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地贫患者铁还原治疗的结果。

Outcome of iron reduction therapy in ex-thalassemics.

机构信息

Department of Haematology, Christian Medical College, Vellore, India.

出版信息

PLoS One. 2021 Jan 22;16(1):e0238793. doi: 10.1371/journal.pone.0238793. eCollection 2021.

Abstract

There is limited data on iron reduction therapy (IRT) after successful allogeneic haematopoietic stem cell transplantation (aHSCT) for patients with thalassemia major (TM). We present the long term outcome of IRT in 149 patients with TM who underwent aHSCT during January, 2001-December, 2012. The median age was 7 years (range:1-18) and 92 (61.7%) belonged to Pesaro class 3 with a median ferritin at aHSCT of 2480ng/ml (range:866-8921). IRT was reinitiated post-aHSCT at a median of 14 months (range:5-53) post aHSCT with phlebotomy alone in 10 (6.7%) patients or iron chelation alone in 60 (40.3%) patients while 79 (53%) were treated with the combination. Reduction in serum ferritin/month [absolute quantity (ng/ml/month) was as follows: 87 (range:33-195), 130 (range:17-1012) and 147 (range:27.7-1427) in the phlebotomy, chelation and combination therapy groups, respectively (p = 0.038). With a median follow up of 80 months (range:37-182), target ferritin level of <300ng/ml was achieved in 59(40%) while a level <500ng/ml was achieved in 88 patients (59%) in a median duration of 41 months of IRT (range: 3-136). Patients in class III risk category and higher starting serum ferritin levels (>2500ng/ml) were associated with delayed responses to IRT. Our data shows that IRT may be needed for very long periods in ex-thalassaemics to achieve target ferritin levels and should therefore be carefully planned and initiated as soon as possible after aHSCT. A combination of phlebotomy and iron chelators is more effective in reducing iron overload.

摘要

对于重型地中海贫血(TM)患者接受异基因造血干细胞移植(aHSCT)成功后的铁还原治疗(IRT),数据有限。我们报告了 2001 年 1 月至 2012 年 12 月期间接受 aHSCT 的 149 例 TM 患者的 IRT 长期结果。中位年龄为 7 岁(范围:1-18 岁),92 例(61.7%)属于佩萨罗 3 级,aHSCT 时中位铁蛋白为 2480ng/ml(范围:866-8921)。aHSCT 后中位 14 个月(范围:5-53 个月)开始再次 IRT,单独放血治疗 10 例(6.7%),单独铁螯合治疗 60 例(40.3%),而 79 例(53%)联合治疗。血清铁蛋白/月的降低量[绝对量(ng/ml/月)分别为:放血组 87(范围:33-195),螯合组 130(范围:17-1012)和联合治疗组 147(范围:27.7-1427)(p = 0.038)。中位随访 80 个月(范围:37-182),59 例(40%)达到目标铁蛋白水平<300ng/ml,88 例(59%)达到目标铁蛋白水平<500ng/ml,中位 IRT 时间为 41 个月(范围:3-136)。III 级风险类别和更高的起始血清铁蛋白水平(>2500ng/ml)的患者对 IRT 的反应延迟。我们的数据表明,ex-thalassaemics 可能需要非常长的时间才能达到目标铁蛋白水平,因此应该在 aHSCT 后尽快仔细计划和启动 IRT。放血和铁螯合剂的联合使用在减少铁过载方面更有效。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b3fd/7822270/9f3705efbdb4/pone.0238793.g001.jpg

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