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All-trans retinoic acid in patients with myelodysplastic syndromes: results
of a pilot study
C Aul, V Runde and N Gattermann
Department of Internal Medicine, Heinrich Heine University, Dusseldorf,
Germany.
Considering the beneficial effect of all-trans retinoic acid (ATRA) in
acute promyelocytic leukemia (APL), it has been speculated that ATRA might
also be useful for treating other hematologic malignancies. To test this
hypothesis, we performed a dose-escalating 3-month-trial of ATRA in 15
patients with primary or secondary myelodysplastic syndromes (MDS).
Morphologic diagnoses were refractory anemia (RA) in 4, RA with ring
sideroblasts (RARS) in 2, RA with excess blasts (RAEB) in 7, and RAEB in
transformation (RAEB/T) in 2 cases. Patients included were required to have
one or more of the following criteria: transfusion- dependent anemia,
pronounced neutropenia (< or = 0.5 x 10(9)/L) or thrombocytopenia (<
or = 20 x 10(9)/L), or increasing blast cells in the peripheral blood or
bone marrow. Therapy was started at an ATRA dose of 30 mg/m2/d,
administered orally as two doses of 15 mg/m2 every 12 hours. The retinoid
dose was increased to 60 mg/m2/d after 4 weeks and to 90 mg/m2/d after 8
weeks. Among 14 patients assessable for response, none obtained a complete
or partial remission. Three patients had a minor response, manifested by
either reduction in transfusion requirements (2 patients) or increase in
neutrophil and platelet counts (1 patient). During the study period, 5
patients progressed to more advanced stages of MDS or overt leukemia. Three
patients with chromosomal abnormalities receiving ATRA for a period of 10
to 12 weeks retained their cytogenetic marker after completion of
treatment. Side effects of ATRA primarily affected the skin and mucous
membranes, with 13 of 15 patients having at least low-grade dermatologic
toxicity. In 2 cases, treatment had to be prematurely stopped because of
intolerable conjunctivitis or progressive neurologic symptoms. These data
suggest that ATRA has little effect on MDS. The lack of response of MDS
patients, as compared with those with APL, may be attributed to the absence
of the t(15;17) translocation that seems to be a prerequisite for clinical
efficacy of ATRA.
Volume 82,
Issue 10,
pp. 2967-2974,
11/15/1993
Copyright © 1993 by The American Society of Hematology
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