Berz F, Weiss M, Belohradsky B H
Abteilung für Infektionsimmunologie, Ludwig-Maximilians-Universität München.
Klin Padiatr. 1996 Mar-Apr;208(2):83-7. doi: 10.1055/s-2008-1044000.
The Hermansky-Pudlak Syndrome (HPS) is a rare autosomal recessively inherited triad of oculocutaneous albinism hemorrhagic diathesis and accumulation of ceroid in tissues. This article comprising the published reports of 232 patients is extended by the personal observation of a 7-year-old boy with HPS and immune deficiency due to a reduced activity of "natural killer" cells. In most cases the syndrome belongs to a form of tyrosinase-positive albinism with great clinical variability. The prolonged bleeding time is due to the lack of storage organelles (dense bodies) in thrombocytes. The most frequent complication of ceroid storage is fibrotic, restrictive lung disease. So far no curative therapy exists; the nature of the defect is still unknown.
赫尔曼斯基-普德拉克综合征(HPS)是一种罕见的常染色体隐性遗传三联征,包括眼皮肤白化病、出血素质以及类蜡质在组织中的蓄积。本文涵盖了232例患者的已发表报告,并通过对一名患有HPS且因“自然杀伤”细胞活性降低而存在免疫缺陷的7岁男孩的个人观察进行了补充。在大多数情况下,该综合征属于酪氨酸酶阳性白化病的一种形式,具有很大的临床变异性。出血时间延长是由于血小板中缺乏储存细胞器(致密体)。类蜡质蓄积最常见的并发症是纤维化、限制性肺病。迄今为止,尚无治愈性疗法;缺陷的本质仍然未知。