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Long-term follow-up of FIP1L1-PDGFRA-mutated patients with eosinophilia: survival and clinical outcome

Leukemia volume 26, pages 2439–2441 (2012)Cite this article

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Identification of the karyotypically occult fusion oncogene FIP1L1-PDGFRA by Cools et al.1 in 2003 provided the molecular basis for the dramatic response to imatinib therapy in a subset of patients with eosinophilia.2, 3, 4 As per the World Health Organization (WHO), patients harboring this molecular abnormality are classified as ‘Myeloid and lymphoid neoplasms associated with eosinophilia and abnormalities of PDGFRA, PDGFRB or FGFR1’.

Ever since the original report was published, several studies have described the clinico-pathological features of this disease entity and confirmed the achievement of rapid and complete clinical and hematological responses with imatinib at doses ranging from 100 to 400 mg/day.5, 6, 7, 8, 9, 10, 11 The majority of patients also achieve molecular remission with imatinib therapy, with clinical responses being durably maintained on imatinib doses as low as 50–200 mg/week.9, 12 Discontinuation of imatinib therapy is associated with molecular relapse within weeks to months, which may be reversible on treatment resumption;8, 10, 11, 13 secondary resistance to imatinib is relatively rare and occurs in the context of accelerated or blast phase disease and acquisition of PDGFRA T674I and/or D842V mutations.14

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References

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Authors and Affiliations

  1. Division of Hematology, Department of Medicine, Mayo Clinic, Rochester, MN, USA

    A Pardanani, A D'Souza & A Tefferi

  2. Division of Cytogenetics, Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, MN, USA

    R A Knudson & R P Ketterling

  3. Division of Hematopathology, Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, MN, USA

    C A Hanson

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  1. A Pardanani
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  2. A D'Souza
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  4. C A Hanson
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  5. R P Ketterling
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  6. A Tefferi
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Correspondence to A Tefferi.

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Author contributions

AP and AT contributed patients. AP, AD and AT reviewed, summarized and analyzed the clinical and laboratory data. RAK and RPK designed, performed and analyzed the FISH assays for FIP1L1-PDGFRA. CAH reviewed the bone marrow histology. All authors contributed towards writing the paper and approved the submitted draft.

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Pardanani, A., D'Souza, A., Knudson, R. et al. Long-term follow-up of FIP1L1-PDGFRA-mutated patients with eosinophilia: survival and clinical outcome. Leukemia 26, 2439–2441 (2012). https://doi.org/10.1038/leu.2012.162

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