Immune Checkpoint Inhibitor-related Acquired Amegakaryocytosis Thrombocytopenia: a Case Report and Literature Review

Overview

Journal Front Oncol

Specialty Oncology

Date 2024 Mar 22

PMID 38515568

Authors

Valerian Rivet

Vincent Sibaud

Jeremie Dion

Thibaut Volosov

Melanie Biteau

Andrea Pastissier

Karen Delavigne

Pierre Cougoul

Odile Rauzy

Thibault Comont

Affiliations

Soon will be listed here.

Abstract

Introduction: Immune 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 Presentation: Herein, 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 × 10/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.

Discussion: Four 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.

Conclusion: TPO-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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