{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,31]],"date-time":"2026-08-31T13:21:15Z","timestamp":1788182475736,"version":"build-2803163510"},"reference-count":55,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"5","license":[{"start":{"date-parts":[[2025,11,5]],"date-time":"2025-11-05T00:00:00Z","timestamp":1762300800000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0"}],"content-domain":{"domain":["lww.com","ovid.com"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2026,9]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Aplastic anaemia (AA) is a rare and life-threatening condition characterized by the failure of bone marrow to produce adequate blood cells. An autoimmune reaction of T lymphocytes against hematopoietic stem cells (HSCs) is the primary etiology. However, less commonly, a congenital deficiency or an acquired injury to these cells can also suppress their division and differentiation. Guidelines for practitioners to manage AA are frequently ambiguous. Close follow-up and monitoring are essential in managing patients with AA, necessitating physicians to comprehend the etiopathology of the disease. Clinical hematologists frequently provide specific treatments, but the physician must stay informed and cognisant of the latest management guidelines for the condition. In the management of AA, both specific and auxiliary modalities hold considerable significance. The databases that were used for the literature study were PubMed\/Medline, Embase, Scopus, and Web of Science. Search terms included \u201cAplastic anaemia.\u201d Articles from after 2006 that were relevant to diagnosis and management instructions were the only ones that were used. The things that were not allowed were scholarly posters, abstracts, and studies with nonhuman subjects. In recent years, there has been a substantial evolution in the concepts related to treating AA. This is attributable to improved outcomes in both familial and nonfamilial donor HSC transplants, alongside current advancements in immunosuppressive and gene therapy. This review article presents the current scientific insights by emphasizing the diagnostic protocols and developments in therapies, including the impacts of gene therapy, immunosuppressive agents, and HSC transplantation.<\/jats:p>","DOI":"10.4103\/aam.aam_196_25","type":"journal-article","created":{"date-parts":[[2025,11,5]],"date-time":"2025-11-05T11:00:09Z","timestamp":1762340409000},"page":"1025-1032","update-policy":"https:\/\/doi.org\/10.1097\/lww.0000000000001000","source":"Crossref","is-referenced-by-count":1,"title":["Aplastic Anemia in Focus: Insights into Diagnosis and Emerging Therapies"],"prefix":"10.4103","volume":"25","author":[{"given":"Anurag","family":"Singh","sequence":"first","affiliation":[{"name":"Department of Pathology, Sanjay Gandhi Postgraduate Institute of Medical Sciences, Lucknow, Uttar Pradesh, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pallavi","family":"Srivastava","sequence":"additional","affiliation":[{"name":"Department of Pathology, Sanjay Gandhi Postgraduate Institute of Medical Sciences, Lucknow, Uttar Pradesh, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alka","family":"Singh","sequence":"additional","affiliation":[{"name":"Department of Pathology, Sanjay Gandhi Postgraduate Institute of Medical Sciences, Lucknow, Uttar Pradesh, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ankita","family":"Singh","sequence":"additional","affiliation":[{"name":"Department of Pathology, Sanjay Gandhi Postgraduate Institute of Medical Sciences, Lucknow, Uttar Pradesh, India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rahul Kumar","family":"Gupta","sequence":"additional","affiliation":[{"name":"Department of Pathology, Sanjay Gandhi Postgraduate Institute of Medical Sciences, Lucknow, Uttar Pradesh, India"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"276","published-online":{"date-parts":[[2025,11,5]]},"reference":[{"key":"R1-20260831","doi-asserted-by":"crossref","first-page":"2660","DOI":"10.1182\/bloodadvances.2021004345","article-title":"Approach to the diagnosis of aplastic anemia","volume":"5","author":"DeZern","year":"2021","journal-title":"Blood