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Clinical Journal of Oncology Nursing 2001
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Indian Journal of Pediatrics May 1970
Review
Topics: Anemia, Aplastic; Bone Marrow Transplantation; Diagnosis, Differential; Humans; Lectins; Prognosis; Splenectomy; Transplantation, Homologous
PubMed: 4933139
DOI: 10.1007/BF02753089 -
Journal of Paediatrics and Child Health Jul 2020Aplastic anaemia is a rare, previously fatal condition with a significantly improved survival rate owing to advances in understanding of the pathophysiology and improved... (Review)
Review
Aplastic anaemia is a rare, previously fatal condition with a significantly improved survival rate owing to advances in understanding of the pathophysiology and improved treatment strategies including haematopoietic stem cell transplantation. Although a rare condition, aplastic anaemia continues to present a high burden for affected patients, their families and the health system due to the prolonged course of disease often associated with high morbidity and the uncertainty regarding clinical outcome. Modern molecular and genetic techniques including next-generation sequencing have contributed to a better understanding of this heterogeneous group of conditions, albeit at a cost of increased complexity of clinical decision-making regarding prognosis and choice of treatment for individual patients. Here we present a concise and comprehensive review of aplastic anaemia and closely related conditions based on extensive literature review and long-standing clinical experience. The review takes the reader across the complex pathophysiology consisting of three main causative mechanisms of bone marrow destruction resulting in aplastic anaemia: direct injury, immune mediated and bone marrow failure related including inherited and clonal disorders. A comprehensive diagnostic algorithm is presented and an up-to-date therapeutic approach to acquired immune aplastic anaemia, the most represented type of aplastic anaemia, is described. Overall, the aim of the review is to provide paediatricians with an update of this rare, heterogeneous and continuously evolving condition.
Topics: Anemia, Aplastic; Hematopoietic Stem Cell Transplantation; Humans; Prognosis
PubMed: 32619069
DOI: 10.1111/jpc.14996 -
British Journal of Haematology Jan 2016
Topics: Aged; Anemia, Aplastic; Benzoates; Blood Component Transfusion; Female; Hematopoietic Stem Cell Transplantation; Hemoglobinuria, Paroxysmal; Humans; Hydrazines; Immunosuppressive Agents; Opportunistic Infections; Pregnancy; Pregnancy Complications, Hematologic; Pyrazoles; Severity of Illness Index
PubMed: 26568159
DOI: 10.1111/bjh.13853 -
JAMA Jul 1999
Review
Topics: Adult; Anemia, Aplastic; Blood Transfusion; Hepatitis; Humans; Immunosuppression Therapy; Male; Pancytopenia
PubMed: 10422997
DOI: 10.1001/jama.282.3.271 -
Journal of the American Pharmaceutical... Aug 1974
Topics: Anemia, Aplastic; Humans
PubMed: 4850618
DOI: 10.1016/s0003-0465(16)34351-8 -
British Journal of Haematology Sep 2021The progress in aplastic anaemia (AA) management is one of success. Once an obscure entity resulting in death in most affected can now be successfully treated with... (Review)
Review
The progress in aplastic anaemia (AA) management is one of success. Once an obscure entity resulting in death in most affected can now be successfully treated with either haematopoietic stem cell transplantation (HSCT) or immunosuppressive therapy (IST). The mechanisms that underly the diminution of haematopoietic stem cells (HSCs) are now better elucidated, and include genetics and immunological alterations. Advances in supportive care with better antimicrobials, safer blood products and iron chelation have greatly impacted AA outcomes. Working somewhat 'mysteriously', anti-thymocyte globulin (ATG) forms the base for both HSCT and IST protocols. Efforts to augment immunosuppression potency have not, unfortunately, led to better outcomes. Stimulating HSCs, an often-sought approach, has not been effective historically. The thrombopoietin receptor agonists (Tpo-RA) have been effective in stimulating early HSCs in AA despite the high endogenous Tpo levels. Dosing, timing and best combinations with Tpo-RAs are being defined to improve HSCs expansion in AA with minimal added toxicity. The more comprehensive access and advances in HSCT and IST protocols are likely to benefit AA patients worldwide. The focus of this review will be on the medical treatment advances in AA.
Topics: Anemia, Aplastic; Animals; Benzoates; Disease Management; Hematopoietic Stem Cell Transplantation; Humans; Hydrazines; Immunosuppression Therapy; Pyrazoles; Severity of Illness Index
PubMed: 33855695
DOI: 10.1111/bjh.17403 -
The New England Journal of Medicine Mar 1982
Review
Topics: Anemia, Aplastic; Animals; Bone Marrow; Bone Marrow Transplantation; Disease Models, Animal; Female; Humans; Immunosuppressive Agents; Pregnancy; Prognosis; Rats
PubMed: 7035946
DOI: 10.1056/NEJM198203183061105 -
Acta Clinica Belgica Apr 2022A 61-year-old female presented with pancytopenia with a hemoglobin of 7.6 g/dL, platelet count of 26.000/µL and neutrophil count of 525/µL. Bone marrow aspirate... (Review)
Review
A 61-year-old female presented with pancytopenia with a hemoglobin of 7.6 g/dL, platelet count of 26.000/µL and neutrophil count of 525/µL. Bone marrow aspirate showed moderately cellular marrow with a dysplastic erythroid lineage and poor megakaryo- and granulopoiesis without excessive blast count. Trephine biopsy revealed profoundly hypocellular marrow with rare hematopoietic elements. Conventional karyotyping was normal and next generation sequencing revealed no mutations. These findings were compatible with transfusion dependent, non-severe aplastic anaemia (AA) with grade 3 thrombopenia and neutropenia. However, diagnostic workup including CT thorax revealed unexpected sclerotic bone conversions in the spine. Additional whole body SPECT with 99mTc-HDP showed multiple bone lesions in the cervical, thoracic and lumbar spine. CT guided biopsy of D12 surprisingly revealed normal trilineage hematopoiesis. These results were very discrepant from the profoundly hypocellular marrow from the trephine biopsy. It is known that in AA residual hyperactive foci of hematopoiesis can persist; so called 'hot pockets'. MRI is the preferred imaging technique in AA; in most cases a homogeneous fatty bone marrow is found, though some patients present with a heterogeneous marrow with foci of decreased intensity, corresponding with residual foci of hematopoiesis. Imaging studies with PET-CT and PET-MRI confirm these different patterns with respectively homogeneous hypometabolism and hypometabolism with focal hyperproliferation. However, there is no previous literature on the aspect of this focal hematopoiesis on computed tomography. This is the first description of a 'hot pocket' manifesting as a sclerotic bone lesion on CT.
Topics: Anemia, Aplastic; Bone Marrow; Bone Neoplasms; Female; Humans; Middle Aged; Pancytopenia; Positron Emission Tomography Computed Tomography
PubMed: 33433292
DOI: 10.1080/17843286.2021.1872310 -
Seminars in Hematology Jan 2000