Zeidan Amer M, Perepezko Kate, Salimi Tehseen, Washington Terri, Epstein Robert S
Yale University and Yale Cancer Center, 333 Cedar Street, PO Box 208028, New Haven, CT 06520-8028, USA.
CorEvitas, LLC, Waltham, MA, USA.
Ther Adv Hematol. 2024 Jul 30;15:20406207241257313. doi: 10.1177/20406207241257313. eCollection 2024.
Hypomethylating agents (HMAs) are guideline-recommended treatment for higher-risk myelodysplastic syndromes/neoplasms (MDS). However, survey of patients with MDS reported challenges with intravenous (IV) and subcutaneous (SC) HMA therapies, including pain related to treatment administration and interference with daily activities; most patients also indicated a preference to switch to an oral therapy if one were available.
This study evaluated the perspectives of US patients with MDS receiving oral decitabine/cedazuridine (DEC-C), an alternative to IV/SC HMAs.
An online survey was conducted among adult patients with MDS in the United States (10 November 2022 to 5 December 2022) who had filled a prescription for oral DEC-C between 2021 and 2022.
A total of 150 patients completed the survey; 61% were aged ⩾60 years and 63% were male. Of these, 123 (82%) were still receiving oral DEC-C, and 27 (18%) had stopped oral DEC-C treatment. Half (50%) of patients had received oral DEC-C for ⩾6 months. The majority reported that treatment was convenient (83%) and that they were satisfied with treatment (86%). Most patients also reported very little/no interference with regular daily activities (82%), social activities (78%), and productivity (78%). When queried about negative impacts on quality of life (QOL), treatment side effects were the most commonly reported (30% of respondents). Among patients who had previously received IV/SC HMAs ( = 91), most agreed that oral DEC-C interfered less with daily life (91%) and had experienced improvement in QOL (85%) compared with previous treatment; 91% reported that oral DEC-C reduced the number of times they needed to travel to a healthcare facility.
Survey results suggest very little/no impact on regular daily activities and improved QOL with oral DEC-C relative to IV/SC HMAs, highlighting the potential for oral DEC-C to reduce the treatment burden associated with parenteral HMA therapy.
低甲基化药物（HMAs）是指南推荐用于治疗高危骨髓增生异常综合征/肿瘤（MDS）的药物。然而，对MDS患者的调查显示，静脉注射（IV）和皮下注射（SC）HMA治疗存在挑战，包括与治疗给药相关的疼痛以及对日常活动的干扰；大多数患者还表示，如果有口服疗法，他们更倾向于改用口服疗法。
本研究评估了接受口服地西他滨/西扎氯铵（DEC-C）（IV/SC HMAs的替代药物）的美国MDS患者的观点。
在美国成年MDS患者中进行了一项在线调查（2022年11月10日至2022年12月5日），这些患者在2021年至2022年期间开具了口服DEC-C的处方。
共有150名患者完成了调查；61%的患者年龄≥60岁，63%为男性。其中，123名（82%）仍在接受口服DEC-C治疗，27名（18%）已停止口服DEC-C治疗。一半（50%）的患者接受口服DEC-C治疗≥6个月。大多数患者报告治疗方便（83%），并且对治疗满意（86%）。大多数患者还报告对日常常规活动（82%）、社交活动（78%）和工作效率（78%）的干扰非常小/没有干扰。当被问及对生活质量（QOL）的负面影响时，治疗副作用是最常被报告的（30%的受访者）。在之前接受过IV/SC HMAs治疗的患者（n = 91）中，大多数人同意口服DEC-C对日常生活的干扰较小（91%），并且与之前的治疗相比生活质量有所改善（85%）；91%的患者报告口服DEC-C减少了他们前往医疗机构的次数。
调查结果表明，与IV/SC HMAs相比，口服DEC-C对日常常规活动的影响非常小/没有影响，并且改善了生活质量，突出了口服DEC-C减轻与胃肠外HMA治疗相关的治疗负担的潜力。
