title: Long-term safety, efficacy, pharmacokinetics and pharmacodynamics of omalizumab in children with severe uncontrolled asthma.
abstract: BACKGROUND
  Omalizumab is effective and well-tolerated in children with moderate to severe allergic asthma. However, the effects of long-term treatment with omalizumab in this population haven't been well investigated. The objective of this study is to evaluate the long-term safety, efficacy, pharmacokinetics and pharmacodynamics of omalizumab in children with uncontrolled severe asthma.
  
  
  METHODS
  Thirty-eight Japanese children (aged 7-16 years) who completed the 24-week treatment core study were included in an uncontrolled extension study, in which treatment with omalizumab continued until the pediatric indication was approved in Japan (ClinicalTrials.gov number: NCT01328886).
  
  
  RESULTS
  Thirty-five patients (92.1%) completed the extension study. The median exposure throughout the core and extension studies was 116.6 weeks (range, 46.9-151.1 weeks). The most common adverse events were nasopharyngitis, influenza, upper respiratory tract infection, and asthma. Serious adverse events developed in 10 patients (26.3%), but resolved completely with additional treatments. Incidence of adverse events didn't increase with extended exposure with omalizumab. Twenty-nine patients (76.3%) achieved completely- or well-controlled asthma compared with 9 patients (23.7%) at the start of the extension study. QOL scores, the rates (per year) of hospitalizations and ER visits were significantly improved compared with the baseline of the core study [39.0 vs 48.0 (median), p < 0.001 for QOL, 1.33 vs 0.16, p < 0.001 for hospitalization, 0.68 vs 0.15, p = 0.002 for ER visits]. Remarkably, the mean total IgE level showed a decreasing trend while exposure to omalizumab remained at steady-state.
  
  
  CONCLUSIONS
  Long-term treatment with omalizumab is well-tolerated and effective in children with uncontrolled severe allergic asthma. No new safety findings were identified.
authors: Department of Pediatrics, Fukuoka National Hospital, Fukuoka, Japan. Electronic address: odaji@mfukuoka2.hosp.go.jp.
authors: Hiroshi
authors: H
authors: Odajima
authors: Department of Allergy, Clinical Research Center for Allergology and Rheumatology, Sagamihara National Hospital, Sagamihara, Japan.
authors: Motohiro
authors: M
authors: Ebisawa
authors: Yoga Allergy Clinic, Tokyo, Japan.
authors: Toshikazu
authors: T
authors: Nagakura
authors: Institute for Clinical Research, Mie National Hospital, Tsu, Japan.
authors: Takao
authors: T
authors: Fujisawa
authors: Division of Allergy, Tokyo Metropolitan Children's Medical Center, Tokyo, Japan.
authors: Akira
authors: A
authors: Akasawa
authors: Department of Allergy, Aichi Children's Health and Medical Center, Obu, Japan.
authors: Komei
authors: K
authors: Ito
authors: Department of Pediatrics, Osaka Prefectural Medical Center for Respiratory and Allergic Diseases, Habikino, Japan.
authors: Satoru
authors: S
authors: Doi
authors: Department of Pediatrics, The Fraternity Memorial Hospital, Tokyo, Japan.
authors: Koichi
authors: K
authors: Yamaguchi
authors: Department of Pediatrics, Jikei Daisan Hospital, Tokyo, Japan.
authors: Toshio
authors: T
authors: Katsunuma
authors: Department of Allergy, Kanagawa Children's Medical Center, Yokohama, Japan.
authors: Kazuyuki
authors: K
authors: Kurihara
authors: Department of Pediatrics, Graduate School of Medicine, Gifu University, Gifu, Japan.
authors: Takahide
authors: T
authors: Teramoto
authors: Department of Pediatrics, Fukuyama Medical Center, Fukuyama, Japan.
authors: Kazuko
authors: K
authors: Sugai
authors: Department of Pediatrics, Tenri Hospital, Tenri, Japan.
authors: Mitsuhiko
authors: M
authors: Nambu
authors: Department of Allergy and Rheumatology, Chiba Children's Hospital, Chiba, Japan.
authors: Akira
authors: A
authors: Hoshioka
authors: Department of Pediatrics, Dokkyo Medical University, Tochigi, Japan.
authors: Shigemi
authors: S
authors: Yoshihara
authors: Clinical Development, Novartis Pharma K.K., Tokyo, Japan.
authors: Norio
authors: N
authors: Sato
authors: Clinical Development, Novartis Pharma K.K., Tokyo, Japan.
authors: Noriko
authors: N
authors: Seko
authors: Department of Pediatrics, Fukuoka National Hospital, Fukuoka, Japan.
authors: Sankei
authors: S
authors: Nishima
mesh_terms: D000293:Adolescent; D001249:Asthma / Q000188:drug therapy*; D002648:Child; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D000069444:Omalizumab / Q000008:administration & dosage* / Q000009:adverse effects / Q000493:pharmacokinetics*; D012720:Severity of Illness Index
chemical_list: D000069444:Omalizumab
keywords: Childhood asthma; Efficacy; Omalizumab; Safety evaluation; Total IgE