Antithymocyte globulin has limited efficacy and substantial toxicity in unselected anemic patients with myelodysplastic syndrome.

Abstract:

: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.

journal_name

Blood

journal_title

Blood

authors

Steensma DP,Dispenzieri A,Moore SB,Schroeder G,Tefferi A

doi

10.1182/blood-2002-09-2867

subject

Has Abstract

pub_date

2003-03-15 00:00:00

pages

2156-8

issue

6

eissn

0006-4971

issn

1528-0020

pii

2002-09-2867

journal_volume

101

pub_type

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