Romiplostim in aplastic anaemia – another tool in the armamentarium

Kulasekararaj, Austin; Marsh, Judith

doi:10.1111/bjh.17189

Cited by 3 publications

(2 citation statements)

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“…Although early-phase clinical trials show the efficacy of high doses of romiplostin in refractory AA, its use is not approved and is not routine practice. [101][102][103][104] Eltrombopag combined with ATG and ciclosporin, as first-line treatment for SAA and VSAA, was associated with a markedly improved CR in a single-arm study 83,105 and also in the recently published pivotal randomised controlled RACE trial. 70 The addition of eltrombopag to ATG and ciclosporin in SAA/VSAA has been approved by the US FDA and several regulatory authorities globally.…”

Section: Tpo Receptor Agonist: Eltrombopagmentioning

confidence: 98%

“…Eltrombopag has been Food and Drug Administration (FDA)‐approved and licensed by the European Medicines Agency (EMA) for SAA refractory to IST or patients heavily pretreated and unsuitable for HSCT. Although early‐phase clinical trials show the efficacy of high doses of romiplostin in refractory AA, its use is not approved and is not routine practice 101–104 …”

Section: Immunosuppressive Therapymentioning

confidence: 99%

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Guidelines for the diagnosis and management of adult aplastic anaemia: A British Society for Haematology Guideline

Kulasekararaj,

Cavenagh,

Dokal

et al. 2024

Br J Haematol

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SummaryPancytopenia 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.

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