Real-World Data on the Efficacy and Safety of Osilodrostat in Patients with Cushing’s Disease in Spain
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| Publicat a: | Journal of Clinical Medicine vol. 14, no. 21 (2025), p. 7575-7588 |
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| Autor principal: | |
| Altres autors: | , , , , , , , , , , , , , , , , , , , , , , , , , , , , , |
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MDPI AG
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| Accés en línia: | Citation/Abstract Full Text + Graphics Full Text - PDF |
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| 001 | 3271046339 | ||
| 003 | UK-CbPIL | ||
| 022 | |a 2077-0383 | ||
| 024 | 7 | |a 10.3390/jcm14217575 |2 doi | |
| 035 | |a 3271046339 | ||
| 045 | 2 | |b d20250101 |b d20251231 | |
| 100 | 1 | |a Araujo-Castro, Marta |u Endocrinology & Nutrition Department, Hospital Universitario Ramón y Cajal & Instituto de Investigación Biomédica Ramón y Cajal (IRYCIS), Colmenar Viejo Street km 9, 28034 Madrid, Spain | |
| 245 | 1 | |a Real-World Data on the Efficacy and Safety of Osilodrostat in Patients with Cushing’s Disease in Spain | |
| 260 | |b MDPI AG |c 2025 | ||
| 513 | |a Journal Article | ||
| 520 | 3 | |a Objective: To evaluate the efficacy and safety of osilodrostat in patients with Cushing’s disease (CD). Methods: A retrospective, multicenter, real-world study of patients with CD. The main efficacy endpoint was the proportion of patients who were complete responders (urinary free cortisol [UFC] < the upper limit of normal and/or with adrenal insufficiency development). Results: Thirty-seven CD patients were enrolled. There were 33 patients who initially received osilodrostat in monotherapy and 4 in combination. However, 3 patients of the monotherapy group were switched to combination therapy. The median duration of osilodrostat treatment was 5 months (range 1–93). All the patients were classified as responders: 33 (89.2%) had complete response and 4 partial response. A positive correlation was detected between the percentage of UFC decrease and the maximum (r = 0.481, p = 0.006) and the maintenance doses (r = 0.440, p = 0.011). The initial doses of osilodrostat were a predictor of complete response (vs. partial) (Odds ratio [OR] 2.82, p = 0.030). The median time to UFC normalization in the group of complete responders was 4 weeks (range 1–20) and UFC normalized before or at month 1 in 67% (n = 20/30) of the patients. Osilodrostat led to a significant decrease in systolic and diastolic blood pressure in parallel with a reduction of antihypertensive medications. Conclusions: Osilodrostat leads to a complete UFC normalization in up to 90% of the patients with CD, in parallel with an improvement in the cardiometabolic profile. A proper titration of osilodrostat is important to achieve a complete response since a positive correlation between the doses and the UFC reduction was observed. | |
| 651 | 4 | |a Spain | |
| 653 | |a Patients | ||
| 653 | |a Variables | ||
| 653 | |a Quality of life | ||
| 653 | |a Combination therapy | ||
| 653 | |a Hormones | ||
| 653 | |a Surgery | ||
| 653 | |a Sinuses | ||
| 653 | |a Drug dosages | ||
| 653 | |a Statistical analysis | ||
| 700 | 1 | |a García-Centeno Rogelio |u Endocrinology &amp; Nutrition Department, Hospital Universitario Gregorio Marañón, 28007 Madrid, Spain | |
| 700 | 1 | |a González, Laura |u Endocrinology &amp; Nutrition Department, Hospital Universitario Gregorio Marañón, 28007 Madrid, Spain | |
| 700 | 1 | |a Hanzu, Felicia A |u Endocrinology &amp; Nutrition Department, Hospital Clinic de Barcelona, 08036 Barcelona, Spain | |
| 700 | 1 | |a Orois Aida |u Endocrinology &amp; Nutrition Department, Hospital Clinic de Barcelona, 08036 Barcelona, Spain | |
| 700 | 1 | |a Camara, Rosa |u Endocrinology &amp; Nutrition Department, Hospital La Fe, 46600 Valencia, Spain | |
| 700 | 1 | |a García María Dolores Ollero |u Endocrinology &amp; Nutrition Department, Hospital Universitario Navarra, 31008 Pamplona, Spain | |
| 700 | 1 | |a Echarri Ana Irigaray |u Endocrinology &amp; Nutrition Department, Hospital Universitario Navarra, 31008 Pamplona, Spain | |
