Hematopoietic Stem Cell Transplantation in Rheumatic Diseases

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.