Mesenchymal stromal cell infusions as rescue therapy for corticosteroid-refractory adult autoimmune enteropathy

Rachele Ciccocioppo, Maria L. Russo, Maria E. Bernardo, Federico Biagi, Laura Catenacci, Maria A. Avanzini, Costanza Alvisi, Alessandro Vanoli, Rachele Manca, Ombretta Luinetti, Franco Locatelli, Gino R. Corazza

Research output: Contribution to journalArticlepeer-review


Adult autoimmune enteropathy (AIE) is a rare cause of malabsorption syndrome unresponsive to dietary restriction. Its diagnostic hallmarks are small-bowel villous atrophy and antienterocyte autoantibodies. Therapy is based mainly on nutritional support and immunosuppression. We treated a 61-year-old woman with corticosteroid-refractory AIE and life-threatening malabsorption syndrome with systemic infusions of autologous, bone marrow-derived, mesenchymal stromal cells (MSCs) as rescue therapy. The MSCs were expanded ex vivo following a previously used Good Manufacturing Practice procedure, and 2 intravenous infusions of 1.8 × 106 MSCs/kg body weight were administered 2 weeks apart. Analysis of circulating and mucosal regulatory T-and B-cell numbers, and of serum and secretory immunoglobulin levels, was performed before and after treatment. The MSC infusions were safe and effective, leading to disappearance of disease hallmarks and recovery from the life-threatening condition. Increases in mucosal regulatory T-cell numbers and secretory immunoglobulin levels were also observed. The benefit, however, was transient, and a further MSC infusion resulted in the same short efficacy. This case encourages the use of MSCs to treat patients with lifethreatening, corticosteroid-refractory AIE and suggests that MSC infusion can attenuate, albeit transiently, the autoimmune attack.

Original languageEnglish
Pages (from-to)909-914
Number of pages6
JournalMayo Clinic Proceedings
Issue number9
Publication statusPublished - 2012

ASJC Scopus subject areas

  • Medicine(all)


Dive into the research topics of 'Mesenchymal stromal cell infusions as rescue therapy for corticosteroid-refractory adult autoimmune enteropathy'. Together they form a unique fingerprint.

Cite this