Congenital Hypothyroidism

2023年9月27日-10月1日，美国甲状腺协会（ATA）年会于美国华盛顿举办了百年庆典暨2023年年会，世界各地甲状腺领域的临床医生和研究专家同聚一堂，共话甲状腺疾病最新的临床和基础研究进展，为提升甲状腺疾病诊疗水平添砖加瓦。借着此次年会的平台，THYROID的主编Anna M. Sawka教授代表杂志社对中国医生和科学家的既往工作给予了高度肯定，并诚挚邀请更多国内专家学者以撰稿人和同行评审的角色共同推进甲状腺疾病领域的发展。此次年会设置了百余个讲题，涉及甲减、甲亢、甲癌和低风险甲状腺疾病等多个领域的内容，同时还涵盖了妊娠、甲癌等多个权威指南的更新讨论。会上精彩纷呈，多个话题引起了专家学者们的热切关注和讨论。回顾甲状腺诊疗的发展趋势蓄力下一征程大会主要从甲状腺基础科学、临床研究和外科诊疗三个方面剖析了过去一年里甲状腺疾病的主要进展，全方位地展现甲状腺疾病诊疗的新趋势，为下一年的发展奠定基调，这也是ATA能够在甲状腺疾病领域成为标杆的基础。关注基础和转化研究探索新的突破点在“影响人类和小鼠甲状腺功能的遗传缺陷”专题中，多位专家分享了人和鼠甲状腺功能的遗传缺陷、甲状腺激素信号传导在动物模型中的作用。Nadia Schoenmakers教授的研究结果表明，SLC26A7的纯合子功能缺失突变可导致甲状腺功能减退；Mingyao Liu教授团队的研究结果表明，XB130缺乏可引起甲状腺细胞极性紊乱和先天性甲状腺功能减退，这些研究有望转化为临床成果，为治疗甲状腺疾病提供新的切入和突破点。关注特殊人群推进全面管理进程在老年衰弱群体、儿童和妊娠群体中需要关注患者的治疗依从性、甲状腺疾病的并发症和对胎儿的潜在风险。Maria Papaleontiou教授分享的质性研究发现老年患者对医生的信任、医患共情以及健康的医患沟通是医生开处方的正向影响因素，保持良好的医患关系可能有助于提升治疗依从性。Ingbar和Braverman杰出奖颁布为行业树立榜样Ingbar和Braverman杰出奖是美国内分泌学领域的重量级奖项，旨在表彰在该领域做出杰出贡献的科学家，2023年这两个奖项分别颁给了Yaron Tomer教授和Rosalind Brown教授。在获奖报告中，Tomer教授分享了关于自噬在自身免疫性甲状腺疾病中作用的见解，其团队的研究表明CD40 ED的构象是CD40L激活其的关键以及甲状腺细胞可能需要内质网相关蛋白降解来清除突变的TG蛋白，这些结果有助于开发靶向治疗Graves病和甲状腺功能减退的药物。Brown教授则回顾了儿童甲状腺疾病的50年发展历程，其中先天性甲状腺功能减退症（CH）仍然是智力障碍最常见的原因之一，大多数CH的病因尚不明确，但碘代谢状态似乎在CH的致病机制中发挥关键作用。【链接到2023ATA年会系列报道｜探索甲亢治疗的演变】结合前沿科技优化甲状腺疾病的诊疗水平人工智能（AI）在甲状腺癌的诊断中也具有发展潜力，会上多位专家共同探讨了AI在甲状腺疾病中的探索性应用。有研究发现基于大量超声图像训练的AI模型可辅助判断分化型甲状腺癌（DTC）的淋巴结转移，基于卷积神经网络的AI模型可鉴别甲状腺结节的良恶性，准确率堪比高年资超声科医生。Einas Alkhatib教授采用电子甲状腺仪表板对新发Graves病患儿进行追踪可将3个月内达到游离甲状腺素(FT4)正常的比例从47%提高到90%，科技进步无疑为未来的研究和诊疗提供了崭新的途径。定期更新ATA系列指南为甲状腺疾病领域提供权威指导会上，新版甲状腺与妊娠指南编写委员会介绍了指南进程和特色内容，新指南包含97条推荐意见。指南的目标主要有三点：一是通过广泛的代表工作组提高指导方针影响；二是使设定的指导方针更加现代化；三是优化指南的证据基础。通过问卷调查确定了六个领域的主题和结果，如妊娠期及产后甲状腺疾病的定义和甲状腺功能测试和筛查等【链接到2023ATA年会系列报道｜孕育爱与健康，聚焦妊娠期甲状腺管理】。同时，甲状腺癌专家组也展示了指南在甲状腺结节、分化型甲状腺癌和小儿甲状腺癌三个方面的更新：新版甲状腺结节指南添加了新的ROMs分类，纳入了先前不可分类的结节；分化型甲状腺癌更新了推荐意见及证据的等级；儿童甲状腺癌指南根据现有的最佳科学证据或专家意见制定了新的框架并增加了动态风险分层及相应的管理措施，以期为甲状腺结节或分化型甲状腺癌患儿提供最高水平的内科和外科治疗【链接到2023ATA年会系列报道｜前沿进展助力甲状腺癌优化管理】。2023年ATA年会已圆满结束，但甲状腺疾病领域的研究和创新永不止步。我们期待在未来看到这些研究成果为患者带来更多获益！免责声明：本文仅作最新前沿信息交流之目的，文中所有观点不代表梅斯医学立场，亦不代表梅斯医学支持或反对文中观点。如需获得治疗方案指导，请前往正规医院就诊！

Online Pharmacy to Offer Hypothyroidism Treatments at Low Cost PARSIPPANY, N.J., March 7, 2023 /PRNewswire/ -- IBSA Pharma announced today that it has entered into an agreement with Mark Cuban Cost Plus Drug Company, PBC (Cost Plus Drugs) to offer Tirosint® (levothyroxine sodium) oral capsules at a low cash price via the Cost Plus Drugs online pharmacy. The agreement is the first of its kind for a branded levothyroxine drug. Levothyroxine is a synthetic hormone used to treat hypothyroidism. It is one of the most widely dispensed prescription medications in the United States with an estimated 116 million prescriptions filled in 2016.