New England Research Institutes, 9 Galen Street, Watertown, MA, 02472, USA.
Health Qual Life Outcomes. 2012 Dec 7;10:148. doi: 10.1186/1477-7525-10-148.
Understanding patients' views about medication is crucial to maximize adherence. Thalassemia is a congenital blood disorder requiring chronic blood transfusions and daily iron chelation therapy.
The Beliefs in Medicine Questionnaire (BMQ) was used to assess beliefs in chelation in thalassemia patients from North America and London in the Thalassemia Longitudinal Cohort (TLC) of the Thalassemia Clinical Research Network (TCRN). Chelation adherence was based on patient report of doses administered out of those prescribed in the last four weeks.
Of 371 patients (ages 5-58y, mean 24y), 93% were transfused and 92% receiving chelation (26% deferoxamine (DFO; a slow subcutaneous infusion via portable pump), 63% oral, 11% combination). Patients expressed high "necessity" for transfusion (96%), DFO chelation (92%) and oral chelation (89%), with lower "concern" about treatment (48%, 39%, 19% respectively). Concern about oral chelation was significantly lower than that of DFO (p<0.001). Self-reported adherence to chelation was not associated with views about necessity or concerns, but negatively correlated with perceived sensitivity to DFO (Sensitive Soma scale; r=-0.23, p=0.01) and side effects of oral chelation (r=-0.14, p=0.04). High ferritin iron levels, potentially indicating lower adherence, were found in 41% of patients reporting low necessity of oral chelation compared to 24% reporting high necessity (p=0.048). Concerns about treatment were associated with lower quality of life and more symptoms of anxiety and depression.
Despite their requirement for multimodal therapy, thalassemia patients have positive views about medicine, more so than in other disease populations. Patients may benefit from education about the tolerability of chelation and strategies to effectively cope with side effects, both of which might be beneficial in lowering body iron burden. CLINICALTRIALS.GOV IDENTIFIER: NCT00661804.
了解患者对药物的看法对于最大限度地提高依从性至关重要。地中海贫血是一种需要长期输血和每日铁螯合治疗的先天性血液疾病。
使用信念量表（BMQ）评估北美和伦敦地中海贫血纵向队列（TLC）中的地中海贫血患者对螯合治疗的信念，该队列是地中海贫血临床研究网络（TCRN）的一部分。螯合治疗的依从性是根据患者在过去四周内服用的处方剂量与实际服用剂量之比来评估的。
在 371 名患者（年龄 5-58 岁，平均 24 岁）中，93%接受输血，92%接受螯合治疗（26%使用可携带皮下输注泵的地拉罗司，63%使用口服药物，11%联合治疗）。患者对输血（96%）、地拉罗司螯合治疗（92%）和口服螯合治疗（89%）的“必要性”表达较高，而对治疗的“担忧”程度较低（分别为 48%、39%和 19%）。与地拉罗司相比，患者对口服螯合治疗的担忧显著较低（p<0.001）。自我报告的螯合治疗依从性与对必要性或担忧的看法无关，但与对地拉罗司的敏感性感知（敏感躯体量表；r=-0.23，p=0.01）和口服螯合治疗的副作用（r=-0.14，p=0.04）呈负相关。在报告对口服螯合治疗的必要性较低的患者中，有 41%的患者铁蛋白水平较高，这可能表明其依从性较低，而在报告对口服螯合治疗的必要性较高的患者中，这一比例为 24%（p=0.048）。对治疗的担忧与较低的生活质量以及更多的焦虑和抑郁症状相关。
尽管地中海贫血患者需要接受多种治疗，但他们对药物的看法比其他疾病患者更为积极。患者可能受益于关于螯合治疗耐受性和有效应对副作用策略的教育，这两者都可能有助于降低体内铁负荷。临床试验注册编号：NCT00661804。
