@article{MTMT:34829112,
	title = {CROCuS, a Phase II Study Evaluating the Antiviral Activity, Clinical Outcomes, and Safety of Rilematovir in Children Aged ≥ 28 Days and ≤ 3 Years with Acute Respiratory Tract Infection Due to Respiratory Syncytial Virus},
	url = {https://m2.mtmt.hu/api/publication/34829112},
	author = {Ferrero, F. and Lin, C.-Y. and Liese, J. and Luz, K. and Stoeva, T. and Németh, Ágnes and Gijón, M. and Calvo, C. and Natalini, S. and Toh, T.-H. and Deleu, S. and Chen, B. and Rusch, S. and Sánchez, B.L. and Leipoldt, I. and Vijgen, L. and Huntjens, D. and Baguet, T. and Bertzos, K. and Gamil, M. and Stevens, M.},
	doi = {10.1007/s40272-024-00625-x},
	journal-iso = {PEDIATR DRUGS},
	journal = {PEDIATRIC DRUGS},
	unique-id = {34829112},
	issn = {1174-5878},
	year = {2024},
	eissn = {1179-2019},
	orcid-numbers = {Németh, Ágnes/0000-0002-5846-7071}
}

@article{MTMT:34729555,
	title = {Early onset of abnormal glucose tolerance in patients with cystic fibrosis : A systematic review and meta-analysis},
	url = {https://m2.mtmt.hu/api/publication/34729555},
	author = {Ferencsikné Kéri, Adrienn Krisztina and Bajzát, Dorina Rita and Andrásdi, Zita and Juhász, Márk Félix and Nagy, Rita and Kói, Tamás and Kovács, Gábor and Hegyi, Péter and Párniczky, Andrea},
	doi = {10.1016/j.jcf.2024.02.010},
	journal-iso = {J CYST FIBROS},
	journal = {JOURNAL OF CYSTIC FIBROSIS},
	unique-id = {34729555},
	issn = {1569-1993},
	abstract = {Despite translational evidences suggesting that cystic fibrosis-related abnormal glucose tolerance (CF-related AGT) may begin early in life and is known to be associated with increased morbidity and mortality, current guidelines recommend screening for AGT only from 10 years of age, thus missing the opportunity for early detection and intervention.A systematic review and meta-analysis (PROSPERO number: CRD42021282516) was conducted on studies that reported data on the prevalence of AGT or its subtypes in CF populations. Pooled proportions, risk, and odds ratios with 95 % confidence intervals (CI) were calculated. One-stage dose-response random-effect meta-analysis was used to assess the effect of age on CF-related diabetes (CFRD).The quantitative analysis included 457 studies and data from 520,544 patients. Every third child with CF (chwCF) (0.31 [95 % CI 0.25-0.37]) and every second adult with CF (awCF) (0.51 [95 % CI 0.45-0.57]) were affected by AGT. Even in the 5-10 years of age subgroup, the proportion of AGT was 0.42 [95 % CI 0.34-0.51]. The prevalence of prediabetes remained unchanged (impaired glucose tolerance in chwCF:0.14 [95 % CI 0.10-0.18]) vs. awCF:0.19 [95 % CI 0.14-0.25]), whereas the proportion of CFRD increased with age (0-5: 0.005 [95 % CI 0.0001-0.15]; 5-10: 0.05 [95 % CI 0.01-0.27]; 10-18: 0.11 [95 % CI 0.08-0.14]; >18 years of age: 0.27 [95 % CI 0.24-0.30]).CF-related AGT is common under 10 years of age. Our study suggests considering earlier AGT screening, starting from 5 years of age. This highlights the imperative for additional research for guideline adjustments and provides the opportunity for early intervention.},
	keywords = {PREVALENCE; EPIDEMIOLOGY; Meta-analysis; cystic fibrosis (CF); Glucose abnormalities; OGTT (Oral Glucose Tolerance Test)},
	year = {2024},
	eissn = {1873-5010},
	orcid-numbers = {Nagy, Rita/0000-0002-2663-4912; Kovács, Gábor/0000-0001-9924-1645; Hegyi, Péter/0000-0003-0399-7259}
}

