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Evaluation of the impact of hematopoietic stem cell transplantation on the vascular aspects of Systemic Sclerosis

Grant number: 18/20339-6
Support Opportunities:Scholarships in Brazil - Doctorate (Direct)
Effective date (Start): March 01, 2019
Effective date (End): February 09, 2020
Field of knowledge:Health Sciences - Medicine - Medical Clinics
Principal Investigator:Maria Carolina de Oliveira Rodrigues
Grantee:Marianna Yumi Kawashima Vasconcelos
Host Institution: Faculdade de Medicina de Ribeirão Preto (FMRP). Universidade de São Paulo (USP). Ribeirão Preto , SP, Brazil


Systemic Sclerosis (SSc) is a rheumatic disease that causes progressive fibrosis of the skin and may affect internal organs. It presents high morbidity and mortality, and in severe cases, where cutaneous involvement is diffuse or in which there is involvement of the lungs, heart or kidneys, mortality may reach 30 to 50% in five years, even under conventional immunosuppressive treatment. Autologous Hematopoietic Stem Cell Transplantation (AHSCT) has been investigated as an experimental therapeutic alternative, and has been shown effective in controlling the progression and even reversing some of the manifestations of the disease. Recently, clinical studies have demonstrated that AHSCT is superior to conventional treatments, providing longer survival, better control of disease progression and improvement of patient quality of life. However, despite the clinical benefit, there are still mechanistic questions about the therapeutic potential of AHSCT. Vasculopathy is an important component of Systemic Sclerosis pathogenesis, reflecting on Raynaud's phenomenon, Digital Ulcers, Pulmonary Arterial Hypertension and Vascular Nephropathy. These manifestations correlate with the severity of the disease, poor quality of life and lower survival. The present project aims, therefore, to prospectively evaluate the impact of AHSCT on the vascular aspects of the systemic sclerosis. We will prospectively evaluate before and at 6 and 12 months after AHSCT: levels of adhesion molecules, coagulation and platelet aggregation, systemic inflammation and oxidative stress, in addition to skin biopsies, capillaroscopy, quantification of digital ulcers and progenitor and circulating endothelial cells, quality of life and functional capacity. Results will be correlated with clinical data of the patients and compared with those from healthy subjects. (AU)

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