Pioneering Research on AHSCT/HSCT for Multiple Sclerosis and Autoimmune Diseases

pioneering research

Compilation of Published Research on HSCT in Global Medical Journals

Autologous Hematopoietic Stem Cell Transplantation (AHSCT), commonly referred to as HSCT, is rapidly reshaping the conversation around treatment for Multiple Sclerosis (MS) and other stubborn autoimmune conditions. Unlike conventional therapies that only manage symptoms or slow progression, HSCT takes a bold step—it essentially “resets” the immune system. For patients who have exhausted traditional options, this offers something rare: genuine hope.

In the last decade, clinical trials from leading research canters across the globe have reported striking outcomes—fewer relapses, improved day-to-day function, and, for some, years of sustained remission.

We have compiled below a curated list of key research papers and clinical trials based on recent searches from PubMed and ClinicalTrials.gov. This is not an exhaustive list (as there are hundreds of publications), but it focuses on prominent, recent (up to 2025), and influential studies. For full access, many PubMed links lead to abstracts; full texts may require institutional access or purchase.

Broad Conclusions from Research and Clinical Trials on HSCT/AHSCT for Multiple Sclerosis and other Auto immune conditions.

Hematopoietic Stem Cell Transplantation (HSCT), particularly its autologous form (AHSCT), represents a high-intensity, immune-reprogramming therapy for multiple sclerosis (MS). In AHSCT, a patient’s own hematopoietic stem cells are harvested, followed by myeloablative or non-myeloablative chemotherapy (e.g., cyclophosphamide-based regimens) to deplete autoreactive immune cells, and then reinfusion of the stem cells to rebuild a tolerant immune system.

This “reset” aims to halt inflammatory activity in aggressive relapsing-remitting MS (RRMS), where disease-modifying therapies (DMTs) like ocrelizumab or natalizumab fail. Since the first MS AHSCT case in 1995, over 3,000 procedures have been performed globally, with accumulating evidence from meta-analyses, cohort studies, and randomized trials supporting its role as a one-time, potentially curative option for select patients.

Efficacy of HSCT proven : Halting Disease Activity and Improving Disability

AHSCT offers transformative potential for aggressive RRMS, with meta-analytic evidence of durable remissions outperforming DMTs, at the cost of manageable short-term risks. Long-term data affirm its role in immune reset, but progressive MS remains challenging. As of 2025, EBMT-endorsed protocols are expanding access, with ongoing trials addressing equity and optimization. Recent meta-analyses and reviews consistently demonstrate AHSCT’s superior efficacy over standard DMTs in highly active RRMS, with high rates of

Long-Term Outcomes: Durability and Quality of Life

Follow-up data (5–15 years) from registries like EBMT and ASTCT highlight durable remissions, though ~20–30% experience late relapses or progression.

Survival and Remission Rates:

  • Progression-free survival: 80% at 10 years in early RRMS; drops to 50–60% in progressive subtypes.
  • Danish cohort (2023, n=81 aggressive RRMS): 92% relapse-free at 5 years; EDSS stable/improved in 75%.
  • UK experience (2025, n=364): 70% NEDA at 7 years; factors for better outcomes: age <35, disease duration <5 years, pre-AHSCT EDSS <4.

Younger patients with shorter disease duration and fewer prior DMTs fare best, with some achieving 15+ years of remission.

Safety and Risks: Balancing Intensity with Modern Protocols HSCT’s risks have declined with refined conditioning (e.g., low-dose cyclophosphamide + ATG) and supportive care, but it’s not risk-free. Treatment-related mortality (TRM) is now <1% in experienced centers. Risk Mitigation: Prior rituximab exposure protects against post-AHSCT inflammation. Neuropsychological reviews (2024) show cognitive stability/improvement in 80%, with minimal decline.

Title

Publication Date

Link

Key Insights

Autologous Hematopoietic Stem-Cell Transplantation in Multiple Sclerosis: 2022 Update on the European Society for Blood and Marrow Transplantation Clinical Practice Recommendations

2022

PubMed Link

Updates on safety improvements due to technical advances and patient selection in MS treatment.

Autologous Hematopoietic Stem Cell Transplant in Multiple Sclerosis

Feb 2021

PubMed Link

Supports AHSCT as a viable option for relapsing MS per the National Multiple Sclerosis Society.