Adv"},{"key":"R2-20260831","doi-asserted-by":"crossref","first-page":"809","DOI":"10.1080\/17474086.2019.1645003","article-title":"Management of aplastic anemia after failure of frontline immunosuppression","volume":"12","author":"Pierri","year":"2019","journal-title":"Expert Rev Hematol"},{"key":"R3-20260831","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1007\/s11864-017-0511-z","article-title":"Diagnosis and treatment of aplastic anemia","volume":"18","author":"Peslak","year":"2017","journal-title":"Curr Treat Options Oncol"},{"key":"R4-20260831","doi-asserted-by":"crossref","first-page":"559","DOI":"10.1080\/16078454.2019.1642548","article-title":"Pathogenesis of aplastic anemia","volume":"24","author":"Wang","year":"2019","journal-title":"Hematology"},{"key":"R5-20260831","doi-asserted-by":"crossref","first-page":"277","DOI":"10.1080\/17474086.2017.1302797","article-title":"Why is the incidence of aplastic anemia higher in Asia?","volume":"10","author":"Kojima","year":"2017","journal-title":"Expert Rev Hematol"},{"key":"R6-20260831","doi-asserted-by":"crossref","first-page":"236","DOI":"10.2174\/1381612825666190313113601","article-title":"Current concepts of the pathogenesis of aplastic anemia","volume":"25","author":"Liu","year":"2019","journal-title":"Curr Pharm Des"},{"key":"R7-20260831","doi-asserted-by":"crossref","first-page":"221","DOI":"10.1586\/ehm.11.11","article-title":"Clinical management of aplastic anemia","volume":"4","author":"Dezern","year":"2011","journal-title":"Expert Rev Hematol"},{"key":"R8-20260831","doi-asserted-by":"crossref","first-page":"1023","DOI":"10.1111\/jpc.14996","article-title":"Aplastic anaemia: Current concepts in diagnosis and management","volume":"56","author":"Furlong","year":"2020","journal-title":"J Paediatr Child Health"},{"key":"R9-20260831","doi-asserted-by":"crossref","first-page":"49","DOI":"10.5045\/br.2022.2022012","article-title":"Overview of inherited bone marrow failure syndromes","volume":"57","author":"Park","year":"2022","journal-title":"Blood Res"},{"key":"R10-20260831","doi-asserted-by":"crossref","first-page":"2257","DOI":"10.1182\/blood-2017-05-781799","article-title":"Inherited bone marrow failure syndromes: considerations pre- and posttransplant","volume":"130","author":"Alter","year":"2017","journal-title":"Blood"},{"key":"R11-20260831","doi-asserted-by":"crossref","first-page":"e157","DOI":"10.15190\/d.2022.16","article-title":"Aplastic anemia severity and IL-6 and IL-8 blood levels","volume":"10","author":"Bhargawa","year":"2022","journal-title":"Discoveries"},{"key":"R12-20260831","doi-asserted-by":"crossref","first-page":"446","DOI":"10.4103\/aam.aam_106_22","article-title":"Interleukin-6 and interleukin-8 levels in children with aplastic anemia and its correlation with disease severity and response to immunosuppressive therapy","volume":"22","author":"Singh","year":"2023","journal-title":"Ann Afr Med"},{"key":"R13-20260831","first-page":"139","article-title":"Immunoexpression of CD34, CD117, and p53 in hypocellular bone marrow disorders","volume":"14","author":"Sharma","year":"2021","journal-title":"J Lab Physicians"},{"key":"R14-20260831","doi-asserted-by":"crossref","first-page":"711","DOI":"10.1111\/ejh.13153","article-title":"Aplastic anemia: Etiology, molecular pathogenesis, and emerging concepts","volume":"101","author":"Shallis","year":"2018","journal-title":"Eur J Haematol"},{"key":"R15-20260831","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1053\/j.seminhematol.2021.12.002","article-title":"Aplastic anemia: Pathophysiology","volume":"59","author":"Giudice","year":"2022","journal-title":"Semin Hematol"},{"key":"R16-20260831","doi-asserted-by":"crossref","first-page":"581","DOI":"10.1016\/j.hoc.2018.03.001","article-title":"The pathophysiology of acquired aplastic anemia: Current concepts revisited","volume":"32","author":"Schoettler","year":"2018","journal-title":"Hematol Oncol Clin"},{"key":"R17-20260831","doi-asserted-by":"crossref","first-page":"1643","DOI":"10.1056\/NEJMra1413485","article-title":"Aplastic anemia","volume":"379","author":"Young","year":"2018","journal-title":"N Engl J Med"},{"key":"R18-20260831","doi-asserted-by":"crossref","first-page":"79","DOI":"10.1016\/j.critrevonc.2009.12.001","article-title":"Abnormal immunity and stem\/progenitor cells in acquired aplastic anemia","volume":"75","author":"Li","year":"2010","journal-title":"Crit Rev Oncol Hematol"},{"key":"R19-20260831","doi-asserted-by":"crossref","first-page":"185","DOI":"10.1097\/MPH.0000000000001737","article-title":"Changes in different cytokines (IL-2, TNF-\u03b1, and IFN-\u03b3) profile in acquired aplastic anemia patients: A study from Eastern India","volume":"42","author":"Dutta","year":"2020","journal-title":"J Pediatr Hematol Oncol"},{"key":"R20-20260831","doi-asserted-by":"crossref","first-page":"84","DOI":"10.1080\/10245332.2018.1512391","article-title":"Comparative study of bone marrow and blood plasma levels of IL-2 in aplastic anaemia and their relationship with disease severity","volume":"24","author":"De","year":"2019","journal-title":"Hematology"},{"key":"R21-20260831","doi-asserted-by":"crossref","first-page":"553","DOI":"10.18632\/aging.204500","article-title":"Alterations of mesenchymal stem cells on regulating Th17 and Treg differentiation in severe aplastic anemia","volume":"15","author":"Li","year":"2023","journal-title":"Aging (Albany NY)"},{"key":"R22-20260831","first-page":"1463","article-title":"Th17 cells and aplastic anemia","volume":"22","author":"Zhang","year":"2014","journal-title":"Zhongguo Shi Yan Xue Ye Xue Za Zhi"},{"key":"R23-20260831","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1111\/j.1365-2567.2011.03470.x","article-title":"CD8+CD28- and CD8+CD57+ T cells and their role in health and disease","volume":"134","author":"Strioga","year":"2011","journal-title":"Immunology"},{"key":"R24-20260831","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1016\/j.cca.2015.07.031","article-title":"Decreased circulating Th22 and Th17 cells in patients with aplastic anemia","volume":"450","author":"Lu","year":"2015","journal-title":"Clin Chim Acta"},{"key":"R25-20260831","doi-asserted-by":"crossref","first-page":"88","DOI":"10.1016\/j.humimm.2009.10.002","article-title":"Association of human leukocyte antigen class II alleles with response to immunosuppressive therapy in Korean aplastic anemia patients","volume":"71","author":"Song","year":"2010","journal-title":"Hum Immunol"},{"key":"R26-20260831","doi-asserted-by":"crossref","first-page":"595","DOI":"10.1016\/j.hoc.2018.03.002","article-title":"Somatic mutations in aplastic anemia","volume":"32","author":"Mufti","year":"2018","journal-title":"Hematol Oncol Clin"},{"key":"R27-20260831","doi-asserted-by":"crossref","first-page":"301","DOI":"10.1007\/s12015-023-10631-0","article-title":"The protective effect of chronic intermittent hypobaric hypoxia on preventing the destruction of CD34+ haematopoietic stem cells in aplastic anaemia by modulating the Th1\/Th2 balance","volume":"20","author":"Guo","year":"2024","journal-title":"Stem Cell Rev Rep"},{"key":"R28-20260831","doi-asserted-by":"crossref","first-page":"4083","DOI":"10.4238\/2014.May.30.3","article-title":"Fas\/FasL in the immune pathogenesis of severe aplastic anemia","volume":"13","author":"Liu","year":"2014","journal-title":"Genet Mol Res"},{"key":"R29-20260831","doi-asserted-by":"crossref","first-page":"2775","DOI":"10.1182\/blood-2014-05-526285","article-title":"Bone marrow failure and the telomeropathies","volume":"124","author":"Townsley","year":"2014","journal-title":"Blood"},{"key":"R30-20260831","doi-asserted-by":"crossref","first-page":"1849","DOI":"10.1016\/j.celrep.2018.01.057","article-title":"Shwachman-diamond syndrome protein SBDS maintains human telomeres by regulating telomerase recruitment","volume":"22","author":"Liu","year":"2018","journal-title":"Cell Rep"},{"key":"R31-20260831","doi-asserted-by":"crossref","first-page":"949435","DOI":"10.3389\/fonc.2022.949435","article-title":"Fanconi anemia and dyskeratosis congenita\/telomere biology disorders: Two inherited bone marrow failure syndromes with genomic instability","volume":"12","author":"Fiesco-Roa","year":"2022","journal-title":"Front Oncol"},{"key":"R32-20260831","doi-asserted-by":"crossref","first-page":"217","DOI":"10.1007\/s12185-024-03715-1","article-title":"Aplastic anemia: History and recent