Stem Cell Transplantation (HSCT) in Patients with Autoimmune Disease (ADs). Results of a Single Center in Brazil.
Authors: Rodrigues Morgani , Ribeiro Andreza Alice Feitosa , Kutner Jose Mauro , Assis Reijane Alves de , Nascimento Claudia Mac Donald Bley , Scheimberg Morton Aaron , Carneiro Roberto , Tilbery Charles Peter , Junior Pedro Paulo Porto , Hamerschlak Nelson 
Published: October 2019

Abstract
Abstract 4360
Autoimmune disease (ADs) are a group of heterogeneous disease, affecting 10-12% of the population. Nowadays, more than 1300 patients worldwide have received an HSCT for ADs. Autologous HSCT trials showed that in patients with a favourable outcome, a resetting of a disregulated auto-aggressive immune system may occur, together with the ablation of auto-reactive cells. The results of Allogeneic HSCT are still unclear, due to small number and heterogeneous groups of patients and conditioning regimens. We related here the experience in the use of HSCT in ADs at the Israelita Albert Einstein Hospital during the period between 1996 to 2009. During this period 28 patients received autologous HSCT: 23 (82%) patients with multiple sclerosis (MS), 2 (7,1%) patients with Sytemic Lupus erytomatosus (SLE),, one (3,5%) with Autoimmune hemolytic anemia (AIHA), one (3,5%) Antiphospholipid sydrome (AFS). and one (3,5%) with Systemic Sclerosis. The first auto HSCT was done in 1996 in one patient (female, 54 years old) with refractory AIHA, the conditioning was cyclophpsphamide/horse antilymphocyte globulin based regime (cyclophosphamide 50mg/Kg D-5 and D-4; Fludarabine 30mg/Kg D-6 to D-3, and horse globulin 15mg/Kg on Days –5, –3, –1, +1, +2, and +3). The patient was in remission for 11 years, and was salvage with rituxmab after that. Our BMT Program for MS started in 2001. A total number of 23 patients were transplanted. The condition was BEAM/horse antilymphocyte globulin in the first 8 patient, (BCNU at a dose of 300 mg/m2 on Day –7; cytarabine at a dose of 200 mg/m2 and etoposide at a dose of 200 mg/m2 from Day –6 to Day –3,melphalan at a dose of 140 mg/m2 on Day –2; and horse antilymphocyte globulin at a dose of 15 mg/kg on Days –5, –3, –1, +1, +2, and +3, together with methylprednisolone at a dose of 2 mg/kg/day.) and the remaining 15 patients received cyclophosphamide/rabbit antilymphocyte globulin based regimen (cyclophosphamide, at a dose of 50 mg/kg of body weight, used on Days –5, –4, –3 and –2, together with rabbit antimonocyte globulin at a dose of 0.5 mg/kg on Day –6 and 1 mg/kg/day on Days –5, –4, –3 and –2 followed by methylprednisolone 2 mg/kg.). The inicial extended disability status score (EDSS) median was 6,0 (5,5-7,0); 16 (70%) were female. The mediam age was 39 (27-50) years at the time of SCT and the MS subtype “Secondary Progressive” was the most common, observed in19 (82%) patient. The period of follow up was 4 years. At the BEAM group, 7 improve their neurologic score and one became stable. At the Cyclophosphamide group, 7 remaining stable, 4 improve, and 4 worse the EDSS score. At total, 19(82%) had improve/stable of their disease disability and 4 (17%) got worse. No death was observed in both groups. The two patient with SLE refractory to previews treatment received cyclophosphamide/horse antilymphocyte globulin conditioning (the same used in AIHA patient). One (male, 38 years old) is alive 9 years after SCT in clinical remission of his disease and the second one (female, 44 years old) died due therapy related mortality. The patient with AFS (male, 52 years old) had partial “remission” and died 2 years after SCT due other cause not related to transplant. The patient with SS (female, 63 years old), also received a cyclophosphamide conditioning based regimen (cyclophosphamide 50mg/Kg D-5 and D-4; Fludarabine 30mg/Kg D-6 to D-3, and rabbit antimonocyte globulin at a dose of 0.5 mg/kg on Day –6 and 1 mg/kg/day on Days –5, –4, –3 and –2 followed by methylprednisolone 2 mg/kg) and after 2 month of follow up she has improving the dermal fibrosis, but still persisting the alteration in the lung function. In Conclusion, the Autologous HSCT had a reasonable indication in a significant subset of patients that still shows an unsatisfactory response to both conventionall and new immunomodulationg agents. Our results, although in a very small number, showed a feseable treatment in that kind of patient. Long-term follow-up would be required to fully assess in therapeutic effectiveness. We believe that the development of multicentric randomized trials, is necessary, to determine the whole use of HSCT in ADs.

Disclosures:
No relevant conflicts of interest to declare.