A language-based sum score for the course and therapeutic intervention in primary progressive aphasia

TY - JOUR

T1 - A language-based sum score for the course and therapeutic intervention in primary progressive aphasia

AU - Semler, Elisa

AU - Anderl-Straub, Sarah

AU - Uttner, Ingo

AU - Diehl-Schmid, Janine

AU - Danek, Adrian

AU - Einsiedler, Beate

AU - Fassbender, Klaus

AU - Fliessbach, Klaus

AU - Huppertz, Hans-Jürgen

AU - Jahn, Holger

AU - Kornhuber, Johannes

AU - Landwehrmeyer, Bernhard

AU - Lauer, Martin

AU - Muche, Rainer

AU - Prudlo, Johannes

AU - Schneider, Anja

AU - Schroeter, Matthias L

AU - Ludolph, Albert C

AU - Otto, Markus

AU - FTLD consortium

PY - 2018/4/25

Y1 - 2018/4/25

N2 - BACKGROUND: With upcoming therapeutic interventions for patients with primary progressive aphasia (PPA), instruments for the follow-up of patients are needed to describe disease progression and to evaluate potential therapeutic effects. So far, volumetric brain changes have been proposed as clinical endpoints in the literature, but cognitive scores are still lacking. This study followed disease progression predominantly in language-based performance within 1 year and defined a PPA sum score which can be used in therapeutic interventions.METHODS: We assessed 28 patients with nonfluent variant PPA, 17 with semantic variant PPA, 13 with logopenic variant PPA, and 28 healthy controls in detail for 1 year. The most informative neuropsychological assessments were combined to a sum score, and associations between brain atrophy were investigated followed by a sample size calculation for clinical trials.RESULTS: Significant absolute changes up to 20% in cognitive tests were found after 1 year. Semantic and phonemic word fluency, Boston Naming Test, Digit Span, Token Test, AAT Written language, and Cookie Test were identified as the best markers for disease progression. These tasks provide the basis of a new PPA sum score. Assuming a therapeutic effect of 50% reduction in cognitive decline for sample size calculations, a number of 56 cases is needed to find a significant treatment effect. Correlations between cognitive decline and atrophy showed a correlation up to r = 0.7 between the sum score and frontal structures, namely the superior and inferior frontal gyrus, as well as with left-sided subcortical structures.CONCLUSION: Our findings support the high performance of the proposed sum score in the follow-up of PPA and recommend it as an outcome measure in intervention studies.

AB - BACKGROUND: With upcoming therapeutic interventions for patients with primary progressive aphasia (PPA), instruments for the follow-up of patients are needed to describe disease progression and to evaluate potential therapeutic effects. So far, volumetric brain changes have been proposed as clinical endpoints in the literature, but cognitive scores are still lacking. This study followed disease progression predominantly in language-based performance within 1 year and defined a PPA sum score which can be used in therapeutic interventions.METHODS: We assessed 28 patients with nonfluent variant PPA, 17 with semantic variant PPA, 13 with logopenic variant PPA, and 28 healthy controls in detail for 1 year. The most informative neuropsychological assessments were combined to a sum score, and associations between brain atrophy were investigated followed by a sample size calculation for clinical trials.RESULTS: Significant absolute changes up to 20% in cognitive tests were found after 1 year. Semantic and phonemic word fluency, Boston Naming Test, Digit Span, Token Test, AAT Written language, and Cookie Test were identified as the best markers for disease progression. These tasks provide the basis of a new PPA sum score. Assuming a therapeutic effect of 50% reduction in cognitive decline for sample size calculations, a number of 56 cases is needed to find a significant treatment effect. Correlations between cognitive decline and atrophy showed a correlation up to r = 0.7 between the sum score and frontal structures, namely the superior and inferior frontal gyrus, as well as with left-sided subcortical structures.CONCLUSION: Our findings support the high performance of the proposed sum score in the follow-up of PPA and recommend it as an outcome measure in intervention studies.

KW - Journal Article

U2 - 10.1186/s13195-018-0345-3

DO - 10.1186/s13195-018-0345-3

M3 - SCORING: Journal article

C2 - 29695300

VL - 10

SP - 41

JO - ALZHEIMERS RES THER

JF - ALZHEIMERS RES THER

SN - 1758-9193

IS - 1

ER -