Leahey A M, Teunissen H, Friedman D L, Moshang T, Lange B J, Meadows A T
Department of Pediatrics, The Children's Hospital of Philadelphia, University of Pennsylvania, 19104, USA.
Med Pediatr Oncol. 1999 Mar;32(3):163-9. doi: 10.1002/(sici)1096-911x(199903)32:3<163::aid-mpo1>3.0.co;2-#.
As more pediatric patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) survive, comparison of the late effects of various therapies becomes increasingly important. This study of survivors of AML is the largest to date comparing the late effects of patients treated with chemotherapy (CT) with or without irradiation (RT) or CT followed by bone marrow transplantation (BMT).
In a retrospective review of 228 patients with AML or MDS from 1970 to 1995, 62 survived and had follow-up data available more than 1 year following completion of therapy. Ten patients with Down syndrome were excluded. Twenty-six received CT and 26 underwent BMT. Weight and height Z scores, endocrine, ophthalmologic, renal, and cardiac function following CT +/- RT or BMT +/- total body irradiation (TBI) were compared at a mean follow-up of 7.4 and 5.6 years, respectively.
Both groups experienced a decrement in height and increase in weight. The mean height Z score in the CT group fell from -0.29 to -0.72 (P = 0.02) and mean weight Z score rose from -0.06 at diagnosis (T0) to 0.51 at last follow-up (T2) (P = 0.02), a finding no longer significant when patients who received RT were excluded. The mean height Z score in the BMT group fell from -0.17 at TO to -0.65 at T2 (P = 0.02), while the mean weight rose from 0.29 at T0 to 0.84 at T2, (P = 0.07). Six of 9 BMT adolescent girls experienced ovarian failure versus 0 of 11 girls treated with CT (P = 0.002). Seven adolescent CT males and seven BMT males showed normal pubertal progression. Two BMT patients require thyroid hormone supplementation, and one receives growth hormone. Six BMT patients and one CT patient developed cataracts, all of whom received irradiation (P = 0.10). Serum creatinine level, hypertension, or left ventricular shortening fraction were not different in the two groups. One BMT patient has chronic graft versus host disease.
Growth, renal, and cardiac functions were similar in the two groups. The need for estrogen supplementation was more frequent following BMT. Recommendations concerning therapy for AML should depend on the probability of cure.
随着越来越多的急性髓系白血病（AML）和骨髓增生异常综合征（MDS）患儿存活下来，比较各种治疗方法的远期效应变得越来越重要。这项针对AML幸存者的研究是迄今为止规模最大的，比较了接受化疗（CT）加或不加放疗（RT）或CT后进行骨髓移植（BMT）的患者的远期效应。
对1970年至1995年期间的228例AML或MDS患者进行回顾性研究，其中62例存活且在治疗结束后有超过1年的随访数据。10例唐氏综合征患者被排除。26例接受化疗，26例接受骨髓移植。分别在平均随访7.4年和5.6年时，比较化疗±放疗或骨髓移植±全身照射（TBI）后患者的体重和身高Z评分、内分泌、眼科、肾脏和心脏功能。
两组患者身高均下降，体重均增加。化疗组的平均身高Z评分从-0.29降至-0.72（P = 0.02），平均体重Z评分从诊断时（T0）的-0.06升至最后随访时（T2）的0.51（P = 0.02），排除接受放疗的患者后这一结果不再显著。骨髓移植组的平均身高Z评分从T0时的-0.17降至T2时的-0.65（P = 0.02），而平均体重从T0时的0.29升至T2时的0.84（P = 0.07）。9例接受骨髓移植的青春期女孩中有6例出现卵巢功能衰竭，而接受化疗的11例女孩中无一例出现（P = 0.002）。7例接受化疗的青春期男性和7例接受骨髓移植的男性青春期发育正常。2例骨髓移植患者需要补充甲状腺激素，1例接受生长激素治疗。6例骨髓移植患者和1例化疗患者出现白内障，他们均接受过放疗（P = 0.10）。两组患者的血清肌酐水平、高血压或左心室缩短分数无差异。1例骨髓移植患者患有慢性移植物抗宿主病。
两组患者的生长、肾脏和心脏功能相似。骨髓移植后雌激素补充的需求更频繁。关于AML治疗的建议应取决于治愈的可能性。
