title: Efficacy and safety of granulocyte, monocyte/macrophage adsorptive in pediatric ulcerative colitis.
abstract: OBJECTIVE
  To investigate efficacy and safety for granulocyte, monocyte apheresis in a population of pediatric patients with ulcerative colitis.
  
  
  METHODS
  The ADAPT study was a prospective, open-label, multicenter study in pediatric patients with moderate, active ulcerative colitis with pediatric ulcerative colitis activity index (PUCAI) of 35-64. Patients received one weekly apheresis with Adacolumn(®) granulocyte, monocyte/macrophage adsorptive (GMA) apheresis over 5 consecutive weeks, optionally followed by up to 3 additional apheresis treatments over 3 consecutive weeks. The primary endpoint was the change in mean PUCAI between baseline and week 12; the secondary endpoint was improvement in PUCAI categorized as (Significant Improvement, PUCAI decrease of ≥ 35), Moderate Improvement (PUCAI decrease of 20 < 35), Small Improvement (PUCAI decrease of 10 < 20) or No change (PUCAI decrease of < 10).
  
  
  RESULTS
  Twenty-five patients (mean age 13.5 years; mean weight 47.7 kg) were enrolled. In the intention-to-treat set (ITT), the mean value for PUCAI improvement was 22.3 [95%CI: 12.9-31.6; n = 21]. In the per-protocol (PP) set, the mean improvement was 36.3 [95%CI: 31.4-41.1; n = 8]. Significant Improvement was recorded for 9 out of 20 patients (45%); 5 out of 20 patients (25%) had Moderate Improvement and one patient (5%) had No Change in PUCAI score at week 12. In the PP set, six out of eight patients (75%) showed Significant Improvement; and in two out of eight patients (25%) Moderate Improvement was recorded. The endoscopic activity index (EAI) decreased by 3 points on average. Seven (7) out of 21 (33%) patients in ITT and 4 out of 8 (50%) patients in PP have used steroids during the clinical investigation. The mean steroid dosage for these patients in the ITT set decreased from a mean 12.4 mg to 10 mg daily on average from Baseline to week 12.
  
  
  CONCLUSIONS
  Adacolumn(®) GMA apheresis treatment was effective in pediatric patients with moderate active Ulcerative Colitis. No new safety signals were reported. The present data contribute to considering GMA apheresis as a therapeutic option in pediatric patients having failed first line therapy.
authors.affiliation: Tarja Ruuska, Department of Pediatrics, Tampere University Hospital, 33521 Tampere, Finland.
authors.forename: Tarja
authors.initials: T
authors.lastname: Ruuska
authors.affiliation: Tarja Ruuska, Department of Pediatrics, Tampere University Hospital, 33521 Tampere, Finland.
authors.forename: Peter
authors.initials: P
authors.lastname: Küster
authors.affiliation: Tarja Ruuska, Department of Pediatrics, Tampere University Hospital, 33521 Tampere, Finland.
authors.forename: Lena
authors.initials: L
authors.lastname: Grahnquist
authors.affiliation: Tarja Ruuska, Department of Pediatrics, Tampere University Hospital, 33521 Tampere, Finland.
authors.forename: Fredrik
authors.initials: F
authors.lastname: Lindgren
authors.affiliation: Tarja Ruuska, Department of Pediatrics, Tampere University Hospital, 33521 Tampere, Finland.
authors.forename: Anne Vibeke
authors.initials: AV
authors.lastname: Wewer
mesh_terms: D000293:Adolescent; D001781:Blood Component Removal / Q000009:adverse effects; D002648:Child; D003093:Colitis, Ulcerative / Q000628:therapy*; D005260:Female; D006098:Granulocytes; D006801:Humans; D008264:Macrophages; D008297:Male; D009000:Monocytes; D011446:Prospective Studies
keywords: Clinical trial; Granulocyte-monocyte apheresis; Inflammatory bowel disease; Pediatric; Steroids; Therapy; Ulcerative colitis