Haematopoietic stem-cell transplantation for patients with Crohn's disease: primum non nocere

Crohn's disease is a lifelong and disabling condition characterised by progressive inflammatory damage of the gut wall. 1 Torres J Mehandru S Colombel J-F Peyrin-Biroulet L Crohn's disease. Lancet. 2017; 389: 1741-1755 Summary Full Text Full Text PDF PubMed Scopus (1384) Google Scholar The multifactorial pathogenesis of Crohn's disease means that recognising the contribution of each component in inducing and sustaining tissue injury is hard, leading to high variability in response to therapy and unpredictable outcomes. Despite efforts to identify novel targeted drugs, there is a therapeutic ceiling effect, with remission observed in only 30% of patients worldwide. 2 Raine T Danese S Breaking through the therapeutic ceiling: what will it take?. Gastroenterology. 2022; 162: 1507-1511 Summary Full Text Full Text PDF PubMed Scopus (25) Google Scholar Additionally, standard treatments are not curative, rarely restore organ function, and require long-term administration. Therefore, elimination, rather than inhibition, of inflammatory mediators seems to be a reasonable strategy to achieve treatment-free remission. Safety and efficacy of autologous haematopoietic stem-cell transplantation with low-dose cyclophosphamide mobilisation and reduced intensity conditioning versus standard of care in refractory Crohn's disease (ASTIClite): an open-label, multicentre, randomised controlled trialAlthough HSCT with an immune-ablative regimen of reduced intensity decreased endoscopic disease activity, significant adverse events deem this regimen unsuitable for future clinical use in patients with refractory Crohn's disease. Full-Text PDF Open Access