Hematopoietic stem cell transplantation for systemic sclerosis: Brazilian experience

Abstract Background: In 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 methods: This 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. Results: Median (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. Conclusions: Autologous 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.(AU)

Virtual learning object in hematopoietic stem cell transplantation for autoimmune diseases / Objeto virtual de aprendizaje en el trasplante de células madre hematopoyéticas para enfermedades autoinmunes / Objeto virtual de aprendizagem no transplante de células-tronco hematopoéticas para doenças autoimunes

ABSTRACT 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.

RESUMEN Objetivo: 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 iconos y menús fueron creados para atraer al usuario, facilitando la búsqueda de información y permitiendo el máximo aprovechamiento de los recursos disponibles en el sitio web. Conclusión: la metodología utilizada permitió el desarrollo del objeto de aprendizaje virtual, que puede ser utilizado como una herramienta para guiar y difundir el conocimiento sobre este tratamiento.

RESUMO Objetivo: 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.

Contribuições da pesquisa para os cuidados de enfermagem em transplante pediátrico de células-tronco hematopoiéticas / Research contributions for the nursing care in pediatric transplantation of hematopoietic stem cells / Contribuciones del estudio para los cuidados de enfermería en el trasplante de células madre hematopoyéticas pediátrico

Objetivo: Propor cuidados de Enfermagem ambulatoriais que subsidiem o tratamento de crianças em pós-transplante de células-tronco hematopoiéticas a partir dos dados advindos de pesquisa sobre perfil sociodemográfico e clínico. Método: Ensaio repercussivo sobre proposição de cuidados de Enfermagem baseados em resultados de pesquisa e por diagnósticos de Enfermagem segundo a taxonomia da North American Nursing Diagnosis Association. Resultados: Os principais diagnósticos foram risco de infecção, risco de função hepática prejudicada e risco de função cardiovascular prejudicada. Os cuidados de Enfermagem incluíram controle de sinais vitais, avaliação da ingesta alimentar, cuidados com cateteres, controle de exames laboratoriais, avaliação do funcionamento dos sistemas corporais, administração de medicamentos e transfusões, medidas de controle de transmissão de micro-organismos, educação em saúde/serviço e comunicação integrada com a equipe interdisciplinar. Conclusão: Os cuidados de Enfermagem foram sustentados por evidências e buscou-se o atendimento da criança em todas as suas necessidades

Objective: This paper aims to propose outpatient Nursing care that helps the children's treatment after hematopoietic stem cell transplantation, using data from research on sociodemographic and clinical profile. Method: Essay on Nursing care proposition was used, on the basis of the research results and Nursing diagnoses, in accordance with the taxonomy of the North American Nursing Diagnosis Association. Results: The main diagnoses were infection risk, impaired liver function risk, and impaired cardiovascular function. Nursing care included vital signs control, food intake evaluation, catheter care, control of laboratory tests, evaluation of the body systems functioning, administration of medications and transfusions, control measures of microorganisms' transmission, health/service education, and integrated communication with the interdisciplinary team. Conclusion: Nursing care was supported by evidence and the child's care was sought in all his/her needs

Objetivo: Proponer cuidados de Enfermería ambulatoria que subsidien el tratamiento de niños en el post trasplante de células madre hematopoyéticas a partir de los datos provenientes de estudios sobre el perfil sociodemográfico y clínico. Método: Ensayo repercusivo sobre proposición de cuidados de Enfermería basados en resultados de estudios y diagnósticos de Enfermería según la taxonomía de la North American Nursing Diagnosis Association. Resultados: los principales diagnósticos fueron el riesgo de infección, el riesgo de función hepática perjudicada y el riesgo de función cardiovascular perjudicada. Los cuidados de Enfermería incluyeron el control de señales vitales, evaluación de la ingesta alimentaria, cuidados con catéteres, control de exámenes de laboratorios, evaluación del funcionamiento de los sistemas corporales, administración de medicamentos y trasfusiones, medidas de control de trasmisión de microorganismos, educación en salud/servicio y comunicación integrada con el equipo interdisciplinar. Conclusiones: Los cuidados de Enfermería se sostuvieron por las evidencias y se buscó el atendimiento del niño en todas sus necesidades