重型β-地中海贫血患儿在迪拜地中海贫血中心的生长和内分泌疾病情况。
Growth and endocrinopathies among children with β-Thalassemia major treated at Dubai Thalassemia centre.
机构信息
Department of Clinical Sciences, College of Medicine, University of Sharjah, Sharjah, United Arab Emirates.
Department of Family & Community Medicine and Behavioral Sciences, College of Medicine, University of Sharjah, Sharjah, United Arab Emirates.
出版信息
BMC Pediatr. 2024 Apr 5;24(1):244. doi: 10.1186/s12887-024-04670-w.
BACKGROUND
β-Thalassemia major (BTM) is one of the most common hereditary anemias worldwide. Patients suffer from iron overload that results from repeated blood transfusion This in turn leads to multiple organ damage and endocrinopathies. This study aims to assess the prevalence of growth retardation, hypothyroidism, and diabetes mellitus in children and adolescents with BTM treated at Dubai Thalassemia Centre.
METHODS
A total of 105 children and adolescents were included in this retrospective observational study.
RESULTS
39 children and 66 adolescents' data were analyzed. Females composed 51.3% (n = 20) of children and 53.0% (n = 35) of adolescents. Pretransfusion hemoglobin below 9 gm/dl was observed in 10.8% (n = 4) and 10.6% (n = 7) in children and adolescents, respectively. The mean age of menarche was 13.5 years. Among all study participants, 22.6% (n = 14) had normal height velocity whereas 37.1% (n = 23) had reduced height velocity in one year and 40.3% (n = 25) had reduced height velocity in two consecutive years. The proportion of children and adolescents showing reduced height velocity was significantly higher in females compared to the males (90.6% versus 63.3%, respectively, Chi-square = 6.597, p-value = 0.010). Although none of the study participants had diabetes mellitus, 26.1% (n = 12/46) had pre-diabetes. Elevated TSH was observed in 14.7% (n = 5) children and 8.1% (n = 5) adolescents while low FT4 was reported in one child and one adolescent.
CONCLUSION
Of all endocrinopathies seen among children and adolescents with BTM, growth delay remains the main concern for this group of patients. Effective treatment is key to further reducing endocrinopathies. Although the sample size is limited, we postulate that the low percentage of endocrinopathies among children with BTM treated at Dubai thalassemia center and the low level of pretransfusion anemia reflect the effective transfusion and chelation at the center.
背景
β-地中海贫血（BTM）是全球最常见的遗传性贫血症之一。患者因反复输血而遭受铁过载，进而导致多器官损伤和内分泌疾病。本研究旨在评估在迪拜地中海贫血中心接受治疗的 BTM 患儿和青少年生长迟缓、甲状腺功能减退和糖尿病的患病率。
方法
本回顾性观察研究共纳入 105 名患儿和青少年。
结果
分析了 39 名儿童和 66 名青少年的数据。女性分别占儿童的 51.3%（n=20）和青少年的 53.0%（n=35）。儿童和青少年中，输血前血红蛋白低于 9gm/dl 的比例分别为 10.8%（n=4）和 10.6%（n=7）。初潮的平均年龄为 13.5 岁。在所有研究参与者中，22.6%（n=14）的身高增长速度正常，而 37.1%（n=23）在一年内身高增长速度降低，40.3%（n=25）在连续两年内身高增长速度降低。女性中身高增长速度降低的儿童和青少年比例明显高于男性（分别为 90.6%和 63.3%，卡方=6.597，p 值=0.010）。尽管没有研究参与者患有糖尿病，但 26.1%（n=12/46）患有糖尿病前期。儿童中 TSH 升高的比例为 14.7%（n=5），青少年为 8.1%（n=5），而一名儿童和一名青少年的 FT4 水平较低。
结论
在 BTM 患儿和青少年中，所有内分泌疾病中，生长迟缓仍然是这群患者的主要关注点。有效的治疗是进一步减少内分泌疾病的关键。尽管样本量有限，但我们推测，在迪拜地中海贫血中心接受治疗的 BTM 儿童中，内分泌疾病的比例较低，输血前贫血程度较低，反映了中心有效的输血和螯合作用。
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