Weekly Subcutaneous Efalizumab for the Treatment of Steroid Refractory Graft-Versus-Host Disease of the Skin and Liver
- Assess the general safety of efalizumab in patients with cutaneous graft-vs-host
- Study the feasibility of digital imaging to objectively quantify cutaneous GVHD.
- Evaluate the feasibility of serial skin biopsies to monitor disease response to
efalizumab in patients with cutaneous GVHD.
- Assess the overall complete response rate in patients treated with this drug.
- Assess the overall cutaneous response rate (complete cutaneous response rate and
partial cutaneous response rate) in patients treated with this drug.
- Assess the overall hepatic response rate (complete hepatic response rate and partial
hepatic response rate) in patients treated with this drug.
- Assess the duration of any responses observed.
- Assess the effect of this drug on overall patient survival.
- Use the preliminary efficacy and toxicity data collected in this small exploratory
study to decide on the appropriateness of a larger, subsequent phase II trial to more
formally assess toxicity and efficacy of this drug in this patient population.
- Collect pharmacokinetic data on this drug in these patients.
OUTLINE: Patients receive efalizumab subcutaneously once weekly for 8 weeks (total of 8
Digital photographs of body regions are taken for determination of disease involved body
surface area. Skin biopsies are obtained before and after treatment and analyzed for LFA-1,
ICAM-1, CD4, CD8, and possibly CD20 by immunohistochemistry.
After completion of study therapy, patients are followed at 1 and 9 weeks.
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Supportive Care
Number of subjects experiencing adverse events
The primary objective of this exploratory study is to evaluate the general tolerability of efalizumab in patients suffering from steroid refractory GVHD Subjects will be evaluated for drug toxicity each visit. Toxicity will be graded using the NCTCAE common criteria.
Thomas C. Shea, MD
UNC Lineberger Comprehensive Cancer Center
United States: Institutional Review Board
|Lineberger Comprehensive Cancer Center at University of North Carolina - Chapel Hill||Chapel Hill, North Carolina 27599-7570|