Personalized Acromegaly Treatment Approach Yields Faster Hormonal Control: ACROFAST study
Spain: The prospective ACROFAST trial indicated that a tailored treatment protocol was more effective for managing acromegaly, also known as gigantism, than the conventional trial-and-error approach.
When a personalized approach was tailored using predictive biomarker testing, 78% of patients with acromegaly achieved hormonal control, compared to 53% of patients treated traditionally after one year, the researchers reported in The Journal of Clinical Endocrinology & Metabolism.
They found that after adjusting for age and sex, patients had more than double the likelihood of achieving hormonal control with the personalized approach (HR 2.53). Hormonal control was defined as normalized insulin-like growth factor 1 (IGF1) standard deviation scores.
Currently, acromegaly is medically treated through a trial-error approach using first-generation somatostatin receptor ligands (fgSRLs) as first-line drugs having an effectiveness of about 50%, and subsequent drugs are indicated through clinical judgment. Certain biomarkers have predictive capabilities for response to fgSRLs. Joan Gil, Endocrine Research Unit, Germans Trias i Pujol Research Institute (IGTP), Badalona, Spain, and colleagues report the results of the ACROFAST study, a clinical trial in which a protocol based on predictive biomarkers of fgSRLs was evaluated.
In the prospective trial involving 21 university hospitals, the effectiveness and time-to-control of two treatment protocols were compared over 12 months: A) A personalized protocol where the initial treatment option was fgSRLs either as monotherapy or in combination with pegvisomant, or pegvisomant as monotherapy, based on results from the short Acute Octreotide Test (sAOT), tumor T2 Magnetic Resonance Imaging (MRI) signal, or immunostaining for E-cadherin. B) A control group where treatment always began with fgSRLs, with additional drugs added only after inadequate control was demonstrated.
Eighty-five patients participated; 45 in the personalized and 40 in the control group.
The following were the key findings of the study:
- More patients in the personalized protocol achieved hormonal control than those in the control group (78% versus 53%).
- Survival analysis revealed a hazard ratio for achieving hormonal control adjusted by age and sex of 2.53.
- Patients from the personalized arm were controlled for a shorter period.
"Implementing personalized medicine through a straightforward protocol enables a greater number of patients to achieve control in a shorter timeframe," the researchers concluded.
Reference:
Gil, J., Valassi, E., Biagetti, B., Hernández, M., MartÃnez, S., Carrato, C., Blanco, C., Xifra, G., Vázquez, F., Pavón, I., Rosado, J. A., Zavala, R., Hanzu, F. A., Mora, M., Aulinas, A., Vilarrasa, N., Librizzi, S., Calatayud, M., De Miguel, P., . . . Marazuela, M. Personalized medicine in acromegaly: The ACROFAST study. The Journal of Clinical Endocrinology & Metabolism. https://doi.org/10.1210/clinem/dgae444
