Autoimmune diseases (AID) include a wide range of chronic illnesses and are of major importance in modern medicine.
Usually, AID affects young adults, and causes a gradual decrease in their functioning and quality of life (QoL).
High-dose immunosuppressive therapy (HDIT) with AHSCT was proposed as a new and promising therapy for AID patients.
The rationale for this method is that ablation of aberrant immune system followed by reconstitution of the new immune system from hematopoietic stem cells may alter the characteristics of the T-cell responses and other immunological properties. This may improve the clinical course of AID. The first HDIT+ AHSCT in AID patient (multiple sclerosis, MS) was performed in Europe in 1997. In Russia the first transplantation in MS was performed by A. Novik and colleagues in 1999. By now centers in Europe, North and South America, Russia, China, Israel and Australia have the experience of use of HDIT+AHSCT for AID treatment. Prof A. Novik proposed modem concept of HSCT in AID. It includes 3 strategies of AHST: "Early HSCT" is performed soon after diagnosis at early disease stage. "Conventional HSCT" is performed in patients with secondary refractory disease. "Salvage/late HSCT" is an option in case of high disease activity and rapid deterioration of functioning.
Also, in this report we present the long-term outcomes of a prospective single center study with the analysis of the safety and efficacy of HDIT+AHSCT with reduced-intensity conditioning regimen in 502 MS patients (mean age - 39 years old; male/female —178/324; mean EDSS=4.0; 257 relapsing/remitting MS, 161 - secondary progressive MS and 84 - primary progressive MS). Reduced-intensity BM/BEAM-like or Cyclophosphamide based conditioning was used. Efficacy was evaluated based on clinical and quality of life outcomes. The mobilization and transplantation procedures were well tolerated. Clinical response in 6 and 12 months after HSCT was observed in almost all patients. At long term follow-up stabilization and improvement had 95% of patients. Event-free and progressive-free survival was better in younger patients, in those with less EDSS score, in relapsing-remitting MS, in those with less disease duration. Also, AHSCT was accompanied by a significant improvement in patient's QoL: statistically significant changes were registered for the majority of QoL scales a year post-transplant. Improved QoL was preserved during the entire period of follow-up. Thus, the risk/benefit ratio of HDIT+AHSCT in our population of MS patients is very favorable. QoL assessment is important end-point for comprehensive outcomes assessment in MS patients undergoing HSCT.
Further studies are needed for better assessment of treatment outcomes in AID patients.