Olivieri N F, Koren G, Harris J, Khattak S, Freedman M H, Templeton D M, Bailey J D, Reilly B J
Division of Haematology/Oncology, Hospital for Sick Children, University of Toronto, Ontario, Canada.
Am J Pediatr Hematol Oncol. 1992 Spring;14(1):48-56. doi: 10.1097/00043426-199221000-00007.
We reviewed the linear growth and growth plate morphology in all children with homozygous beta thalassemia followed in Toronto, for whom monthly height percentiles were available before, and for a 36-month period after, the initiation of nightly subcutaneous deferoxamine therapy. All patients were less than 7 years of age when begun on deferoxamine, and had received nightly deferoxamine for a minimum of 36 months. Marked abnormalities of the metaphyseal growth plate were readily observed in the distal ulnar, radial, and tibial metaphyses in 11 of 37 patients in whom a significant decline in mean height percentile was also noted. (In 10 of these 11 patients, height was less than the 15th percentile after 36 months.) These 11 patients had received a significantly greater (p less than 0.025) initial and average daily dose of deferoxamine, and had maintained a significantly lower (p less than 0.025) mean serum ferritin concentration over the 36 months, than the remainder of the cohort. To determine whether deferoxamine played a causative role in growth failure, growth in patients who began deferoxamine before the age 2 years was compared to that of patients who began after age 5 years, for the period between 2 and 5 years of age. Only patients begun on deferoxamine prior to age 2 years demonstrated a significant (p less than 0.01) decline in height percentile by the third year, implicating deferoxamine therapy as the cause of growth failure. We conclude that both the decline in height percentile and the bony changes observed in well-chelated patients are directly related to deferoxamine therapy.(ABSTRACT TRUNCATED AT 250 WORDS)
我们回顾了多伦多所有接受纯合子β地中海贫血治疗儿童的线性生长情况及生长板形态,这些儿童在开始夜间皮下注射去铁胺治疗前及治疗后的36个月期间,每月均有身高百分位数数据。所有患者在开始使用去铁胺时年龄均小于7岁,且每晚接受去铁胺治疗至少36个月。在37例患者中,有11例尺骨远端、桡骨远端和胫骨干骺端的干骺端生长板出现明显异常,同时这些患者的平均身高百分位数也显著下降。(在这11例患者中,有10例在36个月后身高低于第15百分位数。)与队列中的其他患者相比,这11例患者接受的去铁胺初始剂量和平均每日剂量显著更高(p<0.025),且在36个月期间平均血清铁蛋白浓度显著更低(p<0.025)。为了确定去铁胺是否在生长发育迟缓中起因果作用,将2岁前开始使用去铁胺的患者与5岁后开始使用去铁胺的患者在2至5岁期间的生长情况进行了比较。只有2岁前开始使用去铁胺的患者在第三年身高百分位数出现显著下降(p<0.01),这表明去铁胺治疗是生长发育迟缓的原因。我们得出结论,身高百分位数的下降以及在螯合良好的患者中观察到的骨骼变化均与去铁胺治疗直接相关。(摘要截短至250字)