简介：

Procysbi(cysteamine bitartrate)获准用于治疗肾病型胱氨酸症
近日，美国食品药品管理局(FDA)和Raptor Pharmaceuticals宣布，Procysbi(半胱胺重酒石酸氢盐)缓释胶囊已获准用于治疗成年人和≥6岁儿童的肾病型胱氨酸症。胱氨酸耗竭是肾病型胱氨酸症的主要治疗策略。
美国初始批准：1994
适应症和用法
PROCYSBI是胱氨酸消耗代理人表示对肾病胱氨酸病在成人和儿童6岁岁及以上的管理。
用法用量
PROCYSBI应规定由经验丰富的肾病胱氨酸病管理医生。
•吞咽胶囊或全部洒在食物或液体的建议后。与食物混合后经胃管（12 F或更大）的管理。
•总日剂量为1.3克/平方米/天，分成两次剂量给药，每12小时一次。
•采取PROCYSBI后至少2小时，至少30分钟后再吃。
•治疗目标是维持白细胞（WBC）胱氨酸的水平<1纳摩尔半胱胺酸/ mg蛋白或血浆中半胱胺浓度>0.1毫克/升。
由速释半胱胺切换到PROCYSBI
•每日总剂量PROCYSBI等于其之前的每日总剂量的速释半胱胺重酒石酸的。
初始剂量的半胱胺初治患者
•起始剂量：至¼PROCYSBI的维持剂量.
•维持剂量：1.3克/平方米/天，分两次服用，每12小时
请参阅完整的处方信息进行管理上的细节。
剂型和规格
缓释胶囊：25毫克和75毫克
禁忌
过敏青霉胺
警告和注意事项
•埃勒斯 - 当洛综合征类：减少用量，如果皮肤和骨骼发生病变。
•皮疹：如果严重的皮疹，如多形红斑大疱或中毒性表皮坏死松解症发生时，请停止PROCYSBI。
•胃肠道：监视消化道溃疡和出血的症状。
•中枢神经系统：监测癫痫发作，嗜睡，嗜睡，抑郁和脑病。
•白细胞减少症和碱性磷酸酶升高级别：监视白细胞计数及碱性磷酸酶升高的水平。
•良性颅内压增高：用于监控和体征良性颅内压增高症状。
不良反应
最常见的不良反应（≥5％）是呕吐，腹部疼痛/不适，头痛，恶心，腹泻，厌食/食欲下降，口臭，乏力，头晕，皮肤的气味，和皮疹。
药物相互作用
PROCYSBI可施用与电解质（除碳酸氢盐）和必要的范可尼综合症，管理矿物替换以及维生素D和甲状腺激素.
特殊人群中使用
哺乳期妇女：哺乳，不建议同时服用PROCYSBI。
包装规格：
25mg胶囊/粒*60粒/胶囊/盒
75mg胶囊/粒*250粒/胶囊/盒

PROCYSBI® (cysteamine bitartrate) delayed-release capsules
What is PROCYSBI?
PROCYSBI is the first is cystine-depleting agent given every 12 hours that is approved in the United States for the treatment of nephropathic cystinosis in adults and children 2 years of age and older. Nephropathic cystinosis is a rare metabolic disorder with an estimated preva lence as high as 1 in 100,000 live births.1,2 There are believed to be 500 nephropathic cystinosis patients in the U.S., and 2,000 worldwide.
Indications and Usage
In the United States, PROCYSBI (cysteamine bitartrate) delayed-release capsules is a prescription medicine used to treat a medical condition called nephropathic cystinosis in adults and children 2 years of age and older.
Important Safety Information
CONTRAINDICATIONS:
•Hypersensitivity to penicillamine or cysteamine.
WARNINGS AND PRECAUTIONS:
•Ehlers-Danlos-like Syndrome: Skin and bone lesions that resemble clinical findings for Ehlers-Danlos-like syndrome have been reported in patients treated with high doses of immediate-release cysteamine bitartrate or other cysteamine salts.
◦These include molluscoid pseudotumors (purplish hemorrhagic lesions), skin striae, bone lesions (including osteopenia, compression fractures, scoliosis and genu valgum), leg pain, and joint hyperextension.
◦One patient on immediate-release cysteamine bitartrate with serious skin lesions subsequently died of acute cerebral ischemia with marked vasculopathy.
◦Monitor patients for development of skin or bone lesions and interrupt PROCYSBI dosing if patients develop these lesions. PROCYSBI may be restarted at a lower dose under close supervision, then slowly increase to the appropriate therapeutic dose.
•Skin Rash: Severe skin rashes such as erythema multiforme bullosa or toxic epidermal necrolysis have been reported in patients receiving immediate-release cysteamine bitartrate. If severe skin rashes develop, permanently discontinue use of PROCYSBI.
•Gastrointestinal Ulcers and Bleeding: Gastrointestinal (GI) ulceration and bleeding have been reported in patients receiving immediate-release cysteamine bitartrate.
◦GI tract symptoms including nausea, vomiting, anorexia and abdominal pain, sometimes severe, have been associated with cysteamine. If severe GI tract symptoms develop, consider decreasing the dose of PROCYSBI.
•Central Nervous System Symptoms: Central Nervous System (CNS) symptoms such as seizures, lethargy, somnolence, depression, and encephalopathy have been associated with immediate-release cysteamine.
◦Neurological complications have also been described in some patients with cystinosis who have not been treated with cysteamine.
◦Carefully eva luate and monitor patients who develop CNS symptoms. Interrupt medication or adjust the dose as necessary for patients with severe symptoms or with symptoms that persist or progress.
◦Inform patients that PROCYSBI may impair their ability to perform tasks such as driving or operating machinery.
•Leukopenia and/or Elevated Alkaline Phosphatase Levels: Cysteamine has been associated with reversible leukopenia and elevated alkaline phosphatase levels. Monitor white blood cell counts and alkaline phosphatase levels. If tests values remain elevated, consider decreasing the dose or discontinuing the drug until values revert to normal.
•Benign Intracranial Hypertension: Benign intracranial hypertension (pseudotumor cerebri; PTC) and/or papilledema has been reported in patients receiving immediate- release cysteamine bitartrate treatment.
◦Monitor patients for signs and symptoms of PTC, including headache, tinnitus, dizziness, nausea, diplopia, blurry vision, loss of vision, pain behind the eye or pain with eye movement. If signs/symptoms persist, interrupt dosing or decrease the dose and refer the patient to an ophthalmologist. If the diagnosis is confirmed, permanently discontinue use of PROCYSBI.
ADVERSE REACTIONS:
The most common adverse reactions (≥5%) in patients treated in clinical trials are vomiting, nausea, abdominal pain, breath odor, diarrhea, skin odor, fatigue, rash, and headache.
DRUG INTERACTIONS:
•PROCYSBI should be administered at least 1 hour before or 1 hour after medications containing bicarbonate or carbonate.
•Consumption of alcohol with PROCYSBI may increase the rate of cysteamine release and/or adversely alter the pharmacokinetic properties, as well as the effectiveness and safety of PROCYSBI.
•PROCYSBI can be administered with electrolyte (except bicarbonate) and mineral replacements necessary for management of Fanconi Syndrome as well as vitamin D and thyroid hormone.