Estimating the treatment effect from non-randomized studies: The example of reduced intensity conditioning allogeneic stem cell transplantation in hematological diseases.

Abstract : BACKGROUND: In some clinical situations, for which RCT are rare or impossible, the majority of the evidence comes from observational studies, but standard estimations could be biased because they ignore covariates that confound treatment decisions and outcomes. METHODS: Three observational studies were conducted to assess the benefit of Allo-SCT in hematological malignancies of multiple myeloma, follicular lymphoma and Hodgkin's disease. Two statistical analyses were performed: the propensity score (PS) matching approach and the inverse probability weighting (IPW) approach. RESULTS: Based on PS-matched samples, a survival benefit in MM patients treated by Allo-SCT, as compared to similar non-allo treated patients, was observed with an HR of death at 0.35 (95%CI: 0.14-0.88). Similar results were observed in HD, 0.23 (0.07-0.80) but not in FL, 1.28 (0.43-3.77). Estimated benefits of Allo-SCT for the original population using IPW were erased in HR for death at 0.72 (0.37-1.39) for MM patients, 0.60 (0.19-1.89) for HD patients, and 2.02 (0.88-4.66) for FL patients. CONCLUSION: Differences in estimated benefits rely on whether the underlying population to which they apply is an ideal randomized experimental population (PS) or the original population (IPW). These useful methods should be employed when assessing the effects of innovative treatment in non-randomized experiments.
Type de document :
Article dans une revue
BMC Blood Disorders, BioMed Central, 2012, 12 (1), pp.10. 〈10.1186/1471-2326-12-10〉
Liste complète des métadonnées

Littérature citée [58 références]  Voir  Masquer  Télécharger

http://www.hal.inserm.fr/inserm-00769142
Contributeur : Ed. Bmc <>
Soumis le : vendredi 28 décembre 2012 - 17:06:58
Dernière modification le : jeudi 15 mars 2018 - 10:12:30
Document(s) archivé(s) le : vendredi 29 mars 2013 - 03:49:59

Fichiers

Identifiants

Collections

Citation

Matthieu Resche-Rigon, Romain Pirracchio, Marie Robin, Regis De Latour, David Sibon, et al.. Estimating the treatment effect from non-randomized studies: The example of reduced intensity conditioning allogeneic stem cell transplantation in hematological diseases.. BMC Blood Disorders, BioMed Central, 2012, 12 (1), pp.10. 〈10.1186/1471-2326-12-10〉. 〈inserm-00769142〉

Partager

Métriques

Consultations de la notice

364

Téléchargements de fichiers

237