Pickard A Simon, Huynh Lynn, Ivanova Jasmina I, Totev Todor, Graham Sophia, Mühlbacher Axel C, Roy Anuja, Duh Mei Sheng
Second City Outcomes Research, Chicago, IL USA.
2Analysis Group, Inc., 111 Huntington Avenue, 14th Floor, Boston, MA 02199 USA.
J Patient Rep Outcomes. 2017;2(1):13. doi: 10.1186/s41687-018-0032-y. Epub 2018 Mar 1.
Aplastic anemia is a rare, serious blood disorder due to bone marrow failure to produce blood cells. Transfusions are used to reduce risk of bleeding, infection and relieve anemia symptoms. In severe patients, transfusions may be required more than once/week. It is unclear from the patient perspective the impact that transfusions have on quality of life. This study aimed to elicit patient preferences for attributes associated with severe aplastic anemia (SAA) treatment, including transfusion independence.
An online discrete choice experiment (DCE) was conducted among patients with SAA who experienced insufficient response to immunosuppressive therapy and transfusion dependence for ≥3 months in the past 2 years. Recruitment occurred through the Aplastic Anemia and Myelodysplastic Syndromes International Foundation and referrals from clinical sites in the US and France. Respondents chose between hypothetical treatment pairs characterized by a common set of attributes: transfusions frequency, fatigue, risk of infection, and risk of serious bleeding. Conditional logit model with effects coding was used to estimate part-worth utilities for different attribute levels and the relative importance of each attribute. Predicted utility scores for transfusion frequency levels were reported.
Thirty patients completed the survey. Most were age ≥ 40 years (73.3%), female (70.0%), and from the US (86.7%). 33.3% underwent bone marrow transplant; 36.7% received iron chelation therapy. Patients largely agreed that transfusion independence would result in less burden on time and costs, greater control and quality of life, less fatigue (86.7% noted each) and less scheduling around medical appointments (83.3%). The DCE found highest relative importance for risk of bleeding (0.30), followed by risk of infection (0.28), fatigue (0.23), and frequency of transfusions (0.20). More frequent transfusions resulted in lower utility, particularly when increasing monthly transfusions frequency from 4 (0.57) to 8 (0.35).
Our study showed that higher utility was associated with fewer transfusions in SAA patients with insufficient response to immunosuppressive therapy. While risk of bleeding, risk of infection, and fatigue were more important for patient treatment preferences, frequency of transfusions was also important.
再生障碍性贫血是一种罕见的严重血液疾病，由于骨髓无法产生血细胞所致。输血用于降低出血、感染风险并缓解贫血症状。在重症患者中，可能每周需要输血不止一次。从患者角度来看，输血对生活质量的影响尚不清楚。本研究旨在了解患者对与重型再生障碍性贫血（SAA）治疗相关属性的偏好，包括不依赖输血。
对过去两年中对免疫抑制治疗反应不足且输血依赖≥3个月的SAA患者进行了一项在线离散选择实验（DCE）。通过再生障碍性贫血和骨髓增生异常综合征国际基金会以及美国和法国临床机构的转诊进行招募。受访者在以一组共同属性为特征的假设治疗方案对之间进行选择：输血频率、疲劳、感染风险和严重出血风险。使用带有效应编码的条件logit模型来估计不同属性水平的部分价值效用以及每个属性的相对重要性。报告了输血频率水平的预测效用分数。
30名患者完成了调查。大多数患者年龄≥40岁（73.3%），女性（70.0%），来自美国（86.7%）。33.3%的患者接受了骨髓移植；36.7%的患者接受了铁螯合治疗。患者普遍认为不依赖输血将减少时间和成本负担，增强控制感和生活质量，减轻疲劳（86.7%的患者提到了每一项）并减少围绕医疗预约的安排（83.3%）。DCE发现出血风险的相对重要性最高（0.30），其次是感染风险（0.28）、疲劳（0.23）和输血频率（0.20）。输血频率越高，效用越低，特别是当每月输血频率从4次增加到8次时（从0.57降至0.35）。
我们的研究表明，对于免疫抑制治疗反应不足的SAA患者，输血次数越少，效用越高。虽然出血风险、感染风险和疲劳对患者的治疗偏好更为重要，但输血频率也很重要。
