@article{MTMT:34999595, title = {Acromegaly Disease Control Maintained After Switching From Injected Somatostatin Receptor Ligands to Oral Paltusotine.}, url = {https://m2.mtmt.hu/api/publication/34999595}, author = {Gadelha, Mônica R and Casagrande, Alessandra and Strasburger, Christian J and Bidlingmaier, Martin and Snyder, Peter J and Guitelman, Mirtha A and Boguszewski, Cesar L and Buchfelder, Michael and Shimon, Ilan and Raverot, Gerald and Tóth, Miklós and Mezősi, Emese and Doknic, Mirjana and Fan, Xiaolin and Clemmons, David and Trainer, Peter J and Struthers, R Scott and Krasner, Alan and Biller, Beverly M K}, doi = {10.1210/clinem/dgae385}, journal-iso = {J CLIN ENDOCR METAB}, journal = {JOURNAL OF CLINICAL ENDOCRINOLOGY AND METABOLISM}, volume = {110}, unique-id = {34999595}, issn = {0021-972X}, abstract = {Paltusotine is a nonpeptide selective somatostatin receptor 2 agonist in development as once-daily oral treatment for acromegaly.To evaluate the efficacy and safety of paltusotine in the treatment of patients with acromegaly previously controlled with injected somatostatin receptor ligands (SRLs).This phase 3, randomized, double-blind, placebo-controlled trial enrolled adults with acromegaly who had insulin-like growth factor I (IGF-I) ≤1.0 times the upper limit of normal (×ULN) while receiving a stable dose of depot octreotide or lanreotide. Patients were switched from injected SRLs and randomized to receive paltusotine or placebo orally for 36 weeks. The primary endpoint was proportion of patients maintaining IGF-I ≤1.0×ULN. Secondary endpoints were change in IGF-I level, change in Acromegaly Symptom Diary (ASD) score, and maintenance of mean 5-sample growth hormone (GH) <1.0 ng/mL.The primary endpoint was met: 83.3% (25/30) of patients receiving paltusotine and 3.6% (1/28) receiving placebo maintained IGF-I ≤1.0×ULN (odds ratio: 126.53; 95% CI: 13.73, >999.99; P<.0001). Paltusotine was also superior to placebo for all secondary endpoints: mean (±SE) change in IGF-I of 0.04±0.09×ULN versus 0.83±0.1×ULN (P<.0001); mean (±SE) change in ASD score of -0.6±1.5 versus 4.6±1.6 (P=.02); mean GH maintained at <1.0 ng/mL in 20/23 (87.0%) versus 5/18 (27.8%) patients (odds ratio: 16.61; 95% CI: 2.86, 181.36; P=.0003). The most common adverse events were acromegaly symptoms and gastrointestinal effects characteristic of SRLs.Replacement of injected SRLs by once-daily oral paltusotine was effective in maintaining both biochemical and symptom control in patients with acromegaly and was well tolerated.}, keywords = {SOMATOSTATIN; clinical trial; IGF-I; Acromegaly; somatostatin receptor 2; paltusotine}, year = {2025}, eissn = {1945-7197}, pages = {228-237}, orcid-numbers = {Tóth, Miklós/0000-0002-8701-408X; Mezősi, Emese/0000-0001-9367-3877} } @article{MTMT:35705583, title = {MALIGNUS RENINOMA}, url = {https://m2.mtmt.hu/api/publication/35705583}, author = {Bényei, Erik and Remport, Ádám and Katonai, A and Tőke, Judit and Piros, László and Illés, Anett and Nagy, P and Kósa, János and Lakatos, Péter and Tóth, Miklós}, journal-iso = {MBA}, journal = {MAGYAR BELORVOSI ARCHIVUM}, volume = {77}, unique-id = {35705583}, issn = {0133-5464}, year = {2024}, pages = {331-331}, orcid-numbers = {Piros, László/0000-0003-4841-220X; Illés, Anett/0000-0001-5351-9015; Lakatos, Péter/0000-0002-7652-3671; Tóth, Miklós/0000-0002-8701-408X} } @article{MTMT:34863484, title = {Clinical utility of S-GRAS prognostic system in adrenocortical carcinomas: confirmatory results from a single-centre institutional registry}, url = {https://m2.mtmt.hu/api/publication/34863484}, author = {Bényei, Erik and Tőke, Judit and Huszty, Gergely and Borka, Katalin and Sápi, Zoltán and Jakab, Zsuzsanna