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Gaucher disease (GD) is a genetic autosomic disorder for which treatment has been funded by the Brazilian government since the 1990s. In our state most patients are treated with enzyme replacement therapy (ERT) and followed by our Reference Center under the recommendation of the Ministry of Health Brazilian guidelines. There is a lack in the literature about adherence of patients to treatment. The objective was to describe adherence to the treatment in a cohort of all GD patients in the southern state of Brazil.
This was a cohort study of all GD patients treated with velaglucerase α, taliglucerase α and imiglucerase from January 2010 to January 2015. Adherence was measured as recommended by the Brazilian guidelines as to perform more than 50 percent of the anticipated infusions per year.
Our study included thirty-seven patients of both genders. Doses of ERT varied from 15 to 45IU/kg for type 1 patients and from 30 to 60 IU/kg for type 3 patients. A mean of 83 percent of anticipated infusions were performed and from all patients only one did not adhere to the treatment during the 5 years of our study. The majority of the patients performed at least 50 percent of all anticipated infusions.
We noted a very high rate of adherence to treatment with a very few adverse effects. Our data might be showing that the very high rate of adherence in these chronic disease patients may be attributed to the value of treatment by patients and their family, and also due to the existence of a multidisciplinary team at the reference center. These data might be useful for public health policy making in other countries.
Within the Brazilian Health System, Referral Centers (RCs) are care facilities that provide specialized services. The objective of this study was to evaluate the efficacy of care provided to patients with multiple myeloma (MM) at a specialized Referral Centers (Hospital de Clínicas de Porto Alegre Referral Center for Multiple Myeloma, CRMM-HCPA) and to compare quality of life between patients with MM treated at CRMM-HCPA and those treated at non-RC facilities.
A 6-month cohort study was conducted in patients with MM receiving thalidomide from the State Health Department and treated at CRMM-HCPA, and patients receiving treatment at other non-RC facilities. Thirty-two patients were included in the study, nineteen from CRMM-HCPA and thirteen from other institutions. To analyze the efficacy of care provided at CRMM-HCPA, the main outcome measure was the time from diagnosis to referral for autologous hematopoietic stem cell transplantation.This outcome measure was assessed using questionnaires specifically designed for this study. Quality of life was also assessed, using the Short-Form 36 Item Health Survey (SF-36) questionnaire.
Time from MM diagnosis to referral for autologous hematopoietic stem cell transplantation in each group was measured only in patients aged 65 years (n = 25); of these, 15 were recruited from CRMM-HCPA and 10 from other institutions. In this analysis, there was a significant difference (p = .036) in time elapsed between diagnosis and referral for autologous hematopoietic stem cell transplantation, which was significantly shorter for patients treated at CRMM-HCPA (median, 9 months; Interquartile Range, IQR, 8.5–14.5) than for those treated elsewhere (median, 24 months; IQR, 16–24). On quality of life analysis, there was a significant difference in the Social Functioning, which relates to performance of social activities (p = .02).
The Referral Centers model provided seems to be a more efficient treatment strategy as compared with other health care facilities, as it enabled a reduction in time to transplantation. Patients treated at CRMM-HCPA demonstrated greater ease in performing social activities, with less interference from physical or emotional problems.
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