Osband M E
Pediatric Hematology/Oncology, Boston University School of Medicine, Massachusetts.
Hematol Oncol Clin North Am. 1987 Dec;1(4):737-51.
Histiocytosis X is a complex and poorly understood entity. Nevertheless, it would appear as if certain themes are found recurrently throughout the literature dealing with this disease and a review of them serves as a useful summary. 1. Problems with Nomenclature. To name or categorize a disease based on end-organ pathology is generally not clinically useful, but this is what we have done with histiocytosis X. It has caused substantial confusion among physicians and patients alike concerning diagnosis, prognosis, and treatment. Further attempts at improving the nosology of this disease will not be useful unless those new names also reflect scientific advances in our understanding of etiology, pathogenesis, and therapy. 2. Identification of the Langerhans' Cell as the Consistent Pathognomonic Cell in the Lesions of Histiocytosis X. Although the Langerhans' cell was identified more than a century ago, it has only recently been recognized as the cell that proliferates in this disease. Nevertheless, several important questions remain regarding the relationship of the Langerhans' cell to histiocytosis. Foremost among these questions is whether the Langerhans' cell is a truly normal Langerhans' cell, responding appropriately to immune system signals, or if it is an abnormal variant, possibly even neoplastic. 3. Recognition that Immune System Dysfunction Is a Critical Part of Histiocytosis X. The immune system is the focus of most recent clinical research. Results of these studies are obviously important with regard to both the biology and management of this disease. 4. Histiocytosis X Is an Extremely Heterogeneous Clinical Disorder. As mentioned before, the term histiocytosis X was originally intended by Lichtenstein to describe a pathologic, and not clinical, entity. It is rare to find two patients with this disease who are exactly alike. To make matters even more confusing, the disease includes both infants with disseminated fatal disease as well as middle-aged adults with solitary bony lesions. 5. The Disease Requires Improved Therapy, but it Is a Difficult Setting in which to Perform Clinical Studies. Improved therapy is required in patients with this disease, especially those with the disseminated form. But it will be difficult to develop improved therapy until definitive answers are provided to some of the basic questions of etiology and pathogenesis. Unfortunately, these clinical studies are not readily available because of the rare occurrence of this disease and its extreme clinical heterogeneity.(ABSTRACT TRUNCATED AT 400 WORDS)
组织细胞增多症X是一种复杂且了解甚少的病症。然而，在有关这种疾病的文献中似乎反复出现某些主题，对这些主题进行综述可作为一个有用的总结。1. 命名问题。基于终末器官病理学对一种疾病进行命名或分类通常在临床上并无用处，但我们对组织细胞增多症X就是这么做的。这在医生和患者中都造成了关于诊断、预后和治疗的严重混乱。除非新的名称也能反映我们在病因学、发病机制和治疗理解方面的科学进展，否则进一步改进这种疾病的疾病分类学将毫无用处。2. 将朗格汉斯细胞鉴定为组织细胞增多症X病变中一致的病理诊断性细胞。尽管朗格汉斯细胞在一个多世纪前就已被鉴定出来，但直到最近才被确认为在这种疾病中增殖的细胞。然而，关于朗格汉斯细胞与组织细胞增多症的关系仍有几个重要问题。这些问题中最首要的是朗格汉斯细胞是真正正常的朗格汉斯细胞，对免疫系统信号做出适当反应，还是它是一种异常变体，甚至可能是肿瘤性的。3. 认识到免疫系统功能障碍是组织细胞增多症X的关键部分。免疫系统是最近临床研究的重点。这些研究结果对于这种疾病的生物学和治疗显然都很重要。4. 组织细胞增多症X是一种极其异质性的临床病症。如前所述，“组织细胞增多症X”一词最初由利希滕斯坦用来描述一种病理学而非临床实体。很少能找到两个完全相同的患有这种疾病的患者。更令人困惑的是，这种疾病既包括患有播散性致命疾病的婴儿，也包括患有孤立性骨病变的中年成年人。5. 这种疾病需要改进治疗方法，但进行临床研究的环境很困难。患有这种疾病的患者，尤其是那些患有播散性形式的患者，需要改进治疗方法。但在对病因学和发病机制的一些基本问题提供明确答案之前，很难开发出改进的治疗方法。不幸的是，由于这种疾病罕见且临床异质性极大，这些临床研究并不容易开展。（摘要截选至400字）
