FIP1L1 (Factor Interacting With PAPOLA And CPSF1)

P3, N=14, Recruiting, AstraZeneca | Trial primary completion date: Oct 2026 --> Jul 2027

Our data highlights mutational variation across demographic cohorts. These patterns are vital to future studies into identifying diagnostic markers or therapeutic targets.

Molecular docking and 200 ns molecular dynamics simulations supported stable dual binding of 9d within the ATP-binding pocket of PDGFR and the catalytic cleft of CA IX. By simultaneously targeting oncogenic PDGFRA signaling and hypoxia-driven pH regulation (CA IX/XII), 9d represents a promising lead for preclinical development in PDGFR/CA IX/XII-driven leukemias.

P2, N=80, Recruiting, National Institute of Allergy and Infectious Diseases (NIAID) | N=60 --> 80

The case highlights how delayed recognition of clonal eosinophilia can permit extensive organ injury, whereas early molecular testing and prompt initiation of imatinib yield dramatic clinical and hematologic remission. Persistent hypereosinophilia, particularly with cutaneous or gastrointestinal involvement, should prompt evaluation for PDGFRA-rearranged disease to enable early intervention and prevent irreversible tissue damage.

Acute promyelocytic leukemia (APL) is a distinct subtype of acute myeloid leukemia characterized by the promyelocytic leukemia-retinoic acid receptor alpha (PML-RARA) fusion gene and exceptional responsiveness to differentiation therapy with all-trans retinoic acid (ATRA) and arsenic trioxide (Arsenic Trioxide)...Anthracycline-based chemotherapy was subsequently administered, followed by azacitidine combined with venetoclax in the subsequent course of treatment...A literature review suggests that FIP1L1-RARA-positive APL represents a biologically and clinically distinct entity with highly variable treatment responses and poor prognosis. Early molecular diagnosis and prompt implementation of conventional chemotherapy or targeted therapies such as venetoclax may be essential to improving outcomes in this rare APL subtype.

Cardiac involvement in hypereosinophilic syndromes requires multidisciplinary management combining cytoreductive therapy, anticoagulation when thrombus is present, and serial cardiac magnetic resonance. Testing for FIP1L1-platelet-derived growth factor receptor α is disease-defining and therapy-guiding given the marked response to imatinib.

P3, N=134, Active, not recruiting, AstraZeneca | Trial completion date: Sep 2026 --> Dec 2026

We sequenced genomic DNA from patients (n = 20) with a known or suspected TK gene fusion and identified rearrangements in 18 cases, including all cases with a known TK fusion, typical and atypical BCR::ABL1 rearrangements, an 843Kb deletion causing a FIP1L1::PDGFRA fusion, novel AGAP2::PDGFRB and NFIA::PDGFRB fusions, and a complex CCDC88C::PDGFRB rearrangement with multiple translocation events. The approach was fast (<72 h/sample from DNA to result), flexible with minimal hands-on laboratory time, and provided accurate, patient-specific characterisation of genomic breakpoints.

Meanwhile, the FIP1L1::PDGFRA fusion gene detection results strongly support the use of molecular diagnosis in ABL. In addition, the patient successfully achieved a complete response to treatment and was followed for a year and a half without relapse, thus offering valuable insights for treatment strategies in similar cases.