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移植前未完全缓解与 4a 期转移性视网膜母细胞瘤的整体生存预后不良无关。

Lack of complete response pretransplant is not associated with inferior overall survival for stage 4a metastatic retinoblastoma.

机构信息

Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, New York, USA.

Department of Surgery, Memorial Sloan Kettering Cancer Center, New York City, New York, USA.

出版信息

Pediatr Blood Cancer. 2023 Jan;70(1):e29921. doi: 10.1002/pbc.29921. Epub 2022 Aug 8.

Abstract

BACKGROUND

Stage 4a metastatic retinoblastoma (RB) is curable with intensive multimodality therapy including myeloablative chemotherapy with autologous stem cell transplant (HDC-ASCT) and involved field radiation therapy (IFRT). To our knowledge, no data exist on the impact of (a) pre-ASCT disease status, and (b) IFRT to sites of metastatic disease post ASCT on survival.

PROCEDURE

We retrospectively reviewed patients with stage 4a metastatic RB who underwent induction chemotherapy followed by HDC-ASCT, with or without IFRT, to residual tumor sites at Memorial Sloan Kettering Cancer Center (MSKCC) (n = 24).

RESULTS

The degree of postinduction response prior to ASCT did not affect outcome, with 5-year overall survival (OS) of 68% and 86% in patients who achieved complete response (CR) and very good partial response (VGPR)/partial response (PR) prior to ASCT, respectively. IFRT administered post ASCT in patients with possible residual bony metastatic disease increases the likelihood of developing osteosarcoma in the radiation field.

CONCLUSION

OS for patients with stage 4a metastatic RB treated with ASCT with VGPR or PR to pretransplant chemotherapy was not significantly different from patients with CR. In addition, IFRT does not seem to be required for bony disease control and increased the likelihood of developing osteosarcoma.

摘要

背景

4a 期转移性视网膜母细胞瘤(RB)可通过强化多模式治疗治愈,包括含自体干细胞移植(HDC-ASCT)的骨髓清除化疗和累及野放射治疗(IFRT)。据我们所知,尚无关于(a)ASCT 前疾病状态和(b)ASCT 后转移性疾病部位 IFRT 对生存的影响的数据。

程序

我们回顾性分析了在纪念斯隆凯特琳癌症中心(MSKCC)接受诱导化疗后行 HDC-ASCT 治疗,伴或不伴残余肿瘤部位 IFRT 的 4a 期转移性 RB 患者(n=24)。

结果

ASCT 前诱导后反应程度不影响预后,分别有 5 年总生存率(OS)为 68%和 86%的患者在 ASCT 前达到完全缓解(CR)和很好的部分缓解(VGPR)/部分缓解(PR)。ASCT 后在可能有残留骨转移疾病的患者中给予 IFRT 会增加放射野发生骨肉瘤的可能性。

结论

接受 ASCT 治疗的 4a 期转移性 RB 患者,接受移植前化疗的 VGPR 或 PR 与 CR 患者的 OS 无显著差异。此外,IFRT 似乎对骨病控制没有必要,反而增加了发生骨肉瘤的可能性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a06f/9701149/921145d0208c/nihms-1825847-f0001.jpg

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