Background/Objective We sought to evaluate if autologous hematopoietic stem cell transplantation (AHSCT) influences the functional status of systemic sclerosis (SSc) patients. Methods From 2014 to 2018, a cohort of 27 SSc patients was assessed before, and at 6 and 12 months after AHSCT for modified Rodnan’s skin score (mRSS), mouth opening, hand grip strength, range of motion (ROM), functional ability of upper limbs (DASH questionnaire and Cochin hand function scale—CHFS), 6-minute walk test (6MWT), and quality of life (SF-36 questionnaire). Linear regression models with random effects and Spearman’s test were used for statistical analysis. Results At 6 and 12 months after AHSCT, respectively, we observed significant improvement of mRSS (p < 0.01 and p < 0.01), mouth opening (p = 0.02 and p < 0.01), hand function (DASH, p < 0.01 and p < 0.01; CHFS, p < 0.01 and p < 0.01; strength, p < 0.01 and p < 0.01), physical capacity (6MWT, p = 0.02 and p = 0.03) and physical (p < 0.01 and p < 0.01) and mental (ns and p = 0.02) component scores of SF-36. At 12 months after AHSCT, ROM measurements improved (p < 0.05) in five out of six evaluated joints in both hands, compared to baseline. Correlation was significant between physical capacity and quality of life (R = 0.62; p < 0.01), between DASH and quality of life (R = −0.48; p = 0.03), and between skin involvement and wrist ROM measures (dominant hand, R = −0.65, p < 0.01; non-dominant hand, R = −0.59; p < 0.01). Conclusions AHSCT enhances the functional status of SSc patients in the first year of follow-up, significantly improving hand function, physical capacity and quality of life. These results are interpreted as positive outcomes of AHSCT for SSc.

Objective: describe the development of a virtual learning object to provide information about autologous transplantation of hematopoietic stem cells to autoimmune diseases. Methods: methodological study of a website development, using the instructional design model that includes Analysis, Design, Development and Implementation. Results: the virtual object, available at http://www.transplantardai.com.br, was developed in a web platform, in the Hypertext Markup Language, using the software WebAcappella -Responsive Website Creator (Intuisphere, France 2016). The content was structured in the modules: History, Transplant, Autoimmune Diseases, Links, Guidelines, Speech Team and Doubts. The icons and menus were created in order to attract the user, facilitating the search for information and allowing maximum use of the resources available on the website. Conclusion: the methodology used allowed the development of the virtual learning object, which can be used as a tool to guide and disseminate knowledge about this treatment. RESUMOObjetivo: descrever o desenvolvimento de um objeto virtual de aprendizagem para disponibilização de informações sobre transplante autólogo de células-tronco hematopoéticas para doenças autoimunes. Métodos: estudo metodológico de desenvolvimento de um website, empregando o modelo de design instrucional que envolve Análise, Design, Desenvolvimento e Implementação. Resultados: o objeto virtual, disponível no endereço eletrônico http://www.transplantardai.com.br, foi desenvolvido em plataforma web, na linguagem de marcação Hypertext Markup Language, utilizando-se o software WebAcappella -Responsive Website Creator (Intuisphere, França 2016). O conteúdo foi estruturado nos seguintes módulos: História, Transplante, Doenças Autoimunes, Links, Orientações, Fala Equipe e Dúvidas. Os ícones e menus foram criados de modo a atrair o usuário, facilitando a busca de informações e permitindo máximo uso dos recursos disponíveis no website. Conclusão: a metodologia empregada permitiu o desenvolvimento do objeto virtual de aprendizagem, que poderá ser utilizado como ferramenta para orientar e disseminar o conhecimento sobre esse tratamento. Descritores: Transplante de Células-Tronco Hematopoéticas; Doenças Autoimunes; Materiais de Ensino; Educação a Distância; Internet. RESUMENObjetivo: describir el desarrollo de un objeto de aprendizaje virtual para proporcionar información sobre el trasplante autólogo de células madre hematopoyéticas en las enfermedades autoinmunes. Métodos: estudio metodológico del desarrollo del sitio web, utilizando el modelo de diseño instruccional (Análisis, Diseño, Desarrollo e Implementación). Resultados: el objeto virtual, disponible en http://www.transplantardai.com.br, fue desarrollado en una plataforma web, en el lenguaje de marcación Hypertext Markup Language, utilizando el software WebAcappella -Responsive Website Creator (Intuisphere, Francia 2016). El contenido se estructuró en los módulos: Historia, Trasplante, Enfermedades Autoinmunes, Links, Guías, Habla Equipo y Dudas. Los...

AGRADECIMENTOS

Background Takayasu's arteritis is a rare autoimmune vasculitis that affects medium and large vessels, especially the aorta and its main branches. Autologous stem cell transplantation for autoimmune disease has been shown to be effective in some refractory cases. Objectives Describe our experience with autologous hematopoietic stem cell transplantation (AHSCT) in Takayasu's arteritis. Methods Three patients with refractory Takayasu's arteritis were treated with AHSCT. Hematopoietic stem cells were mobilized with 2g/m2 cyclophosphamide plus G-CSF, and subsequently harvested by leukoapheresis and cryopreserved, nonmanipulated. Patients then received 200mg/kg IV cyclophosphamide and 4.5mg/kg rabbit anti-thymocyte globulin (ATG) over five consecutive days, followed by infusion of the cryopreserved cells. Results The procedure was well tolerated by all patients. Side effects included fever, rash, nausea, vomiting and neutropenic fever. None of the patients presented severe side effects during or after AHSCT. One patient presented CMV reactivation after transplant and was preemptively treated, without clinical manifestations of disease.Case 1: 41 year-old female, with a history of dizziness, claudication of upper and lower limbs, non-palpable right radial pulse and transient visual impairment for over 13 years. Arteriography showed irregularity and stenosis of abdominal aorta, right and left iliac arteries, left subclavian and carotid arteries. She had been unsuccessfully treated with steroids, methotrexate, cyclophosphamide, chlorambucil and mycophenolate mofetil. On follow-up of 127 months after AHSCT, she presented clinical and laboratory remission. Case 2: 30 year-old female, with intermittent visual deficits, upper limb claudication, subclavian steal syndrome and 3 episodes of transient ischemic strokes. Previous treatments included steroids, azathioprine, cyclophosphamide and mycophenolate mofetil, besides a stent placed in the right carotid artery. On 43 months follow-up, she remained symptomatic with high levels of acute phase reactants, indicating refractoriness to AHSCT. After transplant, she also failed etanercept injections, but disease activity was further controlled with tocilizumab. Case 3: 39 year-old male with intestinal claudication, renovascular hypertension, upper limb claudication and dizziness. Magnetic angioresonance (NMR) images evidenced right renal and mesenteric artery stenosis, and bilateral critical narrowing of carotid, subclavian, brachial and axilary arteries, and irregularities along the aorta. On 15 months follow-up, there was clinical and laboratorial improvement. Patients 1 and 3 presented partial reversal of vessel stenosis detected by NMR images. Conclusions Very few cases of vasculitides treated with AHSCT have been reported in the literature. To the best of our knowledge, this is the largest reported series of transplanted Takayasu's patients. Although response to transplant was not universal, beneficial effects were verified in two out of three patients, who remained in...