Outcomes of autologous hematopoietic stem cell transplantation in patients with refractory Crohn's disease: a single-center retrospective case series.

IF 1.2 4区医学 Q4 GASTROENTEROLOGY & HEPATOLOGY

Acta gastro-enterologica Belgica Pub Date : 2026-04-01 DOI:10.51821/89.2.15503

G Geebelen, A Van Besien, J Van Ham, A T D'Hooghe, M Ferrante, J Sabino, B Verstockt, D Dierickx, S Vermeire

{"title":"Outcomes of autologous hematopoietic stem cell transplantation in patients with refractory Crohn's disease: a single-center retrospective case series.","authors":"G Geebelen, A Van Besien, J Van Ham, A T D'Hooghe, M Ferrante, J Sabino, B Verstockt, D Dierickx, S Vermeire","doi":"10.51821/89.2.15503","DOIUrl":null,"url":null,"abstract":"Background and aims: Despite advances in biological therapies, a subset of patients with Crohn's disease (CD) remains or becomes refractory to all available treatments. Emerging evidence indicates that autologous hematopoietic stem cell transplantation (aHSCT) may offer a viable therapeutic option for carefully selected treatment-refractory CD patients.Patients and methods: We retrospectively analyzed the outcomes of all CD patients who underwent aHSCT for treatment-refractory disease at our IBD referral center between 2011 and 2023. All included patients had severe disease, unsuitable for surgery.Results: We included 24 CD patients (15 females, median age at transplantation 31.0 [27.0-34.5] years, median disease duration 13.0 [9.8-17.3] years) who underwent mobilization, of whom 23 patients proceeded to transplantation. All patients had CD with small bowel involvement, while 10 patients (41.7%) had concomitant perianal disease. After a median follow-up of 114.6 [66.0-138.4] months, 8 patients (34.8%) were still in remission without need for advanced therapy. Persistent disease activity after aHSCT, defined as total SES-CD >3, occurred in 15 patients (65.2%) with median time to restarting advanced therapy of 7.0 [5.0-25.0] months. At last follow-up, all patients are still alive. There were a few ICU admissions because of hemodynamic instability due to neutropenic fever following mobilization and transplantation. In the follow-up period, infectious complications occurred in 12 patients (52.2%).Conclusions: Autologous hematopoietic stem cell transplantation is a safe and effective therapy for inducing and maintaining remission in carefully selected therapy-refractory CD patients after extensive multidisciplinary discussion. Although many patients didn't achieve remission, most regained response to previously ineffective therapies.","PeriodicalId":7322,"journal":{"name":"Acta gastro-enterologica Belgica","volume":"89 2","pages":"325-332"},"PeriodicalIF":1.2000,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Acta gastro-enterologica Belgica","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.51821/89.2.15503","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"GASTROENTEROLOGY & HEPATOLOGY","Score":null,"Total":0}

引用次数: 0

Abstract

Background and aims: Despite advances in biological therapies, a subset of patients with Crohn's disease (CD) remains or becomes refractory to all available treatments. Emerging evidence indicates that autologous hematopoietic stem cell transplantation (aHSCT) may offer a viable therapeutic option for carefully selected treatment-refractory CD patients.

Patients and methods: We retrospectively analyzed the outcomes of all CD patients who underwent aHSCT for treatment-refractory disease at our IBD referral center between 2011 and 2023. All included patients had severe disease, unsuitable for surgery.

Results: We included 24 CD patients (15 females, median age at transplantation 31.0 [27.0-34.5] years, median disease duration 13.0 [9.8-17.3] years) who underwent mobilization, of whom 23 patients proceeded to transplantation. All patients had CD with small bowel involvement, while 10 patients (41.7%) had concomitant perianal disease. After a median follow-up of 114.6 [66.0-138.4] months, 8 patients (34.8%) were still in remission without need for advanced therapy. Persistent disease activity after aHSCT, defined as total SES-CD >3, occurred in 15 patients (65.2%) with median time to restarting advanced therapy of 7.0 [5.0-25.0] months. At last follow-up, all patients are still alive. There were a few ICU admissions because of hemodynamic instability due to neutropenic fever following mobilization and transplantation. In the follow-up period, infectious complications occurred in 12 patients (52.2%).

Conclusions: Autologous hematopoietic stem cell transplantation is a safe and effective therapy for inducing and maintaining remission in carefully selected therapy-refractory CD patients after extensive multidisciplinary discussion. Although many patients didn't achieve remission, most regained response to previously ineffective therapies.

查看原文本刊更多论文

自体造血干细胞移植治疗难治性克罗恩病患者的疗效：单中心回顾性病例系列

背景和目的：尽管生物治疗取得了进展，但仍有一部分克罗恩病（CD）患者对所有可用的治疗仍然或变得难以治愈。新出现的证据表明，自体造血干细胞移植（aHSCT）可能为精心挑选的难治性CD患者提供一种可行的治疗选择。患者和方法：我们回顾性分析了2011年至2023年在IBD转诊中心接受aHSCT治疗难治性疾病的所有CD患者的结果。所有患者均患有严重疾病，不适合手术。结果：我们纳入了24例CD患者（15例女性，移植时中位年龄31.0[27.0-34.5]岁，中位病程13.0[9.8-17.3]年），其中23例患者进行了移植。所有患者均有乳糜泻伴小肠受累，10例患者（41.7%）伴有肛周疾病。中位随访114.6[66.0-138.4]个月后，8例患者（34.8%）仍处于缓解期，无需进一步治疗。aHSCT后持续疾病活动，定义为总SES-CD bb0.3, 15例患者（65.2%）出现，重新开始高级治疗的中位时间为7.0[5.0-25.0]个月。最后随访时，所有患者均存活。有少数因动员和移植后中性粒细胞减少热引起的血流动力学不稳定而入院ICU。随访期间发生感染性并发症12例（52.2%）。结论：经过广泛的多学科讨论，对于精心挑选的难治性CD患者，自体造血干细胞移植是一种安全有效的诱导和维持缓解的治疗方法。虽然许多患者没有达到缓解，但大多数患者对先前无效的治疗恢复了反应。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Acta gastro-enterologica Belgica Medicine-Gastroenterology

CiteScore

2.30

自引率

20.00%

发文量

期刊介绍： The Journal Acta Gastro-Enterologica Belgica principally publishes peer-reviewed original manuscripts, reviews, letters to editors, book reviews and guidelines in the field of clinical Gastroenterology and Hepatology, including digestive oncology, digestive pathology, as well as nutrition. Pure animal or in vitro work will not be considered for publication in the Journal. Translational research papers (including sections of animal or in vitro work) are considered by the Journal if they have a clear relationship to or relevance for clinical hepato-gastroenterology (screening, disease mechanisms and/or new therapies). Case reports and clinical images will be accepted if they represent an important contribution to the description, the pathogenesis or the treatment of a specific gastroenterology or liver problem. The language of the Journal is English. Papers from any country will be considered for publication. Manuscripts submitted to the Journal should not have been published previously (in English or any other language), nor should they be under consideration for publication elsewhere. Unsolicited papers are peer-reviewed before it is decided whether they should be accepted, rejected, or returned for revision. Manuscripts that do not meet the presentation criteria (as indicated below) will be returned to the authors. Papers that go too far beyond the scope of the journal will be also returned to the authors by the editorial board generally within 2 weeks. The Journal reserves the right to edit the language of papers accepted for publication for clarity and correctness, and to make formal changes to ensure compliance with AGEB’s style. Authors have the opportunity to review such changes in the proofs.