Stem Cell Transplants Offer Vital Hope for Rare Autoimmune Disease

Systemic sclerosis (SSc) is a rare and challenging autoimmune disorder characterised by fibrosis—a progressive thickening and scarring of tissue that can severely damage internal organs, most notably the lungs. For patients battling rapidly progressive diffuse cutaneous SSc combined with interstitial lung disease (ILD), standard therapies often struggle to halt the decline. However, a robust review of medical evidence points to a transformative solution: autologous haematopoietic stem cell transplantation (AHSCT).

This procedure involves harvesting a patient’s own stem cells before using chemotherapy to eliminate their malfunctioning immune system. The stored cells are then returned to the body to rebuild a healthier immune defence. Randomised controlled trials have highlighted AHSCT as one of the most effective disease-modifying strategies available, offering a proven survival benefit compared to traditional treatments. By facilitating immune reconstitution, the therapy aims to stop the body from attacking its own tissues.

Despite its success, the medical community continues to refine the approach. Current scientific discussions focus on optimising the 'conditioning intensity'—the strength of the chemotherapy used prior to transplant—and the precise selection of CD34+ cells. As researchers analyse real-world data and long-term efficacy, AHSCT stands out as a critical lifeline for patients facing the most aggressive forms of this connective tissue disease.