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Imatinib mesylate for the treatment of steroid-refractory sclerotic-type cutaneous chronic graft-versus-host disease.

Publication ,  Journal Article
Baird, K; Comis, LE; Joe, GO; Steinberg, SM; Hakim, FT; Rose, JJ; Mitchell, SA; Pavletic, SZ; Figg, WD; Yao, L; Flanders, KC; Takebe, N ...
Published in: Biol Blood Marrow Transplant
June 2015

Sclerotic skin manifestations of chronic graft-versus-host disease (ScGVHD) lead to significant morbidity, including functional disability from joint range of motion (ROM) restriction. No superior second-line therapy has been established for steroid-refractory disease. Imatinib mesylate is a multikinase inhibitor of several signaling pathways implicated in skin fibrosis with in vitro antifibrotic activity. We performed an open-label pilot phase II trial of imatinib in children and adults with corticosteroid-refractory ScGVHD. Twenty patients were enrolled in a 6-month trial. Eight received a standard dose (adult, 400 mg daily; children, 260 mg/m(2) daily). Because of poor tolerability, 12 additional patients underwent a dose escalation regimen (adult, 100 mg daily initial dose up to 200 mg daily maximum; children, initial dose 65 mg/m(2) daily up to 130 mg/m(2) daily). Fourteen patients were assessable for primary response, improvement in joint ROM deficit, at 6 months. Primary outcome criteria for partial response was met in 5 of 14 (36%), stable disease in 7 of 14 (50%), and progressive disease in 2 of 14 (14%) patients. Eleven patients (79%), including 5 with partial response and 6 with stable disease, demonstrated a positive gain in ROM (range of 3% to 94% improvement in deficit). Of 13 patients with measurable changes at 6 months, the average improvement in ROM deficit was 24.2% (interquartile range, 15.5% to 30.5%; P = .011). This trial is registered at http://clinicaltrials.gov as NCT007020689.

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Published In

Biol Blood Marrow Transplant

DOI

EISSN

1523-6536

Publication Date

June 2015

Volume

21

Issue

6

Start / End Page

1083 / 1090

Location

United States

Related Subject Headings

  • Transplantation, Homologous
  • Tacrolimus
  • Skin Diseases
  • Recurrence
  • Range of Motion, Articular
  • Prednisone
  • Pilot Projects
  • Mycophenolic Acid
  • Middle Aged
  • Male
 

Citation

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Baird, K., Comis, L. E., Joe, G. O., Steinberg, S. M., Hakim, F. T., Rose, J. J., … Cowen, E. W. (2015). Imatinib mesylate for the treatment of steroid-refractory sclerotic-type cutaneous chronic graft-versus-host disease. Biol Blood Marrow Transplant, 21(6), 1083–1090. https://doi.org/10.1016/j.bbmt.2015.03.006
Baird, Kristin, Leora E. Comis, Galen O. Joe, Seth M. Steinberg, Fran T. Hakim, Jeremy J. Rose, Sandra A. Mitchell, et al. “Imatinib mesylate for the treatment of steroid-refractory sclerotic-type cutaneous chronic graft-versus-host disease.Biol Blood Marrow Transplant 21, no. 6 (June 2015): 1083–90. https://doi.org/10.1016/j.bbmt.2015.03.006.
Baird K, Comis LE, Joe GO, Steinberg SM, Hakim FT, Rose JJ, et al. Imatinib mesylate for the treatment of steroid-refractory sclerotic-type cutaneous chronic graft-versus-host disease. Biol Blood Marrow Transplant. 2015 Jun;21(6):1083–90.
Baird, Kristin, et al. “Imatinib mesylate for the treatment of steroid-refractory sclerotic-type cutaneous chronic graft-versus-host disease.Biol Blood Marrow Transplant, vol. 21, no. 6, June 2015, pp. 1083–90. Pubmed, doi:10.1016/j.bbmt.2015.03.006.
Baird K, Comis LE, Joe GO, Steinberg SM, Hakim FT, Rose JJ, Mitchell SA, Pavletic SZ, Figg WD, Yao L, Flanders KC, Takebe N, Sarantopoulos S, Booher S, Cowen EW. Imatinib mesylate for the treatment of steroid-refractory sclerotic-type cutaneous chronic graft-versus-host disease. Biol Blood Marrow Transplant. 2015 Jun;21(6):1083–1090.
Journal cover image

Published In

Biol Blood Marrow Transplant

DOI

EISSN

1523-6536

Publication Date

June 2015

Volume

21

Issue

6

Start / End Page

1083 / 1090

Location

United States

Related Subject Headings

  • Transplantation, Homologous
  • Tacrolimus
  • Skin Diseases
  • Recurrence
  • Range of Motion, Articular
  • Prednisone
  • Pilot Projects
  • Mycophenolic Acid
  • Middle Aged
  • Male