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1,155 results on '"Cefalù, A."'

1. Autoregressive models for panel data causal inference with application to state-level opioid policies

Author

Antonelli, Joseph, Rubinstein, Max, Agniel, Denis, Smart, Rosanna, Stuart, Elizabeth, Cefalu, Matthew, Schell, Terry, Eagan, Joshua, Stone, Elizabeth, Griswold, Max, Sorbero, Mark, and Griffin, Beth Ann

Subjects
Statistics - Methodology, Statistics - Applications
Abstract

Motivated by the study of state opioid policies, we propose a novel approach that uses autoregressive models for causal effect estimation in settings with panel data and staggered treatment adoption. Specifically, we seek to estimate of the impact of key opioid-related policies by quantifying the effects of must access prescription drug monitoring programs (PDMPs), naloxone access laws (NALs), and medical marijuana laws on opioid prescribing. Existing methods, such as differences-in-differences and synthetic controls, are challenging to apply in these types of dynamic policy landscapes where multiple policies are implemented over time and sample sizes are small. Autoregressive models are an alternative strategy that have been used to estimate policy effects in similar settings, but until this paper have lacked formal justification. We outline a set of assumptions that tie these models to causal effects, and we study biases of estimates based on this approach when key causal assumptions are violated. In a set of simulation studies that mirror the structure of our application, we also show that our proposed estimators frequently outperform existing estimators. In short, we justify the use of autoregressive models to provide robust evidence on the effectiveness of four state policies in combating the opioid crisis.

Published
2024

3. Modeling Outcome in Patients With Acquired Brain Injuries (MOF-ABI)

Author

Istituto S.Anna Crotone, IRCCS Centro Neurolesi 'Bonino-Pulejo', Centro Cardinal Ferrari, Fontanellato, Parma, Fondazione Istituto G. Giglio, Cefalù (PA), Habilita Istituto di Neuroriabilitazione, Zingonia (BG), Casa Sollievo della Sofferenza IRCCS, IRCCS Istituto delle Scienze Neurologiche di Bologna, IRCCS Sacro Cuore Don Calabria di Negrar, Fondazione Salvatore Maugeri, Istituti Clinici Zucchi, Istituto Clinico Humanitas, Istituto Medicina Fisica E Riabilitazione Gervasutta, Udine, Montecatone Rehabilitation Institute S.p.A., Ospedale Di Riabilitazione Fondazione Santa Lucia, Roma, Ospedale San Giovanni Battista, Foligno (PG), Ospedale Ss. Trinità Di Fossano, Cuneo, Fondazione Don Carlo Gnocchi Onlus, and Antonio Cerasa, PhD, Head of Unit

Published
2024

4. Evaluating the Susceptibility of Pre-Trained Language Models via Handcrafted Adversarial Examples

Author

Branch, Hezekiah J., Cefalu, Jonathan Rodriguez, McHugh, Jeremy, Hujer, Leyla, Bahl, Aditya, Iglesias, Daniel del Castillo, Heichman, Ron, and Darwishi, Ramesh

Subjects
Computer Science - Computation and Language
Abstract

Recent advances in the development of large language models have resulted in public access to state-of-the-art pre-trained language models (PLMs), including Generative Pre-trained Transformer 3 (GPT-3) and Bidirectional Encoder Representations from Transformers (BERT). However, evaluations of PLMs, in practice, have shown their susceptibility to adversarial attacks during the training and fine-tuning stages of development. Such attacks can result in erroneous outputs, model-generated hate speech, and the exposure of users' sensitive information. While existing research has focused on adversarial attacks during either the training or the fine-tuning of PLMs, there is a deficit of information on attacks made between these two development phases. In this work, we highlight a major security vulnerability in the public release of GPT-3 and further investigate this vulnerability in other state-of-the-art PLMs. We restrict our work to pre-trained models that have not undergone fine-tuning. Further, we underscore token distance-minimized perturbations as an effective adversarial approach, bypassing both supervised and unsupervised quality measures. Following this approach, we observe a significant decrease in text classification quality when evaluating for semantic similarity., Comment: 10 pages, 1 figure, 3 tables

Published
2022

5. A tutorial for using propensity score weighting for moderation analysis: an application to smoking disparities among sexual minority adults

Author

Griffin, Beth Ann, Schuler, Megan S., Cefalu, Matt, Ayer, Lynsay, Godley, Mark, Greifer, Noah, Coffman, Donna L., and McCaffrey, Daniel

Subjects
Statistics - Applications, Statistics - Methodology
Abstract

Objective. To provide step-by-step guidance and STATA and R code for using propensity score (PS) weighting to estimate moderation effects. Research Design. Tutorial illustrating the key steps for estimating and testing moderation using observational data. Steps include (1) examining covariate overlap across treatment groups within levels of the moderator, (2) estimating the PS weights, (3) evaluating whether PS weights improved covariate balance, (4) estimating moderated treatment effects, and (5) assessing sensitivity of findings to unobserved confounding. Our illustrative case study uses data from 41,832 adults from the 2019 National Survey on Drug Use and Health to examine if gender moderates the association between sexual minority status (e.g., lesbian, gay, or bisexual [LGB] identity) and adult smoking prevalence. Results. For our case study, there were no noted concerns about covariate overlap and we were able to successfully estimate the PS weights within each level of the moderator. Moreover, balance criteria indicated that PS weights successfully achieved covariate balance for both moderator groups. PS weighted results indicated there was significant evidence of moderation for the case study and sensitivity analyses demonstrated that results were highly robust for one level of the moderator but not the other. Conclusions. When conducting moderation analyses, covariate imbalances across levels of the moderator can cause biased estimates. As demonstrated in this tutorial, PS weighting within each level of the moderator can improve the estimated moderation effects by minimizing bias from imbalance within the moderator subgroups.

Published
2022

7. Association of BMI, lipid-lowering medication, and age with prevalence of type 2 diabetes in adults with heterozygous familial hypercholesterolaemia: a worldwide cross-sectional study