| 700 | 1 | |a Gracia Gimeno Paola |u Endocrinology &amp; Nutrition Department, Hospital Universitario Royo Villanova, 50015 Zaragoza, Spain | |
| 700 | 1 | |a Pascual-Corrales Eider |u Endocrinology &amp; Nutrition Department, Hospital Universitario Ramón y Cajal &amp; Instituto de Investigación Biomédica Ramón y Cajal (IRYCIS), Colmenar Viejo Street km 9, 28034 Madrid, Spain | |
| 700 | 1 | |a Biagetti Betina |u Endocrinology &amp; Nutrition Department, Hospital Universitario Vall de Hebrón &amp; CIBERER Group 747, 08035 Barcelona, Spain | |
| 700 | 1 | |a Cardona Andrés |u Endocrinology &amp; Nutrition Department, Hospital Universitario Vall de Hebrón &amp; CIBERER Group 747, 08035 Barcelona, Spain | |
| 700 | 1 | |a González Molero Inmaculada |u Endocrinology &amp; Nutrition Department, Hospital Regional Universitario de Málaga, 29010 Málaga, Spain | |
| 700 | 1 | |a Simo-Servat Andreu |u Endocrinology &amp; Nutrition Department, Hospital Regional Universitario de Terrasa, 08227 Barcelona, Spain | |
| 700 | 1 | |a Guerrero Pérez Fernando |u <label>10</label>Endocrinology &amp; Nutrition Department, Hospital Universitario de Bellvitge, L’Hospitalet de Llobregat, 08907 Cataluña, Spain | |
| 700 | 1 | |a Villar-Taibo Rocío |u <label>11</label>Endocrinology &amp; Nutrition Department, Hospital Universitario de Santiago de Compostela, 15706 Galicia, Spain | |
| 700 | 1 | |a Bernabéu Ignacio |u <label>11</label>Endocrinology &amp; Nutrition Department, Hospital Universitario de Santiago de Compostela, 15706 Galicia, Spain | |
| 700 | 1 | |a Fajardo-Montañana Carmen |u <label>12</label>Endocrinology &amp; Nutrition Department, Hospital Universitario La Ribera, 46600 Valencia, Spain | |
| 700 | 1 | |a Novo-Rodríguez, Cristina |u <label>13</label>Endocrinology &amp; Nutrition Department, Hospital Universitario Virgen de las Nieves, 18014 Granada, Spain | |
| 700 | 1 | |a Tenorio, Jimenéz Carmen |u <label>13</label>Endocrinology &amp; Nutrition Department, Hospital Universitario Virgen de las Nieves, 18014 Granada, Spain | |
| 700 | 1 | |a Calatayud María |u <label>14</label>Endocrinology &amp; Nutrition Department, Hospital Universitario 12 de Octubre, 28041 Madrid, Spain | |
| 700 | 1 | |a Moure Rodríguez María Dolores |u <label>15</label>Endocrinology &amp; Nutrition Department, Hospital Universitario de Cruces, 48903 Bilbao, Spain | |
| 700 | 1 | |a Cordido Fernando |u <label>16</label>Endocrinology &amp; Nutrition Department, Hospital Universitario A Coruña &amp; Universidad de A Coruña, 15006 A Coruña, Spain | |
| 700 | 1 | |a Castro, Ana |u <label>17</label>Endocrinology &amp; Nutrition Department, Hospital Universitario Toledo, 45007 Toledo, Spain | |
| 700 | 1 | |a Valero Lucía Manzano |u <label>17</label>Endocrinology &amp; Nutrition Department, Hospital Universitario Toledo, 45007 Toledo, Spain | |
| 700 | 1 | |a Paja Fano Miguel |u <label>18</label>Endocrinology &amp; Nutrition Department, OSI Bilbao-Basurto, Hospital Universitario de Basurto, 48013 Bilbao, Spain | |
| 700 | 1 | |a Goi Jessica |u Endocrinology &amp; Nutrition Department, Hospital Universitario Ramón y Cajal &amp; Instituto de Investigación Biomédica Ramón y Cajal (IRYCIS), Colmenar Viejo Street km 9, 28034 Madrid, Spain | |
| 700 | 1 | |a Aulinas, Anna |u <label>20</label>Endocrinology &amp; Nutrition Department, Hospital de la Santa Creu i Sant Pau, IR-SANT PAU, CIBERER U747 (ISCIII), 08025 Barcelona, Spain | |
| 700 | 1 | |a Abellán Pablo |u <label>21</label>Endocrinology &amp; Nutrition Department, Hospital Universitario de Valencia, 46010 Valencia, Spain | |
| 700 | 1 | |a Iglesias, Pedro |u <label>22</label>Endocrinology &amp; Nutrition Department, Hospital Universitario Puerta de Hierro, 28222 Madrid, Spain | |
| 700 | 1 | |a Soto-Moreno, Alfonso |u <label>23</label>Unidad de Gestión de Endocrinología y Nutrición, Instituto de Biomedicina de Sevilla (IBiS), Hospital Universitario Virgen del Rocío/CSIC/Universidad de Sevilla, 14004 Córdoba, Spain | |
| 773 | 0 | |t Journal of Clinical Medicine |g vol. 14, no. 21 (2025), p. 7575-7588 | |
| 786 | 0 | |d ProQuest |t Health & Medical Collection | |
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