{"title":"用数字液滴PCR检测多发性骨髓瘤BM-FFPE组织中BRAF V600E和NRAS Q61R突变的流行、检测和临床意义","authors":"Niyati S Sharma, K S Nataraj, Bibha Choudhary","doi":"10.1007/s12032-025-03032-5","DOIUrl":null,"url":null,"abstract":"<p><p>Multiple myeloma (MM) exhibits significant genetic heterogeneity with mutations in the MAPK pathway being increasingly recognized as drivers of progression and treatment resistance. This study is the first to employ droplet digital PCR (DD-PCR) on bone marrow formalin-fixed paraffin-embedded (BM-FFPE) tissue to quantify BRAF V600E and NRAS Q61R mutations in MM, and the first such investigation conducted in an Indian MM cohort. Mutations were identified in > 80% of samples. BRAF V600E displayed stage-specific variation, being lowest in intermediate MM and significantly higher in advanced disease (MMII vs MMIII, p = 0.0453). Burden declined after treatment (p = 0.038) and correlated with stage (β = - 36.95, p = 0.0041) and lytic lesions (β = - 25.97, p = 0.0268). NRAS Q61R was enriched in progressive disease (p = 0.02) and showed an upward trend with advancing stage (β = 14.38, p = 0.0686). Higher BRAF and NRAS burdens were associated with reduced 2-year progression-free survival (PFS). We show that DD-PCR applied to BM-FFPE samples is both feasible and informative. However, our study has inherent limitations: DD-PCR measurements can be influenced by sampling biases, droplet variability and FFPE DNA quality. Our single-centre design and small cohort size limit generalizability but demonstrate the potential for DD-PCR to complement sequencing and clinical stratification if validated in larger populations.</p>","PeriodicalId":18433,"journal":{"name":"Medical Oncology","volume":"42 11","pages":"478"},"PeriodicalIF":3.5000,"publicationDate":"2025-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Prevalence, detection, and clinical implications of BRAF V600E and NRAS Q61R mutations in multiple myeloma BM-FFPE tissues using digital droplet PCR.\",\"authors\":\"Niyati S Sharma, K S Nataraj, Bibha Choudhary\",\"doi\":\"10.1007/s12032-025-03032-5\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Multiple myeloma (MM) exhibits significant genetic heterogeneity with mutations in the MAPK pathway being increasingly recognized as drivers of progression and treatment resistance. This study is the first to employ droplet digital PCR (DD-PCR) on bone marrow formalin-fixed paraffin-embedded (BM-FFPE) tissue to quantify BRAF V600E and NRAS Q61R mutations in MM, and the first such investigation conducted in an Indian MM cohort. Mutations were identified in > 80% of samples. BRAF V600E displayed stage-specific variation, being lowest in intermediate MM and significantly higher in advanced disease (MMII vs MMIII, p = 0.0453). Burden declined after treatment (p = 0.038) and correlated with stage (β = - 36.95, p = 0.0041) and lytic lesions (β = - 25.97, p = 0.0268). NRAS Q61R was enriched in progressive disease (p = 0.02) and showed an upward trend with advancing stage (β = 14.38, p = 0.0686). Higher BRAF and NRAS burdens were associated with reduced 2-year progression-free survival (PFS). We show that DD-PCR applied to BM-FFPE samples is both feasible and informative. However, our study has inherent limitations: DD-PCR measurements can be influenced by sampling biases, droplet variability and FFPE DNA quality. Our single-centre design and small cohort size limit generalizability but demonstrate the potential for DD-PCR to complement sequencing and clinical stratification if validated in larger populations.</p>\",\"PeriodicalId\":18433,\"journal\":{\"name\":\"Medical Oncology\",\"volume\":\"42 11\",\"pages\":\"478\"},\"PeriodicalIF\":3.5000,\"publicationDate\":\"2025-09-18\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Medical Oncology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1007/s12032-025-03032-5\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"ONCOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Medical Oncology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s12032-025-03032-5","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"ONCOLOGY","Score":null,"Total":0}
引用次数: 0
摘要
多发性骨髓瘤(MM)表现出显著的遗传异质性,MAPK通路的突变越来越被认为是进展和治疗耐药的驱动因素。本研究首次在骨髓福尔马林固定石蜡包埋(BM-FFPE)组织上采用液滴数字PCR (DD-PCR)来量化MM中BRAF V600E和NRAS Q61R突变,也是首次在印度MM队列中进行此类研究。在80%的样本中发现了突变。BRAF V600E表现出分期特异性变异,在中度MM中最低,在晚期疾病中显著升高(MMII vs MMIII, p = 0.0453)。治疗后负担下降(p = 0.038),并与分期(β = - 36.95, p = 0.0041)和溶性病变(β = - 25.97, p = 0.0268)相关。NRAS Q61R在病程进展时表达丰富(p = 0.02),随着病程进展呈上升趋势(β = 14.38, p = 0.0686)。较高的BRAF和NRAS负担与2年无进展生存期(PFS)降低相关。我们表明,将DD-PCR应用于BM-FFPE样品是可行的,而且信息丰富。然而,我们的研究存在固有的局限性:DD-PCR测量可能受到采样偏差、液滴可变性和FFPE DNA质量的影响。我们的单中心设计和小队列规模限制了通用性,但如果在更大的人群中得到验证,则证明了DD-PCR补充测序和临床分层的潜力。
Prevalence, detection, and clinical implications of BRAF V600E and NRAS Q61R mutations in multiple myeloma BM-FFPE tissues using digital droplet PCR.
Multiple myeloma (MM) exhibits significant genetic heterogeneity with mutations in the MAPK pathway being increasingly recognized as drivers of progression and treatment resistance. This study is the first to employ droplet digital PCR (DD-PCR) on bone marrow formalin-fixed paraffin-embedded (BM-FFPE) tissue to quantify BRAF V600E and NRAS Q61R mutations in MM, and the first such investigation conducted in an Indian MM cohort. Mutations were identified in > 80% of samples. BRAF V600E displayed stage-specific variation, being lowest in intermediate MM and significantly higher in advanced disease (MMII vs MMIII, p = 0.0453). Burden declined after treatment (p = 0.038) and correlated with stage (β = - 36.95, p = 0.0041) and lytic lesions (β = - 25.97, p = 0.0268). NRAS Q61R was enriched in progressive disease (p = 0.02) and showed an upward trend with advancing stage (β = 14.38, p = 0.0686). Higher BRAF and NRAS burdens were associated with reduced 2-year progression-free survival (PFS). We show that DD-PCR applied to BM-FFPE samples is both feasible and informative. However, our study has inherent limitations: DD-PCR measurements can be influenced by sampling biases, droplet variability and FFPE DNA quality. Our single-centre design and small cohort size limit generalizability but demonstrate the potential for DD-PCR to complement sequencing and clinical stratification if validated in larger populations.
期刊介绍:
Medical Oncology (MO) communicates the results of clinical and experimental research in oncology and hematology, particularly experimental therapeutics within the fields of immunotherapy and chemotherapy. It also provides state-of-the-art reviews on clinical and experimental therapies. Topics covered include immunobiology, pathogenesis, and treatment of malignant tumors.