{"title":"自体外周干细胞移植治疗系统性硬化症:向前迈出了重要一步,但我们必须缓和我们的热情!]","authors":"L Mouthon","doi":"10.1016/j.revmed.2023.11.010","DOIUrl":null,"url":null,"abstract":"<p><p>Three prospective randomized studies have demonstrated the efficacy of autologous hematopoietic stem cell (HSC) transplantation in systemic sclerosis (SSc) on survival. These results encourage us to offer this therapy to patients who have a rapidly progressive disease and who have early symptoms but no advanced visceral involvement. HSC autograft can thus be discussed in patients with diffuse cutaneous SSc with a duration of the disease since the first visceral manifestations (cutaneous, cardiac, digestive, pulmonary, or renal) excluding Raynaud's phenomenon of less than 5 years. However, the indications for HSC autograft in SSc validated at European level and in the national diagnostic and care protocol (PNDS) are broader and some of these indications are debatable, in particular in patients with worsening diffuse interstitial lung disease. These indications are discussed in a reasoned way, taking into account the level of evidence and the toxicity of the HSC autograft.</p>","PeriodicalId":94122,"journal":{"name":"La Revue de medecine interne","volume":" ","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2023-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"[Autologous peripheral stem cell transplantation in systemic sclerosis: An important step forward, but we must temper our enthusiasm!]\",\"authors\":\"L Mouthon\",\"doi\":\"10.1016/j.revmed.2023.11.010\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Three prospective randomized studies have demonstrated the efficacy of autologous hematopoietic stem cell (HSC) transplantation in systemic sclerosis (SSc) on survival. These results encourage us to offer this therapy to patients who have a rapidly progressive disease and who have early symptoms but no advanced visceral involvement. HSC autograft can thus be discussed in patients with diffuse cutaneous SSc with a duration of the disease since the first visceral manifestations (cutaneous, cardiac, digestive, pulmonary, or renal) excluding Raynaud's phenomenon of less than 5 years. However, the indications for HSC autograft in SSc validated at European level and in the national diagnostic and care protocol (PNDS) are broader and some of these indications are debatable, in particular in patients with worsening diffuse interstitial lung disease. These indications are discussed in a reasoned way, taking into account the level of evidence and the toxicity of the HSC autograft.</p>\",\"PeriodicalId\":94122,\"journal\":{\"name\":\"La Revue de medecine interne\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2023-12-05\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"La Revue de medecine interne\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1016/j.revmed.2023.11.010\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"La Revue de medecine interne","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1016/j.revmed.2023.11.010","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
[Autologous peripheral stem cell transplantation in systemic sclerosis: An important step forward, but we must temper our enthusiasm!]
Three prospective randomized studies have demonstrated the efficacy of autologous hematopoietic stem cell (HSC) transplantation in systemic sclerosis (SSc) on survival. These results encourage us to offer this therapy to patients who have a rapidly progressive disease and who have early symptoms but no advanced visceral involvement. HSC autograft can thus be discussed in patients with diffuse cutaneous SSc with a duration of the disease since the first visceral manifestations (cutaneous, cardiac, digestive, pulmonary, or renal) excluding Raynaud's phenomenon of less than 5 years. However, the indications for HSC autograft in SSc validated at European level and in the national diagnostic and care protocol (PNDS) are broader and some of these indications are debatable, in particular in patients with worsening diffuse interstitial lung disease. These indications are discussed in a reasoned way, taking into account the level of evidence and the toxicity of the HSC autograft.