Author

Elshorbagy, Amany, Lyons, Alexander R.M., Vallejo-Vaz, Antonio J., Stevens, Christophe A.T., Dharmayat, Kanika I., Brandts, Julia, Catapano, Alberico L., Freiberger, Tomas, Hovingh, G. Kees, Mata, Pedro, Raal, Frederick J., Santos, Raul D., Soran, Handrean, Watts, Gerald F., Abifadel, Marianne, Aguilar-Salinas, Carlos A., Alhabib, Khalid F., Alkhnifsawi, Mutaz, Almahmeed, Wael, Alonso, Rodrigo, Al-Rasadi, Khalid, Al-Sarraf, Ahmad, Ashavaid, Tester F., Banach, Maciej, Binder, Christoph J., Bourbon, Mafalda, Brunham, Liam R., Chlebus, Krzysztof, Corral, Pablo, Cruz, Diogo, Davletov, Kairat, Descamps, Olivier S., Ezhov, Marat, Gaita, Dan, Groselj, Urh, Harada-Shiba, Mariko, Holven, Kirsten B., Kayikcioglu, Meral, Khovidhunkit, Weerapan, Lalic, Katarina, Latkovskis, Gustavs, Laufs, Ulrich, Liberopoulos, Evangelos, Lima-Martinez, Marcos M., Lin, Jie, Maher, Vincent, Marais, A. David, März, Winfried, Mirrakhimov, Erkin, Miserez, André R., Mitchenko, Olena, Nawawi, Hapizah, Nordestgaard, Børge G., Panayiotou, Andrie G., Paragh, György, Petrulioniene, Zaneta, Pojskic, Belma, Postadzhiyan, Arman, Reda, Ashraf, Reiner, Željko, Reyes, Ximena, Sadiq, Fouzia, Sadoh, Wilson E., Schunkert, Heribert, Shek, Aleksandr B., Stroes, Erik, Su, Ta-Chen, Subramaniam, Tavintharan, Susekov, Andrey V., Tilney, Myra, Tomlinson, Brian, Truong, Thanh-Huong, Tselepis, Alexandros D., Tybjærg-Hansen, Anne, Vázquez, Alejandra C., Viigimaa, Margus, Vohnout, Branislav, Wang, Luya, Yamashita, Shizuya, Arca, Marcello, Averna, Maurizio, Schreier, Laura, Pang, Jing, Ebenbichler, Christoph, Dieplinger, Hans, Innerhofer, Reinhold, Winhofer-Stöckl, Yvonne, Greber-Platzer, Susanne, Krychtiuk, Konstantin, Speidl, Walter, Toplak, Hermann, Widhalm, Kurt, Stulnig, Thomas, Huber, Kurt, Höllerl, Florian, Rega-Kaun, Gersina, Kleemann, Lucas, Mäser, Martin, Scholl-Bürgi, Sabine, Säly, Christoph, Mayer, Florian J., Sperone, Alexandra, Tanghe, Chloé, Gérard, Anne-Catherine, Pojskic, Lamija, Sisic, Ibrahim, Durak Nalbantic, Azra, Ejubovic, Malik, Jannes, Cinthia E., Pereira, Alexandre C., Krieger, Jose E., Petrov, Ivo, Goudev, Assen, Nikolov, Fedya, Tisheva, Snejana, Yotov, Yoto, Tzvetkov, Ivajlo, Baass, Alexis, Bergeron, Jean, Bernard, Sophie, Brisson, Diane, Cermakova, Lubomira, Couture, Patrick, Francis, Gordon A., Gaudet, Daniel, Hegele, Robert A., Khoury, Etienne, Mancini, G.B. John, McCrindle, Brian W., Paquette, Martine, Ruel, Isabelle, Iatan, Iulia, Cuevas, Ada, Wang, Xumin, Meng, Kang, Song, Xiantao, Yong, Qiang, Jiang, Tao, Liu, Ziyou, Duan, Yanyu, Hong, Jing, Ye, Pucong, Chen, Yan, Qi, Jianguang, Liu, Zesen, Li, Yuntao, Zhang, Chaoyi, Peng, Jie, Yang, Ya, Yu, Wei, Wang, Qian, Yuan, Hui, Cheng, Shitong, Jiang, Long, Chong, Mei, Jiao, Jian, Wu, Yue, Wen, Wenhui, Xu, Liyuan, Zhang, Ruiying, Qu, Yichen, He, Jianxun, Fan, Xuesong, Wang, Zhenjia, Chow, Elaine, Pećin, Ivan, Perica, Dražen, Symeonides, Phivos, Vrablik, Michal, Ceska, Richard, Soska, Vladimir, Tichy, Lukas, Adamkova, Vera, Franekova, Jana, Cifkova, Renata, Kraml, Pavel, Vonaskova, Katerina, Cepova, Jana, Dusejovska, Magdalena, Pavlickova, Lenka, Blaha, Vladimir, Rosolova, Hana, Nussbaumerova, Barbora, Cibulka, Roman, Vaverkova, Helena, Cibickova, Lubica, Krejsova, Zdenka, Rehouskova, Katerina, Malina, Pavel, Budikova, Milena, Palanova, Vaclava, Solcova, Lucie, Lubasova, Alena, Podzimkova, Helena, Bujdak, Juraj, Vesely, Jiri, Jordanova, Marta, Salek, Tomas, Urbanek, Robin, Zemek, Stanislav, Lacko, Jan, Halamkova, Hana, Machacova, Sona, Mala, Sarka, Cubova, Eva, Valoskova, Katerina, Burda, Lukas, Benn, Marianne, Bendary, Ahmed, Daoud, Ihab, Emil, Sameh, Elbahry, Atef, Rafla, Samir, Sanad, Osama, Kazamel, Ghada, Ashraf, Dr Mohamed, Sobhy, Mohamed, El-Hadidy, Amro, Shafy, Mohamed Abdoul, Kamal, Saif, Bendary, Mohamed, Talviste, Grete, Christmann, Jutta, Dressel, Alexander, Fath, Felix, Ferraro, Chiara, Frenzke, Lydia, Gopon, Alica, Klein, Isabel, Pienkowska, Dominika, Sietmann, Tobias, Sonntag, Antonia, Adjan, Omar, Bahrmann, Philipp, Baessler, Andrea, Barkowski, Rasmus, Beckerdjian, Raffi, Berr, Christina, Birkenfeld, Andreas, Böll, Gereon, Carstensen, Avisha, Demuth, Ilya, Finkernagel, Holger, Gouni-Berthold, Ioanna, Hahmann, Harry, Hamerle, Michael, Halder, Julian, Heide, Maria, Julius, Ulrich, Kassner, Ursula, Katzmann, Julius L, Kirschbaum, Anja, Klose, Gerald, Könemann, Stephanie, König, Christel, König, Wolfgang, Krämer, Bernhard, Kuprat, Gerrit, Koschker, Ann-Cathrin, Kilic, Özlem, Lindenmeier, Gerd, Van de Loo, Iris, Lorenz, Babette, Lorenz, Elke, Löhr, Birgit, McChord, Johanna, Maslarska, Mariya, Methe, Heiko, Merkel, Martin, Moussaoui, Zineb, Müller-Kozarez, Irina, Olivier, Christoph B, Ong, Peter, Otte, Britta, Parhofer, Klaus, Partsch, Carl-Joachim, Paulus, Michael, Pehlivanli, Sinan, Pflederer, Tobias, Pusl, Thomas, Richter, Veronika, Rosner, Stefanie, Sanin, Veronika, Schäfer, Sebastian, Schäfer, Christoph, Schatz, Ulrike, Schirmer, Stephan, Schmidt, Christine, Seeger, Wolfgang, Sisovic, Snezna, Spens, Antje, Jablonski, Ksenija Stach, Stadelmann, Alexander, Steinhagen-Thiessen, Elisabeth, Stürzebecher, Paulina, Tafelmeier, Maria, Tillack, Dörthe, Tselmin, Sergey, Tünnemann-Tarr, Adrienn, Vogt, Anja, Beckerath, Jens von, Wilke, Andreas, Wolf, Ulrich, Zemmrich, Claudia, Rizos, Christos V., Skoumas, Ioannis, Tziomalos, Konstantinos, Rallidis, Loukianos, Kotsis, Vasileios, Doumas, Michalis, Athyros, Vasileios, Skalidis, Emmanouil, Kolovou, Genovefa, Kolovou, Vana, Garoufi, Anastasia, Bilianou, Eleni, Koutagiar, Iosif, Kiouri, Estela, Antza, Christina, Zacharis, Evangelos, Attilakos, Achilleas, Sfikas, George, Koumaras, Charalambos, Anagnostis, Panagiotis, Anastasiou, Georgia, Liamis, George, Koutsogianni, Amalia-Despoina, Petkou, Ermioni, Milionis, Haralambos, Koulouri, Anastasia, Prodromiadou, Elisavet, Karányi, Zsolt, Harangi, Mariann, Bajnok, László, Audikovszky, Mária, Márk, László, Benczúr, Béla, Reiber, István, Nagy, Gergely, Nagy, András, Reddy, Lakshmi Lavanya, Shah, Swarup A. V, Ponde, Chandrashekhar K., Dalal, Jamshed J., Sawhney, Jitendra P.S., Verma, Ishwar C., Altaey, Mays, Al-Jumaily, Khalid, Rasul, Dilshad, Abdalsahib, Ali Fawzi, Jabbar, Amer Abdl, Al-ageedi, Mohanad, Dhamin, Mohammed, AlFil, Sarmad, Khadhim, Foad, Miahy, Sabah, Agar, Ruth, Catapano, Alberico Luigi, Calandra, Sebastiano, Tarugi, Patrizia, Casula, Manuela, Galimberti, Federica, Olmastroni, Elena, Sarzani, Riccardo, Ferri, Claudio, Repetti, Elena, Piro, Salvatore, Suppressa, Patrizia, Meregalli, Giancarla, Borghi, Claudio, Muntoni, Sandro, Calabrò, Paolo, Cipollone, Francesco, Purrello, Francesco, Pujia, Arturo, Passaro, Angelina, Marcucci, Rossella, Pecchioli, Valerio, Pisciotta, Livia, Mandraffino, Giuseppe, Pellegatta, Fabio, Mombelli, Giuliana, Branchi, Adriana, Fiorenza, Anna Maria, Pederiva, Cristina, Werba, Josè Pablo, Parati, Gianfranco, Carubbi, Francesca, Iughetti, Lorenzo, Fortunato, Giuliana, Iannuzzi, Arcangelo, Iannuzzo, Gabriella, Cefalù, Angelo Baldassare, Biasucci, Giacomo, Zambon, Sabina, Pirro, Matteo, Sbrana, Francesco, Trenti, Chiara, D'Erasmo, Laura, Federici, Massimo, Ben, Maria Del, Bartuli, Andrea, Giaccari, Andrea, Pipolo, Antonio, Citroni, Nadia, Guardamagna, Ornella, Lia, Salvatore, Benso, Andrea, Biolo, Gianni, Maroni, Lorenzo, Lupi, Alessandro, Bonanni, Luca, Rinaldi, Elisabetta, Zenti, Maria Grazia, Matsuki, Kota, Hori, Mika, Ogura, Masatsune, Masuda, Daisaku, Kobayashi, Takuya, Nagahama, Kumiko, Al-Jarallah, Mohammed, Radovic, Mirjana, Lunegova, Olga, Bektasheva, Erkayim, Abilova, Saamay, Erglis, Andrejs, Gilis, Dainus, Nesterovics, Georgijs, Saripo, Vita, Meiere, Ruta, Skudrina, Gunda, Terauda, Elizabete, Jambart, Selim, Ayoub, Carine, Ghaleb, Youmna, Aliosaitiene, Urte, Kutkiene, Sandra, Abdul Kadir, Siti Hamimah Sheikh, Kasim, Noor Alicezah Mohd, Nor, Noor Shafina Mohd, Abdul Hamid, Hasidah, Abdul Razak, Suraya, Al-Khateeb, Alyaa, Abd Muid, Suhaila, Abdul Rahman, Thuhairah, Kasim, Sazzli Shahlan, Radzi, Ahmad Bakhtiar Md, Ibrahim, Khairul Shafiq, Rosli, Marshima Mohd, Razali, Rafezah, Chua, Yung An, Razman, Aimi Zafira, Nazli, Sukma Azureen, Aziz, Nazirul, Rosman, Azhari, Abdul Murad, NorAzian, Jalaludin, Mohd Amin, Abdul Latif, Ahmad Zubaidi, Azzopardi, C., Mehta, Roopa, Martagon, Alexandro J., Ramirez, Gabriela A. Galan, Villa, Neftali E Antonio, Vazquez, Arsenio Vargas, Elias-Lopez, Daniel, Retana, Gustavo Gonzalez, Rodriguez, Betsabel, Macías, Jose J. Ceballos, Zazueta, Alejandro Romero, Alvarado, Rocio Martinez, Portano, Julieta D. Morales, Lopez, Humberto Alvares, Sauque-Reyna, Leobardo, Herrera, Laura G. Gomez, Mendia, Luis E. Simental, Aguilar, Humberto Garcia, Cooremans, Elizabeth Ramirez, Aparicio, Berenice Peña, Zubieta, Victoria Mendoza, Gonzalez, Perla A. Carrillo, Ferreira-Hermosillo, Aldo, Portilla, Nacu Caracas, Dominguez, Guadalupe Jimenez, Garcia, Alinna Y. Ruiz, Cazares, Hector E. Arriaga, Gonzalez, Jesus R., Valencia, Carla V. Mendez, Padilla, Francisco G., Prado, Ramon Madriz, Ibarra, Manuel O. De los Rios, Villicaña, Ruy D. Arjona, Rivera, Karina J. Acevedo, Carrera, Ricardo Allende, Alvarez, Jose A., Martinez, Jose C. Amezcua, Bustillo, Manuel de los Reyes Barrera, Vargas, Gonzalo Carazo, Chacon, Roberto Contreras, Andrade, Mario H. Figueroa, Ortega, Ashanty Flores, Alcala, Hector Garcia, de Leon, Laura E. Garcia, Guzman, Berenice Garcia, Garcia, Jose J. Garduño, Cuellar, Juan C. Garnica, Cruz, Jose R. Gomez, Garcia, Anell Hernandez, Almada, Jesus R. Holguin, Herrera, Ursulo Juarez, Sobrevilla, Fabiola Lugo, Rodriguez, Eduardo Marquez, Sibaja, Cristina Martinez, Rodriguez, Alma B. Medrano, Oyervides, Jose C. Morales, Vazquez, Daniel I. Perez, Rodriguez, Eduardo A. Reyes, Osorio, Ma. Ludivina Robles, Saucedo, Juan Rosas, Tamayo, Margarita Torres, Talavera, Luis A. Valdez, Arroyo, Luis E. Vera, Carrillo, Eloy A. Zepeda, Stroes, Erik S, Defesche, J, Zuurbier, L, Reeskamp, L, Ibrahim, S, Roeters van Lennep, Jeanine, Wiegman, Albert, Isara, Alphonsus, Obaseki, Darlington E., Al-Waili, Khalid, Al-Zadjali, Fahad, Al-Zakwani, Ibrahim, Al-Kindi, Mohammed, Al-Mukhaini, Suad, Al-Barwani, Hamida, Rana, Asim, Shah, Lahore Saeed Ullah, Al-Nouri, Fahad, Starostecka, Ewa, Konopka, Agnieszka, Bielecka-Dabrowa, Agata, Lewek, Joanna, Sosnowska, Bozena, Gąsior, Mariusz, Dyrbuś, Krzysztof, Jóźwiak, Jacek, Pajkowski, Marcin, Romanowska-Kocejko, Marzena, Żarczyńska-Buchowiecka, Marta, Chmara, Magdalena, Wasąg, Bartosz, Stróżyk, Aneta, Michalska-Grzonkowska, Aleksandra, Medeiros, Ana Margarida, Alves, Ana Catarina, Silva, Francisco, Lobarinhas, Goreti, Palma, Isabel, de Moura, Jose Pereira, Rico, Miguel Toscano, Rato, Quitéria, Pais, Patrícia, Correia, Susana, Moldovan, Oana, Virtuoso, Maria João, Araujo, Francisco, Salgado, Jose Miguel, Colaço, Ines, Dumitrescu, Andreea, Lengher, Calin, Mosteoru, Svetlana, Meshkov, Alexey, Ershova, Alexandra, Rozhkova, Tatiana, Korneva, Victoria, Yu, Kuznetsova T., Zafiraki, Vitaliy, Voevoda, Mikhail, Gurevich, Victor, Duplyakov, Dmitry, Ragino, Yulia, Chubykina, Uliana, Shaposhnik, Igor, Alkaf, Fahmi, Khudari, Alia, Rwaili, Nawal, Al-Allaf, Faisal, Alghamdi, Mohammad, Batais, Mohammed A, Almigbal, Turky H, Kinsara, Abdulhalim, AlQudaimi, Ashraf Hammouda Ahmed, Awan, Zuhier, Elamin, Omer A, Altaradi, Hani, Popovic, Ljiljana, Singh, Sandra, Rasulic, Iva, Petakov, Ana, Lalic, Nebojsa M., Lam, Carolyn, Le, Tan Ju, Siang, Eric Lim Tien, Dissanayake, Sanjaya, I-Shing, Justin Tang, Shyong, Tai E, Jin, Terrance Chua Siang, Ting, Sharon Pek Li, Ming, Jeremy Hoe Kian, Drum, Chester Lee, Nastar, Fathima Ashna, Jia, Loh Wann, Ya, Natalie Koh Si, Jie, Marvin Chua Wei, Dalan, Rinkoo, Wei, Yong Quek, sian, Tiong Yee, Keong, Yeo Khung, Rong, Siau Kai, Jin, Darren Seah Ee, Ming, Ian Koh Jan, Chang, Tan Hong, Peng, Fabian Yap Kok, Vasanwala, Rashida Farhad, Raslova, Katarina, Balinth, Karin, Buganova, Ingrid, Fabryova, Lubomira, Kadurova, Michaela, Klabnik, Alexander, Kozárová, Miriam, Sirotiakova, Jana, Battelino, Tadej, Cevc, Matija, Debeljak, Marusa, Torkar, Ana Drole, Fras, Zlatko, Jug, Borut, Cugalj, Barbara Kern, Kovac, Jernej, Mlinaric, Matej, Sikonja, Jaka, Pilcher, Gillian Joan, Blom, D J, Wolmarans, K H, Brice, B C, Muñiz-Grijalvo, Ovidio, Díaz-Díaz, Jose Luis, de Isla, Leopoldo Pérez, Fuentes, Francisco, Badimon, Lina, Martin, François, Miserez, Eleonore B., Shipton, Janine L., Ganokroj, Poranee, Chattranukulchai, Pairoj, Jiamjarasrungsi, Wiroj, Thongtang, Nuntakorn, Krittayaphong, Rungroj, Vathesatogkit, Prin, Sriphrapradang, Chutintorn, Phimphilai, Mattabhorn, Leelawattana, Rattana, Anthanont, Pimjai, Suraamornkul, Swangjit, Deerochanawong, Chaicharn, Senthong, Vichai, Torpongpun, Artit, Suteerayongprasert, Panuwat, Pengpong, Nawarat, Sathavarodom, Nattapol, Sunanta, Usanee, Porntharukchareon, Thachanun, Kiatpanabhikul, Phatharaporn, Kaewkrasaesin, Chatchon, Kongkit, Jaruwan, Umphonsathien, Mongkontida, Akbulut, Mehmet, Alici, Gökhan, Bayram, Fahri, Can, Levent Hürkan, Celik, Ahmet, Ceyhan, Ceyhun, Coskun, Fatma Yilmaz, Demir, Mesut, Demircan, Sabri, Dogan, Volkan, Durakoglugil, Emre, Dural, İbrahim Etem, Gedikli, Omer, Hacioglu, Aysa, Ildizli, Muge, Kilic, Salih, Kirilmaz, Bahadir, Kutlu, Merih, Oguz, Aytekin, Ozdogan, Oner, Onrat, Ersel, Ozer, Savas, Sabuncu, Tevfik, Sahin, Tayfun, Sivri, Fatih, Sonmez, Alper, Temizhan, Ahmet, Topcu, Selim, Tokgozoglu, Lale, Tuncez, Abdullah, Vural, Mirac, Yenercag, Mustafa, Yesilbursa, Dilek, Yigit, Zerrin, Yildirim, Aytul Belgi, Yildirir, Aylin, Yilmaz, Mehmet Birhan, Atallah, Bassam, Traina, Mahmoud, Sabbour, Hani, Abdul Hay, Dana, Luqman, Neama, Elfatih, Abubaker, Abdulrasheed, Arshad, Manla, Yosef, Kwok, See, DellOca, Nicolas, Alieva, Rano B., Fozilov, Khurshid G., Hoshimov, Shavkat U., Nizamov, Ulugbek I., Kan, Liliya E., Kim, Andrey R., Abdullaeva, Guzal J., Abdullaev, Alisher A., Do, Doan Loi, Nguyen, Mai Ngoc Thi, Kim, Ngoc Thanh, Le, Thanh Tung, Le, Hong An, and Ray, Kausik K.

