Peer Reviewed Journals

Pattern of reported infections among paediatric patients with sickle cell disease: a single-centre cohort study in Nigeria

Idris, M.A.; Ruggieri, L.; Ahmad, H.R.; Hassan, A; Ibrahim, I.N.; Faruk, J.A.; Adebiyi, N.M.; Awwalu, S.; Usman, N.; Wada, R.; Muhammad, M.; Abdulkadir, S.; Bonifazi, F.; Atoyebi, W.; Inusa, B.P.D.
Hemato. 2026;7:11. doi:10.3390/hemato7020011.

Challenges in the maintenance of FAIR-compliant paediatric data-sharing repositories[

Pansieri C., Sen A.; D’Ercole A.; Cornet R., Hedley V.; Leary R.; Betourne A.; Bhattacharya S.; Shaunessy S.; Walls R.L.; Bonifazi F.; Felisi M.
Journal of the Society for Clinical Data Management. 2026;6(1). doi:10.47912/jscdm.427.

Ethics and regulatory guidance to address the challenges encountered in research on rare diseases in a multinational dimension: the experience of a European funded programme

Landi A., Mimouni Y., Demoisy A., Namorado J., Torretta S., D’Ercole A., Del Alamo M., Gibson S., Julkowska D., Giannuzzi V.
BMC Medical Ethics. 2026. doi:10.1186/s12910-025-01370-7

Understanding the Needs of Healthcare Professionals Involved in Sickle Cell Disease Management in Nigeria: A Grounded Theory Study

Abdulkareem A., Ruggieri L., Landi A., Didio A., Bonifazi F., Gayus Dogara L., Baba I.
Information Science Trends 2026; 3(11): 20-34. doi: 10.61882/ist.202603.11.02

Mapping Expertise and Educational Needs for Hemoglobinopathies: Insights from HELIOS COST Action Healthcare Professionals

Mezzalira, E.; D’Agnolo, M.; Antić, D.; Basile, F.; Bonifazi, F.; Collado Gimbert, A.; Cremonesi, F.; Durmaz, B.; Gavriilaki, E.; Glenthøj, A.; Inusa, B.; Landi, A.; Mañú Pereira, M. del M.; Orphanou, K.; Pellegrini, M.; Rühluel, D.; Stephanou, C.; Chatzimatthaiou, S.; Traeger-Synodinos, J.; Kountouris, P.; Colombatti, R.
Blood Global Hematology. 2025;100060. doi:10.1016/j.bglo.2025.100060

HELIOS Action: Advancing research, education, and equity in hemoglobinopathies across Europe and beyond

Chatzimatthaiou, S.; Bonifazi, F.; Gimbert, A. C.; Colombatti, R.; Cremonesi, F.; Glenthøj, A.; Mezzalira, E.; Stephanou, C.; Traeger-Synodinos, J.; Antic, D.; Durmaz, B.; Gavriilaki, E.; Inusa, B.; Landi, A.; Pellegrini, M.; Basile, F.; Muth, S.; Cario, H.; Katsantoni, E.; Ruhluel, D.; Lederer, C. W.; Mañú-Pereira, M. del M.; Kountouris, P.
HemaSphere. 2025;9(12):e70258. doi: 10.1002/hem3.70258

Pharmacokinetic and Pharmacodynamic Modeling of Clonidine and Midazolam for Sedation in Pediatric Intensive Care

Bardol, M.; Sheng, Y.; Baarslag, M.; Ceci, A.; Dörje, F.; Ilmoja, M.-L.; Larsson, P.; Lönnqvist, P.-A.; Methsvat, T.; Pokorna, P.; Rascher, W.; van Rosmalen, J.; Schroth, M.; Simonetti, A.; Tibboel, D.; Toni, I.; Tuleu, C.; Völkl, T. M. K.; Anderson, B. J.; Wimmer, S.; Standing, J. F.; Neubert, A.; CloSed Consortium.
Paediatric Anaesthesia. 2025;35(12):1053-1062. doi:10.1111/pan.70050

Characterizing local and systemic exposure to clobetasol propionate in healthy subjects and patients with atopic dermatitis

Duong, J. K.; van Dijkman, S.; Ong, G.; Marta, A.; Ceci, A.; Bonifazi, E.; Della Pasqua, O.
British Journal of Clinical Pharmacology. 2025;91(11):3150-3166. doi: 10.1002/bcp.70102

Scientific and regulatory progress in advancing paediatric oncology drug development in the EU and in the US

Reggiardo, G.; Natale, A.; Nicola Santoro, N.; Giannuzzi, V.; Pansieri, C.; Felisi, M.; Bonifazi, D.; Ceci, A.
Frontiers in Medicine. 2025;12:1642279. doi:10.3389/fmed.2025.1642279

Ethical and regulatory requirements for conducting researcher-driven large-scale multinational genetic haematological studies: the INHERENT experience

Didio, A.; Giannuzzi, V.; Archer, N.; Gani, E.; Sblano, S.; Peprah, E.; Kountouris, P.; Bonifazi, F.; The International Hemoglobinopathy Research Network (INHERENT)
Health Research Policy and Systems. 2025;23(1):101. doi:10.1186/s1296102501375z

The Potential to Leverage Real-World Data for Pediatric Clinical Trials: A Proof-of-Concept Study

Declerck, J.; Lee, J.; Sen, A.; Palmeri, A.; Oostenbrink, R.; Giannuzzi, V.; Woodworth, S.; Malik, S.; Mahler, F.; Aurich, B.; Leary, R.; Kalra, D.; Straub, V.
Journal of Medical Internet Research. 2025;27:e72573. doi:10.2196/72573

Highlighting healthcare disparities in sickle cell disease management in resource-constrained settings: A cross-sectional study in Lebanon

Bou Zerdan, M.; Watfa, A.; Kfoury, B.; Hoballah, J.; El-Hachem, R.; Ruggieri, L.; Bonifazi, F.; Abboud, M. R.; Yazbeck, S.
Pediatric Blood & Cancer. 2025;72(11):e31994. doi:10.1002/pbc.31994

The potential of MAO inhibitors as chemotherapeutics in cancer: A literature survey

