Metabolic Efficiency of Combined Pancreatic Islet and Lung Transplant for the Treatment of End-Stage Cystic Fibrosis (PIM)

University Hospital, Strasbourg, France

Status and phase

Completed

Phase 2

Phase 1

Conditions

Diabetes Related Cystic Fibrosis

Insulin-dependent Diabetes

Cystic Fibrosis

Treatments

Procedure: Combined pancreatic islet and lung transplantation

Study type

Interventional

Funder types

Other

Identifiers

NCT01548729

4790

Details and patient eligibility

About

Patients with end-stage cystic fibrosis (CF) and severe CF-related diabetes (CFRD) may benefit from combined lung-pancreatic islet transplantation. A recent case series showed that combined bilateral lung and pancreatic islet transplantation is a viable therapeutic option for patients with end-stage CF and CFRD. The use of different organs from a single donor may lead to reduced immunogenicity. As the prevalence of CFRD has increased dramatically with the improved life expectancy of patients with CF, islet transplantation should be considered at the end-stage CF. By restoring metabolic control, the investigators hypothesize that islet transplantation may improve the management of CF patients undergoing lung transplant and decrease the complication rate in the early postoperative period.

Enrollment

14 patients

Sex

All

Ages

14+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion criteria:

Patient with cystic fibrosis
Patient able to respect the protocol procedures
Patient with end-stage respiratory insufficiency indicating a lung transplant
Clinical history of cystic fibrosis related diabetes and/or insulin-dependent diabetes, with no residual insulin secretion (C-peptide < 0,5 ng/mL) and/or no response to IV glucagon stimulation: [peak stimulated C-peptide (T6min)/basal plasma C-peptide(T0)] < 2. The absence of insulin secretion will be verified 2 times before inclusion
Evolution of diabetes for over 3 years
Patient whose glycaemic control obtained with insulin therapy is not satisfactory and could significantly alter quality of life (HbA1c > 7% and/or MAGE index > 1,25). This situation is assessed by a diabetologist.
Social Security membership or benefit from Social Welfare
Patient who received the results of the medical evaluation required

Non-inclusion criteria:

Patient with contra-indication for undergo a lung transplant
Patient with an indication of heart, liver or kidney transplantation
Patient for which poor therapeutic compliance is expected
Patient under oral antidiabetic drug
In women of childbearing potential: pregnancy test (urine and / or blood) positive, lactation, or unwilling to use effective contraception for the duration of the study until 3 months after the end . Men: procreation project during the study period up to 3 months after its end, or unwilling to use effective contraception patient.
Active infection, including hepatitis B, hepatitis C, HIV
Any history of malignancy within the past 5 years, with the exception of cutaneous basal cell carcinomas completely resected. The history of breast or melanoma cancers are an absolute contraindication
Alcoholic intoxication or drug addiction
Anemia (hemoglobin Hb <10g / dL in women and Hb <11 g / dL in men), lymphopenia (<1000 / uL), neutropenia (<1500 / uL) or thrombocytopenia (<100,000 / uL).
Persistent elevated liver enzymes at baseline
Portal hypertension identified by oesophageal varice and/or hypersplenism (platelets < 120 000 /mm3) or Child-Pugh score > 6.
Use of a medical treatment under investigation within 4 weeks before inclusion
All medical situation assessed by an investigator which could interfere with the good management of the project
Patient restricted of freedom or unable to give his consent
Patient has been included in another study that could interfere with the results of the study
Contraindications to the use of experimental drugs (Cellcept®, prednisone, Prograf, prednisolone, methylprednisolone, and pancreatic islets)