Division of Pediatric Hematology/Oncology, King Hussein Cancer Center, Amman, Jordan.
Division of Radiology, King Hussein Cancer Center, Amman, Jordan.
JCO Glob Oncol. 2021 Sep;7:1442-1453. doi: 10.1200/GO.21.00127.
Medulloblastoma is composed of four clinically and prognostically distinct molecular subgroups (WNT, SHH, group 3, and group 4). However, the clinical implications of these subgroups in the context of the unique challenges of low- to middle-income countries are rarely reported.
We assembled an institutional cohort of children (3-18 years) diagnosed with medulloblastoma and treated in Jordan between 2003 and 2016. Tumors were subgrouped by NanoString and correlated with clinical and radiologic characteristics.
Eighty-eight patients were identified (63% male); median age was 6.9 years (interquartile range 4.8-9.2) and median symptom duration was 6 weeks (interquartile range 4-11). Radiotherapy was implemented as standard-risk in 41 patients (47%) and high-risk in 47 patients (53%). Subgrouping revealed 17 WNT (19%), 22 SHH (25%), 21 group 3 (24%), and 28 group 4 tumors (32%). Median time between craniotomy and radiotherapy was 45 days (17-155); 44% of them > 49 days. Median duration of radiotherapy was 44 days (36-74). Seventy-two patients (82%) received chemotherapy afterward. With a median follow-up of 4.6 years (0.2-14.9), 5-year progression-free survival (PFS) and overall survival were 73.5% and 69.4%, respectively, with no statistically significant survival difference between standard-risk and high-risk patients. Metastasis was significant for overall survival ( = .011). Patients with SHH and group 4 tumors had very good PFS (83.4% and 87.0%, respectively) and those with group 3 tumors had dismal outcomes (PFS 44.9%), whereas WNT tumors had less-than expected PFS (70.5%). PFS was statistically significant in patients with nonmetastatic tumors receiving radiotherapy ≤ 49 days ( = .011), particularly group 3 tumors.
Patients with SHH and group 4 medulloblastoma had excellent survival comparable with high-income countries. Compliance with treatment protocols and avoiding radiotherapy delays are important in achieving adequate survival in low- to middle-income country settings. Subgroup-driven treatment protocols should be considered in countries with limited resources.
成神经管细胞瘤由四个在临床上和预后上明显不同的分子亚组（WNT、SHH、第 3 组和第 4 组）组成。然而，在中低收入国家的独特挑战背景下，这些亚组的临床意义很少有报道。
我们收集了一组在 2003 年至 2016 年期间在约旦诊断为成神经管细胞瘤并接受治疗的儿童（3-18 岁）的机构队列。通过 NanoString 对肿瘤进行分组，并与临床和影像学特征相关联。
共确定了 88 名患者（63%为男性）；中位年龄为 6.9 岁（四分位距 4.8-9.2），中位症状持续时间为 6 周（四分位距 4-11）。41 名患者（47%）接受标准风险放疗，47 名患者（53%）接受高风险放疗。亚组分析显示 17 例 WNT（19%）、22 例 SHH（25%）、21 例第 3 组（24%）和 28 例第 4 组肿瘤（32%）。从开颅手术到放疗的中位时间为 45 天（17-155）；其中 44%的患者>49 天。放疗的中位持续时间为 44 天（36-74）。72 名患者（82%）随后接受化疗。中位随访时间为 4.6 年（0.2-14.9），5 年无进展生存率（PFS）和总生存率分别为 73.5%和 69.4%，标准风险和高风险患者的生存率无统计学差异。转移对总生存率有显著影响（P=0.011）。SHH 和第 4 组肿瘤患者的 PFS 非常好（分别为 83.4%和 87.0%），而第 3 组肿瘤患者的 PFS 结果较差（PFS 44.9%），而 WNT 肿瘤患者的 PFS 低于预期（70.5%）。无转移肿瘤患者接受放疗≤49 天的 PFS 有统计学意义（P=0.011），特别是第 3 组肿瘤。
SHH 和第 4 组成神经管细胞瘤患者的生存率与高收入国家相当。在中低收入国家，遵守治疗方案和避免放疗延迟对于获得足够的生存率非常重要。在资源有限的国家应考虑采用基于亚组的治疗方案。
Zotero 插件