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. 2025 Feb 25;9(12):3090–3103. doi: 10.1182/bloodadvances.2024013953

Table 3.

Recommended assessment of additional organ functions

Organ Assessment Interval
Growth Height, weight, and body mass index Yearly ×2
Hormone levels if short stature (IGF-1 and IGFBP-3) Subsequently yearly if abnormal and within growth period
Bone age
Thyroid function Thyroid-stimulating hormone At 6 months, 1 year, and then annually
Free thyroxine level
Glucose metabolism Fasting blood sugar Annual
Glucose tolerance test As needed
Lipid metabolism Fasting lipid panel Annual
Bone health Vitamin D3 level Annual
Bone mineral density 2 years and subsequently if intervention planned
Dental health Dental evaluation Annual
Visual function Vision test Annual
Glaucoma screening
Retinal vessel assessment
Iron accumulation Serum ferritin and transferrin saturation Before intervention
Cardiac and liver MRI 6 months, 1 year, and yearly until normal with therapy/phlebotomy

IGF-1, insulin-like growth factor 1; IGFBP-3, insulin-like growth factor binding protein-3.