药物名称：苯丁酸钠 sodium phenylbutyrate

药物别名：Buphenyl powder、Ucyclyd Pharma

分子式成分：1996年FDA批准在美国上市的新药，商品名Buphenyl powder (Ucyclyd Pharma)。本品对于因氨基甲酰磷酸合成酶(CPS)、鸟氨酸氨甲酰转移酶(OTC)或精氨基琥珀酸合成酶(AS)缺乏而致慢性脲循环紊乱的病人，可用作辅助治疗。

药理毒理：
　　为了观察鸟氨酸氨甲酰转移酶缺乏(OTCD)患者体内的代谢能力，有人用质谱法测定口服同位素标记的15　N H4Cl后血中15 N-尿素和(5-15 N)谷氨酸的浓度。结果表明：①此测定方法可有效地测定体内含氮代谢物，也许能在将来取代对OTCD患者做肝脏活检法测定酶活性的需要。②无症状OTCD 患者以正常速率生成尿素，表明在酶活性低于正常水平时仍然有能力生成尿素。③虽然无症状者生成尿素的速率正常，但其含氮代谢物水平不正常，即(5-15 N)谷氨酸的浓度较高。④此测定新方法对于评价肝移植和基因治疗等方法治疗OTCD后的疗效有十分重要意义。 　　
    新生儿期发生高血氨脑病则通常在一岁以内死亡。血透、服用苯丁酸等药物、限制蛋白饮食，可使在出生后一个月内确诊患者的存活率提高到约80%，但智力发育障碍的发生率较高。后天发病并服用苯丁酸钠等治疗的病人可保留认知能力。治疗不能使已有的神经系统损害发生逆转，某些病人还可能出现神经系统损伤的恶化。 　　
    由于OTC是X染色体携带的线粒体酶，直接催化鸟氨酸与氨酰磷酸合成瓜氨酸。对32名1岁～17岁发作过至少一次高血氨脑病的女孩作长期疗效观察发现， 5年存活率在90%以上，并维持适宜的身高体重比，随着年龄的增长，高血氨症的发作次数减少，认知力下降不明显。说明对有症状OTCD女孩的药物治疗促进了体内含氮废物的排泄。

药动学：
　　苯乙酸盐是相对无毒的抗肿瘤药物，但因其不良气味而致部分患者无法接受。前体药物苯丁酸钠无不良气味，在体内可经β-氧化作用而迅速转换成苯乙酸盐。 　　
    HPLC测得苯乙酸钠和苯丁酸钠在血浆中的游离分数分别为0.442±0.008和0.188±0.001，血浆pH值对其蛋白结合率影响不大，若白蛋白水平低，则蛋白结合率亦下降。 　　
    Ⅰ期临床研究中对14名癌症患者，年龄(51.8±13.8)岁，分别给予3个剂量的苯丁酸钠(600，1 200和2 000 mg*m-2)，静脉输注15 min，分别采集血样和24 h尿，用HPLC测定苯丁酸钠、苯乙酸盐、苯乙酰谷酰胺。苯丁酸钠的峰浓度为0.5 mmol*L-1～2.0 mmol*L-1，消除呈饱和性［(Km为(34.1±18.1)μg*ml-1，vmax为(18.1±18)mg*h-1*kg-1］。苯乙酸盐和苯乙酰谷酰胺在体内达到峰浓度的时间分别为1 h和2 h。由苯丁酸钠转换成苯乙酸盐的转换率为(80±12.6)%。苯乙酸盐又迅速转换成苯乙酰谷酰胺，因而其血清水平很低。

适应证：
　　所有新生儿酶缺乏(出生后28 d内表现为完全酶缺乏的)、有高血氨性脑病家族史的迟发性酶缺乏(出生的第一个月后表现为部分酶缺乏)的患者。应尽早诊断、及早治疗以提高生存率。给予苯丁酸钠时应限制饮食蛋白摄入，有时需补充必需氨基酸。

不良反应：
　　常见的副作用为闭经或月经失调(经期患者中的发生率为23%)，食欲下降(4%)，体臭(3%，可能是苯基乙盐所致)，味觉差或较差(3%)。亦有临床检验指标异常，如酸中毒(14%)、低蛋白血症(11%)和贫血(9%)。 　　
    由于本药物中含有125 mg*g-1的钠，应在下列情况下慎用此药：充血性心衰、严重肾功能不全、水肿和限钠患者。 　　
    本品属孕期用药C级(美国FDA颁布的妊娠期间使用药物危险性等级的分类)，仅在必要时才给孕妇使用。是否在乳汁中排泄尚未知，故哺乳期妇女慎用。 　　
    苯丁酸钠主要在肝脏和肾脏代谢，剂量的80%～100%可在24 h内以苯乙酰谷氨酸的形式由肾脏排出。肝、肾功能不全的患者用药时应密切观察。与丙磺舒合用时排出率下降。

相互作用：
　　苯丁酸钠可降低高血氨症或高血氨性脑病的发作频率，但多数患者在治疗期间亦会出现这类病情，需紧急抢救。氟哌啶醇(haloperidol)和丙戊酸盐 (depakote)会增加血氨浓度，应避免用于尿素循环紊乱的患者。甾体激素类药物可能会促进机体蛋白的利用因而使血氨水平升高。

用法用量：
　　体重低于20 kg者苯丁酸钠的日剂量是450 mg*kg-1～600 mg*kg-1，体重高于20kg者为每日9.9 g*m-2～13.0 g*m-2。每日剂量超过20 g时疗效和安全性尚未知。 　　
    本药可终身服用，或服用至肝移植术后。 　　
    苯丁酸钠片剂为500 mg。口服粉剂为0.94 g*g-1(通过口服、胃管或鼻饲管)。每个包装中有一个勺子，一茶勺相当于3.6g苯丁酸钠，一汤勺相当于8.6 g苯丁酸钠。 　　
    每日剂量可分成3等分与饭同服，粉剂可与饮食混合后服用(但不得用酸性饮料)。苯丁酸钠口感较咸，但无不良气味。

Buphenyl
Pronunciation: SOE-dee-um fen-il-BYOO-ti-rate
Generic Name: Sodium Phenylbutyrate
Brand Name: Buphenyl

Buphenyl is used for:
Treating urea cycle (nitrogen or ammonia excretion) disorders along with a prescribed diet.

