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British Journal of Haematology Mar 2024Pancytopenia with hypocellular bone marrow is the hallmark of aplastic anaemia (AA) and the diagnosis is confirmed after careful evaluation, following exclusion of...
Pancytopenia with hypocellular bone marrow is the hallmark of aplastic anaemia (AA) and the diagnosis is confirmed after careful evaluation, following exclusion of alternate diagnosis including hypoplastic myelodysplastic syndromes. Emerging use of molecular cyto-genomics is helpful in delineating immune mediated AA from inherited bone marrow failures (IBMF). Camitta criteria is used to assess disease severity, which along with age and availability of human leucocyte antigen compatible donor are determinants for therapeutic decisions. Supportive care with blood and platelet transfusion support, along with anti-microbial prophylaxis and prompt management of opportunistic infections remain key throughout the disease course. The standard first-line treatment for newly diagnosed acquired severe/very severe AA patients is horse anti-thymocyte globulin and ciclosporin-based immunosuppressive therapy (IST) with eltrombopag or allogeneic haemopoietic stem cell transplant (HSCT) from a matched sibling donor. Unrelated donor HSCT in adults should be considered after lack of response to IST, and up front for young adults with severe infections and a readily available matched unrelated donor. Management of IBMF, AA in pregnancy and in elderly require special attention. In view of the rarity of AA and complexity of management, appropriate discussion in multidisciplinary meetings and involvement of expert centres is strongly recommended to improve patient outcomes.
Topics: Young Adult; Humans; Aged; Anemia, Aplastic; Immunosuppressive Agents; Cyclosporine; Hematopoietic Stem Cell Transplantation; Bone Marrow Failure Disorders; Unrelated Donors; Pancytopenia; Hematology
PubMed: 38247114
DOI: 10.1111/bjh.19236 -
International Journal of Hematology Mar 2024Acquired aplastic anemia (AA) in children is a rare bone marrow failure that requires several special considerations for its diagnosis and treatment compared with that... (Review)
Review
Acquired aplastic anemia (AA) in children is a rare bone marrow failure that requires several special considerations for its diagnosis and treatment compared with that in adults. The most common issue is the differential diagnosis with refractory cytopenia of childhood and inherited bone marrow failure syndromes, which is crucial for making decisions on the appropriate treatment for pediatric AA. In addition to detailed morphological evaluation, a comprehensive diagnostic work-up that includes genetic analysis using next-generation sequencing will play an increasingly important role in identifying the underlying etiology of pediatric AA. When discussing treatment strategies for children with acquired AA, the long-term sequelae and level of hematopoietic recovery that affect daily or school life should also be considered, although the overall survival rate has reached 90% after immunosuppressive therapy or hematopoietic cell transplantation (HCT). Recent advances in HCT for pediatric patients with acquired AA have been remarkable, with the successful use of upfront bone marrow transplantation from a matched unrelated donor, unrelated cord blood transplantation or haploidentical HCT as salvage treatment, and fludarabine/melphalan-based conditioning regimens. This review discusses current clinical practices in the diagnosis and treatment of acquired AA in children based on the latest data.
Topics: Adult; Child; Humans; Anemia, Aplastic; Bone Marrow Transplantation; Hematopoietic Stem Cell Transplantation; Pancytopenia; Immunosuppression Therapy; Transplantation Conditioning
PubMed: 36867357
DOI: 10.1007/s12185-023-03564-4 -
Acta Neurologica Belgica Aug 2023
Topics: Humans; Levetiracetam; Pancytopenia; Anticonvulsants; Piracetam
PubMed: 36344882
DOI: 10.1007/s13760-022-02138-1 -
American Journal of Therapeutics
Topics: Humans; Leflunomide; Pancytopenia; Antirheumatic Agents; Methotrexate
PubMed: 35404332
DOI: 10.1097/MJT.0000000000001497