Rationale for irradiation of persisting oligo-skeletal metastases to improve survival of metastatic neuroblastoma patients with a poor response to chemotherapy: A retrospective study

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer Pub Date : 2024-10-14 DOI:10.1002/pbc.31350

Lea Rossillon, Véronique Edeline, Laurentiu Agrigoroaie, Claudia Pasqualini, Pablo Berlanga, Stephanie Bolle, Christelle Dufour, Dominique Valteau-Couanet

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Abstract

Background

Persistent metaiodobenzylguanidine (mIBG)-positive skeletal metastases post induction in high-risk neuroblastoma correlate with a poor outcome. The aim of this study was to investigate the potential rationale for a prospective randomized study evaluating the impact on event-free survival of the irradiation of residual oligo-skeletal metastases.

Procedure

Patients over 1 year with a stage M neuroblastoma treated between 2000 and 2020 at Gustave Roussy were identified. Patients with a positive mIBG scan at diagnosis and persistent skeletal metastases after high-dose chemotherapy (HDC) were included. Data were retrospectively collected and mIBG scans reviewed by two nuclear medicine physicians.

Results

Persistent skeletal uptake after HDC was observed in 30/201 patients (15%). Four patients reached a complete response at the end of maintenance treatment and did not relapse (median follow-up [FU] 8 years [1.8–11.8]), while two patients had progressive disease during maintenance. Among the 24 patients with persistent skeletal uptakes at the end of treatment, seven had a persistent response (median FU 8.2 years [4–15.6]). Median SIOPEN (International Society of Paediatric Oncology European Neuroblastoma) scores post consolidation and at the end of treatment were, respectively, 2 [1–6] and 2 [0–4] for patients with persistent responses compared to 4 [1–28] and 2 [1–17] for patients with progressive diseases. Median SIOPEN score at progression was 34 [2–56].

Conclusions

Our study underlines that only a minority of patients had persistent skeletal mIBG-positive scans after HDC. Recurrence mainly occurred in disease sites present at diagnosis that cleared with chemotherapy. On-therapy control of the disease is the main challenge. These results highlight the complexity of conducting a randomized study exploring this strategy.

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查看原文本刊更多论文

对化疗反应不佳的转移性神经母细胞瘤患者进行持续性少骨转移灶照射以提高生存率的理由：一项回顾性研究。

背景：高风险神经母细胞瘤诱导后持续的偏碘苄基胍（mIBG）阳性骨骼转移与不良预后相关。本研究旨在调查一项前瞻性随机研究的潜在理由，评估照射残留的寡骨骼转移瘤对无事件生存期的影响：确定2000年至2020年间在古斯塔夫鲁西医院接受治疗的M期神经母细胞瘤患者。其中包括诊断时mIBG扫描呈阳性、高剂量化疗（HDC）后仍有骨骼转移的患者。由两名核医学医生对数据进行回顾性收集，并对mIBG扫描进行复核：结果：30/201例患者（15%）在HDC后出现持续骨骼摄取。4名患者在维持治疗结束时达到完全应答且未复发（中位随访时间[FU]8年[1.8-11.8]），2名患者在维持治疗期间病情进展。在治疗结束时出现持续骨骼摄取的 24 例患者中，有 7 例出现了持续应答（中位随访时间为 8.2 年 [4-15.6]）。巩固治疗后和治疗结束时，持续应答患者的中位SIOPEN（国际儿科肿瘤学会欧洲神经母细胞瘤）评分分别为2[1-6]和2[0-4]，而进展期患者的中位SIOPEN评分分别为4[1-28]和2[1-17]。进展期SIOPEN评分中位数为34[2-56]：我们的研究强调，只有少数患者在 HDC 后骨骼 mIBG 阳性扫描持续存在。复发主要发生在诊断时就存在的疾病部位，这些部位在化疗后已经消失。在治疗过程中控制病情是主要挑战。这些结果凸显了对这一策略进行随机研究的复杂性。

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来源期刊

Pediatric Blood & Cancer 医学-小儿科

CiteScore

4.90

自引率

9.40%

发文量

546

审稿时长

1.5 months

期刊介绍： Pediatric Blood & Cancer publishes the highest quality manuscripts describing basic and clinical investigations of blood disorders and malignant diseases of childhood including diagnosis, treatment, epidemiology, etiology, biology, and molecular and clinical genetics of these diseases as they affect children, adolescents, and young adults. Pediatric Blood & Cancer will also include studies on such treatment options as hematopoietic stem cell transplantation, immunology, and gene therapy.