AB0718 Autologous hematopoietic stem -cell transplantation in severe and refractory diffused systemic sclerosis. long-term study in a single reference centre. (12th June 2018)

• Record Type: Journal Article
• Authors: Ruiz Roman, A.; Atienza Mateo, B.; Calvo del Rio, V.; Gonzalez-Gay Mantecón, M.A.; Blanco Alonso, R.
• Is Part Of: Annals of the rheumatic diseases. Volume 77(2018)Supplement 2
• Page Start: 1498
• Page End: 1499
• Publication Date: 2018-06-12
• Subjects: Rheumatism -- Periodicals; 616.723005
• DOI: 10.1136/annrheumdis-2018-eular.7007
• Languages: English
• ISSNs: 0003-4967

Abstract

Abstract : Background: Pharmacological therapy does not reverse the natural course of systemic sclerosis (SS). Autologous hematopoietic stem-cell transplantation(AHSCT) has been used in refractory autoimmune diseases, including SS. The autologous hematopoietic stem-cell transplantation is based on the ablation of the immunoreactive system with high doses of chemotherapy and a subsequent regeneration of the immune system. Objectives: Study of the efficacy of autologous hematopoietic stem-cell transplantation in a cohort of patients with SS from a referral centre. Methods: Descriptive study of patients with SS (ACR/ EULAR 2013 criteria) treated with autologous hematopoietic stem-cell transplantation in a single reference centre between 2002 and 2017. These clinical variables were reviewed (Raynaud's phenomenon, pulmonary interstitial involvement, digital ulcers, digestive alterations, presence of sclerodermic renal crisis) and activity index variables (modified Rodnan score, HAQ-DI, SHAQ-VAS). Results: Four patients were included (75% women). The median age at the time of the AHSCT was 36.5 years (range 27–51). In all cases, the initial diagnosis was diffuse cutaneous ES, refractory to corticosteroids and at least one DMARD. Prior to autologous hematopoietic stem-cells transplantation, the clinical manifestations presented were a) severe Raynaud's phenomenon (100%) with significant joint and cutaneous involvement; b) digital ulcers (50%); c) interstitial lung disease (50%) and d) sclerodermal renal crisis (25%). In 3 of the cases (75%) the antitopoisomerase antibodies were positive. The conditioning treatment for the autologous hematopoietic stem-cells transplantation was cyclophosphamide at high doses (50 mg/kg x4 days) and anti-thymocyte globulin. In 3 patients (75%) there were slight post-transplant complications (febrile neutropenia, diarrhoea) after a median follow-up of 6.5 years (range 1–15). The response to AHSCT is summarised in table 1. All patients showed values <1 in the Health Assessment Questionnaire on the Disability Index (HAQ-DI), in 75% with a modified Rodnan skin score (mRSS) lower than 7. Conclusions: Autologous hematopoietic stem-cells transplantation can be a therapeutic option in refractory and severe SS. These hopeful data must be ratified in larger studies. Disclosure of Interest: None declared