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Key Features

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Essentials of Diagnosis
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  • Weakness and pallor

  • Petechiae, purpura, and bleeding

  • Frequent or severe infections

  • Pancytopenia with hypocellular bone marrow

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General Considerations
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  • Approximately 50% of cases in childhood are idiopathic

  • Other cases are secondary to idiosyncratic reactions to drugs such as phenylbutazone, sulfonamides, nonsteroidal anti-inflammatory drugs (NSAIDs), and anticonvulsants

  • Toxic causes include exposure to benzene, insecticides, and heavy metals

  • Infectious causes include viral hepatitis, infectious mononucleosis (Epstein-Barr virus [EBV]), and HIV

  • Has been associated with human parvovirus B19 infection in immunocompromised children

  • Immune mechanisms of marrow suppression are suspected in most cases

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Clinical Findings

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  • Weakness, fatigue, and pallor result from anemia

  • Petechiae, purpura, and bleeding occur due to thrombocytopenia

  • Fevers due to generalized or localized infections are associated with neutropenia

  • Hepatosplenomegaly and significant lymphadenopathy are unusual

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Diagnosis

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Laboratory Findings
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  • Usually normocytic, with a low reticulocyte count

  • Low WBC count, with a marked neutropenia

  • Platelet count is typically below 50,000/μL and is frequently below 20,000/μL

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Diagnostic Procedures
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  • Bone marrow biopsy shows a marked decrease in cellularity, typically < 20% of normal in severe cases

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Treatment

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  • Comprehensive supportive care is essential

  • Febrile illnesses require prompt evaluation and usually parenteral antibiotics

  • Red blood cell transfusions alleviate symptoms of anemia

  • Platelet transfusions

    • May be lifesaving

    • However, should be used sparingly because platelet alloantibodies eventually develop in many patients who then become refractory to platelet transfusions

  • Immunomodulation, usually with antithymocyte globulin and cyclosporine or tacrolimus, is associated with a high response rate and improved overall survival. However, incomplete response, relapse, and progression to myelodysplasia/leukemia may occur

  • Hematopoietic stem cell transplantation

    • Treatment of choice for severe aplastic anemia when an HLA-identical sibling donor is available

    • Because the likelihood of success with transplant is influenced adversely by receipt of transfusions, HLA typing of family members should be undertaken at the time of diagnosis

  • Patients who lack HLA-identical siblings may be able to find matched donors through cord blood banks or the National Marrow Donor Program

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Outcome

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Complications
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  • Infection and hemorrhage are the leading causes of death

  • Other complications are those associated with therapy

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Prognosis
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  • Children receiving early bone marrow transplant from an HLA-identical sibling have a long-term survival rate > 80%

  • Sustained, complete remissions may be seen in 65–80% of patients receiving immunosuppressive therapy

  • However, both therapies are associated with an increased risk of myelodysplastic syndromes, acute leukemia, and other malignancies in long-term survivors

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References

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Korthof  ET, Kekassy  AN, Hussein  AA: Management of acquired aplastic anemia in children. Bone Marrow Transplant 2013;48:191–195
[PubMed: 23292240]
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Samarasinghe  S  et al: Excellent outcome of matched unrelated donor transplantation in paediatric aplastic anaemia following failure with immunosuppressive therapy: ...

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