Personalized Medicine in Acromegaly: The ACROFAST Study.

Autor: Marques-Pamies M; Department of Endocrinology and Nutrition, Hospital Municipal de Badalona, Badalona 08911, Spain., Gil J; Endocrine Research Unit, Germans Trias i Pujol Research Institute (IGTP), Badalona 08916, Spain.; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER, Unidad 747), Instituto de Salud Carlos III (ISCIII), Barcelona 28029, Spain., Sampedro-Nuñez M; Department of Endocrinology and Nutrition, La Princesa University Hospital, Madrid 28006, Spain., Valassi E; Endocrine Research Unit, Germans Trias i Pujol Research Institute (IGTP), Badalona 08916, Spain.; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER, Unidad 747), Instituto de Salud Carlos III (ISCIII), Barcelona 28029, Spain.; Department of Endocrinology and Nutrition, Germans Trias i Pujol University Hospital, Badalona 08916, Spain., Biagetti B; Department of Endocrinology and Nutrition, Vall Hebron University Hospital, Barcelona 08035, Spain., Giménez-Palop O; Department of Endocrinology and Nutrition, Parc Taulí University Hospital, Sabadell 08208, Spain., Hernández M; Department of Endocrinology and Nutrition, Arnau de Vilanova University Hospital, Lleida 25198, Spain.; Endocrine Research Unit, Lleida Institute for Biomedical Research Dr. Pifarré Foundation (IRBLleida), Lleida 25198, Spain., Martínez S; Department Hormonal Laboratory, Germans Trias i Pujol University Hospital, Badalona 08916, Spain., Carrato C; Department of Pathology, Germans Trias i Pujol University Hospital, Badalona 08916, Spain., Villar-Taibo R; Department of Endocrinology and Nutrition, Clínico de Santiago University Hospital, Santiago de Compostela 15706, Spain., Araujo-Castro M; Department of Endocrinology and Nutrition, Ramón y Cajal University Hospital, Madrid 28034, Spain.; Instituto de Investigación Ramón y Cajal (IRYCIS), Madrid 28034, Spain., Blanco C; Department of Endocrinology and Nutrition, Príncipe de Asturias University Hospital, Madrid 28805, Spain., Simón-Muela I; Department of Endocrinology and Nutrition, Joan XXIII University Hospital, Tarragona 43005, Spain.; Endocrine Research Unit, Institut d´Investigació Sanitària Pere Virgili (IISPV), Tarragona 43005, Spain.; Rovira i Virgili University (URV), Tarragona 43003, Spain.; Endocrine Research Unit, Institut d'Investigació Biomèdica de Bellvitge (IDIBELL), Hospitalet de LLobregat 08907, Spain., Simó-Servat A; Department of Endocrinology and Nutrition, Mutua de Terrassa University Hospital, Terrassa 08221, Spain., Xifra G; Department of Endocrinology and Nutrition, Josep Trueta University Hospital, Girona 17007, Spain., Vázquez F; Department of Endocrinology and Nutrition, Germans Trias i Pujol University Hospital, Badalona 08916, Spain., Pavón I; Department of Endocrinology and Nutrition, Getafe University Hospital, Madrid 28905, Spain., Rosado JA; Department of Endocrinology and Nutrition, Getafe University Hospital, Madrid 28905, Spain., García-Centeno R; Department of Endocrinology and Nutrition, Gregorio Marañón University Hospital, Madrid 28007, Spain., Zavala R; Department of Endocrinology and Nutrition, Joan XXIII University Hospital, Tarragona 43005, Spain., Hanzu FA; Department of Endocrinology and Nutrition, Hospital Clinic University Hospital, Barcelona 08036, Spain.; Endocrine Research Unit, Institut d'Investigacions Biomèdiques August Pi I Sunyer (IDIBAPS), Barcelona 08036, Spain., Mora M; Department of Endocrinology and Nutrition, Hospital Clinic University Hospital, Barcelona 08036, Spain.; Endocrine Research Unit, Institut d'Investigacions Biomèdiques August Pi I Sunyer (IDIBAPS), Barcelona 08036, Spain., Aulinas A; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER, Unidad 747), Instituto de Salud Carlos III (ISCIII), Barcelona 28029, Spain.; Department of Endocrinology and Nutrition, Research Center for Pituitary Diseases, Institut de Recerca Sant Pau (IIB-Sant Pau), Hospital Sant Pau, Barcelona 08041, Spain., Vilarrasa N; Endocrine Research Unit, Institut d'Investigació Biomèdica de Bellvitge (IDIBELL), Hospitalet de LLobregat 08907, Spain.; Department of Endocrinology and Nutrition, Bellvitge University Hospital, Hospitalet de Llobregat 08907, Spain.; Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas (CIBERDEM), Instituto de Salud Carlos III (ISCIII), Madrid 28029, Spain., Librizzi S; Department of Endocrinology and Nutrition, 12 de Octubre University Hospital, Madrid 28041, Spain., Calatayud M; Department of Endocrinology and Nutrition, 12 de Octubre University Hospital, Madrid 28041, Spain., de Miguel P; Department of Endocrinology and Nutrition, Clínico San Carlos University Hospital, Madrid 2546, Spain., Alvarez-Escola C; Department of Endocrinology and Nutrition, La Paz University Hospital, Madrid 28046, Spain., Picó A; Department of Endocrinology and Nutrition, General University Hospital Dr Balmis, Miguel Hernández University, Alicante 03010, Spain.; Endocrine Research Unit, Instituto de Investigación Sanitaria y Biomédica de Alicante (ISABIAL), Alicante 03010, Spain., Salinas I; Department of Endocrinology and Nutrition, Germans Trias i Pujol University Hospital, Badalona 08916, Spain., Fajardo-Montañana C; Department of Endocrinology and Nutrition, La Ribera University Hospital, Valencia 46600, Spain., Cámara R; Department of Endocrinology and Nutrition, La Fe University Hospital, Valencia 46026, Spain., Bernabéu I; Department of Endocrinology and Nutrition, Clínico de Santiago University Hospital, Santiago de Compostela 15706, Spain., Jordà M; Endocrine Research Unit, Germans Trias i Pujol Research Institute (IGTP), Badalona 08916, Spain., Webb SM; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER, Unidad 747), Instituto de Salud Carlos III (ISCIII), Barcelona 28029, Spain.; Department of Endocrinology and Nutrition, Research Center for Pituitary Diseases, Institut de Recerca Sant Pau (IIB-Sant Pau), Hospital Sant Pau, Barcelona 08041, Spain.; Departament de Medicina, Universitat Autònoma de Barcelona (UAB), Bellaterra 08193, Spain., Marazuela M; Department of Endocrinology and Nutrition, La Princesa University Hospital, Madrid 28006, Spain., Puig-Domingo M; Endocrine Research Unit, Germans Trias i Pujol Research Institute (IGTP), Badalona 08916, Spain.; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER, Unidad 747), Instituto de Salud Carlos III (ISCIII), Barcelona 28029, Spain.; Department of Endocrinology and Nutrition, Germans Trias i Pujol University Hospital, Badalona 08916, Spain.; Departament de Medicina, Universitat Autònoma de Barcelona (UAB), Bellaterra 08193, Spain.
Jazyk: angličtina
Zdroj: The Journal of clinical endocrinology and metabolism [J Clin Endocrinol Metab] 2024 Dec 18; Vol. 110 (1), pp. 30-40.
DOI: 10.1210/clinem/dgae444
Abstrakt: 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.
(© The Author(s) 2024. Published by Oxford University Press on behalf of the Endocrine Society.)
Databáze: MEDLINE