Increase in Mean MCH

  • MCH/Pg

Fetal Haemoglobin

  • Fetal Hb F g/dl

Serum Ferritin

  • Ferritin nl/ml

Beta thalassemias are a group of genetic blood disorders in which the haemoglobin molecule is incomplete. Depending on the mutation, patients may range from symptom-less to severely anaemic.

Affected individuals may need lifelong blood transfusions. In severe cases this leads to iron overload, and drug therapy is needed to reduce this.

It is estimated that 1 in 100,000 people globally suffer from a form of beta thalassemia.

TriStem’s clinical study

Twenty one patients with beta thalassemia were treated with a single infusion of stem cells created from their own white blood cells using TriStem’s technology. Over six months after treatment, the average number of transfusions was reduced and blood iron levels reduced.

Beta thalassemia is a good analogue for Sickle Cell Disease – both are genetic diseases whereby the patient’s red blood cells are deformed or die prematurely, causing low levels of Mean Corpuscular Haemoglobin (MCH)

The results were published in a peer-reviewed paper, which can be accessed here.

Patients were followed beyond the six months reported in the publication and it was discovered that, with time, their blood measurements trended back to the pre-treatment levels. This was considered to be because the disease is genetically determined, and this result was the rationale for the proposed Sickle Cell study being conducted using allogeneic cells.