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Autologous stem cell transplantation in severe treatment-resistant Crohn's disease: long-term follow-up of UK patients treated on compassionate basis

Lookup NU author(s): Professor Graham Jackson

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Abstract

Background: Although autologous stem cell transplantation (ASCT) may achieve disease control in severe treatment-resistant Crohn's disease (CD), relapse is frequent, and there is little information regarding long-term outcomes in terms of response to subsequent treatments and complications of ASCT.Design: Retrospective evaluation of UK patients treated on a compassionate basis from three UK tertiary centres.Methods: We summarize long-term outcomes of six previously unreported patients with severe treatment-resistant CD treated with ASCT according to international guidelines between 2003 and 2009. Median duration of CD before ASCT was 14 (7-22) years. Following stem cell mobilization, patients were treated with high-dose cyclophosphamide (200 mg/kg) and rabbit anti-thymocyte globulin (7.5 mg/kg) followed by ASCT.Results: All patients tolerated ASCT with routine toxicities and no treatment-related mortality and are alive at 50-123 months post-ASCT. Clinical and endoscopic remissions of CD were confirmed at 3 months post-ASCT in five patients, although median time to next treatment for inflammatory disease was 10 months (range: 3-16 months). Subsequently, disease control was achieved with previously ineffective and newer treatments, with surgery performed predominantly for pre-existing fibrotic strictures. Two patients became independent of home total parenteral nutrition (TPN). Reported late complications of ASCT included hypothyroidism and ovarian failure.Conclusion: Long-term follow-up supports the safety and feasibility of ASCT as a means of achieving short-term control of severe CD whilst potentially re-sensitizing the disease to medical therapy and reducing requirements for surgery and TPN. Given the inevitability of relapse, pre-emptive salvage and/or maintenance treatments post-ASCT should be the focus of future trials.

Publication metadata

Author(s): Snowden JA, Ansari A, Sachchithanantham S, Jackson G, Thompson N, Lobo A, Sanderson J, Kazmi M

Publication type: Article

Publication status: Published

Journal: QJM: An International Journal of Medicine

Year: 2014

Volume: 107

Issue: 11

Pages: 871-877

Print publication date: 01/11/2014

Online publication date: 06/05/2014

Acceptance date: 06/03/2014

ISSN (print): 1460-2725

ISSN (electronic): 1460-2393

Publisher: Oxford University Press

URL: http://dx.doi.org/10.1093/qjmed/hcu095

DOI: 10.1093/qjmed/hcu095

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