¹ Tirosint capsules is the leading branded gel capsule formulation of levothyroxine in the US. It is formulated without excipients that can cause tolerability and drug absorption problems commonly observed with traditional levothyroxine tablet therapies. Tirosint has money savings options available for patients, their availability in the Cost Plus Drugs program is a first for both companies. Cost Plus Drugs launched its online pharmacy in 2021 and was co-founded by investor-entrepreneur Mark Cuban to reduce the cost of prescription medications. It offers the most commonly prescribed drugs at low transparent prices by negotiating prices directly with manufacturers and passing along the savings to consumers. "Everyone should have access to safe, affordable prescription drugs at transparent prices" commented Cuban at the company's founding. Currently, most of Cost Plus Drugs' offerings are generic drugs. The agreement with IBSA marks one of the company's first forays into the branded segment of the US pharmaceutical market. Cuban is currently a Tirosint patient. Commenting on the agreement, IBSA Pharma CEO Nicholas Hart stated that "hypothyroidism is a prevalent condition with significant public health implications. We are delighted to offer Tirosint capsules to patients via Cost Plus Drugs. Tirosint has been clinically proven to provide the benefits of levothyroxine, the standard of care for treating hypothyroidism, in a formulation that is free of gluten, dyes, lactose, sugars and other excipients commonly found in tablet formulations. These can be problematic for many patients. Studies have shown that up to 31% of levothyroxine-treated patients have gastrointestinal and other problems that can lead to underperformance of levothyroxine therapy and suboptimal control of hypothyroidism.²" Tirosint capsules is a prescription medication that IBSA Pharma markets to physicians and patients. Tirosint capsules are indicated to treat hypothyroidism in patients six years and older and available in 13,25,50,75,88, 100,112,125,137, 150, 175 and 200 mcg doses. Additional information, including money saving options, can be found at . About Hypothyroidism Hypothyroidism is an endocrine disorder with numerous causes resulting in a deficiency in thyroid hormone. More than 27 million adults have been diagnosed with hypothyroidism. ² Up to 13 million Americans have undiagnosed hypothyroidism. ³ About 2% of the U.S. population has pronounced hypothyroidism, and as much as 10% has subclinical (mild) hypothyroidism. The condition is most common in women over 40 years of age and in the elderly of both sexes.⁴ The signs and symptoms of hypothyroidism are nonspecific and may include fatigue, forgetfulness, depression, constipation, muscle cramps, weight gain, dry skin and hair loss.⁵ TSH laboratory tests are recommended as first-line screening tests for thyroid dysfunction.