Pillai Raveendran K, Jayasree K
Division of Clinical Laboratory, Regional Cancer Centre, Thiruvananthapuram, India.
Division of Pathology, Regional Cancer Centre, Thiruvananthapuram, India.
Indian J Med Res. 2017 Jan;145(1):17-27. doi: 10.4103/ijmr.IJMR_915_14.
Rare cancers account for about 22 per cent of all cancers diagnosed worldwide, disproportionately affecting some demographic groups, with an occurrence of less than 6 per 100,000 individuals annually. Many rare cancers in adults, adolescents and children are not curable, and patients and care providers have little option to take therapeutic decisions. The epidemiology of rare cancers is a challenging area of study but is inadequately addressed. Despite efforts mainly in some European nations, a few improvements have been observed in the management of rare cancers. Reasons for this obvious stagnation are multifactorial and are mainly inherent to logistical difficulties in carrying out clinical trials in very small patient populations, hesitation of the pharmaceutical industry to spend in small markets and complexity in creating adequate information for the development of cost-effective drugs. Rare cancers also face specific challenges that include late and incorrect diagnosis, lack of clinical expertise and lack of research interest and development of new therapies. The utilization of nationally representative study findings for the patients' evaluation may possibly offer chances to find out pathogenesis and prevalence, and this will eventually lead to control and prevention. Currently, advancing targeted therapies offer a great opportunity for the better management of rare cancers. Conducting clinical trials with small patient population, innovative clinical trial approach, prevailing controlling obstacles for international cooperation and financial support for research are the present challenges for rare cancers. The International Rare Cancers Initiative functions as a main platform for achieving new international clinical trials in rare tumours. This review delineates the current challenges and issues in the interpretation, management and research scenarios of rare cancers.
罕见癌症约占全球确诊癌症总数的22%,对某些人群的影响尤为严重,每年发病率低于十万分之六。许多成人、青少年和儿童的罕见癌症无法治愈,患者和医疗服务提供者几乎没有治疗决策的选择。罕见癌症的流行病学是一个具有挑战性的研究领域,但尚未得到充分解决。尽管主要在一些欧洲国家做出了努力,但在罕见癌症的管理方面仅观察到一些改善。这种明显停滞的原因是多方面的,主要源于在非常小的患者群体中开展临床试验存在后勤困难、制药行业不愿在小市场投入、以及为开发性价比高的药物创建充分信息的复杂性。罕见癌症还面临着特定的挑战,包括诊断延迟和错误、缺乏临床专业知识以及缺乏对新疗法的研究兴趣和开发。利用具有全国代表性的研究结果对患者进行评估,可能会为了解发病机制和患病率提供机会,最终实现控制和预防。目前,推进靶向治疗为更好地管理罕见癌症提供了巨大机遇。在小患者群体中开展临床试验、创新临床试验方法、克服国际合作的主要障碍以及为研究提供资金支持,是罕见癌症目前面临的挑战。国际罕见癌症倡议是在罕见肿瘤领域开展新的国际临床试验的主要平台。本综述阐述了罕见癌症在解释、管理和研究方面当前面临的挑战和问题。