A Pilot Study of Alpha-1-Antitrypsin (AAT) in Steroid Refractory Acute Graft vs Host Disease

Launched by UNIVERSITY OF MICHIGAN ROGEL CANCER CENTER · Oct 3, 2012

Keywords

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Gender

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Eligibility criteria

• Inclusion Criteria:
• • Age \>18 years
• * Patients must have clinical evidence\* of steroid-refractory acute Graft vs Host Disease (any organ) defined as one of the following:
• • No change or progression in the stage of skin GvHD after at least 1 week of 2mg/kg/day methylprednisolone (or po equivalent)
• • lack of response of visceral (liver, GI) GvHD despite treatment with 2mg/kg/day methylprednisolone for at least 72h.
• • progression of visceral GvHD despite treatment with 2mg/kg/day methylprednisolone for at least 48h
• • visceral GvHD progressing to stage 4 after 24h of 2mg/kg/d methylprednisolone
• • Patients with protracted acute GvHD who have not responded to at least 0.5mg/kg/d of prednisone are considered eligible.
• • Ability to understand and the willingness to sign a written informed consent document.
• • \* As GvHD is a clinical diagnosis, and patients will have already been initiated on steroid therapy at the discretion of the attending physician, tissue confirmation of refractory GvHD by biopsy is not required for entry to this study. It is anticipated that most, but not all, patients will have undergone tissue confirmation of the initial diagnosis of GvHD; however lack of tissue confirmation for this clinical syndrome is not exclusionary.
• Exclusion Criteria:
• • As patients with steroid refractory acute GvHD are quite ill with multiple abnormal labs and organ dysfunction, there are no explicit laboratory values or degree of organ dysfunction that specifically preclude enrollment on this study. Baseline lab studies will be obtained and followed throughout this trial as the standard of care for patients with GvHD.
• • Pregnancy or Nursing Mother
• • Vasopressor requirement
• • Patients may not be receiving any other investigational agents for the treatment of GvHD at time of study entry or at any time while on study or be on another investigational agent that can impact on the primary clinical outcome analyses or has known pharmacodynamics or pharmacokinetic effects on AAT.
• • Patients with known antibodies to IgA