CPMs for competing risks and multi-state models

I was reading Frank’s post about CPMs and became curious about how to employ CPMs in competing risks contexts, and multi-state modeling more generally. What would the data setup be, and what would the model setup options be for:

  1. CPM for the purpose of estimating conditional cause-specific cumulative incidence functions

  2. … semi-competing risks context for marginal mean function estimation a la Ghosh-Lin

  3. … general multi-state modeling setup

Thank you.

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Please describe the ultimate goals of the analyses first, taking into account that cause-specific cumulative incidence is not an easy-to-interpret estimand. For example if you are analyzing time until cancer returns, with a competing risk from cardiovascular death, the cancer-specific cumulative incidence is the probability that cancer occurs before cardiovascular death. I’m not sure how to really digest that.

Likely not a helpful comment but for me as a clinician, the probability that a cancer recurs before cardiovascular death is an extremely relevant statistic that I would have no problem using in decision making. Most of my patients are 75 or older and most die because of CV disease long before their cancer recurs and for a large segment of the probability spectrum many would opt out of treatment. In fact many already do so on gut feeling already

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The question is whether the following would be as or more relevant as that:

  • What is the probability of getting cancer within 5y without dying from cardiovascular disease?
  • What is the probability of getting cancer or dying from CVD?
  • What is the probability of getting cancer and dying from CVD?
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@f2harrell

How would you treat cancer and cvd-death-related in CPM?

I mean, cvd and cancer are not different levels of the same outcomes. It’s not exactly what I have in mind when I think of death-illness model…

The underlying thought behind this is gaining some Quality-Adjusted Life Years that might be worth treatment-harm?

I hope that someone can think through this is more detail but my thought is that if a non-cancer death is sure to be a random event that is unrelated to the outcomes of interest, we can use an independent censoring assumption to terminate records for a patient who died from an unrelated cause at the last known time alive.

That’s a big “If” :slight_smile:

I think that both independent-censoring and enforcing different outcomes on the same scale are far from perfect heuristics - specially when facing comorbidities.

For prediction models the best heuristic I can think of is trying several approaches as a sensitivity analysis: Competing, Independent-censoring, Ordinal outcome or just plain composite.

I don’t catch the “different outcomes” reference. Censoring on a non-related outcome does not treat it as an outcome. Please elaborate. Thanks.