Published
2024
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8. Estimating effects within nonlinear autoregressive models: a case study on the impact of child access prevention laws on firearm mortality

Author

Cefalu, Matthew, Schell, Terry, Griffin, Beth Ann, Smart, Rosanna, and Morral, Andrew

Subjects
Statistics - Methodology
Abstract

Autoregressive models are widely used for the analysis of time-series data, but they remain underutilized when estimating effects of interventions. This is in part due to endogeneity of the lagged outcome with any intervention of interest, which creates difficulty interpreting model coefficients. These problems are only exacerbated in nonlinear or nonadditive models that are common when studying crime, mortality, or disease. In this paper, we explore the use of negative binomial autoregressive models when estimating the effects of interventions on count data. We derive a simple approximation that facilitates direct interpretation of model parameters under any order autoregressive model. We illustrate the approach using an empirical simulation study using 36 years of state-level firearm mortality data from the United States and use the approach to estimate the effect of child access prevention laws on firearm mortality.

Published
2021

10. Reducing bias in difference-in-differences models using entropy balancing

Author

Cefalu, Matthew, Vegetabile, Brian G., Dworsky, Michael, Eibner, Christine, and Girosi, Federico

Subjects
Statistics - Methodology, Economics - Econometrics
Abstract

This paper illustrates the use of entropy balancing in difference-in-differences analyses when pre-intervention outcome trends suggest a possible violation of the parallel trends assumption. We describe a set of assumptions under which weighting to balance intervention and comparison groups on pre-intervention outcome trends leads to consistent difference-in-differences estimates even when pre-intervention outcome trends are not parallel. Simulated results verify that entropy balancing of pre-intervention outcomes trends can remove bias when the parallel trends assumption is not directly satisfied, and thus may enable researchers to use difference-in-differences designs in a wider range of observational settings than previously acknowledged., Comment: 20 pages, 7 figures, 4 tables

