Department of Biotechnological and Applied Clinical Sciences, University of L'Aquila, L'Aquila, Italy.
Rheumatology Department, Cochin Hospital, APHP, INSERM U1016, Université Paris Cité, Paris, France.
Autoimmun Rev. 2024 Jul-Aug;23(7-8):103581. doi: 10.1016/j.autrev.2024.103581. Epub 2024 Jul 26.
Inflammatory rheumatic diseases are different pathologic conditions associated with a deregulated immune response, codified along a spectrum of disorders, with autoinflammatory and autoimmune diseases as two-end phenotypes of this continuum. Despite pathogenic differences, inflammatory rheumatic diseases are commonly managed with a limited number of immunosuppressive drugs, sometimes with partial evidence or transferring physicians' knowledge in different patients. In addition, several randomized clinical trials, enrolling these patients, did not meet the primary pre-established outcomes and these findings could be linked to the underlying molecular diversities along the spectrum of inflammatory rheumatic disorders. In fact, the resulting patient heterogeneity may be driven by differences in underlying molecular pathology also resulting in variable responses to immunosuppressive drugs. Thus, the identification of different clinical subsets may possibly overcome the major obstacles that limit the development more effective therapeutic strategies for these patients with inflammatory rheumatic diseases. This clinical heterogeneity could require a diverse therapeutic management to improve patient outcomes and increase the frequency of clinical remission. Therefore, the importance of better patient stratification and characterization is increasingly pointed out according to the precision medicine principles, also suggesting a new approach for disease treatment. In fact, based on a better proposed patient profiling, clinicians could more appropriately balance the therapeutic management. On these bases, we synthetized and discussed the available literature about the patient profiling in regard to therapy in the context of inflammatory rheumatic diseases, mainly focusing on randomized clinical trials. We provided an overview of the importance of a better stratification and characterization of the clinical heterogeneity of patients with inflammatory rheumatic diseases identifying this point as crucial in improving the management of these patients.
炎症性风湿病是一组不同的病理状况，与免疫反应失调有关，沿着一系列疾病谱编码，其中自体炎症和自身免疫性疾病是这一连续体的两个极端表型。尽管发病机制不同，但炎症性风湿病通常采用有限数量的免疫抑制剂进行治疗，有时在不同患者中仅部分证据或转移医生的知识。此外，几项纳入这些患者的随机临床试验并未达到预先设定的主要终点，这些发现可能与炎症性风湿病谱中潜在的分子多样性有关。事实上，患者的异质性可能是由潜在分子病理学的差异驱动的，这也导致了对免疫抑制剂的反应不同。因此，确定不同的临床亚组可能有可能克服限制为这些炎症性风湿病患者开发更有效治疗策略的主要障碍。这种临床异质性可能需要多样化的治疗管理，以改善患者的预后并增加临床缓解的频率。因此，根据精准医学原则，更好地对患者进行分层和特征描述的重要性日益凸显，也为疾病治疗提出了新的方法。事实上，基于更好的患者特征描述，临床医生可以更恰当地平衡治疗管理。在此基础上，我们综合讨论了炎症性风湿病治疗中关于患者特征描述的现有文献，主要侧重于随机临床试验。我们概述了更好地分层和描述炎症性风湿病患者临床异质性的重要性，认为这一点对于改善这些患者的管理至关重要。