@article{MTMT:34544768,
	title = {Risk of subsequent gliomas and meningiomas among 69,460 5-year survivors of childhood and adolescent cancer in Europe: the PanCareSurFup study},
	url = {https://m2.mtmt.hu/api/publication/34544768},
	author = {Heymer, E.J. and Hawkins, M.M. and Winter, D.L. and Teepen, J.C. and Sunguc, C. and Ronckers, C.M. and Allodji, R.S. and Alessi, D. and Sugden, E. and Belle, F.N. and Bagnasco, F. and Byrne, J. and Bárdi, E. and Garwicz, S. and Grabow, D. and Jankovic, M. and Kaatsch, P. and Kaiser, M. and Michel, G. and Schindera, C. and Haddy, N. and Journy, N. and Česen, Mazić M. and Skinner, R. and Kok, J.L. and Gunnes, M.W. and Wiebe, T. and Sacerdote, C. and Maule, M.M. and Terenziani, M. and Jakab, Zsuzsanna and Winther, J.F. and Lähteenmäki, P.M. and Zadravec, Zaletel L. and Haupt, R. and Kuehni, C.E. and Kremer, L.C. and de, Vathaire F. and Hjorth, L. and Reulen, R.C.},
	doi = {10.1038/s41416-024-02577-y},
	journal-iso = {BRIT J CANCER},
	journal = {BRITISH JOURNAL OF CANCER},
	unique-id = {34544768},
	issn = {0007-0920},
	year = {2024},
	eissn = {1532-1827},
	orcid-numbers = {Jakab, Zsuzsanna/0000-0002-8231-0357}
}

@article{MTMT:33770049,
	title = {Cost-effectiveness analysis alongside the inter-B-NHL ritux 2010 trial: rituximab in children and adolescents with B cell non-Hodgkin’s lymphoma},
	url = {https://m2.mtmt.hu/api/publication/33770049},
	author = {Lueza, B. and Aupérin, A. and Rigaud, C. and Gross, T.G. and Pillon, M. and Delgado, R.F. and Uyttebroeck, A. and Amos, Burke G.A. and Zsíros, J. and Csóka, Monika and Simonin, M. and Patte, C. and Minard-Colin, V. and Bonastre, J.},
	doi = {10.1007/s10198-023-01581-y},
	journal-iso = {EUR J HEALTH ECON},
	journal = {EUROPEAN JOURNAL OF HEALTH ECONOMICS},
	volume = {25},
	unique-id = {33770049},
	issn = {1618-7598},
	year = {2024},
	eissn = {1618-7601},
	pages = {307-317},
	orcid-numbers = {Csóka, Monika/0000-0003-4202-891X}
}

@article{MTMT:32764701,
	title = {Parent-child agreement on health-related quality of life: the role of perceived consequences of the child’s chronic illness},
	url = {https://m2.mtmt.hu/api/publication/32764701},
	author = {Papp, Zsuzsanna Katalin and Török, Szabolcs János and Szentes, Annamária and Hosszú, Dalma and Kökönyei, Gyöngyi},
	doi = {10.1080/08870446.2022.2057496},
	journal-iso = {PSYCHOL HEALTH},
	journal = {PSYCHOLOGY AND HEALTH},
	volume = {39},
	unique-id = {32764701},
	issn = {0887-0446},
	year = {2024},
	eissn = {1476-8321},
	pages = {233-251},
	orcid-numbers = {Papp, Zsuzsanna Katalin/0000-0003-3651-177X; Török, Szabolcs János/0000-0003-4653-1707; Szentes, Annamária/0000-0002-1320-7559; Kökönyei, Gyöngyi/0000-0001-6750-2644}
}

