Acquired amegakaryocytic thrombocytopaenia after treatment with immune checkpoint inhibitor as part of neoadjuvant treatment in a patient with bilateral breast cancer

Immune checkpoint inhibitors are a standard treatment for solid metastatic cancer and are now used as neoadjuvant therapy in early triple-negative breast cancer.While haematological immune-related adverse events are known, acquired amegakaryocytic thrombocytopaenia (AAT) is a rare condition characterised by a complete absence of megakaryocytes, leading to severe thrombocytopaenia, with no standardised treatment available.A patient in her early 60s presented with a low platelet count and was initially treated with high-dose methylprednisolone (1 g daily) for suspected immune-mediated thrombocytopaenia, but this was ineffective. A bone marrow biopsy revealed a depletion of megakaryocytes.The treatment was switched to eltrombopag, cyclosporine and tranexamic acid.Despite low platelet levels, the patient successfully underwent bilateral mastectomy with supportive platelet transfusions.Three weeks after starting eltrombopag and cyclosporine, her platelet count returned to normal. AAT in neoadjuvant treatment has not been previously reported, highlighting the need for interdisciplinary collaboration to address these challenges.

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Publication Details

Journal
BMJ Case Reports
Published
2025-03-01
DOI
https://doi.org/10.1136/bcr-2024-263434
Primary Topic
Platelet Disorders and Treatments
Type
article
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article

Acquired amegakaryocytic thrombocytopaenia after treatment with immune checkpoint inhibitor as part of neoadjuvant treatment in a patient with bilateral breast cancer

Henrik Frederiksen, Louise Kristensen, Vivi Quoc Nguyen
1 links
BMJ Case Reports
Platelet Disorders and Treatments
article

Acquired amegakaryocytic thrombocytopaenia after treatment with immune checkpoint inhibitor as part of neoadjuvant treatment in a patient with bilateral breast cancer

Henrik Frederiksen, Louise Kristensen, Vivi Quoc Nguyen
article en

Abstract

Immune checkpoint inhibitors are a standard treatment for solid metastatic cancer and are now used as neoadjuvant therapy in early triple-negative breast cancer.While haematological immune-related adverse events are known, acquired amegakaryocytic thrombocytopaenia (AAT) is a rare condition characterised by a complete absence of megakaryocytes, leading to severe thrombocytopaenia, with no standardised treatment available.A patient in her early 60s presented with a low platelet count and was initially treated with high-dose methylprednisolone (1 g daily) for suspected immune-mediated thrombocytopaenia, but this was ineffective. A bone marrow biopsy revealed a depletion of megakaryocytes.The treatment was switched to eltrombopag, cyclosporine and tranexamic acid.Despite low platelet levels, the patient successfully underwent bilateral mastectomy with supportive platelet transfusions.Three weeks after starting eltrombopag and cyclosporine, her platelet count returned to normal. AAT in neoadjuvant treatment has not been previously reported, highlighting the need for interdisciplinary collaboration to address these challenges.

BMJ Case ReportsVol. 18(3)
Odense University Hospital (DK)
Openalex Percentile: Top 93%
Platelet Disorders and Treatments
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Acquired amegakaryocytic thrombocytopaenia after treatment with immune checkpoint inhibitor as part of neoadjuvant treatment in a patient with bilateral breast cancer — Henrik Frederiksen, Louise Kristensen, et al. · BMJ Case Reports (2025) | TGRS Research Map | TGRS