⁶ Levothyroxine sodium is a synthetic version of a hormone that is normally produced by the thyroid gland. It is used to treat patients who suffer from hypothyroidism or inadequate levels of thyroid stimulating hormone (TSH). About IBSA Pharma Inc. IBSA Pharma is part of IBSA Group, which is headquartered in Lugano, Switzerland. It currently markets 4 prescription medications in the United States: Tirosint capsules and Tirosint-SOL (levothyroxine sodium) oral solution for the treatment of hypothyroidism; Licart (diclofenac epolamine) topical system 1.3% and Flector (diclofenac epolamine) topical system 1.3% for the treatment of acute pain from minor strains, sprains, and contusions. IBSA Institut Biochimique SA IBSA (Institut Biochimique SA) is a Swiss multinational pharmaceutical Company, founded in 1945 in Lugano. Today, its products are present in over 90 Countries on 5 continents, through the Company's 17 subsidiaries located in Europe, China, and the United States. The company has a consolidated turnover of 800 million CHF, and employs over 2,000 people between headquarters, subsidiaries, and production sites. IBSA holds 90 families of approved patents, plus others under development, as well as a vast portfolio of products, covering 10 therapeutic areas: reproductive medicine, endocrinology, pain and inflammation, osteoarticular, aesthetic medicine, dermatology, uro-gynaecology, cardiometabolic, respiratory, consumer health. It is also one of the largest operators worldwide in the area of reproductive medicine, and one of the world's leaders in hyaluronic acid-based products. IBSA has based its philosophy on four pillars: Person, Innovation, Quality and Responsibility. To report SUSPECTED ADVERSE REACTIONS, contact IBSA Pharma Inc. at 1-800-587-3513 or FDA at 1-800- FDA-1088 For more information about IBSA, visit . For full prescribing information, visit . References Verble, M. Inappropriate use and overdose of PPIs: a strategy for discontinuation. Pharmacy Times, August 2018:35. IMS Institute for Healthcare Informatics Report, "Medicine Use and the Shifting Costs of Healthcare"; 2014; pg. 46 Booth, M, 2019. Published online at: Canaris GJ, Manowitz NR, Mayor G, Ridgeway EC, "The Colorado Thyroid Disease Prevalence Study", Arch of Internal Medicine; 2000;160: 526-534 McDermott MT. "In the clinic: hypothyroidism". Annals of Internal Medicine; 2009;151 (11): ITC-6-1 Soh SB. Laboratory testing in thyroid conditions: pitfalls and clinical utility. Ann Lab Med. 2019;39(1):3. IMPORTANT SAFETY INFORMATION Warning: Not for the Treatment of Obesity or for Weight Loss Thyroid hormones, including TIROSINT, either alone or with other therapeutic agents, should not be used for the treatment of obesity or for weight loss. In euthyroid patients, doses within the range of daily hormonal requirements are ineffective for weight reduction. Larger doses may produce serious or even life-threatening manifestations of toxicity, particularly when given in association with sympathomimetic amines such as those used for their anorectic effects. Contraindications Uncorrected adrenal insufficiency Warnings And Precautions Cardiac adverse reactions in the elderly and in patients with underlying cardiovascular disease. Initiate TIROSINT at less than the full replacement dose because of the increased risk of cardiac adverse reactions, including atrial fibrillation Myxedema coma: Do no