Case report: Response to regional melphalan via limb infusion and systemic PD1 blockade in recurrent myxofibrosarcoma: A report of 2 cases Journal Article


Authors: Bartlett, E. K.; D’Angelo, S. P.; Kelly, C. M.; Siegelbaum, R. H.; Fisher, C.; Antonescu, C. R.; Ariyan, C. E.
Article Title: Case report: Response to regional melphalan via limb infusion and systemic PD1 blockade in recurrent myxofibrosarcoma: A report of 2 cases
Abstract: Treatment options for patients with advanced sarcoma remain limited. Promising responses to checkpoint inhibition have been observed, but responses to single-agent PD-1 inhibition are rare. We report on two patients with multiply recurrent myxofibrosarcoma treated with the combination of regionally administered melphalan (via isolated limb infusion) and pembrolizumab. Both patients had recurrent disease after multiple surgical resections and radiation. Analysis of primary tumors demonstrated microsatellite stable tumors with few mutations. After combination treatment, one patient had a significant partial response of 6 months duration, the second patient had a complete response of 2 years duration. Post treatment biopsies demonstrated immune infiltration into the tumor. These promising responses in patients with multiply recurrent myxofibrosarcoma have prompted the development of an investigator-initiated clinical trial to formally study the combination of regional melphalan and pembrolizumab in a systematic fashion (NCT04332874). © Copyright © 2021 Bartlett, D’Angelo, Kelly, Siegelbaum, Fisher, Antonescu and Ariyan.
Keywords: immunohistochemistry; clinical article; human tissue; treatment response; aged; limb salvage; gene mutation; human cell; gene deletion; clinical trial; case report; follow up; genetic analysis; computer assisted tomography; cell infiltration; melphalan; microsatellite dna; protein p53; biopsy; sarcoma; tumor suppressor gene; immunotherapy; microsatellite instability; isolated limb infusion; single drug dose; recurrent disease; inguinal lymph node; cyclin dependent kinase inhibitor 2a; hypothyroidism; lymphocytic infiltration; cd3+ t lymphocyte; cd163 antigen; programmed death 1 receptor; fibromyxosarcoma; myxofibrosarcoma; immune checkpoint inhibitor; human; female; article; pembrolizumab; infusion therapy; transcriptional regulator atrx; lower limb; undifferentiated pleomorphic sarcoma (ups)
Journal Title: Frontiers in Oncology
Volume: 11
ISSN: 2234-943X
Publisher: Frontiers Media S.A.  
Date Published: 2021-10-15
Start Page: 725484
Language: English
DOI: 10.3389/fonc.2021.725484
PROVIDER: scopus
PMCID: PMC8554327
PUBMED: 34722269
DOI/URL:
Notes: Article -- Export Date: 1 December 2021 -- Source: Scopus
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MSK Authors
  1. Cristina R Antonescu
    901 Antonescu
  2. Sandra Pierina D'Angelo
    254 D'Angelo
  3. Charlotte Eielson Ariyan
    154 Ariyan
  4. Charles H Fisher
    8 Fisher
  5. Ciara Marie Kelly
    91 Kelly