A RISK EQUATION TO ESTIMATE IRON OVERLOAD-RELATED COMPLICATION RATES BASED ON ADHERENCE RATES OF IRON CHELATION THERAPY IN TRANSFUSION-DEPENDENT THALASSEMIA
Author(s)
Huang VW1, Kuizenga P2, Barbier M2, Kessabi S2
1Novartis, East Hanover, NJ, USA, 2Wellmera, Basel, Switzerland
Presentation Documents
The aim of this study was to determine an adherence-based risk equation that estimates the annual risk of developing IOL-related complications for any ICT in patients with TDT.
METHODS: A literature search was conducted for publications reporting the risk of IOL-related complications in relation to adherence.
RESULTS: None of the articles described the full risk equation used in their model or study. Therefore, the findings and assumptions from three publications were combined into one risk equation: IOL compl (other trt)=IOL compl(DFO)*(1-rr)^((Ad(other trt)-Ad(DFO))*100)
IOL compl = annual risk of IOL related complications in %, other treatment (trt) = DFX, DFP, DFO/DFP, Ad = adherence in %, rr = risk ratio in %
We assumed a baseline IOL-related complication rate under ICT. DFO was selected as the reference treatment, as published adherence rates and associated complication rates were available. To estimate the IOL-related complication rate for other treatments, the baseline rate was adjusted by the adherence-dependent risk ratio. This risk ratio was complication specific and, with each percentage point increase in adherence, the complication rate was reduced by the risk ratio.
CONCLUSIONS: Risk equations are often used in models, but rarely published. This equation can be used for future economic evaluations of ICT using adherence rates from clinical trials or real-world settings. Further validation of the equation is needed.
Conference/Value in Health Info
Value in Health, Vol. 19, No. 3 (May 2016)
Code
PSY55
Topic
Patient-Centered Research
Topic Subcategory
Adherence, Persistence, & Compliance
Disease
Systemic Disorders/Conditions