Clinical Trial: The Use of Etanercept Enbrel as Sole Treatment for Grade I Acute Graft Versus Host Disease

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: The Use of Etanercept (Enbrel) as Sole Treatment for Grade I Acute Graft Versus Host Disease

Brief Summary:

This is a clinical trial to see if treatment with etanercept for early skin graft-versus-host disease (GVHD) can effectively treat and prevent progression of the disease without using high dose steroids.

GVHD is a common complication following a bone marrow transplant from another donor. GVHD occurs after transplant, when the donor's blood cells (called lymphocytes) recognize parts of your body, such as the skin, as foreign. A certain chemical, called Tumor Necrosis Factor, or TNF, also causes damage to the skin. The main effect on the skin is a red rash, when the skin GVHD is mild, but in more severe forms the skin can blister.

We have been studying GVHD at the University of Michigan for the past decade. We know that high levels of TNF makes GVHD worse. Our research has shown that adding an anti-TNF drug (called etanercept or Enbrel®) to the standard GVHD treatment of high dose steroids leads to improvement in the GVHD in twice as many patients compared to when steroids alone are used. It is now standard practice at the University of Michigan and many other centers to treat GVHD with both steroids and etanercept.

The management of early skin GVHD for most patients involves treatment with steroids, given both as a cream and by either the mouth (in pills) or IV. Early skin GVHD is also called grade I GVHD, which means the skin rash covers less than half of the body. Steroid treatment can be effective; however, it also causes many complications such as an increased risk of infection, weight gain, stomach ulcers, muscle weakness and bone damage, among many others. We have developed this study to test whether starting treatment with etanercept and steroid creams alone can treat the GVHD without requiring the use of high dose steroids. The goal is to avoid the complications

Detailed Summary:
Sponsor: University of Michigan Cancer Center

Current Primary Outcome: The Percentage of Patients Who Progress Within 28 Days of Initiation of Etanercept Treatment [ Time Frame: 28 days ]

We hypothesized that treatment of grade 1 acute GVHD (Graft Versus Host Disease) with etanercept would reduce the proportion of patients who progressed to grade 2 to 4 acute GVHD within 4 weeks of diagnosis from 58%, historically observed at our institution, to 38%.


Original Primary Outcome: To determine with statistical certainty that treatment with etanercept alone for Grade I (stage 1-2 skin only) acute GVHD will reduce the percentage of patients who progress within 28 days of initiation of etanercept treatment, from 58% to 38%. [ Time Frame: 30 days ]

Current Secondary Outcome: The Number of Patients in Complete Remission (CR) at Four Weeks. [ Time Frame: 28 days ]

Estimate the proportion of patients in complete remission (CR) at four weeks who remain alive and never require additional therapy four weeks after the last dose of etanercept.

Complete remission is defined as the resolution of all manifestations of GVHD (Graft Versus Host Disease) within the first four weeks of treatment. All organs must have a Grade 0.



Original Secondary Outcome: Estimate proportion patients in complete remission four weeks survival, never require additional therapy four weeks after the last dose of etanercept. Estimate the proportion of patients who respond to etanercept [ Time Frame: 30 days ]

Information By: University of Michigan Cancer Center

Dates:
Date Received: July 28, 2008
Date Started: May 2008
Date Completion:
Last Updated: December 4, 2015
Last Verified: December 2015