Hematopoietic stem cell transplantation for systemic sclerosis: Brazilian experience

Autor: Luiz Fernando Weffort, Djúlio César Zanin-Silva, Giuliana Martinelli Dotoli, Maria Carolina Oliveira, Vanessa Cristina Leopoldo, Álvaro Henrique-Neto, Talita Graminha Zucoloto, Maynara Santana Gonçalves, Juliana Bernardes Elias Dias, Daniela A. Moraes, Marília de Fátima Cirioli de Oliveira, Marianna Yumi Kawashima Vasconcelos
Jazyk: angličtina
Rok vydání: 2021
Předmět:
Adult
Male
lcsh:Immunologic diseases. Allergy
medicine.medical_specialty
Vital capacity
lcsh:Diseases of the musculoskeletal system
medicine.medical_treatment
Thrombotic thrombocytopenic purpura
Hematopoietic stem cell transplantation
Stem cells
Gastroenterology
Progenitor cells
Pulmonary function testing
03 medical and health sciences
Young Adult
0302 clinical medicine
Rheumatology
Internal medicine
Cause of Death
medicine
Humans
Longitudinal Studies
Progenitor cell
Retrospective Studies
030203 arthritis & rheumatology
Transplantation
Lung
Scleroderma
Systemic
business.industry
Interstitial lung disease
Middle Aged
medicine.disease
DOENÇAS AUTOIMUNES
medicine.anatomical_structure
Treatment Outcome
030220 oncology & carcinogenesis
Systemic sclerosis
Female
lcsh:RC925-935
business
Lung Diseases
Interstitial
lcsh:RC581-607
Brazil
Zdroj: Advances in Rheumatology, Vol 61, Iss 1, Pp 1-10 (2021)
Repositório Institucional da USP (Biblioteca Digital da Produção Intelectual)
Universidade de São Paulo (USP)
instacron:USP
ISSN: 2523-3106
Popis: BackgroundIn the past 20 years, hematopoietic stem cell transplantation (HSCT) has been investigated as treatment for systemic sclerosis (SSc). The goal of HSCT is to eradicate the autoreactive immune system, which is replaced by a new immune repertoire with long-lasting regulation and tolerance to autoantigens. Here, we describe the clinical outcomes of severe and refractory SSc patients that underwent HSCT at a single Brazilian center.Patients and methodsThis is a longitudinal and retrospective study, including 70 adult SSc patients, with an established diagnosis of SSc, and who underwent autologous HSCT from 2009 to 2016. The procedure included harvesting and cryopreservation of autologous hematopoietic progenitor cells, followed by administration of an immunoablative regimen and subsequent infusion of the previously collected cells. Patients were evaluated immediately before transplantation, at 6 months and then yearly until at least 5-years of post-transplantation follow-up. At each evaluation time point, patients underwent clinical examination, including modified Rodnan’s skin score (mRSS) assessment, echocardiography, high-resolution computed tomography of the lungs and pulmonary function.ResultsMedian (range) age was 35.9 (19–59), with 57 (81.4%) female and median (range) non-Raynaud’s disease duration of 2 (1–7) years. Before transplantation, 96% of the patients had diffuse skin involvement, 84.2%, interstitial lung disease and 67%, positive anti-topoisomerase I antibodies. Skin involvement significantly improved, with a decline in mRSS at all post-transplantation time points until at least 5-years of follow-up. When patients with pre-HSCT interstitial lung disease were analyzed, there was an improvement in pulmonary function (forced vital capacity and diffusing capacity of lung for carbon monoxide) over the 5-year follow-up. Overall survival was 81% and progression-free survival was 70.5% at 8-years after HSCT. Three patients died due to transplant-related toxicity, 9 patients died over follow-up due to disease reactivation and one patient died due to thrombotic thrombocytopenic purpura.ConclusionsAutologous hematopoietic progenitor cell transplantation improves skin and interstitial lung involvement. These results are in line with the international experience and support HSCT as a viable therapeutic alternative for patients with severe and progressive systemic sclerosis.
Databáze: OpenAIRE
Externí odkaz: