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Very Severe Aplastic Anemia

MedGen UID:
1732130
•Concept ID:
C5417733
•
Disease or Syndrome

Definition

Aplastic anemia that is characterized by bone marrow (BM) cellularity less than 25% (or less than 50% if BM is comprised of less than 30% hematopoietic cells), and two or more of the following: absolute neutrophil count of less than 200 per microliter; platelet count of less than 20,000 per microliter; or an absolute reticulocyte count of less than 20,000 per microliter. [from NCI]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVVery Severe Aplastic Anemia

Professional guidelines

PubMed

Plain language summary of RACE study results: addition of eltrombopag to standard treatment of severe aplastic anemia.
de Latour RP, Kulasekararaj A, Iacobelli S, Griffin M, Halkes CJ, Dufour C, Risitano AM
Immunotherapy 2024 Feb;16(3):135-142. Epub 2023 Dec 13 doi: 10.2217/imt-2023-0200. PMID: 38088156
Aplastic anemia: clinicohaematological features, treatment and outcome analysis.
Wali R, Fadoo Z, Adil S, Naqvi MA
J Coll Physicians Surg Pak 2011 Apr;21(4):219-22. PMID: 21453618
Treatment of severe aplastic anemia with antilymphocyte globulin and androgens: a report on 33 patients.
Facon T, Walter MP, Fenaux P, Morel P, Dupriez B, Gardin C, Jouet JP, Bauters F
Ann Hematol 1991 Aug;63(2):89-93. doi: 10.1007/BF01707279. PMID: 1912037

Recent clinical studies

Etiology

Development and validation of early death risk score model for emergency status prediction in very severe aplastic anemia.
Liu X, Yang W, Zhang L, Jing L, Ye L, Zhou K, Li Y, Li J, Fan H, Yang Y, Xiong Y, Zhao X, Zhang F
Front Immunol 2023;14:1175048. Epub 2023 Apr 20 doi: 10.3389/fimmu.2023.1175048. PMID: 37153568Free PMC Article
Cytokines help suggest aplastic anemia with pulmonary bacterial or co-fungal infection.
Zhang J, Yang Z, Hu P, Guan X, Zhang C, Zou Y, Li H, Yang T, Cao Y, Zhao R, Li Z
Sci Rep 2022 Nov 1;12(1):18373. doi: 10.1038/s41598-022-22503-7. PMID: 36319826Free PMC Article
Nutritional assessment of patients with aplastic anemia: comparison of four nutritional screening tools.
Tan X, Tian Y, Li Z, Cui S, Wang Z, Zhou Y, Liu Z, Kong F, Wang Y, Xu R
Nutr Hosp 2022 Dec 20;39(6):1289-1297. doi: 10.20960/nh.03957. PMID: 36173198
A New Immunosuppressive Therapy for Very Severe Aplastic Anemia in Children with Autoantibodies.
Wang ZJ, Chen HB, Zhou F, Yu H, Wu XY, Shen YQ, Qiu YN, Jin RM
Curr Med Sci 2022 Apr;42(2):379-386. Epub 2022 Mar 8 doi: 10.1007/s11596-022-2519-2. PMID: 35258748
Adult aplastic anemia in Thailand: incidence and treatment outcome from a prospective nationwide population-based study.
Norasetthada L, Wongkhantee S, Chaipokam J, Charoenprasert K, Chuncharunee S, Rojnuckarin P, Sirijerachai C, Wanachiwanawin W, Issaragrisil S; Thai Aplastic Anemia Study Group
Ann Hematol 2021 Oct;100(10):2443-2452. Epub 2021 Jul 16 doi: 10.1007/s00277-021-04566-0. PMID: 34269837Free PMC Article

Diagnosis

New-onset aplastic anemia after SARS-CoV-2 vaccination.
Chatzikalil E, Kattamis A, Diamantopoulos P, Solomou EE
Int J Hematol 2023 Dec;118(6):667-681. Epub 2023 Sep 28 doi: 10.1007/s12185-023-03666-z. PMID: 37768509
Development and validation of early death risk score model for emergency status prediction in very severe aplastic anemia.
Liu X, Yang W, Zhang L, Jing L, Ye L, Zhou K, Li Y, Li J, Fan H, Yang Y, Xiong Y, Zhao X, Zhang F
Front Immunol 2023;14:1175048. Epub 2023 Apr 20 doi: 10.3389/fimmu.2023.1175048. PMID: 37153568Free PMC Article
Interferon-gamma and perforin-positive T cells in acquired aplastic anemia: implication in therapeutic response.
Sharma V, Kumar P, Kumar R, Chakraborty S, Namdeo M, Sazawal S, Kanga U, Seth T, Mitra DK
Clin Exp Immunol 2022 May 12;207(3):272-278. doi: 10.1093/cei/uxab006. PMID: 35553631Free PMC Article
A New Immunosuppressive Therapy for Very Severe Aplastic Anemia in Children with Autoantibodies.
Wang ZJ, Chen HB, Zhou F, Yu H, Wu XY, Shen YQ, Qiu YN, Jin RM
Curr Med Sci 2022 Apr;42(2):379-386. Epub 2022 Mar 8 doi: 10.1007/s11596-022-2519-2. PMID: 35258748
Supportive care in severe and very severe aplastic anemia.
Höchsmann B, Moicean A, Risitano A, Ljungman P, Schrezenmeier H
Bone Marrow Transplant 2013 Feb;48(2):168-73. Epub 2012 Dec 3 doi: 10.1038/bmt.2012.220. PMID: 23208312

Therapy

New-onset aplastic anemia after SARS-CoV-2 vaccination.
Chatzikalil E, Kattamis A, Diamantopoulos P, Solomou EE
Int J Hematol 2023 Dec;118(6):667-681. Epub 2023 Sep 28 doi: 10.1007/s12185-023-03666-z. PMID: 37768509
Development and validation of early death risk score model for emergency status prediction in very severe aplastic anemia.
Liu X, Yang W, Zhang L, Jing L, Ye L, Zhou K, Li Y, Li J, Fan H, Yang Y, Xiong Y, Zhao X, Zhang F
Front Immunol 2023;14:1175048. Epub 2023 Apr 20 doi: 10.3389/fimmu.2023.1175048. PMID: 37153568Free PMC Article
A New Immunosuppressive Therapy for Very Severe Aplastic Anemia in Children with Autoantibodies.
Wang ZJ, Chen HB, Zhou F, Yu H, Wu XY, Shen YQ, Qiu YN, Jin RM
Curr Med Sci 2022 Apr;42(2):379-386. Epub 2022 Mar 8 doi: 10.1007/s11596-022-2519-2. PMID: 35258748
Supportive care in severe and very severe aplastic anemia.
Höchsmann B, Moicean A, Risitano A, Ljungman P, Schrezenmeier H
Bone Marrow Transplant 2013 Feb;48(2):168-73. Epub 2012 Dec 3 doi: 10.1038/bmt.2012.220. PMID: 23208312
Immunosuppressive therapy in aplastic anemia.
Gupta V, Kumar A, Tilak V, Saini I, Bhatia B
Indian J Pediatr 2012 Dec;79(12):1587-91. Epub 2012 Jan 25 doi: 10.1007/s12098-012-0691-2. PMID: 22274992

Prognosis

Development and validation of early death risk score model for emergency status prediction in very severe aplastic anemia.
Liu X, Yang W, Zhang L, Jing L, Ye L, Zhou K, Li Y, Li J, Fan H, Yang Y, Xiong Y, Zhao X, Zhang F
Front Immunol 2023;14:1175048. Epub 2023 Apr 20 doi: 10.3389/fimmu.2023.1175048. PMID: 37153568Free PMC Article
Nutritional assessment of patients with aplastic anemia: comparison of four nutritional screening tools.
Tan X, Tian Y, Li Z, Cui S, Wang Z, Zhou Y, Liu Z, Kong F, Wang Y, Xu R
Nutr Hosp 2022 Dec 20;39(6):1289-1297. doi: 10.20960/nh.03957. PMID: 36173198
Interferon-gamma and perforin-positive T cells in acquired aplastic anemia: implication in therapeutic response.
Sharma V, Kumar P, Kumar R, Chakraborty S, Namdeo M, Sazawal S, Kanga U, Seth T, Mitra DK
Clin Exp Immunol 2022 May 12;207(3):272-278. doi: 10.1093/cei/uxab006. PMID: 35553631Free PMC Article
Adult aplastic anemia in Thailand: incidence and treatment outcome from a prospective nationwide population-based study.
Norasetthada L, Wongkhantee S, Chaipokam J, Charoenprasert K, Chuncharunee S, Rojnuckarin P, Sirijerachai C, Wanachiwanawin W, Issaragrisil S; Thai Aplastic Anemia Study Group
Ann Hematol 2021 Oct;100(10):2443-2452. Epub 2021 Jul 16 doi: 10.1007/s00277-021-04566-0. PMID: 34269837Free PMC Article
Supportive care in severe and very severe aplastic anemia.
Höchsmann B, Moicean A, Risitano A, Ljungman P, Schrezenmeier H
Bone Marrow Transplant 2013 Feb;48(2):168-73. Epub 2012 Dec 3 doi: 10.1038/bmt.2012.220. PMID: 23208312

Clinical prediction guides

New-onset aplastic anemia after SARS-CoV-2 vaccination.
Chatzikalil E, Kattamis A, Diamantopoulos P, Solomou EE
Int J Hematol 2023 Dec;118(6):667-681. Epub 2023 Sep 28 doi: 10.1007/s12185-023-03666-z. PMID: 37768509
Development and validation of early death risk score model for emergency status prediction in very severe aplastic anemia.
Liu X, Yang W, Zhang L, Jing L, Ye L, Zhou K, Li Y, Li J, Fan H, Yang Y, Xiong Y, Zhao X, Zhang F
Front Immunol 2023;14:1175048. Epub 2023 Apr 20 doi: 10.3389/fimmu.2023.1175048. PMID: 37153568Free PMC Article
Interferon-gamma and perforin-positive T cells in acquired aplastic anemia: implication in therapeutic response.
Sharma V, Kumar P, Kumar R, Chakraborty S, Namdeo M, Sazawal S, Kanga U, Seth T, Mitra DK
Clin Exp Immunol 2022 May 12;207(3):272-278. doi: 10.1093/cei/uxab006. PMID: 35553631Free PMC Article
Adult aplastic anemia in Thailand: incidence and treatment outcome from a prospective nationwide population-based study.
Norasetthada L, Wongkhantee S, Chaipokam J, Charoenprasert K, Chuncharunee S, Rojnuckarin P, Sirijerachai C, Wanachiwanawin W, Issaragrisil S; Thai Aplastic Anemia Study Group
Ann Hematol 2021 Oct;100(10):2443-2452. Epub 2021 Jul 16 doi: 10.1007/s00277-021-04566-0. PMID: 34269837Free PMC Article
Supportive care in severe and very severe aplastic anemia.
Höchsmann B, Moicean A, Risitano A, Ljungman P, Schrezenmeier H
Bone Marrow Transplant 2013 Feb;48(2):168-73. Epub 2012 Dec 3 doi: 10.1038/bmt.2012.220. PMID: 23208312

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