Artigo Acesso aberto Revisado por pares

Personalized Medicine in Acromegaly: The ACROFAST Study

2024; Oxford University Press; Linguagem: Inglês

10.1210/clinem/dgae444

ISSN

1945-7197

Autores

Montserrat Marqués-Pamies, Joan Gil, Miguel Sampedro‐Núñez, Elena Valassi, Betina Biagetti, Olga Giménez‐Palop, Marta Hernández, Silvia Martínez, Cristina Carrato, Rocío Villar-Taibo, Marta Araujo‐Castro, Concepción Blanco, Inmaculada Simón‐Muela, Andreu Simó-Servat, Gemma Xifra, Federico Vázquez, Isabel Pavón, José Rosado, Rogelio García-Centeno, Roxana Zavala, Felicia A. Hanzu, Mireia Mora, Anna Aulinas, Núria Vilarrasa, Soledad Librizzi, María Calatayud, Paz de Miguel, Cristina Álvarez‐Escolá, Antonio Picó, Isabel Salinas, Carmen Fajardo, Rosa Cámara, Ignacio Bernabéu, Mireia Jordà, Susan M. Webb, Mónica Marazuela, Manel Puig‐Domingo,

Tópico(s)

Adrenal and Paraganglionic Tumors

Resumo

Abstract Context Medical treatment of acromegaly is currently performed through a trial-and-error approach using first-generation somatostatin receptor ligands (fgSRLs) as first-line drugs, with an effectiveness of about 50%, and subsequent drugs are indicated through clinical judgment. Some biomarkers can predict fgSRLs response. Objective Here we report the results of the ACROFAST study, a clinical trial in which a protocol based on predictive biomarkers of fgSRLs was evaluated. Methods This was a prospective trial (21 university hospitals) comparing the effectiveness and time-to-control of 2 treatment protocols during 12 months: (A) a personalized protocol in which the first options were fgSRLs as monotherapy or in combination with pegvisomant, or pegvisomant as monotherapy depending on the short acute octreotide test (sAOT) results, tumor T2 magnetic resonance (MRI) signal or immunostaining for E-cadherin; and (B) a control group with treatment always started by fgSRLs and the other drugs included after demonstrating inadequate control. Results Eighty-five patients participated; 45 in the personalized and 40 in the control group. More patients in the personalized protocol achieved hormonal control compared to those in the control group (78% vs 53%, P < .05). Survival analysis revealed a hazard ratio for achieving hormonal control adjusted by age and sex of 2.53 (CI, 1.30-4.80). Patients from the personalized arm were controlled in a shorter period of time (P = .01). Conclusion Personalized medicine is feasible using a relatively simple protocol, and it allows a higher number of patients to achieve control in a shorter period of time.

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