The 51st Annual Meeting of the European Society for Blood and Marrow Transplantation: Physicians Award Winners (O001-O007) - Takeaways - MDSpire

The 51st Annual Meeting of the European Society for Blood and Marrow Transplantation: Physicians Award Winners (O001-O007)

  • November 5, 2025

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  • 1

    Ghosal hematodiaphyseal dysplasia (GHD) is a rare disorder characterized by severe anemia and painful cortical hypertrophy of long bones.

  • 2

    NSAIDs have been shown to be an effective first-line treatment for GHD, providing significant hemoglobin and platelet responses without major adverse effects.

  • 3

    Patients treated with NSAIDs achieved hemoglobin responses within a median of 3 months and platelet responses within 2 months.

  • 4

    Bone marrow biopsies in GHD patients revealed severe hypoplasia and interstitial edema, complicating diagnosis and treatment.

  • 5

    Screening for TBXAS1 mutations should be standard in inherited bone marrow failure syndromes, especially before hematopoietic stem cell transplantation.

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