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Blood, 15 October 2002, Vol. 100, No. 8, pp. 2687-2690
PLENARY PAPER
Response of Diamond-Blackfan anemia to metoclopramide:
evidence for a role for prolactin in erythropoiesis
Janis L. Abkowitz,
Gerard Schaison,
Farid Boulad,
Deborah L. Brown,
George R. Buchanan,
Christine A. Johnson,
Jeffrey C. Murray, and
Kathleen M. Sabo
From the Division of Hematology, Department of
Medicine, University of Washington, Seattle; Pediatric Service Hospital
St Louis, Paris, France; Department of Pediatrics, Memorial
Sloan-Kettering Cancer Center, New York, NY; Children's Memorial
Hospital, Chicago, IL; Department of Pediatrics, University of Texas
Southwestern Medical Center, Dallas; Department of Pediatrics, Wake
Forest University, Winston-Salem, NC; and Cook Children's Medical
Center, Fort Worth, TX.
A 47-year-old woman with severe macrocytic anemia markedly improved
during the second and third trimesters of 3 pregnancies and when
breast-feeding her 2 children. Because the serum prolactin level is
elevated at these times, we later treated her with metoclopramide (10 mg orally 3 times daily), a medication known to induce prolactin release. Her serum prolactin levels increased from 7 to 133 ng/mL (normal < 20 ng/mL) and hematocrit from 17% to 22% to 35%. With continued therapy (now 10 mg orally daily), her hematocrit has ranged
from 30% to 40% for 6 years, although the macrocytosis persists (mean
corpuscular volume, 100-112 fL). On the basis of this observation, a
pilot study was undertaken of metoclopramide therapy in patients with
Diamond-Blackfan anemia who were refractory to low doses of
corticosteroids. Fifteen patients were enrolled and 9 completed the
planned 16 weeks of therapy. Three individuals responded, suggesting
that this therapeutic approach may benefit others. As with the index
case, the anemia did not improve until 12 to 15 weeks of therapy had
been completed.
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