Jani Ashesh B, Kao Johnny, Hellman Samuel
Department of Radiation and Cellular Oncology, University of Chicago, Chicago, Illinois 60637, USA.
Cancer. 2003 Dec 1;98(11):2351-61. doi: 10.1002/cncr.11804.
Hormone therapy commonly is used to treat metastatic, locally advanced, and localized prostate carcinoma. The objective of the current investigation was to determine, using the number-needed-to-treat (NNT) method, the effect of using hormone therapy to treat locally advanced disease, with consideration given to both the complications and the known advantages associated with hormone therapy.
A literature review was performed to determine 1) the absolute benefit, based on available clinical endpoints, associated with the addition of hormone therapy to external beam radiotherapy for locally advanced prostate carcinoma; 2) the incidence of side effects of short-term and long-term hormone therapy; and 3) the stepwise progression from biochemical failure to death. A model was constructed to estimate the complication/utility-adjusted survival detriment resulting from the side effects of short-term (</= 6 months) and long-term (> 6 months) hormone therapy, and the absolute/unadjusted and complication-adjusted NNTs for the addition of short-term and long-term hormone therapy were computed. In all cases, the magnitudes and signs of the NNTs obtained were used to gauge the effect of hormone therapy.
The unadjusted NNTs were positive and in most cases had relatively small magnitudes (the greater the NNT, the smaller the benefit) for both short-term and long-term hormone therapy; these results were expected, and they suggested that there is a strong benefit associated with the use of hormones adjuvant to radiotherapy for locally advanced disease. Adjusted NNTs remained positive and had relatively small magnitudes even after the introduction into the analysis of complications of short-term and long-term hormone therapy. This finding, although weak with respect to the effect of short-term hormone therapy on cause-specific survival, remained robust over the range of values for utility impairment expected from short-term and long-term hormone therapy.
The benefits of short-term and long-term hormone therapy for locally advanced prostate carcinoma appear to be significant and to outweigh the associated side effects. Long-term therapy appears to be better than short-term therapy in terms of virtually all endpoints studied, even when the increased incidence of side effects is considered. The current investigation was successful in the use of the complication-adjusted NNT method for oncologic and radiotherapeutic scenarios in which the results of randomized trials could be summarized, adjusted for treatment toxicity, and individualized to a given patient.
激素疗法常用于治疗转移性、局部晚期和局限性前列腺癌。本研究的目的是采用需治疗人数（NNT）方法，确定激素疗法治疗局部晚期疾病的效果，同时考虑激素疗法相关的并发症和已知优势。
进行文献综述以确定：1）基于现有临床终点，激素疗法联合外照射放疗治疗局部晚期前列腺癌的绝对获益；2）短期和长期激素疗法的副作用发生率；3）从生化失败到死亡的逐步进展。构建一个模型来估计短期（≤6个月）和长期（>6个月）激素疗法副作用导致的并发症/效用调整后的生存损害，并计算短期和长期激素疗法联合应用的绝对/未调整及并发症调整后的NNT。在所有情况下，所获得的NNT的大小和符号用于衡量激素疗法的效果。
短期和长期激素疗法的未调整NNT均为正值，且在大多数情况下数值相对较小（NNT越大，获益越小）；这些结果在意料之中，表明激素疗法联合放疗治疗局部晚期疾病有显著获益。即使在分析中纳入短期和长期激素疗法的并发症后，调整后的NNT仍为正值且数值相对较小。这一发现，尽管在短期激素疗法对特定病因生存的影响方面较弱，但在短期和长期激素疗法预期的效用损害值范围内仍很稳健。
短期和长期激素疗法治疗局部晚期前列腺癌的获益似乎很显著，且超过相关副作用。就几乎所有研究的终点而言，长期疗法似乎优于短期疗法，即使考虑到副作用发生率增加的情况。本研究成功地将并发症调整后的NNT方法用于肿瘤学和放射治疗场景，在这些场景中，可以总结随机试验的结果，针对治疗毒性进行调整，并针对特定患者进行个体化。
