Yarali Neşe, Fişgin Tunç, Duru Feride, Kara Abdurrahman, Ecin Nurhayat, Fitoz Suat, Erden Ilhan
Dr. Sami Ulus Children's Hospital, Department of Hematology, Ankara, Turkey.
J Pediatr Hematol Oncol. 2006 Jan;28(1):11-6.
The objective of this study was to compare the short- and long-term efficacy of deferoxamine (DFO) given by subcutaneous (SC) continuous infusion over 10 hours via a pump (n = 10) versus a twice-daily subcutaneous bolus injection of the same overall dose (n = 10) in 20 thalassemic children. Urinary iron excretion was measured in 24-hour urine samples after DFO treatment in the 20 patients. The patients were randomized to two groups: 10 patients continued SC continuous infusion with a pump and the remaining 10 received the same overall dose of DFO by twice-daily SC bolus injection for a year. Serum ferritin levels and T1-weighted spin-echo and T2-weighted fast spin-echo signal intensities of liver and paraspinal muscle were determined at initiation and 1 year after initiation of the therapy. In 12 patients, six from each group, liver biopsies were performed and hepatic iron concentration was determined at initiation of therapy and 1 year after treatment. A similar and significant decrease in ferritin levels and improvement in signal intensities of the liver were observed in response to chelation therapy with DFO in both groups (P < 0.01, within each group). Hepatic iron concentration decreased in all patients in the SC bolus injection group (P < 0.05) and in four patients in the SC continuous infusion group (P > 0.05). Hepatic iron concentration was noted to be slightly increased in two patients in the SC continuous infusion group, which may be due to poor compliance. Based on these results, twice-daily SC bolus injection of DFO is as effective as administration via SC continuous infusion using a pump. Subcutaneous bolus injection, being more convenient for the patient, may be a more preferable method of DFO administration.
本研究的目的是比较20名地中海贫血儿童中,通过泵持续皮下(SC)输注10小时给予去铁胺(DFO)(n = 10)与每日两次皮下推注相同总剂量(n = 10)的短期和长期疗效。在20例患者接受DFO治疗后,收集24小时尿液样本测量尿铁排泄量。患者被随机分为两组:10例患者继续通过泵进行SC持续输注,其余10例接受每日两次SC推注相同总剂量的DFO,为期一年。在治疗开始时及开始后1年测定血清铁蛋白水平以及肝脏和椎旁肌的T1加权自旋回波和T2加权快速自旋回波信号强度。在12例患者中,每组6例,在治疗开始时及治疗后1年进行肝脏活检并测定肝铁浓度。两组患者在接受DFO螯合治疗后,铁蛋白水平均出现相似且显著的下降,肝脏信号强度得到改善(每组P < 0.01)。SC推注注射组所有患者的肝铁浓度均下降(P < 0.05),SC持续输注组4例患者的肝铁浓度下降(P > 0.05)。SC持续输注组有2例患者的肝铁浓度略有升高,这可能是由于依从性差所致。基于这些结果,每日两次SC推注DFO与通过泵进行SC持续输注给药同样有效。皮下推注对患者来说更方便,可能是DFO给药更可取的方法。