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Weekly Subcutaneous Efalizumab for the Treatment of Steroid Refractory Graft-Versus-Host Disease of the Skin and Liver

18 Years
Not Enrolling
Graft Versus Host Disease

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Trial Information

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
disease (GVHD).

- 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.

Inclusion Criteria


- Diagnosis of acute or chronic cutaneous graft-versus-host disease (GVHD), as
evidenced by an erythematous maculopapular rash which is felt to be clinically
consistent with GVHD

- Pathologic findings from skin biopsy consistent with GVHD

- Sclerodermatous skin changes may be present but will not by themselves be
considered adequate for study enrollment

- Patients with concurrent hepatic GVHD are eligible

- Patients with liver dysfunction are encouraged but not required to undergo
hepatic biopsy in order to document that liver injury is the result of GVHD

- Patients with a pretreatment serum bilirubin ≥ 2.0 mg/dL and biopsy-confirmed
cutaneous GVHD will be assumed to demonstrate hepatic GVHD if no other cause for
the bilirubin elevation can be identified

- Underwent allogeneic hematopoietic stem cell transplantation (peripheral blood stem
cells and/or bone marrow, regardless of the degree of HLA matching) ≥ 30 days prior
to study enrollment

- Steroid refractory disease, defined by 1 of the following criteria:

- Worsening skin or liver disease despite 1 week of treatment with the equivalent
of 1 mg/kg of methylprednisolone

- Failed to achieve a 50% reduction in the body surface area involved by GVHD or a
50% reduction in the total serum bilirubin after 4 weeks of treatment with the
equivalent of at least 0.5 mg/kg of methylprednisolone

- Requires the equivalent of at least 0.5 mg/kg of methylprednisolone to maintain
a response after 8 weeks of steroid therapy

- Progression of cutaneous or hepatic GVHD after a prior history of treatment with
at least 8 weeks of corticosteroids now requiring the reintroduction of
corticosteroids (the equivalent of greater than 10 mg/day of methylprednisolone)

- Not improving or progressing on alternative immunosuppressive agents after prior
steroid refractoriness had been established


- Not pregnant or nursing

- Fertile patients must use effective contraception

- Absolute neutrophil count (ANC) > 1,000/μL

- Platelet count ≥ 20,000/μL

- Serum creatinine ≤ 3.0 mg/dL

- No HIV infection


- See Disease Characteristics

- At least 2 terminal half-lives since prior and no concurrent infliximab, daclizumab,
etanercept, rituximab, antithymocyte globulin (ATG), or denileukin diftitox

Type of Study:


Study Design:

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Supportive Care

Outcome Measure:

Number of subjects experiencing adverse events

Outcome Description:

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.

Outcome Time Frame:

120 days

Safety Issue:


Principal Investigator

Thomas C. Shea, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

UNC Lineberger Comprehensive Cancer Center


United States: Institutional Review Board

Study ID:

LCCC 0605



Start Date:

May 2007

Completion Date:

October 2008

Related Keywords:

  • Graft Versus Host Disease
  • Graft Versus Host Disease
  • GVHD
  • Efalizumab
  • Raptiva
  • Steroid
  • Refractory
  • Skin
  • Liver
  • Lineberger
  • University of North Carolina
  • Graft vs Host Disease



Lineberger Comprehensive Cancer Center at University of North Carolina - Chapel HillChapel Hill, North Carolina  27599-7570