Investigations

1st investigations to order

haemoglobin (Hb)

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Hb will be normal in alpha-thalassaemia silent carrier and may be slightly low in alpha-thalassaemia trait.

The Hb is variable in Hb H disease: approximately 80-120 g/L (8-12 g/dL) in deletional disease and 60-110 g/L (6-11 g/dL) in non-deletional disease.[43][53]

Result

normal to low

mean corpuscular volume (MCV)

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The MCV correlates with phenotypic and genotypic worsening of the disease. Patients with alpha-thalassaemia silent carrier have a normal to slightly low MCV, whereas those with deletional alpha-thalassaemia trait usually have an MCV of <78 femtolitres.[48]​​ Those with deletional Hb H disease will have an MCV in the mid-60s.[43] Patients with Hb H/Constant Spring will have an MCV that is normal to mildly low, and those homozygous for Hb Constant Spring will often have a normal MCV.

Result

low

mean corpuscular haemoglobin (MCH)

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MCH will be normal to low (24-29 picograms/cell) in those with 1 alpha-thalassaemia silent carrier, and it will be low (<27 picograms/cell) in those with >1 alpha-globin deletion or in those with non-deletional mutations. Mean MCH is approximately 20 picograms/cell in Hb H disease.[43]

Result

low

red blood cell count

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Should be performed in all patients.

Result

increased; variable, corresponding to severity

peripheral smear

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Microcytosis, hypochromia, increased polychromasia, target cells, and anisopoikilocytosis; red cell fragments may be seen; basophilic stippling may be prominent in Hb H/Constant Spring and homozygous Hb Constant Spring.[6][48]​​

Result

abnormal shape and size of cells

reticulocyte percentage

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Elevated (5% to 10%), and may be further increased during acute infections or haemolytic crises.[48]​​

Result

increased

serum iron

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Result

normal or elevated

serum ferritin

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Iron chelation therapy can be considered when the ferritin is >1797.6 picomol/L (800 nanograms/mL).[50]

Serum ferritin levels may underestimate liver iron concentration.[52]

Result

normal or elevated

Investigations to consider

brilliant cresyl blue staining of red blood cells

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Numerous inclusion bodies are typically seen in Hb H disease.[16] Felt by some experts to be more reliable than Hb electrophoresis for diagnosis of Hb H disease. 

May be reduced or absent in the presence of heterozygous beta-thalassaemia such as Hb S, Hb C, Hb E, or beta-thalassaemia.​​[43][54]​​​

Result

demonstrates Hb H inclusions in peripheral blood red cells

Hb electrophoresis

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Hb H and Hb Bart can be detected as fast-moving haemoglobins. Will not pick up alpha-thalassaemia silent carrier or alpha-thalassaemia trait outside of the newborn period (Hb Barts), and will not differentiate deletional from non-deletional forms of Hb H (except for Hb Constant Spring). Patients with Hb H disease and concomitant heterozygous beta-haemoglobinopathies such as Hb S, Hb C, Hb E, or beta-thalassaemia have low or absent Hb H, which may confound the diagnosis of Hb H disease.

Result

presence of Hb H, Hb Bart, and concomitant haemoglobinopathies (Hb E, Hb S, Hb C, Hb D)

Hb fractionation by high-performance liquid chromatography (HPLC)

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Hb fractionation by HPLC provides fast separation and precise estimation of haemoglobin variants.

Will not pick up alpha-thalassaemia silent carrier or alpha-thalassaemia trait outside of the newborn period (Hb Barts), and will not differentiate deletional from non-deletional forms of Hb H (except for Hb Constant Spring). Patients with Hb H disease and concomitant heterozygous beta-haemoglobinopathies such as Hb S, Hb C, Hb E, or beta-thalassaemia have low or absent Hb H, which may confound the diagnosis of Hb H disease.

Result

presence of Hb H, Hb Bart, and concomitant haemoglobinopathies (Hb E, Hb S, Hb C, Hb D)

gap-polymerase chain reaction (gap-PCR)

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Seven of the most common alpha-thalassaemia deletions (-alpha(3.7), -alpha(4.2), --(FIL), --(THAI), --(MED), -(alpha)(20.5) , --(SEA)) can be diagnosed by gap-PCR.[48]​​​

Result

detects specific deletions

multiplex ligation-dependent probe amplification

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Used to detect deletions and duplications in the alpha-globin gene cluster causing alpha-thalassaemia.[48]

Result

detects deletion alleles

direct sequencing/reverse dot blot

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Used to detect non-deletional alpha-thalassaemia mutations.[48]​​[49]

Result

detect non-deletional alpha-thalassaemia mutations

MRI (hepatic or cardiac)

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Hepatic iron overload can be assessed by R2 or R2* MRI.[51]

MRI is the gold standard for measurement of hepatic iron concentration.​[16]​​

Cardiac iron loading is assessed by T2* cardiac MRI.[48] Cardiac iron loading is uncommon in non-transfused patients.

Result

hepatic or cardiac iron in patients with iron overload is demonstrated by difference in organ-to-muscle signal intensity

superconducting quantum interference devices (SQUID)

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Result
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If iron status is significantly elevated as evident by a serum ferritin >1797.6 picomol/L (>800 nanograms/mL), hepatic iron overload can be assessed by superconducting quantum interference devices (SQUID).[50][51]

Result

hepatic iron in iron overload

liver biopsy

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Result
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If iron status is significantly elevated as evident by a serum ferritin >1797.6 picomol/L (>800 nanograms/mL), hepatic iron overload can be assessed by liver biopsy.[50][51]

MRI is preferred to liver biopsy for assessment of iron overload where possible as it has better accuracy and fewer procedural risks. Liver biopsy may be used where assessment of histology is needed.[21]

Result

liver iron ≥5 mg/gram dry weight on liver biopsy indicates need for iron chelation therapy

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