De Simone M, Di Bartolomeo P, Olioso P, Di Girolamo G, Palumbo M, Farello G, Aconito P, Papalinetti G, Bavaro P, Criscione S
Clinica Pediatrica-Dipartimento Medicina Sperimentale, Universita' di L'Aquila, Italy.
Bone Marrow Transplant. 1997 Oct;20(7):567-73. doi: 10.1038/sj.bmt.1700931.
The aim of this study was to evaluate the treatment effects with recombinant human growth hormone (rhGH) in a group of patients after bone marrow transplantation for thalassemia major. At the end of treatment we divided the subjects into two groups according to the outcome of the therapy: responder and nonresponder. Responder group: after 24 months of rhGH administration, growth rate was still significantly higher in respect to start of treatment (P < 0.0001). Plasma levels of IGF-I rose significantly (P < 0.003). The serum levels of serum asparate aminotransferase (SGOT) and alanine aminotransferase (SGPT) were higher compared to normal values but improved in non-responder patients. There was no difference in the mean concentration of these parameters before and after treatment (P = NS). Non-responder group: these patients had a worsening of the growth rate during rhGH administration. There was no increase of the IGF-I levels. Single values of transaminase and ferritin levels were higher than in responder patients before and after treatment. There was a significant correlation between IGF-I, SGOT, SGPT and ferritin in all patients before and after therapy. It appears from these data that rhGH administration is worth serious consideration in patients after BMT for thalassemia major presenting impaired growth hormone secretion. This treatment can offer good results only in cases where the normal hepatic synthesis of IGF-I is conserved and where liver damage has not reached irreversible conditions, as we have seen in the responder group.
本研究的目的是评估重组人生长激素（rhGH）对一组重型地中海贫血骨髓移植后患者的治疗效果。在治疗结束时，我们根据治疗结果将受试者分为两组：反应者和无反应者。反应者组：rhGH给药24个月后，生长速率仍显著高于治疗开始时（P < 0.0001）。胰岛素样生长因子-I（IGF-I）的血浆水平显著升高（P < 0.003）。血清天冬氨酸氨基转移酶（SGOT）和丙氨酸氨基转移酶（SGPT）水平高于正常值，但在无反应患者中有所改善。这些参数治疗前后的平均浓度无差异（P = 无显著性差异）。无反应者组：这些患者在rhGH给药期间生长速率恶化。IGF-I水平没有升高。转氨酶和铁蛋白水平的单个值在治疗前后均高于反应者患者。在所有患者治疗前后，IGF-I、SGOT、SGPT和铁蛋白之间均存在显著相关性。从这些数据看来，对于重型地中海贫血骨髓移植后生长激素分泌受损的患者，rhGH给药值得认真考虑。正如我们在反应者组中所见，这种治疗仅在IGF-I的正常肝脏合成得以保留且肝损伤尚未达到不可逆状态的情况下才能取得良好效果。
