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转移性视网膜母细胞瘤的治疗

Treatment of metastatic retinoblastoma.

作者信息

Rodriguez-Galindo Carlos, Wilson Matthew W, Haik Barrett G, Lipson Mindy J, Cain Alvida, Merchant Thomas E, Kaste Sue, Pratt Charles B

机构信息

Department of Hematology-Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee 38105, USA.

出版信息

Ophthalmology. 2003 Jun;110(6):1237-40. doi: 10.1016/S0161-6420(03)00258-6.

DOI:10.1016/S0161-6420(03)00258-6
PMID:12799253
Abstract

PURPOSE

The risk for death in patients with retinoblastoma is increased in those who present with metastatic disease, and the role of intensive chemotherapy and autologous hematopoietic stem cell rescue in these patients remains unclear.

DESIGN

Nonrandomized interventional case series.

PARTICIPANTS

Four consecutive patients with metastatic retinoblastoma.

METHODS

We treated four patients with retinoblastoma metastatic to the bone and bone marrow with intensive chemotherapy, consolidation with megatherapy, and autologous hematopoietic stem cell rescue. Chemotherapy included courses of carboplatin and etoposide alternating with cyclophosphamide, etoposide, and either carboplatin or cisplatin. Radiation therapy was delivered to areas of bone metastases.

MAIN OUTCOME MEASURES

Patient survival.

RESULTS

All patients completed and responded to the scheduled therapy; complete response of the bone marrow disease was documented after two courses of chemotherapy in all cases. Two patients are long-term survivors.

CONCLUSIONS

The treatment described has been successful in obtaining disease-free survival in patients with metastatic retinoblastoma.

摘要

目的

患有视网膜母细胞瘤且出现转移性疾病的患者死亡风险会增加,强化化疗和自体造血干细胞救援在这些患者中的作用仍不明确。

设计

非随机干预性病例系列。

参与者

连续4例转移性视网膜母细胞瘤患者。

方法

我们对4例发生骨和骨髓转移的视网膜母细胞瘤患者进行了强化化疗、大剂量治疗巩固及自体造血干细胞救援。化疗方案包括卡铂和依托泊苷疗程与环磷酰胺、依托泊苷及卡铂或顺铂交替使用。对骨转移部位进行放射治疗。

主要观察指标

患者生存率。

结果

所有患者均完成了预定治疗并出现反应;所有病例在两个化疗疗程后均记录到骨髓疾病完全缓解。2例患者为长期存活者。

结论

所描述的治疗方法已成功使转移性视网膜母细胞瘤患者实现无病生存。

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1
Treatment of metastatic retinoblastoma.转移性视网膜母细胞瘤的治疗
Ophthalmology. 2003 Jun;110(6):1237-40. doi: 10.1016/S0161-6420(03)00258-6.
2
Intensive multimodality therapy for patients with stage 4a metastatic retinoblastoma.4a 期转移性视网膜母细胞瘤患者的强化多模态治疗。
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High-dose chemotherapy with autologous stem cell rescue in children with retinoblastoma.视网膜母细胞瘤患儿的大剂量化疗及自体干细胞救援
Bone Marrow Transplant. 2003 Feb;31(4):281-4. doi: 10.1038/sj.bmt.1703832.
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Metastatic retinoblastoma clinical features, treatment, and prognosis.转移性视网膜母细胞瘤的临床特征、治疗及预后
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A multidisciplinary treatment strategy that includes high-dose chemotherapy for metastatic retinoblastoma without CNS involvement.一种多学科治疗策略,包括对无中枢神经系统受累的转移性视网膜母细胞瘤进行大剂量化疗。
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Br J Ophthalmol. 2009 Sep;93(9):1163-6. doi: 10.1136/bjo.2008.148932. Epub 2009 Jun 24.
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High-dose chemotherapy with autologous hematopoietic stem cell rescue for stage 4B retinoblastoma.大剂量化疗联合自体造血干细胞移植治疗 4B 期视网膜母细胞瘤。
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Recurrent disseminated retinoblastoma in a 7-year-old girl treated successfully by high-dose chemotherapy and CD34-selected autologous peripheral blood stem cell transplantation.一名7岁女孩复发性播散性视网膜母细胞瘤经大剂量化疗及CD34选择的自体外周血干细胞移植治疗成功。
Bone Marrow Transplant. 2001 Mar;27(6):653-5. doi: 10.1038/sj.bmt.1702824.

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Metastatic retinoblastoma at presentation: Clinical presentation, treatment, and outcomes.初诊时的转移性视网膜母细胞瘤:临床表现、治疗及预后
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Lack of complete response pretransplant is not associated with inferior overall survival for stage 4a metastatic retinoblastoma.
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Pediatr Blood Cancer. 2023 Jan;70(1):e29921. doi: 10.1002/pbc.29921. Epub 2022 Aug 8.
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