抗儿童白血病新药Clolar获美国FDA批准 2005年1月2日：Genzyme药剂公司发言人日前宣布，美国食品及药物管理局FDA已批准其生产的Clolar可以用于治疗对其他常规疗法预后不佳甚至全无反应的儿童急性淋巴性白血病(ALL)，但仍然要求该公司继续其临床研究以进一步确证患儿存活率的改善。这也是十多年来首个获准的专用抗儿童白血病新药。FDA是依据近期完成的一项临床治...... 临床快报 药市动态 违法广告 医保动态 药品价格 流感疫情 保健常识 妇科课堂 医改动态 2005年1月2日：Genzyme药剂公司发言人日前宣布，美国食品及药物管理局FDA已批准其生产的Clolar可以用于治疗对其他常规疗法预后不佳甚至全无反应的儿童急性淋巴性白血病(ALL)，但仍然要求该公司继续其临床研究以进一步确证患儿存活率的改善。也是十多年来首获准的专用抗儿童白血病新药。 FDA是依据近期完成的临床治疗研究结果做出上述批准的。总共有49例急性淋巴性白血病患儿参加了该项由St. Jude儿童医院白血病研究中心的Sima Jeha医学博士等实施的临床研究，此前的联合化疗均对他们无效，但在接受Clolar治疗后有30%的患儿表现出不同程度的改善。专家们同时注意到该药的潜在严重副作用之一是骨髓抑制，这不仅会降低抗击严重感染所必需的免疫细胞数量，而且还可能相应影响骨髓细胞转化为红细胞和血小板。另据报道，一项用Clolar治疗儿童急性骨髓性白血病(AML)的临床研究也得到了类似结果，42例对其他常规疗法无反应的患儿中有26%表现出不同程度的改善，但FDA尚未批准该药用于治疗AML。，但本研究还没回答某些问题，如eculizumab应用多长时间，以及本药是否可改善骨髓功能。 What is Clolar? Clolar (clofarabine) is a cancer medication that interferes with the growth of cancer cells and slows their growth and spread in the body.Clolar is used to treat acute lymphoblastic leukemia (a type of blood cancer) in children and young adults up to 21 years old.Clolar is usually given after other cancer medicines have been tried without successful treatment. Important information about Clolar Do not use Clolar without telling your doctor if you are pregnant. It could harm the unborn baby. Use birth control to prevent pregnancy while you are receiving Clolar, whether you are a man or a woman. Clolar use by either parent may cause birth defects. If you have liver or kidney disease, you may need a dose adjustment or special tests to safely receive Clolar. To be sure Clolar is not causing harmful effects, your blood cells, kidney function, and liver function may need to be tested on a regular basis. Do not miss any follow-up visits to your doctor. Clolar can lower the blood cells that help your body fight infections. This can make it easier for you to bleed from an injury or get sick from being around others who are ill. Avoid being near people who have colds, the flu, or other contagious illnesses. Contact your doctor at once if you develop signs of infection. Clolar can be harmful to the liver or kidneys, and these effects are increased when you also use other medicines that can harm the liver or kidneys. During your 5-day treatment with Clolar, you may need to avoid using certain medications. Discuss all treatment options with your doctor if you regularly use any other medications. Tell your caregivers at once if you have a serious side effect such as rapid breathing and heart rate, trouble breathing, painful swelling, numbness or tingling, feeling like you might pass out, lower back pain, blood in your urine, urinating less than usual, or jaundice (yellowing of the skin or eyes). Before receiving Clolar If you have liver or kidney disease, you may need a dose adjustment or special tests to safely receive Clolar.FDA pregnancy category D. Clolar can cause harm to an unborn baby or cause birth defects. Before you receive this medicine, tell your doctor if you are pregnant. Use birth control to prevent pregnancy while you are receiving Clolar, whether you are a man or a woman. Clolar use by either parent may cause birth defects. It is not known whether clofarabine passes into breast milk or if it could harm a nursing baby. Do not breast-feed while you are receiving Clolar. How is Clolar given? Clolar is given as an injection through a needle placed into a vein. You will receive this injection in a clinic or hospital setting. The medicine must be given slowly through an IV infusion, and can take at least 2 hours to complete. Clolar is usually given daily for 5 days in a row during one or more treatment cycles. Your doctor will determine how many treatment cycles you will receive and how often. You may receive other medications to help prevent certain side effects of Clolar. Clolar can lower the blood cells that