Safety and efficacy of eltrombopag in patients with post-CAR T cytopenias

Eur J Haematol. 2024 Apr;112(4):538-546. doi: 10.1111/ejh.14141. Epub 2023 Dec 3.

Abstract

Background: While chimeric antigen receptor (CAR) T-cell therapy has revolutionized the treatment outcomes of relapsed/refractory hematological malignancies, this therapy is associated with post-treatment cytopenias, which can pose a challenge to its safe administration. This study describes the management of post-CAR T cytopenias using the thrombopoietin mimetic eltrombopag.

Methods: This retrospective analysis included adult patients with lymphoma or myeloma who received CAR T-cell therapy at two academic medical centers. Eltrombopag was initiated for patients who had persistent high-grade leukopenia and/or thrombocytopenia beyond 21 days post-CAR T infusion. Risk factors and outcomes were assessed and compared for patients who did or did not receive eltrombopag.

Results: Among the 185 patients analyzed, a majority (88%) experienced thrombocytopenia or leukopenia at day +30 post-CAR T infusion. A total of 42 patients met the criteria for eltrombopag treatment and initiated therapy. Patients who received eltrombopag were more likely to have pre-existing cytopenias at lymphodepletion, receive bridging therapy, experience an infection, or require intensive care. Recovery from cytopenias occurred within 180 days for a majority (94%) of patients.

Conclusions: The use of eltrombopag for post-CAR T leukopenia and thrombocytopenia was considered safe without any significant toxicities. The use of eltrombopag for post-CAR T cytopenias might be effective in a high-risk patient population but requires further study.

Keywords: CAR T; Eltrombopag; leukopenia; lymphoma; myeloma; thrombocytopenia.

MeSH terms

  • Adult
  • Anemia* / etiology
  • Benzoates*
  • Cytopenia*
  • Hematologic Neoplasms* / complications
  • Hematologic Neoplasms* / therapy
  • Humans
  • Hydrazines*
  • Immunotherapy, Adoptive / adverse effects
  • Leukopenia*
  • Neoplasm Recurrence, Local
  • Pyrazoles*
  • Receptors, Chimeric Antigen*
  • Retrospective Studies
  • Thrombocytopenia* / drug therapy
  • Thrombocytopenia* / etiology

Substances

  • Receptors, Chimeric Antigen
  • eltrombopag
  • Benzoates
  • Hydrazines
  • Pyrazoles