Sblano, S.; Boccarelli, A.; Deruvo, C.; La Spada, G.; De Candia, M.; Purgatorio, R. et al.
European Journal of Medicinal Chemistry. 2025;117159. doi:10.1016/j.ejmech.2024.117159

Effects of the Paediatric Regulation funding on the development of off-patent medicines in children

Ruggieri, L.; Torretta, S.; Giannuzzi, V.; Natale, A.; Felisi, M.; Ceci, A.; Bonifazi, F.
Frontiers in Medicine. 2025;11:1473862. doi:10.3389/fmed.2024.1473862

Descriptive Analysis of Pediatric Studies Included in the European Union Post-Authorization Study Register from 2010 to 2023

Landi, A.; Reggiardo, G.; Didio, A.; D’Ercole, A.; Ceci, A.; Govere, G. S.; Bonifazi, D.; Bonifazi, F.; Crisafulli, S.; Trifirò, G.; Kaguelidou, F.; Hakkarainen, K. M.; Gvozdanović, K.; Barone-Adesi, F.; Ucciero, A.; Felisi, M.
Pediatric Reports. 2025;17(1):24. doi:10.3390/pediatric17010024

Paediatric-specific content in data standards for health

Turner, M. A.; Kalra, D.; Cornet, R.; Palmeri, A.; Sen, A.; Owen, J.; Pansieri, C.; Lee, J.; Hedley, V.; Nally, S.; Bonifazi, F.; Leary, R.; Straub, V.
Archives of Disease in Childhood. 2025;110(3):177–179. doi:10.1136/archdischild-2024-327931

Paediatric formulations—part of the repurposing concept?

Wang, S.; Giannuzzi, V.
Frontiers in Medicine. 2024;11:1456247. doi:10.3389/fmed.2024.1456247

Innovative research methodologies in the EU regulatory framework: an analysis of EMA qualification procedures from a paediatric perspective

Giannuzzi, V.; Bertolani, A.; Torretta, S.; Reggiardo, G.; Toich, E.; Bonifazi, D.; Ceci, A.
Frontiers in Medicine. 2024;11:1369547. doi:10.3389/fmed.2024.1369547

TISBE: A Public Web Platform for the Consensus-Based Explainable Prediction of Developmental Toxicity

Mastrolorito, F.; Togo, M. V.; Gambacorta, N.; Trisciuzzi, D.; Giannuzzi, V.; Bonifazi, F.; Liantonio, A.; Imbrici, P.; De Luca, A.; Altomare, C. D.; Ciriaco, F.; Amoroso, N.; Nicolotti, O.
Chemical Research in Toxicology. 2024;37(2):323–339. doi:10.1021/acs.chemrestox.3c00310

Design and Implementation of a Sickle Cell Disease Electronic Registry in Resource Limited Setting in Nigeria - A Pilot Study

Idris, M. A.; Ruggieri, L.; Rufai, H. A.; Hassan, A.; Ibrahim, I. N.; Adullahi, F. J.; Awwalu, S.; Nasiru, U.; Bonifazi, F.; Inusa, B.
Hemato. 2024;5(3):340–349. doi:10.3390/hemato5030025

Harmonizing Quality Improvement Metrics Across Global Trial Networks to Advance Paediatric Clinical Trials Delivery

Attar, S.; Price, A.; Hovinga, C.; Stewart, B.; Lacaze-Masmonteil, T.; Bonifazi, F. et al.
Therapeutic Innovation & Regulatory Science. 2024;58(5):953–964. doi:10.1007/s43441-024-00663-0

Mapping of Data-Sharing Repositories for Paediatric Clinical Research—A Rapid Review

Felisi, M.; Bonifazi, F.; Toma, M.; Pansieri, C.; Leary, R.; Hedley, V.; Cornet, R.; Reggiardo, G.; Landi, A.; D’Ercole, A.; Malik, S.; Nally, S.; Sen, A.; Palmeri, A.; Bonifazi, D.; Ceci, A.
Data. 2024;9(4):59. doi:10.3390/data904

Learning from conect4children: A Collaborative Approach towards Standardisation of Disease-Specific Paediatric Research Data

Sen, A.; Hedley, V.; Degraeuwe, E.; Hirschfeld, S.; Cornet, R.; Walls, R.; Owen, J.; Robinson, P. N.; Neilan, E. G.; Liener, T.; Nisato, G.; Modi, N.; Woodworth, S.; Palmeri, A.; Gaentzsch, R.; Walsh, M.; Berkery, T.; Lee, J.; Persijn, L.; Baker, K.; An Haack, K.; Segovia Simon, S.; Jacobsen, J. O. B.; Reggiardo, G.; Kirwin, M. A.; Trueman, J.; Pansieri, C.; Bonifazi, D.; Nally, S.; Bonifazi, F.; Leary, R.; Straub, V.
Data. 2024;9(4):55. doi:10.3390/data9040055

Sharing Data and Transferring Samples Within Pediatric Clinical Studies: How to Overcome Challenges and Make Them a Science Opportunity

Landi, A.; D’Ambrosio, F.; Faggion, S.; Rocchi, F.; Paganin, C.; Lain, M. G.; Ceci, A.; Giannuzzi, V. on behalf of the EPIICAL Consortium
Healthcare. 2024;12(23):2473. doi:10.3390/healthcare12232473

Getting your DUCs in a row - standardising the representation of Digital Use Conditions

Jeanson, F.; Gibson, S. J.; Alper, P.; Bernier, A.; Woolley, J. P.; Mietchen, D.; Strug, A.; Becker, R.; Kamerling, P.; Sanchez Gonzalez, M.; Mah, N.; Novakowski, A.; Wilkinson, M. D.; Benhamed, O. M.; Landi, A.; Krog, G. P.; Müller, H.; Riaz, U.; Veal, C.; Holub, P.; van Enckevort, E.; Brookes, A. J.
Scientific Data. 2024;11:464. doi:10.1038/s41597-024-03280-6

Common conditions of use elements. Atomic concepts for consistent and effective information governance

