Aplastic Anemia Definition Pancytopenia with bone marrow hypocellularity

Aplastic Anemia Definition Pancytopenia with bone marrow hypocellularity cf. iatrogenic marrow aplasia Epidemiology Europe 2/1, 000; China 5 -7/1, 000 Gender; equal distribution Age; biphasic, peaks in older children, young adulthood/ elderly

Clinical Features • Onset – Abrupt or insidious, adolescent or young adulthood • Symptoms – Bleeding, weakness, shortness of breath – Confined to hematologic system cf. rare infection at onset cf. systemic complaints and weight loss-consider other etiology • Physical examination – Pallor, petechiae, ecchymoses – Café au lait spots, short stature→Fanconi’s anemia – Peculiar nails, tongue leukoplakia→dyskeratosis congenita cf. splenomegaly, lymphadenopathy→ other diagnosis

Diagnosis • Pancytopenia with empty, fatty marrow in adolescent or young adults, with typical symptoms – DDx: variant MDS (morphologic abnormality, cytogenetic abnormality), PNH (hemolysis, thrombosis) • Severe AA (any 2 of 3): 85% 6 month survival rate – Blood: 1. ANC < 500/ul (very severe if ANC < 200/ul) 2. Platelet < 20, 000/ul 3. Corrected reti <1% or absolute reti count <50, 000/ul – Marrow: 1. Severe hypocellularity (<25%) 2. Moderate hypocellularity (25 -50%) with hematopoietic cells representing <30% of residual cells

Treatment (I) • Bone marrow transplantation – Best tx for young pts with fully matched sibling donors – Transfusion minimized for prevention of alloimmunization (특히 가족으로부터의 수혈은 금기이다) – Long-term survival >80% if fully matched sibling donor – Unrelated donors: half success rate • Immunosuppression – ALG or ATG + cyclosporine: 70% hematologic recovery, frequent relapse, anaphylaxis, serum sickness, hypertension, nephrotoxicity, infection – High dose cyclophosphamide

Treatment (II) • Androgen – Occasional response, but not verified by controlled studies – Virilization • G-CSF/GM-CSF/IL-3 – Not recommended

Supportive Care • Infection control • G-CSF • Transfusion – Platelet > 10, 000/ul, hemoglobin > 7. 0 g/d. L • Menstruation suppression • Avoidance of aspirin/other NSAID’s

Serum ferritin 철중독증 : Serum Ferritin >1000 ng/m. L 참고치 : 남자 30 -400 ng/m. L 여자 13 -150 ng/m. L Ferritin ↓: 철결핍성 빈혈(IDA), 갑상선기능저하증(hypothyroidism), Vitamin C 결핍증 Ferritin ↑: hematochromatosis, hemosiderosis, porphyria