help your body fight infections. This can make it easier for you to bleed from an injury or get sick from being around others who are ill. To be sure your blood cells do not get too low, your blood will need to be tested on a regular basis. Your cancer treatments may be delayed based on the results of these tests. Your kidney or liver function may also need to be tested. Do not miss any follow-up visits to your doctor. Contact your doctor at once if you develop signs of infection such as fever, chills, sore throat, flu symptoms, pale skin, easy bruising or bleeding (nosebleeds, bleeding gums), purple or red pinpoint spots under your skin, loss of appetite, mouth sores, or unusual weakness. What should I avoid while receiving Clolar? Avoid being near people who have colds, the flu, or other contagious illnesses. Contact your doctor at once if you develop signs of infection. Clolar side effects Get emergency medical help if you have any of these signs of an allergic reaction to Clolar: hives; difficult breathing; swelling of your face, lips, tongue, or throat. Tell your caregivers at once if you have a serious side effect such as:feeling like you might pass out;runny or stuffy nose, cough, rapid breathing and heart rate, trouble breathing, swelling and pain in any part of your body;lower back pain, blood in your urine, urinating less than usual or not at all;numbness or tingly feeling around your mouth;muscle weakness, tightness, or contraction, overactive reflexes;fast or slow heart rate, weak pulse, feeling short of breath, confusion, fainting;numbness or redness on the palms of your hands or the soles of your feet;jaundice (yellowing of the skin or eyes); or signs of infection such as fever, chills, sore throat, flu symptoms, pale skin, easy bruising or bleeding (nosebleeds, bleeding gums), purple or red pinpoint spots under your skin, loss of appetite, mouth sores, unusual weakness. 美国FDA批准Clolar(clofarabine)用于儿童急性淋巴细胞白血病 Genzyme公司于2004年12月29日宣布，美国FDA已经批准其clofarabine（Clolar）用于治疗儿童顽固性或复发性急性淋巴细胞白血病（ALL）。本品为十多年来首个获准专门用于儿童的白血病治疗新药。考虑到这一严重患者群的新疗法相当匮乏，Genzyme公司将尽可能在2005年1月份上市本品。 业内人士认为，FDA此次批准本品是一种积极信号，表明医药行业和主管部门已经开始采取行动，针对儿童肿瘤患者的治疗需求，开发抗肿瘤药。这将鼓励该行业去满足严重儿科疾病的治疗需求。据估计，美国在2005年将新增3400例儿科急性白血病患者。ALL是最常见的儿科白血病，而对于初始疗法无应答或反复发作的患儿，其预后不利。 本品获准用于1～21岁顽固性或复发性ALL患者，患者此前至少接受过2种优先使用的疗法。本品此项适应证是基于患者对本品的完全应答所作出的结论。在提高患者生存率和其它临床益处方面尚无随机临床研究进行证明。 本品已被授予成人及儿童ALL和急性髓样白血病（AML）治疗方面的罕用药物地位。现在，本品将在治疗儿科ALL方面拥有7年的市场独占权。FDA还根据《儿童最佳药物法案》将本品的市场独占权期限延长了6个月。 本品必须由专业医师给药，患者接受本品治疗后可能会出现骨髓功能抑制，这种抑制作用是可逆的且是剂量相关性的，这可能会增加包括严重败血症在能的感染风险。医师必须评估和监测患者细胞因子释放以及肿瘤溶解综合征的体征和症状，因为这些可能引起患者系统性炎症反应综合征/毛细血管漏综合征和器官机能障碍。在输注本品期间，还应监测患者的呼吸和血压。 一旦患者出现临床上的系统性炎症反应综合征或毛细血管漏综合征的显著体征，则应立即停用本品，并对患者采取适当的支持性医护措施。如果不考虑诱发因素，本品最常见的不良反应为：胃肠道症状，包括恶心、呕吐、腹泻；对血液方面的影响，包括贫血、白细胞减少症、血小板减少症、嗜中性白细胞减少症、嗜中性白细胞减少症合并发热；感染。在治疗期间，对患者密切进行血液学监测是相当重要的。患者在接受本品治疗前和治疗期间应接受肝功能和肾功能评估。有35%的患者出现心脏方面的障碍，包括心动过速、心包积液和左心室收缩功能障碍等。但是，这些症状是在患者接受本品治疗前、治疗早期还是由患者本身的疾病引起的，在病因学上尚未明确 ------------------------------------------------------------- The diagnosis of acute lymphoblastic leukemia (ALL) can be a frightening thing to hear from your doctor. And if your child has not responded to therapy, you may have many questions about what to do next. By now you may know a lot about childhood leukemia and what to expect from therapy. This website is designed to help answer many of the questions you may have about Clolar, but is not medical advice and does not replace discussions you should have with your healthcare team. Healthcare professionals are an excellent resource for information about ALL and treatment options. How Clolar is Used  怎样使用Clolar Clolar is a type of medication to treat children, ages 1 to 21 with a type of leukemia called relapsed or refractory acute lymphoblastic leukemia (ALL), after at least 2 other treatment attempts have failed. Clolar