Sanchez Gonzalez, M.; Kamerling, P.; Iermito, M.; Casati, S.; Riaz, U.; Veal, C. D.; Maini, M.; Jeanson, F.; Benhamed, O. M.; van Enckevort, E.; Landi, A.; Mimouni, Y.; Le Cornec, C.; Coviello, D. A.; Franchin, T.; Fusco, F.; Ramírez García, J. A.; van der Zanden, L. F. M.; Bernier, A.; Wilkinson, M. D.; Mueller, H.; Gibson, S. J.; Brookes, A. J.
Scientific Data. 2024;11:465. doi:10.1038/s41597-024-03279-z

The creation of an adaptable informed consent form for research purposes to overcome national and institutional bottlenecks in ethics review: experience from rare disease registries

Landi, A.; Mimouni, Y.; Giannuzzi, V.; Schaefer, F.; Altavilla, A.; Gibson, S.; Daria Julkowska, D.
Frontiers in Medicine. 2024;11:1384026. doi:10.3389/fmed.2024.1384026

High mortality following early initiation of antiretroviral therapy in infants living with HIV from three African countries

Tagarro, A.; Domínguez-Rodríguez, S.; Cotton, M.; Otwombe, K.; Klein, N.; Lain, M. G.; Nhampossa, T.; Maiga, A. I.; Barnabas, S.; Vaz, P.; Violari, A.; Fernández-Luis, S.; Behuhuma, O.; Sylla, M.; López-Varela, E.; Naniche, D.; Janse-Van-Rensburg, A.; Liberty, A.; Ramsagar, N.; Smit, T.; Makhari, S.; Ismael, N.; Giaquinto, C.; Rossi, P.; Kuhn, L.; Palma, P.; Spyer, M.; Lichterfeld, M.; Nastuoli, E.; Giannuzzi, V. et al.
eClinicalMedicine. 2024;73:102648. doi:10.1016/j.eclinm.2024.102648

Special Issue: “Personalized Medicine in Blood Disease of Children”

Ceci, A.; Kountouris, P.; Didio, A.; Bonifazi, F.
Journal of Personalized Medicine. 2024;14(3):285. doi:10.3390/jpm14030285

Ethical and regulatory requirements for conducting researcher-driven large-scale multi-national genetic hematological studies: the INHERENT experience

Didio, A.; Giannuzzi, V.; Archer, N.; Gani, E.; Peprah, E.; Kountouris, P.; Bonifazi, F.
HemaSphere. 2024;8:e70009; p. 133. doi:10.1002/hem3.70009

How to live well with SCD for patients, parents, caregivers, families and public

Mande, A.; Ruggieri, L.; Abdulakadir, I.; Bonifazi, F.; Conte, R.; Didio, A.; Faleye, M.; Hassan, A.; Muhammad, A.I.; Aliyu, R.M.; Rufai, H.A.
HemaSphere. 2024;8:e70009; p. 143. doi:10.1002/hem3.70009

Pattern of infections reported among paediatric sickle cell patients attending a tertiary hospital in Nigeria

Idris, M.A.; Ruggieri, L.; Rufai, H.A.; Hassan, A.; Ibrahim, I.N.; Abdullahi, F.J.; Awwalu, S.A.; Nasiru, U.; Muhammad, M.; Abdulkadir, S.; Bonifazi, F.; Atoyebi, W.
HemaSphere. 2024;8:e70009; p. 30. doi:10.1002/hem3.70009

Haemoglobinopathies in European Liaison of Medicine and Science (HELIOS) CA22119 COST Action

Chatzimatthaiou, S.C.; Bonifazi, F.; Colombatti, R.F.; Cremonesi, F.; Glenthøj, A.; Synodinos, J.T.; Gimbert, A.; Stephanou, C.; Antic, D.; Durmaz, B.; Gavriilaki, E.; Inusa, B.; Landi, A.; Pellegrini, M.; Pereira, M.; Kountouris, P.
HemaSphere. 2024;8:e70009; p. 38. doi:10.1002/hem3.70009

Analysis of the pilot study of the International Hemoglobinopathy Research Network (INHERENT)

Archer, N.M.; Stephanou, C.; Xenophontos, M.; Tshilolo, L.; Nzengu, F.; Fazili, S.; Nnodu, O.E.; Nwegbu, M.M.; Waziri, A.D.; Awwalu, S.; Christou, S.; Savvidou, I.; Rekleiti, A.; Minaidou, A.; Diamantidis, M.D.; Mohd Yasin, N.Y.; Abdul Aziz, N.A.; Selvaratnam, V.S.; Esa, E.E.; Glenthøj, A.; Delicou, S.; Dogara, L.G.; Dimopoulou, M.N.; Raja Sabudin, R.Z.A.; Jalil, N.; Loh, C.K.; Lau, S.C.D.; Zilfalil, B.A.; Norsarwany, M.; Syahiran, S.; Ahmad Fikri, Z.A.; Hashim, H.F.; Oni, M.; Lukangu, A.; Alves, L.; Brito, M.; Giannuzzi, V.; Bonifazi, F.; Michailidou, K.; Chatzimatthaiou, S.; Lederer, C.W.; Kountouris, P.
HemaSphere. 2024;8:e70009; pp. 107-108. doi:10.1002/hem3.70009

Editorial: Reviews in: regulatory science

Kerpel-Fronius, S.; Giannuzzi, V.; Stoyanova-Beninska, V.; Sun, Z.
Frontiers in Medicine. 2023;10:1206753. doi:10.3389/fmed.2023.1206753

PedCRIN tool for the biosamples management in paediatric clinical trials

Giannuzzi, V.; Ruggieri, L.; Conte, R.; Manfredi, C.; Felisi, M.; Kubiak, C.; Matei, M.; Malik, S.; Demotes, J.; Ceci, A.; Bonifazi, D.
Clinical and Translational Science. 2023;16(5):797–809. doi:10.1111/cts.13489

Integrations of pharmacokinetic and pharmacodynamic models to multi-criteria decision analysis (MCDA) for prospective assessment of the risk-benefit ratio of oral iron chelators in paediatric patients with transfusion-dependent haemoglobinopathies

Della Pasqua, O.; Ceci, A.; Ruggieri, L.; Oosterholt, S.; Di Deo, A.; Bonifazi, F.
HemaSphere. 2023;7(Suppl):18-19. doi:10.1097/01.HS9.0000928260.45846.86