and Kiss, Gergely and Kalina, Ildikó and Reismann, Péter and Uhlyarik, Andrea and Igaz, Péter and Tóth, Miklós}, doi = {10.1530/endoabs.99.EP254}, journal-iso = {ENDOCR ABSTR}, journal = {ENDOCRINE ABSTRACTS}, volume = {99}, unique-id = {34863484}, issn = {1470-3947}, year = {2024}, eissn = {1479-6848}, orcid-numbers = {Borka, Katalin/0000-0002-8956-0770; Kalina, Ildikó/0000-0002-2647-9123; Reismann, Péter/0000-0002-2696-6132; Igaz, Péter/0000-0003-2192-554X; Tóth, Miklós/0000-0002-8701-408X} } @article{MTMT:35596775, title = {12531 Long-term Safety And Efficacy Of Once-daily Oral Paltusotine In The Treatment Of Patients With Acromegaly: Update From Acrobat Advance}, url = {https://m2.mtmt.hu/api/publication/35596775}, author = {Gadelha, M and Randeva, H S and Gordon, M B and Doknic, M and Mezosi, E and Tóth, Miklós and Boguszewski, C L and Davidson, C and Ferrara-Cook, C T and Casagrande, A and Krasner, A}, doi = {10.1210/jendso/bvae163.1269}, journal-iso = {J ENDOCRINE SOC}, journal = {JOURNAL OF THE ENDOCRINE SOCIETY}, volume = {8}, unique-id = {35596775}, abstract = {Disclosure: M. Gadelha: Consulting Fee; Self; Crinetics, Ipsen, Novo Nordisk, and Recordati. Grant Recipient; Self; Crinetics and Recordati. Speaker; Self; Ipsen, Novo Nordisk, and Recordati. H.S. Randeva: None. M.B. Gordon: Consulting Fee; Self; Crinetics Pharmaceuticals, HRA Pharma, Novo Nordisk, and Recordati Rare Diseases. Grant Recipient; Self; Ascendis, Camurus, Chiasma, Corcept, Crinetics, Ipsen, Novartis, Novo Nordisk, Opko, Pfizer, and Strongbridge. M. Doknic: Research Investigator; Self; Crinetics Pharmaceuticals. Speaker; Self; Merck, Novartis, Novo Nordisk, Pfizer, and Sandoz. E. Mezosi: Research Investigator; Self; Crinetics Pharmaceuticals. M. Toth: Consulting Fee; Self; Pfizer, Ipsen, Novo Nordisk, and Recordati. Research Investigator; Self; Crinetics Pharmaceuticals. Other; Self; Pfizer, Ipsen, Novo Nordisk, and Recordati. C.L. Boguszewski: Advisory Board Member; Self; Novo Nordisk and Recordati. Consulting Fee; Self; Ipsen, Novo Nordisk, and Recordati. Other; Self; Ipsen, Novo Nordisk, and Recordati. C. Davidson: Employee; Self; Crinetics Pharmaceuticals. C.T. Ferrara-Cook: Employee; Self; Crinetics Pharmaceuticals. A. Casagrande: Employee; Self; Crinetics Pharmaceuticals. A. Krasner: Employee; Self; Crinetics Pharmaceuticals.Paltusotine is a non-peptide, highly selective SST2 receptor agonist in development as a once-daily, oral treatment for patients with acromegaly or carcinoid syndrome. ACROBAT Advance is an ongoing, 6-year, single-arm, open-label extension study of paltusotine in the treatment of patients with acromegaly. Interim results as the first enrolled patients approach 4 years of treatment are reported here. Enrolled patients had completed either the ACROBAT Edge or Evolve phase 2 parent studies. In Edge, at enrollment all patients were candidates for combination drug therapy: either sub-optimally controlled on an injected SRL (octreotide or lanreotide) alone or in combination with cabergoline, or required combination therapy or pasireotide to achieve normal IGF-I levels. In Evolve, enrolled patients had normal IGF-I levels on injected SRL monotherapy. When the Advance study was initiated, paltusotine was formulated as a capsule (dose range, 10-40 mg); all patients were switched to the tablet formulation (dose range, 20-60 mg) during the third year of the study. As of this analysis, all patients had at least 2 assessments after switching to tablet formulation. Adjunctive treatment with cabergoline or pegvisomant was allowed in patients who did not attain normal IGF-I levels on the maximum dose of paltusotine. Forty-three patients were enrolled in