can reduce the number of leukemia cells in the blood. At this time we do not know if Clolar will help a child with ALL live longer or cure him or her of the cancer. Important Safety Information for Patients Serious side effects 主要副作用 Clolar can cause serious side effects that include: Systemic inflammatory response syndrome (SIRS)/capillary leak syndrome (CLS). Signs include fast breathing, fast heartbeat, low blood pressure, and difficulty breathing. These signs should be reported to the physician right away, as SIRS and CLS can be life-threatening if not treated right way. If your child experiences clinically significant signs of SIRS or CLS, your physician should stop Clolar immediately and consider giving your child steroids, diuretics, and albumin. When your child has stabilized, Clolar can be continued, usually at a lower dose. Bone marrow suppression and infection. Clolar can stop your child’s bone marrow from making enough red blood cells, white blood cells, and platelets. Serious side effects can result from this, and include severe infection (sepsis), bleeding, and anemia. Effects on pregnancy and breastfeeding. Females should not become pregnant or breastfeed during treatment with Clolar because Clolar may harm the baby. Other side effects 其他副作用 The most common side effects with Clolar are stomach problems (including vomiting, diarrhea, and nausea), and effects on blood cells (including low red and white blood cells count, low platelet count, fever, and infection). A fast heartbeat has been noted in some patients taking Clolar. Clolar can also affect the liver and kidneys. For these reasons, your child’s healthcare professional will do blood tests to monitor his or her blood cells, kidney function, and liver function. Treatment with Clolar quickly reduces the number of leukemia cells in your child’s blood. For this reason, your doctor should monitor your child for signs and symptoms of tumor lysis syndrome (TLS), as well as signs and symptoms of cytokine release, which can develop into SIRS, CLS, and organ problems. Your doctor is encouraged to give continuous IV fluids throughout the five days of Clolar treatment to reduce certain side effects. Your doctor may also prescribe allopurinol to reduce the build-up of uric acid that occurs with TLS. Your doctor should stop the Clolar treatment if your child develops low blood pressure for any reason during the five days of treatment. ----------------------------------------------------------------------- 部分中文 CLOLAR 处方信息（仅供参考） 化学名称: 2-氯-9-(2-去氧-2-氟-β-D-阿拉伯呋喃)-9H-嘌呤-6-胺,2-chloro-9-(2-deoxy-2-fluoro-β-D-arabinofuranosyl)-9H-purin-6-amine 英文名称: Clofarabine 药品名称：氯法拉滨 又名：Clofarabine，Clolar，卢伐拉滨，克罗拉滨 美国Bioenvision公司开发, Genzyme公司生产，04.12.28FDA快速审评通道批准本品上市。现本品未申请进口，原料制剂均为3.1类。Bioenvision公司于2006年5月宣布，欧盟委员会已经批准其clofarabine（Evoltra）上市。用于治疗急性淋巴细胞性白血病。本品适用范围为复发的或对至少2种疗法无应答且没有其它疗法可选用的儿科急性淋巴细胞性白血病患者。此外，本品还可用于初次诊断为白血病的21岁及以下患者。 适应症、用法用量：用于1～21岁顽固性或复发性急性淋巴细胞白血病（ALL）患者，患者此前至少接受过2种优先使用的疗法。本品先用5％葡萄糖注射液或0.9％NaCl注射液稀释。儿童推荐剂量为52mg/m2，每天输注2小时，连续5天。器官功能回复到基线后，治疗周期循环，大约每2～6周一次。 本品结合了氟达拉滨和克拉屈滨 的优点，既抑制DNA聚合酶，又抑制核糖核酸还原酶，是目前唯一可以特异性用于儿童白血病的药物；治疗白血病有效率高。两次常规化疗无应答的患者, 对该药的总反应率为31%；病人耐受性好，无不可预知的不良反应；具有潜在广谱抗肿瘤特性。 注：以下产品不同规格和不同价格，购买时请以电话咨询为准！ --------------------------------------------------------------- 原产地英文商品名: CLOLAR 1MG/ML 20MLS/VIAL 原产地英文药品名: CLOFARABINE 原产地英文化合物名称: 2-chloro-9-(2-deoxy-2-fluoro-β-D-arabinofuranosyl)-9H-purin-6-amine 中文参考商品译名: 克罗拉 1毫克/毫升 20毫升/瓶 中文参考药品译名: 氯法拉滨 中文参考化合物名称: 2-氯-9-(2-去氧-2-氟-β-D-阿拉伯呋喃)-9H-嘌呤-6-胺 生产厂家中文参考译名: 健赞 生产厂家英文名: GENZYME --------------------------------------------------------------- 原产地英文商品名: CLOLAR 1MG/ML 20MLS/VIAL 4VIALS/BOX 原产地英文药品名: CLOFARABINE 中文参考商品译名: 克罗拉 1毫克/毫升 20毫升/瓶 4瓶/盒 中文参考药品译名: 氯法拉滨 生产厂家中文参考译名: 健赞 生产厂家英文名: Genzyme --------------------------------------------------------------