Published
2020

11. Familial hypercholesterolaemia in children and adolescents from 48 countries: a cross-sectional study

Author

Dharmayat, Kanika Inamdar, Vallejo-Vaz, Antonio J., Stevens, Christophe A.T., Brandts, Julia M., Lyons, Alexander R.M., Groselj, Urh, Abifadel, Marianne, Aguilar-Salinas, Carlos A., Alhabib, Khalid, Alkhnifsawi, Mutaz, Almahmeed, Wael, Alnouri, Fahad, Alonso, Rodrigo, Al-Rasadi, Khalid, Ashavaid, Tester F., Banach, Maciej, Béliard, Sophie, Binder, Christoph, Bourbon, Mafalda, Chlebus, Krzysztof, Corral, Pablo, Cruz, Diogo, Descamps, Olivier S., Drogari, Euridiki, Durst, Ronen, Ezhov, Marat V., Genest, Jacques, Harada-Shiba, Mariko, Holven, Kirsten B., Humphries, Steve E., Khovidhunkit, Weerapan, Lalic, Katarina, Laufs, Ulrich, Liberopoulos, Evangelos, Roeters van Lennep, Jeanine, Lima-Martinez, Marcos Miguel, Lin, Jie, Maher, Vincent, März, Winfried, Miserez, André R., Mitchenko, Olena, Nawawi, Hapizah, Panayiotou, Andrie G., Paragh, György, Postadzhiyan, Arman, Reda, Ashraf, Reiner, Željko, Reyes, Ximena, Sadiq, Fouzia, Sahebkar, Amirhossein, Schunkert, Heribert, Shek, Aleksandr B., Stroes, Eric, Su, Ta-Chen, Subramaniam, Tavintharan, Susekov, Andrey, Vázquez Cárdenas, Alejandra, Huong Truong, Thanh, Tselepis, Alexandros D., Vohnout, Branislav, Wang, Luya, Yamashita, Shizuya, Al-Sarraf, Ahmad, Al-Sayed, Nasreen, Davletov, Kairat, Dwiputra, Bambang, Gaita, Dan, Kayikcioglu, Meral, Latkovskis, Gustavs, Marais, A. David, Thushara Matthias, Anne, Mirrakhimov, Erkin, Nordestgaard, Børge G., Petrulioniene, Zaneta, Pojskic, Belma, Sadoh, Wilson, Tilney, Myra, Tomlinson, Brian, Tybjærg-Hansen, Anne, Viigimaa, Margus, Catapano, Alberico L., Freiberger, Tomas, Hovingh, G. Kees, Mata, Pedro, Soran, Handrean, Raal, Frederick, Watts, Gerald F., Schreier, Laura, Bañares, Virginia, Greber-Platzer, Susanne, Baumgartner-Kaut, Margot, de Gier, Charlotte, Dieplinger, Hans, Höllerl, Florian, Innerhofer, Reinhold, Karall, Daniela, Lischka, Julia, Ludvik, Bernhard, Mäser, Martin, Scholl-Bürgi, Sabine, Thajer, Alexandra, Toplak, Hermann, Demeure, Fabian, Mertens, Ann, Balligand, Jean-Luc, Stephenne, Xavier, Sokal, Etienne, Petrov, Ivo, Goudev, Assen, Nikolov, Fedya, Tisheva, Snejana, Yotov, Yoto, Tzvetkov, Ivajlo, Hegele, Robert A, Gaudet, Daniel, Brunham, Liam, Ruel, Isabelle, McCrindle, Brian, Cuevas, Ada, Perica, Dražen, Symeonides, Phivos, Trogkanis, Efstratios, Kostis, Andreas, Ioannou, Andreas, Mouzarou, Angeliki, Georgiou, Anthoula, Stylianou, Andreas, Miltiadous, George, Iacovides, Paris, Deltas, Constantinos, Vrablik, Michal, Urbanova, Zuzana, Jesina, Pavel, Tichy, Lukas, Hyanek, Josef, Dvorakova, Jana, Cepova, Jana, Sykora, Josef, Buresova, Kristyna, Pipek, Michal, Pistkova, Eva, Bartkova, Ivana, S|ulakova, Astrid, Toukalkova, Lenka, Spenerova, Michaela, Maly, Jan, Benn, Marianne, Bendary, Ahmed, Elbahry, Atef, Ferrières, Jean, Ferrieres, Dorota, Peretti, Noel, Bruckert, Eric, Gallo, Antonio, Valero, René, Mourre, Florian, Aouchiche, Karine, Reynaud, Rachel, Tounian, Patrick, Lemale, Julie, Boccara, Franck, Moulin, Philippe, Charrières, Sybil, Di Filippo, Mathilde, Cariou, Bertrand, Paillard, François, Dourmap, Caroline, Pradignac, Alain, Verges, Bruno, Simoneau, Isabelle, Farnier, Michel, Cottin, Yves, Yelnik, Cecile, Hankard, Regis, Schiele, François, Durlach, Vincent, Sultan, Ariane, Carrié, Alain, Rabès, Jean-Pierre, Sanin, Veronika, Schmieder, Raphael S., Ates, Sara, Rizos, Christos V., Skoumas, Ioannis, Tziomalos, Konstantinos, Rallidis, Loukianos, Kotsis, Vasileios, Doumas, Michalis, Skalidis, Emmanouil, Kolovou, Genovefa, Kolovou, Vana, Garoufi, Anastasia, Koutagiar, Iosif, Polychronopoulos, Georgios, Kiouri, Estela, Antza, Christina, Zacharis, Evangelos, Attilakos, Achilleas, Sfikas, George, Koumaras, Charalambos, Anagnostis, Panagiotis, Anastasiou, Georgia, Liamis, George, Adamidis, Petros-Spyridon, Milionis, Haralambos, Lambadiari, Vaia, Stabouli, Stella, Filippatos, Theodosios, Mollaki, Vicky, Tsaroumi, Anastasia, Lamari, Frida, Proyias, Pavlos, Harangi, Mariann, Reddy, Lakshmi Lavanya, Shah, Swarup A. V, Ponde, Chandrashekhar K., Dalal, Jamshed J., Sawhney, Jitendra P.S., Verma, Ishwar C., Hosseini, Susan, Jamialahmadi, Tannaz, Alareedh, Mohammed, Shaghee, Foaad, Rhadi, Sabah Hasan, Abduljalal, Maryam, Alfil, Sarmad, Kareem, Huda, Cohen, Hofit, Leitersdorf, Eran, Schurr, Daniel, Shpitzen, Shoshi, Arca, Marcello, Averna, Maurizio, Bertolini, Stefano, Calandra, Sebastiano, Tarugi, Patrizia, Casula, Manuela, Galimberti, Federica, Gazzotti, Marta, Olmastroni, Elena, Sarzani, Riccardo, Ferri, Claudio, Repetti, Elena, Giorgino, Francesco, Suppressa, Patrizia, Bossi, Antonio Carlo, Borghi, Claudio, Muntoni, Sandro, Cipollone, Francesco, Scicali, Roberto, Pujia, Arturo, Passaro, Angelina, Berteotti, Martina, Pecchioli, Valerio, Pisciotta, Livia, Mandraffino, Giuseppe, Pellegatta, Fabio, Mombelli, Giuliana, Branchi, Adriana, Fiorenza, Anna Maria, Pederiva, Cristina, Werba, José Pablo, Parati, Gianfranco, Nascimbeni, Fabio, Iughetti, Lorenzo, Fortunato, Giuliana, Cavallaro, Raimondo, Iannuzzo, Gabriella, Calabrò, Paolo, Cefalù, Angelo Baldassare, Capra, Maria Elena, Zambon, Alberto, Pirro, Matteo, Sbrana, Francesco, Trenti, Chiara, Minicocci, Ilenia, Federici, Massimo, Del Ben, Maria, Buonuomo, Paola Sabrina, Moffa, Simona, Pipolo, Antonio, Citroni, Nadia, Guardamagna, Ornella, Lia, Salvatore, Benso, Andrea, Biolo, Gianni Biolo, Maroni, Lorenzo, Lupi, Alessandro, Bonanni, Luca, Rinaldi, Elisabetta, Zenti, Maria Grazia, Masuda, Daisaku, Mahfouz, Linda, Jambart, Selim, Ayoub, Carine, Ghaleb, Youmna, Kasim, Noor Alicezah Mohd, Nor, Noor Shafina Mohd, Al-Khateeb, Alyaa, Kadir, Siti Hamimah Sheikh Abdul, Chua, Yung-An, Razman, Aimi Zafira, Nazli, Sukma Azureen, Ranai, Norashikin Mohd, Latif, Ahmad Zubaidi Abd, Torres, María Teresa Magaña, Mehta, Roopa, Martagon, Alexandro J., Ramirez, Gabriela A. Galan, Antonio-Villa, Neftali Eduardo, Vargas-Vazquez, Arsenio, Elias-Lopez, Daniel, Retana, Gustavo Gonzalez, Encinas, Bethsabel Rodrıguez, Macıas, Jose J. Ceballos, Zazueta, Alejandro Romero, Alvarado, Rocio Martinez, Portano, Julieta D. Morales, Lopez, Humberto Alvares, Sauque-Reyna, Leobardo, Gomez Herrera, Laura G., Simental Mendia, Luis E., Aguilar, Humberto Garcia, Cooremans, Elizabeth Ramirez, Aparicio, Berenice Pe~na, Zubieta, Victoria Mendoza, Gonzalez, Perla A. Carrillo, Ferreira-Hermosillo, Aldo, Portilla, Nacu Caracas, Dominguez, Guadalupe Jimenez, Garcia, Alinna Y. Ruiz, Arriaga Cazares, Hector E., Gonzalez Gonzalez, Jesus R., Mendez Valencia, Carla V., Padilla Padilla, Francisco G., Prado, Ramon Madriz, De los Rios Ibarra, Manuel O., Arjona Villica~na, Ruy D., Acevedo Rivera, Karina J., Carrera, Ricardo Allende, Alvarez, Jose A., Amezcua Martinez, Jose C., Barrera Bustillo, Manuel de los Reyes, Vargas, Gonzalo Carazo, Chacon, Roberto Contreras, Figueroa Andrade, Mario H., Ortega, Ashanty Flores, Alcala, Hector Garcia, Garcia de Leon, Laura E., Guzman, Berenice Garcia, Gardu~no Garcia, Jose J., Garnica Cuellar, Juan C., Gomez Cruz, Jose R., Garcia, Anell Hernandez, Holguin Almada, Jesus R., Herrera, Ursulo Juarez, Sobrevilla, Fabiola Lugo, Rodriguez, Eduardo Marquez, Sibaja, Cristina Martinez, Medrano Rodriguez, Alma B., Morales Oyervides, Jose C., Perez Vazquez, Daniel I., Reyes Rodriguez, Eduardo A., Osorio, Ma. Ludivina Robles, Saucedo, Juan Rosas, Tamayo, Margarita Torres, Valdez Talavera, Luis A., Vera Arroyo, Luis E., Zepeda Carrillo, Eloy A., Galema-Boers, Annette, Weigman, Albert, Bogsrud, Martin P., Malik, Munir, Shah, Saeedullah, Khan, Sabeen Abid, Rana, Muhammad Asim, Batool, Hijab, Starostecka, Ewa, Konopka, Agnieszka, Lewek, Joanna, Bielecka-Dąbrowa, Agata, Gach, Agnieszka, Jóźwiak, Jacek, Pajkowski, Marcin, Romanowska-Kocejko, Marzena, Żarczyńska-Buchowiecka, Marta, Hellmann, Marcin, Chmara, Magdalena, Wasąg, Bartosz, Parczewska, Aleksandra, Gilis-Malinowska, Natasza, Borowiec-Wolna, Justyna, Stróżyk, Aneta, Michalska-Grzonkowska, Aleksandra, Chlebus, Izabela, Kleinschmidt, Mariola, Wojtecka, Agnieszka, Zdrojewski, Tomasz, Myśliwiec, Małgorzata, Hennig, Matylda, Medeiros, Ana Margarida, Alves, Ana Catarina, Almeida, Ana Filipa, Lopes, Andreia, Guerra, António, Bilhoto, Carla, Simões, Fernando, Silva, Francisco, Lobarinhas, Goreti, Gama, Guida, Palma, Isabel, Salgado, José Miguel, Matos, Luísa Diogo, Moura, Márcio de, Virtuoso, Maria João, Tavares, Mónica, Ferreira, Patrícia, Pais, Patrícia, Garcia, Paula, Coelho, Raquel, Ribeiro, Raquel, Correia, Susana, Sadykova, Dinara, Slastnikova, Evgenia, Alammari, Dalal, Mawlawi, Horia Ahmed, Alsahari, Atif, Khudary, Alia Abdullah, Alrowaily, Nawal Lafi, Rajkovic, Natasa, Popovic, Ljiljana, Singh, Sandra, Rasulic, Iva, Petakov, Ana, Lalic, Nebojsa M., Peng, Fabian Kok, Vasanwala, Rashida Farhan, Venkatesh, Sreedharan Aravind, Raslova, Katarina, Fabryova, Lubomira, Nociar, Jan, Šaligova, Jana, Potočňáková, Ludmila, Kozárová, Miriam, Varga, Tibor, Kadurova, Michaela, Debreova, Marianna, Novodvorsky, Peter, Gonova, Katarina, Klabnik, Alexander, Buganova, Ingrid, Battelino, Tadej, Bizjan, Barbara Jenko, Debeljak, Marusa, Kovac, Jernej, Mlinaric, Matej, Molk, Neza, Sikonja, Jaka, Sustar, Ursa, Podkrajsek, Katarina Trebusak, Muñiz-Grijalvo, Ovidio, Díaz-Díaz, Jose Luis, de Andrés, Raimundo, Fuentes-Jiménez, Francisco, Blom, Dirk, Miserez, Eleonore B., Shipton, Janine L., Ganokroj, Poranee, Futema, Marta, Ramaswami, Uma, Alieva, Rano B., Fozilov, Khurshid G., Khoshimov, Shavkat U., Nizamov, Ulugbek I., Abdullaeva, Guzal J., Kan, Liliya E., Abdullaev, Alisher A., Zakirova, Daria V., Do, Doan-Loi, Nguyen, Mai-Ngoc-Thi, Kim, Ngoc-Thanh, Le, Thanh-Tung, Le, Hong-An, Santos, Raul, and Ray, Kausik K.

Published
2024
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14. Nonparametric Estimation of Population Average Dose-Response Curves using Entropy Balancing Weights for Continuous Exposures

Author

Vegetabile, Brian G., Griffin, Beth Ann, Coffman, Donna L., Cefalu, Matthew, and McCaffrey, Daniel F.

Subjects
Statistics - Methodology
Abstract

Weighted estimators are commonly used for estimating exposure effects in observational settings to establish causal relations. These estimators have a long history of development when the exposure of interest is binary and where the weights are typically functions of an estimated propensity score. Recent developments in optimization-based estimators for constructing weights in binary exposure settings, such as those based on entropy balancing, have shown more promise in estimating treatment effects than those methods that focus on the direct estimation of the propensity score using likelihood-based methods. This paper explores recent developments of entropy balancing methods to continuous exposure settings and the estimation of population dose-response curves using nonparametric estimation combined with entropy balancing weights, focusing on factors that would be important to applied researchers in medical or health services research. The methods developed here are applied to data from a study assessing the effect of non-randomized components of an evidence-based substance use treatment program on emotional and substance use clinical outcomes., Comment: 26 pages, 10 figures

Published
2020

15. Refinement of the diagnostic approach for the identification of children and adolescents affected by familial hypercholesterolemia: Evidence from the LIPIGEN study

Author

Anesi, Adriano, Arca, Marcello, Auricchio, Renata, Averna, Maurizio, Baldera, Davide, Banderali, Giuseppe, Beccuti, Guglielmo, Benso, Andrea, Berteotti, Martina, Bertolini, Stefano, Bianconi, Vanessa, Biasucci, Giacomo, Biolo, Gianni, Bonanni, Luca, Borghi, Claudio, Bossi, Antonio Carlo, Branchi, Adriana, Bruzzi, Patrizia, Bucci, Marco, Buonuomo, Paola Sabrina, Calabrò, Paolo, Calandra, Sebastiano, Carubbi, Francesca, Cavallaro, Raimondo, Cefalù, Angelo Baldassarre, Cesaro, Arturo, Cipollone, Francesco, Citroni, Nadia, Colombo, Emanuela, Coppola, Chiara, D'Addato, Sergio, Dal Pino, Beatrice, Dalla Nora, Edoardo, De Corrado, Giuseppe, Del Ben, Maria, Di Molfetta, Sergio, Di Taranto, Maria Donata, Fainelli, Giulia, Federici, Massimo, Ferri, Claudio, Fiorenza, Anna Maria, Formisano, Elena, Fortunato, Giuliana, Giaccari, Andrea, Giorgino, Francesco, Grigore, Liliana, Guardamagna, Ornella, Iannuzzi, Arcangelo, Iannuzzo, Gabriella, Iughetti, Lorenzo, Lia, Salvatore, Longo, Susanna, Lupi, Alessandro, Mandraffino, Giuseppe, Marcucci, Rossella, Maroni, Lorenzo, Massini, Giulia, Mazza, Elisa, Melchioda, Elena, Meregalli, Giancarla, Minicocci, Ilenia, Moffa, Simona, Mombelli, Giuliana, Muntoni, Sandro, Nascimbeni, Fabio, Negri, Emanuele Alberto, Notargiacomo, Serena, Panfili, Filippo Maria, Parati, Gianfranco, Passaro, Angelina, Pavanello, Chiara, Pecchioli, Valerio, Pecchioli, Lorenzo, Pellegatta, Fabio, Perla, Francesco Massimo, Pipolo, Antonio, Piro, Salvatore, Pirro, Matteo, Pisciotta, Livia, Pujia, Roberta, Putotto, Carolina, Repetti, Elena, Rinaldi, Elisabetta, Romandini, Alessandra, Sani, Elena, Sarnari, Silvia, Sarzani, Riccardo, Sbrana, Francesco, Scicali, Roberto, Scuruchi, Michele, Suppressa, Patrizia, Tarugi, Patrizia, Trenti, Chiara, Vinci, Pierandrea, Werba, José Pablo, Zambon, Sabina, Zambon, Alberto, Zenti, Maria Grazia, Casula, Manuela, Gazzotti, Marta, Capra, Maria Elena, Olmastroni, Elena, Galimberti, Federica, Catapano, Alberico L., and Pederiva, Cristina

Published
2023
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18. Synthetic estimation for the complier average causal effect

Author

Agniel, Denis, Han, Bing, and Cefalu, Matthew

Subjects
Statistics - Methodology
Abstract

We propose an improved estimator of the complier average causal effect (CACE). Researchers typically choose a presumably-unbiased estimator for the CACE in studies with noncompliance, when many other lower-variance estimators may be available. We propose a synthetic estimator that combines information across all available estimators, leveraging the efficiency in lower-variance estimators while maintaining low bias. Our approach minimizes an estimate of the mean squared error of all convex combinations of the candidate estimators. We derive the asymptotic distribution of the synthetic estimator and demonstrate its good performance in simulation, displaying a robustness to inclusion of even high-bias estimators.

