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Autologous hematopoietic stem cell transplantation in patients with refractory Crohn's disease.

Abstract：

BACKGROUND & AIMS:Crohn's disease (CD) is an immunologically mediated inflammatory disease of the gastrointestinal tract. Due to a high morbidity and/or an increase in mortality in refractory cases, a new treatment approach is needed. In theory, maximum immune ablation by autologous hematopoietic stem cell transplantation (HSCT) can induce a remission. METHODS:We conducted a phase 1 HSCT study in 12 patients with refractory CD. Candidates were younger than 60 years of age with a Crohn's Disease Activity Index (CDAI) of 250-400 despite conventional therapies including infliximab. Peripheral blood stem cells were mobilized with cyclophosphamide and granulocyte colony-stimulating factor and CD34 + enriched. The immune ablative (conditioning) regimen consisted of 200 mg/kg cyclophosphamide and 90 mg/kg equine antithymocyte globulin. RESULTS:The procedure was well tolerated with anticipated cytopenias, neutropenic fever, and disease-related fever, diarrhea, anorexia, nausea, and vomiting. The median days for neutrophil and platelet engraftment were 9.5 (range, 8-11) and 9 (range, 9-18), respectively. The initial median CDAI was 291 (range, 250-358). Symptoms and CDAI improved before hospital discharge, whereas radiographic and colonoscopy findings improved gradually over months to years following HSCT. Eleven of 12 patients entered a sustained remission defined by a CDAI < or =150. After a median follow-up of 18.5 months (range, 7-37 months), only one patient has developed a recurrence of active CD, which occurred 15 months after HSCT. CONCLUSIONS:Autologous HSCT may be performed safely and has a marked salutary effect on CD activity. A randomized study will be needed to confirm the efficacy of this therapy.

journal_name

Gastroenterology

journal_title

Gastroenterology

authors

Oyama Y,Craig RM,Traynor AE,Quigley K,Statkute L,Halverson A,Brush M,Verda L,Kowalska B,Krosnjar N,Kletzel M,Whitington PF,Burt RK

doi

10.1053/j.gastro.2004.11.051

keywords:

subject

Has Abstract

pub_date

2005-03-01 00:00:00

pages

552-63

issue

3

eissn

0016-5085

issn

1528-0012

pii

S0016508504021560

journal_volume

128

pub_type

临床试验,杂志文章

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