Arboretti R, Tognoni G, Alberti D
Dipartimento di Farmacologia Clinica ed Epidemiologia, Consorzio Mario Negri Sud, Via Nazionale, I-66030S Maria Imbaro, CH, Italy.
Eur J Clin Pharmacol. 2001 Mar;56(12):915-22. doi: 10.1007/s002280000251.
For adolescent thalassaemic patients, parenteral iron-chelation therapy is still a burden and a major reason for unsatisfactory compliance. The introduction of a new pharmaceutical preparation of deferoxamine (Desferal) claimed to be better tolerated was accompanied by an epidemiological study on the quality of care and life of the Italian thalassaemic population and on the acceptability and safety profile of the new preparation compared with the old one.
This was a two-period prospective survey of 867 patients (12% of the registered thalassaemic patient population) in a sample of centres representative of the different contexts of care.
The majority of patients (81%) reported an overall positive opinion about the quality of care (median satisfaction score 7.4). Judgement was more critical among patients over 15 years, unemployed, less compliant with chelation treatment and with higher mean serum ferritin levels. The co-morbidity profile did not appear to influence patients' satisfaction scores, but 21% of respondents associated great discomfort with the use of Desferal, which appeared in a multivariate analysis to be a strong predictor [odds ratio (OR) 2.15, 95% confidence interval (CI) 1.20-3.83] of negative perception. Quality of life was reported as fair or poor (score below 6) by 21% of patients. Over a total of 29,066 infusions included in the comparison of the safety of the new versus the old preparation, the reported incidences of any adverse event (AE) were 51% versus 57%, respectively, and 8.2% versus 9.3% for severe AEs. Comparing the new with the old preparation, the adjusted relative risk of severe AEs was 0.79 (95% CI 0.65-0.96).
While oral chelation therapies are still under evaluation, the overall quality of care and perceived quality of life of a representative sample of the Italian population of thalassaemic patients show that room for improvement depends more on the contexts of life and care than on strictly medical conditions. Comprehensive epidemiological surveillance is needed as a routine component of the care of this highly morbidity-burdened population to ensure timely appreciation of unmet needs and to assess the yield of innovative treatment schedules.
对于青少年地中海贫血患者而言，肠胃外铁螯合疗法仍然是一项负担，也是导致依从性不尽人意的主要原因。一种声称耐受性更好的去铁胺新制剂（得斯芬）问世后，针对意大利地中海贫血人群的护理质量与生活质量，以及该新制剂与旧制剂相比的可接受性和安全性开展了一项流行病学研究。
这是一项为期两阶段的前瞻性调查，对来自不同护理背景的代表性中心样本中的867名患者（占登记地中海贫血患者总数的12%）进行了调查。
大多数患者（81%）对护理质量给出了总体积极评价（满意度中位数为7.4）。15岁以上、失业、铁螯合治疗依从性较差且平均血清铁蛋白水平较高的患者给出的评价更为严苛。共病情况似乎并未影响患者的满意度评分，但21%的受访者表示使用得斯芬会带来极大不适，在多变量分析中，这似乎是负面看法的一个有力预测因素[比值比（OR）为2.15，95%置信区间（CI）为1.20 - 3.83]。21%的患者报告生活质量为中等或较差（评分低于6分）。在对新制剂与旧制剂安全性进行比较的总计29,066次输液中，报告的任何不良事件（AE）发生率分别为51%和57%，严重不良事件发生率分别为8.2%和9.3%。将新制剂与旧制剂进行比较，严重不良事件的调整相对风险为0.79（95% CI为0.65 - 0.96）。
虽然口服螯合疗法仍在评估中，但意大利地中海贫血患者代表性样本的总体护理质量和感知生活质量表明，改善空间更多地取决于生活和护理背景，而非严格意义上的医疗状况。需要进行全面的流行病学监测，作为对这一高发病负担人群护理的常规组成部分，以确保及时了解未满足的需求，并评估创新治疗方案的效果。
