近日，美国FDA批准Pomalyst（pomalidomide，泊马度胺）胶囊治疗其它抗癌药治疗后病情依然进展的多发性骨髓瘤患者。

多发性骨髓瘤是一种血癌，主要影响老龄人群，自骨髓的浆细胞诱发。据美国国家癌症研究院估计，每年约有2.17万名美国人被诊断出患有多发性骨髓瘤，1.071万名死于这种疾病。

Pomalyst是一种药丸，它调节人体的免疫系统，摧毁癌细胞并抑制其生长。它适宜用于先前至少已经接受过两种治疗药物，包括来那度胺（lenalidomide）和硼替佐米（bortezomib），对治疗没有应答（未能奏效）和在最后一次治疗后60天内进展（复发和难治性）的患者。

Pomalyst是免疫调节剂一类的，继来那度胺和沙利度胺（thalidomide）之后第三个药物。多发性骨髓瘤治疗需要“度身定制”，以满足各别患者的需要，Pomalyst的获准为其它药物治疗无效的患者提供了新的选择。

批准日期：2013年1月8日 公司：Celgene Corporation 

POMALYST（泊马度胺 pomalidomide）胶囊，用于口服

美国最初批准：2013年

警告：

胚胎 - 胎儿毒性和静脉和动脉血栓栓塞

查看完整的盒装警告的完整处方信息

胚胎毒性

POMALYST在怀孕期间禁忌。 POMALYST是沙利度胺类似物。沙利度胺是一种已知的人类致畸因子，可导致严重危及生命的出生缺陷。

对于具有生殖潜力的女性：在开始治疗前排除怀孕。通过使用2种可靠的避孕方法预防治疗期间的怀孕。

POMALYST仅通过名为POMALYSTREMS®的受限程序提供。

静脉和动脉血栓栓塞

用POMALYST治疗的多发性骨髓瘤患者发生深静脉血栓形成（DVT），肺栓塞（PE），心肌梗死和中风。建议进行抗血栓预防。

最近的重大变化

警告和注意事项：03/18

作用机制

Pomalidomide是沙利度胺的类似物，具有免疫调节，抗血管生成和抗肿瘤特性。 pomalidomide的细胞活性是通过其目标cereblon介导的，cullin环E3泛素连接酶复合物的组成部分。在体外，在药物存在下，底物蛋白（包括Aiolos和Ikaros）被靶向用于遍在蛋白化和随后的降解，导致直接的细胞毒性和免疫调节作用。在体外细胞测定中，pomalidomide抑制增殖并诱导造血肿瘤细胞的凋亡。另外，在来那度胺敏感和来那度胺抗性细胞系中，pomalidomide抑制来那度胺抗性多发性骨髓瘤细胞系的增殖并与地塞米松协同诱导肿瘤细胞凋亡。

Pomalidomide增强T细胞和自然杀伤（NK）细胞介导的免疫并抑制单核细胞产生促炎细胞因子（例如TNF-α和IL-6）。Pomalidomide在小鼠肿瘤模型和体外脐带模型中显示出抗血管生成活性。

适应症和用法

POMALYST是一种沙利度胺类似物，与地塞米松联合用于多发性骨髓瘤患者，这些患者至少接受过两种治疗，包括来那度胺和蛋白酶体抑制剂，并且在最后一次治疗完成后60天或之内证实了疾病进展。

剂量和给药

多发性骨髓瘤：每天4毫克，在重复的28天周期的第1-21天口服，直至疾病进展。地塞米松给药参见第14.1节。

剂量形式和强度

胶囊：1毫克，2毫克，3毫克和4毫克

禁忌症

怀孕

警告和注意事项

死亡率增加：当pembrolizumab加入地塞米松和沙利度胺类似物时，在多发性骨髓瘤患者中观察到。

血液学毒性：中性粒细胞减少症是最常报告的3/4级不良事件。监测患者的血液学毒性，尤其是中性粒细胞减少症。

肝毒性：肝功能衰竭，包括死亡;每月监测肝功能检查。

包括过敏反应的严重皮肤反应：已报道血管性水肿和严重的皮肤反应，包括史蒂文斯 - 约翰逊综合征，中毒性表皮坏死松解症，嗜酸性粒细胞增多的药物反应和全身症状。停止POMALYST治疗血管性水肿和严重反应。

肿瘤裂解综合征（TLS）：监测有TLS风险的患者（即肿瘤负荷高的患者）并采取适当的预防措施。

不良反应

最常见的不良反应（≥30％）包括疲劳和虚弱，中性粒细胞减少，贫血，便秘，恶心，腹泻，呼吸困难，上呼吸道感染，背痛和发热。

要报告疑似不良反应，请致电1-888-423-5436联系Celgene Corporation或致电1-800-FDA-1088或www.fda.gov/medwatch联系FDA。

