Kuttesch John F, Krailo Mark D, Madden Timothy, Johansen Mary, Bleyer Archie
Department of Pediatrics, Vanderbilt School of Medicine and Vanderbilt Ingram Cancer Center, Nashville, Tennessee, USA.
Pediatr Blood Cancer. 2009 Oct;53(4):590-3. doi: 10.1002/pbc.22133.
A Phase II trial was developed to determine the efficacy and toxicity of intravenous vinorelbine, a semi-synthetic vinca alkaloid, in children, adolescent, and young adults with recurrent or refractory solid malignancies.
Fifty patients were enrolled among three strata: soft tissue sarcomas [rhabdomyosarcoma (RMS), non-rhabdomyosarcoma, primitive neuroepithelial tumor] (20 patients); brain tumors [astrocytoma (4 patients), medulloblastoma (2 patients), other (16 patients)] (22 patients); neuroblastoma (8 patients). Vinorelbine was given weekly for 6 consecutive weeks during an 8-week interval. The response rate and toxicity profile was assessed.
Among the first 35 patients treated at 33.75 mg/m(2)/dose, 25 experienced grades 3-4 neutropenia (75%). The dose was decreased to 30 mg/m(2)/dose in the remaining 15 patients. The median age was 10 years (range, 1-25). Four responses (one complete, three partial) occurred within the soft tissue sarcoma strata (all with RMS) and two occurred in the brain tumor group (medulloblastoma and astrocytoma). The most common toxicities were hematological and neurological.
Vinorelbine at dose of 30 mg/m(2) can be safely administered to children with recurrent or refractory solid malignancies. The study design identified vinorelbine to be active in the sarcoma category, with a response rate of 36% (4/11) among RMS patients.
开展了一项II期试验，以确定半合成长春花生物碱静脉注射长春瑞滨对复发或难治性实体恶性肿瘤儿童、青少年及年轻成人的疗效和毒性。
50名患者被纳入三个分层：软组织肉瘤[横纹肌肉瘤（RMS）、非横纹肌肉瘤、原始神经上皮肿瘤]（20例）；脑肿瘤[星形细胞瘤（4例）、髓母细胞瘤（2例）、其他（16例）]（22例）；神经母细胞瘤（8例）。在8周的间隔期内，长春瑞滨每周给药1次，连续给药6周。评估缓解率和毒性特征。
在最初接受33.75mg/m²/剂量治疗的35例患者中，25例出现3-4级中性粒细胞减少（75%）。其余15例患者的剂量降至30mg/m²/剂量。中位年龄为10岁（范围1-25岁）。软组织肉瘤分层内出现4例缓解（1例完全缓解，3例部分缓解）（均为RMS），脑肿瘤组出现2例缓解（髓母细胞瘤和星形细胞瘤）。最常见的毒性反应为血液学和神经学毒性反应。
30mg/m²剂量的长春瑞滨可安全用于复发或难治性实体恶性肿瘤患儿。该研究设计确定长春瑞滨在肉瘤类别中具有活性，RMS患者的缓解率为36%（4/11）。
