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Updated: Mar 14, 2026

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Hematopoietic Stem Cell Transplantation in Rheumatic Diseases.

Nandana Suresh1, D R Spoorthy Raj2, Harshwardhan Patil1

  • 1Doctor of Pharmacy, Department of Pharmacy Practice, JSS College of Pharmacy, Mysore, India.

International Journal of Rheumatic Diseases
|March 13, 2026
PubMed
Summary

Hematopoietic stem cell transplantation (HSCT) offers a promising treatment for severe autoimmune rheumatic diseases (AIRD) when other therapies fail. This approach can lead to long-term remission by regenerating a self-tolerant immune system, though careful patient selection is crucial.

Keywords:
autoimmune diseaseshematopoietic stem cell transplantationjuvenile idiopathic arthritisrheumatic diseasessystemic lupus erythematosussystemic sclerosistreatment outcome

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Area of Science:

  • Immunology
  • Rheumatology
  • Transplantation Medicine

Background:

  • Hematopoietic stem cell transplantation (HSCT) is an emerging therapy for severe autoimmune rheumatic diseases (AIRD) unresponsive to conventional treatments.
  • This review examines HSCT's role in systemic sclerosis (SSc), systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), deficiency of adenosine deaminase 2 (DADA2), ANCA-associated vasculitis, Takayasu arteritis (TA), and juvenile idiopathic arthritis (JIA).

Purpose of the Study:

  • To evaluate the efficacy, immunological mechanisms, patient selection, conditioning regimens, and outcomes of HSCT for specific AIRD.
  • Focus on systemic sclerosis-associated interstitial lung disease (SSc-ILD) outcomes.

Main Methods:

  • Comprehensive literature review of clinical trials, observational studies, and preclinical research.
  • Analysis of pulmonary outcomes, immune reconstitution, CD34+ cell selection, and post-transplant immunosuppression.
  • Keywords: SSc, SLE, RA, DADA2, ANCA-associated vasculitis, Takayasu arteritis, JIA.

Main Results:

  • HSCT shows promise in diffuse cutaneous SSc with ILD, refractory SLE, RA, and JIA.
  • HSCT can reverse hematological, immunological, and vascular phenotypes in DADA2.
  • While effective for ANCA-associated vasculitis and TA, relapses and complications are concerns; benefits include immune system regeneration, but early mortality necessitates careful selection and reduced-toxicity conditioning.

Conclusions:

  • HSCT is a transformative option for select patients with refractory AIRD, potentially achieving long-term, drug-free remission.
  • Future research should optimize conditioning, refine patient selection, and assess long-term outcomes to maximize benefits and minimize risks.