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Imatinib is effective in some PDGFRA/B-negative hypereosinophilic syndromes: A step closer to unveiling underlying

Giuseppe G Loscocco1, Grzegorz Helbig2

  • 1Department of Experimental and Clinical Medicine, CRIMM, Center of Research and Innovation of Myeloproliferative Neoplasms, Azienda Ospedaliero-Universitaria Careggi, University of Florence, Florence, Italy.

British Journal of Haematology
|October 29, 2024
PubMed

Hypereosinophilic syndromes (HES) comprise different clonal, reactive, or idiopathic disorders characterized by elevated eosinophil levels and subsequent organ damage. Kim et al. in a multicentre, single-arm, prospective phase II study, treated 32 patients with PDGFRA/B-negative HES with imatinib at the dose of 100-400 mg daily. Respective overall and complete haematological response rates were 46.9% and 18.8%, and the median time to response was 1.5 months. The molecular basis of responses was identified by using whole-exome and whole-transcriptome sequencing in 11 patients. STAT5B::RARA, PAK2::PIGX, and FIP1L1::CHIC2 fusions were identified in responders, whereas RNF130::BRAF and WNK1::KDM5A were identified in non-responders. Imatinib could be a therapeutic option for some, possibly clonal, PDGFRA/B-negative HES. Commentary on: Kim et al. Phase II trial of imatinib mesylate in patients with PDGFRA/B-negative hypereosinophilic syndrome. Br J Haematol 2024; 205:2305-2314.

Keywords:
PDGFRAPDGFRBeosinophiliahypereosinophilic syndromeimatinib

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