Dr Ruben Mesa on the Importance of Gauging Quality of Life in MPNs

Quality of life is an important end point in clinical trials, and it is important to discuss that with regulatory agencies, said Ruben Mesa, MD, director of UT Health San Antonio MD Anderson Cancer Center.

For myeloproliferative neoplasms, why is quality of life not used as much as an end point?
Quality of life as an end point is certainly something that's evolving in cancer and in the blood diseases overall. In myeloproliferative neoplasms [MPNs], in many ways they've now become a leader in this regard. When we started this journey back in the mid-2000s, before the first testing with the JAK inhibitors, [quality of life was] pretty much was not looked at it in any disease. Now, we had worked to develop the instruments, the myelofibrosis symptom assessment form, as well as the MPN symptom assessment form that now have become the standard particularly for phase 3 clinical trials.

Now, usually the end point that's included in studies is actual symptom improvement. There have been much discussion with the regulatory bodies regarding the different between the 2, but at least now, actual very specific symptoms, such as itching, such as night sweats, you know can now be quantified at least in a subjective way with a validated instruments with improvement, then potentially as an end point.

There's even studies now that have it as either a coprimary, or even a primary end point. Quality of Life, I think is a very important secondary end point. Part of even health-related quality of life has many aspects, which include in financial toxicity and other things that perhaps are still more nuanced. But I think they are important, without question discussions with the regulatory agencies that are very sensitive to clinical benefit as being important for patients, and quality of life, of course, is an important distillation of clinical benefit.
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