Horowitz M E, Kinsella T J, Wexler L H, Belasco J, Triche T, Tsokos M, Steinberg S M, McClure L, Longo D L, Steis R G
Pediatric Branche, National Cancer Institute, National Institutes of Health, Bethesda, MD.
J Clin Oncol. 1993 Oct;11(10):1911-8. doi: 10.1200/JCO.1993.11.10.1911.
In an effort to improve outcome in patients with metastatic or high-risk localized Ewing's sarcoma family of tumors (ESF) and rhabdomyosarcoma (RMS), we explored the role of consolidation therapy with total-body irradiation (TBI) plus autologous bone marrow transplantation (ABMT).
Ninety-one patients were entered onto one of three consecutive protocols from 1981 to 1986. Induction therapy consisted of four or five cycles of vincristine, doxorubicin, and cyclophosphamide (VAdriaC); in the earlier series, patients received one or two cycles with dactinomycin instead of doxorubicin. Irradiation of the primary site was used for local control. Patients who attained a complete response (CR) to induction therapy were eligible for consolidation with 8 Gy TBI plus VAdriaC and ABMT.
Nineteen patients were ineligible for consolidation after failing to achieve or maintain a CR following induction therapy; all 19 are dead of disease. Seven eligible patients elected to forgo consolidation; three of seven are long-term event-free survivors. Sixty-five patients received consolidation therapy; 20 of 65 are long-term event-free survivors. A local control rate of 83% was achieved using radiation therapy as the primary modality of local control. Patients with metastatic disease at diagnosis fared substantially worse than did patients with localized tumors (6-year event-free survival [EFS] rate, 14% v 38%; two-sided P [P2] = .008).
Consolidation of patients with metastatic or high-risk localized pediatric sarcomas with 8 Gy TBI plus ABMT has failed to improve the outcome of this group of patients. Metastatic disease at diagnosis continues to confer the poorest prognosis. New therapeutic strategies are needed to consolidate more effectively the remissions that can be achieved in the majority of these patients.
为改善转移性或高危局限性尤因肉瘤家族性肿瘤（ESF）及横纹肌肉瘤（RMS）患者的治疗结局，我们探讨了全身照射（TBI）联合自体骨髓移植（ABMT）巩固治疗的作用。
1981年至1986年期间，91例患者纳入三个连续方案之一。诱导治疗包括四或五个周期的长春新碱、多柔比星和环磷酰胺（VAdriaC）；在早期系列研究中，患者接受一或两个周期的放线菌素D而非多柔比星治疗。对原发部位进行照射以实现局部控制。对诱导治疗达到完全缓解（CR）的患者有资格接受8 Gy TBI联合VAdriaC及ABMT进行巩固治疗。
19例患者在诱导治疗后未达到或未维持CR，不符合巩固治疗条件；这19例患者均死于疾病。7例符合条件的患者选择放弃巩固治疗；7例中有3例是长期无事件生存者。65例患者接受了巩固治疗；65例中有20例是长期无事件生存者。以放射治疗作为局部控制的主要方式，局部控制率达到83%。诊断时患有转移性疾病的患者比局限性肿瘤患者的预后差得多（6年无事件生存率[EFS]，14%对38%；双侧P[P2]=0.008）。
用8 Gy TBI联合ABMT对转移性或高危局限性儿童肉瘤患者进行巩固治疗未能改善该组患者的结局。诊断时的转移性疾病仍然预示着最差的预后。需要新的治疗策略来更有效地巩固大多数此类患者所能实现的缓解。
