Hemofilia: validação da versão brasileira do VERITAS-Pro para avaliação da adesão à profilaxia e custo-análise do tratamento

Abstract

Prophylaxis is the therapeutic regimen of choice for individuals with severe hemophilia. However, its effectiveness may be limited by the patient's low adherence, compromising the results, despite the high costs of treatment. The objectives of this study were: 1) to validate the Brazilian version of the VERITAS-Pro questionnaire for assessing adherence to prophylaxis and 2) to assess the direct medical costs of hemophilia treatment from the perspective of the Unified Health System, underscoring the increase in public spending after the incorporation of therapeutic modalities such as prophylaxis, immunotolerance, and the use of recombinant products. The study was conducted at the Juiz de Fora Regional Blood Center between 2015 and 2016. For validation, the US version of the VERITAS-Pro was translated and adapted to Brazilian Portuguese and administered to 32 persons responsible for the prophylaxis of hemophilia patients, and to 5 observers of the treatment conducted. Measures for validation included results obtained from visual analogue scales of adherence, medications dispensed by the blood center pharmacy, and percentage of recommended doses administered, obtained from patient infusion diaries. The internal consistency was very good for VERITAS-Pro's overall score, excellent for the subscales "remember", "skip", and "communicate", good for the subscale "dose" and acceptable for "time" and "plan". Twelve participants answered the questionnaire on more than one occasion, to evaluate reproducibility. The intraclass correlation coefficient was excellent. Regarding convergent validity, the VERITAS-Pro scores were moderately correlated with the visual scale and with the infusion diaries, but showed poor correlation with pharmacy dispensing records. Thus, the Brazilian version of the VERITAS-Pro proved to be a valid and reliable instrument that can facilitate understanding of specific factors related to adherence and permit more targeted interventions. For the cost analysis study, sociodemographic, clinical, and therapeutic data were collected retrospectively from the medical records of 98 hemophilia patients treated between January 2011 and December 2015. Consultations, examinations, hospitalizations, and medications were assigned costs according to the Management System for the Table of Procedures, Medications, and Orthoses, Prostheses, and Materials, and the Medications Price List of the Brazilian Health Regulatory Agency. From the total number of patients, 43.3% had severe hemophilia and 32.7% had moderate hemophilia; 36 adhered to the prophylaxis, with 29 in the tertiary form; two presented positive inhibitor screening; and one received immunotolerance. The number of consultations and the consumption of blood products were higher in the group with severe hemophilia. Hospitalizations were rare. Arthropathy was not staged and there were no orthopedic surgeries. Direct costs increased 286.8% over the period evaluated. The mean annual cost per patient was 57,416.43 Brazilian Reais (2,962.10 BR in mild hemophilia - 89,467.77 BR in severe cases), with no significant difference between hemophilia A and B. Blood products corresponded to 99.46% of the total cost. The implementation of prophylaxis has considerably increased the direct costs of treating hemophilia. The inclusion of patients with unmeasured arthropathy in prophylactic regimens makes it impossible to assess the cost-effectiveness of this approach, making it difficult to define priorities given the scarcity of financial resources.