Phase II study of rabbit anti-thymocyte globulin, cyclosporine and granulocyte colony-stimulating factor in patients with aplastic anemia and myelodysplastic syndrome.
We investigated efficacy and safety of rabbit anti-thymocyte globulin (rATG), cyclosporine and granulocyte colony-stimulating factor (G-CSF) as first-line therapy for patients with aplastic anemia (AA) and low or intermediate-1 or hypoplastic myelodysplastic syndrome (MDS). rATG 3.5 mg/kg (or 2.5 mg/kg per day for patients >or=55 years with MDS) was given for 5 days. Cyclosporine (5 mg/kg) and G-CSF (5 microg/kg) were given daily and continued for up to 6 months or longer. Responses were assessed about 3 and 6 months after therapy. Thirty-six patients have been enrolled on study and 32 patients treated; 25 were evaluable for a response (13 with AA, 12 with MDS); the rest are too early. The median age was 62 years (range, 20-83) for patients with AA and 63 (range, 42-80) for patients with MDS. Of 13 patients, 12 (92%) patients with AA responded (5 complete response (CR), 7 partial response (PR)), whereas of 12 patients, 4 (33%) patients with MDS responded (1 CR, 3 PR). For patients with AA, the median time to response (TTR) was 93 days (range, 79-623), whereas in the MDS group the median TTR was 111 days (range, 77-139). Grade III/IV toxicities were mainly cytopenias and neutropenic fever. Combination of rATG, cyclosporine and G-CSF is safe and effective as first-line treatment of AA and has significant activity in low-risk MDS.
Garg
R
R
Department of Leukemia, The University of Texas, MD Anderson Cancer Center, Houston, TX 77030, USA.
Faderl
S
S
Garcia-Manero
G
G
Cortes
J
J
Koller
C
C
Huang
X
X
York
S
S
Pierce
S
S
Brandt
M
M
Beran
M
M
Borthakur
G
G
Kantarjian
H
H
Ravandi
F
F
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000741:Anemia, Aplastic / Q000188:drug therapy*; D000818:Animals; D000961:Antilymphocyte Serum / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D016572:Cyclosporine / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D016179:Granulocyte Colony-Stimulating Factor / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy*; D011817:Rabbits
D000961:Antilymphocyte Serum; D016179:Granulocyte Colony-Stimulating Factor; D016572:Cyclosporine