دورية أكاديمية
Application of Item Response Theory to Modeling of Expanded Disability Status Scale in Multiple Sclerosis.
| العنوان: | Application of Item Response Theory to Modeling of Expanded Disability Status Scale in Multiple Sclerosis. |
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| المؤلفون: | Novakovic AM; Department of Pharmaceutical Biosciences, Uppsala University, Box 591, 751 24, Uppsala, Sweden. ana.kalezic@farmbio.uu.se., Krekels EH; Department of Pharmaceutical Biosciences, Uppsala University, Box 591, 751 24, Uppsala, Sweden.; Division of Pharmacology, Leiden Academic Centre of Drug Research, Leiden University, Leiden, Netherlands., Munafo A; Merck Institute for Pharmacometrics, Merck Serono, Lausanne, Switzerland., Ueckert S; Department of Pharmaceutical Biosciences, Uppsala University, Box 591, 751 24, Uppsala, Sweden., Karlsson MO; Department of Pharmaceutical Biosciences, Uppsala University, Box 591, 751 24, Uppsala, Sweden. |
| المصدر: | The AAPS journal [AAPS J] 2017 Jan; Vol. 19 (1), pp. 172-179. Date of Electronic Publication: 2016 Sep 15. |
| نوع المنشور: | Journal Article |
| اللغة: | English |
| بيانات الدورية: | Publisher: American Association of Pharmaceutical Scientists Country of Publication: United States NLM ID: 101223209 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1550-7416 (Electronic) Linking ISSN: 15507416 NLM ISO Abbreviation: AAPS J Subsets: MEDLINE |
| أسماء مطبوعة: | Original Publication: Arlington, Va., USA : American Association of Pharmaceutical Scientists, [2004]- |
| مواضيع طبية MeSH: | Disability Evaluation* , Models, Theoretical* , Severity of Illness Index*, Multiple Sclerosis, Relapsing-Remitting/*diagnosis, Cladribine/therapeutic use ; Clinical Trials, Phase III as Topic ; Disease Progression ; Humans ; Immunosuppressive Agents/therapeutic use ; Logistic Models ; Multiple Sclerosis, Relapsing-Remitting/drug therapy |
| مستخلص: | In this study, we report the development of the first item response theory (IRT) model within a pharmacometrics framework to characterize the disease progression in multiple sclerosis (MS), as measured by Expanded Disability Status Score (EDSS). Data were collected quarterly from a 96-week phase III clinical study by a blinder rater, involving 104,206 item-level observations from 1319 patients with relapsing-remitting MS (RRMS), treated with placebo or cladribine. Observed scores for each EDSS item were modeled describing the probability of a given score as a function of patients' (unobserved) disability using a logistic model. Longitudinal data from placebo arms were used to describe the disease progression over time, and the model was then extended to cladribine arms to characterize the drug effect. Sensitivity with respect to patient disability was calculated as Fisher information for each EDSS item, which were ranked according to the amount of information they contained. The IRT model was able to describe baseline and longitudinal EDSS data on item and total level. The final model suggested that cladribine treatment significantly slows disease-progression rate, with a 20% decrease in disease-progression rate compared to placebo, irrespective of exposure, and effects an additional exposure-dependent reduction in disability progression. Four out of eight items contained 80% of information for the given range of disabilities. This study has illustrated that IRT modeling is specifically suitable for accurate quantification of disease status and description and prediction of disease progression in phase 3 studies on RRMS, by integrating EDSS item-level data in a meaningful manner. |
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| فهرسة مساهمة: | Keywords: cladribine tablets; disease progression model; expanded disability status scale; item response theory; multiple sclerosis |
| المشرفين على المادة: | 0 (Immunosuppressive Agents) 47M74X9YT5 (Cladribine) |
| تواريخ الأحداث: | Date Created: 20160917 Date Completed: 20171128 Latest Revision: 20181113 |
| رمز التحديث: | 20231215 |
| DOI: | 10.1208/s12248-016-9977-z |
| PMID: | 27634384 |
| قاعدة البيانات: | MEDLINE |
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