developments in diagnosis and treatment","volume":"119","author":"Onishi","year":"2024","journal-title":"Int J Hematol"},{"key":"R33-20260831","doi-asserted-by":"crossref","first-page":"11765","DOI":"10.3390\/ijms231911765","article-title":"Aplastic anemia as a roadmap for bone marrow failure: An overview and a clinical workflow","volume":"23","author":"Solimando","year":"2022","journal-title":"Int J Mol Sci"},{"key":"R34-20260831","doi-asserted-by":"crossref","first-page":"e31070","DOI":"10.1002\/pbc.31070","article-title":"Treatment of newly diagnosed severe aplastic anemia in children: Evidence-based recommendations","volume":"71","author":"Shimano","year":"2024","journal-title":"Pediatr Blood Cancer"},{"key":"R35-20260831","doi-asserted-by":"crossref","first-page":"1378432","DOI":"10.3389\/fimmu.2024.1378432","article-title":"The state of the art in the treatment of severe aplastic anemia: Immunotherapy and hematopoietic cell transplantation in children and adults","volume":"15","author":"Piekarska","year":"2024","journal-title":"Front Immunol"},{"key":"R36-20260831","first-page":"172","article-title":"Guidelines for the diagnosis and management of adult aplastic anaemia","volume":"2","author":"Killick","year":"2016","journal-title":"Br J Haematol"},{"key":"R37-20260831","doi-asserted-by":"crossref","first-page":"1784","DOI":"10.3324\/haematol.2014.109355","article-title":"First-line treatment for severe aplastic anemia in children: Bone marrow transplantation from a matched family donor versus immunosuppressive therapy","volume":"99","author":"Yoshida","year":"2014","journal-title":"Haematologica"},{"key":"R38-20260831","doi-asserted-by":"crossref","first-page":"565","DOI":"10.1111\/bjh.13297","article-title":"Outcome of aplastic anaemia in children. A study by the severe aplastic anaemia and paediatric disease working parties of the European group blood and bone marrow transplant","volume":"169","author":"Dufour","year":"2015","journal-title":"Br J Haematol"},{"key":"R39-20260831","doi-asserted-by":"crossref","first-page":"1428","DOI":"10.1182\/blood-2016-08-693481","article-title":"How I treat acquired aplastic anemia","volume":"129","author":"Bacigalupo","year":"2017","journal-title":"Blood"},{"key":"R40-20260831","doi-asserted-by":"crossref","first-page":"2351","DOI":"10.1182\/blood-2010-12-327536","article-title":"Alemtuzumab with fludarabine and cyclophosphamide reduces chronic graft-versus-host disease after allogeneic stem cell transplantation for acquired aplastic anemia","volume":"118","author":"Marsh","year":"2011","journal-title":"Blood"},{"key":"R41-20260831","doi-asserted-by":"crossref","first-page":"1291","DOI":"10.3324\/haematol.2017.164459","article-title":"Effect of antithymocyte globulin source on outcomes of bone marrow transplantation for severe aplastic anemia","volume":"102","author":"Kekre","year":"2017","journal-title":"Haematologica"},{"key":"R42-20260831","doi-asserted-by":"crossref","first-page":"585","DOI":"10.1111\/bjh.13614","article-title":"Similar outcome of upfront-unrelated and matched sibling stem cell transplantation in idiopathic paediatric aplastic anaemia. A study on behalf of the UK Paediatric BMT Working Party, Paediatric Diseases Working Party and Severe Aplastic Anaemia Working Party of EBMT","volume":"171","author":"Dufour","year":"2015","journal-title":"Br J Haematol"},{"key":"R43-20260831","doi-asserted-by":"crossref","first-page":"288","DOI":"10.1038\/bmt.2015.231","article-title":"Allogeneic hematopoietic cell transplantation for severe aplastic anemia: Similar long-term overall survival after transplantation with related donors compared to unrelated donors","volume":"51","author":"Mortensen","year":"2016","journal-title":"Bone Marrow Transplant"},{"key":"R44-20260831","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1038\/bmt.2016.223","article-title":"Immunosuppressive therapy versus alternative donor hematopoietic stem cell transplantation for children with severe aplastic anemia who lack an HLA-matched familial donor","volume":"52","author":"Choi","year":"2017","journal-title":"Bone Marrow Transplant"},{"key":"R45-20260831","doi-asserted-by":"crossref","first-page":"11","DOI":"10.3324\/haematol.10075","article-title":"Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade: