Brazilian experience with two conditioning regimens in patients with multiple sclerosis: BEAM/horse ATG and CY/rabbit ATG

• Published in: Bone marrow transplantation (Basingstoke) Vol. 45; no. 2; pp. 239 - 248
• Main Authors: Hamerschlak, N, Rodrigues, M, Moraes, D A, Oliveira, M C, Stracieri, A B P L, Pieroni, F, Barros, G M N, Madeira, M I A, Simões, B P, Barreira, A A, Brum, D G, Ribeiro, A A F, Kutner, J M, Tylberi, C P, Porto, P P, Santana, C L, Neto, J Z, Barros, J C, Paes, A T, Burt, R K, Oliveira, E A, Mastropietro, A P, Santos, A C, Voltarelli, J C
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 01.02.2010; Nature Publishing Group
• Subjects: Adult; Alveoli; Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy; Animals; Antilymphocyte Serum - administration & dosage; Antineoplastic Combined Chemotherapy Protocols - administration & dosage; Autografts; Biological and medical sciences; Bone marrow, stem cells transplantation. Graft versus host reaction; Care and treatment; Carmustine - administration & dosage; Cell Biology; Conditioning; Cytarabine - administration & dosage; Development and progression; Etoposide - administration & dosage; Female; Graft Rejection - prevention & control; Hematology; Hematopoietic Stem Cell Mobilization; Hematopoietic Stem Cell Transplantation - methods; Hematopoietic stem cells; Hemorrhage; Horses; Humans; Immunosuppressive agents; Internal Medicine; Male; Medical sciences; Medicine; Medicine & Public Health; Melphalan - administration & dosage; Middle Aged; Multiple sclerosis; Multiple Sclerosis - therapy; Multiple sclerosis and variants. Guillain barré syndrome and other inflammatory polyneuropathies. Leukoencephalitis; Neurology; original-article; Public Health; Quality of Life; Rabbits; Sepsis; Stem cell transplantation; Stem Cells; Toxicity; Transfusions. Complications. Transfusion reactions. Cell and gene therapy; Transplantation; Transplantation Conditioning - methods; South America; Brazil; America; hematopoietic SCT; hematopoietic stem cell mobilization; multiple sclerosis; CY; BEAM regimen; Multiple sclerosis; Stem cell; Hematopoietic cell; Rabbit; Inflammatory disease; Horse; Conditioning; Antithymocyte globulin; Ungulata; Hematopoietic stem cell transplantation; Human; Nervous system diseases; Hematology; Lagomorpha; Mobilization; Vertebrata; Mammalia; Animal; Central nervous system disease; Perissodactyla; Therapeutic protocol
• ISSN: 0268-3369; 1476-5365; 1476-5365
• DOI: 10.1038/bmt.2009.127

Studies have shown that autologous hematopoietic SCT (HSCT) can be used as an intensive immunosuppressive therapy to treat refractory patients and to prevent the progression of multiple sclerosis (MS). This is a prospective multicentric Brazilian MS trial comparing two conditioning regimens: BEAM/horse ATG and CY/rabbit ATG. Most (80.4%) of the 41 subjects in the study had the secondary progressive MS subtype and the mean age was 42 years. The baseline EDSS score in 58.5% of the subjects was 6.5 and 78% had a score of 6.0 or higher, respectively. The complication rate during the intra-transplantation period was 56% for all patients: 71.4% of the patients in the BEAM/hATG group and 40% in the CY/rATG group ( P =0.04). Three subjects (7.5%) died of cardiac toxicity, sepsis and alveolar hemorrhage, all of them in the BEAM/ATG group. EFS was 58.54% for all patients: 47% in the BEAM/hATG group and 70% in the CY/rATG group ( P =0.288). In conclusion, the CY/rATG regimen seems to be associated with similar outcome results, but presented less toxicity when compared with the BEAM/hATG regimen. Long-term follow-up would be required to fully assess the differences in therapeutic effectiveness between the two regimens.