# Aplastic Anaemia in Children > Aplastic anaemia in children for NEET-PG Paediatrics, covering severity criteria, marrow findings, transplant versus immunosuppression and transfusion support. - Canonical URL: https://prepelephant.com/topics/neet-pg/paediatrics/aplastic-anaemia-in-children - Exam / course: NEET-PG · Subject: Paediatrics - Publisher: PrepElephant (https://prepelephant.com) — Prepared and reviewed by the PrepElephant Academic Review Team - First published: 2026-10-08 - Last updated: 2026-10-08 - Reference basis: Nathan and Oski's Hematology and Oncology of Infancy and Childhood; Nelson Textbook of Pediatrics - How to cite: "Aplastic Anaemia in Children", PrepElephant, https://prepelephant.com/topics/neet-pg/paediatrics/aplastic-anaemia-in-children ## Direct answer Aplastic anaemia is bone marrow failure with pancytopenia and a hypocellular marrow, usually immune-mediated in children and not caused by infiltration or fibrosis. Severe disease needs at least two of three counts critically low, namely neutrophils below 0.5 × 10^9/L, platelets below 20 × 10^9/L and reticulocytes below 20 × 10^9/L, with marrow cellularity below 25 per cent. A matched sibling donor makes transplant first-line, while antithymocyte globulin with cyclosporine is the alternative. ## What you must remember - **Pancytopenia:** reduced red cells, neutrophils and platelets with a hypocellular marrow on aspirate and trephine biopsy. - **Severity criteria:** severe disease needs two of three cytopenias with marrow cellularity below 25 per cent; very severe disease has neutrophils below 0.2 × 10^9/L. - **Pathogenesis:** T-cell mediated destruction of haematopoietic stem cells, with interferon-gamma and tumour necrosis factor implicated. - **Causes:** idiopathic in most children, but drugs such as chloramphenicol, viral infections including hepatitis and Epstein-Barr virus, and toxins such as benzene are recognised triggers. - **Exclude mimics:** inherited marrow failure, paroxysmal nocturnal haemoglobinuria and infiltration must be considered before the diagnosis is made. - **Supportive care:** irradiated, leucodepleted blood products and antimicrobial prophylaxis for neutropenia, with transfusion kept to need to limit alloimmunisation. ## A pathway from confirmation to definitive therapy Treatment choice depends on donor availability and severity, and a matched sibling transplant offers the best prospects in children. 1. **Confirm and stage:** repeat counts, marrow cellularity and cytogenetics, and check for inherited marrow failure before committing to acquired disease. 2. **Support while assessing:** transfuse to thresholds, use irradiated and leucodepleted products, and give antimicrobial prophylaxis for profound neutropenia. 3. **Search for a donor:** HLA-type siblings and start an unrelated donor search, since donor availability decides the first choice of therapy. 4. **Transplant first line:** matched sibling donor transplantation is preferred in children, with generally favourable outcomes. 5. **Immunosuppression alternative:** antithymocyte globulin with cyclosporine, with a thrombopoietin receptor agonist added in selected cases, for children without a suitable donor. 6. **Monitor response and relapse:** track counts and clonal evolution, since relapse and late myelodysplasia can follow immunosuppression. Outcome depends on age, severity, donor type and the response to immunosuppression, so each decision is documented carefully. Supportive care continues throughout, because infection and bleeding remain the main early threats. ## Where the exam tries to catch you Aplastic anaemia questions often test whether the student has excluded a mimic before choosing treatment. - **Trap:** starting immunosuppression before excluding Fanconi anaemia — **Correct:** breakage testing is needed, because transplant conditioning and treatment choices differ. - **Trap:** equating a low reticulocyte count with iron deficiency — **Correct:** hypoproliferation indicates marrow failure, not iron lack. - **Trap:** insisting on transplant without a donor — **Correct:** without a suitable donor, antithymocyte globulin with cyclosporine is the standard alternative. - **Trap:** ignoring transfusion-related alloimmunisation — **Correct:** HLA sensitisation from repeated transfusions makes later donor matching harder. ## Frequently asked questions ### What defines severe aplastic anaemia in children? Severe aplastic anaemia is defined by marrow cellularity below 25 per cent with at least two of three counts critically low: neutrophils below 0.5 × 10^9/L, platelets below 20 × 10^9/L and reticulocytes below 20 × 10^9/L. Very severe disease has neutrophils below 0.2 × 10^9/L. ### Why is transplant preferred for paediatric aplastic anaemia? Haematopoietic stem cell transplantation from a matched sibling donor is preferred in children because it offers a potential cure with generally favourable outcomes. Immunosuppression is the alternative when no suitable donor is available. ### Which drugs are associated with aplastic anaemia? Chloramphenicol is the classic drug associated with aplastic anaemia, and other medicines and toxins such as benzene are also implicated. Most paediatric cases remain idiopathic, so a thorough exposure history is essential. ### What is the standard immunosuppressive regimen for aplastic anaemia? Antithymocyte globulin combined with cyclosporine is the standard immunosuppressive regimen for children without a matched donor, with a thrombopoietin receptor agonist added in selected cases. Response and relapse are tracked with blood counts and marrow monitoring. ### Why are blood products irradiated and leucodepleted in aplastic anaemia? Irradiated products reduce the risk of transfusion-associated graft versus host disease, while leucodepletion reduces HLA alloimmunisation that can complicate later donor matching. These measures are routine in children with marrow failure.