A report from the European Group for Blood and Marrow Transplantation","volume":"92","author":"Locasciulli","year":"2007","journal-title":"Haematologica"},{"key":"R46-20260831","first-page":"83","article-title":"Nontransplant therapy for bone marrow failure","author":"Townsley","year":"2016","journal-title":"Hematol Am Soc Hematol Educ Program 2016"},{"key":"R47-20260831","doi-asserted-by":"crossref","first-page":"4434","DOI":"10.1182\/blood-2010-08-304071","article-title":"A randomized controlled study in patients with newly diagnosed severe aplastic anemia receiving antithymocyte globulin (ATG), cyclosporine, with or without G-CSF: A study of the SAA Working Party of the European Group for Blood and Marrow Transplantation","volume":"117","author":"Tichelli","year":"2011","journal-title":"Blood"},{"key":"R48-20260831","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1056\/NEJMoa2109965","article-title":"Eltrombopag added to immunosuppression in severe aplastic anemia","volume":"386","author":"Peffault de Latour","year":"2022","journal-title":"N Engl J Med"},{"key":"R49-20260831","doi-asserted-by":"crossref","first-page":"e382","DOI":"10.3324\/haematol.2017.171215","article-title":"Long-term risk of cancer development in adult patients with idiopathic aplastic anemia after treatment with anti-thymocyte globulin","volume":"102","author":"Van der Hem","year":"2017","journal-title":"Haematologica"},{"key":"R50-20260831","doi-asserted-by":"crossref","first-page":"277","DOI":"10.1016\/j.jcyt.2022.11.014","article-title":"CRISPR\/Cas9-mediated gene editing. A promising strategy in hematological disorders","volume":"25","author":"Ugalde","year":"2023","journal-title":"Cytotherapy"},{"key":"R51-20260831","doi-asserted-by":"crossref","first-page":"78","DOI":"10.1038\/nrd.2017.267","article-title":"JAK inhibition as a therapeutic strategy for immune and inflammatory diseases","volume":"17","author":"Schwartz","year":"2017","journal-title":"Nat Rev Drug Discov"},{"key":"R52-20260831","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1186\/s13287-023-03496-0","article-title":"Impaired immunosuppressive effect of bone marrow mesenchymal stem cell-derived exosomes on T cells in aplastic anemia","volume":"14","author":"Wang","year":"2023","journal-title":"Stem Cell Res Ther"},{"key":"R53-20260831","doi-asserted-by":"crossref","first-page":"1389","DOI":"10.1080\/10428194.2023.2215614","article-title":"Clonal evolution in aplastic anemia: Failed tumor surveillance or maladaptive recovery?","volume":"64","author":"Gurnari","year":"2023","journal-title":"Leuk Lymphoma"},{"key":"R54-20260831","doi-asserted-by":"crossref","first-page":"2328","DOI":"10.1038\/s41375-022-01636-8","article-title":"Predictors of clonal evolution and myeloid neoplasia following immunosuppressive therapy in severe aplastic anemia","volume":"36","author":"Groarke","year":"2022","journal-title":"Leukemia"},{"key":"R55-20260831","doi-asserted-by":"crossref","first-page":"784","DOI":"10.1111\/bjh.19236","article-title":"BSH Committee. Guidelines for the diagnosis and management of adult aplastic anaemia: A British Society for Haematology guideline","volume":"204","author":"Kulasekararaj","year":"2024","journal-title":"Br J Haematol"}],"container-title":["Annals of African Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.ovid.com\/10.4103\/aam.aam_196_25","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,8,31]],"date-time":"2026-08-31T13:00:16Z","timestamp":1788181216000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.ovid.com\/10.4103\/aam.aam_196_25"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,11,5]]},"references-count":55,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2026]]}},"URL":"https:\/\/doi.org\/10.4103\/aam.aam_196_25","relation":{},"ISSN":["1596-3519","0975-5764"],"issn-type":[{"value":"1596-3519","type":"print"},{"value":"0975-5764","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,11,5]]},"assertion":[{"value":"2025-05-23","name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-07-25","name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}}]}}