Effects of zoledronic acid therapy in fibrous dysplasia of bone: a single-center experience.

Objective: Fibrous dysplasia (FD) is a rare bone disorder that can involve any part of the skeleton, leading to bone pain, deformities, and fractures. Treatment with intravenous bisphosphonates has been used with variable results. Therefore, we aimed to evaluate the effects of zoledronic acid (ZA) therapy in patients with monostotic or polyostotic FD. Methods: The medical records of thirteen patients with FD evaluated between 2015 and 2020 were retrospectively analyzed. In the subgroup of patients treated with ZA (n = 7), data on pain relief, changes in bone turnover markers (BTMs), and adverse events following ZA infusions were retrieved. Moreover, radiological changes in response to treatment were recorded in patients who underwent radiological follow-up. Results: Of the patients, 5 (38%) presented with monostotic whereas 8 (62%) had polyostotic FD. Bone pain was a common finding (69%), and most patients (62%) exhibited elevated baseline BTMs. Partial or complete pain relief was reported in 6 of 7 patients treated with ZA. BTMs, especially C-telopeptide of type I collagen (CTX), significantly decreased after therapy (change rate: -61.8% [IQR -71, -60%]), and median CTX levels were significantly lower than at baseline (0.296 ng/mL [0.216, 0.298] vs. 0.742 ng/mL [0.549, 0.907], respectively; P = 0.04). No radiological improvement was observed in cases with radiological follow-up (n = 3). No serious adverse effects of ZA were reported. Conclusion: ZA treatment was well tolerated and provided beneficial effects in relieving bone pain and reducing BTMs, especially CTX. Our data reinforce the role of ZA in the treatment of FD-related bone pain.

Effects of zoledronic acid therapy in fibrous dysplasia of bone: a single-center experience

ABSTRACT Objective: Fibrous dysplasia (FD) is a rare bone disorder that can involve any part of the skeleton, leading to bone pain, deformities, and fractures. Treatment with intravenous bisphosphonates has been used with variable results. Therefore, we aimed to evaluate the effects of zoledronic acid (ZA) therapy in patients with monostotic or polyostotic FD. Subjects and methods: The medical records of thirteen patients with FD evaluated between 2015 and 2020 were retrospectively analyzed. In the subgroup of patients treated with ZA (n = 7), data on pain relief, changes in bone turnover markers (BTMs), and adverse events following ZA infusions were retrieved. Moreover, radiological changes in response to treatment were recorded in patients who underwent radiological follow-up. Results: Of the patients, 5 (38%) presented with monostotic whereas 8 (62%) had polyostotic FD. Bone pain was a common finding (69%), and most patients (62%) exhibited elevated baseline BTMs. Partial or complete pain relief was reported in 6 of 7 patients treated with ZA. BTMs, especially C-telopeptide of type I collagen (CTX), significantly decreased after therapy (change rate: −61.8% [IQR −71, −60%]), and median CTX levels were significantly lower than at baseline (0.296 ng/mL [0.216, 0.298] vs. 0.742 ng/mL [0.549, 0.907], respectively; P = 0.04). No radiological improvement was observed in cases with radiological follow-up (n = 3). No serious adverse effects of ZA were reported. Conclusion: ZA treatment was well tolerated and provided beneficial effects in relieving bone pain and reducing BTMs, especially CTX. Our data reinforce the role of ZA in the treatment of FD-related bone pain.

Percepção médica quanto aos protocolos clínicos / Physician perception of clinical protocols

Os protocolos clínicos são condutas e procedimentos desenvolvidos com suporte em evidências atualizadas e consistentes, que objetivam promover uma melhor prática da medicina. Esses protocolos vêm auxiliar o médico em decisões sobre a melhor e mais apropriada conduta em situações clínicas específicas, permitindo resolução mais rápida e eficiente das enfermidades, gerando melhor qualidade de vida aos pacientes. Neste sentido, os guidelines buscam aumento da precisão diagnóstica, qualidade da assistência médica, dos serviços de saúde e controle de custos. Em estudo, verificou-se a percepção médica em relação ao uso de protocolos, no tocante às vantagens e limitações da prática, principais beneficiados, influência nos custos e possível diferença entre os protocolos adotados nos sistemas de saúde. Aplicou-se questionário contendo nove perguntas, respondidas individualmente e voluntariamente por 80 médicos. Constatou-se que 98,75% dos médicos tem conhecimento do uso de protocolos; 95% concordam com seu uso, sendo que 83,75% dos médicos o fazem parcialmente. Os entrevistados reconheceram as limitações de sua aplicabilidade. Dentre eles, 38,75% consideram que o julgamento clínico é mais importante, e 72,5% avaliam que os protocolos trazem benefícios, sendo que 77,5% reconhecem a diminuição nos custos. Concluiu-se que, apesar de haver limitações na aplicabilidade deste recurso, o conhecimento e uso dos protocolos por parte dos médicos já são significativos, sendo, portanto, instrumentos de auxílio em um contexto, no qual a experiência clínica deve ser integrada à informação científica, de forma crítica e racional, objetivando melhorar a qualidade da assistência médica. Desta forma, os protocolos devem ser amplamente divulgados, constantemente atualizados e adequados à realidade de cada paciente.

Clinical protocols are evidence-based sets of procedures and approaches aiming to improve medical practice and inform clinical decision-taking. With a body of information about better and more appropriate approaches to specific clinical situations, clinical protocols allow for quicker and more efficient responses to disease to be made, while providing patients with better quality of life. Guidelines seek to increase diagnostic accuracy, medical care quality, health services quality and cost control. The study investigated the following items regarding medical protocols: advantages and limitations as seen by physicians; their main beneficiaries; economic impact; and possible differences among adopted protocols. A nine-question questionnaire was answered individually and voluntarily by 80 physicians. As a result it was observed that 98.75% were aware of protocol use; 95% agreed with the use of protocols, of whom 83.75% did it partially; those interviewed recognized the limitations of protocol applicability; 38.75% considered clinical judgment to be paramount; 72.5% believed the protocols to be beneficial; 77.5% recognized cost reduction with their use. In spite of limitations to their applicability, knowledge about and use of clinical protocols by physicians are already significant. Because clinical protocols are helpful tools to improve medical care, in a context in which clinical experience must be critically and rationally integrated with scientific information, they must be widely spread, constantly updated and adapted to each patient`s reality.