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Long-term follow-up of treatment with imatinib in eosinophilia-associated myeloid/lymphoid neoplasms with PDGFR rearrangements in blast phase

Leukemia volume 27, pages 2254–2256 (2013)Cite this article

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Several cooperative study groups and single institutions have reported on high rates of rapid and durable complete hematologic (CHR) and complete molecular remissions (CMR) on imatinib in eosinophilia-associated myeloid/lymphoid neoplasms (MLN-Eo) with rearrangements of PDGFR (PDGFRA and PDGFRB) in chronic phase (CP). These responses have translated into vastly improved progression-free and overall survival (OS).1, 2, 3, 4, 5, 6, 7, 8, 9 In this report, we present data from the ‘German Registry on Disorders of Eosinophils and Mast Cells’ on long-term follow-up of 17 patients primarily diagnosed in blast phase (BP). BP was defined as (i) myeloid BP (>20% blasts in peripheral blood (PB) and/or bone marrow (BM)), (ii) chloroma (extramedullary tissue infiltration by myeloid blasts) or (iii) extramedullary lymphoid BP (lymph node (LN) infiltration by lymphoblasts of T-cell origin).

Thirteen patients were FIP1L1-PDGFRA-positive (myeloid BP, n=8; chloroma, n=2; lymphoid BP, n=3). In all three patients investigated, FIP1L1-PDGFRA could be identified contemporaneously in myeloid cells derived from BM and CD3-positive lymphoblasts in LN biopsies. Four patients (myeloid BP, n=2; lymphoid BP, n=2) were positive for a rearrangement of PDGFRB (partner genes: ETV6, n=1; SART3, n=1; unknown partner gene, n=2).10 All patients were male, median age was 46 years (range 37–66). Significant eosinophilia >1.5 × 109/l (median 7.7 × 109/l, range 1.3–20.7) was present in 12 of 13 (92%) FIP1L1-PDGFRA-positive patients and 2 of 4 (50%) patients (median 1.2 × 109/l, range 0.1–33.0) with rearrangements of PDGFRB. In addition to the specific morphologic features of PB, BM and LN, the most common clinical characteristics included splenomegaly (17/17, 100%), involvement of lung (4/17, 24%; pleural effusion, n=4), skin (4/17, 24%) and heart (2/17, 12%; endo-/myocarditis, n=1; intracardial thrombus, n=1).

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Acknowledgements

This work was supported by the ‘Deutsche José Carreras Leukämie Stiftung e.V.’ (R09/29f and H11/03), Germany.

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Authors and Affiliations

  1. III. Medizinische Klinik, Universitätsmedizin Mannheim, Mannheim, Germany

    G Metzgeroth, J Schwaab, D Gosenca, A Fabarius, W-K Hofmann & A Reiter

  2. Münchner Leukämie Labor, München, Germany

    C Haferlach

  3. Abteilung Hämatologie/Onkologie, Klinik für Innere Medizin II, Universitätsklinikum Jena, Jena, Germany

    A Hochhaus

  4. Wessex Regional Genetics Laboratory, Salisbury, UK

    N C P Cross

  5. Faculty of Medicine, University of Southampton, Southampton, UK

    N C P Cross

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  1. G Metzgeroth
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Correspondence to A Reiter.

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Competing interests

GM, AH, NCPC, WKH and AR received honoraria and travel support from Novartis Pharma.

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Metzgeroth, G., Schwaab, J., Gosenca, D. et al. Long-term follow-up of treatment with imatinib in eosinophilia-associated myeloid/lymphoid neoplasms with PDGFR rearrangements in blast phase. Leukemia 27, 2254–2256 (2013). https://doi.org/10.1038/leu.2013.129

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