医疗专业人员

Feb. 24, 2023

An Alzheimer's Association work group has reassessed what to consider clinically meaningful in trials of therapies to slow progression of Alzheimer's disease.

The group's findings and recommendations are published online in Alzheimer's & Dementia: The Journal of the Alzheimer's Association and include:

• Slowing rather than halting disease progression has measurable and meaningful benefits for patients and their families, especially in early Alzheimer's disease.
• A statistically significant benefit in an 18-month clinical trial may signify and lead to an even more meaningful change when projected over succeeding years.
• Any single intervention on dementia-related brain changes is unlikely to have a large clinical effect on its own.

"The work group recognized that we need to modify expectations of a single intervention on a complex set of neurodegenerative processes," says Ronald C. Petersen, M.D., Ph.D., a neurologist at Mayo Clinic in Rochester, Minnesota, and lead author of the journal article. "However, recent progress with newly approved Alzheimer's drugs is a tremendous first step."

The Alzheimer's Association convened the work group as new clinical data are expected soon from several studies of emerging therapeutics. Interpretation of clinical meaningfulness will be part of discussions clinicians will have with patients and their families when they are considering potential benefits, risks and challenges of disease-modifying therapies newly approved to treat Alzheimer's disease.

"As we discuss the clinical meaningfulness of slowing the Alzheimer's disease progression, realistic expectations of benefits and risks must be conveyed," Dr. Petersen says. "But slowing brain deterioration even 4 to 6 months in the early stages of Alzheimer's disease may lead to preservation of function for patients that can be very meaningful to them and their families."

For more information

Petersen RC, et al. Expectations and clinical meaningfulness of randomized controlled trials. Alzheimer's & Dementia: The Journal of the Alzheimer's Association. In press.

Refer a patient to Mayo Clinic.