Litzman J, Jones A, Hann I, Chapel H, Strobel S, Morgan G
Molecular Immunology Unit, University of London.
Arch Dis Child. 1996 Nov;75(5):436-9. doi: 10.1136/adc.75.5.436.
To assess the results of supportive treatment with intravenous immunoglobulin (IVIG) and antibiotic prophylaxis in combination with splenectomy in patients with Wiskott-Aldrich syndrome.
Retrospective review of case records of 21 patients from March 1984 to February 1996.
Thrombocytopenia was cured in 14 of 15 patients who had splenectomy, but it recurred intermittently in three. Mean platelet volume (MPV) was normal transiently in some patients, but all MPV values were subnormal 8-23 months after splenectomy. Antibiotic and IVIG prophylaxis may have contributed to the lack of a detectable increase in the number of severe acute bacterial infections in the 451 months after splenectomy. Four patients died in 2205 months of observation before and after splenectomy (median 82, range 16-248): two of cerebral B cell lymphoma, one of progressive multifocal leucoencephalopathy, and one with severe chronic chest disease of pneumonia.
Adequate supportive treatment with IVIG and antibiotic prophylaxis together with splenectomy enables good survival and quality of life in the short and medium term in patients with Wiskott-Aldrich syndrome. Persistence of infection, bleeding, and vasculitic and allergic symptoms in a significant minority and the risk of development of lymphoma, however, suggest that bone marrow transplantation may be indicated if an HLA identical donor is available.
评估静脉注射免疫球蛋白（IVIG）及抗生素预防联合脾切除术对维斯科特-奥尔德里奇综合征患者的支持治疗效果。
回顾性分析1984年3月至1996年2月期间21例患者的病例记录。
15例行脾切除术的患者中，14例血小板减少症得到治愈，但3例出现间歇性复发。部分患者平均血小板体积（MPV）曾短暂恢复正常，但脾切除术后8 - 23个月时所有患者的MPV值均低于正常水平。抗生素及IVIG预防措施可能有助于减少脾切除术后451个月内严重急性细菌感染数量的明显增加。在脾切除术前及术后的2205个月观察期内，4例患者死亡（中位数82，范围16 - 248）：2例死于脑B细胞淋巴瘤，1例死于进行性多灶性白质脑病，1例死于严重慢性肺炎。
IVIG及抗生素预防联合脾切除术进行充分的支持治疗，可使维斯科特-奥尔德里奇综合征患者在短期和中期获得良好的生存率及生活质量。然而，仍有相当一部分患者存在持续感染、出血、血管炎及过敏症状，且有发生淋巴瘤的风险，提示若有HLA匹配的供体，可考虑进行骨髓移植。
