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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5334413PMC
http://dx.doi.org/10.1007/s00277-017-2938-5DOI Listing

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CRLF2 rearrangements occur in >50% of Ph-like and Down syndrome (DS)-associated B-acute lymphoblastic leukemia (ALL) and induce constitutive kinase signaling targetable by the JAK1/2 inhibitor ruxolitinib under current clinical investigation. While chimeric antigen receptor T cell (CART) immunotherapies have achieved remarkable remission rates in children with relapsed/refractory B-ALL, ~50% of CD19CART-treated patients relapse again, many with CD19 antigen loss. We previously reported preclinical activity of thymic stromal lymphopoietin receptor-targeted cellular immunotherapy (TSLPRCART) against CRLF2-overexpressing ALL as an alternative approach.

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Objectives: Myelofibrosis is a rare bone marrow disorder associated with a high symptom burden, poor prognosis, and shortened survival. While allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment for myelofibrosis, the only approved and reimbursed pharmacotherapy for non-HSCT candidates in Belgium is ruxolitinib.

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Background: After ruxolitinib discontinuation, the outcome of patients with myelofibrosis (MF) is poor with scarce therapeutic possibilities.

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Article Synopsis
  • Myelofibrosis is a serious blood disorder characterized by symptoms like anemia, enlarged spleen, and bone marrow issues, with current treatments mainly focused on JAK inhibitors, particularly ruxolitinib.
  • Ruxolitinib is effective for many patients but often leads to side effects and resistance over time, making ongoing treatment challenging.
  • Newer JAK inhibitors like fedratinib, pacritinib, and momelotinib show promise for patients who do not respond well to ruxolitinib, with some improving symptoms and spleen size significantly.
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