Collaborative approach to enhance the knowledge on paediatric rare diseases: the European Paediatric Translational Research Infrastructure. Meeting abstracts from the 11th edition of the European conference on Rare Diseases & Orphan Products (ECRD) 2022

Ruggieri, L.; Ceci, A.; Georgieva, B.; Bertolani, A.; Bartoloni, F.; Bonifazi, F.; Migliaccio, G.; Altavilla, A.; Della Pasqua, O.; Mikros, E.; Kubova, H.; Martelli, P.; Allegaert, K.; Dimitri, P.; Staud, F.; Smits, E.; Kleanthous, M.; Giannattasio, S.; Denora, N.; Tuleu, C.; Bonifazi, D.
Orphanet Journal of Rare Diseases. 2023; 18(Suppl 1):118; p.13. doi:10.1186/s13023-023-02707-4

Off-label is not always off-evidence: authorising paediatric indications for old medicines

de Wildt, S. N.; Foeldvari, I.; Siapkara, A.; Lepola, P.; Kriström, B.; Ruggieri, L.; Eichler, I.; Egger, G. F.
The Lancet Child & Adolescent Health. 2023;7(6):371–372. doi:10.1016/S2352-4642(23)00083-4

Dose rationale for gabapentin and tramadol in pediatric patients with chronic pain

Healy, P.; Verrest, L.; Felisi, M.; Ceci, A.; Della Pasqua, O.
Pharmacology Research & Perspectives. 2023;11(5):e01138. doi:10.1002/prp2.1138

Target therapy for high-risk neuroblastoma treatment:integration of regulatory and scientific tools is needed

Ceci, A.; Conte, R.; Didio, A.; Landi, A.; Ruggieri, L.; Giannuzzi, V.; Bonifazi, F.
Frontiers in Medicine. 2023;10:1113460. doi:10.3389/fmed.2023.1113460

Moving towards a question-centric approach for regulatory decision-making in the context of drug assessment

Musuamba Tshinanu, F.; Cheung, S. Y. A.; Colin, P.; Davies, E. H.; Barret, J. S.; Pappalardo, F.; Chappell, M.; Dogne, J. M.; Ceci, A.; Della Pasqua, O.; Rusten, I. S.
Clinical Pharmacology and Therapeutics. 2023;114(1):41–50. doi:10.1002/cpt.2856

Editorial: The use of real world data for regulatory purposes in the rare diseases setting

Giannuzzi, V.; Stoyanova-Beninska, V.; Hivert, V.
Frontiers in Pharmacology. 2023;14:1089033. doi:10.3389/fphar.2022.1089033

Overview of the EU PAS register post-authorization studies performed in Europe from September 2010 to December 2018

Sultana, J.; Crisafulli, S.; Almas, M.; Antonazzo, I.C.; Baan, E.; Bartolini, C.; Bertuccio, M.P., Bonifazi, F.; Capuano, A.; Didio, A.; Ehrenstein, V.; Felisi, M.; Ferrajolo, C.; Fontana, A.; Francisca, R.; Fourrier-Reglat, A.; Fortuny, J; Gini, R.; Hyeraci, G.; Hoeve, C.; Kontogiorgis, C.; Isgrò, V.; Lalagka, P.N.; L'Abbate, L.; Layton, D.; Landi, A.; Narduzzi, S.; Roque Pereira, L.; Poulentzas, G.; Rafaniello, C.; Roberto, G.; Scondotto, G.; Sportiello, L.; Toma, M.; Toussi, M.; Verhamme, K.; Volpe, E.; Trifirò, G.; On behalf the Working Group 3 of Uncap (Inventory of EU data sources and methodological approaches for multisource studies).
Pharmacoepidemiology and Drug Safety. 2022;31(6):689–705. doi:10.1002/pds.5413.

Random Forest Clustering Identifies Three Subgroups of β-Thalassemia with Distinct Clinical Severity

Vitrano, A.; Musallam, K. M.; Meloni, A.; Addario Pollina, S.; Karimi, M.; El-Beshlawy, A.; Hajipour, M.; Di Marco, V.; Ansari, S. H.; Filosa, A.; Ricchi, P.; Ceci, A.; Daar, S.; Vlachaki, E.; Singer, S. T.; Naserullah, Z. A.; Pepe, A.; Scondotto, S.; Dardanoni, G.; Bonifazi, F.; Sankaran, V. G.; Vichinsky, E.; Taher, A. T.; Maggio, A.; International Working Group on Thalassemia.
Thalassemia Reports. 2022;12(1):14–23. doi:10.3390/thalassrep12010004

Primary HBB gene mutation severity and long-term outcomes in a global cohort of β-thalassaemia

Musallam, K. M.; Vitrano, A.; Meloni, A.; Addario Pollina, S.; Di Marco, V.; Hussain Ansari, S.; Filosa, A.; Ricchi, P.; Ceci, A.; Daar, S.; Vlachaki, E.; Singer, S. T.; Naserullah, Z. A.; Pepe, A.; Scondotto, S.; Dardanoni, G.; Karimi, M.; El-Beshlawy, A.; Hajipour, M.; Bonifazi, F.; Vichinsky, E.; Taher, A. T.; Sankaran, V. G.; Maggio, A.; International Working Group on Thalassemia (IWG-THAL)
British Journal of Haematology. 2022;196(2):414–423. doi:10.1111/bjh.17897

Informed consent and assent guide for paediatric clinical trials in Europe

Lepola, P.; Kindred, M.; Giannuzzi, V.; Glosli, H.; Dehlinger-Kremer, M.; Dalrymple, H.; Neubauer, D.; Boylan, G. B.; Conway, J.; Dewhurst, J.; Hoffman, D.
Archives of Disease in Childhood. 2022;107(6):582–590. doi:10.1136/archdischild2021322798

Health technology assessment of paediatric medicines: European landscape, challenges and opportunities inside the conect4children project

Moretti, F.; Ruiz, F.; Bonifazi, F.; Pizzo, E.; Kindblom, J. M.; the c4c HTA expert group
British Journal of Clinical Pharmacology. 2022;88(12):5052–5059. doi:10.1111/bcp.15190

Ethical and procedural issues for applying researcher-driven multi-national paediatric clinical trials in and outside the European Union: the challenging experience of the DEEP project

Giannuzzi, V.; Felisi, M.; Bonifazi, D.; Devlieger, H.; Papanikolaou, G.; Ragab, L.; Fattoum, S.; Tempesta, B.; Reggiardo, G.; Ceci, A.
BMC Medical Ethics. 2021;22(1):49. doi:10.1186/s12910021006182

Paediatric Medicines in Europe: The Paediatric Regulation—Is It Time for Reform?

