title: Clinical trial: cyclophosphamide pulse therapy - a promising therapeutic alternative in refractory Crohn's disease.
abstract: BACKGROUND
  In severe steroid-refractory Crohn's disease (CD), established therapies fail in a relevant proportion of patients. Recent pilot studies indicated the efficacy of cyclophosphamide pulse therapy in these patients.
  
  
  OBJECTIVE
  To provide further and substantial evidence for the rationale to apply cyclophosphamide pulse therapy as therapeutic option in severe courses of CD.
  
  
  METHODS
  Fifteen patients with steroid-refractory (n = 13) or steroid-dependent (n = 2) CD received 2-6 (median 3) monthly pulses of 750 mg cyclophosphamide in an open-label fashion. Eleven patients were on concomitant immunosuppression (azathioprine/mercaptopurine n = 9; methotrexate n = 2).
  
  
  RESULTS
  Thirteen of 15 patients (87%) had a clinical response (CDAI decrease >100). Ten patients (67%) went into remission (CDAI <150) after 8 weeks. Steroid-free remission was achieved in eight patients (54%). Two patients (13%) failed to respond. Median CDAI decreased from 420 (245-550) to 100 (26-538) at week 8. Remission lasted 16 months (median, range 4-40). In three patients, arthritis, erythema nodosum and episcleritis completely resolved. Cyclophosphamide pulse therapy administration was well tolerated in all subjects.
  
  
  CONCLUSIONS
  Cyclophosphamide pulse therapy is safe and highly effective for induction and maintenance of remission in steroid-refractory/-dependent CD. There is a strong need for additional experience to improve the setting of the encouraging cyclophosphamide treatment in CD.
authors.affiliation: Department of Internal Medicine I, University Hospital of Schleswig-Holstein, Lübeck, Germany. klausjuergen.schmidt@uk-sh.de
authors.forename: K J
authors.initials: KJ
authors.lastname: Schmidt
authors.forename: K
authors.initials: K
authors.lastname: Fellermann
authors.forename: P
authors.initials: P
authors.lastname: Wellhöner
authors.forename: G
authors.initials: G
authors.lastname: Weitz
authors.forename: N
authors.initials: N
authors.lastname: Homann
authors.forename: K
authors.initials: K
authors.lastname: Herrlinger
authors.forename: H
authors.initials: H
authors.lastname: Lehnert
authors.forename: D
authors.initials: D
authors.lastname: Ludwig
authors.forename: J
authors.initials: J
authors.lastname: Büning
mesh_terms: D000328:Adult; D000368:Aged; D003424:Crohn Disease / Q000188:drug therapy*; D003520:Cyclophosphamide / Q000008:administration & dosage*; D004305:Dose-Response Relationship, Drug; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage*; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D020551:Pulse Therapy, Drug; D012074:Remission Induction; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D007166:Immunosuppressive Agents; D003520:Cyclophosphamide