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Patient-reported effectiveness and safety of Pamidronate in NSAIDs-refractory chronic recurrent multifocal
Bartłomiej Juszczak1, Jerzy Sułko2
1Department of Orthopaedics, Children's University Hospital of Cracow, ul. Wielicka 265, 30-663, Cracow, Poland. jushchuck.bartlomiej@gmail.com.
Insights
Pamidronate effectively treats pediatric Chronic Recurrent Multifocal Osteomyelitis (CRMO) refractory to NSAIDs, significantly reducing pain and improving social engagement. While generally recommended by patients, it can cause adverse drug reactions.
Area of Science:
- Pediatric Rheumatology
- Pediatric Orthopedics
- Pediatric Pharmacology
Background:
- Chronic Recurrent Multifocal Osteomyelitis (CRMO) in children is often refractory to non-steroidal anti-inflammatory drugs (NSAIDs).
- Pamidronate, a bisphosphonate, is used to treat bone disorders and has shown potential in refractory CRMO.
- Patient-reported outcomes are crucial for evaluating treatment effectiveness and impact on quality of life.
Purpose of the Study:
- To assess patient-reported effectiveness, safety, and social impact of Pamidronate in children with NSAIDs-refractory CRMO.
- To analyze symptom alleviation, adverse drug reactions (ADRs), and changes in daily activities and social engagement.
Main Methods:
- A retrospective analysis of self-created questionnaires from 42 pediatric patients diagnosed with NSAIDs-refractory CRMO.
- Patients received intravenous Pamidronate (1 mg/kg/day for 3 days).
- Questionnaires assessed pain intensity, ADRs, and self-reliance in daily activities.
Main Results:
- Significant pain reduction observed after an average of 2.5 Pamidronate series, with complete resolution after 5.9 series.
- Nearly 50% of patients reported increased social participation after the first infusion.
- 93% considered Pamidronate effective; 95% would recommend it, despite an average of 2.5 ADRs per patient.
Conclusions:
- Pamidronate demonstrates significant patient-reported effectiveness in NSAIDs-refractory CRMO, improving pain and social engagement.
- The therapy is generally well-tolerated and recommended by patients, though ADRs are common.
- Further research quantifying quality of life is needed to fully assess Pamidronate's impact on daily activities.
Abstract:
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.