Buphenyl is a nutritional modifier. It works by helping rid the body of excess nitrogen waste by bypassing the urea cycle.

Do NOT use Buphenyl if:
you are allergic to any ingredient in Buphenyl
you have sudden high levels of ammonia in the blood
Contact your doctor or health care provider right away if any of these apply to you.

Before using Buphenyl:
Some medical conditions may interact with Buphenyl. Tell your doctor or pharmacist if you have any medical conditions, especially if any of the following apply to you:
if you are pregnant, planning to become pregnant, or are breast-feeding
if you are taking any prescription or nonprescription medicine, herbal preparation, or dietary supplement
if you have allergies to medicines, foods, or other substances
if you have edema (swelling or water retention), heart failure or other heart problems, high blood pressure, or kidney or liver disease

Some MEDICINES MAY INTERACT with Buphenyl. Tell your health care provider if you are taking any other medicines, especially any of the following:
Corticosteroids (eg, prednisone), haloperidol, or valproic acid because these medicines increase blood ammonia levels
Probenecid because the actions and side effects of Buphenyl may be increased

This may not be a complete list of all interactions that may occur. Ask your health care provider if Buphenyl may interact with other medicines that you take. Check with your health care provider before you start, stop, or change the dose of any medicine.

How to use Buphenyl:
Use Buphenyl as directed by your doctor. Check the label on the medicine for exact dosing instructions.

Take Buphenyl with meals.
It is very important that your full dose for a 24-hour period is taken. The total daily dose should be taken in equally divided amounts (3 to 6 times per day).
It is very important to follow the prescribed diet given to you by your doctor.
Buphenyl comes with an additional patient information sheet. Read it carefully and reread it each time you get Buphenyl refilled.
If you miss a dose of Buphenyl, take it as soon as possible that same day.

Ask your health care provider any questions you may have about how to use Buphenyl.

Important safety information:
Wear a MedicAlert tag that states that you have a urea cycle disorder.
LAB TESTS, such as blood tests for ammonia and amino acid levels, will be required to monitor your progress. Be sure to keep all doctor and lab appointments.
Buphenyl is not recommended for use in CHILDREN weighing less than 44 pounds (20 kg). Safety and effectiveness in this age group have not been confirmed.
PREGNANCY and BREAST-FEEDING: It is not known if Buphenyl causes harm to the developing fetus. If you become pregnant, discuss with your doctor the benefits and risks of using Buphenyl during pregnancy. It is unknown if Buphenyl is excreted in breast milk. If you are or will be breast-feeding while taking Buphenyl, consult your doctor or pharmacist to discuss the risks to your baby.

Possible side effects of Buphenyl:
All medicines may cause side effects, but many people have no, or minor, side effects. Check with your doctor if any of these most COMMON side effects persist or become bothersome:
Bad taste in mouth; body odor; decreased appetite; absent or irregular menstrual periods.

Seek medical attention right away if any of these SEVERE side effects occur:
Severe allergic reactions (rash; hives; itching; difficulty breathing; tightness in the chest; swelling of the mouth, face, lips, or tongue); decreased hearing; disorientation; headache; lightheadedness; memory problems; sleepiness.

This is not a complete list of all side effects that may occur. If you have questions about side effects, contact your health care provider. Call your doctor for medical advice about side effects. To report side effects to the appropriate agency, please read the Guide to Reporting Problems to FDA.

If OVERDOSE is suspected:
Contact 1-800-222-1222 (the American Association of Poison Control Centers), your local poison control center, or emergency room immediately.

Proper storage of Buphenyl:
Store Buphenyl between 59 and 86 degrees F (15 and 30 degrees C). Store away from heat, moisture, and light. After opening, keep the bottle tightly closed. Do not store in the bathroom. Keep Buphenyl out of the reach of children and away from pets.

General information:
If you have any questions about Buphenyl, please talk with your doctor, pharmacist, or other health care provider.
Buphenyl is to be used only by the patient for whom it is prescribed. Do not share it with other people.
If your symptoms do not improve or if they become worse, check with your doctor.
Check with your pharmacist about how to dispose of unused medicine.

This information is a summary only. It does not contain all information about Buphenyl. If you have questions about the medicine you are taking or would like more information, check with your doctor, pharmacist, or other health care provider.

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原产地英文商品名:
BUPHENYL POWDER 250g/bottle
原产地英文药品名:
SODIUM PHENYLBUTYRATE
中文参考商品译名:
BUPHENYL粉剂 250克/瓶
中文参考药品译名:
苯丁酸钠
生产厂家中文参考译名:
MEDICIS
生产厂家英文名:
MEDICIS
处方药:处方药
包装规格: 250克/瓶
销售单价(美元 US$): 6,727 美元
销售单价(人民币 RMB): 44,398 元
计价单位: 瓶