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Early Results of a Phase I Trial Using an Adipose-Derived Mesenchymal Stem Cell-Coated Fistula Plug for the Treatment of Transsphincteric Cryptoglandular Fistulas

Dozois, Eric J., M.D.1; Lightner, Amy L., M.D.1; Mathis, Kellie L., M.D.1; Chua, Heidi K., M.D.1; Kelley, Scott R., M.D.1; Fletcher, Joel G., M.D.2; Dietz, Allan B., Ph.D.3; Friton, Jessica J., B.S., M.A.4; Butler, Greg W., B.S.3; Faubion, William A., M.D.4

Diseases of the Colon & Rectum: May 2019 - Volume 62 - Issue 5 - p 615–622
doi: 10.1097/DCR.0000000000001333
Original Contribution: Anorectal
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BACKGROUND: Management of transsphincteric cryptoglandular fistulas remains a challenging problem and the optimal surgical approach remains elusive. Mesenchymal stem cells, increasingly being utilized for perianal Crohn’s disease, offer a novel therapy to treat cryptoglandular fistulas.

OBJECTIVES: This study aimed to determine safety and feasibility of using an autologous mesenchymal stem cell-coated fistula plug in patients with transsphincteric cryptoglandular fistulas.

DESIGN: This study is a phase I clinical trial.

SETTING: This study was conducted at a tertiary academic medical center.

PATIENTS: Adult (>18 years) male and female patients with transsphincteric cryptoglandular fistulas were selected.

MAIN OUTCOMES MEASURES: The primary outcomes measured were the safety, feasibility, and efficacy of a mesenchymal stem cell-coated fistula plug in patients with transsphincteric fistulas.

RESULTS: Fifteen patients (8 women, mean age 39.8 years) with a single-tract transsphincteric fistula received a mesenchymal stem cell-loaded fistula plug and were followed for 6 months. Duration of disease at the time of study enrollment was a median of 3.0 years (range, 1–13 years) with a median of 3.5 (range, 1–20) prior surgical interventions. Adverse events included 1 plug extrusion, 1 abdominal wall seroma, 3 perianal abscesses requiring drainage, and 1 patient with perianal cellulitis. There were no serious adverse events. At 6 months, 3 patients had complete clinical healing, 8 had partial healing, and 4 patients showed no clinical improvement. Radiographic improvement was seen in 11 of 15 patients.

LIMITATIONS: This study was limited by the small cohort and short follow-up.

CONCLUSIONS: Autologous mesenchymal stem cell-coated fistula plug treatment of transsphincteric cryptoglandular fistulas was safe and feasible and resulted in complete or partial healing in a majority of patients. See Video Abstract at http://links.lww.com/DCR/A897.

1 Division of Colon and Rectal Surgery, Mayo Clinic, Rochester, Minnesota

2 Department of Radiology, Mayo Clinic, Rochester, Minnesota

3 Division of Transfusion Medicine, Mayo Clinic, Rochester, Minnesota

4 Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, Minnesota

Funding/Support: This work was supported by the Mayo Clinic Discovery Translation Award.

Financial Disclosure: Dr Faubion is a Consultant and on the Advisory Board for AbbVie, a consultant for Boehringer Ingelheim Pharma and Celgene, Consultant and Advisory Board member for Janssen, and a consultant for Robarts, Takeda, and MediBeacon. Dr Lightner is a consultant for Takeda. Dr Dietz is an inventor of technology used as a tool in this research; the technology has been licensed to a commercial entity (PLTMax; Mill Creek LifeScienes). Dr Dietz and the Mayo Clinic have equity in the company and have contractual rights to receive royalties from the licensing of this technology. Greg W. Butler is an inventor of technology used as a tool in this research; the technology has been licensed to a commercial entity (PLTMax; Mill Creek LifeScienes). Greg Butler and the Mayo Clinic have equity in the company and have contractual rights to receive royalties from the licensing of this technology. Joel G. Fletcher is a consultant for Medtronics and received a grant to institution from Siemens Healthineers.

Podium presentation at the meeting of The American Society of Colon and Rectal Surgeons, Nashville, TN, May 19 to 23, 2018.

Correspondence: Eric J. Dozois, M.D., 200 1st St SW, Rochester, MN 55905. E-mail: dozois.eric@mayo.edu

© 2019 The American Society of Colon and Rectal Surgeons