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Hematology›Condition·Updated Jun 27, 2026·v1

Aplastic Anemia

Aplastic anemia is a rare but life-threatening bone marrow failure syndrome characterized by pancytopenia and hypocellular marrow. Prompt diagnosis with bone marrow biopsy and severity classification using Camitta criteria is essential. Treatment depends on age and donor availability: HSCT for young patients with matched sibling donor, IST (horse ATG + cyclosporine) for others. Supportive care with restrictive transfusion thresholds and infection prophylaxis is critical. Long-term monitoring for clonal evolution to MDS/AML is required.

High Evidence53 references·1,566 words·7 min read·v1
aplastic anemiabone marrow failurepancytopeniaimmunosuppressive therapyhematopoietic stem cell transplantationCamitta criteriaPNH

Quick Reference

RxDrug of choiceHorse antithymocyte globulin (hATG) 40 mg/kg/day IV for 4 days + cyclosporine 5-10 mg/kg/day PO (trough 200-400 ng/mL) for immunosuppressive therapy; allogeneic HSCT for eligible patients.
AltAlternativesRabbit ATG (rATG) 3.5 mg/kg/day IV for 5 days; eltrombopag 50-150 mg/day PO; romiplostim; alemtuzumab; high-dose cyclophosphamide.
⊘AvoidLive vaccines; NSAIDs (bleeding risk); corticosteroids as monotherapy; G-CSF in patients with clonal cytogenetics or MDS; transfusions from family members.
DxTest of choiceBone marrow aspiration and trephine biopsy with flow cytometry, cytogenetics, and molecular testing for PNH clone and inherited syndromes.
ScKey scoreCamitta criteria for severity: severe AA requires marrow cellularity <25% and at least two of ANC <500/μL, platelets <20,000/μL, reticulocytes <60,000/μL; very severe AA has ANC <200/μL.
→When to referAll suspected or confirmed aplastic anemia should be referred urgently to a hematologist with expertise in bone marrow failure for evaluation and treatment planning.
Aplastic anemia is a medical emergency requiring prompt diagnosis via bone marrow biopsy, severity classification, and initiation of IST or HSCT. Supportive care with restrictive transfusion thresholds and infection prophylaxis is critical. Long-term monitoring for clonal evolution is essential.
Aplastic anemia (AA) is a rare, life-threatening bone marrow failure syndrome defined by pancytopenia and a hypocellular marrow with no infiltration or fibrosis [2]. It is a diagnosis of exclusion, requiring workup for inherited syndromes and hypocellular MDS. Severe AA carries >70% 1-year mortality with supportive care alone, but outcomes improve with immunosuppressive therapy or hematopoietic stem cell transplantation.

Overview and Recommendations

Background

  • •Aplastic anemia (AA) is a rare, life-threatening bone marrow failure syndrome defined by pancytopenia and a hypocellular marrow with no infiltration or fibrosis. The incidence is 2-3 per million per year, with a bimodal age distribution (peaks at 15-25 years and >60 years). Untreated severe AA carries a 1-year mortality >70%, but outcomes improve dramatically with immunosuppressive therapy (IST) or hematopoietic stem cell transplantation (HSCT).
  • •The majority of acquired AA is immune-mediated: activated cytotoxic T cells destroy hematopoietic stem cells, often triggered by an inciting event (drugs, viruses, toxins) in a genetically susceptible host. This immune attack is the rationale for IST with antithymocyte globulin (ATG) and cyclosporine.
  • •Severity is classified by the : severe AA (SAA) requires bone marrow cellularity <25% and at least two of ANC <500/μL, platelets <20,000/μL, reticulocytes <60,000/μL; very severe AA (VSAA) has ANC <200/μL. Non-severe AA does not meet these thresholds.
  • •Inherited bone marrow failure syndromes (e.g., , ) must be excluded, especially in children and young adults. Paroxysmal nocturnal hemoglobinuria (PNH) clones are present in 40-70% of acquired AA, indicating immune pressure and often predicting response to IST.
  • •AA can be associated with thymoma, hepatitis, or drugs (e.g., chloramphenicol, NSAIDs). The AA-PNH overlap syndrome carries additional risks of thrombosis and hemolysis.

