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A Cost-effectiveness Study of Intravenous Immunoglobulin in Childhood Idiopathic Thrombocytopenia Purpura Patients with Life-Threatening Bleeding

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Abstract

Background

Although the international guideline recommends intravenous immunoglobulin (IVIG) as the first-line treatment for childhood idiopathic thrombocytopenia purpura (ITP) with life-threatening bleeding, ITP patients may not be able to access IVIG because of the limitation in health benefit packages especially in developing countries. There remains an important policy question as to whether IVIG used as a first-line treatment is worth the money spent. Thus, the objective of this study was to perform a cost-effectiveness analysis of adding IVIG to the standard treatment of platelet transfusion and corticosteroids, for the treatment of childhood ITP with life-threatening bleeding in the context of Thailand.

Methods

A cost-effectiveness analysis using a hybrid model consisting of a decision tree and Markov models was conducted with a societal perspective. The effectiveness and utility parameters were determined by systematic reviews, while costs and mortality parameters were determined using a retrospective electronic hospital database analysis. All costs were presented in 2012 US$. The discount rate of 3 % was applied for both costs and outcomes. One-way and probabilistic sensitivity analyses were also performed.

Results

The incremental cost-effectiveness ratio (ICER) was $3,172 per quality-adjusted life-year gained ($/QALY) for the addition of IVIG versus standard treatment alone. The probability of response to corticosteroids was the most influential parameter on ICER. According to the willingness-to-pay of Thailand, of approximately $3,861/QALY, the probability of IVIG being cost effective was 33 %.

Conclusions

The addition of IVIG to standard treatment in the treatment of childhood ITP with life-threatening bleeding is possibly a cost-effective intervention in Thailand. However, our findings were highly sensitive. Policy makers may consider our findings as part of the information for their decision making

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Acknowledgments

This study was supported by grants from the subcommittees of the National List of Essential Medicine, Thailand and the Thailand Research Fund (TRF) through the Royal Golden Jubilee PhD program (Grant No. PHD/0356/2550 to P.D. and Grant No. PHD/0127/2552 to R.S.). The authors gratefully acknowledge the support of Associate Professor Dr. Sanan Visuthisakchai, Division of Hematology, Department of Medicine, Dr. Chanin Limwongse, Division of Molecular Genetics, Department of Research and Development and Assistant Professor Dr. Bunchoo Pongtanakul, Department of Pediatrics, Faculty of Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand for comments on economic model development and input parameter suggestions.

Conflict of interest

The authors declare that they have no conflicts of interest.

Role of the funding source

No sponsors were involved in this study.

Author contributions

RS and PD participated in the design of the study, developed the cost-effectiveness model, conducted stake-holder meetings, searched input parameters, and conducted the cost-effectiveness analyses. NC participated in the design of the study, developed the cost-effectiveness models, identified data sources, conducted stake-holder meetings, and conducted the cost-effectiveness analysis. CP participated in the design of the study and identified data sources. All authors have read and approved the final manuscript. Each author also contributed to the interpretation of data and results, drafting the manuscript, critically reviewing the manuscript for important intellectual content, and has approved the final version.

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Correspondence to Nathorn Chaiyakunapruk.

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Sruamsiri, R., Dilokthornsakul, P., Pratoomsoot, C. et al. A Cost-effectiveness Study of Intravenous Immunoglobulin in Childhood Idiopathic Thrombocytopenia Purpura Patients with Life-Threatening Bleeding. PharmacoEconomics 32, 801–813 (2014). https://doi.org/10.1007/s40273-014-0171-9

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