Stem Cell Transplantation for Refractory Crohn Disease

• Autores: Richard K. Burt, Milton A. Ruiz, Roberto L. Kaiser Jr, Christopher J. Hawkey, James Lindsay, John G. Gribben
• Localización: JAMA: the journal of the American Medical Association, ISSN 0098-7484, Vol. 315, Nº. 23, 2016, págs. 2620-2621
• Idioma: inglés
• Títulos paralelos:
  • Stem Cell Transplantation for Refractory Crohn Disease—Reply
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• Resumen
  • English

    The Autologous Stem Cell Transplantation International Crohn Disease (ASTIC) trial by Dr Hawkey and colleagues1 was interpreted as a failure of autologous hematopoietic stem cell transplantation (HSCT) compared with standard care. However, the ASTIC trial used a complex multifaceted primary end point that included all of the following: Crohn disease activity index less than 150; no active treatment; and endoscopic and radiologic remission. This end point has not been used in any prior study. The data from such a small cohort of patients would have been interpreted as encouraging if the authors had used some of their secondary outcomes as the primary end point, for example, Crohn disease activity index change from baseline (P = .04) or simple endoscopic score of Crohn disease change from baseline (P = .03).

  • English

    The ASTIC trial was designed to investigate the possibility, suggested by a number of case reports, that long-term, drug-free cure of Crohn disease could be achieved through autologous HSCT.1 The lack of statistical significance of the primary end point showed that this experience is rare. However, we agree with Dr Burt and colleagues that the extent of improvement in secondary end points suggests possible therapeutic efficacy, albeit falling short of cure.