondary outcomes, neither clinical nor magnetic resonance imaging (MRI) measures showed any significant differences.CONCLUSION: This study provides class II evidence that treatment with high-dose EPO is not an effective treatment in patients with moderately advanced progressive MS.
AB - BACKGROUND: Erythropoietin (EPO) is a part of an endogenous neuroprotective system in the brain and may address pathophysiological mechanisms in progressive multiple sclerosis (MS).OBJECTIVE: To evaluate a treatment effect of EPO on progressive MS.METHODS: This was a single-center, randomized, double-blind, placebo-controlled phase 2 trial, in which 52 patients with secondary or primary progressive MS were allocated to treatment with recombinant EPO (48,000 IU) or placebo, administered intravenously 17 times during 24 weeks. Patients had an Expanded Disability Status Score (EDSS) from 4 to 6.5 and clinical progression without relapses in the 2 preceding years. The primary outcome was the change in a composite measure of maximum gait distance, hand dexterity, and cognition from baseline to 24 weeks.RESULTS: A total of 50 patients completed the study. Venesection was performed often but no thromboembolic events occurred. We found no difference in the primary outcome between the EPO and the placebo group using the intention-to-treat principle ( p = 0.22). None of the secondary outcomes, neither clinical nor magnetic resonance imaging (MRI) measures showed any significant differences.CONCLUSION: This study provides class II evidence that treatment with high-dose EPO is not an effective treatment in patients with moderately advanced progressive MS.
KW - Adult
KW - Brain/drug effects
KW - Disability Evaluation
KW - Disease Progression
KW - Double-Blind Method
KW - Erythropoietin/administration & dosage
KW - Female
KW - Humans
KW - Magnetic Resonance Imaging/methods
KW - Male
KW - Middle Aged
KW - Multiple Sclerosis/drug therapy
KW - Treatment Outcome
U2 - 10.1177/1352458516661048
DO - 10.1177/1352458516661048
M3 - Journal article
C2 - 27481206
SN - 1352-4585
VL - 23
SP - 675
EP - 685
JO - Multiple Sclerosis Journal
JF - Multiple Sclerosis Journal
IS - 5
ER -