EBMT 2025, Florence, Italy скачать в хорошем качестве

EBMT 2025, Florence, Italy

PhD, MD, Dr. Denis Fedorenko speech on the 51st Annual Meeting of the EBMT in Florence, Italy. Topic: Effectiveness of non-myeloablative AHSCT in MS Patients and factors associated with better outcomes Background: AHSCT is highly effective and promising treatment option for MS. We evaluated clinical outcomes and associated factors in patients with MS undergoing non-myeloablative AHSCT. Methods: Patients with various types of MS who underwent AHSCT from 2006 to 2023 were enrolled in a single-center study. Three non-myeloablative conditioning regimens were used – BEAM-like with Rituximab (25%), Cyclophosphamide with Rituximab (62%), and Fludarabine+Cyclophosphamide with Rituximab (13%). For assessment of clinical outcomes EDSS evaluation and MRI were performed, for QoL assessment SF-36 was used at baseline and at different time points after AHSCT. EDSS decrease of ≥1.0 was considered as improvement, an increase of ≥1.0 as worsening. For comparisons Wilcoxon test and ANOVA were used. Event-free survival (EFS) in terms of relapse-free survival (RFS) and progression-free survival (PFS) were evaluated by Kaplan-Meyer method and compared by log-rank test. Univariate logistic regression was used for identification of factors associated with EDSS worsening (AHSCT failure) and three risk groups were identified – low (0-1 factor), intermediate (2-3 factors), and high risk (4-5 factors). Results: Totally, 898 patients with relapsing-remitting (RRMS, n=477, 53%), primary progressive (PPMS, n=141, 16%), and secondary progressive MS (SPMS, n=280, 31%) were enrolled. Median age – 40 yrs. (Q1; Q3 33; 48), 40% were males. Mean (SD) disease duration - 6.8 (5.9) yrs. Median baseline EDSS – 4. There were 6 cases of treatment-related mortality (TRM): RRMS - 3, SPMS - 2, PPMS - 1; Cyclophosphamide was used in all cases. There was no TRM in BEAM-like and Fludarabine-based conditioning. Toxicity profile was the best for Fludarabine-based conditioning. Median follow-up after AHSCT was 30 months (Q1; Q3 12-54). EDSS improvement for the entire group at all-time intervals after transplantation as compared with baseline was observed (p less 0.001). At 12 months after AHSCT the decrease of EDSS in RRMS was from median 3.0 to 1.5, in SPMS – from 6.0 tо 5.0, and in PPMS – from 6.0 tо 4.0. The estimated 5-years EFS probability for the entire group was 83.6% (95% CI 79.8-87.4), RFS for RRMS – 87.3% (95% CI 82.3-92.4), PFS for SPMS – 81.9% (95% CI 75-88.8), and for PPMS – 78.7% (95%CI 68.8-88.5). No differences in EFS depending on conditioning regimen were found. Factors associated with AHSCT failure were – age ≥ 40 (OR=2.34, 95% CI 1.38-3.99, p=0.002), progressive MS (OR=2.51, 95%CI 1.5-4.05, p less 0.001), baseline EDSS ≥ 4 (OR=4.73, 95%CI 2.73-8.21, p less 0.001), previous standard treatment (OR=2.31, 95%CI 1.08-4.95, p=0.031), baseline physical health less 40 scores by SF-36 (OR=1.04, 95%CI 1.01-1.06, p=0.004). In high risk group EFS was worse than in low and intermediate risk groups (low risk group – 89.7% [95%CI 79.7-99.7], intermediate risk group – 79.3% [95%CI 64.4-94.2], and high risk group – 58.2%, [95%CI 34.9-81.5]); p less 0.05. Conclusions: The results demonstrate that non-myeloablative AHSCT is effective in patients with different types of MS. The safest non-myeloablative conditioning is Fludarabine-based. The best candidates for AHSCT are patients aged less 40 years, with relapsing MS, treatment-naive, with baseline EDSS less 4, and with baseline physical health ≥40 scores. EFS is better in MS patients of low and intermediate risk than in patients of high risk. AHSCT RUSSIA [https://hsct-russia.com/] It's a complex treatment program against Autoimmune Diseases such as: Multiple Sclerosis (MS) Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Ankylosing Spondylitis (AS) Neuromyelitis Optica Spectrum Disorders (NMOSD) Rheumatoid Arthritis (RA) Nonspecific Ulcerative Colitis (UC) Stiff-person Syndrome (SPS) Systemic Sclerosis (SSc) Scleroderma Systemic Lupus Erythematosus (SLE) Psoriatic Arthritis (PA) Myasthenia Gravis (MG) Sjögren's Syndrome (SjS) Crohn's Disease How to Apply: Complete our questionnaire, describing you previous treatment and current functional status, to define whether HSCT is a meaningful and safe treatment option for you. This online form must be accompanied by the report of last MRI (Please attach only report, no MRI images are required) Link to the application for MS patients: https://hsct-russia.com/application-f... Link to the application for patients with other autoimmune diseases: https://hsct-russia.com/cidp-applicat... Your medical records will be personally reviewed by MD, PhD, Dr. Denis Fedorenko.