TY - JOUR
T1 - Unusual Causes of β Thalassemia Trait
T2 - Discovery of another Three Novel SUPT5H Variants
AU - Nik Mohd Hasan, Nik Fatma Fairuz
AU - Achour, Ahlem
AU - Koopmann, Tamara
AU - Gammeren, Adriaan van
AU - van der Leeuw, Joep
AU - Ceelie, Huib
AU - Stieber, Daniel
AU - Baas, Frank
AU - Harteveld, Cornelis L.
N1 - Publisher Copyright:
© 2025 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group.
PY - 2025/3/30
Y1 - 2025/3/30
N2 - Beta (β) thalassemia is an inherited disorder that occurs following mutations or deletions in the β globin gene. Rarely, it is caused by variants in genes coding for erythroid transcriptional factors or trans-acting factors. Here, we report three novel variants of SUPT5H revealed by next generation sequencing. This, gene has been progressively acknowledged as a mimicker of β thalassemia trait in two independent individuals and one family. These individuals have the same features, including hypochromic microcytic indices, increased Hb A2 levels, without mutations in the β globin gene. The three novel SUPT5H variants identified in this study (c.1168_1169del, c.2688del and c.307+1G>A) are frameshift variants leading to a premature stop codon or an intronic variant predicted to alter the splice site consensus sequence by in silico software. All three variants are characterized as Loss-of-Function variants either by generating a truncated protein or haplo-insufficiency due to nonsense-mediated decay. These findings confirm the general observation that most variants in SUPT5H associated with a β thalassemia trait phenotype are Loss-of-Function variants. This gene should be considered as a potential target gene in the genetic diagnosis of any unsolved cases of increased HbA2 and unexplained inconsistency of phenotype and genotype of β thalassemia intermedia.
AB - Beta (β) thalassemia is an inherited disorder that occurs following mutations or deletions in the β globin gene. Rarely, it is caused by variants in genes coding for erythroid transcriptional factors or trans-acting factors. Here, we report three novel variants of SUPT5H revealed by next generation sequencing. This, gene has been progressively acknowledged as a mimicker of β thalassemia trait in two independent individuals and one family. These individuals have the same features, including hypochromic microcytic indices, increased Hb A2 levels, without mutations in the β globin gene. The three novel SUPT5H variants identified in this study (c.1168_1169del, c.2688del and c.307+1G>A) are frameshift variants leading to a premature stop codon or an intronic variant predicted to alter the splice site consensus sequence by in silico software. All three variants are characterized as Loss-of-Function variants either by generating a truncated protein or haplo-insufficiency due to nonsense-mediated decay. These findings confirm the general observation that most variants in SUPT5H associated with a β thalassemia trait phenotype are Loss-of-Function variants. This gene should be considered as a potential target gene in the genetic diagnosis of any unsolved cases of increased HbA2 and unexplained inconsistency of phenotype and genotype of β thalassemia intermedia.
KW - Increased Hb A
KW - mutation
KW - SUPT5H
KW - β thalassemia
UR - http://www.scopus.com/inward/record.url?scp=105001940502&partnerID=8YFLogxK
U2 - 10.1080/03630269.2025.2484230
DO - 10.1080/03630269.2025.2484230
M3 - Article
AN - SCOPUS:105001940502
SN - 0363-0269
VL - 49
SP - 145
EP - 148
JO - Hemoglobin
JF - Hemoglobin
IS - 2
ER -