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Background <p>For patients with unresectable pseudomyxoma peritonei (PMP), intra-tumoral administration of a mucolytic drug combination comprising bromelain and acetylcysteine (BromAc) reduced mucinous tumor volume in phase 1/2 non-U.S. trials. This report describes the initial outcomes of an expanded access BromAc treatment program at a single U.S. center.</p> Methods <p>Eligible patients with progressive unresectable PMP underwent image-guided placement of percutaneous catheters in one or more mucinous tumors. BromAc was administered via the catheters, and after a 24&#xa0;h dwell period, the dissolved mucus was aspirated. This treatment was repeated up to six times per tumor. Changes in tumor volume, post-treatment complications, and patient-reported quality-of-life (QOL) symptoms were recorded.</p> Results <p>Ten appendiceal PMP patients with 23 individual tumors underwent 107 intra-tumoral BromAc treatments (median, 4 treatments; interquartile range [IQR], 4–6 treatments per tumor). The median pre-treatment tumor volume by imaging was 728&#xa0;ml (IQR, 350–2104&#xa0;ml). Among eight patients that completed post-treatment imaging, 14 (82.4 %) of 17 tumors exhibited a reduction in tumor volume (median tumor volume reduction, 48.6 %; IQR, 14.9–75.4 %). The median cumulative volume of dissolved mucus aspirated after treatment completion was 370&#xa0;ml (IQR, 155–756&#xa0;ml). There were 6 CTCAE grade 1, 20 grade 2, and 6 grade 3 adverse events. Two patients died within 40-days after protocol initiation from disease progression, clinical deterioration, or both. Seven patients completed post-treatment QOL surveys, with four patients (57.1 %) reporting improved symptoms.</p> Conclusions <p>Repeated intra-tumoral BromAc administration decreases mucinous tumor volume and may improve short-term QOL symptoms. Tumor characteristics influence treatment response, whereas patient factors impact safety and tolerability of therapy.</p>

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Intra-Tumoral Mucolytic Therapy for Unresectable Pseudomyxoma Peritonei: Results of a Single-Center Expanded Access Program

  • 期刊论文

  • 发表日期：

    2026年1月3日

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  • Shannon Altpeter,
  • Michael M. Wach,
  • Erika Giran,
  • Joshua Derby,
  • Scott Beasley,
  • James F. Pingpank,
  • Melanie Ongchin,
  • Haroon A Choudry

摘要

Background

For patients with unresectable pseudomyxoma peritonei (PMP), intra-tumoral administration of a mucolytic drug combination comprising bromelain and acetylcysteine (BromAc) reduced mucinous tumor volume in phase 1/2 non-U.S. trials. This report describes the initial outcomes of an expanded access BromAc treatment program at a single U.S. center.

Methods

Eligible patients with progressive unresectable PMP underwent image-guided placement of percutaneous catheters in one or more mucinous tumors. BromAc was administered via the catheters, and after a 24 h dwell period, the dissolved mucus was aspirated. This treatment was repeated up to six times per tumor. Changes in tumor volume, post-treatment complications, and patient-reported quality-of-life (QOL) symptoms were recorded.

Results

Ten appendiceal PMP patients with 23 individual tumors underwent 107 intra-tumoral BromAc treatments (median, 4 treatments; interquartile range [IQR], 4–6 treatments per tumor). The median pre-treatment tumor volume by imaging was 728 ml (IQR, 350–2104 ml). Among eight patients that completed post-treatment imaging, 14 (82.4 %) of 17 tumors exhibited a reduction in tumor volume (median tumor volume reduction, 48.6 %; IQR, 14.9–75.4 %). The median cumulative volume of dissolved mucus aspirated after treatment completion was 370 ml (IQR, 155–756 ml). There were 6 CTCAE grade 1, 20 grade 2, and 6 grade 3 adverse events. Two patients died within 40-days after protocol initiation from disease progression, clinical deterioration, or both. Seven patients completed post-treatment QOL surveys, with four patients (57.1 %) reporting improved symptoms.

Conclusions

Repeated intra-tumoral BromAc administration decreases mucinous tumor volume and may improve short-term QOL symptoms. Tumor characteristics influence treatment response, whereas patient factors impact safety and tolerability of therapy.