Effectiveness of omalizumab (Xolair) in the first patients treated in real-life practice in France.
OBJECTIVE
To describe omalizumab (Xolair) effectiveness in the first patients treated on compassionate grounds before its commercialisation in France.


METHODS
In a historic-prospective study, data were obtained by questionnaire from the physicians whose patients had received a nominative temporary use authorisation (ATU) for omalizumab from July 2003 to January 2006. Anonymised patient data regarding demographics, asthma-related treatments and events in the year previous to the start of omalizumab treatment as well as the details of omalizumab treatment itself were obtained at inclusion. Follow-up data at more than 3 months following inclusion were also obtained and regarded asthma-related treatment (including omalizumab), events and undesirable effects suspected to be linked to omalizumab treatment.


RESULTS
Data were obtained for 147 of the 154 patients treated via ATU. 31.3% received inappropriate monthly doses of omalizumab. Of the 28 patients (19%) who discontinued for unsatisfactory therapeutic effect, 7 were treated for less than the 16 weeks recommended to evaluate efficacy and 9 who were treated for a longer period of time were underdosed. During the treatment period and compared to the previous year, patients with follow-up data at 5 months or more had experienced 62% fewer exacerbations requiring oral corticosteroids, 65% fewer emergency department visits and 29% fewer hospitalisations per year. The nature of adverse effects reported was similar to that reported in omalizumab clinical trials.


CONCLUSIONS
Results strongly suggest that omalizumab in the first patients treated in real-life setting provided a similar benefit to that observed in clinical trials. Underdosing of patients may limit this therapeutic effect.
Molimard
Mathieu
M
INSERM, Unité 657, IFR 99, Université Victor Segalen, Département de Pharmacologie, CHU de Bordeaux, 33076 Bordeaux Cedex, France. Mathieu.Molimard@pharmaco.u-bordeaux2.fr
de Blay
Frédéric
F
Didier
Alain
A
Le Gros
Vincent
V
D018927:Anti-Asthmatic Agents / Q000008:administration & dosage* / Q000009:adverse effects; D000888:Antibodies, Anti-Idiotypic; D000911:Antibodies, Monoclonal / Q000008:administration & dosage* / Q000009:adverse effects; D061067:Antibodies, Monoclonal, Humanized; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D016903:Drug Monitoring / Q000379:methods*; D005260:Female; D005602:France; D006801:Humans; D008297:Male; D008875:Middle Aged; D000069444:Omalizumab; D011446:Prospective Studies; D011795:Surveys and Questionnaires; D016896:Treatment Outcome
D018927:Anti-Asthmatic Agents; D000888:Antibodies, Anti-Idiotypic; D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D000069444:Omalizumab