ความปลอดภัยและประสิทธิภาพของยาเดเฟอริโพรนเพียงอย่างเดียว หรือร่วมกับยาเดสเฟอรอกซามีนในผู้ป่วยเด็กโรคโลหิตจางธาลัสซีเมีย

Abstract

Safety and Efficacy of Deferiprone Monotherapy and Combined Therapy with
Desferrioxamine in Thalassemia Children
Saraburi Hospital Medical Journal 2009; 34: 13-22
Objective To assess safety and efficacy of deferiprone monotherapy and combined therapy
with desferrioxamine in thalassemia children with iron overload (serum ferritin> 1,000
ng/ml)

Patients and Methods A descriptive retrospective study involved 50 thalassemia
children with iron overload age 18 years and below between July 2007 and December
2008. There were 28 children who received treatment for at least 6 months with
completed data, deferiprone monotherapy (n = 1 o) and combined therapy with
desferrioxamine (n = 18). The dose of deferiprone in deferiprone monotherapy ranged
from 29.70 - 62.00 mg/kg/day with a mean of 42.84 ± 10.16 mg/kg/day. In combined
therapy, the dose of deferiprone ranged from 29.70- 62.00 mg/kg/day with a mean of
42.84 ± 10.16 mg/kglday and desferrioxamine ranged from 23.50 - 42.10 mg/kg/day
with a mean of 33.25 ± 5.97 mglkg/day, subcutaneously about 8 - 10 hours for 3 - 4
days weekly. Changes in complete blood count, serum alanine aminotransferase,
serum zinc, and serum ferritin were assessed.
Results In deferiprone monotherapy, serum ferritin decreased from mean initial level
of 2,044 ± 535 ng/ml to mean final level of 1,353 ± 487 ng/ml (8 in 10, 80%), (p =
0.008). In combined therapy with desferrioxamine, serum ferritin decreased from mean
initial level of 3,750 ± 1,564 ng/ml to mean final level of 1,641 ± 701 ng/ml (15 in 18,
83.3%), (p = o.ooo). One (5 .5%) patient in combined therapy developed agranulocytosis which resolved to normal on interruption of treatment. Serum AL T levels were
decreased in the most of patients of both regimens. Zinc deficiency occurred in deferiprone
monotherapy (1 in 10, 10%) and in combined therapy (7 in 17, 41.2%). Arthropathy
appeared in 5 (27 .8o/o) patients in combined therapy. There were nausea-vomiting in
deferiprone monotherapy (3 in 10, 30%) and combined therapy (sin 18, 27.8%). But
there was abdominal pain only in combined therapy (2 in 18, 11.1%).
Conclusion Deferiprone 30 - 60 mg/kg/day is effective in reducing serum ferritin in
both deferiprone monotherapy and combined therapy with desferrioxamine (p = 0.008
and p = o.ooo respectively). The common side effects of deferiprone are zinc deficiency,
arthropathy, gastrointestinal symptoms, but agranulocytosis is the most serious ·side
effect that needs close monitoring during therapy