do Nascimento Ana Clara Kneese Virgilio, Annichino-Bizzacchi Joyce Maria, Maximo Claudia de Alvarenga, Minowa Eimy, Julian Guilherme Silva, Dos Santos Rafael Freitas
a Departamento de Clínica Médica , Santa Casa de Misericórdia de São Paulo , São Paulo , SP , Brazil.
b Centro de Hematologia de São Paulo , São Paulo , SP , Brazil.
J Med Econ. 2017 Aug;20(8):884-892. doi: 10.1080/13696998.2017.1341415. Epub 2017 Jul 4.
Although several therapeutic options are available for chronic immune thrombocytopenic purpura (cITP), little is known about the treatment of cITP in Brazil.
A multi-center, retrospective chart review, observational study was designed to describe the treatment patterns, clinical burden, resources use, and associated costs for adult patients diagnosed with cITP and treated in public and private institutions in Brazil. Patient charts were screened in reverse chronological order based on their last visit post January 1, 2012. (All costs were calculated using 1.00 USD = 3.9571 BRL, from February 2016.) Results: Of 340 patient charts screened, 50 patients were eligible for inclusion in the study. Single-drug therapy (prednisone, dexamethasone, or dapsone) was the most commonly used treatment, followed by combination therapies (azathioprine + prednisone, azathioprine + prednisone + danazol, and prednisone + dapsone). Splenectomy was performed in 22% of patients after at least first-line treatment. Platelet count and number of bleeding episodes at diagnosis were 31,561.1/mm (SD = ±26,396.1) and 40 episodes, respectively; in first-line, 92,631.1/mm (SD = ±79,955.3) and 19 episodes, respectively; in second-line, 96,950.0/mm (SD = ±76,476.4) and 17 episodes, respectively. Private system patients had a higher median cost compared to public system patients (USD 17.49/month, range = 0-2,020.77 vs USD 9.51/month, range = 0-192.64, respectively).
This study does not allow conclusions for causal explanations due to the cohort study design, and treatment patterns represent only the practices of physicians who have agreed to participate in the study.
The data indicate that available therapeutic strategies for second- and third-line therapies appear to be limited.
尽管慢性免疫性血小板减少性紫癜（cITP）有多种治疗选择，但巴西对cITP的治疗了解甚少。
设计了一项多中心、回顾性病历审查观察性研究，以描述巴西公立和私立机构中诊断为cITP并接受治疗的成年患者的治疗模式、临床负担、资源使用及相关成本。根据患者2012年1月1日后的最后一次就诊情况，按时间倒序筛选病历。（所有成本均使用2016年2月1.00美元 = 3.9571巴西雷亚尔的汇率计算。）结果：在筛选的340份患者病历中，50例符合纳入研究标准。单药治疗（泼尼松、地塞米松或氨苯砜）是最常用的治疗方法，其次是联合治疗（硫唑嘌呤 + 泼尼松、硫唑嘌呤 + 泼尼松 + 达那唑、泼尼松 + 氨苯砜）。至少经过一线治疗后，22%的患者接受了脾切除术。诊断时血小板计数和出血发作次数分别为31,561.1/mm（标准差 = ±26,396.1）和40次发作；一线治疗时分别为92,631.1/mm（标准差 = ±79,955.3）和19次发作；二线治疗时分别为96,950.0/mm（标准差 = ±76,476.4）和17次发作。与公立系统患者相比，私立系统患者的中位成本更高（分别为17.49美元/月，范围 = 0 - 2,020.77美元 vs 9.51美元/月，范围 = 0 - 192.64美元）。
由于队列研究设计，本研究无法得出因果解释结论，且治疗模式仅代表同意参与研究的医生的做法。
数据表明，二线和三线治疗的可用治疗策略似乎有限。
