Thalidomide for the treatment of chronic graft-versus-host disease

Georgia Boyce Vogelsang, Evan R. Farmer, Allan D. Hess, Viki Altamonte, William E. Beschorner, Douglas Jabs, Russell L. Corio, L. Stefon Levin, O. Michael Colvin, John R. Wingard, George W. Santos

Research output: Contribution to journalArticle

Abstract

Background. Allogeneic bone marrow transplantation is an accepted therapy for hematologic cancer, aplastic anemia, and inherited immunodeficiencies. Chronic graft-versus-host disease (GVHD) is the principal complication in patients surviving more than 100 days. Ttialidomide has been shown experimentally to be 1ffective in treating GVHD. Methods. We treated 23 patients with chronic GVHD refractory to conventional treatment and 21 patients with "high-risk" chronic GVHD (identified as having at least two of the following three risk factors: chronic GVHD that has evolved from acute GVHD, lichenoid skin or mucous-membrane changes, and hepatic dysfunction; such patients have a high mortality rate) with thalidomide in a dose that produced a plasma level of 5 pg per milliliter two hours after administration. Therapy was continued for three months after a complete response or for six months after a partial response. Results. The overall actuarial survival of all enrolled patients was 64 percent. Survival was 76 percent among the patients receiving salvage therapy for refractory GVHD and 48 percent among those with high-risk GVHD. A complete response was observed in 14 patients, a partial response in 12 patients, and no response in 18. Side effects were minor, most notably sedation in almost all patients. Conclusions. In this preliminary trial, thalidomide appeared to be safe and effective for the treatment of chronic GVHD. A trial comparing thalidomide with prednisone in patients with newly diagnosed chronic GVHD will be required to demonstrate its relative efficacy.

Original languageEnglish (US)
Pages (from-to)1055-1058
Number of pages4
JournalNew England Journal of Medicine
Volume326
Issue number16
StatePublished - Apr 16 1992

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Thalidomide
Graft vs Host Disease
Therapeutics
Salvage Therapy
Survival
Aplastic Anemia
Homologous Transplantation
Prednisone
Bone Marrow Transplantation
Mucous Membrane
Skin

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Vogelsang, G. B., Farmer, E. R., Hess, A. D., Altamonte, V., Beschorner, W. E., Jabs, D., ... Santos, G. W. (1992). Thalidomide for the treatment of chronic graft-versus-host disease. New England Journal of Medicine, 326(16), 1055-1058.

Thalidomide for the treatment of chronic graft-versus-host disease. / Vogelsang, Georgia Boyce; Farmer, Evan R.; Hess, Allan D.; Altamonte, Viki; Beschorner, William E.; Jabs, Douglas; Corio, Russell L.; Levin, L. Stefon; Colvin, O. Michael; Wingard, John R.; Santos, George W.

In: New England Journal of Medicine, Vol. 326, No. 16, 16.04.1992, p. 1055-1058.

Research output: Contribution to journalArticle

Vogelsang, GB, Farmer, ER, Hess, AD, Altamonte, V, Beschorner, WE, Jabs, D, Corio, RL, Levin, LS, Colvin, OM, Wingard, JR & Santos, GW 1992, 'Thalidomide for the treatment of chronic graft-versus-host disease', New England Journal of Medicine, vol. 326, no. 16, pp. 1055-1058.
Vogelsang GB, Farmer ER, Hess AD, Altamonte V, Beschorner WE, Jabs D et al. Thalidomide for the treatment of chronic graft-versus-host disease. New England Journal of Medicine. 1992 Apr 16;326(16):1055-1058.
Vogelsang, Georgia Boyce ; Farmer, Evan R. ; Hess, Allan D. ; Altamonte, Viki ; Beschorner, William E. ; Jabs, Douglas ; Corio, Russell L. ; Levin, L. Stefon ; Colvin, O. Michael ; Wingard, John R. ; Santos, George W. / Thalidomide for the treatment of chronic graft-versus-host disease. In: New England Journal of Medicine. 1992 ; Vol. 326, No. 16. pp. 1055-1058.
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AU - Farmer, Evan R.

AU - Hess, Allan D.

AU - Altamonte, Viki

AU - Beschorner, William E.

AU - Jabs, Douglas

AU - Corio, Russell L.

AU - Levin, L. Stefon

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AU - Santos, George W.

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N2 - Background. Allogeneic bone marrow transplantation is an accepted therapy for hematologic cancer, aplastic anemia, and inherited immunodeficiencies. Chronic graft-versus-host disease (GVHD) is the principal complication in patients surviving more than 100 days. Ttialidomide has been shown experimentally to be 1ffective in treating GVHD. Methods. We treated 23 patients with chronic GVHD refractory to conventional treatment and 21 patients with "high-risk" chronic GVHD (identified as having at least two of the following three risk factors: chronic GVHD that has evolved from acute GVHD, lichenoid skin or mucous-membrane changes, and hepatic dysfunction; such patients have a high mortality rate) with thalidomide in a dose that produced a plasma level of 5 pg per milliliter two hours after administration. Therapy was continued for three months after a complete response or for six months after a partial response. Results. The overall actuarial survival of all enrolled patients was 64 percent. Survival was 76 percent among the patients receiving salvage therapy for refractory GVHD and 48 percent among those with high-risk GVHD. A complete response was observed in 14 patients, a partial response in 12 patients, and no response in 18. Side effects were minor, most notably sedation in almost all patients. Conclusions. In this preliminary trial, thalidomide appeared to be safe and effective for the treatment of chronic GVHD. A trial comparing thalidomide with prednisone in patients with newly diagnosed chronic GVHD will be required to demonstrate its relative efficacy.

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