use oral thyroid hormone drug products to treat myxedema coma Acute adrenal crisis in patients with concomitant adrenal insufficiency: Treat with replacement glucocorticoids prior to initiation of TIROSINT treatment Prevention of hyperthyroidism or incomplete treatment of hypothyroidism: Proper dose titration and careful monitoring is critical to prevent the persistence of hypothyroidism or the development of hyperthyroidism Worsening of diabetic control: Therapy in patients with diabetes mellitus may worsen glycemic control and result in increased antidiabetic agent or insulin requirements. Carefully monitor glycemic control after starting, changing, or discontinuing thyroid hormone therapy Decreased bone mineral density associated with thyroid hormone over-replacement. Over-replacement can increase bone reabsorption and decrease bone mineral density. Give the lowest effective dose Use for the suppression of nontoxic diffuse goiter or nodular thyroid disease: Use is not recommended in iodine-insufficient patients Limitations of Use Not indicated for suppression of benign thyroid nodules and nontoxic diffuse goiter in iodine-sufficient patients Not indicated for treatment of transient hypothyroidism during the recovery phase of subacute thyroiditis Adverse Reactions Common adverse reactions with levothyroxine therapy are primarily those of hypothyroidism due to therapeutic overdosage. They include the following: General: fatigue, increased appetite, weight loss, heat intolerance, fever, excessive sweating Central Nervous System: headache, hyperactivity, nervousness, anxiety, irritability, emotional lability, insomnia Musculoskeletal: tremors, muscle weakness Cardiovascular: palpitations, tachycardia, arrythmias, increased pulse and blood pressure, heart failure, angina, myocardial infarction, cardiac arrest Respiratory: dyspnea Gastrointestinal (GI): diarrhea, vomiting, abdominal cramps, elevations in liver function tests Dermatologic: hair loss, flushing Endocrine: decreased bone mineral density Reproductive: menstrual irregularities, impaired fertility Adverse reactions in Children Pseudotumor cerebri and slipped capital femoral epiphysis have been reported in children receiving levothyroxine therapy. Overtreatment may result in craniosynostosis in infants and premature closure of the epiphyses in children with resultant compromised adult height. Seizures have been reported rarely with the institution of levothyroxine therapy. Hypersensitivity Reactions Hypersensitivity reactions to inactive ingredients (in this product or other levothyroxine products) have occurred in patients treated with thyroid ho0rmone products. These include urticaria, pruritis, skin rash, flushing, angioedema, various GI symptoms (abdominal pain, nausea, vomiting and diarrhea), fever, arthralgia, serum sickness and wheezing. Hypersensitivity to levothyroxine itself is not known to occur. PM-04-23-0001 View original content to download multimedia: SOURCE IBSA Pharma Inc.

