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Remission of refractory C rohn's disease by high‐dose cyclophosphamide and autologous peripheral blood stem cell transplantation
Author(s) -
Hasselblatt P.,
Drognitz K.,
Potthoff K.,
Bertz H.,
Kruis W.,
Schmidt C.,
Stallmach A.,
SchmittGraeff A.,
Finke J.,
Kreisel W.
Publication year - 2012
Publication title -
alimentary pharmacology and therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 3.308
H-Index - 177
eISSN - 1365-2036
pISSN - 0269-2813
DOI - 10.1111/apt.12032
Subject(s) - medicine , cyclophosphamide , refractory (planetary science) , transplantation , immunosuppression , surgery , granulocyte colony stimulating factor , chemotherapy , clinical trial , gastroenterology , physics , astrobiology
Summary Background Despite advances in immunosuppressive therapy, up to 10% of patients with severe C rohn's disease ( CD ) remain refractory to conventional treatment. Limited evidence from pilot trials suggests that high‐dose immunosuppression and autologous peripheral blood stem cell transplantation (auto PBSCT ) may induce remission in these patients, but there is substantial controversy regarding the safety and efficacy of this approach. Aim To address this issue, a monocentre phase I / II trial of auto PBSCT was performed in patients with refractory CD in our hospital. Methods Here, we report on the outcome of 12 patients with refractory CD treated with auto PBSCT . Briefly, CD 34 + ‐selected PBSC s were harvested after mobilisation therapy with cyclophosphamide and granulocyte‐colony stimulating factor. Later, immunoablative conditioning therapy with high‐dose cyclophosphamide followed by auto PBSCT was applied and clinical and endoscopic responses were analysed after a mean follow‐up of 3.1 years (range 0.5–10.3 years). Results PBSC harvest following mobilisation chemotherapy was successful in 11/12 patients and resulted in a clinical and endoscopic improvement in 7/12 patients. Subsequent conditioning and auto PBSCT were performed in nine patients and were relatively well tolerated. Among those, five patients achieved a clinical and endoscopic remission within 6 months after auto PBSCT . However, relapses occurred in 7/9 patients during follow‐up, but disease activity could be controlled by low‐dose corticosteroids and conventional immunosuppressive therapy. Conclusion Immunoablation by cyclophosphamide and autologous peripheral blood stem cell transplantation is safe and effective to induce remission of refractory Crohn's disease, and should be further evaluated in randomised controlled trials.