Trastuzumab Emtansine-Associated Porto-Sinusoidal Vascular Disorder: Clinical Features and Outcomes from Published...

Area of Science:

• Hepatology
• Oncology
• Pharmacology

Background:

• Ado-trastuzumab emtansine (T-DM1) is a targeted therapy for HER2-positive breast cancer.
• T-DM1 combines trastuzumab with DM1, a microtubule inhibitor.
• Portal hypertension can occur with T-DM1 treatment, even without cirrhosis, consistent with porto-sinusoidal vascular disorder (PSVD).

Purpose of the Study:

• To summarize the clinical, biochemical, imaging, histological, therapeutic, and prognostic features of T-DM1-associated PSVD.
• To identify key diagnostic and management considerations for this rare complication.

Main Methods:

• Systematic literature search of PubMed and Web of Science for T-DM1-associated PSVD cases.
• Inclusion criteria: T-DM1 exposure and liver biopsy confirming PSVD criteria.
• Exclusion criteria: Lack of liver biopsy or features of overt cirrhosis.

Main Results:

• Eight patients from seven articles were identified; all were female, with a mean age of 60.38 years.
• PSVD diagnosis occurred 6-30 months after T-DM1 initiation; symptoms appeared around 18.3 months.
• Common findings included thrombocytopenia, splenomegaly, mild liver enzyme elevations, and histological evidence of disorganized hepatic plates with nodular regeneration.

Conclusions:

• T-DM1-associated PSVD is a rare but significant complication presenting months after treatment initiation.
• Clinical presentation includes thrombocytopenia, splenomegaly, gastrointestinal bleeding, or mild liver abnormalities without overt cirrhosis.
• Early recognition and potential discontinuation or modification of T-DM1 may lead to clinical improvement.