药物相互作用

强CYP1A2抑制剂：避免同时使用强效CYP1A2抑制剂。如果必须使用强效CYP1A2抑制剂，则将POMALYST剂量减少50％。

用于特定人群

哺乳期：建议女性不要母乳喂养。

肾功能损害：严重肾功能不全患者的POMALYST剂量减少至少25％

如何提供/存储和处理

如何提供

深蓝色不透明帽和黄色不透明的身体，用白色墨水在帽子上印上“POML”，在黑色墨水上用“1毫克”印在身上

1毫克21瓶（NDC 59572-501-21）

1毫克100瓶（NDC 59572-501-00）

深蓝色不透明帽和橙色不透明的身体，在帽子上印有“POML”，在身体上印有“2毫克”的白色墨水

2毫克21瓶（NDC 59572-502-21）

2毫克100瓶（NDC 59572-502-00）

深蓝色不透明帽和绿色不透明的身体，帽子上印有“POML”，白色墨水上印有“3毫克”的身体

3毫克21瓶（NDC 59572-503-21）

3毫克100瓶（NDC 59572-503-00）

深蓝色不透明帽和蓝色不透明的身体，帽子上印有“POML”，白色墨水上印有“4毫克”的身体

4毫克21瓶（NDC 59572-504-21）

4毫克100瓶（NDC 59572-504-00）

存储

储存在20°C-25°C（68°F-77°F）;允许的偏差为15°C-30°C（59°F-86°F）。 [见USP受控室温]。

处理和处置

在处理POMALYST时应小心谨慎。 POMALYST胶囊不应打开或压碎。如果来自POMALYST的粉末接触皮肤，请立即用肥皂和水彻底清洗皮肤。如果POMALYST接触粘膜，请用水彻底冲洗。

遵循正确处理和处置抗癌药物的程序

完整资料附件：https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=2b25ef01-5c9e-11e1-b86c-0800200c9a66

Pomalyst® (pomalidomide) capsules

POMALYST® (pomalidomide) is a prescription medicine, taken along with the medicine dexamethasone, used to treat people with multiple myeloma who have previously received at least 2 medicines to treat multiple myeloma, including a proteasome inhibitor and lenalidomide, and whose disease has become worse during treatment or within 60 days of finishing the last treatment. It is not known if POMALYST is safe and effective in children.

Important Safety Information

What is the most important information I should know about POMALYST?

Before you begin taking POMALYST, you must read and agree to all of the instructions in the POMALYST REMS® program. Before prescribing POMALYST, your healthcare provider (HCP) will explain the POMALYST REMS program to you and have you sign the Patient-Physician Agreement Form.

POMALYST can cause serious side effects, including:

Possible birth defects (deformed babies) or death of an unborn baby. Females who are pregnant or plan to become pregnant must not take POMALYST. –POMALYST is similar to the medicine thalidomide (THALOMID®), which is known to cause severe life-threatening birth defects. POMALYST has not been tested in pregnant females. POMALYST has harmed unborn animals in animal testing.

–Females must not get pregnant for at least 4 weeks before starting POMALYST, while taking POMALYST, during any breaks (interruptions) in your treatment with POMALYST, and for at least 4 weeks after stopping POMALYST.

–Females who can become pregnant: ◦–Must have pregnancy tests weekly for 4 weeks once treatment has started, then every 4 weeks if your menstrual cycle is regular or every 2 weeks if your menstrual cycle is irregular. If you miss your period or have unusual bleeding, you will need to have a pregnancy test and receive counseling.

◦–Must agree to use 2 different forms of effective birth control at the same time, for at least 4 weeks before, while taking, during any breaks (interruptions) in treatment, and for at least 4 weeks after stopping POMALYST. Talk with your HCP to find out about options for acceptable forms of birth control that you may use to prevent pregnancy.

–If you become pregnant while taking POMALYST, stop taking it right away and call your HCP. If your HCP is not available, you can call Celgene Customer Care Center at 1-888-423-5436. Healthcare providers and patients should report all cases of pregnancy to FDA MedWatch at 1-800-FDA-1088, and Celgene Corporation at 1-888-423-5436. There is a pregnancy exposure registry that monitors the outcomes of females who take POMALYST during pregnancy, or if their male partner takes POMALYST and they are exposed during pregnancy. You can enroll in this registry by calling Celgene Corporation at the phone number listed above.

–POMALYST can pass into human semen. Males, including those who have had a vasectomy, must always use a latex or synthetic condom during any sexual contact with a pregnant female or a female that can become pregnant while taking POMALYST, during any breaks (interruptions) in your treatment with POMALYST, and for at least 4 weeks after stopping POMALYST. ◦–If a female becomes pregnant with your sperm, you should call your HCP right away. The baby may be exposed to POMALYST and may be born with birth defects.

◦–Do not have unprotected sexual contact with a female who is or could become pregnant. Tell your HCP if you have unprotected sexual contact with a female who is or could become pregnant.

