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Background <p>Neurodegenerative diseases present an escalating global health burden with limited disease-modifying therapies. Erythropoietin (EPO), well-known for its hematological effects, has demonstrated neuroprotective properties in preclinical studies, but its clinical utility in neurodegeneration remains unclear. Therefore, this study aims to systematically appraise clinical and translational evidence on the efficacy and safety of EPO and its analogues in AD, PD, and dementia syndromes.</p> Methods <p>A systematic review was conducted following JBI and PRISMA guidelines. PubMed and Scopus were searched up to October 2024, for original studies evaluating EPO interventions in neurodegenerative diseases. Eligibility, data extraction, and quality assessment were performed independently by two reviewers using standardized criteria.</p> Results <p>Eleven studies were included, covering randomized controlled trials, cohort, cross-sectional, and clinical studies across diverse populations. In AD, randomized trials of intranasal NeuroEPO reported significant cognitive improvement versus placebo, supported by mechanistic evidence of EPO receptor upregulation in affected brain regions. In PD, Cuban clinical trials found cognitive and motor benefits with EPO-based therapies and favorable safety profiles. Observational data suggested EPO may reduce risk for vascular and unspecified dementia, particularly in high-risk cohorts. However, substantial heterogeneity and small sample sizes limit the generalizability of findings.</p> Conclusions <p>EPO and its analogues appear safe and may offer symptomatic and neuroprotective benefits in AD and PD. Larger, multicenter trials are required to clarify clinical efficacy, biomarker utility, and long-term safety in neurodegenerative disease management.</p>

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Therapeutic and biomarker potential of erythropoietin in neurodegenerative diseases: a systematic review

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年1月27日

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  • Maryam Babazadeh,
  • Sarvin Sanaie,
  • Sama Rahnemayan

摘要

Background

Neurodegenerative diseases present an escalating global health burden with limited disease-modifying therapies. Erythropoietin (EPO), well-known for its hematological effects, has demonstrated neuroprotective properties in preclinical studies, but its clinical utility in neurodegeneration remains unclear. Therefore, this study aims to systematically appraise clinical and translational evidence on the efficacy and safety of EPO and its analogues in AD, PD, and dementia syndromes.

Methods

A systematic review was conducted following JBI and PRISMA guidelines. PubMed and Scopus were searched up to October 2024, for original studies evaluating EPO interventions in neurodegenerative diseases. Eligibility, data extraction, and quality assessment were performed independently by two reviewers using standardized criteria.

Results

Eleven studies were included, covering randomized controlled trials, cohort, cross-sectional, and clinical studies across diverse populations. In AD, randomized trials of intranasal NeuroEPO reported significant cognitive improvement versus placebo, supported by mechanistic evidence of EPO receptor upregulation in affected brain regions. In PD, Cuban clinical trials found cognitive and motor benefits with EPO-based therapies and favorable safety profiles. Observational data suggested EPO may reduce risk for vascular and unspecified dementia, particularly in high-risk cohorts. However, substantial heterogeneity and small sample sizes limit the generalizability of findings.

Conclusions

EPO and its analogues appear safe and may offer symptomatic and neuroprotective benefits in AD and PD. Larger, multicenter trials are required to clarify clinical efficacy, biomarker utility, and long-term safety in neurodegenerative disease management.