Toma, M.; Felisi, M. G.; Bonifazi, D.; Bonifazi, F.; Giannuzzi, V.; Reggiardo, G.; de Wildt, S.; Ceci, A.; TEDDY European Network of Excellence for Paediatric Research.
Frontiers in Medicine. 2021;8:593281. doi:10.3389/fmed.2021.593281

Paediatric clinical research in Europe: an insight on experts' needs and perspectives

Ruggieri, L.; Ceci, A.; Bartoloni, F.; Elie, V.; Felisi, M.; Jacqz-Aigrain, E.; Lupo, M.; Malik, S.; Manfredi, C.; Reggiardo, G.; Demotes, J.; Bonifazi, D.
Contemporary Clinical Trials Communications. 2021;21:100735. doi:10.1016/j.conctc.2021.100735

Medical Device Development for Children and Young People—Reviewing the Challenges and Opportunities

Dimitri, P.; Pignataro, V.; Lupo, M.; Bonifazi, D.; Henke, M.; Musazzi, U. M.; Ernst, F.; Minghetti, P.; Redaelli, D. F.; Antimisiaris, S. G.; Migliaccio, G.; Bonifazi, F.; Marciani, L.; Courtenay, A. J.; Denora, N.; Lopedota, A.
Pharmaceutics. 2021;13(12):2178. doi:10.3390/pharmaceutics13122178

The conect4children (c4c) Consortium: Potential for Improving European Clinical Research into Medicines for Children

Turner, M. A.; Hildebrand, H.; Fernandes, R. M.; de Wildt, S. N.; Mahler, F.; Hankard, R.; Leary, R.; Bonifazi, F.; Nobels, P.; Cheng, K.; Attar, S.; Rossi, P.; Rocchi, F.; Clavero, J.; Nafria, B.; Giaquinto, C.
Pharmaceutical Medicine. 2021;35(2):71–79. doi:10.1007/s40290020003736

Human Microbiome in Children, at the Crossroad of Social Determinants of Health and Personalized Medicine

Sainz, T.; Pignataro, V.; Bonifazi, D.; Ravera, S.; Mellado, M. J.; Pérez-Martínez, A.; Escudero, A.; Ceci, A.; Calvo, C.
Children. 2021;8(12):1191. doi:10.3390/children8121191

Survival and causes of death in 2,033 patients with non-transfusion-dependent β-thalassemia

Musallam, K. M.; Vitrano, A.; Meloni, A.; Pollina, S. A.; Karimi, M.; El-Beshlawy, A.; Hajipour, M.; Di Marco, V.; Ansari, S. H.; Filosa, A.; Ricchi, P.; Ceci, A.; Daar, S.; Vlachaki, E.; Singer, S. T.; Naserullah, Z. A.; Pepe, A.; Scondotto, S.; Dardanoni, G.; Bonifazi, F.; Sankaran, V. G.; Vichinsky, E.; Taher, A. T.; Maggio, A.
Haematologica. 2021;106(9):2489–2492. doi:10.3324/haematol.2021.278684

Ethical, Legal and Regulatory Issues of Paediatric Translational Research. Call for an Adequate Model of Governance

Altavilla, A.; Giannuzzi, A.; Lupo, M.; Bonifazi, D.; Ceci, A.
European Journal of Health Law. 2020;27(3):213–231. doi:10.1163/15718093-BJA10010

Machine Learning Systems Applied to Health Data and System

Bonifazi, F.; Volpe, E.; Digregorio, G.; Giannuzzi, V.; Ceci, A.
European Journal of Health Law. 2020;27(3):242–258. doi:10.1163/15718093-BJA10009

Different strategies to execute multi-database studies for medicines surveillance in real world setting: a reflection on the European model

Gini, R.; Sturkenboom, M.; Sultana, J.; Cave, A.; Landi, A.; Pacurariu, A.; Roberto, G.; Schink, T.; Candore, G.; Slattery, J.; Trifirò, G.; The Working Group 3 of ENCePP.
Clinical Pharmacology & Therapeutics. 2020;108(2):228–235. doi:10.1002/cpt.1833.

A complication risk score to evaluate clinical severity of thalassaemia syndromes

Vitrano, A.; Meloni, A.; Addario Pollina, W.; Karimi, M.; El-Beshlawy, A.; Hajipour, M.; Di Marco, V.; Hussain Ansari, S.; Filosa, A.; Ricchi, P.; Ceci, A.; Daar, S.; Titi Singer, S.; Naserullah, Z. A.; Pepe, A.; Scondotto, S.; Dardanoni, G.; Bonifazi, F.; Vichinsky, E.; Maggio, A.
British Journal of Haematology. 2021;192(3):626–633. doi:10.1111/bjh.17203

European research networks to facilitate drug research in children

Turner, M.; Cheng, K.; de Wildt, S.; Hildebrand, H.; Attar, S.; Rossi, P.; Bonifazi, D.; Ceci, A.; Claverol, J.; Nafria, B.; Giaquinto, C.
British Journal of Clinical Pharmacology. 2020;88(10):4258–4266. doi:10.1111/bcp.14545.

Evaluation of the efficacy and safety of deferiprone compared with deferasirox in paediatric patients with transfusion-dependent haemoglobinopathies (DEEP-2): a multicentre, randomised, open-label, non-inferiority, phase 3 trial

Maggio, A.; Kattamis, A.; Felisi, M.; Reggiardo, G.; El-Beshlawy, A.; Bejaoui, M.; Sherief, L.; Christou, S.; Cosmi, C.; Della Pasqua, O.; Del Vecchio, G.; Filosa, A.; Cuccia, L.; Hassab, H.; Kreka, M.; Origa, R.; Putti, M.; Spino, M.; Telfer, P.; Tempesta, B.; Vitrano, A.; Tsang, Y. C.; Zaka, A.; Tricta, F.; Bonifazi, D.; Ceci, A.
The Lancet Haematology. 2020;7(6):e469–e478. doi:10.1016/S2352-3026(20)30100-9.

