Patient-reported outcomes · Maimonides Medical Center
What do patients with nocturia actually want treated?
Nocturia trials are built on a countable outcome: voids per night. It is easy to measure and easy to compare, and it may not be the thing patients are trying to change. A treatment that removes one void from three may or may not be a treatment that changes a life.
Status. Manuscript V3.
This hub is still being built. The analysis is underway; the derivation, tables and figures will be posted here as they are finished, the way the completed projects on this site are. What is below is the design and the current state, written up as it stands.
The question
What outcomes do patients with nocturia actually want from treatment, and how much nighttime urination will they tolerate before it matters to them?
How it is being done
- Patients define their own treatment goals rather than choosing from a fixed instrument.
- Tolerability thresholds are elicited directly — how many voids a night is acceptable — instead of inferred from symptom scores.
- Patient-defined goals are compared against the void-count endpoints trials use, to see how far apart the two actually sit.
The manuscript
Manuscript V3
With Oliver Menken. Behind a passphrase while the work is unpublished — open it here if I sent you one, or email me.
What comes next
If patient-defined goals diverge from void count, the argument follows for a goal-attainment endpoint in nocturia trials.
This page grows as the work does.