The goal of this prospective phase 2 study is to assess the efficacy and safety of intestinal or multivisceral transplantation for participants with PMP not amenable to other curative-intent treatments. Participants will undergo intestinal/multivisceral transplantation. Participants will be followed for 12 months to assess efficacy and safety.
Pseudomyxoma peritonei (PMP) is a rare clinical entity (approximately 2-4 cases per million people) characterized by extensive dissemination of mucinous ascites in the abdominal cavity. Relentless accumulation of mucin causes progressive abdominal distention, intestinal obstruction, malnutrition, cachexia, and ultimately death. As a rare disease, diagnosis is often late, and usually occurs when the disease is in a clinically advanced stage. The prognosis of PMP has been dramatically improved by the introduction of cytoreductive surgery (CRS) and hyperthermic intraperitoneal chemotherapy (HIPEC). While outcomes are favorable for participants with disease amenable to CRS+HIPEC, the therapeutic options for participants with unresectable PMP are limited. Intestinal transplantation represents a therapeutic option in participants with unresectable PMP. Overall survival has been shown to improve with participants with unresectable PMP during an Oxford Transplant Center study. The goal of this study is to corroborate the Oxford results on an American cohort.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Enrolled participants will enter the active transplant waiting list within one month of signing informed consent for study participation. Participants can be listed for: * Isolated small bowel transplant (SBT): transplantation of the donor small intestine * Modified multivisceral tran I lant (MMVT): transplantation of the donor pancreas and small intestine, with or without stomach * Multivisceral transplant (MVT): transplantation of the donor pancreas, small intestine, and liver, with or without stomach
A post-transplant, steroid-free immunosuppressive regimen will be utilized and will include Alemtuzumab as Antibody Induction Therapy. Participants will be administered two doses of Alemtuzumab (30 mg IV) on days 0 and 1.
Cleveland Clinic Digestive Disease & Surgery Institute (DDSI), Case Comprehensive Cancer Center
Cleveland, Ohio, United States
Overall Rate of Survival
To determine overall 12-month survival after intestinal or multivisceral transplantation in participants with unresectable PMP.
Time frame: 12 months post operative
Overall Rate of Morbidity
Determine the morbidity of intestinal or multivisceral transplantation for participants with unresectable at 90 days by monitoring severe adverse events as classified according to Clavien-Dindo grade and comprehensive complication index.
Time frame: 90 days post operative
Overall Rate of Morbidity
Determine the morbidity of intestinal or multivisceral transplantation for participants with unresectable at 12 months by monitoring severe adverse events as classified according to Clavien-Dindo grade and comprehensive complication index.
Time frame: 12 months post operative
Overall Rate of Mortality
Determine cancer-related and transplant-related mortality after intestinal or multivisceral transplantation in participants with unresectable PMP.
Time frame: 12 months post operative
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
A post-transplant, steroid-free immunosuppressive regimen will be utilized and will include Tacrolimus for maintenance. Participants will have Tacrolimus for the first 3 months. Dosing of Tacrolimus will depend on participant target level, starting with 0.05 mg/Kg bid.
A post-transplant, steroid-free immunosuppressive regimen will be utilized and will include Sirolimus for maintenance. Participants will have Sirolimus after 3 months of Tacrolimus. Dosing of Sirolimus will depend on participant target level, starting with 2 mg od.