@article{MTMT:34474596,
	title = {Misfolding of fukutin-related protein (FKRP) variants in congenital and limb girdle muscular dystrophies},
	url = {https://m2.mtmt.hu/api/publication/34474596},
	author = {Esapa, Christopher T. and Mcilhinney, R. A. Jeffrey and Waite, Adrian J. and Benson, Matthew A. and Mirzayan, Jasmin and Pikó, Henriett and Herczegfalvi, Ágnes and Horvath, Rita and Karcagi, Veronika and Walter, Maggie C. and Lochmueller, Hanns and Rizkallah, Pierre J. and Lu, Qi L. and Blake, Derek J.},
	doi = {10.3389/fmolb.2023.1279700},
	journal-iso = {FRONT MOL BIOSCI},
	journal = {FRONTIERS IN MOLECULAR BIOSCIENCES},
	volume = {10},
	unique-id = {34474596},
	abstract = {Fukutin-related protein (FKRP, MIM ID 606596) variants cause a range of muscular dystrophies associated with hypo-glycosylation of the matrix receptor, alpha-dystroglycan. These disorders are almost exclusively caused by homozygous or compound heterozygous missense variants in the FKRP gene that encodes a ribitol phosphotransferase. To understand how seemingly diverse FKRP missense mutations may contribute to disease, we examined the synthesis, intracellular dynamics, and structural consequences of a panel of missense mutations that encompass the disease spectrum. Under non-reducing electrophoresis conditions, wild type FKRP appears to be monomeric whereas disease-causing FKRP mutants migrate as high molecular weight, disulfide-bonded aggregates. These results were recapitulated using cysteine-scanning mutagenesis suggesting that abnormal disulfide bonding may perturb FKRP folding. Using fluorescence recovery after photobleaching, we found that the intracellular mobility of most FKRP mutants in ATP-depleted cells is dramatically reduced but can, in most cases, be rescued with reducing agents. Mass spectrometry showed that wild type and mutant FKRP differentially associate with several endoplasmic reticulum (ER)-resident chaperones. Finally, structural modelling revealed that disease-associated FKRP missense variants affected the local environment of the protein in small but significant ways. These data demonstrate that protein misfolding contributes to the molecular pathophysiology of FKRP-deficient muscular dystrophies and suggest that molecules that rescue this folding defect could be used to treat these disorders.},
	keywords = {MUTATIONS; ENDOPLASMIC-RETICULUM; CYSTIC-FIBROSIS; missense mutation; chaperone; MUTANT; Protein misfolding; MENTAL-RETARDATION; muscular dystrophy; PHENOTYPIC SPECTRUM; myoclonus-dystonia; epsilon-sarcoglycan; FUKUTIN-RELATED PROTEIN; RIBITOL-PHOSPHATE},
	year = {2023},
	eissn = {2296-889X},
	orcid-numbers = {Pikó, Henriett/0000-0002-3579-5451; Herczegfalvi, Ágnes/0000-0002-8013-4775}
}

@article{MTMT:34471018,
	title = {Zoledronic Acid Add-on Therapy for Standard-Risk Ewing Sarcoma Patients in the Ewing 2008R1 Trial},
	url = {https://m2.mtmt.hu/api/publication/34471018},
	author = {Koch, R. and Haveman, L. and Ladenstein, R. and Brichard, B. and Jürgens, H. and Cyprova, S. and van, den Berg H. and Hassenpflug, W. and Raciborska, A. and Ek, T. and Baumhoer, D. and Egerer, G. and Kager, L. and Renard, M. and Hauser, Péter and Burdach, S. and Bovee, J.V.M.G. and Hong, A.M. and Reichardt, P. and Kruseova, J. and Streitbürger, A. and Kühne, T. and Kessler, T. and Bernkopf, M. and Butterfaß-Bahloul, T. and Dhooge, C. and Bauer, S. and Kiss, János and Paulussen, M. and Bonar, F. and Ranft, A. and Timmermann, B. and Rascon, J. and Vieth, V. and Kanerva, J. and Faldum, A. and Hartmann, W. and Hjorth, L. and Bhadri, V.A. and Metzler, M. and Gelderblom, H. and Dirksen, U.},
	doi = {10.1158/1078-0432.CCR-23-1966},
	journal-iso = {CLIN CANCER RES},
	journal = {CLINICAL CANCER RESEARCH},
	volume = {29},
	unique-id = {34471018},
	issn = {1078-0432},
	year = {2023},
	eissn = {1557-3265},
	pages = {5057-5068},
	orcid-numbers = {Hauser, Péter/0000-0002-8307-8975}
}

@article{MTMT:34412424,
	title = {GENOTYPE-PHENOTYPE ASSOCIATION OF PEDIATRIC NEUROFIBROMATOSIS TYPE 1},
	url = {https://m2.mtmt.hu/api/publication/34412424},
	author = {Botos, Péter Barnabás and Osztermayer, Dorka Réka and Kovacs, A and Garami, Miklós and Bodor, C and Brückner, Edit and Jenei, Sámuel},
	journal-iso = {ARCH HUNG MED ASSOC AM},
	journal = {ARCHIVES OF THE HUNGARIAN MEDICAL ASSOCIATION OF AMERICA},
	volume = {31},
	unique-id = {34412424},
	issn = {1070-0773},
	year = {2023},
	pages = {38},
	orcid-numbers = {Garami, Miklós/0000-0003-4298-2746; Brückner, Edit/0000-0002-2140-3357}
}

