High-Dose Immunosuppressive Therapy with Autologous Hematopoietic Stem Cell Transplantation

Autologous Hematopoietic Stem Cell Transplantation (AHSCT) is a treatment that uses a patient’s own hematopoietic stem cells, which are typically collected from the peripheral blood. The stem cells are collected and cryopreserved in advance, then reinfused after the patient has received high-dose chemotherapy or immunosuppressive treatment. By resetting the immune system, AHSCT can halt disease progression in many patients and may help preserve their quality of life.

AHSCT is a well-established treatment that has been used in clinical practice for more than three decades. It is clinically approved for the treatment of various hematological malignancies, including lymphoma and leukemia. The development of hematopoietic stem cell transplantation has significantly improved treatment outcomes for patients with blood cancers, providing many patients with a chance of long-term remission and recovery where treatment options were previously limited. Research has also shown that autoimmune diseases are associated with abnormalities in the regulation and function of the immune system.

Following AHSCT, patients with multiple sclerosis generally do not require ongoing maintenance disease-modifying therapy. AHSCT should not be confused with so-called “cell therapy” approaches that have become increasingly popular in Russia. Unlike these approaches, AHSCT is a well-established medical procedure with a defined treatment protocol and clinical evidence supporting its use. Clinical studies have shown that the transplantation procedure is generally well tolerated by patients. The key findings of the study are presented below.

The purpose of lymphoablative conditioning is to substantially reduce the population of autoreactive immune cells that may contribute to the development and progression of autoimmune diseases. The chemotherapy used for conditioning is administered at lower doses than those typically used in established high-dose protocols for patients with hematological malignancies such as lymphoma or leukemia. The use of less intensive conditioning regimens is based on the understanding that AHSCT may act not only through immunosuppression but also by promoting a profound and sustained modulation of the immune system. Moderate-intensity, less toxic conditioning regimens may provide durable, long-term disease remission comparable to that achieved with more intensive approaches, while potentially reducing treatment-related toxicity and transplant-related mortality.

We suggest, that the best candidates for transplantation are relatively young patients with active inflammatory lesions of relatively short duration and rapidly progressive disease, but still low disability scores, resistant to conventional therapy. Cumulative incidence of disease progression was quite low both for Relapsing-Remitting Multiple Sclerosis (RRMS) and Progressive Multiple Sclerosis (PrMS). It was higher in patients with progressive course of the disease than in those with relapsing-remitting MS: 21.3% vs 13.2% For long-term follow-up (median 48.9 months), in the group with RRMS event-free survival rate was 83.3% and in the group with progressive course – 75.5%.