Aplastic Anemia

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Aplastic Anemia — is a rare but serious blood disorder in which the bone marrow fails to produce enough new blood cells, including red blood cells, white blood cells, and platelets. This leads to fatigue, higher risk of infection, and uncontrolled bleeding.

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🧬 What Happens

• The bone marrow (the soft tissue inside bones) becomes hypocellular — meaning it lacks the stem cells needed to make blood.

• As a result:

  • Red cells ↓ → anemia → fatigue, shortness of breath

  • White cells ↓ → leukopenia → infections

  • Platelets ↓ → thrombocytopenia → bleeding or bruising

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⚠️ Causes

• Autoimmune attack on bone marrow stem cells (most common)

• Exposure to toxins (benzene, pesticides, arsenic)

• Certain drugs (chemotherapy, antibiotics like chloramphenicol, NSAIDs)

• Radiation exposure

• Viral infections (Hepatitis, EBV, CMV, Parvovirus B19, HIV)

• Inherited conditions (e.g., Fanconi anemia, Shwachman–Diamond syndrome)

• Idiopathic (unknown cause in ~50% of cases)

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🩸 Symptoms

• Extreme fatigue, weakness

• Shortness of breath

• Pale skin

• Frequent infections (low white cells)

• Easy bruising or bleeding, nosebleeds, bleeding gums

• Prolonged healing of wounds

• Rapid or irregular heartbeat (from anemia)

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🧪 Diagnosis

• Complete Blood Count (CBC) – low counts of all three types of blood cells

• Reticulocyte count – low immature red cells

• Bone marrow biopsy – shows few blood-forming cells (hypocellular marrow)

• Blood tests for viruses or autoimmune antibodies

• Chromosomal tests to rule out inherited bone marrow failure syndromes

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💊 Treatment Options

1. Immunosuppressive Therapy (IST)

Used when bone marrow transplant isn’t possible.

• Antithymocyte globulin (ATG) – suppresses immune attack

• Cyclosporine (CSA) – prevents immune destruction

• Often combined for best results

• Sometimes add Eltrombopag (Promacta) to stimulate marrow

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2. Bone Marrow (Stem Cell) Transplant

• Preferred for younger patients (<40–50 yrs) with matched sibling donor

• Replaces damaged marrow with healthy donor stem cells

• High success rates (70–90% in good matches)

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3. Supportive Care

• Blood transfusions (RBCs and platelets as needed)

• Antibiotics and antivirals to fight infections

• Growth factors – Erythropoietin (EPO) or G-CSF to stimulate production

• Avoid toxins and unnecessary medications

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4. Experimental / Emerging Treatments

• Eltrombopag and other TPO receptor agonists to stimulate marrow

• Gene therapy (in inherited forms) – ongoing research

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🚨 Complications

• Severe infections

• Hemorrhage (due to very low platelets)

• Heart failure (from chronic anemia)

• Transformation to myelodysplastic syndrome (MDS) or leukemia

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💡 Prognosis & Monitoring

• With treatment, many recover partially or fully

• Lifelong follow-up with hematologist