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Patient handout

Thalassaemia (TDT / NTDT / α-thal-HbH / trait — chronic management)

PRODUCTION

1. Your condition

This handout is for thalassaemia (tdt / ntdt / α-thal-hbh / trait — chronic management). Your care team identified this based on: microcytic hypochromic anaemia on cbc — discriminate thalassaemia trait vs ida (tif 5th ed 2025 pmid 40045934).

Other reasons your team may use this plan: known thalassaemia on problem list — comprehensive clinic visit (tif 5th ed 2025); abnormal hb electrophoresis/hplc (↑hba2 / ↑hbf / hbh / hbe) (tif 5th ed 2025); positive antenatal/newborn carrier screen → genetic counselling (tif 5th ed 2025).

2. Your medications

Take these medications exactly as prescribed. Do not stop or change a dose without talking to your provider.

MedicationStarting doseHowWhenWhat it does
deferasirox14 mg/kg/day (film-coated tablet); titrate 7–28 mg/kgPOonce dailyFirst-line PO chelator; titrate by ferritin/LIC trend. Boxed warning: renal/hepatic failure, GI haemorrhage — monitor eGFR + LFT + GI symptoms (TIF 5th ed 2025 PMID 40045934)
deferoxamine20–40 mg/kg/day SC over 8–12 h; up to 50–60 mg/kg/day IV (intensive) for siderotic cardiomyopathySC5–7 nights/week (continuous IV if cardiac decompensation)Pregnancy-preferred chelator; 24-h IV for iron cardiomyopathy; backbone of DFP+DFO combination (Tanner Circulation 2007 PMID 17372174)
deferiprone75 mg/kg/day divided TID (up to 99 mg/kg/day)POthree times dailySuperior myocardial iron clearance; combination with deferoxamine for cardiac T2* <10 ms (Tanner PMID 17372174). MANDATORY weekly ANC — agranulocytosis ~1–2% (TIF 5th ed 2025)
folic acid5 mgPOonce dailySupports increased erythropoietic folate demand in chronic haemolysis / ineffective erythropoiesis (TIF NTDT 2023 NBK599489)

Plan: Iron chelation (compartment- and cardiac-T2*-directed)

3. When to call your provider

Contact your care team if any of the following happen:

  • Cardiac T2* <10 ms / new HF symptoms → cardiology + intensive combination chelation + admit (Kirk/Pennell PMID 19801505)
  • Acute Hb drop with low reticulocytes → aplastic crisis (parvovirus) → transfusion + admit (TIF 5th ed 2025)
  • Fever in splenectomised patient → ED, treat as encapsulated-organism sepsis (TIF 5th ed 2025)
  • New thrombosis / pulmonary hypertension in NTDT → ED + anticoagulation assessment (TIF NTDT 2023 NBK599489)
  • Deferiprone ANC <0.5 → STOP deferiprone, urgent assessment (TIF 5th ed 2025)

4. When to seek emergency care

Call 911 or go to the nearest emergency room right away if you have:

  • Cardiac T2* <10 ms and/or new heart failure / arrhythmia from myocardial siderosis (RR≈160 for HF vs T2* >10 ms; 47% HF within 1 yr if T2* <6 ms) (Kirk/Pennell Circulation 2009 PMID 19801505)(life-threatening)
  • Acute Hb drop below baseline with LOW reticulocyte count — parvovirus B19 transient red-cell aplasia (TIF 5th ed 2025 PMID 40045934)
  • Fever in a splenectomised / functionally asplenic thalassaemia patient — overwhelming encapsulated-organism infection until proven otherwise (TIF 5th ed 2025 PMID 40045934)(life-threatening)
  • New venous/arterial thrombosis or pulmonary hypertension in NTDT or post-splenectomy thalassaemia (high prothrombotic state) (TIF NTDT 2023 NBK599489)
  • Deferiprone-associated ANC <0.5 ×10⁹/L (agranulocytosis ~1–2%) (TIF 5th ed 2025 PMID 40045934)(life-threatening)

5. Follow-up

Annual endocrinopathy screen — hypogonadism, growth failure, diabetes, hypothyroidism, hypoparathyroidism, osteoporosis (bone densitometry; calcium + vitamin D); cardiac + hepatic surveillance (HCC screening if cirrhotic — route gi.cirrhosis.core.v1); fertility / pregnancy planning + pre-conception chelation washout; vaccination + lifelong penicillin if splenectomised; transition pediatric→adult care; partner + family genetic counselling, prenatal/PGT options (TIF 5th ed 2025; NTDT 2023 NBK599489)

6. Sources

Guideline: TIF Guidelines for the Management of Transfusion-Dependent β-Thalassaemia, 5th edition 2025 (Musallam/Taher HemaSphere, PMID 40045934) + TIF NTDT 3rd edition 2023 (NBK599489) + BELIEVE/BEYOND luspatercept + beti-cel HGB-207/212 + exa-cel CLIMB THAL-111 + cardiac-T2* (Kirk/Pennell) + combined chelation (Tanner)

  1. pubmed.ncbi.nlm.nih.gov/40045934
  2. pubmed.ncbi.nlm.nih.gov/32212518
  3. pubmed.ncbi.nlm.nih.gov/36007538