肢端肥大症:病理生理学考虑因素和治疗方案,包括口服生长抑素类似物的演变作用。

IF 2.7 Q2 PATHOLOGY Pathophysiology Pub Date : 2023-09-01 DOI:10.3390/pathophysiology30030029
Charles P Daniel, Maxwell J Wagner, Grant E Borne, Connor J Plaisance, Shahab Ahmadzadeh, Alfonso Aquino, Sahar Shekoohi, Adam M Kaye, Elyse M Cornett, Alan D Kaye
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引用次数: 0

摘要

肢端肥大症是人生第五个十年最常见的诊断疾病,有多种治疗选择。在这方面,Mycapssa®是美国食品药品监督管理局批准的第一种用于治疗肢端肥大症的口服奥曲肽胶囊,它结合了生长抑素受体配体奥曲肽的功效,以及每天两次的口服胶囊。在不能选择手术治疗的情况下,生长抑素类似物,包括奥曲肽,是肢端肥大症的第一道医疗治疗线,需要患者的医疗保健提供者定期皮下或肌肉注射。奥曲肽胶囊(Mycapssa®)通过将奥曲肽与其他赋形剂结合,产生一种瞬态渗透增强技术,改善奥曲肽通过胃肠壁进入小肠的细胞旁转运,为这些生长抑素受体配体注射提供了一种替代方案。在多项试验中,包括开放标签(CH-ACM-01)、双盲安慰剂对照(CHIASMA OPTIMAL)和开放标签延长试验期(CHIASMA OPTIMAL OLE),Mycapssa®奥曲肽胶囊保持了IGF-1和GH水平的一致生化标准化,安全性与注射的生长抑素受体配体相似,以及患者对奥曲肽胶囊持续治疗的偏好。虽然临床试验数据支持奥曲肽胶囊(Mycapssa®)在GH和IGF-1水平的药理学管理中的使用,但关于该药物的疗效、耐受性以及在女性或儿童特定人群中的使用的数据很少。更好地了解口服奥曲肽胶囊在不同人群肢端肥大症长期医疗管理中的疗效、应用和作用,对于最好地确定临床医生的风险/益处至关重要。
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Acromegaly: Pathophysiological Considerations and Treatment Options Including the Evolving Role of Oral Somatostatin Analogs.

Acromegaly is a condition most commonly diagnosed in the fifth decade of life and has numerous treatment options. In this regard, Mycapssa® is the first FDA-approved oral octreotide capsule for treating acromegaly, combining the efficacy of the somatostatin receptor ligand, octreotide, with the ease of a twice-daily oral capsule. Where surgical treatment is not an option, somatostatin analogs, including octreotide, are the first line of medical treatment for acromegaly, requiring regular subcutaneous or intramuscular injections administered by a patient's healthcare provider. Octreotide capsules (Mycapssa®) provide an alternative to these somatostatin receptor ligand injections by combining octreotide with other excipients to produce a transient permeability enhancer technology that improves paracellular transport of octreotide across the gastrointestinal wall into the small intestine. Across multiple trials, including open-label (CH-ACM-01), double-blind placebo-controlled (CHIASMA OPTIMAL), and open-label extension of the trial period (CHIASMA OPTIMAL OLE), Mycapssa® octreotide capsules maintained a consistent biochemical normalization of IGF-1 and GH levels, safety profiles similar to injected somatostatin receptor ligands, and patient preference to continued treatment with octreotide capsules. While clinical trial data supports the use of octreotide capsules (Mycapssa®) in the pharmacological management of GH and IGF-1 levels, very little data exist regarding the drug's efficacy, tolerability, and use in female or pediatric-specific populations. A better understanding of the efficacy, application, and role of oral octreotide capsules in the long-term medical management of acromegaly in a diversity of populations is imperative to best determine the risks/benefits for the clinician.

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来源期刊
Pathophysiology
Pathophysiology Medicine-Pathology and Forensic Medicine
CiteScore
3.10
自引率
0.00%
发文量
48
期刊介绍: Pathophysiology is an international journal which publishes papers in English which address the etiology, development, and elimination of pathological processes. Contributions on the basic mechanisms underlying these processes, model systems and interdisciplinary approaches are strongly encouraged.
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