AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION (HSCT) FOR NEUROLOGICAL DISORDERS: A SYSTEMATIC REVIEW & META-ANALYSIS OF CLINICAL OUTCOMES AND COMPLICATIONS



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Abstract

Introduction: Neurological disorders, including MS and pediatric neurodegenerative diseases, pose a significant global health burden with limited therapeutic options. Autologous HSCT has emerged as a promising intervention to halt disease progression, reduce disability, and modulate immune responses in the central nervous system. This systematic review and meta-analysis evaluate the clinical outcomes and safety of autologous HSCT in patients with neurological disorders.

Methods: Following PRISMA guidelines, we searched PubMed, Embase, Cochrane Library, and Web of Science for studies on autologous HSCT in neurological disorders. Included studies reported clinical outcomes (e.g., Expanded Disability Status Scale [EDSS] changes, progression-free survival [PFS]) and complications. Data were synthesized using random-effects meta-analyses to calculate standardized mean differences (SMD) for EDSS changes, pooled proportions for PFS and relapse-free survival, and treatment-related mortality (TRM) rates. Heterogeneity was assessed with I² statistics, and predictors of outcomes were explored via meta-regression and subgroup analyses.

Results: Fifteen studies (n=1,378 patients) were included, predominantly focusing on MS (relapsing-remitting, progressive, and aggressive subtypes). Autologous HSCT significantly reduced disability, with a pooled SMD in EDSS of -1.02 (95% CI: -1.42, -0.62; p < 0.01; I² = 88.9%). PFS was 73% (95% CI: 0.61–0.84; I² = 0%), and relapse-free survival in relapsing-remitting MS was 82% (95% CI: 0.70–0.92; I² = 5%). TRM was low at 2% (95% CI: 0.00–0.04; I² = 38.9%), with common adverse events including febrile neutropenia and infections. Younger age, shorter disease duration, and relapsing-remitting MS subtype predicted better outcomes (p < 0.05). Conditioning regimen influenced safety, with BEAM-based protocols showing lower TRM (p = 0.0049).

Conclusion: Autologous HSCT demonstrates significant efficacy in reducing disability and preventing disease progression in neurological disorders, particularly MS, with a favorable safety profile. However, high heterogeneity and limited controlled trials highlight the need for larger, randomized studies to confirm comparative efficacy against standard therapies and optimize patient selection and treatment protocols.

About the authors

Seema Awasthi

Teerthanker Mahaveer Medical college and research centre

Email: seemaawasthi285@gmail.com
India

Vinod Kumar Singh

Teerthanker Mahaveer Medical college and research centre

Email: drvinodkumarsingh85@gmail.com
India

Sanjeev Kumar Jain

Teerthanker Mahaveer Medical College and Research Centre

Email: jainsanjeevkumar77@gmail.com
ORCID iD: 0009-0003-6959-859X
India

Soniya Sharma

TMMC&RC

Author for correspondence.
Email: soniyasharma19922@gmail.com
India

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СМИ зарегистрировано Федеральной службой по надзору в сфере связи, информационных технологий и массовых коммуникаций (Роскомнадзор).
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