Survey by TEDDY European Network of Excellence for Paediatric Clinical Research demonstrates potential for Europe‐wide trials

Ruggieri, L.; Bonifazi, D.; Landi, A.; Bonifazi, F.; Bartoloni, F.; Costello, M.; Felisi, M. G.; Gasthuys, E.; Godo, A.; Torres, F. M.; Nadal, D.; Nuytinck, L.; Rocchi, F.; Turner, M.; Ceci, A.
Acta Paediatrica. 2020;109(3):607–612. doi:10.1111/apa.15020.

The “A” of FAIR – As Open as Possible, as Closed as Necessary

Landi, A.; Thompson, M.; Giannuzzi, V.; Bonifazi, F.; Labastida, I.; Bonino da Silva Santos, L. O.; Roos, M.
Data Intelligence. 2020;2(1-2):47–55. doi:10.1162/dint_a_00027.

Development of a Severity Score System for Thalassemia Syndromes

Vitrano, A.; Meloni, A.; Addario Pollina, W.; Karimi, M.; El-Beshlawy, A.; Hajipour, M.; Di Marco, V.; Hussain Ansari, S.; Filosa, A.; Ricchi, P.; Ceci, A.; Daar, S.; Titi Singer, S.; Borgio, J. F.; Pepe, A.; Scondotto, S.; Dardanoni, G.; Di Maggio, R.; Sacco, M.; Vichinsky, E. P.; Maggio, A.
Blood. 2019;134(Supplement_1):2225. doi:10.1182/blood-2019-12467

An overview of the efficacy and safety of deferiprone in paediatric patients with congenital haemoglobinopathies and chronic iron overload

Ceci, A.; Conte, R.; Didio, A.; Bonifazi, D.; Felisi, M.; Giannuzzi, V.; Bonifazi, F.
Expert Opinion on Orphan Drugs. 2019;7(4):181–197. doi:10.1080/21678707.2019.1613977.

The Secondary Use of Paediatric Data Under GDPR: Looking for New Safeguards for Research

Altavilla, A.; Herveg, J.; Giannuzzi, V.; Landi, A.; Ceci, A.
European Pharmaceutical Law Review. 2019;3(4):156–164. doi:10.21552/eplr/2019/4/6.

Abandoned OMPs. 9th European Conference on Rare Diseases & Orphan Products (ECRD Vienna 2018)

Giannuzzi, V.; Landi, A.; Ceci, A.
Orphanet Journal of Rare Diseases. 2018;13(Suppl 2):26 (Speaker presentation S5). doi:10.1186/s13023-018-0895-2.

Safe, Innovative and Accessible Medicines in Europe: A Renewed Strategy for Patients and the Pharmaceutical Sector

Altavilla A.
European Journal of Health Law. 2018;25(2):133–156. doi: 10.1163/15718093-12520381

Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs

Giannuzzi, V.; Landi, A.; Bosone, E.; Giannuzzi, F.; Nicotri, S.; Torrent-Farnell, J.; Bonifazi, F.; Felisi, M.; Bonifazi, D.; Ceci, A.
BMJ Open. 2017;7(9):e017358. doi:10.1136/bmjopen-2017-017358

The CLOSED trial; CLOnidine compared with midazolam for SEDation of paediatric patients in the intensive care unit: study protocol for a multicentre randomised controlled trial

Neubert, A.; Baarslag, M. A.; van Dijk, M.; van Rosmalen, J.; Standing, J. F.; Sheng, Y.; Rascher, W.; Roberts, D.; Winslade, J.; Rawcliffe, L.; Hanning, S. M.; Metsvaht, T.; Giannuzzi, V.; Larsson, P.; Pokorná, P.; Simonetti, A.; Tibboel, D. on behalf of CLOSED Consortium.
BMJ Open. 2017;7(6):e016031. doi:10.1136/bmjopen-2017-016031

Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen

Giannuzzi, V.; Conte, R.; Landi, A.; Ottomano, S. A.; Bonifazi, D.; Baiardi, P.; Bonifazi, F.; Ceci, A.
Orphanet Journal of Rare Diseases. 2017;12(1):64. doi:10.1186/s13023-017-0617-1

The ethical framework for performing research with rare inherited neurometabolic disease patients

Giannuzzi, V.; Devlieger, H.; Margari, L.; Odlind, V.; Ragab, L.; Bellettato, C.M.; D’Avanzo, F.; Lampe, C.; Cassis, L.; Cortès-Saladelafont, E.; Garcia Cazorla, Á.; Barić, I.; Cvitanović-Šojat, L.; Fumić, K.; Dali, C.I.; Bartoloni, F.; Bonifazi, F.; Scarpa, M.; Ceci, A.
European Journal of Pediatrics. 2017;176(3):395–405. doi:10.1007/s00431-017-2852-9

Pattern of complications and burden of disease in patients affected by beta thalassemia major

Bonifazi, F.; Conte, R.; Baiardi, P.; Bonifazi, D.; Felisi, M.; Giordano, P.; Giannuzzi, V.; Iacono, A.; Padula, R.; Pepe, A.; Putti, M. C.; Ruggieri, L.; Del Vecchio, G. C.; Filosa, A.; Maggio, A.; Ceci, A. on behalf of the HTA-THAL Multiregional Registry.
Current Medical Research and Opinion. 2017;33(8):1525–1533. doi:10.1080/03007995.2017.1326890.

Investigating the roles and training of paediatric research nurses working across Europe: a questionnaire-based survey

Veal, G. J.; Malik, S.; Lupo, M.; MacFarlane, S.; Lepola, P.; Costello, M.; Ceci, A.; Boué, C.; Lecour, C.; Otto, A.; Rastegari, M.; Berry, P.
BMJ Paediatrics Open. 2017;1(1):e000170. doi:10.1136/bmjpo-2017-000170.