Guidance Supports Use of Tirosint®-SOL In Pediatric Population PARSIPPANY, N.J., Jan. 12, 2023 /PRNewswire/ -- The American Academy of Pediatrics (AAP), the Pediatric Endocrine Society (PES) and the American Thyroid Association (ATA) have published new guidelines for the treatment of congenital hypothyroidism (CH). For the first time, these leading medical societies cite levothyroxine oral solution as an option for managing patients with CH. Congenital hypothyroidism is a condition affecting up to 1 in every 2,000-4,000 live births in the United States annually. It is a leading cause of intellectual deficiency among infants.1 CH is typically treated with levothyroxine, a synthetic thyroid hormone that is administered daily to restore thyroid hormones to normal levels. Tirosint-SOL (levothyroxine sodium) oral solution is the leading FDA-approved levothyroxine oral solution currently available in the US. New Treatment Guidelines for Congenital Hypothyroidism Published Mention Levothyroxine Oral Solution The newly published treatment guidelines reinforce the use of Tirosint-SOL in the pediatric population. A randomized clinical study published in the journal Hormone Research in Pediatrics examined the efficacy and safety of treating infants newly diagnosed with CH with levothyroxine oral solution in lieu of traditional levothyroxine tablet formulations. In total, 78 infants with congenital hypothyroidism (CH) were studied.2 The study's authors concluded that levothyroxine oral solution was able to successfully treat CH patients to their therapeutic goals. Equally important, no study subject experienced cranial stenosis, a serious adverse effect experienced by infants who are exposed to excessive doses of levothyroxine. "The mention of levothyroxine oral solution in newly published guidelines is an acknowledgement of the utility of drugs like Tirosint-SOL for treating infants with congenital hypothyroidism" stated Susan Rose, Professor Emeritus of the University of Cincinnati College of Medicine, and Cincinnati Children's Hospital Medical Center. "However, liquid formulations like Tirosint-SOL are more easily absorbed than traditional tablet formulations, the current standard of care. Clinicians wishing to prescribe it should take this into consideration." Rose went on to add that "the availability of FDA-approved levothyroxine oral solutions makes it critical to avoid the use of extemporaneously compounded liquid levothyroxine preparations which have been demonstrated to be pharmacologically inconsistent. Clinicians wishing to prescribe liquid levothyroxine for children now have an FDA-approved oral solution." The new treatment guidelines for congenital hypothyroidism were developed jointly by the American Academy of Pediatrics (AAP), the Pediatric Endocrine Society (PES) and the American Thyroid Association (ATA). The new guidelines were published in Pediatrics, the Journal of the American Academy of Pediatrics. The journal is available in print and online. Commenting on the publication of the new guidelines, IBSA Pharma Chief Business and Medical Officer Dr. Carolyn Kong stated that "congenital hypothyroidism is a serious condition with significant public health implications. Traditional levothyroxine tablet formulations have been used for years to treat this population. These formulations require caregivers to crush a child's medication into powder and then to mix it into water before administration. The availability of a levothyroxine oral solutions like Tirosint®-SOL, which has been demonstrated to be effective and safe for infants with CH, now provides patients and healthcare practitioners with a simple, consistent, and convenient option for treating CH." Tirosint®-SOL is the only FDA-approved levothyroxine oral solution available in monodose packaging for convenience and dosing precision. It does not contain gluten, dyes, colorants, lactose, and other ingredients that can cause tolerability and absorption problems. Its unique formulation consists of only three ingredients – levothyroxine, glycerol, and water – which is packaged in patented monodose ampules for precision and convenience. Tirosint®-SOL is indicated to treat the full spectrum of hypothyroid patients regardless of age. It can be administered by pouring the contents of the monodose ampule directly into the mouth or using a spoon or mixing it with water. It is currently available in 15 dosage strengths including unique 13, 37.5, 44 and 62.5 microgram dosing options, giving it the widest dosing range of any levothyroxine therapy available. To help provide patients with cost-effective access to Tirosint®-SOL, IBSA recently enhanced the Tirosint®-SOL Copay Savings Coupon Program. With the new program, eligible patients with commercial insurance can pay as little as $4 for a one-month supply of Tirosint®-SOL or $0 for a 3-month supply. Additional information, including money-saving options for patients without commercial insurance or with high copays or deductibles, can be found at . About Congenital Hypothyroidism (CH) Congenital hypothyroidism (CH) is an endocrine disorder present in up to 1 in every 2,000 - 4,000 live births in the United States each year. It is the result of inadequate thyroid function which results in low or inexistent levels of thyroid hormones. Thyroid hormones are key to the development of human infants. Untreated, or sub-optimally treated, CH is a leading cause of intellectual disability.