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Cord Blood Successfully Restores Immune System in Severe Aplastic Anaemia


What is aplastic anaemia?

Aplastic anaemia is a serious condition caused by the failure of the bone marrow to produce enough new blood cells. Aplastic anaemia causes:

  • Less oxygen sent to organs, tissues and cells (from too few red blood cells)
  • Increased risk of infection (from too few white blood cells)
  • Increased risk of bleeding problems (from too few platelets)

What causes aplastic anaemia?

Aplastic anaemia has many causes. It can be acquired or passed down from parent to child. Causes include:

  • Infections such as hepatitis, liver infection and many viral illnesses
  • Cancers such as leukaemia, myeloma and lymphoma
  • Autoimmune diseases such as lupus and rheumatoid arthritis
  • Medicines including some antibiotics
  • Toxins including heavy metals, pesticides, and benzene
  • Radiation therapy and chemotherapy

What are the symptoms of aplastic anaemia?

Low levels of red blood cells can cause pale skin, chest pain, irregular heartbeat and an enlarged heart. Low levels of white blood cells can cause fevers, mouth sores and infections and low levels of platelets can cause easy bruising, nosebleeds, bleeding gums and heavy periods.

Without treatment, aplastic anaemia can cause problems with growth and development, cancers, heart failure, uncontrolled bleeding and severe infections.

Treating aplastic anaemia

It is now possible to ‘reboot’ a patient’s immune system and restore healthy bone marrow function with a stem cell transplant. This process has proven successful in cancers affecting the blood and immune systems, as well as in an ever-expanding range of conditions including MS, Sickle Cell disease and

with autologous umbilical cord blood (auto-UCB) transplant in a 3-year-old boy with severe aplastic anemia.

Case Report

The patient received auto-UCB containing 0.3 × 105 CD34+ cells per kilogram of body weight. Recovery of leukocyte count above 1000/μL was reached on post-transplant day +39, and recovery of granulocytes above 500/μL was reached on day +40. The final regular transfusions of packed red blood cells and platelet concentrate were performed on day +55. The complications that occurred in the post-transplant period were nausea, diarrhoea, septic fever, and hepatic abscess formation. Post-transplant immunosuppression with cyclosporine A was discontinued 17.5 months after auto-UCB, and the patient remained in complete remission with normal blood counts and bone marrow morphology.

Summary

Auto-UCB transplantation without chemotherapy conditioning can be considered a therapeutic option for children with stored cord blood cells.


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