Department of Orthopaedics, Children's University Hospital of Cracow, ul. Wielicka 265, 30-663, Cracow, Poland.
Rheumatol Int. 2022 Apr;42(4):699-706. doi: 10.1007/s00296-021-04886-4. Epub 2021 May 20.
To evaluate patient-reported effectiveness, safety and social influence of Pamidronate in the therapy of NSAIDs-refractory Chronic Recurrent Multifocal Osteomyelitis in children. Authors reviewed self-created questionnaires, which asked patients for symptoms alleviation, adverse drug reactions frequency and degree of severity and daily activities self-reliance. Only surveys with complete answers, which were returned to authors by an e-mail from juvenile patients treated for NSAIDs-refractory Chronic Recurrent Multifocal Osteomyelitis at the University Children's Hospital of Cracow were analyzed. Between 2010 and 2019, 61 children were diagnosed with NSAIDs-refractory Chronic Recurrent Multifocal Osteomyelitis at our department. Out of 61 requests sent, 42 complete replies (33 females, 9 males) were gathered and analyzed. All patients included in this research were administered with at least one set of Pamidronate intravenously in the dose of 1 mg/kg/day for 3 consecutive days. Our analysis shows remarkable in terms of patient's impressions decrease of pain intensity after 2.5 series of Pamidronate on average, and total pain resolution after 5.9 series on average. Overall number of adverse drug reaction events reported by responders was 105. One patient developed drug-dependent renal insufficiency in the course of therapy. Outcome assessment indicates that nearly 50% of the studied population was more eager to participate in social life just after the first infusion of the drug. 95% of the surveyed unanimously agreed to recommend Pamidronate therapy to cure NSAIDs-refractory CRMO. 39 out of 42 (93%) patients considered Pamidronate effective at the end of the treatment. Onset of Pamidronate's action is gradual and differs in terms of symptoms alleviation between sexes. The therapy can induce considerable number of adverse drug reactions (2.5 per patient). Only 3 out of 42 (7%) patients were free from any ADRs. To demonstrate the impact of the use of Pamidronate on daily activities more precisely, further research with quantification of the quality of life is warranted.
评估帕米膦酸二钠治疗儿童非甾体类抗炎药难治性慢性复发性多灶性骨炎的患者报告疗效、安全性和社会影响。作者回顾了自行创建的问卷，这些问卷询问了患者症状缓解、药物不良反应频率和严重程度以及日常活动自理能力。仅分析了 2010 年至 2019 年在克拉科夫儿童大学医院接受非甾体类抗炎药难治性慢性复发性多灶性骨炎治疗的青少年患者通过电子邮件寄回给作者的完整问卷。在我们科室诊断出 61 例非甾体类抗炎药难治性慢性复发性多灶性骨炎患儿。在发出的 61 份请求中，共收集并分析了 42 份完整回复（33 名女性，9 名男性）。本研究中所有患者均接受至少一组帕米膦酸二钠治疗，剂量为 1mg/kg/天，连续 3 天。我们的分析表明，在接受 2.5 个疗程帕米膦酸二钠治疗后，平均疼痛强度显著下降，在接受 5.9 个疗程治疗后，疼痛完全缓解。报告不良反应事件的患者总数为 105 例。1 例患者在治疗过程中出现药物依赖肾功能不全。结果评估表明，近 50%的研究人群在接受药物首次输注后更渴望参与社会生活。95%的被调查者一致同意推荐帕米膦酸二钠治疗以治愈非甾体类抗炎药难治性 CRMO。42 名患者中的 39 名（93%）在治疗结束时认为帕米膦酸二钠有效。帕米膦酸二钠的作用是逐渐发生的，在性别之间缓解症状方面存在差异。该疗法会引起相当数量的药物不良反应（每名患者 2.5 次）。只有 42 名患者中的 3 名（7%）没有任何不良反应。为了更准确地展示帕米膦酸二钠使用对日常生活的影响，需要进一步进行生活质量量化的研究。
