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What are the Current ADA-deficiency Treatments?

There are currently three treatments to help patients diagnosed with adenosine deaminase deficiency (ADA). These are:

  • Bone Marrow Transplantation
  • Blood Erythrocyte Transfusion
  • PEG-ADA Enzyme Replacement

Bone Marrow Transplantation

Bone marrow transplantation is the treatment of choice as the cure rate is high (1). The donors are usually HLA-matched siblings. In total, the percentage of patients with suitable donors is approximately 20-30%. As a result, this treatment is unable to treat all patients with this disease. The alternative is PEG-ADA treatment.

Blood Erythrocyte Transfusion

Blood erythrocyte transfusion is an enzyme replacement therapy involving repeated transfusions with normal, healthy, frozen, irradiated red blood cells into patients lacking proper ADA function (2). However, antibodies develop against the foreign material, and thus the use of RBC replacement is limited.

PEG-ADA Enzyme Replacement

PEG-ADA is a polyethylene glycol-modified bovine adenosine deaminase enzyme replacement. This treatment has an immense advantage over other current treatments as this treatment has excellent clinical results with few, if any, clinical difficulties.

References

  1. Hershfield, M.S., Chaffee, S., Sorensen, R.U. (1993). Enzyme Replacement Therapy with Polyetheylene Glycol-Adenosine Deaminase in Adenosine Deaminase Deficiency: Overview and Case Reports of Three Patients, Including Two Now Receiving Gene Therapy. Pediatr. Res. 33: S42-S47.

  2. Bory, C., Boulieu, R., Souillet, G., Chantin, C., Rolland, MO., Mathieu, M., Hershfield, M. (1990). Comparison of Red Cell Transfusion and Polyethylene Glycol-Modified Adenosine Deaminase Therapy in an Adenosine Deaminase-Deficient Child: Measurement of Erythrocyte Deoxyadenosine Triphosphate as a Useful Tool. Pediatr. Res. 28: 127-130.
 
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