Delayed Physical Growth and Related Factors in Pediatric Patients with Transfusion-Dependent Thalassemia

Objectives To investigate the physical growth status of pediatric patients with transfusion-dependent thalassemia (TDT) and assess the clinical and economic factors associated with delayed physical growth.

Methods We extracted sociodemographic and clinical data on 338 pediatric patients with TDT, including physical growth indicators (height and weight), family economic status and disease related treatment, from thalassemia disease database at a grade-A tertiary hematological specialty hospital in China between October 2023 and May 2024. Length/height-for-age and body mass index-for-age (BMI-for-age) were assessed using the ‘Growth Standard for Children under 7 Years of Age’ and the ‘Standard for Height Level Classification among Children and Adolescents Aged 7-18 Years’ issued by the National Health Commission, as well as the ‘Dietary Guidelines for Chinese Residents’. Logistic regression was performed to explore the impact of sociodemographic and clinical factors on length/height-for-age and BMI-for-age, respectively.

Results Among the 338 pediatric patients with TDT, 192 were male and 146 were female, with a median age of 12 (0.8-18.0) years and a median time since diagnosis of 10.3 (0.5-17.9) years. Of these patients, 21 (6.2%) had ⍺ thalassemia, 288 (85.2%) had β thalassemia, and 29 (8.6%) had mixed ⍺ and β thalassemia. Overall, 88 (26.00%) children were stunted as their length/height failing below -2 standard deviations (SDs) from the national growth standard median. There were 95 (28.1%) children with length/height between -2 SDs and -1SD, and 155(45.9%) children with length/height≥-1SD. Meanwhile, 77 (22.8%) patients were underweight, 241 (71.3%) were at a healthy weight, and 20 (5.9%) were classified as overweight/obese based on their BMI-for-age. The majority of TDT children had a family monthly income falling within the ranges of 3000-5000 yuan and 5000-10000 yuan, accounting for 135 (39.9%) and 118 (34.9%) cases, respectively. Pre-transfusion hemoglobin (HGB) levels were categorized as ≤70 g/L, 70-90 g/L, and >90 g/L in 19 (5.6%), 123 (36.4%), and 196 (58.0%) cases, respectively. Thirty (8.9%) pediatric patients exhibited serum ferritin (SF) levels of ≥5000ug/L, 321 (95.0%) received chelation therapy, and 16 (4.7%) underwent splenectomy. Multivariate regression analysis revealed that pre-transfusion HGB≤70g/L, SF≥5000ug/L, and a monthly family income below 10000 yuan were all significantly associated with an increased risk of stunting (all P<0.05). Moreover, a longer time since diagnosis was a hazardous factor for being underweight (P=0.013).

Conclusions Pediatric patients with TDT who had pre-transfusion HGB≤70g/L, SF≥5000ug/L, lower monthly family income or longer time since diagnosis were at a higher risk for delayed physical growth.