Advance (Edge, n=32; Evolve, n=11; 88% of eligible patients): at baseline, mean (±SD) age 53.0±11.6 years, 56% female, 86% previous pituitary surgery, and none had prior radiotherapy. IGF-I control in Edge and Evolve subsets remained stable at parent study baseline values. For all patients pooled, median (IQR) IGF-I levels were 1.15× ULN (0.84, 1.46; n=43) at parent study baseline; in Advance, 1.14× ULN (0.89, 1.29; n=40), 1.06× ULN (0.87, 1.24; n=35), and 1.08× ULN (0.87, 1.57; n=10) at months 12, 24, and 42, respectively. As expected, patients receiving adjunctive medication during Advance largely derived from the Edge study. Acromegaly symptoms, as measured using the patient-reported Acromegaly Symptom Diary (score range, 0-70; higher values indicate greater symptom burden), were stably controlled: median (IQR) score of 8.6 (3.6, 20.1; n=21) at parent study baseline; in Advance, 10.5 (5.0, 18.5; n=40), 10.0 (5.0, 25.0; n=34), and 13.5 (6.0, 22.0; n=10) at months 12, 24, and 42, respectively. The most common AEs reported through month 42 were arthralgia (37.2%), headache (30.2%), and fatigue (23.3%). One serious drug-related AE (cholelithiasis) was reported. Of the 8 patients who discontinued the study, 2 were due to AEs (mild or moderate). Glycemic control, as measured by HbA1c, was stable during paltusotine treatment. In conclusion, long-term results show that once-daily oral paltusotine treatment was well tolerated, with stable biochemical and symptom control relative to that observed with injected SRLs. Support: Crinetics Pharmaceuticals.Presentation: 6/3/2024}, year = {2024}, eissn = {2472-1972}, pages = {A661-A662}, orcid-numbers = {Tóth, Miklós/0000-0002-8701-408X} } @misc{MTMT:35596786, title = {ORAL PALTUSOTINE MAINTAINS IGF-I, GH, AND SYMPTOM CONTROL IN PATIENTS WITH ACROMEGALY SWITCHED FROM INJECTED SOMATOSTATIN RECEPTOR LIGAND MONOTHERAPY: THE PHASE 3, PATHFNDR-1 STUDY RESULTS.}, url = {https://m2.mtmt.hu/api/publication/35596786}, author = {Gadelha, Monica R and Casagrande, Alessandra and Strasburger, Christian J and Bidlingmaier, Martin and Snyder, Peter and Guitelman, Mirtha A and Boguszewski, Cesar L and Buchfelder, Michael and Shimon, Ilan and Raverot, Gerald and Tóth, Miklós and Mezősi, Emese and Doknic, Mirjana and Fan, Xiaolin and Clemmons, David and Keeley, Michael and Trainer, Peter J and Struthers, R. Scott and Krasner, Alan and Biller, Beverly M.K.}, unique-id = {35596786}, year = {2024}, orcid-numbers = {Tóth, Miklós/0000-0002-8701-408X} } @article{MTMT:34452033, title = {High prevalence of venous thrombotic events in Cushing's syndrome. data from ERCUSYN and details in relation to surgery.}, url = {https://m2.mtmt.hu/api/publication/34452033}, author = {Isand, Kristina and Feelders, Richard and Brue, Thierry and Tóth, Miklós and Deutschbein, Timo and Reincke, Martin and Krsek, Michal and Santos, Alicia and Demtröder, Frank and Chabre, Olivier and Strasburger, Christian J and Aulinas Maso, Anna and Volke, Vallo and Pereira, Alberto M and Lohmann, Rüdiger and Gich Saladich, Ignasi and Webb, Susan M and Wass, John and Valassi, Elena}, doi = {10.1093/ejendo/lvad176}, journal-iso = {EUR J ENDOCRINOL}, journal = {EUROPEAN JOURNAL OF ENDOCRINOLOGY}, volume = {190}, unique-id = {34452033}, issn = {0804-4643}, abstract = {The aim of this study was to evaluate the prevalence of venous thromboembolism (VTE) in patients included in the European Registry on Cushing's syndrome (ERCUSYN), compare their clinical characteristics with those who did not develop VTE and identify risk factors for VTE.A retrospective observational cohort study.Data extraction from the registry was taken on the 07.02.2022. At the time there were 2174 patients diagnosed