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

OBJECTIVES: Patients with β-thalassemia major (TDT) require frequent blood transfusions and therefore have a high risk of developing chronic iron overload (IOL). Iron chelation therapy (ICT), consisting of deferoxamine (DFO), deferasirox (DFX) or Deferiprone (DFP), is an important therapy to prevent the related long-term morbidity and mortality related to iron toxicity. Several studies have described the link between suboptimal adherence to ICT and an increased risk of IOL-related complications such as cardiac disease, diabetes, hypogonadism and liver disease.

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

2016-05, ISPOR 2016, Washington DC, USA

Value in Health, Vol. 19, No. 3 (May 2016)

Code

PSY55

Topic

Patient-Centered Research

Topic Subcategory

Adherence, Persistence, & Compliance

Disease

Systemic Disorders/Conditions

Explore Related HEOR by Topic


Your browser is out-of-date

ISPOR recommends that you update your browser for more security, speed and the best experience on ispor.org. Update my browser now

×