1.
A cost-effectiveness study of intravenous immunoglobulin in childhood idiopathic thrombocytopenia purpura patients with life-threatening bleeding
Sruamsiri R, Dilokthornsakul P, Pratoomsoot C, Chaiyakunapruk N
Pharmacoeconomics. 2014;32((8):):801-13.
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.
2.
Economic assessment of recombinant factor IX in prophylaxis for hemophilia B in young patients in Mexico
Mucino-Ortega E, Salinas-Escudero G, Galindo-Suarez RM
Value in Health. 2013;16((3):):A118.. Abstract No. PSY42.
3.
Canadian cost- utility analysis of intravenous immunoglobulin for acute childhood idiopathic thrombocytopenic purpura
Blackhouse G, Xie F, Levine MA, Campbell K, Assasi N, Gaebel K, O'Reilly D, Tarride J, Goeree R
Journal of Population Therapeutics & Clinical Pharmacology. 2012;19((2):):e166-78.
Abstract
Background Idiopathic thrombocytopenic purpura (ITP) is a hematological disorder and can be classified as acute or chronic. The main goal of treatment for acute childhood ITP is the prevention of potentially fatal bleeding complications, the most serious of which is intracranial hemorrhage (ICH). Treatment options for acute childhood ITP include splenectomy, corticosteroids, and blood products such as intravenous immunoglobulin.Objectives The objective was to evaluate, from a Canadian perspective, the cost-effectiveness of intravenous immunoglobulin (IVIG) compared to alternative inpatient treatments for acute childhood idiopathic thrombocytopenic purpura (ITP). Methods A Markov model with a lifelong time horizon was used to evaluate the costs and quality-adjusted life years (QALYs) for 5 treatments for children hospitalized for ITP: 1) no treatment; 2) IVIG; 3) Anti-D; 4) prednisone; and 5) methylprednisolone. The model predicted the probability of intracranial hemorrhage for each treatment strategy based on the time children spent with platelet counts <20,000microL. The time patients spent with platelet counts <20,000microL with each treatment was estimated by pooling data from published randomized clinical trials. In the basecase analysis, the cohort was assumed to weigh 20kg. Cost and utility model variables were based upon various literature sources. Parameter uncertainty was assessed using probabilistic sensitivity analysis. Results The treatment strategies that comprised the efficiency frontier were prednisone, Anti-D and IVIG. The incremental cost per QALY was $53,333 moving from prednisone to Anti-D and $53,846 moving from Anti-D to IVIG. Results were sensitive to patient weight. If patient weight is 10kg, IVIG dominates all other strategies and if weight is increased to 30kg, the cost per QALY of IVIG is $163,708. Conclusion Based on common willingness to pay thresholds, IVIG might be considered a cost effective treatment for acute childhood ITP. Cost effectiveness is highly dependent on patient weight.
4.
A cost-utility analysis of treatment for acute childhood idiopathic thrombocytopenic purpura (ITP)
O'Brien SH, Ritchey AK, Smith KJ
Pediatric Blood & Cancer. 2007;48((2):):173-80.