Abstract: SA-PO1239
Long-Term Kidney Outcomes in Patients with Chronic Myeloid Leukemia (CML) Treated with Ponatinib: A Real-World Cohort Study
Session Information
- Onconephrology: Epidemiological Trends, Risk Stratification, and Clinical Outcomes
October 24, 2026 | Location: Exhibit Hall A, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Onconephrology
- 1600 Onconephrology
Authors
- Shukla, Parnika, Medical College of Wisconsin, Milwaukee, Wisconsin, United States
- Eschweiler, Eilis M., Western Michigan University, Kalamazoo, Michigan, United States
- Gudsoorkar, Prakash Shashikant, University of Cincinnati, Cincinnati, Ohio, United States
- Gupta, Shruti, Mass General Brigham Inc, Boston, Massachusetts, United States
- Hanna, Paul, Medical College of Wisconsin, Milwaukee, Wisconsin, United States
Background
Ponatinib, a 3rd-generation BCR-ABL1 tyrosine kinase inhibitor for resistant CML, is linked to vascular toxicity via VEGFR inhibition & may also cause nephrotoxicity. However, renal safety data remain limited to case reports, with no real-world evidence on renal outcomes.
Methods
Using data from the TriNetX Research Network, we conducted a retrospective cohort study of 804 real-world patients with CML. Exclusions were those with advanced CKD, on dialysis, prior kidney transplant, multiple myeloma, structural kidney disease, or limited follow-up data. Primary outcome was estimated glomerular filtration rate (eGFR) trajectory. Secondary outcomes included a composite adverse kidney outcome defined as sustained >40% eGFR decline, eGFR <15 mL/min/1.73sqm, or dialysis initiation following initiation of ponatinib (Kaplan-Meier analysis)
Results
A median per-patient eGFR slope was −2.16 mL/min/yr (IQR −7.3 to +0.5) over a median follow-up of 18 months (IQR 5–45) compared to the previously established decline of 0.75 mL/min/1.73sqm among healthy controls with normal aging (Fig 1). By the end of year one, 25% of patients reached the composite kidney endpoint, increasing to 43% by year five. Patients who went 2 or more years without early kidney events had their eGFR stabilize near age-expected rates. The composite adverse kidney outcome occurred in 28.4% overall, with the majority of events attributed to sustained >40% eGFR decline; only 8 initiated dialysis. Cumulative incidence of the composite outcome reached 25.2% (95% CI 21.7–28.5%) at one year and 42.9% (95% CI 37.6–47.7%) at five years. Among patients with ≥24 months of follow-up free of early kidney events (n=321), median eGFR slope was −1.90 mL/min/yr and was statistically indistinguishable from expected age-related decline, suggesting relative stabilization in long-term survivors.
Conclusion
In this large real-world cohort, ponatinib was associated with significant kidney function decline, with 25% of patients reaching an adverse renal endpoint within one year. These findings support routine renal monitoring and prospective studies of kidney outcomes during ponatinib therapy.