When viewing this topic in a different language, you may notice some differences in the way the content is structured, but it still reflects the latest evidence-based guidance.

Aplastic anemia in adults

  • Overview
  • Theory
  • Diagnosis
  • Management
  • Follow up
  • Resources
Last reviewed: 10 Apr 2025
Last updated: 08 Apr 2022

Summary

Definition

History and exam

Other diagnostic factors

  • history of recurrent infection
  • fatigue
  • pallor
  • history of bleeding or easy bruising
  • tachycardia
  • dyspnea
  • persistent warts
  • hearing loss or deafness
  • short stature, pigmentation abnormalities, or urogenital abnormalities
  • nail malformations, reticular rash, oral leukoplakia, or epiphora
  • osteoporosis
  • premature hair loss/graying
  • hyperhidrosis
  • dysphagia
  • extensive dental caries or tooth loss
  • steatorrhea
  • skeletal dysplasia
  • monocytopenia
  • nontuberculous mycobacterial infections
  • pulmonary alveolar proteinosis
  • congenital lymphedema, Emberger syndrome
  • immunodeficiency (DCML [dendritic cell, monocyte, B cell, and NK cell deficiency])
Full details

Risk factors

  • drug or toxin exposure
  • paroxysmal nocturnal hemoglobinuria (PNH)
  • recent hepatitis
  • pregnancy
  • autoimmune disease
  • family history
Full details

Diagnostic tests

1st tests to order

  • complete blood count with differential
  • reticulocyte count
  • bone marrow biopsy and cytogenetic analyses
Full details

Tests to consider

  • serum B12 and folate levels
  • HIV testing
  • liver function tests (LFTs)
  • autoantibody screen
  • flow cytometry for glycosylphosphatidylinositol (GPI)-anchored proteins
  • chest x-ray
  • abdominal ultrasound
  • appropriate genetic tests
  • computed tomography scan
Full details

Treatment algorithm

ACUTE

non-severe acquired disease

severe/very severe acquired disease

inherited marrow failure syndrome

Contributors

Authors

Austin G. Kulasekararaj, MD, MRCP, FRCPath

Consultant Haematologist and Honorary Senior Clinical Lecturer

Department of Haematological Medicine

King's College Hospital NHS Foundation Trust and King’s College London

London

UK

Disclosures

AGK is on the Speakers' Panel/Advisory Board/has consulted for Novartis, Pfizer, Alexion, Roche, Ra Pharma, BioCryst, and Celgene/BMS. He is an author of a reference cited in this topic. AGK is the unpaid chairman of the writing committee of the British Society of Haematology guidelines on diagnosis and management of adult aplastic anaemia.

Acknowledgements

Dr Kulasekararaj would like to gratefully acknowledge the assistance of Professor Judith C. W. Marsh, Dr Ziyi Lim, Dr Philippe Armand, and Professor Joseph H. Antin, previous contributors to this topic. ZL, PA, and JHA declared that they had no competing interests. JCWM has received fees for consulting for Novartis Global, Amgen, Alexion, Jazz Pharmaceuticals, and GSK and has also consulted for Pfizer and Sanofi; received a fee for lectures given for Novartis and has also given lectures for Alexion and Sanofi; was awarded a research grant from Sanofi for King’s College London and has received a research grant from Novartis Global; and has received travel expenses from Novartis and Adienne to attend meetings.

Peer reviewers

Christoph Pechlaner, MD

Associate Professor of Medicine

Innsbruck Medical University

Innsbruck

Austria

Disclosures

CP declares that he has no competing interests.

References

Our in-house evidence and editorial teams collaborate with international expert contributors and peer reviewers to ensure that we provide access to the most clinically relevant information possible.

Key articles

Townsley DM, Dumitriu B, Young NS. Bone marrow failure and the telomeropathies. Blood. 2014 Oct 30;124(18):2775-83.Full text  Abstract

Bluteau O, Sebert M, Leblanc T, et al. A landscape of germ line mutations in a cohort of inherited bone marrow failure patients. Blood. 2018 Feb 15;131(7):717-32. Abstract

Kordasti S, Costantini B, Seidl T, et al. Deep phenotyping of Tregs identifies an immune signature for idiopathic aplastic anemia and predicts response to treatment. Blood. 2016 Sep 1;128(9):1193-205.Full text  Abstract

Killick SB, Bown N, Cavenagh J, et al; British Society for Standards in Haematology. Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol. 2016 Jan;172(2):187-207.Full text  Abstract

Bacigalupo A, Socié G, Hamladji RM, et al. Current outcome of HLA identical sibling versus unrelated donor transplants in severe aplastic anemia: an EBMT analysis. Haematologica. 2015 May;100(5):696-702.Full text  Abstract

Reference articles

A full list of sources referenced in this topic is available to users with access to all of BMJ Best Practice.

  • Differentials

    • Radiation injury
    • Chemotherapy
    • HIV infection
    More Differentials

  • Guidelines

    • Monitoring and management of iron overload in patients with haemoglobinopathies and rare anaemias
    • Guidelines for the diagnosis and management of acquired aplastic anaemia
    More Guidelines
  • padlock-lockedLog in or subscribe to access all of BMJ Best Practice

Use of this content is subject to our disclaimer