Moving forward from spleen response as an endpoint in randomized controlled trials in myelofibrosis | Haematologica

Cancer research · Myelofibrosis

We argue that there is no convincing evidence for the use of spleen response as a surrogate for clinical outcome in MF and that use of surrogate endpoints in RCT for MF should be avoided altogether.

Anticancer drugs should make patients live longer and/or feel better. Ideally, endpoints of cancer randomized controlled trials (RCT) should demonstrate that a drug leads to an increase in overall survival and/or improvement in quality of life. With the aim of including smaller numbers of patients, running shorter trials and thus getting new drugs to patients faster, cancer RCT are increasingly using (putative) surrogate endpoints. However, changes in surrogate endpoints often do not reliably predict improvements in overall survival and/or quality of life. ... In myelofibrosis, spleen response has extensively been used as a surrogate for clinical outcome. In this review we argue that there is no convincing evidence for the use of spleen response or other surrogate endpoints in myelofibrosis, and that the use of surrogate endpoints in RCT in myelofibrosis should be avoided altogether.

Spleen response is widely adopted as a primary endpoint in myelofibrosis trials, yet it has not been validated at the trial level to predict overall survival or quality of life, potentially leading to accelerated approval of therapies that may not actually improve patient survival or well‑being.

Evidence level: Feltételezés / hírjelzés. Nincs önálló tudományos bizonyíték.

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