Evaluation

  • •Suspect AA in any patient presenting with unexplained pancytopenia: fatigue, pallor, dyspnea (anemia); fever, recurrent infections (neutropenia); petechiae, ecchymoses, mucosal bleeding (thrombocytopenia). Ask about drug/toxin exposure, viral infections, family history of bone marrow failure, and symptoms of PNH (dark urine, abdominal pain, thrombosis).
  • •Examine for pallor, petechiae, ecchymoses, gingival bleeding, and signs of infection (fever, oral ulcers, perianal tenderness). Look for café-au-lait spots or short stature (suggesting Fanconi anemia), nail dystrophy or oral leukoplakia (dyskeratosis congenita), and splenomegaly (extramedullary hematopoiesis).
  • •Order a complete blood count with differential and reticulocyte count. Pancytopenia with low reticulocyte count is typical. Peripheral smear shows no blasts, no dysplastic cells, and no abnormal cells.
  • •Perform bone marrow aspiration and trephine biopsy (≥1.5-2 cm core) from the posterior iliac crest. The marrow is markedly hypocellular (<25% cellularity in SAA) with fatty replacement. Residual cells are lymphocytes, plasma cells, and stromal cells; megakaryocytes are absent or rare.
  • •Send bone marrow for flow cytometry to assess CD34+ progenitor cells (markedly reduced) and to detect a PNH clone (GPI-anchored protein deficiency). Also send for cytogenetics (typically normal in AA; abnormal suggests MDS) and molecular testing for somatic mutations (e.g., BCOR, PIGA).
  • •Exclude inherited syndromes: perform chromosome breakage test (diepoxybutane or mitomycin C) for Fanconi anemia, telomere length measurement for dyskeratosis congenita, and next-generation sequencing for germline mutations in patients <40 years or with suggestive features.
  • •Apply the to classify severity: SAA requires marrow cellularity <25% and at least two of ANC <500/μL, platelets <20,000/μL, reticulocytes <60,000/μL. VSAA has ANC <200/μL.
  • •Consider imaging: MRI of the spine shows diffuse fatty replacement (hyperintense T1 signal) and can quantify marrow cellularity noninvasively. FLT-PET shows reduced proliferation. Use imaging if biopsy is contraindicated or to guide biopsy site.
  • •Rule out hypocellular myelodysplastic syndrome (MDS): look for dysplasia in ≥10% of cells, blasts ≥5%, abnormal cytogenetics, or CD34+ clusters on immunohistochemistry. AA has no dysplasia and blasts <5%.
  • •Also consider other causes of pancytopenia: vitamin B12/folate deficiency, HIV, parvovirus B19, systemic lupus erythematosus, and drug-induced agranulocytosis. Bone marrow biopsy is essential to confirm AA.