Published
2019

19. Averaging causal estimators in high dimensions

Author

Antonelli, Joseph and Cefalu, Matthew

Subjects
Statistics - Methodology
Abstract

There has been increasing interest in recent years in the development of approaches to estimate causal effects when the number of potential confounders is prohibitively large. This growth in interest has led to a number of potential estimators one could use in this setting. Each of these estimators has different operating characteristics, and it is unlikely that one estimator will outperform all others across all possible scenarios. Coupling this with the fact that an analyst can never know which approach is best for their particular data, we propose a synthetic estimator that averages over a set of candidate estimators. Averaging is widely used in statistics for problems such as prediction, where there are many possible models, and averaging can improve performance and increase robustness to using incorrect models. We show that these ideas carry over into the estimation of causal effects in high-dimensional scenarios. We show theoretically that averaging provides robustness against choosing a bad model, and show empirically via simulation that the averaging estimator performs quite well, and in most cases nearly as well as the best among all possible candidate estimators. Finally, we illustrate these ideas in an environmental wide association study and see that averaging provides the largest benefit in the more difficult scenarios that have large numbers of confounders.

Published
2019

20. Heterogeneity of Diabetes: β-Cells, Phenotypes, and Precision Medicine: Proceedings of an International Symposium of the Canadian Institutes of Health Research’s Institute of Nutrition, Metabolism and Diabetes and the U.S. National Institutes of Health’s National Institute of Diabetes and Digestive and Kidney Diseases

Author

Rosenblum, Norman, Cefalu, William, Andersen, Dana K., Arreaza-Rubín, Guillermo, Dhara, Christine, James, Stephen P., Makarchuk, Mary-Jo, Pin, Christopher L., Sato, Sheryl, Verchere, Bruce, Woo, Minna, Powers, Alvin, Estall, Jennifer, Hoesli, Corrine, Millman, Jeffrey, Linnemann, Amelia, Johnson, James, Hawkins, Meredith, Gloyn, Anna, Huising, Mark O., Benninger, Richard K.P., Almaça, Joana, Hull-Meichle, Rebecca L., MacDonald, Patrick, Lynn, Francis, Melero-Martin, Juan, Yoshihara, Eiji, Stabler, Cherie, Sander, Maike, Evans-Molina, Carmella, Engin, Feyza, Thompson, Peter, Shalev, Anath, Redondo, Maria J., Nadeau, Kristen, Bellin, Melena, Udler, Miriam S., Dennis, John, Dash, Satya, Zhou, Wenyu, Snyder, Michael, Booth, Gillian, Butte, Atul, Florez, Jose, Cefalu, William T., Verchere, C. Bruce, and Rosenblum, Norman D.

Published
2021
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21. WED-336 RESIST-NASH: a regional network for identification and referral of masld patients at risk for liver fibrosis

Author

Pennisi, Grazia, primary, Buscemi, Silvio, additional, Bertino, Gaetano, additional, Frittitta, Lucia, additional, Purrello, Francesco, additional, Raimondo, Giovanni, additional, Cartabellotta, Fabio, additional, Russello, Maurizio, additional, Malizia, Giuseppe, additional, Carroccio, Antonio, additional, Tuttolomondo, Antonino, additional, Camilleri, Salvatore, additional, Magrì, Giovanni, additional, Licata, Anna, additional, Cefalù, Angelo Baldassare, additional, Distefano, Marco, additional, Giordano, Carla, additional, Iaria, Chiara, additional, Scalisi, Ignazio, additional, Tullio, Prestileo, additional, Arpi, Maria Luisa, additional, Marco, Vito Di, additional, Camma, Calogero, additional, and Petta, Salvatore, additional

Published
2024
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25. Worldwide experience of homozygous familial hypercholesterolaemia: retrospective cohort study

Author

Tromp, Tycho R., Hartgers, Merel L., Hovingh, G. Kees, Vallejo-Vaz, Antonio J., Ray, Kausik K., Soran, Handrean, Freiberger, Tomas, Bertolini, Stefano A., Harada-Shiba, Mariko, Pang, Jing, Watts, Gerald F., Greber-Platzer, Susanne, Mäser, Martin, Stulnig, Thomas M., Ebenbichler, Christoph F., Bin Thani, Khalid, Cassiman, David, Descamps, Olivier S., Rymen, Daisy, Witters, Peter, Santos, Raul D., Brunham, Liam R., Francis, Gordon A., Genest, Jacques, Hegele, Robert A., Kennedy, Brooke A., Ruel, Isabelle, Sherman, Mark H., Jiang, Long, Wang, Luya, Reiner, Željko, Blaha, Vladimir, Ceska, Richard, Dvorakova, Jana, Dlouhy, Lubomir, Horak, Pavel, Soska, Vladimir, Tichy, Lukas, Urbanek, Robin, Vaverkova, Helena, Vrablik, Michal, Zemek, Stanislav, Zlatohlavek, Lukas, Emil, Sameh, Naguib, Tarek, Reda, Ashraf, Béliard, Sophie, Bruckert, Eric, Gallo, Antonio, Elisaf, Moses S., Kolovou, Genovefa, Cohen, Hofit, Durst, Ronen, Dann, Eldad J., Elis, Avishay, Hussein, Osama, Leitersdorf, Eran, Schurr, Daniel, Setia, Nitika, Verma, Ishwar C., Alareedh, Mohammed D., Al-Khnifsawi, Mutaz, Abdalsahib Al-Zamili, Ali F., Rhadi, Sabah H., Shaghee, Foaad K., Arca, Marcello, Averna, Maurizio, Bartuli, Andrea, Bucci, Marco, Buonuomo, Paola S., Calabrò, Paolo, Calandra, Sebastiano, Casula, Manuela, Catapano, Alberico L., Cefalù, Angelo B., Cicero, Arrigo F.G., D'Addato, Sergio, D'Erasmo, Laura, Di Costanzo, Alessia, Fasano, Tommaso, Gazzotti, Marta, Giammanco, Antonina, Iannuzzo, Gabriella, Ibba, Anastasia, Negri, Emanuele A., Pasta, Andrea, Pavanello, Chiara, Pisciotta, Livia, Rabacchi, Claudio, Ripoli, Carlo, Sampietro, Tiziana, Sbrana, Francesco, Sileo, Fulvio, Suppressa, Patrizia, Tarugi, Patrizia, Trenti, Chiara, Zenti, Maria G., Hori, Mika, Ayesh, Mahmoud H., Azar, Sami T., Bitar, Fadi F., Fahed, Akl C., Moubarak, Elie M., Nemer, Georges, Nawawi, Hapizah M., Madriz, Ramón, Mehta, Roopa, Cupido, Arjen J., Defesche, Joep C., Reijman, M. Doortje, Roeters-van Lennep, Jeanine E., Stroes, Erik S.G., Wiegman, Albert, Zuurbier, Linda, Al-Waili, Khalid, Sadiq, Fouzia, Chlebus, Krzysztof, Bourbon, Mafalda, Gaspar, Isabel M., Lalic, Katarina S., Ezhov, Marat V., Susekov, Andrey V., Groselj, Urh, Charng, Min-Ji, Khovidhunkit, Weerapan, Aktan, Melih, Altunkeser, Bulent B., Demircioglu, Sinan, Kose, Melis, Gokce, Cumali, Ilhan, Osman, Kayikcioglu, Meral, Kaynar, Leyla G., Kuku, Irfan, Kurtoglu, Erdal, Okutan, Harika, Ozcebe, Osman I., Pekkolay, Zafer, Sag, Saim, Salcioglu, Osman Z., Temizhan, Ahmet, Yenercag, Mustafa, Yilmaz, Mehmet, Yilmaz Yasar, Hamiyet, Mitchenko, Olena, Lyons, Alexander R.M., Stevens, Christophe A.T., Brothers, Julie A., Hudgins, Lisa C., Nguyen, Christina, Alieva, Rano, Shek, Aleksandr, Do, Doan-Loi, Kim, Ngoc-Thanh, Le, Hong-An, Le, Thanh-Tung, Nguyen, Mai-Ngoc T., Truong, Thanh-Huong, Blom, Dirk J., Raal, Frederick J., Cuchel, Marina, Tromp, Tycho R, Hartgers, Merel L, Hovingh, G Kees, Vallejo-Vaz, Antonio J, Ray, Kausik K, Bertolini, Stefano, Blom, Dirk J, and Raal, Frederick J

Published
2022
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26. Posterior Predictive Treatment Assignment for Estimating Causal Effects with Limited Overlap

Author

Zigler, Corwin M and Cefalu, Matthew

Subjects
Statistics - Methodology
Abstract

Estimating causal effects with propensity scores relies upon the availability of treated and untreated units observed at each value of the estimated propensity score. In settings with strong confounding, limited so-called "overlap" in propensity score distributions can undermine the empirical basis for estimating causal effects and yield erratic finite-sample performance of existing estimators. We propose a Bayesian procedure designed to estimate causal effects in settings where there is limited overlap in propensity score distributions. Our method relies on the posterior predictive treatment assignment (PPTA), a quantity that is derived from the propensity score but serves different role in estimation of causal effects. We use the PPTA to estimate causal effects by marginalizing over the uncertainty in whether each observation is a member of an unknown subset for which treatment assignment can be assumed unconfounded. The resulting posterior distribution depends on the empirical basis for estimating a causal effect for each observation and has commonalities with recently-proposed "overlap weights" of Li et al. (2016). We show that the PPTA approach can be construed as a stochastic version of existing ad-hoc approaches such as pruning based on the propensity score or truncation of inverse probability of treatment weights, and highlight several practical advantages including uncertainty quantification and improved finite-sample performance. We illustrate the method in an evaluation of the effectiveness of technologies for reducing harmful pollution emissions from power plants in the United States.

