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  1. Article ; Online: Guidelines for the diagnosis and management of adult aplastic anaemia: A British Society for Haematology Guideline.

    Kulasekararaj, Austin / Cavenagh, Jamie / Dokal, Inderjeet / Foukaneli, Theodora / Gandhi, Shreyans / Garg, Mamta / Griffin, Morag / Hillmen, Peter / Ireland, Robin / Killick, Sally / Mansour, Sahar / Mufti, Ghulam / Potter, Victoria / Snowden, John / Stanworth, Simon / Zuha, Roslin / Marsh, Judith

    British journal of haematology

    2024  Volume 204, Issue 3, Page(s) 784–804

    Abstract: 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 ... ...

    Abstract 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.
    MeSH term(s) Young Adult ; Humans ; Aged ; Anemia, Aplastic/therapy ; Immunosuppressive Agents/therapeutic use ; Cyclosporine/therapeutic use ; Hematopoietic Stem Cell Transplantation ; Bone Marrow Failure Disorders/drug therapy ; Unrelated Donors ; Pancytopenia/drug therapy ; Hematology
    Chemical Substances Immunosuppressive Agents ; Cyclosporine (83HN0GTJ6D)
    Language English
    Publishing date 2024-01-21
    Publishing country England
    Document type Journal Article
    ZDB-ID 80077-6
    ISSN 1365-2141 ; 0007-1048
    ISSN (online) 1365-2141
    ISSN 0007-1048
    DOI 10.1111/bjh.19236
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  2. Article: Long-term outcomes of previously untreated myeloma patients: responses to induction chemotherapy and high-dose melphalan incorporated within a risk stratification model can help to direct the use of novel treatments.

    Alvares, Caroline L / Davies, Faith E / Horton, Clive / Patel, Gita / Powles, Ray / Sirohi, Bhawna / Zuha, Roslin / Gatt, Alex / Saso, Radovan / Treleaven, Jennifer G / Dearden, Claire E / Potter, Michael N / Ethell, Mark E / Morgan, Gareth J

    British journal of haematology

    2005  Volume 129, Issue 5, Page(s) 607–614

    Abstract: Induction chemotherapy followed by high-dose melphalan (HDM) is the standard treatment for fitter patients with myeloma. The place of bortezomib and the thalidomide analogues within this treatment paradigm is yet to be established. We sought to identify ... ...

    Abstract Induction chemotherapy followed by high-dose melphalan (HDM) is the standard treatment for fitter patients with myeloma. The place of bortezomib and the thalidomide analogues within this treatment paradigm is yet to be established. We sought to identify patients who may benefit from the introduction of novel agents during their initial management. An intention-to-treat analysis was performed on 383 patients with newly diagnosed myeloma eligible for HDM to determine whether the extent of response to induction therapy and HDM correlated with long-term survival. Early response [complete response (CR) and partial response (PR)] to induction therapy was predictive of overall survival (OS) [median OS, 7.47 years for responders (CR and PR) versus 4.89 years for non-responders; P = 0.035]. The attainment of CR at 3 months post-HDM correlated with a prolonged progression-free survival (PFS) (median PFS, 7.4 years in CR group versus 5.3 years in non-CR group; P = 0.023). This data suggests that, at every stage of treatment, the aim should be to achieve CR. Patients with suboptimal responses could be offered alternative therapy. We propose a multiparametric risk-adapted model that includes response to induction chemotherapy and HDM, for identifying patients who may benefit from novel approaches to treatment.
    MeSH term(s) Adult ; Aged ; Antineoplastic Agents, Alkylating/therapeutic use ; Antineoplastic Combined Chemotherapy Protocols/therapeutic use ; Bone Marrow Transplantation ; Drug Administration Schedule ; Follow-Up Studies ; Humans ; Melphalan/administration & dosage ; Melphalan/therapeutic use ; Middle Aged ; Multiple Myeloma/drug therapy ; Multiple Myeloma/mortality ; Multiple Myeloma/therapy ; Remission Induction ; Risk ; Survival Rate ; Time Factors ; Treatment Outcome
    Chemical Substances Antineoplastic Agents, Alkylating ; Melphalan (Q41OR9510P)
    Language English
    Publishing date 2005-06
    Publishing country England
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 80077-6
    ISSN 1365-2141 ; 0007-1048
    ISSN (online) 1365-2141
    ISSN 0007-1048
    DOI 10.1111/j.1365-2141.2005.05514.x
    Database MEDical Literature Analysis and Retrieval System OnLINE

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