title: Chronic graft-versus-host disease in 52 patients: adverse natural course and successful treatment with combination immunosuppression.
abstract: Fifty-two of 175 (30%) survivors of allogeneic marrow transplantation developed chronic graft-versus-hose diseases (GVHD). Five with limited chronic GVHD had an indolent clinical course with involvement of only the skin and liver. Forty-seven with extensive chronic GVHD had an unfavorable multiorgan disorder that resembled several autoimmune diseases. Thirteen patients with extensive disease (group I) were not treated and only 2 survive with Karnofsky scores >- 70%. Mortality resulted from infections and morbidity from sica syndrome, pulmonary and hepatic insufficiency, scleroderma-like skin disease, and contractures. Another 13 (group II) received a median of 8 mo prednisone and/or a brief course of antithymocyte globulin, and 3 survive without disability. The other 21 (group III) were treated with a combination of prednisone (1.0 mg/kg/q.o.d.) and either cyclophosphamide, procarbazine, or azathioprine (all 1.5 mg/kg/day) for a median of 13 mo. Combination therapy was well tolerated with only modest myelotoxicity. Fifteen in group III had a good and 4 a fair response to treatment while 2 with no response died. Azathioprine and prednisone was the most effective regimen. All therapy has been discontinued in 12 group III patients: GVHD returned in 5 (including 2 who died in spite of retreatment) while 7 remain free of GVHD for a median of 11 (range 6-30) mo observation. Only I group III survivor is disabled and 16 of the original 21 are alive 2-4 yr after transplant with Karnofsky scores of 70%-100%. Thus, combination immmunosuppression appears to favorably affect and, in some cases, premanently arrest the adverse natural course of extensive chronic GVHD.
mesh_terms: D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D001379:Azathioprine / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation; D002908:Chronic Disease; D004359:Drug Therapy, Combination; D005500:Follow-Up Studies; D006087:Graft vs Host Reaction; D006801:Humans; D011241:Prednisone / Q000627:therapeutic use*; D014184:Transplantation, Homologous
chemical_list: D000961:Antilymphocyte Serum; D001379:Azathioprine; D011241:Prednisone