UNLABELLED: The utility of predictive systems in patients with haematological malignancy stems both from the heterogeneity of disease presentation and from the inherent disadvantages of therapy. There is an acute morbidity and mortality associated with chemotherapy and bone marrow transplantation, a need for long term follow-up, the possibility of treatment failure and the issue of cost. In general, the value of a scoring system is in its ability to quantify disease burden, classify patient subgroups and predict disease course. Successful scoring systems are disease-specific, have been validated in an independent population and are useful in different countries, centres and individuals. There should be good discrimination of poorly responsive and optimally responsive populations and a high rate of prediction of the disease course for individual patients. Optimal utility should be provided by a few variables which are independent, highly predictive and easy to obtain. Other considerations include economic evaluation and adaptation for population changes over time.CONCLUSION: Experience gained from the use of staging and scoring systems in haematology provides useful insights into their potential use in Fabry disease and other lysosomal storage diseases. Such systems may help to predict whom to treat, when to treat and what response is likely to be achieved.
Health Status Indicators Hematologic Diseases