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.

Abstract Background The reproducibility and reliability of the modified Rodnan's Skin Score (mRSS) are debated due to investigator-related subjectivity. Here, we evaluate if durometry correlates with mRSS in patients with diffuse systemic sclerosis (SSc). Methods This cross-sectional study was conducted from December 2018 to June 2019, including 58 diffuse SSc patients. Two certified researchers, blind to each other's scores, performed the mRSS, followed by durometry at 17 predefined skin sites. For durometry and mRSS, individual scores per skin site were registered. Durometry and mRSS results measured by each researcher, as well as scores from different researchers, were compared. Skin thickness measurements from forearm skin biopsies were available in a subset of the patients, for comparisons. Statistical analyses included Cohen's Kappa Coefficient, Intraclass Correlation Coefficient, Kendall's Coefficient and Spearman's test. Results Mean (standard deviation, SD) patient age was 44.8 (12.9) years, and 88% were female. Inter-rater agreement varied from 0.88 to 0.99 (Intraclass correlation coefficient) for durometry, and 0.54 to 0.79 (Cohen's Kappa coefficient) for mRSS, according to the specific evaluated sites. When data were compared with skin thickness assessed in forearm biopsies, durometry correlated better with skin thickness than mRSS. Conclusion Durometry may be considered as an alternative method to quantify skin involvement in patients with diffuse SSc. The strong inter-rater agreement suggests that the method may be useful for the assessment of patients by multiple researchers, as in clinical trials.

RESUMO As doenças falciformes são as hemoglobinopatias mais frequentes mundialmente. Nas últimas décadas vivenciamos melhora na sobrevida de portadores destas patologias com a introdução de medidas preventivas e o uso precoce da hidroxiurea. O transplante de medula óssea alogênico (alo TMO) é a única opção terapêutica curativa para as hemoglobinopatias. O mesmo tem sido indicado para pacientes com complicações neurológicas, crises vasoclusivas repetidas, alguma lesão orgânica e alosensibilizados. O uso de doadores relacionados de medula óssea ou cordão umbilical mostrou em 1000 procedimentos realizados sobrevida global de 95% e sobrevida livre de ventos de 90%. O uso de doadores não aparentados não mostrou resultados tão expressivos, mas no futuro métodos melhores de tipagem de HLA e de medidas de suporte podem melhorar estes resultados. No Brasil apenas recentemente o alo TMO foi incluído no âmbito do sistema único de saúde (SUS) como opção terapêutica para portadores de doenças falciformes. O uso de doadores aparentados de MO ou de SCU com regime mieloablativo é considerado hoje tratamento estabelecido, sendo que o uso de doadores alternativos não aparentados ou haploidenticos e o uso de transplante com regime não mieloablativo deve ser considerado apenas em estudos clínicos.

SUMMARY Sickle-cell diseases are the most common inherited hemoglobinopathies worldwide. Improvement in survival has been seen in the last decades with the introduction of careful screening and prevention of complications and the introduction of hydroxyurea. Stem-cell transplantation is currently the only curative option for these patients and has been indicated for patients with neurological events, repeated vaso-occlusive crisis, any organ damage or presence of red blood cell antibodies. Related bone-marrow or cord-blood transplant has shown an overall survival of more than 90% with a disease-free survival of 90% in 1,000 patients transplanted in the last decades. The use of unrelated donors unfortunately has not shown the same good results, but better typing methods and improved support may improve the outcome with this source of stem cells in the future. In Brazil, only recently stem cell transplant from related donors has been included in the procedures performed in the public health system. The use of related bone marrow or cord blood and a myeloablative conditioning regimen are considered standard of care for patients with sickle-cell diseases. Transplants with non-myeloablative regimens, unrelated donors or haploidentical donors should be performed only in controlled clinical trials.

O artigo se propõe a destacar algumas contribuições da teoria dos processos civilizadores do sociólogo alemão Norbert Elias para estudos na área da educação e da cultura que abordam a leitura, seus objetos e práticas, e, enfim, para os que versam sobre a produção e o consumo dos livros destinados à formação das novas gerações. Num primeiro momento do texto, introduz-se a obra em que o autor desenvolve a teoria sobre os processos civilizadores, apresentando sua organização e premissas gerais. A seguir, discute-se como a compreensão sobre o processo de constituição das sociedades modernas ocidentais - seguindo as proposições de Elias - oportuniza fecundas contribuições para uma abordagem histórica e sociológica dos objetos de consumo cultural voltados à infância, especialmente os livros: objetos por excelência da "civilidade".

The article intends to highlight some contributions of the theory of the German sociologist Norbert Elias for studies in the area of education and culture which deal with reading, its objects and practices, and, especially, for studies regarding the production and consumption of books destined for new generations' education. Firstly, the text introduces the work in which the author develops the theory on civilizing processes, with its organization and general assumptions. Next, it discusses how the understanding of the process for the formation of modern western societies - according to Norbert Elias' ideas - presents productive contributions to a historical and sociological approach of the objects of cultural consumption destined to childhood, specially the books: objects by excellence of civility.

OBJETIVO: Relatar uma série de casos de endoleaks, com descrição da classificação vigente. MATERIAIS E MÉTODOS: Realizou-se um estudo retrospectivo dos endoleaks diagnosticados em nossa instituição, entre 2005 e 2009. Foram incluídos 20 casos, utilizados para ilustrar os diferentes tipos de endoleaks. RESULTADOS: Setenta por cento dos pacientes eram do sexo masculino. A idade variou entre 43 e 91 anos, média de 76,3 anos. Treze casos foram observados na aorta abdominal infrarrenal, quatro na aorta torácica, dois nas artérias ilíacas e um no território carotídeo. A ultrassonografia foi o método utilizado para o diagnóstico em 3 casos e a tomografia computadorizada, nos outros 17 casos. Classificação: tipo I, 60%; tipo II, 25%; tipo III, 15%. Não foram observados os demais tipos nesta série. CONCLUSÃO: O diagnóstico precoce e a correta classificação são fundamentais para o manejo adequado dos casos de endoleaks, tornando o conhecimento de seus subtipos conceito fundamental na formação do médico especialista em radiologia e diagnóstico por imagem e para o cirurgião vascular.

OBJECTIVE: To describe a series of endoleak cases and their respective classification. MATERIALS AND METHODS: The authors developed a retrospective study of endoleaks diagnosed at their institution in the period between 2005 and 2009. Twenty cases were included to illustrate the different types of endoleaks. RESULTS: Seventy percent of the patients were men, and the ages ranged from 43 to 91 years (mean, 76.3 years). Thirteen cases were observed in the infrarenal abdominal aorta, four in the thoracic aorta, two in the iliac artery, and one in the carotid territory. Ultrasonography was the method utilized for diagnosis in three cases, and computed tomography in the other 17 cases. Classification: 60% type I, 25% type II, 15% type III. Other endoleak types were not observed in the present series. CONCLUSION: Early diagnosis and correct classification of endoleaks are crucial for an appropriate management of cases. The knowledge of endoleaks subtypes is fundamental in the education of physicians specialized in radiology and imaging diagnosis as well as for vascular surgeons.