Frontiers in Pediatrics (Jul 2024)
Rapid genome sequencing for critically ill infants: an inaugural pilot study from Turkey
- Bengisu Guner Yilmaz,
- Ozlem Akgun-Dogan,
- Ozlem Akgun-Dogan,
- Ozlem Akgun-Dogan,
- Ozkan Ozdemir,
- Ozkan Ozdemir,
- Ozkan Ozdemir,
- Bayram Yuksel,
- Ozden Hatirnaz Ng,
- Ozden Hatirnaz Ng,
- Kaya Bilguvar,
- Kaya Bilguvar,
- Beril Ay,
- Gulsah Sebnem Ozkose,
- Eylul Aydin,
- Ayca Yigit,
- Aybike Bulut,
- Fatma Nisa Esen,
- Serdar Beken,
- Selma Aktas,
- Atalay Demirel,
- Baran Cengiz Arcagok,
- Ebru Kazanci,
- İbrahim Bingol,
- Ozge Umur,
- Guntulu Sik,
- Ugur Isik,
- Melike Ersoy,
- Ayse Korkmaz,
- Agop Citak,
- Adil Mardinoglu,
- Adil Mardinoglu,
- Adil Mardinoglu,
- Ugur Ozbek,
- Ugur Ozbek,
- Ugur Ozbek,
- Yasemin Alanay,
- Yasemin Alanay,
- Yasemin Alanay
Affiliations
- Bengisu Guner Yilmaz
- Department of Pediatrics, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Ozlem Akgun-Dogan
- Division of Pediatric Genetics, Department of Pediatrics, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Ozlem Akgun-Dogan
- Acibadem Mehmet Ali Aydinlar University Rare Diseases and Orphan Drugs Application and Research Center (ACURARE), Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Ozlem Akgun-Dogan
- Department of Transitional Medicine, Health Sciences Institute, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Ozkan Ozdemir
- Acibadem Mehmet Ali Aydinlar University Rare Diseases and Orphan Drugs Application and Research Center (ACURARE), Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Ozkan Ozdemir
- Department of Genome Studies, Health Sciences Institute, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Ozkan Ozdemir
- Division of Medical Biology, Department of Basic Sciences, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Bayram Yuksel
- Genetic Diagnosis Center, SZA OMICS, Istanbul, Turkey
- Ozden Hatirnaz Ng
- Acibadem Mehmet Ali Aydinlar University Rare Diseases and Orphan Drugs Application and Research Center (ACURARE), Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Ozden Hatirnaz Ng
- Division of Medical Biology, Department of Basic Sciences, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Kaya Bilguvar
- Acibadem Mehmet Ali Aydinlar University Rare Diseases and Orphan Drugs Application and Research Center (ACURARE), Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Kaya Bilguvar
- Department of Medical Genetics, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Beril Ay
- School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Gulsah Sebnem Ozkose
- Department of Genome Studies, Health Sciences Institute, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Eylul Aydin
- Department of Genome Studies, Health Sciences Institute, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Ayca Yigit
- Department of Genome Studies, Health Sciences Institute, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Aybike Bulut
- Department of Genome Studies, Health Sciences Institute, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Fatma Nisa Esen
- 0Acibadem Labgen Genetic Diagnosis Center, Istanbul, Turkey
- Serdar Beken
- 1Division of Neonatology, Department of Pediatrics, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Selma Aktas
- 1Division of Neonatology, Department of Pediatrics, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Atalay Demirel
- 1Division of Neonatology, Department of Pediatrics, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Baran Cengiz Arcagok
- 1Division of Neonatology, Department of Pediatrics, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Ebru Kazanci
- 1Division of Neonatology, Department of Pediatrics, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- İbrahim Bingol
- 2Division of Intensive Care, Department of Pediatrics, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Ozge Umur
- 2Division of Intensive Care, Department of Pediatrics, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Guntulu Sik
- 2Division of Intensive Care, Department of Pediatrics, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Ugur Isik
- 3Division of Neurology, Department of Pediatrics, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Melike Ersoy
- 4Division of Pediatric Metabolism, Department of Pediatrics, University of Health Sciences, Bakırkoy Dr. Sadi Konuk Training and Research, Istanbul, Turkey
- Ayse Korkmaz
- 1Division of Neonatology, Department of Pediatrics, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Agop Citak
- 2Division of Intensive Care, Department of Pediatrics, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Adil Mardinoglu
- Genetic Diagnosis Center, SZA OMICS, Istanbul, Turkey
- Adil Mardinoglu
- 5Faculty of Dentistry, Oral & Craniofacial Sciences, Centre for Host-Microbiome Interactions, King's College London, London, United Kingdom
- Adil Mardinoglu
- 6Science for Life Laboratory, KTH-Royal Institute of Technology, Stockholm, Sweden
- Ugur Ozbek
- Acibadem Mehmet Ali Aydinlar University Rare Diseases and Orphan Drugs Application and Research Center (ACURARE), Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Ugur Ozbek
- Department of Genome Studies, Health Sciences Institute, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Ugur Ozbek
- Department of Medical Genetics, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Yasemin Alanay
- Division of Pediatric Genetics, Department of Pediatrics, School of Medicine, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Yasemin Alanay
- Acibadem Mehmet Ali Aydinlar University Rare Diseases and Orphan Drugs Application and Research Center (ACURARE), Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- Yasemin Alanay
- Department of Genome Studies, Health Sciences Institute, Acibadem Mehmet Ali Aydinlar University, Istanbul, Turkey
- DOI
- https://doi.org/10.3389/fped.2024.1412880
- Journal volume & issue
-
Vol. 12
Abstract
IntroductionRare and ultra-rare genetic conditions significantly contribute to infant morbidity and mortality, often presenting with atypical features and genetic heterogeneity that complicate management. Rapid genome sequencing (RGS) offers a timely and cost-effective approach to diagnosis, aiding in early clinical management and reducing unnecessary interventions. This pilot study represents the inaugural use of next-generation sequencing (NGS) as a diagnostic instrument for critically ill neonatal and pediatric ICU patients in a Turkish hospital setting.MethodsTen infants were enrolled based on predefined inclusion criteria, and trio RGS was performed. The mean age of the participants was 124 days, with congenital abnormalities being the most common indication for testing. Three patients had consanguineous parents. The mean turnaround time from enrollment to delivery of results was 169 h, with a diagnostic yield of 50%.ResultsThree patients received a definitive molecular diagnosis, impacting their clinical management. Two patients benefited from the exclusion of Mendelian conditions, leading to alternative diagnoses.DiscussionThis study demonstrates the feasibility and results of RGS in Turkish hospital settings, emphasizing the importance of timely genetic diagnosis in reducing the diagnostic odyssey for families and improving patient care. Further research is needed to evaluate the cost-effectiveness and applicability of RGS in the Turkish healthcare system for children with diseases of uncertain etiology.
Keywords
- rapid genome sequencing
- critically ill patient
- pediatric intensive care unit
- neonatal intensive care unit
- trio sequencing