ArticleHaematologica2013
Ponatinib as targeted therapy for FGFR1 fusions associated with the 8p11 myeloproliferative syndrome.
Article in Haematologica, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 papers.
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Who cites it
32 citing papers in PubMed.
- Olverembatinib for 8p11 myeloproliferative syndrome with a positive BCR-FGFR1 fusion gene: a case report.Annals of hematology · 2025Article
- Protein-Ligand Interactions in Cardiometabolic Drug Targets: Focus on Weight Loss and Cardioprotection.Molecules (Basel, Switzerland) · 2025Review
- Acute Lymphoblastic Leukemia Characterized by RareCase reports in hematology · 2025Article
- Pemigatinib therapy in myeloid/lymphoid neoplasm with FGFR1 rearrangement manifesting as aggressive B-cell lymphoma.Journal of clinical and experimental hematopathology : JCEH · 2025Article
- Biological and clinical implications of FGFR aberrations in paediatric and young adult cancers.Oncogene · 2023Review
- Article
- Identification of a novel HOOK3-FGFR1 fusion gene involved in activation of the NF-kappaB pathway.Cancer cell international · 2022Article
- The 8p11 myeloproliferative syndrome: Genotypic and phenotypic classification and targeted therapy.Frontiers in oncology · 2022Review
- [Ponatinib inhibits growth of patient-derived xenograft of cholangiocarcinoma expressing FGFR2-CCDC6 fusion protein in nude mice].Nan fang yi ke da xue xue bao = Journal of Southern Medical University · 2020Article
- Cardiotoxicity of the BCR-ABL1 tyrosine kinase inhibitors: Emphasis on ponatinib.International journal of cardiology · 2020Review
- Article
- Ponatinib-induced cardiotoxicity: delineating the signalling mechanisms and potential rescue strategies.Cardiovascular research · 2019Article
- Ponatinib Inhibits Proliferation and Induces Apoptosis of Liver Cancer Cells, but Its Efficacy Is Compromised by Its Activation on PDK1/Akt/mTOR Signaling.Molecules (Basel, Switzerland) · 2019Article
- Detection of novel fusion-transcripts by RNA-Seq in T-cell lymphoblastic lymphoma.Scientific reports · 2019Article
- Myelodysplastic Syndrome/Myeloproliferative Neoplasm (MDS/MPN) Overlap Syndromes: Molecular Pathogenetic Mechanisms and Their Implications.Indian journal of hematology & blood transfusion : an official journal of Indian Society of Hematology and Blood Transfusion · 2019Review
- Mate pair sequencing improves detection of genomic abnormalities in acute myeloid leukemia.European journal of haematology · 2019Article
- Review
- A novel computational approach for drug repurposing using systems biology.Bioinformatics (Oxford, England) · 2018Article
- Activity of fibroblast growth factor receptor inhibitors TKI258, ponatinib and AZD4547 against TPR‑FGFR1 fusion.Molecular medicine reports · 2017Article
- Antitumor effect of FGFR inhibitors on a novel cholangiocarcinoma patient derived xenograft mouse model endogenously expressing an FGFR2-CCDC6 fusion protein.Cancer letters · 2016Article
Corrections and comments
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Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The 8p11 myeloproliferative syndrome is a rare, aggressive myeloproliferative neoplasm characterized by constitutively active FGFR1 fusion proteins that arise from specific chromosomal translocations and which drive aberrant proliferation. Although FGFR1 inhibitors have shown in vitro activity against FGFR1 fusions, none are in use clinically and there is a need to assess additional compounds as potential therapy. Here we use cell lines and primary cells to investigate ponatinib (AP24534). Ponatinib-treated Ba/F3 cells transformed by ZMYM2-FGFR1 and BCR-FGFR1 and the FGFR1OP2-FGFR1 positive KG1A cell line showed reduced proliferation and decreased survival when compared to control cells. Inhibition induced apoptosis and reduced phosphorylation of the FGFR1 fusion proteins and substrates. Ponatinib-treated cells from 8p11 myeloproliferative syndrome patients (n=5) showed reduced colony growth compared to controls. In one evaluable patient, ponatinib specifically reduced numbers of FGFR1-fusion gene positive colonies. Ponatinib, therefore, shows considerable promise for the treatment of patients with 8p11 myeloproliferative syndrome.
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