Utilization and efficacy of palivizumab for children with Down syndrome
Takeshi Kimura1, Masato Takeuchi1, Koji Kawakami1
1Department of Pharmacoepidemiology, Graduate School of Medicine and Public Health, Kyoto University, Kyoto, Japan.
Insights
Palivizumab prophylaxis in children with Down syndrome (DS) significantly reduced respiratory syncytial virus (RSV) hospitalizations. This study analyzed Japanese health insurance data to assess palivizumab
Area of Science:
- Pediatric Infectious Diseases
- Immunoprophylaxis
- Genetic Syndromes
Background:
- Palivizumab is a monoclonal antibody used for respiratory syncytial virus (RSV) prevention in high-risk infants.
- Japan expanded palivizumab eligibility for children with Down syndrome (DS) in 2013, irrespective of other risk factors.
- Evidence supporting universal palivizumab use in DS populations remains limited.
Purpose of the Study:
- To investigate the prescription patterns of palivizumab in children with DS in Japan.
- To evaluate the efficacy of palivizumab in reducing RSV-related hospitalizations among children with DS.
Main Methods:
- Analysis of Japanese health insurance claims data (JMDC) from 2007-2015.
- Inclusion of children aged ≤24 months with DS at the start of the RSV season.
- Comparison of RSV-related admission risks between children receiving palivizumab prophylaxis and those not, using mixed-effects logistic regression.
Main Results:
- Palivizumab use increased significantly after the 2013 program expansion, particularly in children with DS but without additional risk factors (0% to 73%).
- Overall, RSV-related hospitalization occurred in 4.2% of children with prophylaxis versus 6.0% without.
- Multivariate analysis indicated that palivizumab was associated with a reduced risk of RSV-related hospitalization (OR: 0.41, 95% CI: 0.18-0.92, P=0.03).
Conclusions:
- Palivizumab use is now widespread among children with DS in Japan.
- Palivizumab prophylaxis demonstrated an association with decreased RSV-related hospitalizations in this population.
- Further research is needed to determine optimal, risk-tailored versus universal, prophylaxis strategies for children with DS.
Background:
Palivizumab is a prophylactic drug used for severe respiratory syncytial virus (RSV) infection in young children with high-risk factors, such as prematurity and congenital heart diseases. Although Japan expanded the use of palivizumab for Down syndrome (DS) in 2013 regardless of additional high-risk factors, there is not enough evidence to support its use. We analyzed health insurance claim data from the JMDC (Tokyo, Japan) to investigate the prescription pattern and efficacy of palivizumab in children with DS.
Methods:
We enrolled children of ≤24 months of age with DS at the start of the RSV epidemic season (2007-2015). We compared the risk of RSV-related admission with and without palivizumab prophylaxis using mixed effects logistic regression models accounting for patient-level clustering.
Results:
Of 632 children, 30% (81/268) and 83% (303/364) received palivizumab before and after the expanded program (2007-2012 and 2013-2015), respectively. Among children with DS but without additional high-risk factors (n = 135), palivizumab use surged from 0% (0/62) to 73% (53/73). In the whole study population, RSV-related hospitalization occurred in 4.2% (16/384) patients with prophylaxis and 6.0% (15/248) patients without prophylaxis. The multivariate analysis revealed that palivizumab was associated with reduced RSV-related hospitalization (odds ratio: 0.41, 95% confidence interval: 0.18-0.92, P = 0.03).
Conclusions:
Virtually all children with DS in Japan have now received palivizumab, the use of which was associated with a reduction in RSV-related hospitalization in DS. Further evidence is required to clarify whether palivizumab prophylaxis should be risk-tailored or universal for DS.
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