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Blood, Vol. 93 No. 1 (January 1), 1999:
pp. 66-70
Etretinate Therapy for Refractory Sclerodermatous Chronic
Graft-Versus-Host Disease
D.C. Marcellus,
V.L. Altomonte,
E.R. Farmer,
T.D. Horn,
C.S. Freemer,
J. Grant, and
G.B. Vogelsang
From the Departments of Oncology, of Dermatology and Pathology, and
of Rehabilitation Medicine, Johns Hopkins University, Baltimore, MD;
the Department of Dermatology, Indiana University, Indianapolis; and
the Department of Dermatology, University of Arkansas, Little Rock.
Chronic graft-versus-host disease (GVHD) is the most common late
complication of allogeneic bone marrow transplantation (BMT). The
sclerodermatous form of the disease is often refractory to standard
treatment modalities. Based on reports of response to etretinate, a
synthetic retinoid, among patients with scleroderma, we have added
etretinate to the treatment regimen of 32 patients with refractory
sclerodermatous chronic GVHD. This case series is comprised mainly of
patients who had chronic GVHD of long duration (median of 30 months
before the initiation of etretinate). Most had failed to respond to
three or more agents before etretinate treatment was started. Clinical
response was assessed after 3 months of therapy. Five patients did not
complete a 3-month trial. Among the 27 patients evaluable for response,
20 showed improvement including softening of the skin, flattening of
cutaneous lesions, increased range of motion, and improved
performance status. Four showed no response after 3 months of
therapy and 3 had progression of their sclerosis. Overall, etretinate
has been fairly well tolerated in our patients, with skin breakdown
and/or ulceration leading to its discontinuation in 6 patients.
We believe the results in our patients are encouraging and suggest that
further evaluation of etretinate in the treatment of sclerodermatous
chronic GVHD is warranted.
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