SP020HEALTH-RELATED QUALITY OF LIFE FOR PATIENTS WITH TUBEROUS SCLEROSIS COMPLEX AND ASSOCIATED MANIFESTATIONS
Notice bibliographique
Résumé
INTRODUCTION AND AIMS: Renal angiomyolipomas (AMLs), epilepsy and subependymal giant cell astrocytoma (SEGA) are manifestations of tuberous sclerosis complex (TSC) that pose an important disease burden for patients with TSC. This study assessed the health-related quality of life (HRQoL) of patients with TSC who have associated AML, epilepsy and/or SEGA manifestations. METHODS: Patients with TSC managed in a TSC specialty center at the University Medical Center Utrecht (UMCU), Netherlands from 1990-2015 had their HRQoL measured in 2012 via a generic Health Utility Index (HUI), with a 66% response rate (242 respondents / 369 patients with TSC, with or without associated manifestations). The HUI evaluates 8 dimensions of health status including vision, hearing, speech, ambulation, dexterity, emotion, cognition and pain (HUI version 3). HUI values range from −0.371 to 1, with 0 corresponding to death and 1 to perfect health. Differences of 0.03 or greater in mean HUI score are generally considered clinically relevant. Mean HUI score and corresponding 95% confidence intervals (CIs) are reported for patients with different TSC manifestations and for additional subgroups of interest. Subgroups were identified based on the information collected from the patients’ medical charts at the UMCU stored in the TSC-U database. RESULTS: The study sample included 216 patients with TSC and associated manifestations that had completed the HUI. Median age was 39.0 years (92% adults), 113 (52%) were male, and all patients were Caucasian. Most patients with TSC manifestations had two or more different manifestations (156/216; 72%). TSC-AML was observed in 194 patients, TSC-epilepsy in 168 patients, and TSC-SEGA in 43 patients. Patients with multiple TSC manifestations appeared to have lower HRQoL scores than those with a single AML manifestation (mean HUI scores 0.31 to 0.33 vs. 0.41 to 0.78; Table). Epilepsy manifestations of TSC and epilepsy-associated conditions appeared to be associated with the greatest burden on the patient’s HRQoL vs. AML only: mean HUI score 0.41 for patients with epilepsy only, −0.15 for patients with TSC-epilepsy and paralysis, 0.16 for patients with TSC-epilepsy and low and very low intelligence coefficient, and 0.25 for patients with TSC-epilepsy and autism (vs. 0.78 for AML only; Table). Patients with TSC-epilepsy and prior use of antiepileptic drugs and patients with TSC-SEGA also had low HRQoL scores (0.25 and 0.41, respectively; Table). CONCLUSIONS: Most subgroups of patients with TSC in this study had mean HUI scores below the 0.80 HUI reference score for the US for non-Hispanic non-Black general adult population (Health Utilities, Inc) and also below the 0.67 HUI score previously reported among Canadian patients with both cancer and diabetes (Bowker et al. 2006). Scores for the subgroup of patients with both TSC and epilepsy were especially very low, with the exception of the subgroup that had prior epilepsy surgery. Identifying treatments to delay or prevent the progression of these TSC-related manifestations could improve the HRQoL of patients with TSC. SP020 Table Mean HUI scores in pts with different TSC manifestations and for additional subgroups of interest
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Comment cette classification a été obtenuedéplier
Prédiction distillée sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Apprise à partir de 10 348 étiquettes directes de Codex et de 10 348 étiquettes directes de Gemma. Le mode candidate est l'union des têtes enseignantes seuillées; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont ni des étiquettes humaines ni des étiquettes directes de modèles de pointe.
Scores Codex et Gemma par catégorie
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,001 | 0,000 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,001 | 0,000 |
| Bibliométrie | 0,000 | 0,000 |
| Études des sciences et des technologies | 0,001 | 0,000 |
| Communication savante | 0,000 | 0,000 |
| Science ouverte | 0,000 | 0,000 |
| Intégrité de la recherche | 0,000 | 0,000 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,000 | 0,000 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule tête enseignante, pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».