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Prepublished online as a Blood First Edition Paper on October 31, 2002; DOI 10.1182/blood-2002-09-2867.
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Blood, 15 March 2003, Vol. 101, No. 6, pp. 2156-2158
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Brief report
Antithymocyte globulin has limited efficacy and
substantial toxicity in unselected anemic patients with
myelodysplastic syndrome
David P. Steensma,
Angela Dispenzieri,
S.
Breanndan Moore,
Georgene Schroeder, and
Ayalew Tefferi
From the Division of Hematology, Department of Internal
Medicine, Mayo Clinic, Rochester, MN; Tissue Typing Laboratory,
Division of Transfusion Medicine, Department of Laboratory Medicine and
Pathology, Mayo Clinic, Rochester, MN; and Division of Biostatistics,
Department of Health Sciences Research, Mayo Clinic, Rochester, MN.
Antithymocyte globulin (ATG) has recently been popularized as an
effective treatment in myelodysplastic syndrome (MDS). We treated 8 anemic MDS patients (refractory anemia [RA] and refractory anemia
with excess blasts [RAEB-1]) with ATG (40 mg/kg/d for 4 days) and
prednisone in a phase 2 trial. The study was stopped early according to
a preset termination rule because of lack of efficacy. There were no
salutary responses. Toxicities included serum sickness (in all
patients), transient neutropenia and thrombocytopenia, diarrhea,
vomiting, and syncope with a generalized seizure. At least 3 patients
had the HLA-DR15 (DR2) allele. We conclude that the risk-benefit ratio
of ATG in an unselected population of MDS patients may be unfavorable,
and more work is needed to define the subset of patients who will
respond to ATG before its widespread use can be recommended.
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