کد مقاله | کد نشریه | سال انتشار | مقاله انگلیسی | نسخه تمام متن |
---|---|---|---|---|
8648106 | 1570392 | 2018 | 7 صفحه PDF | دانلود رایگان |
عنوان انگلیسی مقاله ISI
Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project
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کلمات کلیدی
GD1CCL18/PARCBUABMDDXASOSenzyme replacement therapy - آنزیم جایگزین درمانEfficacy - اثرEMG - الکترومیوگرافیelectromyography - الکترومیوگرافیstandard deviation - انحراف معیارSafety - ایمنیBone mineral density - تراکم معدنی استخوانstandard error - خطای استانداردDual x-ray absorptiometry - دوز جذبی سنجی اشعه ایکسspeed of sound - سرعت صوتbroadband ultrasound attenuation - ضعیف التراسوند باند پهنMaintenance - عملیات نگهداری و تعمیراتMiglustat - مگولاستاGaucher disease type 1 - نوع بیماری گوچه 1ERT - هستندhaemoglobin - هموگلوبینchitotriosidase - کیتوتریوزیداز
موضوعات مرتبط
علوم زیستی و بیوفناوری
بیوشیمی، ژنتیک و زیست شناسی مولکولی
زیست شناسی مولکولی
پیش نمایش صفحه اول مقاله
چکیده انگلیسی
We report data from a prospective, observational study (ZAGAL) evaluating miglustat 100 mg three times daily orally. in treatment-naïve patients and patients with type 1 Gaucher Disease (GD1) switched from previous enzyme replacement therapy (ERT). Clinical evolution, changes in organ size, blood counts, disease biomarkers, bone marrow infiltration (S-MRI), bone mineral density by broadband ultrasound densitometry (BMD), safety and tolerability annual reports were analysed. Between May 2004 and April 2016, 63 patients received miglustat therapy; 20 (32%) untreated and 43 (68%) switched. At the time of this report 39 patients (14 [36%] treatment-naïve; 25 [64%] switch) remain on miglustat. With over 12-year follow-up, hematologic counts, liver and spleen volumes remained stable. In total, 80% of patients achieved current GD1 therapeutic goals. Plasma chitotriosidase activity and CCL-18/PARC concentration showed a trend towards a slight increase. Reductions on S-MRI (p = 0.042) with an increase in BMD (p < 0.01) were registered. Gastrointestinal disturbances were reported in 25/63 (40%), causing miglustat suspension in 11/63 (17.5%) cases. Thirty-eight patients (60%) experienced a fine hand tremor and two a reversible peripheral neuropathy. Overall, miglustat was effective as a long-term therapy in mild to moderate naïve and ERT stabilized patients. No unexpected safety signals were identified during 12-years follow-up.
ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: Blood Cells, Molecules, and Diseases - Volume 68, February 2018, Pages 173-179
Journal: Blood Cells, Molecules, and Diseases - Volume 68, February 2018, Pages 173-179
نویسندگان
Pilar Giraldo, Marcio Andrade-Campos, Pilar Alfonso, Pilar Irun, Koldo Atutxa, Antonio Acedo, Abelardo Barez, Margarita Blanes, Vicente Diaz-Morant, Ma Angeles Fernández-Galán, Rafael Franco, Cristina Gil-Cortes, Vicente Giner, Angela Ibañez,