Song Hyun Jin, Lee Jun Ah, Han Euna, Lee Eui-Kyung
School of Pharmacy, Sungkyunkwan University, 2066 Seobu-ro, Jangan-gu, Suwon, Gyeonggi-do, 440-746, Republic of Korea.
Department of Pediatrics, Korea Cancer Center Hospital, Seoul, Republic of Korea.
Tumour Biol. 2015 Sep;36(9):6773-9. doi: 10.1007/s13277-015-3405-3. Epub 2015 Apr 3.
The mortality and progression rates in osteosarcoma differ depending on the presence of metastasis. A decision model would be useful for estimating long-term effectiveness of treatment with limited clinical trial data. The aim of this study was to explore the lifetime effectiveness of the addition of mifamurtide to chemotherapy for patients with metastatic and nonmetastatic osteosarcoma. The target population was osteosarcoma patients with or without metastasis. A Markov process model was used, whose time horizon was lifetime with a starting age of 13 years. There were five health states: disease-free (DF), recurrence, post-recurrence disease-free, post-recurrence disease-progression, and death. Transition probabilities of the starting state, DF, were calculated from the INT-0133 clinical trials for chemotherapy with and without mifamurtide. Quality-adjusted life-years (QALY) increased upon addition of mifamurtide to chemotherapy by 10.5 % (10.13 and 9.17 QALY with and without mifamurtide, respectively) and 45.2 % (7.23 and 4.98 QALY with and without mifamurtide, respectively) relative to the lifetime effectiveness of chemotherapy in nonmetastatic and metastatic osteosarcoma, respectively. Life-years gained (LYG) increased by 10.1 % (13.10 LYG with mifamurtide and 11.90 LYG without mifamurtide) in nonmetastatic patients and 42.2 % (9.43 LYG with mifamurtide and 6.63 LYG without mifamurtide) in metastatic osteosarcoma patients. The Markov model analysis showed that chemotherapy with mifamurtide improved the lifetime effectiveness compared to chemotherapy alone in both nonmetastatic and metastatic osteosarcoma. Relative effectiveness of the therapy was higher in metastatic than nonmetastatic osteosarcoma over lifetime. However, absolute lifetime effectiveness was higher in nonmetastatic than metastatic osteosarcoma.
骨肉瘤的死亡率和进展率因是否存在转移而异。决策模型对于在临床试验数据有限的情况下评估治疗的长期有效性很有用。本研究的目的是探讨米伐木肽联合化疗对转移性和非转移性骨肉瘤患者的终生有效性。目标人群为有或无转移的骨肉瘤患者。使用了马尔可夫过程模型，其时间范围为终生，起始年龄为13岁。有五个健康状态：无病（DF）、复发、复发后无病、复发后疾病进展和死亡。起始状态DF的转移概率根据INT - 0133临床试验计算得出，该试验涉及使用和不使用米伐木肽的化疗。与单独化疗在非转移性和转移性骨肉瘤中的终生有效性相比，米伐木肽联合化疗使质量调整生命年（QALY）分别增加了10.5%（使用和不使用米伐木肽时分别为10.13和9.17 QALY）和45.2%（使用和不使用米伐木肽时分别为7.23和4.98 QALY）。非转移性患者的生命年增益（LYG）增加了10.1%（使用米伐木肽时为13.10 LYG，不使用米伐木肽时为11.90 LYG），转移性骨肉瘤患者增加了42.2%（使用米伐木肽时为9.43 LYG，不使用米伐木肽时为6.63 LYG）。马尔可夫模型分析表明，在非转移性和转移性骨肉瘤中，与单独化疗相比，米伐木肽联合化疗提高了终生有效性。在整个生命过程中，该疗法在转移性骨肉瘤中的相对有效性高于非转移性骨肉瘤。然而，非转移性骨肉瘤的绝对终生有效性高于转移性骨肉瘤。
