2016
DOI: 10.1038/bmt.2016.211
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Autologous hematopoietic stem cell transplantation has better outcomes than conventional therapies in patients with rapidly progressive systemic sclerosis

Abstract: We retrospectively evaluated the efficacy of autologous hematopoietic stem cell transplantation (AHSCT) in 18 patients with rapidly progressive diffuse cutaneous systemic sclerosis (rp-dcSSc), and compared their disease outcomes with those of 36 demographically-and clinically-matched patients treated with conventional therapies. Cutaneous involvement, by performing modified Rodnan skin score (mRss), lung diffusion capacity, by measuring diffusing capacity of lung for carbon monoxide (DLCO), and disease activit… Show more

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Cited by 59 publications
(33 citation statements)
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“…1022 One retrospective study compared outcomes between 18 AHCT recipients with rapidly progressive diffuse cutaneous systemic sclerosis and a demographically- and clinically-matched cohort of 36 patients receiving conventional therapies. 17 Compared to the transplantation cohort, control patients had significantly lower overall survival (hazard ratio [HR] 6.94, P<0.002), including the subset of control patients who had received cyclophosphamide-based regimens (HR 5.98, P<0.006). AHCT recipients had a significantly higher likelihood of improving skin sclerosis and disease activity and preserving lung function.…”
Section: Summary Of Evidencementioning
confidence: 99%
See 1 more Smart Citation
“…1022 One retrospective study compared outcomes between 18 AHCT recipients with rapidly progressive diffuse cutaneous systemic sclerosis and a demographically- and clinically-matched cohort of 36 patients receiving conventional therapies. 17 Compared to the transplantation cohort, control patients had significantly lower overall survival (hazard ratio [HR] 6.94, P<0.002), including the subset of control patients who had received cyclophosphamide-based regimens (HR 5.98, P<0.006). AHCT recipients had a significantly higher likelihood of improving skin sclerosis and disease activity and preserving lung function.…”
Section: Summary Of Evidencementioning
confidence: 99%
“…A subsequent systematic review and meta-analysis included three randomized trials (including SCOT trial) and one comparative observational study. 17, 2325, 27 The control arm was monthly cyclophosphamide in all three trials and the majority of patients in the observational study. Compared to controls, patients receiving AHCT experienced lower all-cause mortality (risk ratio [RR] 0.50, [95% confidence intervals, 0.33–0.75] P=0.0007), and improved skin thickness, forced vital capacity, total lung capacity, and quality of life.…”
Section: Summary Of Evidencementioning
confidence: 99%
“…A miopatia é um preditor de desfecho desfavorável em pacientes com ES (JUNG et al, 2014;MORRISROE et al, 2000;PAIK et al, 2017;WALKER et al, 2017 (BURT et al, 2001;FARGE et al, 2002FARGE et al, , 2004OYAMA et al, 2007;VAN LAAR et al, 2014;HENES;SCHMALIZING;VOGEL, 2014;DEL PAPA et al, 2017). Embora a avaliação cutânea não tenha sido objetivo do estudo, a melhora do escore de Rodnan mostra que o grupo de pacientes foi responsivo ao tratamento a exemplo dos outros pacientes com ES transplantados no nosso centro e em outros centros.…”
Section: Comorbidades N(%)unclassified
“…O projeto brasileiro de TACTH para ES iniciou-se no Hospital das Clínicas da FMRP-USP em setembro de 2001, incluindo até hoje 84 pacientes e tem revelado resultados terapêuticos promissores. A maioria dos pacientes transplantados apresentou resposta clínica, com melhora do espessamento cutâneo e, pelo menos, estabilização da função pulmonar (BURT et al, 2013), corroborando dados da literatura internacional (DEL PAPA et al, 2016;FARGE et al, 2010;VAN LAAR et al, 2014). Além disso, considerando-se que os casos graves de ES apresentam altas taxas de morbimortalidade, o TACTH mostrou-se alternativa terapêutica relativamente segura, com aproximadamente 6% de morte relacionada ao tratamento.…”
Section: Reimpressões Dos Manuscritos Prólogounclassified
“…O racional para a utilização do TACTH no tratamento das DAIs se baseia na depleção inespecífica do sistema imunológico autorreativo seguido da reconstituição de um sistema imune autotolerante, no contexto ideal em que os fatores ambientais desencadeantes não estarão mais presentes ou efetivos (MURARO et al, 2017;SWART et al, 2017 HÜGLE;DAIKELER, 2010;MURARO et al, 2017;DOUEK, 2006;SWART et al, 2017). Apesar das taxas crescentes de sucesso clínico associadas ao TACTH na ES (DEL PAPA et al, 2016;SULLIVAN et al, 2016;SWART et al, 2017;VAN LAAR et al, 2014) (FARGE et al, 2010;MCSWEENEY et al, 2002), uma vez que a reconstituição adequada da função tímica é essencial para restituição da imunidade protetora a patógenos após transplante bem assim como no resetting imunológico (ALEXANDER et al, 2016;HAKIM et al, 2005 (FARGE et al, 2005;SAKKAS et al, 2002). Tais perturbações no repertório do TCR se relacionam ao estado inflamatório crônico no qual os pacientes se encontram (BARAUT et al, 2010), exemplificado pelos altos níveis séricos de IL-6, IL-8, IFN-g, TNF-a e MCP-1, como apresentado no Manuscrito 6.…”
Section: Discussão 6 Discussãounclassified