Abstract

The genotype–phenotype correlation of the X-linked Alport syndrome (XLAS) has been well elucidated in males, whereas it remains unclear in females. In this multicenter retrospective study, we analyzed the genotype–phenotype correlation in 216 Korean patients (male:female = 130:86) with XLAS between 2000 and 2021. The patients were divided into three groups according to their genotypes: the non-truncating group, the abnormal splicing group, and the truncating group. In male patients, approximately 60% developed kidney failure at the median age of 25.0 years, and kidney survival showed significant differences between the non-truncating and truncating groups (P < 0.001, hazard ratio (HR) 2.8) and splicing and truncating groups (P = 0.002, HR 3.1). Sensorineural hearing loss was detected in 65.1% of male patients, while hearing survival periods showed a highly significant difference between the non-truncating and truncating groups (P < 0.001, HR 5.1). In female patients, approximately 20% developed kidney failure at the median age of 50.2 years. The kidney survival was significantly different between the non-truncating and truncating groups (P = 0.006, HR 5.7). Our findings support the presence of genotype–phenotype correlation not only in male patients but also in female patients with XLAS.

Details

Title
Genotype–phenotype correlation of X-linked Alport syndrome observed in both genders: a multicenter study in South Korea
Author
Kim, Ji Hyun 1 ; Lim, Seon Hee 2 ; Song, Ji Yeon 2 ; Cho, Myung Hyun 3 ; Hyun, HyeSun 4 ; Yang, Eun Mi 5 ; Lee, Jung Won 6 ; Cho, Min Hyun 7 ; Park, Min Ji 7 ; Lee, Joo Hoon 8 ; Jung, Jiwon 8 ; Yoo, Kee Hwan 9 ; Jang, Kyung Mi 10 ; Pai, Ki Soo 11 ; Suh, Jin-Soon 12 ; Namgoong, Mee Kyung 13 ; Chung, Woo Yeong 14 ; Kim, Su Jin 15 ; Cho, Eun Young 16 ; Kim, Kyung Min 16 ; Kim, Nam Hee 17 ; Kim, Minsun 18 ; Paik, Jin Ho 19 ; Kang, Hee Gyung 20 ; Ahn, Yo Han 20 ; Cheong, Hae Il 21 

 Seoul National University Bundang Hospital, Department of Pediatrics, Seongnam, South Korea (GRID:grid.412480.b) (ISNI:0000 0004 0647 3378); Seoul National University College of Medicine, Department of Pediatrics, Seoul, South Korea (GRID:grid.31501.36) (ISNI:0000 0004 0470 5905) 
 Pusan National University Yangsan Children’s Hospital and School of Medicine, Department of Pediatrics, Yangsan, South Korea (GRID:grid.262229.f) (ISNI:0000 0001 0719 8572) 
 Hallym University Sacred Heart Hospital, Department of Pediatrics, Anyang, South Korea (GRID:grid.488421.3) (ISNI:0000000404154154) 
 The Catholic University of Korea, Department of Pediatrics, College of Medicine, St. Vincent’s Hospital, Seoul, South Korea (GRID:grid.411947.e) (ISNI:0000 0004 0470 4224) 
 Chonnam National University and School of Medicine, Department of Pediatrics, Gwangju, South Korea (GRID:grid.14005.30) (ISNI:0000 0001 0356 9399) 
 Ewha Womans University School of Medicine, Department of Pediatrics, Seoul, South Korea (GRID:grid.255649.9) (ISNI:0000 0001 2171 7754) 
 Kyungpook National University, School of Medicine, Department of Pediatrics, Daegu, South Korea (GRID:grid.258803.4) (ISNI:0000 0001 0661 1556) 
 University of Ulsan College of Medicine, Department of Pediatrics, Asan Medical Center Children’s Hospital, Seoul, South Korea (GRID:grid.267370.7) (ISNI:0000 0004 0533 4667) 
 Woori Children’s Hospital, Department of Nephrology, Seoul, South Korea (GRID:grid.267370.7) 
10  Yeungnam University College of Medicine, Department of Pediatrics, Gyeongsan, South Korea (GRID:grid.413028.c) (ISNI:0000 0001 0674 4447) 
11  Ajou University School of Medicine, Department of Pediatrics, Suwon, South Korea (GRID:grid.251916.8) (ISNI:0000 0004 0532 3933) 
12  The Catholic University of Korea, Department of Pediatrics, Bucheon St. Mary’s Hospital, College of Medicine, Seoul, South Korea (GRID:grid.411947.e) (ISNI:0000 0004 0470 4224) 
13  Yonsei University Wonju College of Medicine, Department of Pediatrics, Wonju Severance Christian Hospital, Wonju, South Korea (GRID:grid.15444.30) (ISNI:0000 0004 0470 5454) 
14  WooYeong Chung Pediatrics Clinic, Busan, South Korea (GRID:grid.15444.30) 
15  Inha University College of Medicine, Inha University Hospital, Department of Pediatrics, Incheon, South Korea (GRID:grid.15444.30) 
16  Chungnam National University Hospital, Department of Pediatrics, Daejeon, South Korea (GRID:grid.411665.1) (ISNI:0000 0004 0647 2279) 
17  Inje University Ilsan Paik Hospital, Department of Pediatrics, Goyang, South Korea (GRID:grid.411633.2) (ISNI:0000 0004 0371 8173) 
18  Jeonbuk National University Medical School, Department of Pediatrics, Jeonju, South Korea (GRID:grid.411545.0) (ISNI:0000 0004 0470 4320) 
19  Seoul National University Bundang Hospital and Seoul National University College of Medicine, Department of Pathology, Seongnam, South Korea (GRID:grid.412480.b) (ISNI:0000 0004 0647 3378) 
20  Seoul National University College of Medicine, Department of Pediatrics, Seoul, South Korea (GRID:grid.31501.36) (ISNI:0000 0004 0470 5905); Seoul National University Children’s Hospital, Department of Pediatrics, Seoul, South Korea (GRID:grid.412482.9) (ISNI:0000 0004 0484 7305); Seoul National University, Kidney Research Institute, Medical Research Center, Seoul, South Korea (GRID:grid.31501.36) (ISNI:0000 0004 0470 5905) 
21  Seoul Red Cross Hospital, Department of Pediatrics, Seoul, South Korea (GRID:grid.31501.36) 
Pages
6827
Publication year
2023
Publication date
2023
Publisher
Nature Publishing Group
e-ISSN
20452322
Source type
Scholarly Journal
Language of publication
English
ProQuest document ID
2806316104
Copyright
© The Author(s) 2023. This work is published under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.