Outcomes of clinical utility in amyloid-PET studies: state of art and future perspectives.

PURPOSE: To review how outcomes of clinical utility are operationalized in current amyloid-PET validation studies, to prepare for formal assessment of clinical utility of amyloid-PET-based diagnosis. METHODS: Systematic review of amyloid-PET research studies published up to April 2020 that included outcomes of clinical utility. We extracted and analyzed (a) outcome categories, (b) their definition, and (c) their methods of assessment. RESULTS: Thirty-two studies were eligible. (a) Outcome categories were clinician-centered (found in 25/32 studies, 78%), patient-/caregiver-centered (in 9/32 studies, 28%), and health economics-centered (5/32, 16%). (b) Definition: Outcomes were mainly defined by clinical researchers; only the ABIDE study expressly included stakeholders in group discussions. Clinician-centered outcomes mainly consisted of incremental diagnostic value (25/32, 78%) and change in patient management (17/32, 53%); patient-/caregiver-centered outcomes considered distress after amyloid-pet-based diagnosis disclosure (8/32, 25%), including quantified burden of procedure for patients' outcomes (n = 8) (1/8, 12.5%), impact of disclosure of results (6/8, 75%), and psychological implications of biomarker-based diagnosis (75%); and health economics outcomes focused on costs to achieve a high-confidence etiological diagnosis (5/32, 16%) and impact on quality of life (1/32, 3%). (c) Assessment: all outcome categories were operationalized inconsistently across studies, employing 26 different tools without formal rationale for selection. CONCLUSION: Current studies validating amyloid-PET already assessed outcomes for clinical utility, although non-clinician-based outcomes were inconsistent. A wider participation of stakeholders may help produce a more thorough and systematic definition and assessment of outcomes of clinical utility and help collect evidence informing decisions on reimbursement of amyloid-PET.

Caregivers' perceptions of the therapeutic benefits of drug treatments for dementia.

BACKGROUND AND AIMS: To collect opinions, perceptions, and expectations on the therapeutic benefits of drug treatment for dementia and the impact on the care of the patient in a large sample of caregivers. Only few studies deal with this topic in a small number of participants. METHODS: This used an ad-hoc online questionnaire to collect the opinions of caregivers of patients with dementia and assess their expectations and perceptions of the therapeutic benefits of drug treatments. The questionnaire was accessible for nearly 4 months on the Federazione Alzheimer Italia website and had three sections: (1) information on the patient with dementia; (2) information on the caregiver's perception of the therapeutic benefits of drug treatments; (3) information on caregivers. To evaluate the relationship between the caregiver's expectations of the therapeutic benefits of dementia treatments and some characteristics of the patients and the caregivers, we used the Chi-square test. RESULTS: During the access time, 439 questionnaires were filled, and 369 were validated for inclusion in the analysis; of these, 329 also had information on caregivers. The expectations of drug treatment effects were not statistically significantly influenced by any variables considered about the patients or the caregivers. Caregivers' beliefs about the effectiveness of dementia treatment, their expectations and changes in their lives were clear. CONCLUSIONS: This study addresses a gap in knowledge about caregivers' experiences and their views of drug treatments, and highlights the need for a pharmaceutical treatment that helps to resolve the symptoms and outcomes of dementia.