@article{MTMT:34271695,
	title = {The EMT factor ZEB1 paradoxically inhibits EMT in BRAF-mutant carcinomas.},
	url = {https://m2.mtmt.hu/api/publication/34271695},
	author = {Sánchez-Tilló, Ester and Pedrosa, Leire and Vila, Ingrid and Chen, Yongxu and Győrffy, Balázs and Sánchez-Moral, Lidia and Siles, Laura and Lozano, Juan J and Esteve-Codina, Anna and Darling, Douglas S and Cuatrecasas, Miriam and Castells, Antoni and Maurel, Joan and Postigo, Antonio},
	doi = {10.1172/jci.insight.164629},
	journal-iso = {JCI INSIGHT},
	journal = {JCI INSIGHT},
	volume = {8},
	unique-id = {34271695},
	abstract = {Despite being in the same pathway, mutations of KRAS and BRAF in colorectal carcinomas (CRCs) determine distinct progression courses. ZEB1 induces an epithelial-to-mesenchymal transition (EMT) and is associated with worse progression in most carcinomas. Using samples from patients with CRC, mouse models of KrasG12D and BrafV600E CRC, and a Zeb1-deficient mouse, we show that ZEB1 had opposite functions in KRAS- and BRAF-mutant CRCs. In KrasG12D CRCs, ZEB1 was correlated with a worse prognosis and a higher number of larger and undifferentiated (mesenchymal or EMT-like) tumors. Surprisingly, in BrafV600E CRC, ZEB1 was associated with better prognosis; fewer, smaller, and more differentiated (reduced EMT) primary tumors; and fewer metastases. ZEB1 was positively correlated in KRAS-mutant CRC cells and negatively in BRAF-mutant CRC cells with gene signatures for EMT, cell proliferation and survival, and ERK signaling. On a mechanistic level, ZEB1 knockdown in KRAS-mutant CRC cells increased apoptosis and reduced clonogenicity and anchorage-independent growth; the reverse occurred in BRAFV600E CRC cells. ZEB1 is associated with better prognosis and reduced EMT signature in patients harboring BRAF CRCs. These data suggest that ZEB1 can function as a tumor suppressor in BRAF-mutant CRCs, highlighting the importance of considering the KRAS/BRAF mutational background of CRCs in therapeutic strategies targeting ZEB1/EMT.},
	keywords = {Oncology; colorectal cancer; GASTROENTEROLOGY},
	year = {2023},
	eissn = {2379-3708},
	orcid-numbers = {Győrffy, Balázs/0000-0002-5772-3766}
}

@article{MTMT:34265523,
	title = {Luminal breast cancer identity is determined by loss of glucocorticoid receptor activity},
	url = {https://m2.mtmt.hu/api/publication/34265523},
	author = {Prekovic, S. and Chalkiadakis, T. and Roest, M. and Roden, D. and Lutz, C. and Schuurman, K. and Opdam, M. and Hoekman, L. and Abbott, N. and Tesselaar, T. and Wajahat, M. and Dwyer, A.R. and Mayayo-Peralta, I. and Gomez, G. and Altelaar, M. and Beijersbergen, R. and Győrffy, Balázs and Young, L. and Linn, S. and Jonkers, J. and Tilley, W. and Hickey, T. and Vareslija, D. and Swarbrick, A. and Zwart, W.},
	doi = {10.15252/emmm.202317737},
	journal-iso = {EMBO MOL MED},
	journal = {EMBO MOLECULAR MEDICINE},
	volume = {15},
	unique-id = {34265523},
	issn = {1757-4676},
	abstract = {Glucocorticoid receptor (GR) is a transcription factor that plays a crucial role in cancer biology. In this study, we utilized an in silico-designed GR activity signature to demonstrate that GR relates to the proliferative capacity of numerous primary cancer types. In breast cancer, the GR activity status determines luminal subtype identity and has implications for patient outcomes. We reveal that GR engages with estrogen receptor (ER), leading to redistribution of ER on the chromatin. Notably, GR activation leads to upregulation of the ZBTB16 gene, encoding for a transcriptional repressor, which controls growth in ER-positive breast cancer and associates with prognosis in luminal A patients. In relation to ZBTB16's repressive nature, GR activation leads to epigenetic remodeling and loss of histone acetylation at sites proximal to cancer-driving genes. Based on these findings, epigenetic inhibitors reduce viability of ER-positive breast cancer cells that display absence of GR activity. Our findings provide insights into how GR controls ER-positive breast cancer growth and may have implications for patients' prognostication and provide novel therapeutic candidates for breast cancer treatment. © 2023 Netherlands Cancer Institute. Published under the terms of the CC BY 4.0 license.},
	keywords = {NUCLEAR RECEPTORS; GLUCOCORTICOIDS; breast cancer; Zbtb16; luminal breast cancer subtypes},
	year = {2023},
	eissn = {1757-4684},
	orcid-numbers = {Győrffy, Balázs/0000-0002-5772-3766}
}