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Immunotherapy: clinical trials – optimal trial and clinical outcomes

Bousquet, Philippe Jean; Demoly, Pascal; Passalacqua, Giovanni; Canonica, G Walter; Bousquet, Jean

Current Opinion in Allergy and Clinical Immunology: December 2007 - Volume 7 - Issue 6 - p 561–566
doi: 10.1097/ACI.0b013e3282f1d6a4
Immunotherapy: Edited by David Broide and Giovanni Passalacqua

Purpose of review The methodology of randomized clinical trials is essential to assess and register treatment interventions. Although specific immunotherapy was introduced a century ago, very few trials meet the criteria for pivotal studies. The paper reviews the methods which should be used to investigate specific immunotherapy.

Recent findings Large pivotal trials on specific immunotherapy were published within the last year and made sublingual immunotherapy an accepted treatment in Europe. Moreover, a study proposed the guidelines for conducting trials on immunotherapy. Phase I studies were also published on sublingual immunotherapy. A large trial on subcutaneous immunotherapy confirmed its efficacy, but showed that this form of treatment is associated with side effects.

Summary The clinical efficacy of subcutaneous and sublingual immunotherapy with pollen and mites is well established for both rhinitis and asthma, but the methodology of many specific immunotherapy trials was found to be insufficient until recent large pivotal studies. The present paper reviews the requirements for conducting trials in allergen-specific immunotherapy including allergen standardization, patient inclusion and exclusion criteria, phase I trials to assess safety, dose-ranging studies, and pivotal trials. These need to be randomized, parallel group, placebo-controlled designs. The number of patients should be sufficient. Primary and secondary outcome measures are listed. Trials for asthma need specific requirements. In all trials, safety should be carefully monitored. Studies in children are required.

aCHU Montpellier, Montpellier, France

bDIMI, University of Genoa, Genoa, Italy

Correspondence to Professor Jean Bousquet, Clinique des Maladies Respiratoires, Hôpital Arnaud de Villeneuve, Centre Hospitalier Universitaire, 34295 Montpellier Cedex 5, France Tel: +33 4 67 33 61 05; fax: +33 4 67 04 27 08; e-mail:

All authors are members of the GA2LEN (Global Allergy and Asthma European Network), supported by the EU Framework Programme for Research (contract FOOD-CT-2004-506378).

Copyright © 2007 Wolters Kluwer Health, Inc. All rights reserved.