Personalized Medicine in Acromegaly: The ACROFAST Study.

Area of Science:

• Endocrinology
• Oncology
• Pharmacogenomics

Background:

• Acromegaly treatment relies on a trial-and-error approach with first-generation somatostatin receptor ligands (fgSRLs), achieving ~50% effectiveness.
• Predictive biomarkers for fgSRLs response exist, but are underutilized in clinical practice.

Purpose of the Study:

• To evaluate a novel clinical trial protocol (ACROFAST) utilizing predictive biomarkers to personalize acromegaly treatment.
• To compare the effectiveness and time-to-control of a personalized protocol versus a standard care protocol.

Main Methods:

• A prospective trial involving 21 university hospitals compared two 12-month treatment protocols.
• Protocol A (personalized): treatment selection based on short acute octreotide test (sAOT), MRI T2 signal, or E-cadherin immunostaining.
• Protocol B (control): standard treatment initiation with fgSRLs, with other drugs added upon inadequate control.

Main Results:

• Eighty-five patients were enrolled (45 personalized, 40 control).
• Higher hormonal control rates were observed in the personalized group (78%) versus the control group (53%, P < .05).
• The personalized protocol led to achieving hormonal control in a significantly shorter time (P = .01).

Conclusions:

• Personalized medicine for acromegaly is feasible with a simple, biomarker-guided protocol.
• This approach increases the number of patients achieving hormonal control and reduces the time to achieve it.