Sustained Complete Molecular Remission With Imatinib Monotherapy in a Child Presenting With Blast Phase FIP1L1-PDGFRA-Associated Myeloid Neoplasm With Eosinophilia

• Type of Material: Article
• Authors: Juhi Jain, Children's Healthcare of Atlanta; Elizabeth Weinzierl, Emory University; Debra Saxe, Emory University; Daniel Bergsagel, Emory University; Jason Gotlib, Stanford University; Andreas Reiter, University Hospital Mannheim; Sunil Raikar, Emory University
• Language: English
• Date: 2020-12-01
• Publisher: LIPPINCOTT WILLIAMS & WILKINS
• Publication Version: Final Published Version
• Copyright Statement: © 2020 the Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the European Hematology Association.
• License: Creative Commons Attribution 4.0 International
• Final Published Version (URL): http://dx.doi.org/10.1097/HS9.0000000000000486
• Title of Journal or Parent Work: HEMASPHERE
• Volume: 4
• Issue: 6
• Start Page: e486
• End Page: e486
• Keywords: Hematology; Life Sciences & Biomedicine; Science & Technology
• Research Categories: Health Sciences, Medicine and Surgery; Biology, Molecular

Abstract

The etiology of hypereosinophilia is divided into 2 categories, clonal or reactive in origin. The clonal form typically presents as a chronic myeloproliferative neoplasm (MPN), associated with rearrangements involving PDGFRA/B, FGFR1 or with the PCM1-JAK2 fusion.1 These fusion genes result in a constitutively active tyrosine kinase, and the PDGFR-rearrangements are very sensitive to tyrosine kinase inhibitors (TKIs) such as imatinib. More rarely, these rearrangements can be seen in cases of acute myeloid leukemia (AML), T-cell lymphoblastic leukemia/lymphoma (T-ALL/T-LLy), or mixed phenotype acute leukemia (MPAL), generally associated with eosinophilia.1 Given the varied presentation, the World Health Organization now classifies these malignancies under the category of “myeloid/lymphoid neoplasms with eosinophilia (MLN-Eo) and rearrangement of PDGFRA, PDGFRB, or FGFR1, or with PCM1-JAK2”. This disease is extremely uncommon in children and only 8 FIP1L1-PDGRFA cases have previously been described.