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Autologous hematopoietic stem cell transplantation in patients with refractory Crohn's disease.
Oyama, Yu; Craig, Robert M; Traynor, Ann E; Quigley, Kathleen; Statkute, Laisvyde; Halverson, Amy; Brush, Mary; Verda, Larissa; Kowalska, Barbara; Krosnjar, Nela; Kletzel, Morris; Whitington, Peter F; Burt, Richard K.
Afiliação
  • Oyama Y; Division of Immunotherapy, Department of Medicine, Northwestern University Medical Center, Chicago, Illinois 60611, USA. y-oyama@northwestern.edu
Gastroenterology ; 128(3): 552-63, 2005 Mar.
Article em En | MEDLINE | ID: mdl-15765390
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.
Assuntos
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Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Crohn / Transplante de Células-Tronco Hematopoéticas Tipo de estudo: Clinical_trials Limite: Adolescent / Adult / Female / Humans / Male Idioma: En Ano de publicação: 2005 Tipo de documento: Article
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Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Crohn / Transplante de Células-Tronco Hematopoéticas Tipo de estudo: Clinical_trials Limite: Adolescent / Adult / Female / Humans / Male Idioma: En Ano de publicação: 2005 Tipo de documento: Article