Management

  • •Initiate supportive care immediately: transfuse platelets to maintain >10 × 10⁹/L (restrictive strategy) or >20 × 10⁹/L if febrile or bleeding. Transfuse red blood cells for hemoglobin ≤7 g/dL or symptomatic anemia. Use irradiated, leukoreduced products to prevent alloimmunization and transfusion-associated GVHD.
  • •For severe AA (SAA/VSAA) in patients <40-50 years with a matched sibling donor, proceed to allogeneic hematopoietic stem cell transplantation (HSCT) as first-line therapy. Preferred graft source is bone marrow. Conditioning regimen: fludarabine, ATG, busulfan, thiotepa (FABT) with post-transplant cyclophosphamide for haploidentical donors.
  • •For patients without a matched sibling donor or age >40-50 years, start immunosuppressive therapy (IST) with horse antithymocyte globulin (hATG) 40 mg/kg/day IV for 4 days plus cyclosporine 5-10 mg/kg/day PO (target trough 200-400 ng/mL). Premedicate with corticosteroids and antihistamines to reduce infusion reactions.
  • •Add eltrombopag (TPO-RA) to IST for refractory or relapsed AA: start at 50 mg/day PO, titrate to 150 mg/day based on platelet response. Monitor for liver function abnormalities and clonal evolution.
  • •For patients who fail hATG, consider rabbit ATG (rATG) 3.5 mg/kg/day IV for 5 days, though response rates are lower. Alternatively, use alemtuzumab or high-dose cyclophosphamide in clinical trials.
  • •Administer granulocyte colony-stimulating factor (G-CSF) 5 μg/kg/day SC for severe neutropenia (ANC <200/μL) to reduce infection risk. Discontinue when ANC >500/μL. Avoid G-CSF in patients with known MDS or clonal cytogenetics due to theoretical risk of progression.
  • •Monitor for treatment response: hematologic recovery typically begins at 3-6 months after IST. Complete response is defined as normalization of blood counts (ANC >1500, platelets >150K, Hb >11 g/dL). Partial response is transfusion independence with counts not meeting normal.
  • •Manage infections aggressively: febrile neutropenia requires empiric broad-spectrum antibiotics (e.g., cefepime or piperacillin-tazobactam). Add antifungal coverage (voriconazole or liposomal amphotericin B) for persistent fever. Use antiviral prophylaxis (acyclovir) and Pneumocystis jirovecii prophylaxis (trimethoprim-sulfamethoxazole) during IST.
  • •Avoid live vaccines (MMR, varicella, yellow fever) during immunosuppression. Administer inactivated vaccines (influenza, pneumococcal, COVID-19) as indicated.
  • •Monitor for complications of IST: cyclosporine can cause nephrotoxicity, hypertension, neurotoxicity (tremor, PRES). Monitor serum creatinine, blood pressure, and cyclosporine trough levels. ATG can cause serum sickness (fever, rash, arthralgias) 7-14 days after infusion; treat with corticosteroids.
  • •For patients with PNH clone, consider eculizumab or ravulizumab if hemolysis or thrombosis occurs. Anticoagulation for thrombosis per standard guidelines.
  • •Refer to a hematologist with expertise in bone marrow failure for all patients. Early referral for HSCT evaluation is critical.
  • •Discharge criteria: stable blood counts with transfusion independence or predictable transfusion needs, no active bleeding, infection resolved, and outpatient follow-up arranged. Educate patient on signs of bleeding, infection, and need for urgent medical attention.
  • •Long-term monitoring: serial CBCs every 1-3 months. Bone marrow biopsy at 6-12 months to assess response and exclude clonal evolution. Monitor for late complications: MDS/AML (risk ~5-10% at 10 years), iron overload from transfusions (consider chelation if ferritin >1000 ng/mL), and secondary malignancies.
  • •What NOT to do: Do not use corticosteroids as monotherapy (ineffective). Do not use G-CSF alone without IST. Do not transfuse family members (risk of alloimmunization for future HSCT). Do not delay HSCT in eligible patients.

Board Review — High Yield

  • •Camitta criteria, Defines severe AA as bone marrow cellularity <25% and at least two of ANC <500/μL, platelets <20,000/μL, reticulocytes <60,000/μL; very severe AA has ANC <200/μL.
  • •PNH clone, Present in 40-70% of acquired AA; detected by flow cytometry for GPI-anchored proteins; indicates immune-mediated disease and predicts response to IST.
  • •Horse ATG, First-line IST for severe AA; given 40 mg/kg/day IV for 4 days with cyclosporine; response rates ~60-70%.
  • •HSCT, Curative for young patients (<40-50 years) with matched sibling donor; bone marrow preferred graft source; conditioning with fludarabine, ATG, busulfan, thiotepa.
  • •Eltrombopag, TPO-RA used in refractory/relapsed AA; start 50 mg/day, titrate to 150 mg/day; monitor LFTs and clonal evolution.
  • •Hypocellular MDS, Main differential; distinguished by dysplasia in ≥10% of cells, blasts ≥5%, abnormal cytogenetics, or CD34+ clusters on IHC.
  • •Fanconi anemia, Inherited cause of AA; test with diepoxybutane-induced chromosome breakage; affects conditioning regimen and surveillance.
  • •Restrictive platelet transfusion, Threshold ≤10 × 10⁹/L in stable patients; reduces alloimmunization and transfusion reactions without increasing bleeding risk.

Deep Dive — Evidence Details

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