Haroche Julien, Amoura Zahir, Trad Salim G, Wechsler Bertrand, Cluzel Philippe, Grenier Philippe A, Piette Jean-Charles
Hôpital Pitié-Salpêtrière, Paris, France.
Arthritis Rheum. 2006 Oct;54(10):3330-6. doi: 10.1002/art.22165.
Erdheim-Chester disease (ECD) is a rare, non-Langerhans form of histiocytosis of unknown origin, characterized by infiltration of tissues by spumous histiocytes. ECD features heterogeneous systemic manifestations, and the general prognosis remains poor despite various treatment options.
We treated 8 patients with multisystemic ECD with subcutaneous interferon-alpha (IFNalpha) at a dosage of 3-9 x 10(6) units 3 times weekly, for a median duration of 23 months (range 1-46 months).
Treatment was generally well tolerated, and side effects remained limited to fever following injections. Treatment was discontinued in 1 patient, because of severe depression. During treatment, some manifestations of ECD disappeared (i.e., xanthelasma, exophthalmos, papilledema, and intracranial hypertension). The efficacy of IFNalpha on cardiovascular ECD was variable, however. Treatment resulted in partial regression of "coated aorta" in some cases and clear failure in others; 2 patients died. The level of C-reactive protein diminished sharply in 5 patients.
IFNalpha might be a valuable first-line therapy for prolonged treatment of ECD. However, the efficacy of IFNalpha varies among patients and according to the sites of disease involvement, and symptoms may fail to respond to treatment, especially in patients with severe multisystemic forms of ECD with central nervous system and cardiovascular involvement.
厄尔海姆-切斯特病（ECD）是一种罕见的、起源不明的非朗格汉斯细胞组织细胞增多症，其特征为泡沫状组织细胞浸润组织。ECD具有异质性的全身表现，尽管有多种治疗选择，但总体预后仍然较差。
我们对8例多系统ECD患者采用皮下注射α干扰素（IFNα）进行治疗，剂量为3 - 9×10⁶单位，每周3次，中位治疗持续时间为23个月（范围1 - 46个月）。
治疗总体耐受性良好，副作用仅限于注射后发热。1例患者因严重抑郁而停药。治疗期间，ECD的一些表现消失（即睑黄瘤、眼球突出、视乳头水肿和颅内高压）。然而，IFNα对心血管ECD的疗效不一。治疗在某些情况下导致“主动脉包被征”部分消退，而在其他情况下则明显无效；2例患者死亡。5例患者的C反应蛋白水平急剧下降。
IFNα可能是ECD长期治疗的一种有价值的一线治疗方法。然而，IFNα的疗效因患者个体以及疾病受累部位而异，症状可能对治疗无反应，尤其是在患有严重多系统形式的ECD且累及中枢神经系统和心血管系统的患者中。
