Journal Publication

Small pilot links disitamab vedotin-toripalimab-radiotherapy to bladder preservation

A 10-patient pilot combining an HER2-directed antibody-drug conjugate, an anti-PD-1 antibody, and radiotherapy reported 90% bladder preservation, a hypothesis for larger testing rather than a practice-changing result.

A pilot study combining disitamab vedotin, toripalimab, and concurrent radiotherapy in HER2-positive invasive bladder cancer reported its safety and efficacy results.

By AppliedXLJuly 16, 2026

Executive Summary

A small single-arm study tested whether adding an HER2-directed antibody-drug conjugate and an anti-PD-1 antibody to radiotherapy could let patients with HER2-positive invasive bladder cancer avoid having their bladder removed.
Most patients who completed the regimen kept their bladder and remained free of disease progression at extended follow-up, with adverse events concentrated in low-grade radiation-related toxicity and one serious blood-cell count drop.
The cohort is too small to isolate which component of the three-drug-plus-radiation combination drives the benefit, and two patients developed radiation-related bladder toxicity more than a year and a half after treatment, a signal that needs tracking in a larger group before it changes practice.

The stake

Patients with invasive bladder cancer who qualify for radical cystectomy but decline the surgery have limited standardized alternatives that preserve the bladder while controlling the disease. This pilot tested a bladder-preserving regimen built around disitamab vedotin, an antibody-drug conjugate targeting HER2, paired with the anti-PD-1 antibody toripalimab and concurrent radiotherapy, in patients whose tumors were confirmed HER2-positive after resection. The authors describe it as a novel strategy for comprehensive bladder preservation in this population, independent of PD-L1 status. Safety

How it was done

The study enrolled 10 patients between January 2023 and September 2025 who met criteria for radical cystectomy but refused it. All patients first underwent maximal transurethral resection of the bladder tumor, with or without neoadjuvant disitamab vedotin plus toripalimab, after pathology confirmed HER2 positivity. Radiotherapy was then given concurrently with continued disitamab vedotin and toripalimab. Patients were followed through March 2026 or until death, with a median follow-up of 17.2 months ranging from 6 to 31 months. There was no comparator arm; this was a single-arm pilot cohort. Safety

The results

Nine of 10 patients completed the full treatment regimen, with one patient stopping radiotherapy early at 46 Gy for personal reasons. The bladder preservation success rate was 90% and the event-free survival rate was 80% at last follow-up. The patient who did not complete radiotherapy had a T3 recurrence at 5 months, underwent salvage cystectomy, and later died of intestinal obstruction complications 13 months afterward. A separate patient had a T1 recurrence at 7 months and continued bladder-preserving treatment for 31 months. Safety

Safety profile

One of 10 patients had a grade 3 or higher adverse event, leukopenia (low white blood cell count). Grade 1-2 radiation cystitis occurred in 9 of 10 patients and hand-foot numbness in 2 of 10. Two patients developed hemorrhagic cystitis from radiotherapy at 20 and 22 months after treatment, a late-onset toxicity that emerged well beyond the acute treatment window. With a cohort this size, these are raw counts rather than stable rate estimates, but the delayed cystitis cases mark a toxicity pattern that longer follow-up in more patients would need to characterize. Safety

What it changes

The authors frame the combination as a potential bladder-preserving option for HER2-positive invasive bladder cancer patients who decline cystectomy, regardless of PD-L1 status, while stating explicitly that the finding requires validation in a larger cohort. No prior trial data on this exact three-component combination in this population is cited in the results. The pilot establishes feasibility and a preliminary signal in a resection-eligible, surgery-averse population; it does not establish comparative efficacy against existing bladder-preserving approaches or against cystectomy itself. Safety

This analysis was produced using AI-assisted reporting systems, AppliedXL data, and official public records. These systems undergo editorial review, quality checks, and regular audits by human experts. Errors may still occur, as with any automated system. Always consult the linked primary sources. Read our AI Editorial Policy.