DAILY VS WEEKLY PREDNISONE THERAPY IN DUCHENNE MUSCULAR DYSTROPHY

杜氏肌营养不良症的每日与每周泼尼松治疗

• 批准号: 7374997
• 负责人: Timothy Edward Lotze
• 金额: $ 0.34万
• 依托单位: BAYLOR COLLEGE OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2005
• 资助国家: 美国
• 起止时间: 2005-12-01 至 2006-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7374997
• 关键词: DAILY; VS; WEEKLY; PREDNISONE; THERAPY

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Duchenne muscular dystrophy (DMD) is among the most common of genetic disorders worldwide. Yet, to date, the only clinical treatments that have been shown to improve the health and mobility of DMD patients are the corticosteroids prednisone and deflazacort. Both agents shoe immune modulatory function, and both decreased the invasion of T-cells in DMD muscle. However there is growing evidence that additional pathophysiological pathways are responsible, in part, for the efficacy of corticosteroids. Including improvement of calcium homeostasis, inhibition of muscle protein degradation, and improvement of energy metabolism. While many physicians consider chronic steroid administration as "standard of care" in DMD, only about half of physicians and patient families in the USA opt to use steroids due to the side effects associated with its chronic use. Many of those that use steroid, delay therapy until the disease is advanced because of concern that risks outweigh the benefits of therapy. Some alternate regimes use for other autoimmune diseases have been found to be ineffective in DMD; however, large dose steroid given intermittently, which have been found to be effective and have fewer side effects in other autoimmune disorders, have not been evaluated in DMD in adequately powered controlled trials. This study will evaluate the high dose prednisone adminsitered on an intermittent schedule in pediatric patients with DMD. Finding an alternate dose regimen that would be at least as effective as daily therapy, but has fever side effects is the goal of this proposal. If successful, this regimen would likely become a standard of care in DMD and permit onset of therapy earlier, which might be even more beneficial.

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得主要资金，因此可以在其他CRISP条目中表示。所列机构为中心，不一定是研究者所在机构。杜氏肌营养不良症（DMD）是世界上最常见的遗传性疾病之一。然而，到目前为止，已被证明可以改善DMD患者健康和活动能力的唯一临床治疗方法是皮质类固醇泼尼松和地夫可特。两种药物均具有免疫调节功能，并且均降低DMD肌肉中T细胞的侵袭。然而，越来越多的证据表明，额外的病理生理途径是负责，部分，对皮质类固醇的疗效。包括改善钙稳态、抑制肌肉蛋白质降解、改善能量代谢。虽然许多医生认为慢性类固醇给药是DMD的“护理标准”，但由于长期使用类固醇的副作用，美国只有大约一半的医生和患者家庭选择使用类固醇。许多使用类固醇的人推迟治疗，直到疾病进展，因为担心风险超过治疗的好处。已发现用于其他自身免疫性疾病的一些替代方案对DMD无效;然而，间歇性给予大剂量类固醇已被发现在其他自身免疫性疾病中有效且副作用较少，但尚未在充分把握度的对照试验中对DMD进行评估。本研究将评估间歇性给予高剂量泼尼松治疗儿童DMD患者的效果。找到一种替代剂量方案，至少与日常治疗一样有效，但有发热副作用，这是该提案的目标。如果成功的话，这种方案可能会成为DMD的标准治疗，并允许更早开始治疗，这可能会更有益。