The Italian multiregional thalassemia registry: Centers characteristics, services, and patients' population

Conte, R.; Ruggieri, L.; Gambino, A.; Bartoloni, F.; Baiardi, P.; Bonifazi, D.; Bonifazi, F.; Felisi, M.; Giannuzzi, V.; Padula, R.; Pepe, A.; Putti, M. C.; Del Vecchio, G. C.; Maggio, A.; Filosa, A.; Iacono, A.; Mangiarini, L.; Ceci, A.
Hematology. 2016;21(7):415–424. doi:10.1080/10245332.2015.1101971.

Clinical Trial Application in Europe: What Will Change with the New Regulation?

Giannuzzi, V.; Altavilla, A.; Ruggieri, L.; Ceci, A.
Science and Engineering Ethics. 2016;22(2):451–466. doi:10.1007/s11948-015-9662-0.

Safety Profile of Oral Iron Chelator Deferiprone in Chinese Children with Transfusion-Dependent Thalassaemia

Botzenhardt, S.; Sing, C. W.; Wong, I. C.; Chan, G. C.; Wong, L. Y.; Felisi, M.; Rascher, W.; Ceci, A.; Neubert, A.
Current Drug Safety. 2016;11(2):137–144. doi:10.2174/1574886310666150930113957

Review and evaluation of the methodological quality of the existing guidelines and recommendations for inherited neurometabolic disorders

Cassis, L.; Cortès-Saladelafont, E.; Molero-Luis, M.; Yubero, D.; González, M. J.; Ormazábal, A.; Fons, C.; Jou, C.; Sierra, C.; Ponce, E. C.; Ramos, F.; Armstrong, J.; O'Callaghan, M. M.; Casado, M.; Montero, R.; Meavilla-Olivas, S.; Artuch, R.; Barić, I.; Bartoloni, F.; Bellettato, C. M.; Bonifazi, F.; Ceci, A.; Cvitanović-Šojat, L.; Dali, C. I.; D'Avanzo, F.; Fumic, K.; Giannuzzi, V.; Lampe, C.; Scarpa, M.; Garcia-Cazorla, Á.
Orphanet Journal of Rare Diseases. 2015;10:164. doi:10.1186/s13023-015-0376-9.

Successful private-public funding of paediatric medicines research: lessons from the EU programme to fund research into off-patent medicines

Ruggieri, L.; Giannuzzi, V.; Baiardi, P.; Bonifazi, F.; Davies, E. H.; Giaquinto, C.; Bonifazi, D.; Felisi, M.; Chiron, C.; Pressler, R.; Rabe, H.; Whitaker, M. J.; Neubert, A.; Jacqz-Aigrain, E.; Eichler, I.; Turner, M. A.; Ceci, A.; on behalf of the GRiP Consortium
European Journal of Pediatrics. 2015 Apr;174(4):481–491. doi:10.1007/s00431-014-2398-z.

Multidisciplinary care in haemoglobinopathies

Ceci, A.; Mangiarini, L.; Bonifazi, F.; Conte, R. and on behalf of the HTA-THAL Multiregional Registry
Thalassemia Reports. 2014;4(3):4875. doi:10.4081/thal.2014.4875

Iron chelation therapy in thalassemia major: A systematic review with meta-analyses of 1520 patients included on randomized clinical trials

Maggio, A.; Filosa, A.; Vitrano, A.; Aloj, G.; Kattamis, A.; Ceci, A.; Fucharoen, S.; Cianciulli, P.; Grady, R. W.; Prossomariti, L.; Porter, J. B.; Iacono, A.; Cappellini, M. D.; Bonifazi, F.; Cassarà, F.; Harmatz, P.; Wood, J.; Gluud, C.
Blood Cells, Molecules & Diseases. 2011;47(3):166–175. doi:10.1016/j.bcmd.2011.07.002

The management of iron chelation therapy: preliminary data from a national registry of thalassaemic patients

Ceci, A.; Mangiarini, L.; Felisi, M.; Bartoloni, F.; Ciancio, A.; Capra, M.; D'Ascola, D.; Cianciulli, P.; Filosa, A.
Anemia. 2011;2011:435683. doi:10.1155/2011/435683

Personalised medicine in paediatrics: individualising treatment in children with rare neurological diseases

Scarpa, M.; Ceci, A.; Tomanin, R.; Mincarone, P.; Begley, D.
EPMA Journal. 2011;2(2):231–239. doi:10.1007/s13167-011-0081-2

Survey of the presence of patulin in fruit juices

Bonerba, E.; Ceci, E.; Conte, R.; Tantillo, G.
Food Additives & Contaminants: Part B Surveillance. 2010;3(2):114–119. doi:10.1080/19393210.2010.490882.

Assessment of dietary intake of patulin from baby foods

Bonerba, E.; Ceci, E.; Conte, R.; Tantillo, G.
Journal of Food Science. 2010;75(7):T123–T125. doi:10.1111/j.1750-3841.2010.01743.x.

In-label and off-label use of respiratory drugs in the Italian paediatric population

Baiardi, P.; Ceci, A.; Felisi, M.; Cantarutti, L.; Girotto, S.; Sturkenboom, M.; Baraldi, E.
Acta Paediatrica. 2010 Apr;99(4):544–9. doi:10.1111/j.1651-2227.2009.01668.x

The european paediatric legislation: benefits and perspectives

Rocchi, F.; Paolucci, P.; Ceci, A.; Rossi, P.
Italian Journal of Pediatrics. 2010 Aug 17;36:56. doi:10.1186/1824-7288-36-56

Availability of medicines for rare diseases in EU countries

Trama, A.; Pierannunzio, D.; Loizzo, A.; Taruscio, D.; Ceci, A.
Pharmaceuticals, Policy and Law. 2009;11(1–2):101–109. doi:10.3233/PPL-2009-0215.