¹ Levothyroxine is the current standard for treating CH. It is a synthetic thyroid hormone that is administered daily and is available in intravenous and oral formulations. Oral tablets are the most popular form of levothyroxine and must be crushed and added to water before administration to infants. Levothyroxine therapy for CH, once started, continues for three years to life depending on the severity of the disease. Levothyroxine is available by prescription only. About IBSA Pharma Inc. IBSA Pharma is part of IBSA Group which is headquartered in Lugano, Switzerland. IBSA Pharma currently markets Tirosint® (levothyroxine sodium) capsules and Tirosint®-SOL (levothyroxine sodium) oral solution for the treatment of hypothyroidism and Licart® (diclofenac epolamine) topical system 1.3% and Flector® (diclofenac epolamine) topical system 1.3% for the treatment of acute pain due to strains, sprains, and contusions. IBSA Institut Biochimique SA IBSA (Institut Biochimique SA) is a Swiss multinational pharmaceutical Company, founded in 1945 in Lugano. Today, its products are present in over 90 Countries on 5 continents, through the Company's 17 subsidiaries located in Europe, China, and the United States. The company has a consolidated turnover of 800 million CHF, and employs over 2,000 people between headquarters, subsidiaries and production sites. IBSA holds 90 families of approved patents, plus others under development, as well as a vast portfolio of products, covering 10 therapeutic areas: reproductive medicine, endocrinology, pain and inflammation, osteoarticular, aesthetic medicine, dermatology, uro-gynaecology, cardiometabolic, respiratory, consumer health. It is also one of the largest operators worldwide in the area of reproductive medicine, and one of the world's leaders in hyaluronic acid-based products. IBSA has based its philosophy on four pillars: Person, Innovation, Quality and Responsibility. To report SUSPECTED ADVERSE REACTIONS, contact IBSA Pharma Inc. at 1-800-587-3513 or FDA at 1-800- FDA-1088 For more information about IBSA, visit . For full prescribing information, visit . References 1 Rastogi V et al, "Congenital hypothyroidism", Orphanet Journal of Rare Diseases, 2010; 5:17 2 Peroni et al, "Congenital hypothyroidism treatment in infants: a comparative study between liquid and tablet formulations of levothyroxine", Hormone Res Paediatrics, 2014: (81) 50 -54 IMPORTANT SAFETY INFORMATION WARNING: NOT FOR THE TREATMENT OF OBESITY OR FOR WEIGHT LOSS. See full prescribing information for complete boxed warning Thyroid hormones, including TIROSINT-SOL, should not be used for the treatment of obesity or for weight loss. Doses beyond the range of daily hormonal requirements may produce serious or even life-threatening manifestations of toxicity CONTRAINDICATIONS Hypersensitivity to glycerol Uncorrected adrenal insufficiency WARNINGS AND PRECAUTIONS Cardiac adverse reactions in the elderly and in patients with underlying cardiovascular disease: Initiate TIROSINT-SOL at less than the full replacement dose because of the increased risk of cardiac adverse reactions, including atrial fibrillation Myxedema coma: Do not use oral thyroid hormone drug products to treat myxedema coma Acute adrenal crisis in patients with concomitant adrenal insufficiency: Treat with replacement glucocorticoids prior to initiation of TIROSINT-SOL treatment Prevention of hyperthyroidism or incomplete treatment of hypothyroidism: Proper dose titration and careful monitoring is critical to prevent the persistence of hypothyroidism or the development of hyperthyroidism Worsening of diabetic control: Therapy in patients with diabetes mellitus may worsen glycemic control and result in increased antidiabetic agent or insulin requirements. Carefully monitor glycemic control after starting, changing, or discontinuing thyroid hormone therapy Decreased bone mineral density associated with thyroid hormone over-replacement. Over-replacement can increase bone reabsorption and decrease bone mineral density. Give the lowest effective dose Limitations of Use Not indicated for suppression of benign thyroid nodules and nontoxic diffuse goiter in iodine-sufficient patients Not indicated for treatment of transient hypothyroidism during the recovery phase of subacute thyroiditis Adverse Reactions Adverse reactions with TIROSINT-SOL are primarily those of hyperthyroidism due to therapeutic overdosage including arrhythmias, myocardial infarction, dyspnea, muscle spasm, headache, nervousness, irritability, insomnia, tremors, muscle weakness, increased appetite, weight loss, diarrhea, heat intolerance, menstrual irregularities, and skin rash PM 01-22-0102