Autologous Hematopoietic Stem Cell Transplantation (AHSCT) in Multiple Sclerosis: An Updated Review of Current Literature and Future Directions

Mar 2021

PubMed Link

Positions AHSCT as a novel approach for DMT-resistant MS patients.

Ongoing Randomized Clinical Trials on HSCT in Multiple Sclerosis: A Scoping Review

2024

PubMed Link

Reviews emerging AHSCT for highly active relapsing-remitting MS.

Autologous Haematopoietic Stem Cell Transplantation for Multiple Sclerosis: A Cochrane Review

Jun 2025

PubMed Link

Comprehensive review since 1995, assessing long-term outcomes.

Second Autologous Hematopoietic Stem Cell Transplantation in Multiple Sclerosis: A Case Report

Jan 2024

PubMed Link

Explores repeat AHSCT for immunosuppressive therapy in MS.

Sustained Immunotolerance in Multiple Sclerosis after Stem Cell Transplantation

Feb 2022

PubMed Link

Discusses recalibration of pro-inflammatory pathways post-AHSCT.

Comparative Effectiveness of Autologous Hematopoietic Stem Cell Transplant vs Disease-Modifying Therapy in Patients with Multiple Sclerosis

Jul 2023

PubMed Link

Compares AHSCT to standard therapies for highly active MS.

Autologous Hematopoietic Stem Cell Transplantation to Treat Multiple Sclerosis: The Path to Widespread Adoption

2023

PubMed Link

Evidence for durable efficacy in relapsing MS.

Autologous Haematopoietic Stem Cell Transplantation for Treatment of Multiple Sclerosis: A Cochrane Review (Update)

Jan 2025

PubMed Link

Focuses on relapsing MS with aggressive disease.

 Research Papers on AHSCT/HSCT for Other Autoimmune Diseases

These cover a range of conditions like systemic sclerosis, lupus, and Crohn’s disease, excluding MS.

Title

Publication Date

Link

Key Insight

Haematopoietic Stem Cell Transplantation for Autoimmune Diseases: Potential Risks and Benefits

2017

PubMed Link

Reviews immunological mechanisms and regimens for various autoimmune diseases.

Autologous Haematopoiesis Stem Cell Transplantation (AHSCT) for Refractory Autoimmune Diseases in Children and Adolescents: A Single-Center Experience

Jul 2024

PubMed Link

Demonstrates safety and efficacy in pediatric refractory cases.

Hematopoietic Stem Cell Transplantation and Cellular Therapies for Autoimmune Diseases: Overview and Future Considerations

May 2022

PubMed Link

Discusses EBMT perspectives on HSCT for autoimmune conditions.

Infectious Complications After Autologous Hematopoietic Stem Cell Transplantation in Autoimmune Diseases: A Systematic Review

Mar 2025

PubMed Link

Analyzes infection risks post-AHSCT in autoimmune patients.

Autoimmune Diseases After Allogeneic Stem Cell Transplantation: A Clinician’s Guide and Future Outlook

May 2022

PubMed Link

Guide on post-HSCT autoimmune complications.

T-Cell Recovery After Autologous Hematopoietic Stem Cell Transplantation in Autoimmune Diseases

2025

PubMed Link

Covers over 3,000 AHSCT procedures for severe autoimmune diseases.

Autologous Hematopoietic Stem Cell Transplantation with Immunosuppressive Treatment in Autoimmune Diseases: A Retrospective Survey

2020

PubMed Link

Retrospective data on refractory autoimmune patients.

Haematopoietic Stem Cell Transplantation for Severe Autoimmune Diseases in Children: A Review of Current Literature, Registry Activity and Future Directions

2023

PubMed Link

Focuses on pediatric applications and EBMT registry data.

Hematopoietic Stem Cell Therapy for Autoimmune Diseases: Clinical Experience and Mechanisms

Jun 2018

PubMed Link

Explores clinical outcomes and underlying mechanisms.

A Position Statement from the EBMT Autoimmune Diseases Working Party on Haematopoietic Stem Cell Transplantation in Autoimmune Diseases

2019

PubMed Link

Weighs risks vs. benefits compared to standard treatments.

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Fortis.Hospital_India

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