Abstract：
BACKGROUND: Oral treprostinil is a prostacyclin analogue approved to treat pulmonary arterial hypertension (PAH) by delaying disease progression and improving exercise capacity. Higher doses of oral treprostinil correlate with increased treatment benefit. Titrations may be challenging due to common side effects of prostacyclin-class therapies.
METHODS: The multicenter, prospective, real-world, observational ADAPT Registry study followed adult patients with PAH for up to 78 weeks after initiating oral treprostinil (NCT03045029). Dosing, titration, and transitions of oral treprostinil were at the discretion of the prescriber. Patient-reported incidence and treatment of common side effects were collected to understand side effect management and tolerability. Insights from literature and expert recommendations were added to provide a consolidated resource for oral treprostinil use.
RESULTS: In total, 139 participants in ADAPT completed ≥1 weekly survey; (median age 60.0 years, 76 % female). Median treatment duration of oral treprostinil was 13.1 months. During early therapy (Months 1-5), 62 % (78/126) of patients reported headache and diarrhea, and 40 % (50/126) reported nausea. At Month 6, many patients who reported side effects during early therapy reported an improvement (61 % headache, 44 % diarrhea, 70 % nausea). Common side effect treatments, including acetaminophen, loperamide, and ondansetron, were effective. Approximately one-quarter of patients reporting the most common side effects were untreated at Month 6.
CONCLUSIONS: Patient selection for, and initiation and titration of, oral treprostinil should be individualized and may include parenteral treprostinil induction-transition for faster titration. Assertive side effect management may help patients reach higher and more efficacious doses of oral treprostinil.
METHODS: The multicenter, prospective, real-world, observational ADAPT Registry study followed adult patients with PAH for up to 78 weeks after initiating oral treprostinil (NCT03045029). Dosing, titration, and transitions of oral treprostinil were at the discretion of the prescriber. Patient-reported incidence and treatment of common side effects were collected to understand side effect management and tolerability. Insights from literature and expert recommendations were added to provide a consolidated resource for oral treprostinil use.
RESULTS: In total, 139 participants in ADAPT completed ≥1 weekly survey; (median age 60.0 years, 76 % female). Median treatment duration of oral treprostinil was 13.1 months. During early therapy (Months 1-5), 62 % (78/126) of patients reported headache and diarrhea, and 40 % (50/126) reported nausea. At Month 6, many patients who reported side effects during early therapy reported an improvement (61 % headache, 44 % diarrhea, 70 % nausea). Common side effect treatments, including acetaminophen, loperamide, and ondansetron, were effective. Approximately one-quarter of patients reporting the most common side effects were untreated at Month 6.
CONCLUSIONS: Patient selection for, and initiation and titration of, oral treprostinil should be individualized and may include parenteral treprostinil induction-transition for faster titration. Assertive side effect management may help patients reach higher and more efficacious doses of oral treprostinil.
摘要：
背景：口服曲前列环素是一种前列环素类似物，被批准通过延缓疾病进展和改善运动能力来治疗肺动脉高压（PAH）。较高剂量的口服曲前列环素与增加的治疗益处相关。由于前列环素类疗法的常见副作用，滴定可能具有挑战性。
方法：多中心，prospective,真实世界,观察性ADAPT注册研究在开始口服曲前列环素后随访成人PAH患者长达78周(NCT03045029).给药,滴定,口服曲前列环素的过渡由处方者自行决定。收集患者报告的常见副作用的发生率和治疗，以了解副作用的管理和耐受性。增加了文献和专家建议的见解，为口服曲前列环素的使用提供了综合资源。
结果：总计，139名ADAPT参与者完成≥1次每周调查；(中位年龄60.0岁，76%的女性）。口服曲前列环素的中位治疗时间为13.1个月。在早期治疗期间（1-5个月），62%（78/126）的患者报告头痛和腹泻，40%（50/126）报告恶心。在第6个月，许多在早期治疗期间报告副作用的患者报告改善（61％头痛，44%腹泻,70%恶心)。常见的副作用治疗，包括对乙酰氨基酚,洛哌丁胺,昂丹司琼有效.大约四分之一的报告最常见副作用的患者在第6个月未治疗。
结论：患者选择，口服曲前列环素的起始和滴定应个体化，并可能包括肠胃外曲前列环素诱导-转变以加快滴定。可靠的副作用管理可能有助于达到更高和更有效剂量的口服曲前列环素。
方法：多中心，prospective,真实世界,观察性ADAPT注册研究在开始口服曲前列环素后随访成人PAH患者长达78周(NCT03045029).给药,滴定,口服曲前列环素的过渡由处方者自行决定。收集患者报告的常见副作用的发生率和治疗，以了解副作用的管理和耐受性。增加了文献和专家建议的见解，为口服曲前列环素的使用提供了综合资源。
结果：总计，139名ADAPT参与者完成≥1次每周调查；(中位年龄60.0岁，76%的女性）。口服曲前列环素的中位治疗时间为13.1个月。在早期治疗期间（1-5个月），62%（78/126）的患者报告头痛和腹泻，40%（50/126）报告恶心。在第6个月，许多在早期治疗期间报告副作用的患者报告改善（61％头痛，44%腹泻,70%恶心)。常见的副作用治疗，包括对乙酰氨基酚,洛哌丁胺,昂丹司琼有效.大约四分之一的报告最常见副作用的患者在第6个月未治疗。
结论：患者选择，口服曲前列环素的起始和滴定应个体化，并可能包括肠胃外曲前列环素诱导-转变以加快滴定。可靠的副作用管理可能有助于达到更高和更有效剂量的口服曲前列环素。