with Cushing's syndrome (CS) and 95 VTE's reported in the database.Of 95 VTE events 70 (74%) were in pituitary-dependent CS patients, 12 (12.5%) in adrenal-dependant CS, 10 (10.5%) in ectopic CS and 3 (3%) in CS due to other causes.Sex, UFC value at diagnosis, as well as the number of operations remained statistically significant predictors of VTE.Of patients who were treated with at least one surgery, 12 (13%) VTE occured before and 80 (87%) after the surgery. Nearly half of these VTE's occurred within six months since the operation (36; 45%).Over half the centres who reported VTE did not routinely anticoagulate CS patients. Anticoagulation schemes varied widely.Patients with CS have elevated risk of developing VTE for an extended period of time. From ERCUSYN cohort patients have higher risk for VTE if they need multiple surgeries to treat CS, are males and have high UFC values at the diagnosis of CS.Since there is no agreement on thromboprohpylaxis, a protocol for VTE prevention which is widely adopted appears to be necessary in patients with CS.}, keywords = {Risk Factors; venous thromboembolism; Cushing’s syndrome; Ercusyn}, year = {2024}, eissn = {1479-683X}, pages = {75-85}, orcid-numbers = {Tóth, Miklós/0000-0002-8701-408X; Tóth, Miklós/0000-0002-8701-408X} } @article{MTMT:35596764, title = {A peculiar case of ectopic ACTH-syndrome - multiple challenges during diagnostic and therapeutic workup}, url = {https://m2.mtmt.hu/api/publication/35596764}, author = {Kövesdi, Annamária and Tőke, Judit and Eitler, Katalin and Kiss, Gergely and Nyilas, Nóra Luca and Scheich, Bálint and Tóth, Miklós}, doi = {10.1530/endoabs.99.P529}, journal-iso = {ENDOCR ABSTR}, journal = {ENDOCRINE ABSTRACTS}, volume = {99}, unique-id = {35596764}, issn = {1470-3947}, year = {2024}, eissn = {1479-6848}, orcid-numbers = {Kövesdi, Annamária/0000-0001-8067-1792; Tóth, Miklós/0000-0002-8701-408X} } @article{MTMT:35657439, title = {A karcinoid, amely nem válogat – a preoperatív echokardiográfia szerepe karcinoid szívbetegségben}, url = {https://m2.mtmt.hu/api/publication/35657439}, author = {Papp, E and Dénes, M and Zádori, A and Takó, K and Tóth, Miklós and Tőke, Judit and Szolnoky, J and Rácz, R. and Andréka, P}, doi = {10.26430/CHUNGARICA.2024.54.6.439}, journal-iso = {CARDIOL HUNG}, journal = {CARDIOLOGIA HUNGARICA}, volume = {54}, unique-id = {35657439}, issn = {0133-5596}, year = {2024}, eissn = {1588-0230}, pages = {439-443}, orcid-numbers = {Tóth, Miklós/0000-0002-8701-408X} } @article{MTMT:35711219, title = {PRIMER BILATERALIS MACRONODULARIS MELLÉKVESE HYPERPLASIA}, url = {https://m2.mtmt.hu/api/publication/35711219}, author = {Perge, Pál and Mezei, P and József, I and Kiss, E and Huszty, Gergely and Kovács, K and Tőke, Judit and Tóth, Miklós}, journal-iso = {MBA}, journal = {MAGYAR BELORVOSI ARCHIVUM}, volume = {77}, unique-id = {35711219}, issn = {0133-5464}, year = {2024}, pages = {354-354}, orcid-numbers = {Perge, Pál/0000-0002-0722-7684; Tóth, Miklós/0000-0002-8701-408X} } @article{MTMT:35596769, title = {Diagnosis and therapy of medullary thyroid cancer. A real-life, retrospective, multicentric, Hungarian study}, url = {https://m2.mtmt.hu/api/publication/35596769}, author = {Réti, Zsuzsanna and Tőke, Judit and Balla, Reka and Reismann, Péter and Kovács, Attila and Sápi, Zoltán and Bodor, Miklos and Nagy, Endre and Ivanyi, Gabor and Valkusz, Zsuzsanna and Mezosi, Emese and Tóth, Miklós}, doi = {10.1530/endoabs.99.P372}, journal-iso = {ENDOCR ABSTR}, journal = {ENDOCRINE ABSTRACTS}, volume = {99}, unique-id = {35596769}, issn = {1470-3947}, year = {2024}, eissn = {1479-6848}, orcid-numbers = {Reismann, Péter/0000-0002-2696-6132; Tóth, Miklós/0000-0002-8701-408X} }