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首页> 美国卫生研究院文献>Molecular Genetics and Metabolism Reports >Successful reduction of high-sustained anti-idursulfase antibody titers by immune modulation therapy in a patient with severe mucopolysaccharidosis type II
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Successful reduction of high-sustained anti-idursulfase antibody titers by immune modulation therapy in a patient with severe mucopolysaccharidosis type II

机译：免疫调节治疗II型严重黏多糖贮积症患者成功降低了高持续性抗异硫苷酶抗体滴度

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We report on a 6 year old boy with severe MPS II undergoing immune modulation therapy due to high IgG antibody titers to IV idursulfase and no significant decline in urinary GAG levels since initiating enzyme replacement therapy. He has complete deficiency of iduronate-2-sulfatase activity due to a submicroscopic deletion of the X chromosome involving the entire I2S gene but not including in the fragile X locus. At 19 months of age, IV idursulfase therapy at the recommended dose of 0.5 mg/kg/week was initiated and then increased to 1.0 mg/kg/week after no observed clinical improvement and no decline in urine GAG level. After one year of ERT at the increased dose, he had no significant decline in urinary GAG excretion and increase of anti-idursulfase IgG antibody titers to 102,000 with complete neutralizing antibodies. In light of the evidence of lack of efficacy of idursulfase therapy, the patient was started on an immune modulation regimen consisting of ofatumumab, bortezomib, methotrexate and IVIG for a 12 week period. Only a slight decrease in IgG titers and urine GAG levels was observed, leading to increased intensity of bortezomib administration and addition of dexamethasone to the regimen, while continuing with the current schedule ofatumumab, IVIG and methotrexate. Over 18 month period of immune modulation therapy, we observed a significant reduction in anti-idursulfase IgG titers and a moderate reduction in urine GAG levels compared to baseline. Modest clinical improvements were observed. Our experience suggests that future MPS II patients with a complete gene deletion may be likely to develop persistent anti-idursulfase antibody titers and may benefit from immune modulation therapy prior to the development of high titer levels.
机译：我们报告了一个6岁重症MPS II的男孩，由于对IV异硫磺酶的IgG抗体滴度高，正在接受免疫调节治疗，并且自开始酶替代治疗以来尿GAG水平没有明显下降。由于X染色体亚显微缺失，涉及整个I2S基因，但不包括脆弱的X基因座，因此他具有完全缺乏的异氰酸酯2-硫酸酯酶活性。在19个月大时，开始以建议的0.5 mg / kg /周的剂量静脉内静脉注射艾杜硫酶治疗，然后在未观察到临床改善且尿液GAG水平未降低后增加至1.0 mg / kg /周。经过增加剂量的ERT一年后，他的尿GAG排泄没有显着下降，而完全中和抗体后抗异硫脲酶IgG抗体滴度增加至102,000。鉴于缺乏艾杜硫酶治疗功效的证据，该患者开始接受由Ofatumumab，bortezomib，甲氨蝶呤和IVIG组成的免疫调节方案，为期12周。仅观察到IgG滴度和尿液GAG水平略有降低，从而导致硼替佐米的给药强度增加，并且在方案中加入地塞米松，同时继续使用当前的依他莫单抗，IVIG和甲氨蝶呤。在超过18个月的免疫调节治疗期间，与基线相比，我们观察到抗异硫脲酶IgG滴度显着降低，尿液GAG水平适度降低。观察到适度的临床改善。我们的经验表明，未来具有完整基因缺失的MPS II患者可能会产生持续的抗异硫糖苷酶抗体滴度，并且可能会在高滴度水平发展之前受益于免疫调节疗法。

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