吉西他滨-奥沙利铂作为婴儿型脑肿瘤自体造血干细胞移植的桥接治疗。

Gemcitabine-oxaliplatin as a bridge therapy toward autologous hematopoietic stem cell transplantation in infant-type brain tumors.

作者信息

Castelli Barbara, Fonte Carla, Tellini Marco, Di Nicola Marco, Guidi Milena, Giunti Laura, Tirinnanzi Bianca, Marzano Chiara, Buccoliero Anna Maria, D'Incerti Ludovico, Giordano Flavio, Scagnet Mirko, Tintori Veronica, Genitori Lorenzo, Sardi Iacopo

机构信息

Neuro-Oncology Unit, Meyer Children's Hospital IRCCS, Florence, Italy.

Pathology Unit, Meyer Children's Hospital IRCCS, Florence, Italy.

出版信息

Front Oncol. 2025 May 13;15:1476411. doi: 10.3389/fonc.2025.1476411. eCollection 2025.

Abstract

INTRODUCTION

In neuro-oncological pediatric patients under 3 years of age, chemotherapy intensified to high doses (high-dose chemotherapy, HDC) represents the cornerstone to avoid the potential toxicity of radiotherapy. Combination treatment with gemcitabine-oxaliplatin (GemOx) was administered for infant- type cerebral tumors as a bridge toward autologous hematopoietic transplantation to achieve clinical and neuroradiological permissiveness to HDC and to raise the possibility of second-look neurosurgery.

METHODS

From May 2017 to May 2023, at Meyer Children's Hospital IRCSS in Florence (Italy), four patients, with a median age of 19 months (with two high- grade gliomas, a metastatic medulloblastoma, and a choroid plexus carcinoma CNS WHO grade 3), were subjected to partial neurosurgical removal and induction therapy delivered according to the Italian program for malignant cerebral tumors under 3 years. To delay HDC, either for disease reassessment or for temporary unfitness, GemOx cycles were administered. Gemcitabine 1,000 mg/m2 and oxaliplatin 100 mg/m2 were given on day 1 every 21-28 days for one to six cycles.

RESULTS

The treatment was well tolerated overall, except for severe platelet hematological toxicity in a patient, which required dose reduction to 75%. After GemOx, one patient was also subjected to further neurosurgery. Bridge therapy made it possible to submit patients to HDC in safety, in permissive clinical conditions, and after assessment of disease stability.

CONCLUSION

In infant-type cerebral tumors eligible for HDC, GemOx could be a possible strategy in the case of post-induction residual disease to exclude uncertain evolution or when waiting for clinical suitability for second surgery and intensified treatment. The therapy was overall safe and well tolerated. This approach resulted incisive in the therapeutic or palliative choice for extremely young patients with aggressive brain tumors.

摘要

引言

在3岁以下的神经肿瘤学儿科患者中,强化至高剂量的化疗(高剂量化疗,HDC)是避免放疗潜在毒性的基石。吉西他滨 - 奥沙利铂(GemOx)联合治疗用于婴儿型脑肿瘤,作为通向自体造血移植的桥梁,以实现临床和神经放射学上对HDC的允许,并提高二次神经外科手术的可能性。

方法

2017年5月至2023年5月,在意大利佛罗伦萨的迈耶儿童医院IRCSS,4名患者(中位年龄19个月,包括2例高级别胶质瘤、1例转移性髓母细胞瘤和1例中枢神经系统WHO 3级脉络丛癌)接受了部分神经外科切除,并根据意大利3岁以下恶性脑肿瘤治疗方案进行诱导治疗。为了延迟HDC,无论是为了疾病重新评估还是暂时不适合,均给予GemOx周期治疗。吉西他滨1000 mg/m²和奥沙利铂100 mg/m²在第1天给予,每21 - 28天一次,共进行1至6个周期。

结果

总体而言,治疗耐受性良好,除了一名患者出现严重的血小板血液学毒性,需要将剂量降低至75%。GemOx治疗后,一名患者还接受了进一步的神经外科手术。桥接治疗使患者能够在安全、允许的临床条件下,并在评估疾病稳定性后接受HDC。

结论

在适合HDC的婴儿型脑肿瘤中,对于诱导后残留疾病以排除不确定演变的情况,或者在等待二次手术和强化治疗的临床适宜性时,GemOx可能是一种可行的策略。该治疗总体安全且耐受性良好。这种方法对于患有侵袭性脑肿瘤的极年轻患者在治疗或姑息选择方面具有决定性作用。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3dab/12106401/01956cd06d19/fonc-15-1476411-g001.jpg

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