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Blood, 15 November 2000, Vol. 96, No. 10, pp. 3357-3363
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Oral isobutyramide reduces transfusion requirements in some
patients with homozygous  -thalassemia
Susanne Reich,
Christoph Bührer,
Günter Henze,
Dieter Ohlendorf,
Michael Mesche,
Pranav Sinha,
Andreas Kage,
Christian Müller,
Barbara Vetter, and
Andreas E. Kulozik
From the Departments of Pediatric Oncology/Hematology,
General Pediatrics, Neonatology, Clinical Chemistry/Biochemistry, and
Pharmacy, Charité, Humboldt University, Berlin,
Germany.
The butyrate derivative isobutyramide (IBT) increases fetal
hemoglobin (HbF) in patients with  -hemoglobinopathies, but little is
known about its usefulness for prolonged therapeutic use. We treated 8 patients with transfusion-dependent -thalassemia with 350 mg/kg of
body weight per day of oral IBT for 126 to 384 days. During the trial
period, the hemoglobin level was maintained between 85 g/L (range 82-87 g/L) (pretransfusion) and 115 g/L (range 110-119 g/L)
(post-transfusion) (median, interquartile range), corresponding to
4-week transfusion intervals in all patients during the pretreatment phase. Adverse effects (bitter taste, epigastric discomfort) did not
cause discontinuation of IBT. HbF increased in all patients from 3.1%
(range 1.9%-4.8%) to 6.0% (range 3.3%-8.7)
(P = .0017), while free Hb dropped from 0.48 g/L (range
0.39-0.81 g/L) to 0.19 g/L (range 0.16-0.24 g/L)
(P < .0001). Transfusion intervals were consistently
extended to 8 or 9 weeks in 1 patient, resulting in a decrease of daily
iron load from 455 µg/kg per day (range 451-459 µg/kg per day)
before therapy to 211µg/kg per day (range 203-286 µg/kg per day)
during the 12-month treatment period. Prolongation of transfusion
intervals achieved by IBT was less consistent in another patient, whose
parenteral iron load nevertheless decreased from 683 µg/kg per day
(range 618-748 µg/kg per day) to 542 µg/kg per day (340-596 µg/kg
per day). In the other 6 patients, no prolongation of transfusion
intervals was achieved. Response to treatment was associated
with high pretreatment HbF (> 4.5%), high parental HbF, and
increased erythropoietin levels (> 150 IU/L). We conclude that IBT
prolongs transfusion intervals and reduces parenteral iron burden in
some patients with transfusion-dependent -thalassemia.
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