Brommann Simone Juline, Pilgaard Linea, Jensen Jonas Faartoft, Olesen Anne Estrup, Kristensen Daniel Tuyet, Severinsen Marianne Tang
Department of Haematology Clinical Cancer Research Center Aalborg University Hospital Aalborg Denmark.
Department of Clinical Pharmacology Aalborg University Hospital Aalborg Denmark.
EJHaem. 2025 Jun 2;6(3):e70066. doi: 10.1002/jha2.70066. eCollection 2025 Jun.
The hypomethylating agent azacitidine (AZA) is the most widely used first-line treatment for patients with higher-risk myelodysplastic syndromes (MDS) due to its survival benefits. Effective management of higher-risk MDS requires addressing not only clinical outcomes but also the burden of healthcare resource utilisation (hospitalisations and transfusions), which remains poorly characterised.
We conducted a retrospective, population-based study of patients diagnosed with higher-risk MDS treated with AZA between 2010 and 2022. We collected clinical data, including transfusions and hospitalisations, by detailed review of medical records from the initiation of AZA and up to 12 cycles.
In total, 103 patients were included, and 94% required supportive care during treatment. Healthcare resource utilisation peaked during the first four cycles. Hospitalisation was necessary for 75% of the patients, and 28% were hospitalised ≥ 3 times. Red blood cell (RBC) transfusions were required for 78% of the patients in the first cycle, and 30% achieved RBC transfusion independence.
This study highlights the substantial healthcare resource utilisation for patients with higher-risk MDS receiving AZA, which emphasises the importance of considering these factors in treatment planning.
The authors have confirmed clinical trial registration is not needed for this submission.
由于其生存获益，低甲基化药物阿扎胞苷（AZA）是高危骨髓增生异常综合征（MDS）患者最广泛使用的一线治疗药物。有效管理高危MDS不仅需要关注临床结局，还需要关注医疗资源利用负担（住院和输血），而这方面的特征仍不明确。
我们对2010年至2022年期间接受AZA治疗的高危MDS患者进行了一项基于人群的回顾性研究。我们通过详细审查从开始使用AZA直至12个周期的病历，收集了包括输血和住院情况在内的临床数据。
总共纳入了103例患者，94%的患者在治疗期间需要支持性治疗。医疗资源利用在前四个周期达到峰值。75%的患者需要住院治疗，28%的患者住院≥3次。第一个周期中78%的患者需要输注红细胞（RBC），30%的患者实现了RBC输血独立。
本研究强调了接受AZA治疗的高危MDS患者大量的医疗资源利用情况，这凸显了在治疗规划中考虑这些因素的重要性。
作者已确认本研究无需临床试验注册。
