Successful treatment of congenital pulmonary alveolar proteinosis with intravenous immunoglobulin G administration
Successful treatment of congenital pulmonary alveolar proteinosis with intravenous immunoglobulin G administration
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DOI：
10.1111/j.1440-1843.2006.00814.x
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发表时间：
2006-01-01
期刊：
影响因子：
6.9
通讯作者：
Minakami, H
中科院分区：
文献类型：
--
作者：
Cho, K;Nakata, K;Minakami, H
作者报告1例女性先天性肺泡蛋白沉积症。她有两个兄弟死于同一种疾病。BAL未改善其进行性呼吸衰竭。静脉注射免疫球蛋白G（IVIG）治疗复杂性低丙种球蛋白血症后，患者从呼吸衰竭中恢复。IVIG的疗效通过两次不同情况下呼吸状态恶化的恢复和胸部CT结果的改善得到证实。随后，患者在每月IVIG的持续方案下保持无呼吸道症状超过3年。她没有表面活性蛋白（SP）B缺乏症。肺泡巨噬细胞（AM）从她的BAL液中获得的小，并表现出降低的吞噬活性。免疫组化显示PU. 1在她的AM中弱表达，AM成熟的关键蛋白。粒细胞-巨噬细胞集落刺激因子（GMCSF）、GM-CSF-受体和PU. 1的所有核苷酸序列均正常。内毒素诱导的外周血单个核细胞（PMNC）释放GM-CSF和PMNC对GM-CSF的反应性增殖均正常。此外，在成人特发性PAP患者的血清或BAL液中未检测到抗GM-CSF抗体。尽管患者的PMNC仅分泌少量的IgG和IgM，但她的B细胞的EB病毒衍生细胞系分泌与正常对照细胞一样多的IgM。在流式细胞术研究中，IgM在细胞表面上表达。结论：单个基因的异常可能导致患者B细胞分泌免疫球蛋白和AM吞噬活性降低。
The authors report a female patient with congenital pulmonary alveolar proteinosis (PAP). She had two brothers who died from the same disease. BAL did not improve her progressive respiratory failure. After intravenous immunoglobulin G (IVIG) administration for complicated hypogammaglobulinemia, she recovered from respiratory failure. The efficacy of IVIG was confirmed by recovery from deterioration in respiratory status and improvement in chest CT findings on two separate occasions. Subsequently, the patient remains free from respiratory symptoms for more than 3 years on an ongoing regimen of monthly IVIG. She had no surfactant protein (SP) B deficiency. Alveolar macrophages (AM) obtained from her BAL fluid were small and showed decreased phagocytotic activity. Immunostaining revealed weak expression of PU.1 in her AM, a key protein in AM maturation. All nucleotide sequences of granulocyte-macrophage colony stimulating factor (GMCSF), GM-CSF-receptor and PU.1 were normal. Endotoxin-induced GM-CSF release from peripheral mononuclear cells (PMNC), and proliferation of PMNC in response to GM-CSF were normal. In addition, an antibody against GM-CSF, as seen in adult patients with idiopathic PAP, was not detected in the serum or BAL fluid. Although the patient's PMNC secreted only small amounts of IgG and IgM, an EB virus-derived cell line of her B cells secreted IgM as much as normal control cells. In a flow cytometric study, IgM was expressed on the cell surface. In conclusion, an abnormality in a single gene may have decreased secretion of immunoglobulin from the B cells and the AM phagocytotic activity in the patient.