Checklist for routine monitoring in individuals with transfusion-dependent thalassemia (TDT)

Category	Children	Adults
General health	Testing prior to transfusion and possible transplant (all done once at initial evaluation): HLA typing DNA mapping (alpha and beta globin genes) RBC phenotyping	Testing prior to transfusion and possible transplant (all done once if not already available): HLA typing DNA mapping (alpha and beta globin genes) RBC phenotyping
	Assessment of iron overload: Total volume of RBCs transfused – Assess every 6 to 12 months Serum ferritin – Every 3 months Iron, TIBC, TSAT – As clinically indicated (eg, if ferritin level is higher than expected) Baseline liver ultrasound at the time of diagnosis, repeated twice yearly in those with severe liver disease, iron overload, or cirrhosis Liver iron (eg, by MRI) upon increases in ferritin or changes in clinical condition that suggest increased iron burden Cardiac MRI at initial evaluation and if there are significant increases in ferritin	Assessment of iron overload: Total volume of RBCs transfused – Assess every 6 to 12 months Serum ferritin – Every 3 months Iron, TIBC, TSAT – As clinically indicated (eg, if ferritin level is higher than expected) Liver ultrasound twice yearly in those with severe liver disease, iron overload, or cirrhosis Liver iron (eg, by MRI) upon increases in ferritin or changes in clinical condition that suggest increased iron burden Cardiac MRI if there are significant increases in ferritin
	CBC, reticulocyte count, and assessment of kidney function at each visit	CBC, reticulocyte count, and assessment of kidney function at each visit
	Chemistry panel including serum calcium every 3 months	Chemistry panel including serum calcium every 3 to 6 months
	Urinalysis every 6 months
	Growth and development: Weight monthly in infants, every 3 to 4 months in older children and adolescents Head circumference every other month in infants Annual growth velocity in children Tanner stage every 6 months in older children and adolescents	Growth and development: Weight every 3 to 6 months Standing height every 3 to 6 months
	Routine dental evaluation every 6 months	Routine dental evaluation every 6 months
	Vision screening every 3 months; ophthalmology examination annually	Routine ophthalmology examination every 6 months
	Audiology screen annually
Specific organ systems	Hematology: CBC at any transfusion Blood type and antibody screen monthly if receiving chronic transfusions DAT (direct Coombs) as clinically indicated	Hematology: CBC at any transfusion Blood type and antibody screen monthly if receiving chronic transfusions DAT (direct Coombs) as clinically indicated
	Hepatology: Transaminases (AST, ALT) and bilirubin (direct and total) every 3 months Hepatitis A, B, and C serology and serum albumin annually Hepatitis B and C PCR as clinically indicated PT and aPTT as clinically indicated	Hepatology: Transaminases (AST, ALT) and bilirubin (direct and total) every 3 to 6 months Hepatitis A, B, and C serology and serum albumin annually Hepatitis B and C PCR as clinically indicated PT and aPTT as clinically indicated
	Cardiopulmonary: Echocardiography to evaluate for pulmonary hypertension if chronic hemolysis and/or iron overload are present	Cardiopulmonary: Echocardiography to evaluate for pulmonary hypertension if chronic hemolysis and/or iron overload are present
	Endocrinology: T3, free T4, TSH annually starting at age 5 years PTH, calcium, and ionized calcium annually starting at age 5 years Fasting glucose, HbA1c, or oral glucose tolerance test* at ages 10, 12, 14, and 16 years IGF-1 and IGFBP-3 as clinically indicated (for growth delay) LH-ICMA, FSH, and estradiol as clinically indicated (for delayed puberty in females) Testosterone as clinically indicated (for delayed puberty in males) Bone density testing annually after adolescence	Endocrinology: T3, free T4, TSH annually PTH, calcium, and ionized calcium annually Fasting glucose, HbA1c*, or oral glucose tolerance test annually IGF-1 and IGFBP-3 as clinically indicated (for growth delay) Bone density testing annually

This is a general timetable for clinical and laboratory evaluations for individuals with TDT. TDT includes conditions previously referred to as thalassemia major as well as some with thalassemia intermedia; clinical judgment is used to determine the appropriate interval for all testing and the specific testing and timetable in thalassemia intermedia phenotypes. Evaluations for delayed puberty are generally done for females at age 12 years and for males at age 14 years. Refer to UpToDate for additional discussions of thalassemia management.

ALT: alanine aminotransferase; aPTT: activated partial thromboplastin time; AST: aspartate aminotransferase; CBC: complete blood count; DAT: direct antiglobulin test; DNA: deoxyribonucleic acid; FSH: follicle-stimulating hormone; HbA1c: glycosylated hemoglobin; HLA: human leukocyte antigen; IGF-1: insulin-like growth factor-1; IGFBP-3: IGF binding protein 3; LH-ICMA: luteinizing hormone-immunochemiluminometric assay; MRI: magnetic resonance imaging; PCR: polymerase chain reaction; PT: prothrombin time; PTH: parathyroid hormone; RBC: red blood cell; TIBC: total iron binding capacity; TSAT: transferrin saturation; TSH: thyroid-stimulating hormone.

* Chronic hemolysis and transfused RBCs both can artifactually lower the HbA1c and provide misleading information regarding glucose regulation, especially if used as a one-time isolated test. For diabetes screening, fasting blood glucose and an oral glucose tolerance test should be included in the diagnostic thinking. For individuals with steady-state transfusion requirements and clinical stability, the HbA1c may be useful to follow as a means of monitoring changes from their baseline (using their prior values as a reference for detecting changes over time).

Modified with permission from: UCSF Benioff Children's Hospital Oakland, Northern California Comprehensive Thalassemia Center. Treating Thalassemia: Checklist. Copyright © 2017 Children's Hospital & Research Center Oakland.

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Checklist for routine monitoring in individuals with transfusion-dependent thalassemia (TDT)