◦–Do not donate sperm while taking POMALYST, during any breaks (interruptions) in your treatment, and for at least 4 weeks after stopping POMALYST.

–Do not donate blood while you take POMALYST, during any breaks (interruptions) in your treatment, and for at least 4 weeks after stopping POMALYST. If someone who is pregnant gets your donated blood, her baby may be exposed to POMALYST and may be born with birth defects.

Blood clots in your arteries, veins, and lungs; heart attack; and stroke. Most people who take POMALYST will also take a blood thinner medicine to help prevent blood clots. –Before taking POMALYST, tell your HCP if you have had a blood clot in the past, if you have high blood pressure or hyperlipidemia (high level of fat in your blood), or if you smoke. Tell your HCP about all the medicines you take because certain other medicines can also increase your risk for blood clots.

–Call your HCP or get medical help right away if you get any of the following during treatment with POMALYST: (1) signs or symptoms of a blood clot in the lung, arm, or leg, including shortness of breath, chest pain, or arm or leg swelling; (2) signs or symptoms of a heart attack, including chest pain that may spread to the arms, neck, jaw, back, or stomach area (abdomen); feeling sweaty; shortness of breath; feeling sick; or vomiting; or (3) signs or symptoms of stroke, including sudden numbness or weakness, especially on one side of the body; severe headache or confusion; or problems with vision, speech, or balance.

Who should not take POMALYST?

Do not take POMALYST if you are pregnant, plan to become pregnant, or become pregnant during treatment with POMALYST. See "What is the most important information I should know about POMALYST?"

What should I tell my healthcare provider (HCP) before taking POMALYST?

If you smoke cigarettes (POMALYST may not work as well in people who smoke), have any other medical conditions, or are breastfeeding. Do not breastfeed during treatment with POMALYST—it is not known if POMALYST passes into breast milk and can harm the baby.

Tell your HCP about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. POMALYST and other medicines may affect each other, causing serious side effects. Talk with your HCP before taking any new medicines.

How should I take POMALYST?

Take POMALYST exactly as prescribed and follow all the instructions of the POMALYST REMS program.

Swallow POMALYST capsules whole with water 1 time a day. Do not break, chew, or open capsules.

Take POMALYST at the same time each day with or without food.

If you are on hemodialysis, take POMALYST after hemodialysis on hemodialysis days.

Do not open POMALYST capsules or handle them any more than needed. If you touch a broken POMALYST capsule or the medicine in the capsule, wash the area of your body right away with soap and water.

If you miss a dose of POMALYST and it has been less than 12 hours since your regular time, take POMALYST as soon as you remember. If it has been more than 12 hours, just skip your missed dose. Do not take 2 doses at the same time.

If you take too much POMALYST, call your healthcare provider (HCP) right away.

Do not share POMALYST with other people. It may cause birth defects and other serious problems.

What are the possible side effects of POMALYST?

See "What is the most important information I should know about POMALYST?"

POMALYST can cause serious side effects, including: –Low white blood cells (neutropenia), low platelets (thrombocytopenia), and low red blood cells (anemia) are common with POMALYST, but can also be serious. You may need a blood transfusion or certain medicines if your blood counts drop too low. Your blood counts should be checked by your healthcare provider (HCP) weekly for the first 8 weeks of treatment and monthly after that.

–Severe liver problems, including liver failure or death. Your HCP should do blood tests to check your liver function during your treatment with POMALYST. Tell your HCP right away if you develop any of the following symptoms: yellowing of your skin or the white parts of your eyes (jaundice); dark or brown (tea-colored) urine; pain on the upper right side of your stomach area (abdomen); bleeding or bruising more easily than normal, or feeling very tired.

–Severe allergic and skin reactions. Call your HCP if you have any symptoms of an allergic reaction, including: swelling of your lips, mouth, tongue, or throat; trouble breathing; or skin reaction.

–Dizziness and confusion. Avoid taking other medicines that may cause dizziness and confusion during treatment with POMALYST. Avoid situations that require you to be alert until you know how POMALYST affects you.

–Nerve damage. Stop taking POMALYST and call your HCP if you develop numbness, tingling, pain, or a burning sensation in your hands, legs, or feet.

–New cancers (malignancies). New cancers, including certain blood cancers (acute myelogenous leukemia or AML) have been seen in people who received POMALYST. Talk with your HCP about your risk.

–Tumor Lysis Syndrome (TLS). TLS is caused by the fast breakdown of cancer cells. TLS can cause kidney failure and the need for dialysis treatment, abnormal heart rhythm, seizure, and sometimes death. Your HCP may do blood tests to check you for TLS.

The most common side effects of POMALYST include tiredness, weakness, constipation, nausea, diarrhea, shortness of breath, upper respiratory tract infection, back pain, and fever.