Servicio de Reumatología, Hospital J. M. Ramos Mejia, Urquiza 609, Ciudad Autónoma de Buenos Aires, Argentina.
Servicio de Reumatología, Hospital J. M. Ramos Mejia, Urquiza 609, Ciudad Autónoma de Buenos Aires, Argentina.
Reumatol Clin (Engl Ed). 2022 May;18(5):286-292. doi: 10.1016/j.reumae.2021.01.006.
Off-label (OL) drug use is the prescription of a drug for indications other than those authorised in its technical datasheet. The objective of this study was to identify drugs recommended in rheumatology but considered for off-label use in Argentina.
A list of medications for certain selected rheumatic conditions was compiled. A drug was considered recommended if it was endorsed by a) at least one Argentine or Pan-American treatment guideline or consensus, or b) two international treatment guidelines, or c) one international treatment guideline and one selected textbook. Approval of these drugs for any condition in Argentina until December 31st, 2018 was explored, and medicines were divided into those with on-label indications and those considered for OL use.
One hundred and thirty-six medications were analysed in 13 clinical conditions. Sixty-seven OL recommendations (49%) were found, and several drugs had more than one. All the conditions included the recommendation of at least 1 OL drug except osteoporosis and rheumatoid arthritis. The frequency of OL recommendations for the following conditions was 100%: calcium pyrophosphate dihydrate crystal deposition disease, polymyalgia rheumatica, Sjögren syndrome, and systemic sclerosis. The drugs with the highest number of OL recommendations were methotrexate (in 7 conditions), and glucocorticoids and mycophenolate (in 4). There were 2 OL recommendations for rituximab and 1 for abatacept.
Almost all the rheumatic disorders analysed involved the recommendation of at least 1 OL medication, and in 4 conditions all the recommendations were OL. Most OL drugs recommended in rheumatology are neither biological nor small-molecule therapies.
超适应证（OL）用药是指将一种药物用于其技术说明书中未授权的适应证。本研究的目的是确定在阿根廷推荐用于风湿病但被认为是超适应证使用的药物。
编制了某些选定风湿病药物的清单。如果药物得到以下至少一种的认可，则被认为是推荐的：a）至少一份阿根廷或泛美治疗指南或共识，或 b）两份国际治疗指南，或 c）一份国际治疗指南和一本选定的教科书。研究了这些药物在阿根廷治疗任何疾病的适应证批准情况，截至 2018 年 12 月 31 日，将药物分为有适应证和超适应证使用的药物。
在 13 种临床情况下分析了 136 种药物。发现了 67 种超适应证推荐（49%），其中一些药物有不止一种适应证。除骨质疏松症和类风湿关节炎外，所有情况下都推荐了至少 1 种超适应证药物。下列情况下 100%推荐了超适应证药物：焦磷酸钙二水化合物晶体沉积病、巨细胞动脉炎、干燥综合征和系统性硬化症。超适应证推荐药物中使用最多的是甲氨蝶呤（7 种疾病），其次是糖皮质激素和霉酚酸酯（4 种疾病）。利妥昔单抗和阿巴西普各有 2 种超适应证推荐。
几乎所有分析的风湿病疾病都涉及至少 1 种超适应证药物的推荐，在 4 种情况下，所有推荐都是超适应证。风湿病学中推荐的大多数超适应证药物既不是生物制剂也不是小分子药物。