Published
2017

27. Doubly robust matching estimators for high dimensional confounding adjustment

Author

Antonelli, Joseph, Cefalu, Matthew, Palmer, Nathan, and Agniel, Denis

Subjects
Statistics - Methodology
Abstract

Valid estimation of treatment effects from observational data requires proper control of confounding. If the number of covariates is large relative to the number of observations, then controlling for all available covariates is infeasible. In cases where a sparsity condition holds, variable selection or penalization can reduce the dimension of the covariate space in a manner that allows for valid estimation of treatment effects. In this article, we propose matching on both the estimated propensity score and the estimated prognostic scores when the number of covariates is large relative to the number of observations. We derive asymptotic results for the matching estimator and show that it is doubly robust, in the sense that only one of the two score models need be correct to obtain a consistent estimator. We show via simulation its effectiveness in controlling for confounding and highlight its potential to address nonlinear confounding. Finally, we apply the proposed procedure to analyze the effect of gender on prescription opioid use using insurance claims data.

Published
2016

29. Long-term efficacy of lipoprotein apheresis and lomitapide in the treatment of homozygous familial hypercholesterolemia (HoFH): a cross-national retrospective survey

Author

Laura D’Erasmo, Antonio Gallo, Angelo Baldassare Cefalù, Alessia Di Costanzo, Samir Saheb, Antonina Giammanco, Maurizio Averna, Alessio Buonaiuto, Gabriella Iannuzzo, Giuliana Fortunato, Arturo Puja, Tiziana Montalcini, Chiara Pavanello, Laura Calabresi, Giovanni Battista Vigna, Marco Bucci, Katia Bonomo, Fabio Nota, Tiziana Sampietro, Francesco Sbrana, Patrizia Suppressa, Carlo Sabbà, Fabio Fimiani, Arturo Cesaro, Paolo Calabrò, Silvia Palmisano, Sergio D’Addato, Livia Pisciotta, Stefano Bertolini, Randa Bittar, Olga Kalmykova, Sophie Béliard, Alain Carrié, Marcello Arca, and Eric Bruckert

Subjects
Homozygous hypercholesterolemia, Lipoprotein apheresis, Lomitapide, LDL, Therapeutics, Genetic disease, Medicine
Abstract

Abstract Background Homozygous familial hypercholesterolemia (HoFH) is a rare life-threatening condition that represents a therapeutic challenge. The vast majority of HoFH patients fail to achieve LDL-C targets when treated with the standard protocol, which associates maximally tolerated dose of lipid-lowering medications with lipoprotein apheresis (LA). Lomitapide is an emerging therapy in HoFH, but its place in the treatment algorithm is disputed because a comparison of its long-term efficacy versus LA in reducing LDL-C burden is not available. We assessed changes in long-term LDL-C burden and goals achievement in two independent HoFH patients’ cohorts, one treated with lomitapide in Italy (n = 30) and the other with LA in France (n = 29). Results The two cohorts differed significantly for genotype (p = 0.004), baseline lipid profile (p

Published
2021
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32. Efficacy of Long-Term Treatment of Autosomal Recessive Hypercholesterolemia With Lomitapide: A Subanalysis of the Pan-European Lomitapide Study

Author

Laura D’Erasmo, Antonina Giammanco, Patrizia Suppressa, Chiara Pavanello, Gabriella Iannuzzo, Alessia Di Costanzo, Daniele Tramontano, Ilenia Minicocci, Simone Bini, Anja Vogt, Kim Stewards, Jeanine Roeters Van Lennep, Stefano Bertolini, Marcello Arca, the Italian and European Working Group on Lomitapide in HoFH, Maurizio Averna, Eric Boersma, Katia Bonomo, Marco Bucci, Laura Calabresi, Paolo Calabrò, Angelo Baldassare Cefalù, Jaimini Cegla, Arturo Cesaro, Sergio D’Addato, Eugene Daphnis, Maria Donata Di Taranto, Avishay Ellis, Fabio Fimiani, Giuliana Fortunato, Marco Gentile, Meral Kayikcioglu, Genovefa Kolovou, Evangelos Liberopoulos, Karin Littmann, Sergio Martínez-Hervás, Tiziana Montalcini, Fabio Nota, Livia Pisciotta, Arturo Puja, Giovanni José Real, Jeanine Roeters van Lennep, Joost Rutten, Carlo Sabbà, Tiziana Sampietro, Francesco Sbrana, Kim Steward, Fulvio Ventura, Battista Vigna, and Shahenaz Walji

Subjects
Real-world study, rare disease, autosomal recessive hypercholesterolaemia, LDL-C, lomitapide, long-term, Genetics, QH426-470
Abstract

Backgroundand aim: Autosomal recessive hypercholesterolemia (ARH) is a rare autosomal recessive disorder of low-density lipoprotein (LDL) metabolism caused by pathogenic variants in the LDLRAP1 gene. Like homozygous familial hypercholesterolemia, ARH is resistant to conventional LDL-lowering medications and causes a high risk of atherosclerotic cardiovascular diseases (ASCVDs) and aortic valve stenosis. Lomitapide is emerging as an efficacious therapy in classical HoFH, but few data are available for ARH.Results: This is a subanalysis carried out on nine ARH patients included in the Pan-European Lomitapide Study. The age at starting lomitapide was 46 (interquartile range (IQR), 39.0–65.5) years, with a median treatment duration of 31.0 (IQR 14.0–40.5) months. At baseline, four (44.4%) patients had hypertension, one (11.1%) had diabetes mellitus, two (22.2%) were active smokers, and five (55.5%) reported ASCVD. The baseline LDL-C was 257.0 (IQR, 165.3–309.2) mg/dL. All patients were on statins plus ezetimibe, three were receiving Lipoprotein apheresis (LA), and one was also receiving proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9i). The addition of lomitapide (mean dose, 10 mg) resulted in the achievement of a median on-treatment LDL-C of 101.7 mg/dL (IQR, 71.3–138.3; 60.4% reduction from baseline), with a best LDL-C value of 68.0 mg/dL (IQR, 43.7–86.7; 73.5% reduction from baseline). During follow-up, one patient stopped both PCSK9i and LA. Recurrence of ASCVD events was reported in one patient. The median on-treatment aspartate transaminase and alanine transaminase values were 31.1 (IQR, 22.6–48.3) U/L and 31.1 (IQR, 27.2–53.8) U/L, respectively. Among six ARH patients with available fibroscan examination, liver stiffness values recorded at the last visit were within the normal range (median, 4.7 KPa; IQR, 3.6–5.3 KPa).Conclusion: Lomitapide is effective and safe in ARH therapy as well as in classical HoFH.

Published
2022
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33. rs629301 CELSR2 polymorphism confers a ten-year equivalent risk of critical stenosis assessed by coronary angiography

Author

Noto, Davide, Cefalù, Angelo B., Martinelli, Nicola, Giammanco, Antonina, Spina, Rossella, Barbagallo, Carlo M., Caruso, Marco, Novo, Salvatore, Sarullo, Filippo, Pernice, Vincenzo, Brucato, Federica, Ingrassia, Valeria, Fayer, Francesca, Altieri, Grazia I., Scrimali, Chiara, Misiano, Gabriella, Olivieri, Oliviero, Girelli, Domenico, and Averna, Maurizio R.

Published
2021
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36. Effects of PCSK9 inhibitors on HDL cholesterol efflux and serum cholesterol loading capacity in familial hypercholesterolemia subjects: a multi-lipid-center real-world evaluation

Author

Marcella Palumbo, Antonina Giammanco, Francesco Purrello, Chiara Pavanello, Giuliana Mombelli, Antonino Di Pino, Salvatore Piro, Angelo Baldassare Cefalù, Laura Calabresi, Maurizio Averna, Franco Bernini, Francesca Zimetti, Maria Pia Adorni, and Roberto Scicali

Subjects
PCSK9 inhibitors, cholesterol efflux capacity, cholesterol loading capacity, familial hypercholesterolemia, cardiovascular risk, Biology (General), QH301-705.5
Abstract

Proprotein convertase subtilisin/kexin type 9 (PCSK9), beyond regulating LDL cholesterol (LDL-c) plasma levels, exerts several pleiotropic effects by modulating lipid metabolism in extrahepatic cells such as macrophages. Macrophage cholesterol homeostasis depends on serum lipoprotein functions, including the HDL capacity to promote cell cholesterol efflux (CEC) and the serum capacity to promote cell cholesterol loading (CLC). The aim of this observational study was to investigate the effect of PCSK9 inhibitors (PCSK9-i) treatment on HDL-CEC and serum CLC in patients with familial hypercholesterolemia (FH). 31 genetically confirmed FH patients were recruited. Blood was collected and serum isolated at baseline and after 6 months of PCSK9-i treatment. HDL-CEC was evaluated through the main pathways with a radioisotopic cell-based assay. Serum CLC was assessed fluorimetrically in human THP-1 monocyte-derived macrophages. After treatment with PCSK9-i, total cholesterol and LDL-c significantly decreased (−41.6%, p < 0.0001 and −56.7%, p < 0.0001, respectively). Total HDL-CEC was not different between patients before and after treatment. Conversely, despite no changes in HDL-c levels between the groups, ABCG1 HDL-CEC significantly increased after treatment (+22.2%, p < 0.0001) as well as HDL-CEC by aqueous diffusion (+7.8%, p = 0.0008). Only a trend towards reduction of ABCA1 HDL-CEC was observed after treatment. PCSK9-i significantly decreased serum CLC (−6.6%, p = 0.0272). This effect was only partly related to the reduction of LDL-c levels. In conclusion, PCSK9-i treatment significantly increased HDL-CEC through ABCG1 and aqueous diffusion pathways and reduced the serum CLC in FH patients. The favorable effect of PCSK9-i on functional lipid profile could contribute to the cardiovascular benefit of these drugs in FH patients.

Published
2022
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38. Homozygous familial hypercholesterolemia in Italy: Clinical and molecular features

Author

Bartuli, Andrea, Bucci, Marco, Buonuomo, Paola Sabrina, Calabrò, Paolo, Casula, Manuela, Cefalù, Angelo Baldassare, Cicero, Arrigo, D'Addato, Sergio, D'Erasmo, Laura, Fasano, Tommaso, Iannuzzo, Gabriella, Ibba, Anastasia, Negri, Emanuele A., Pasta, Andrea, Pavanello, Chiara, Pisciotta, Livia, Rabacchi, Claudio, Ripoli, Carlo, Sampietro, Tiziana, Sbrana, Francesco, Sileo, Fulvio, Suppressa, Patrizia, Trenti, Chiara, Zenti, Maria Grazia, Bertolini, Stefano, Calandra, Sebastiano, Arca, Marcello, Averna, Maurizio, Catapano, Alberico L., and Tarugi, Patrizia

Published
2020
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39. Lomitapide does not alter PCSK9 and Lp(a) levels in homozygous familial hypercholesterolemia patients: Analysis on cytokines and lipid profile

Author

Maria Giovanna Lupo, Diletta Arcidiacono, Alice Zaramella, Fabio Fimiani, Paolo Calabrò, Angelo Baldassare Cefalù, Maurizio Averna, Laura D'Erasmo, Marcello Arca, Sara De Martin, Alberto Zambon, and Nicola Ferri

Subjects
Lomitapide, pcsk9, lp(a), Familial hypercholesterolemia, Diseases of the circulatory (Cardiovascular) system, RC666-701
Abstract

Lomitapide, a drug for the treatment of homozygous familial hypercholesterolemia patients, reduced total and LDL cholesterol but no significant changes were observed on PCSK9 and Lp(a) plasma levels. Some changes of inflammatory mediators were also observed, including hsCRP, which may suggest an anti-inflammatory effect.

