Focal Segmental Sclerosis Associated with the Novel Multi-tyrosine Kinase Inhibitor Ponatinib

Area of Science:

• Nephrology
• Oncology
• Pharmacology

Background:

• Ponatinib is a multi-tyrosine kinase inhibitor (TKI) effective against refractory chronic myeloid leukemia (CML).
• While clinically efficacious, ponatinib's multi-target nature can lead to diverse adverse events.
• Renal complications are considered rare side effects of ponatinib therapy.

Observation:

• A 76-year-old female patient with CML resistant to imatinib and nilotinib was treated with ponatinib.
• The patient exhibited proteinuria and declining renal function during ponatinib treatment.
• These renal symptoms were not observed during prior imatinib or nilotinib therapy.

Findings:

• This case presents the first documented instance of secondary focal segmental glomerulosclerosis (FSGS) with partial glomerular collapse.
• The renal pathology was directly linked to ponatinib treatment in this patient.
• Ponatinib-induced nephrotoxicity can manifest as FSGS.

Implications:

• This finding highlights a rare but serious renal complication associated with ponatinib use.
• Clinicians should monitor renal function closely in CML patients treated with ponatinib.
• Further investigation into the mechanisms of ponatinib-induced nephrotoxicity is warranted.