Frontiers in Oncology (Mar 2024)

Immune checkpoint inhibitor-related acquired amegakaryocytosis thrombocytopenia: a case report and literature review

  • Valérian Rivet,
  • Vincent Sibaud,
  • Jérémie Dion,
  • Thibaut Volosov,
  • Mélanie Biteau,
  • Andréa Pastissier,
  • Karen Delavigne,
  • Pierre Cougoul,
  • Odile Rauzy,
  • Thibault Comont

DOI
https://doi.org/10.3389/fonc.2024.1353896
Journal volume & issue
Vol. 14

Abstract

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IntroductionImmune checkpoint inhibitors (ICIs) are used in several advanced malignancies and may cause various immune-related adverse events (irAEs). Among them, hematological irAEs are less described. Acquired amegakaryocytic thrombocytopenia (AAT) is a rare immune hematologic disorder characterized by severe thrombocytopenia and complete absence of megakaryocytes in bone marrow.Case presentationHerein, we present the case of a patient in their 40s with metastatic melanoma who developed an AAT after 12 cycles of nivolumab (anti-PD1). His platelet count decreased by ≤5 × 109/l without other cytopenia. Bone marrow biopsy showed normal cellularity with a complete absence of megakaryocyte and T-CD8+ lymphocyte infiltration. Given the failure of systemic steroids, eltrombopag was started, an oral thrombopoietin receptor agonist (TPO-RA), and his platelet count subsequently increased with complete response.DiscussionFour other cases are described on literature with the same features than non-ICI-related AAT. All cases occurred after anti-PD/PD-L1 treatment with a median onset of 5 weeks. The presentation of our case is quite different with delayed cytopenia. Both ciclosporin and TPO-RA seem to be efficient therapies.ConclusionTPO-RA could be preferred in oncologic patients, but safety data are still missing to define clear guidelines for immune-related AAT management.

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