Supplementary materials: Adherence, duration and healthcare costs in a real-world population of patients with acromegaly

<b>These are peer-reviewed supplementary materials for the article</b><b> </b><b>'</b><b>Adherence, duration and healthcare costs in a real-world population of patients with acromegaly</b><b>'</b><b> </b><b>published in the</b><b> </b><b><i>Journal of Comparative Effectiveness Research</i></b><b>.</b><b>Table A: </b>List of CPT and ICD codes for hypophysectomy and stereotactic radiosurgery<b>Table B: </b>List of ICD-9 codes and ICD-10 codes for comorbidities of interest<b>Aim:</b> The aim of this study was to describe treatment patterns among patients with acromegaly who are newly treated with acromegaly medical therapy. <b>Materials &amp;methods: </b>Data from IQVIA Pharmetrics Plus R ?Database from 1 January 2013 to 30 June 2023 were used to identify patientswith acromegaly who started a new acromegaly medical therapy and observe their treatment patterns. Patients were required to have at least 12 months of data without any acromegaly therapy (medication or surgery) prior to the index date and at least 6 months of follow-up. Comorbidities were measured during the baseline period. Adherence, persistence, medication and switching were measured during follow-up. <b>Results:</b> A total of 453 patients with acromegaly who were newly treated with acromegaly medical therapy and had no evidence of acromegaly therapy for at least 12 months were identified. Among these patients, 46.1% (n = 206) were treated with cabergoline as their index treatment, 24.5% (n = 111) with injectable octreotide, 15.0% (n = 68) with lanreotide, 5.5% (n = 25) with bromocriptine, 4.9% (n = 22) with pegvisomant, 2.2% (n = 10) with pasireotide, 1.1% (n = 5) with oral octreotide, 0.4% (n = 2) with cabergoline + octreotide, and 0.2% (n = 1) with cabergoline + lanreotide. By the end of the follow-up period, 54.3% (n = 246) were not on any treatment, 19.6% (n = 89) remained on the index treatment, and the remaining 26.0% (n = 118) switched to another treatment. <b>Conclusion:</b> This study contributed to the growing evidence that patients with acromegaly are not well-served by current therapeutic options, as indicated by high rates of treatment discontinuation, switching and add-on therapy. However, treatment switching and add-on therapy represent ongoing efforts to optimize patient care toward more effective and tolerable treatments. Expanded treatment options may serve an unmet need in this patient population.

本材料为发表于《比较效果研究杂志》（Journal of Comparative Effectiveness Research）的《肢端肥大症真实世界患者队列的治疗依从性、持续时长与医疗成本》一文的同行评议补充材料。
表A：垂体切除术(hypophysectomy)与立体定向放射外科(stereotactic radiosurgery)的当前操作术语(CPT)及国际疾病分类(ICD)编码列表
表B：目标合并症的国际疾病分类第9版(ICD-9)与第10版(ICD-10)编码列表
研究目的：本研究旨在描述首次接受肢端肥大症(acromegaly)药物治疗的患者的治疗模式。
材料与方法：本研究采用2013年1月1日至2023年6月30日期间的艾昆纬(IQVIA) Pharmetrics Plus R数据库数据，筛选出首次启动肢端肥大症药物治疗的患者并观察其治疗模式。所有纳入患者需在索引日期前具备至少12个月的无肢端肥大症治疗（药物或手术）数据，且随访时长至少为6个月。合并症于基线期进行评估，治疗依从性、持续性、用药情况及换药情况则于随访期进行统计。
研究结果：最终共纳入453例首次接受肢端肥大症药物治疗、且此前至少12个月未接受任何肢端肥大症治疗的患者。其中，46.1%（n=206）以卡麦角林作为初始治疗方案，24.5%（n=111）采用注射用奥曲肽，15.0%（n=68）采用兰瑞肽，5.5%（n=25）采用溴隐亭，4.9%（n=22）采用培维索孟，2.2%（n=10）采用帕瑞肽，1.1%（n=5）采用口服奥曲肽，0.4%（n=2）采用卡麦角林联合奥曲肽，0.2%（n=1）采用卡麦角林联合兰瑞肽。随访期末，54.3%（n=246）的患者未接受任何治疗，19.6%（n=89）仍维持初始治疗方案，剩余26.0%（n=118）更换了治疗方案。
研究结论：本研究进一步丰富了现有证据体系：当前治疗方案无法很好地满足肢端肥大症患者的诊疗需求，这一点从较高的治疗中断、换药及联合治疗比例中可见一斑。不过，换药与联合治疗体现了临床为优化患者护理、寻求更有效且耐受更佳的治疗方案所做出的持续努力。拓展治疗选择或可满足该患者群体尚未被满足的医疗需求。