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 Direct antiglobulin test (DAT; direct Coombs) as clinically indicated	Hematology: CBC at any transfusion Blood type and antibody screen monthly if receiving chronic transfusions Direct antiglobulin test (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 transfusion-dependent thalassemia (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 girls at age 12 years and for boys at age 14 years. Refer to UpToDate for additional discussions of thalassemia management.

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

* If there is a concern that hemolysis may falsely lower the HbA1c, the other methods (fasting glucose or oral glucose tolerance test) may be preferred.

Modified with permission from: UCSF Benioff Children's Hospital Oakland, Northern California Comprehensive Thalassemia Center. Treating Thalassemia: Checklist. Available at: http://thalassemia.com/treatment-timetable.aspx#gsc.tab=0 (Accessed on September 27, 2018). Copyright © 2017 Children's Hospital & Research Center Oakland.

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