Published
2021
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44. Familial hypercholesterolaemia in children and adolescents from 48 countries: a cross-sectional study

Author

Dharmayat, Kanika Inamdar, primary, Vallejo-Vaz, Antonio J., additional, Stevens, Christophe A.T., additional, Brandts, Julia M., additional, Lyons, Alexander R.M., additional, Groselj, Urh, additional, Abifadel, Marianne, additional, Aguilar-Salinas, Carlos A., additional, Alhabib, Khalid, additional, Alkhnifsawi, Mutaz, additional, Almahmeed, Wael, additional, Alnouri, Fahad, additional, Alonso, Rodrigo, additional, Al-Rasadi, Khalid, additional, Ashavaid, Tester F., additional, Banach, Maciej, additional, Béliard, Sophie, additional, Binder, Christoph, additional, Bourbon, Mafalda, additional, Chlebus, Krzysztof, additional, Corral, Pablo, additional, Cruz, Diogo, additional, Descamps, Olivier S., additional, Drogari, Euridiki, additional, Durst, Ronen, additional, Ezhov, Marat V., additional, Genest, Jacques, additional, Harada-Shiba, Mariko, additional, Holven, Kirsten B., additional, Humphries, Steve E., additional, Khovidhunkit, Weerapan, additional, Lalic, Katarina, additional, Laufs, Ulrich, additional, Liberopoulos, Evangelos, additional, Roeters van Lennep, Jeanine, additional, Lima-Martinez, Marcos Miguel, additional, Lin, Jie, additional, Maher, Vincent, additional, März, Winfried, additional, Miserez, André R., additional, Mitchenko, Olena, additional, Nawawi, Hapizah, additional, Panayiotou, Andrie G., additional, Paragh, György, additional, Postadzhiyan, Arman, additional, Reda, Ashraf, additional, Reiner, Željko, additional, Reyes, Ximena, additional, Sadiq, Fouzia, additional, Sahebkar, Amirhossein, additional, Schunkert, Heribert, additional, Shek, Aleksandr B., additional, Stroes, Eric, additional, Su, Ta-Chen, additional, Subramaniam, Tavintharan, additional, Susekov, Andrey, additional, Vázquez Cárdenas, Alejandra, additional, Huong Truong, Thanh, additional, Tselepis, Alexandros D., additional, Vohnout, Branislav, additional, Wang, Luya, additional, Yamashita, Shizuya, additional, Al-Sarraf, Ahmad, additional, Al-Sayed, Nasreen, additional, Davletov, Kairat, additional, Dwiputra, Bambang, additional, Gaita, Dan, additional, Kayikcioglu, Meral, additional, Latkovskis, Gustavs, additional, Marais, A. David, additional, Thushara Matthias, Anne, additional, Mirrakhimov, Erkin, additional, Nordestgaard, Børge G., additional, Petrulioniene, Zaneta, additional, Pojskic, Belma, additional, Sadoh, Wilson, additional, Tilney, Myra, additional, Tomlinson, Brian, additional, Tybjærg-Hansen, Anne, additional, Viigimaa, Margus, additional, Catapano, Alberico L., additional, Freiberger, Tomas, additional, Hovingh, G. Kees, additional, Mata, Pedro, additional, Soran, Handrean, additional, Raal, Frederick, additional, Watts, Gerald F., additional, Schreier, Laura, additional, Bañares, Virginia, additional, Greber-Platzer, Susanne, additional, Baumgartner-Kaut, Margot, additional, de Gier, Charlotte, additional, Dieplinger, Hans, additional, Höllerl, Florian, additional, Innerhofer, Reinhold, additional, Karall, Daniela, additional, Lischka, Julia, additional, Ludvik, Bernhard, additional, Mäser, Martin, additional, Scholl-Bürgi, Sabine, additional, Thajer, Alexandra, additional, Toplak, Hermann, additional, Demeure, Fabian, additional, Mertens, Ann, additional, Balligand, Jean-Luc, additional, Stephenne, Xavier, additional, Sokal, Etienne, additional, Petrov, Ivo, additional, Goudev, Assen, additional, Nikolov, Fedya, additional, Tisheva, Snejana, additional, Yotov, Yoto, additional, Tzvetkov, Ivajlo, additional, Hegele, Robert A, additional, Gaudet, Daniel, additional, Brunham, Liam, additional, Ruel, Isabelle, additional, McCrindle, Brian, additional, Cuevas, Ada, additional, Perica, Dražen, additional, Symeonides, Phivos, additional, Trogkanis, Efstratios, additional, Kostis, Andreas, additional, Ioannou, Andreas, additional, Mouzarou, Angeliki, additional, Georgiou, Anthoula, additional, Stylianou, Andreas, additional, Miltiadous, George, additional, Iacovides, Paris, additional, Deltas, Constantinos, additional, Vrablik, Michal, additional, Urbanova, Zuzana, additional, Jesina, Pavel, additional, Tichy, Lukas, additional, Hyanek, Josef, additional, Dvorakova, Jana, additional, Cepova, Jana, additional, Sykora, Josef, additional, Buresova, Kristyna, additional, Pipek, Michal, additional, Pistkova, Eva, additional, Bartkova, Ivana, additional, S|ulakova, Astrid, additional, Toukalkova, Lenka, additional, Spenerova, Michaela, additional, Maly, Jan, additional, Benn, Marianne, additional, Bendary, Ahmed, additional, Elbahry, Atef, additional, Ferrières, Jean, additional, Ferrieres, Dorota, additional, Peretti, Noel, additional, Bruckert, Eric, additional, Gallo, Antonio, additional, Valero, René, additional, Mourre, Florian, additional, Aouchiche, Karine, additional, Reynaud, Rachel, additional, Tounian, Patrick, additional, Lemale, Julie, additional, Boccara, Franck, additional, Moulin, Philippe, additional, Charrières, Sybil, additional, Di Filippo, Mathilde, additional, Cariou, Bertrand, additional, Paillard, François, additional, Dourmap, Caroline, additional, Pradignac, Alain, additional, Verges, Bruno, additional, Simoneau, Isabelle, additional, Farnier, Michel, additional, Cottin, Yves, additional, Yelnik, Cecile, additional, Hankard, Regis, additional, Schiele, François, additional, Durlach, Vincent, additional, Sultan, Ariane, additional, Carrié, Alain, additional, Rabès, Jean-Pierre, additional, Sanin, Veronika, additional, Schmieder, Roland, additional, Ates, Sara, additional, Rizos, Christos V., additional, Skoumas, Ioannis, additional, Tziomalos, Konstantinos, additional, Rallidis, Loukianos, additional, Kotsis, Vasileios, additional, Doumas, Michalis, additional, Skalidis, Emmanouil, additional, Kolovou, Genovefa, additional, Kolovou, Vana, additional, Garoufi, Anastasia, additional, Koutagiar, Iosif, additional, Polychronopoulos, Georgios, additional, Kiouri, Estela, additional, Antza, Christina, additional, Zacharis, Evangelos, additional, Attilakos, Achilleas, additional, Sfikas, George, additional, Koumaras, Charalambos, additional, Anagnostis, Panagiotis, additional, Anastasiou, Georgia, additional, Liamis, George, additional, Adamidis, Petros-Spyridon, additional, Milionis, Haralambos, additional, Lambadiari, Vaia, additional, Stabouli, Stella, additional, Filippatos, Theodosios, additional, Mollaki, Vicky, additional, Tsaroumi, Anastasia, additional, Lamari, Frida, additional, Proyias, Pavlos, additional, Harangi, Mariann, additional, Reddy, Lakshmi Lavanya, additional, Shah, Swarup A. V, additional, Ponde, Chandrashekhar K., additional, Dalal, Jamshed J., additional, Sawhney, Jitendra P.S., additional, Verma, Ishwar C., additional, Hosseini, Susan, additional, Jamialahmadi, Tannaz, additional, Alareedh, Mohammed, additional, Shaghee, Foaad, additional, Rhadi, Sabah Hasan, additional, Abduljalal, Maryam, additional, Alfil, Sarmad, additional, Kareem, Huda, additional, Cohen, Hofit, additional, Leitersdorf, Eran, additional, Schurr, Daniel, additional, Shpitzen, Shoshi, additional, Arca, Marcello, additional, Averna, Maurizio, additional, Bertolini, Stefano, additional, Calandra, Sebastiano, additional, Tarugi, Patrizia, additional, Casula, Manuela, additional, Galimberti, Federica, additional, Gazzotti, Marta, additional, Olmastroni, Elena, additional, Sarzani, Riccardo, additional, Ferri, Claudio, additional, Repetti, Elena, additional, Giorgino, Francesco, additional, Suppressa, Patrizia, additional, Bossi, Antonio Carlo, additional, Borghi, Claudio, additional, Muntoni, Sandro, additional, Cipollone, Francesco, additional, Scicali, Roberto, additional, Pujia, Arturo, additional, Passaro, Angelina, additional, Berteotti, Martina, additional, Pecchioli, Valerio, additional, Pisciotta, Livia, additional, Mandraffino, Giuseppe, additional, Pellegatta, Fabio, additional, Mombelli, Giuliana, additional, Branchi, Adriana, additional, Fiorenza, Anna Maria, additional, Pederiva, Cristina, additional, Werba, José Pablo, additional, Parati, Gianfranco, additional, Nascimbeni, Fabio, additional, Iughetti, Lorenzo, additional, Fortunato, Giuliana, additional, Cavallaro, Raimondo, additional, Iannuzzo, Gabriella, additional, Calabrò, Paolo, additional, Cefalù, Angelo Baldassare, additional, Capra, Maria Elena, additional, Zambon, Alberto, additional, Pirro, Matteo, additional, Sbrana, Francesco, additional, Trenti, Chiara, additional, Minicocci, Ilenia, additional, Federici, Massimo, additional, Del Ben, Maria, additional, Buonuomo, Paola Sabrina, additional, Moffa, Simona, additional, Pipolo, Antonio, additional, Citroni, Nadia, additional, Guardamagna, Ornella, additional, Lia, Salvatore, additional, Benso, Andrea, additional, Biolo, Gianni Biolo, additional, Maroni, Lorenzo, additional, Lupi, Alessandro, additional, Bonanni, Luca, additional, Rinaldi, Elisabetta, additional, Zenti, Maria Grazia, additional, Masuda, Daisaku, additional, Mahfouz, Linda, additional, Jambart, Selim, additional, Ayoub, Carine, additional, Ghaleb, Youmna, additional, Kasim, Noor Alicezah Mohd, additional, Nor, Noor Shafina Mohd, additional, Al-Khateeb, Alyaa, additional, Kadir, Siti Hamimah Sheikh Abdul, additional, Chua, Yung-An, additional, Razman, Aimi Zafira, additional, Nazli, Sukma Azureen, additional, Ranai, Norashikin Mohd, additional, Latif, Ahmad Zubaidi Abd, additional, Torres, María Teresa Magaña, additional, Mehta, Roopa, additional, Martagon, Alexandro J., additional, Ramirez, Gabriela A. Galan, additional, Antonio-Villa, Neftali Eduardo, additional, Vargas-Vazquez, Arsenio, additional, Elias-Lopez, Daniel, additional, Retana, Gustavo Gonzalez, additional, Encinas, Bethsabel Rodriguez, additional, Macias, Jose J. Ceballos, additional, Zazueta, Alejandro Romero, additional, Alvarado, Rocio Martinez, additional, Portano, Julieta D. Morales, additional, Lopez, Humberto Alvares, additional, Sauque-Reyna, Leobardo, additional, Gomez Herrera, Laura G., additional, Simental Mendia, Luis E., additional, Aguilar, Humberto Garcia, additional, Cooremans, Elizabeth Ramirez, additional, Aparicio, Berenice Pe~na, additional, Zubieta, Victoria Mendoza, additional, Gonzalez, Perla A. Carrillo, additional, Ferreira-Hermosillo, Aldo, additional, Portilla, Nacu Caracas, additional, Dominguez, Guadalupe Jimenez, additional, Garcia, Alinna Y. Ruiz, additional, Arriaga Cazares, Hector E., additional, Gonzalez Gonzalez, Jesus R., additional, Mendez Valencia, Carla V., additional, Padilla Padilla, Francisco G., additional, Prado, Ramon Madriz, additional, De los Rios Ibarra, Manuel O., additional, Arjona Villica~na, Ruy D., additional, Acevedo Rivera, Karina J., additional, Carrera, Ricardo Allende, additional, Alvarez, Jose A., additional, Amezcua Martinez, Jose C., additional, Barrera Bustillo, Manuel de los Reyes, additional, Vargas, Gonzalo Carazo, additional, Chacon, Roberto Contreras, additional, Figueroa Andrade, Mario H., additional, Ortega, Ashanty Flores, additional, Alcala, Hector Garcia, additional, Garcia de Leon, Laura E., additional, Guzman, Berenice Garcia, additional, Gardu~no Garcia, Jose J., additional, Garnica Cuellar, Juan C., additional, Gomez Cruz, Jose R., additional, Garcia, Anell Hernandez, additional, Holguin Almada, Jesus R., additional, Herrera, Ursulo Juarez, additional, Sobrevilla, Fabiola Lugo, additional, Rodriguez, Eduardo Marquez, additional, Sibaja, Cristina Martinez, additional, Medrano Rodriguez, Alma B., additional, Morales Oyervides, Jose C., additional, Perez Vazquez, Daniel I., additional, Reyes Rodriguez, Eduardo A., additional, Osorio, Ma. Ludivina Robles, additional, Saucedo, Juan Rosas, additional, Tamayo, Margarita Torres, additional, Valdez Talavera, Luis A., additional, Vera Arroyo, Luis E., additional, Zepeda Carrillo, Eloy A., additional, Galema-Boers, Annette, additional, Weigman, Albert, additional, Bogsrud, Martin P., additional, Malik, Munir, additional, Shah, Saeedullah, additional, Khan, Sabeen Abid, additional, Rana, Muhammad Asim, additional, Batool, Hijab, additional, Starostecka, Ewa, additional, Konopka, Agnieszka, additional, Lewek, Joanna, additional, Bielecka-Dąbrowa, Agata, additional, Gach, Agnieszka, additional, Jóźwiak, Jacek, additional, Pajkowski, Marcin, additional, Romanowska-Kocejko, Marzena, additional, Żarczyńska-Buchowiecka, Marta, additional, Hellman, Marcin, additional, Chmara, Magdalena, additional, Wasąg, Bartosz, additional, Parczewska, Aleksandra, additional, Gilis-Malinowska, Natasza, additional, Borowiec-Wolna, Justyna, additional, Stróżyk, Aneta, additional, Michalska-Grzonkowska, Aleksandra, additional, Chlebus, Izabela, additional, Kleinschmidt, Mariola, additional, Wojtecka, Agnieszka, additional, Zdrojewski, Tomasz, additional, Myśliwiec, Małgorzata, additional, Hennig, Matylda, additional, Medeiros, Ana Margarida, additional, Alves, Ana Catarina, additional, Almeida, Ana Filipa, additional, Lopes, Andreia, additional, Guerra, António, additional, Bilhoto, Carla, additional, Simões, Fernando, additional, Silva, Francisco, additional, Lobarinhas, Goreti, additional, Gama, Guida, additional, Palma, Isabel, additional, Salgado, José Miguel, additional, Matos, Luísa Diogo, additional, Moura, Márcio de, additional, Virtuoso, Maria João, additional, Tavares, Mónica, additional, Ferreira, Patrícia, additional, Pais, Patrícia, additional, Garcia, Paula, additional, Coelho, Raquel, additional, Ribeiro, Raquel, additional, Correia, Susana, additional, Sadykova, Dinara, additional, Slastnikova, Evgenia, additional, Alammari, Dalal, additional, Mawlawi, Horia Ahmed, additional, Alsahari, Atif, additional, Khudary, Alia Abdullah, additional, Alrowaily, Nawal Lafi, additional, Rajkovic, Natasa, additional, Popovic, Ljiljana, additional, Singh, Sandra, additional, Rasulic, Iva, additional, Petakov, Ana, additional, Lalic, Nebojsa M., additional, Peng, Fabian Kok, additional, Vasanwala, Rashida Farhan, additional, Venkatesh, Sreedharan Aravind, additional, Raslova, Katarina, additional, Fabryova, Lubomira, additional, Nociar, Jan, additional, Šaligova, Jana, additional, Potočňáková, Ludmila, additional, Kozárová, Miriam, additional, Varga, Tibor, additional, Kadurova, Michaela, additional, Debreova, Marianna, additional, Novodvorsky, Peter, additional, Gonova, Katarina, additional, Klabnik, Alexander, additional, Buganova, Ingrid, additional, Battelino, Tadej, additional, Bizjan, Barbara Jenko, additional, Debeljak, Marusa, additional, Kovac, Jernej, additional, Mlinaric, Matej, additional, Molk, Neza, additional, Sikonja, Jaka, additional, Sustar, Ursa, additional, Podkrajsek, Katarina Trebusak, additional, Muñiz-Grijalvo, Ovidio, additional, Díaz-Díaz, Jose Luis, additional, de Andrés, Raimundo, additional, Fuentes-Jiménez, Francisco, additional, Blom, Dirk, additional, Miserez, Eleonore B., additional, Shipton, Janine L., additional, Ganokroj, Poranee, additional, Futema, Marta, additional, Ramaswami, Uma, additional, Alieva, Rano B., additional, Fozilov, Khurshid G., additional, Khoshimov, Shavkat U., additional, Nizamov, Ulugbek I., additional, Abdullaeva, Guzal J., additional, Kan, Liliya E., additional, Abdullaev, Alisher A., additional, Zakirova, Daria V., additional, Do, Doan-Loi, additional, Nguyen, Mai-Ngoc-Thi, additional, Kim, Ngoc-Thanh, additional, Le, Thanh-Tung, additional, Le, Hong-An, additional, Santos, Raul, additional, and Ray, Kausik K., additional

