| 简介：
部份中文尼替西农处方资料（仅供参考）
英文名：Nitisinone
商品名：Orfadin Capsules
中文名：尼替西农胶囊
生产商：安斯泰来制药
オーファディンカプセル2mg/オーファディンカプセル5mg/オーファディンカプセル10mg
药用类别名称
I型高酪氨酸-红细胞增多症治疗剂
批准日期：2015年2月
商標名
Orfadin Capsules 2mg
Orfadin Capsules 5mg
Orfadin Capsules 10mg
一般名
ニチシノン（Nitisinone）
化学名
2-[2-Nitro-4-(trifluoromethyl)benzoyl]cyclohexane-1,3-dione
構造式
分子式
C14H10F3NO5
分子量
329.23
融点
140〜142℃
性状：
尼希松是一种白色带淡黄色白色的结晶粉末。 不溶于甲醇或乙醇 (99.5), 不易溶于水。
操作注意事项
从瓶子中取出产品后, 及时服用。
从瓶子上取下产品后, 立即关闭瓶盖, 并将其存放在冰箱中。
批准条件
1. 制定药品风险管理计划并予以适当实施。
2. 由于该国的使用经验极为有限, 在制造和销售后, 通过在重新检查期间对所有病例进行使用结果调查, 掌握使用该制剂的患者的背景信息, 并尽早收集有关该制剂安全性和有效性的数据, 采取必要措施, 正确使用本制剂。
药用药理学
遗传性高酪氨酸i型是一种常染色体隐性遗传病, 由于酪氨酸降解途径(FAH)最后阶段的基因突变。通过降低FAH活性, 是酪氨酸分解途径富马乙酰乙酸(FAA)和马来乙酰乙酸(MAA)的中间代谢物, 以及这些代谢物琥珀酰丙酮(SA)和琥珀酸乙酸(SAA)是在肝脏和肾脏中积累, 它被认为是失败发生在这些器官。在酪氨酸分解途径中, 通过抑制位于fah上游的4-羟基苯基丙酮酸二氧合酶, 据信FAA、MAA可以抑制SA和SAA的产生和积累, 事实上, 在遗传性高酪氨酸血症i型患者中, 经反复口服尼希松(0.1至0.6mgggg/day), 尿Sa和血浆SA可减少。
适应症
I型高酪氨酸血血症
用法与用量
通常, 口服管理的1天 1mgskg 两倍作为尼希西农。虽然根据患者的病情适当增加或减少, 但上限为1天2mg kg。
临床结果
NTBC测试, 已开始作为一个医生驱动的类型, 包括日本高酪氨酸型i患者1例, 第一个推荐剂量是1mg/kg/day/天 (每日两次口服), 非盲, 这是与有限酪氨酸和苯基丙氨酸的饮食治疗相结合, 这是一个非控制测试。报告价值仅作为对照组, 对207例患者进行分析时观察到显著存活率的提高。(见临床结果表 3)
此外, 在药物管理机构对90%或更多患者进行为期一周的药物管理后, 尿中琥珀酸的尿排泄已正常化。此外, 与仅通过饮食治疗相比, 肝细胞癌的发生风险降低(2.3至3.7倍)。如果早期开始治疗, 肝细胞癌的风险进一步降低(出生前12个月开始时的13.5倍)。
临床结果表
表3
|
治療開始時又は診断時の年齢 |
生存率（％）：
ニチシノンによる治療：5年 |
生存率（％）：
ニチシノンによる治療：10年 |
生存率（％）：
食事療法のみの対照群※：5年 |
生存率（％）：
食事療法のみの対照群※：10年 |
|
＜2カ月 |
82 |
- |
28 |
- |
|
＞2〜6カ月 |
95 |
95 |
51 |
34 |
|
＞6カ月 |
92 |
86 |
93 |
59 |
※：Van Spronsen et al.、19945）の図1
包装
胶囊
2mg:60胶囊(瓶)
5mg/60胶囊(瓶)
10mg:60胶囊(瓶)
製造和銷售來源
安斯泰来制药公司
注：以上中文不够完整，使用者以原处方资料为准。
完整说明附件：http://www.info.pmda.go.jp/go/pack/3999036M1029_1_04/
Orfadin Capsules 10mg(Nitisinone オーファディンカプセル)
Brand name:Orfadin Capsules 10mg
Active ingredient:Nitisinone
Dosage form:white capsule, No. 3 capsule
Print on wrapping:
Effects of this medicine
This medicine inhibits 4-hydroxyphenylpyruvate dioxygenase in the tyrosine degradation pathway to restrict thegeneration of toxic metabolites and alleviates diseases.
It is usually used to treatment of hypertyrosinemia type I.
Before using this medicine, be sure to tell your doctor and pharmacist
・If you have previously experienced any allergic reactions (itch, rash, etc.) to any medicines.
・If you are pregnant or breastfeeding.
・If you are taking any other medicinal products. (Some medicines may interact to enhance or diminish medicinaleffects. Beware of over-the-counter medicines and dietary supplements as well as other prescription medicines.)
Dosing schedule (How to take this medicine)
・Your dosing schedule prescribed by your doctor is<< to be written by a healthcare professional>>
・In general, take 1 mg/kg of active ingredient per day, to be taken in 2 divided doses. The dosage should be adjustedaccording to the symptoms. If it is not effective enough, the dosage may be increased up to 2 mg/kg per day. Each
capsule contains 10 mg of the active ingredient. Strictly follow the instructions.
・Take the medicine as soon as possible after taking it out of the bottle, and close the cap of the bottle and store it.
・If you miss a dose, take a dose as soon as possible. However, if you remember your medicine at a point close to thetime for the next dose, proceed with the prescribed schedule of medication starting from the next dose.
・If you accidentally take more than your prescribed dose, consult with your doctor or pharmacist.
・Do not stop taking this medicine unless your doctor instructs you to do so.
Precautions while taking this medicine
・Strictly follow dietary therapy instructed by your doctor.
・You may have vision disorder by taking this medicine. Pay attention when you drive a car, or operate dangerousmachinery.
Possible adverse reactions to this medicine
The most commonly reported adverse reactions include eye disorder, exfoliative dermatitis, erythematous eruption,and itch. If any of these symptoms occur, consult with your doctor or pharmacist.
The symptoms described below are rarely seen as initial symptoms of the adverse reactions indicatedin brackets. If any of these symptoms occur, stop taking this medicine and see your doctorimmediately.
・blurred vision, bloodshot eyes, foreign-body sensation, sensitive to light, eye pain, itch of eyelid [eye disorder]
・nose/gum bleeding, subcutaneous bleeding, sore throat, fever [thrombocytopenia, leukopenia, granulocytopenia]
The above symptoms do not describe all the adverse reactions to this medicine. Consult with yourdoctor or pharmacist if you notice any symptoms of concern other than those listed above.
Storage conditions and other information
・Keep out of the reach of children. Avoid freezing and store it at 2 to 8 ℃ (refrigerator).
・Discard the remainder. Do not store them. Ask the pharmacist or healthcare facility how to discard them.
Internal
Published: 01/2015
The information on this sheet is based on approvals granted by the Japanese regulatory authority. Approvaldetails may vary by country. Medicines have adverse reactions (risks) as well as efficacies (benefits). It isimportant to minimize adverse reactions and maximize efficacy. To obtain a better therapeutic response,patients should understand their medication and cooperate with the treatment. |
