Hamidieh Amir Ali, Mohseni Fariba, Behfar Maryam, Hamidi Zohreh, Alimoghaddam Kamran, Pajouhi Mohamad, Larijani Bagher, Mohajeri-Tehrani Mohammad-Reza, Ghavamzadeh Ardeshir
Pediatric Stem Cell Transplant Department Children's Medical Center, Tehran University of Medical Sciences, Tehran, Iran.
Endocrinology and Metabolism Research Center, Endocrinology and Metabolism Clinical Sciences Institute, Tehran University of Medical Sciences, Tehran, Iran.
Arch Iran Med. 2018 Feb 1;21(2):56-60.
Beta thalassemia major (BTM) and its treatment by hematopoietic stem cell transplantation (HSCT) may have deleterious effects on the endocrine systems. We assessed endocrine complications of HSCT in pediatric patients for 3 months.
In 20 (6 female) pediatric major thalassemic patients (mean age of 10.8 ± 3.9 years old), prolactin, luteinizing hormone (LH), follicle-stimulating hormone (FSH), T4, T3, thyroid-stimulating hormone (TSH), IGF-1, testosterone (in males) or estradiol (in females) were measured as a batch at the Endocrinology and Metabolism Research Center (EMRC) of Tehran University of Medical Sciences (TUMS) laboratories before HSCT and 1 and 3 months afterwards. The cosyntropin test for all and the clonidine test for short stature patients was conducted before HSCT.
Before HSCT, delayed puberty and hypogonadotropic hypogonadism was found in 10% and 20% of patients, respectively. GH deficiency, low IGF1 and short stature was found in 25%, 55% and 40% of patients, respectively. Hypocortisolism, hypothyroidism and panhypopituitarism was found in 15%, 10% and 15% of patients, respectively. Prevalence of hypogonadotropic hypogonadism, low IGF1, hypothyroidism and panhypopituitarism was found in 20%, 40%, 10% and 10% of patients after 3 months, respectively (delayed puberty and short stature prevalence do not change after 3 months). HSCT caused lower T3 and estradiol and higher TSH. Corticosteroid users (15) had higher GH and lower T3 and testosterone or estradiol. Ferritin had a significant (negative) correlation with (before) prolactin and a significant correlation with T3 and T4 after HSCT. Age and acute graft-versus-host disease (GVHD) had no significant effect.
Considering the small sample size and short duration of the study, it is difficult to reach any conclusion however it seems HSCT does not appear to have an overall positive or negative effect on prevalence of pituitary- hypothalamus axis disorders in pediatric thalassemic patients in 3 months.
重型β地中海贫血（BTM）及其造血干细胞移植（HSCT）治疗可能对内分泌系统产生有害影响。我们评估了小儿患者HSCT术后3个月的内分泌并发症。
选取20例（6例女性）小儿重型地中海贫血患者（平均年龄10.8±3.9岁），在德黑兰医科大学（TUMS）内分泌与代谢研究中心（EMRC）实验室于HSCT术前、术后1个月和3个月时批量检测催乳素、黄体生成素（LH）、卵泡刺激素（FSH）、T4、T3、促甲状腺激素（TSH）、胰岛素样生长因子-1（IGF-1）、睾酮（男性）或雌二醇（女性）。所有患者在HSCT术前均进行了促肾上腺皮质激素试验，身材矮小患者进行了可乐定试验。
HSCT术前，分别有10%和20%的患者出现青春期延迟和低促性腺激素性性腺功能减退。分别有25%、55%和40%的患者存在生长激素缺乏、IGF1水平低和身材矮小。分别有15%、10%和15%的患者存在皮质醇减退、甲状腺功能减退和全垂体功能减退。3个月后，分别有20%、40%、10%和10%的患者存在低促性腺激素性性腺功能减退、IGF1水平低、甲状腺功能减退和全垂体功能减退（青春期延迟和身材矮小的患病率在3个月后未改变）。HSCT导致T3和雌二醇水平降低，TSH水平升高。使用皮质类固醇的患者（15例）生长激素水平较高，T3以及睾酮或雌二醇水平较低。铁蛋白与（术前）催乳素呈显著（负）相关，与HSCT术后的T3和T4呈显著相关。年龄和急性移植物抗宿主病（GVHD）无显著影响。
考虑到本研究样本量小且持续时间短，难以得出任何结论，但似乎HSCT在3个月内对小儿地中海贫血患者垂体-下丘脑轴疾病的患病率总体上没有正面或负面影响。