Clinical trials for paediatric medicines in Europe

Baiardi, P.; Girotto, S.; Della Pasqua, O.; Harper, L.; Grosch-Worner, I.; Ceci, A.; Giaquinto, C.
Pharmaceuticals Policy and Law. 2009;11(1-2):71–78. doi:10.3233/PPL-2009-0205

Recommendation for drug development for children

Catapano, M.; Manfredi, C.; Paolucci, P.; Cross, H.; Verhamme, K.; Mellado Pena, M. J.; Grosch-Worner, I.; Knibbe, C.; Ceci, A.
Pharmaceuticals Policy and Law. 2009;11(1–2):61–70. doi:10.3233/PPL-2009-0205

The role of paediatric pharmacogenetic studies in Europe

Krekels, E.; Ceci, A.; Iolascon, A.; Girotto, S.; Della Pasqua, O.
Pharmaceuticals Policy and Law. 2009;11(1–2):23–30. doi:10.3233/PPL-2009-0203

Off-label and unlicensed use of medicines for children

Neubert, A.; Felisi, M.; Bonifazi, A.; Manfredi, C.; Wong, I. C. K.; Ceci, A.
Pharmaceuticals Policy and Law. 2009;11(1–2):41–49. doi:10.3233/PPL-2009-0209

Defining off-label and unlicensed use of medicines for children: Results of a Delphi survey

Neubert, A.; Wong, I. C. K.; Bonifazi, A.; Catapano, M.; Felisi, M.; Baiardi, P.; Giaquinto, C.; Knibbe, C. A.; Sturkenboom, M. C.; Ghaleb, M. A.; Ceci, A.
Pharmacological Research. 2008;58(5–6):316–322. doi:10.1016/j.phrs.2008.09.007.

Drug use in children: cohort study in three European countries

Sturkenboom, M.C.; Verhamm, K.M.; Nicolosi, A.; Murray, M.L.; Neubert, A.; Caudri, D.; Picelli, G.; Sen, E.F.; Giaquinto, C.; Cantarutti, L.; Baiardi, P.; Felisi M.G.; Ceci, A.; Wong, I.C.; TEDDY European Network of Excellence
British Medical Journal. 2008;337:a2245. doi:10.1136/bmj.a2245

Activity of Ethics Committees in Europe on issues related to clinical trials in paediatrics: Results of a survey

Altavilla, A.; Giaquinto, C.; Giocanti, D.; Manfredi, C.; Aboulker, J. P.; Bartoloni, F.; Cattani, E.; Lo Giudice, M.; Mellado Peña, M. J.; Nagler, R.; Peterson, C.; Vajnerova, O.; Bonifazi, F.; Ceci, A.
Pharmaceuticals Policy and Law. 2009;11(1–2):79–87. doi:10.3233/PPL-2009-0208

TEDDY EPMD: A European Paediatric Medicines Database

Felisi, M.; Padula, R.; Bartoloni, F.; Grosch-Worner, I.; Kagedal, B.; Mellado Pena, M. J.; Parry, J.; Stuchlik, A.; Verhamme, K.; Ceci, A.
Pharmaceuticals Policy and Law. 2009;11(1–2):31–39. doi:10.3233/PPL-2009-0211

TEDDY NoE project in the framework of the EU Paediatric Regulation

Ceci, A.; Baiardi, P.; Bonifazi, F.; Giaquinto, C.; Mellado Pena, M. J.; Mincarone, P.; Nicolosi, A.; Sturkenboom, M.; Wong, I.
Pharmaceuticals Policy and Law. 2009;11(1–2):13–21. doi:10.3233/PPL-2009-0206

Italian registry of chemotherapy-induced peripheral neurotoxicity: pilot study phase

Cavaletti, G.; Lanzani, F.; Nobile-Orazio, E.; Gallia, F.; Briani, C.; Lucchetta, M.; Plasmati, R.; Pastorelli, F.; Ceci, A.; Bonifazi, F.
Journal of the Peripheral Nervous System. 2009;14:1–28. doi:10.1111/j.1529-8027.2009.00210.x. Abstracts of the 13th Meeting of the Italian Peripheral Nerve Study Group; 2009 May 14–16; Otranto, Italy.

Challenges in prescribing drugs for children with cancer

Paolucci, P.; Jones, K.P.; del Carmen Cano Garcinuno, M.; Catapano, M.; Iolascon, A.; Ceci A.
The Lancet Oncology. 2008;9(2):176–183. doi:10.1016/S1470-2045(08)70030-5

Medicines for children licensed by the European Medicines Agency (EMEA): the balance after 10 years

Ceci, A.; Felisi, M.; Baiardi, P.; Bonifazi, F.; Catapano, M.; Giaquinto, C.; Nicolosi, A.; Sturkenboom, M.; Neubert, A.; Wong, I.
European Journal of Clinical Pharmacology. 2006;62(11):947–952. doi:10.1007/s00228-006-0193-0

Risk factors for death in patients with beta-thalassemia major: results of a case-control study

Ceci, A.; Baiardi, P.; Catapano, M.; Felisi, M.; Cianciulli, P.; De Sanctis, V.; Del Vecchio, G. C.; Magnano, C.; Meo, A.; Maggio, A.
Haematologica. 2006;91(10):1420–1421. PMID:16963395

Pharmacotherapy of iron overload in thalassaemic patients

Ceci, A.; Felisi, M.; De Sanctis, V.; De Mattia, D.
Expert Opinion on Pharmacotherapy. 2003;4(10):1763–1774. doi:10.1517/14656566.4.10.1763

Medicines for children licensed by the European Agency for the Evaluation of Medicinal Products

Ceci, A.; Felisi, M.; Catapano, M.; Baiardi, P.; Cipollina, L.; Ravera, S.; Bagnulo, S.; Reggio, S.; Rondini, G.
European Journal of Clinical Pharmacology. 2002;58(8):495–500. doi:10.1007/s00228-002-0511-0

Mutua disponibilità, prezzi e rimborsabilità di farmaci autorizzati con procedura centralizzata europea

Cipollina, L.; Folino-Gallo, P.; Grande, D.; Felisi, M.; Ravera, S.; Ceci, A.
Farmeconomia. Health Economics and Therapeutic Pathways. 2002;3(1):15–27. doi:10.7175/fe.v3i1.740.

Drugs and food supplements in sports. The nandrolone lessons

Benzi G, Ceci A.
Minerva Pediatrica. 2001 Oct;53(5):395–396. PMID:11668257

Creatine as nutritional supplementation and medicinal product

Benzi, G.; Ceci, A.
Journal of Sports Medicine and Physical Fitness. 2001;41(1):1–10. PMID:11317142.