Published
2023
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45. Refinement of the diagnostic approach for the identification of children and adolescents affected by familial hypercholesterolemia: Evidence from the LIPIGEN study

Author

Casula, Manuela, primary, Gazzotti, Marta, additional, Capra, Maria Elena, additional, Olmastroni, Elena, additional, Galimberti, Federica, additional, Catapano, Alberico L., additional, Pederiva, Cristina, additional, Anesi, Adriano, additional, Arca, Marcello, additional, Auricchio, Renata, additional, Averna, Maurizio, additional, Baldera, Davide, additional, Banderali, Giuseppe, additional, Beccuti, Guglielmo, additional, Benso, Andrea, additional, Berteotti, Martina, additional, Bertolini, Stefano, additional, Bianconi, Vanessa, additional, Biasucci, Giacomo, additional, Biolo, Gianni, additional, Bonanni, Luca, additional, Borghi, Claudio, additional, Bossi, Antonio Carlo, additional, Branchi, Adriana, additional, Bruzzi, Patrizia, additional, Bucci, Marco, additional, Buonuomo, Paola Sabrina, additional, Calabrò, Paolo, additional, Calandra, Sebastiano, additional, Carubbi, Francesca, additional, Cavallaro, Raimondo, additional, Cefalù, Angelo Baldassarre, additional, Cesaro, Arturo, additional, Cipollone, Francesco, additional, Citroni, Nadia, additional, Colombo, Emanuela, additional, Coppola, Chiara, additional, D'Addato, Sergio, additional, Dal Pino, Beatrice, additional, Dalla Nora, Edoardo, additional, De Corrado, Giuseppe, additional, Del Ben, Maria, additional, Di Molfetta, Sergio, additional, Di Taranto, Maria Donata, additional, Fainelli, Giulia, additional, Federici, Massimo, additional, Ferri, Claudio, additional, Fiorenza, Anna Maria, additional, Formisano, Elena, additional, Fortunato, Giuliana, additional, Giaccari, Andrea, additional, Giorgino, Francesco, additional, Grigore, Liliana, additional, Guardamagna, Ornella, additional, Iannuzzi, Arcangelo, additional, Iannuzzo, Gabriella, additional, Iughetti, Lorenzo, additional, Lia, Salvatore, additional, Longo, Susanna, additional, Lupi, Alessandro, additional, Mandraffino, Giuseppe, additional, Marcucci, Rossella, additional, Maroni, Lorenzo, additional, Massini, Giulia, additional, Mazza, Elisa, additional, Melchioda, Elena, additional, Meregalli, Giancarla, additional, Minicocci, Ilenia, additional, Moffa, Simona, additional, Mombelli, Giuliana, additional, Muntoni, Sandro, additional, Nascimbeni, Fabio, additional, Negri, Emanuele Alberto, additional, Notargiacomo, Serena, additional, Panfili, Filippo Maria, additional, Parati, Gianfranco, additional, Passaro, Angelina, additional, Pavanello, Chiara, additional, Pecchioli, Valerio, additional, Pecchioli, Lorenzo, additional, Pellegatta, Fabio, additional, Perla, Francesco Massimo, additional, Pipolo, Antonio, additional, Piro, Salvatore, additional, Pirro, Matteo, additional, Pisciotta, Livia, additional, Pujia, Roberta, additional, Putotto, Carolina, additional, Repetti, Elena, additional, Rinaldi, Elisabetta, additional, Romandini, Alessandra, additional, Sani, Elena, additional, Sarnari, Silvia, additional, Sarzani, Riccardo, additional, Sbrana, Francesco, additional, Scicali, Roberto, additional, Scuruchi, Michele, additional, Suppressa, Patrizia, additional, Tarugi, Patrizia, additional, Trenti, Chiara, additional, Vinci, Pierandrea, additional, Werba, José Pablo, additional, Zambon, Sabina, additional, Zambon, Alberto, additional, and Zenti, Maria Grazia, additional

Published
2023
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47. Long-term efficacy of lipoprotein apheresis and lomitapide in the treatment of homozygous familial hypercholesterolemia (HoFH): a cross-national retrospective survey

Author

D’Erasmo, Laura, Gallo, Antonio, Cefalù, Angelo Baldassare, Di Costanzo, Alessia, Saheb, Samir, Giammanco, Antonina, Averna, Maurizio, Buonaiuto, Alessio, Iannuzzo, Gabriella, Fortunato, Giuliana, Puja, Arturo, Montalcini, Tiziana, Pavanello, Chiara, Calabresi, Laura, Vigna, Giovanni Battista, Bucci, Marco, Bonomo, Katia, Nota, Fabio, Sampietro, Tiziana, Sbrana, Francesco, Suppressa, Patrizia, Sabbà, Carlo, Fimiani, Fabio, Cesaro, Arturo, Calabrò, Paolo, Palmisano, Silvia, D’Addato, Sergio, Pisciotta, Livia, Bertolini, Stefano, Bittar, Randa, Kalmykova, Olga, Béliard, Sophie, Carrié, Alain, Arca, Marcello, and Bruckert, Eric

Published
2021
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48. An Unexplained Congenital Disorder of Glycosylation-II in a Child with Neurohepatic Involvement, Hypercholesterolemia and Hypoceruloplasminemia

Author

Calvo, Pier Luigi, Spada, Marco, Rabbone, Ivana, Pinon, Michele, Porta, Francesco, Cisarò, Fabio, Reggiani, Stefania, Cefalù, Angelo B., Sturiale, Luisella, Garozzo, Domenico, Lefeber, Dirk J., Jaeken, Jaak, Baumgartner, Matthias, Series Editor, Patterson, Marc, Series Editor, Rahman, Shamima, Series Editor, Peters, Verena, Series Editor, Morava, Eva, Editor-in-Chief, and Zschocke, Johannes, Series Editor

Published
2018
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