DECISION ANALYSIS IN THE TREATMENT PATHWAY FOR USE OF HYDROXUREA IN CHILDREN WITH SICKLE CELL DISEASE

Purpose:

Use a clinical decision tree to analyze a proposed treatment regime using hydroxurea for children diagnosed with Sickle Cell Disease (SCD)

Method:

Model Type – The model type used for this project is a decision tree. This project focused on estimating the quality of life (QALY) for children with SCD.

Target Population – The target population are children who have been diagnosed with sickle cell disease. With no suitable cure in place for the disease, the main form of management of the disease is mainly supportive management in trying to prevent the complications of the disease. Hydroxurea is currently the only recommended drug that is in use for the disease. While not curing the disease, it helps in reducing and preventing the various complications of the disease

Strategies –

No treatment – currently the protocol for children with SCD. Children receive all supportive treatment but no hydroxurea.

Current Regime – Hydroxurea used only in those severe SCD. Protocol used in adults and also used in clinical trials for children with SCD.

Proposed Regime – Hydroxurea used for all children with SCD

Result:

Analysis, of rolling back, or our decision tree revealed that the treatment strategy associated with the highest average utility (quality of life) over a 5-year period was proposed regime (0.84). Average utilities for no treatment and current regime were 0.68 and 0.76 respectively. One-way sensitivity analysis of clinical parameters over plausible ranges based on variation seen in the published literature revealed the proposed regime was the preferred strategy in the large majority of scenarios

Conclusion:

Based on the decision tree the preferred treatment option will be the children with SCD be on hydroxurea irrespective of the severity of the sickle cell disease