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Guest Blog: What Gets Measured Gets Managed

Guest blog post by Bertil Damato, MD, PhD, FRCOphth. Dr. Damato is a Senior Clinical Research Fellow at the Nuffield Department of Clinical Neurosciences in Oxford, United Kingdom and a member of the CURE OM Patient Registry Steering Committee.

There is a saying that ‘what gets measured gets managed.’ By measuring success rates, complication rates, quality of life and patient-reported outcomes such as pain and visual handicap after treatment for ocular melanoma, we can assess the quality of care and improve it.

Another way to improve medical care is to ‘know thy enemy.’ The more we know about uveal melanoma, its causes, effects and patterns of behavior, the greater the likelihood of rational treatment and the better the chances of maintaining or restoring good health. This requires a rigorous scientific approach to discovering the biology of uveal melanoma and the impact of treatment on the disease. This means abandoning intuition and adhering to the scientific method: forming hypotheses which are then tested by statistical analysis. For example, intuitively, we might ask if uveal melanoma biopsy is dangerous because it disseminates tumor cells throughout the body that can cause metastatic disease and death. To find out whether this is indeed the case, we must first formulate the hypothesis that ‘uveal melanoma biopsy causes metastatic disease’ and then to attempt to disprove that hypothesis by comparing survival in patients who have had a biopsy with patients who have not had this procedure. If such a study does not compare ‘like with like,’ it will show that metastasis is more common in patients who have had a biopsy. However, this will probably be because of selection bias: patients who have a biopsy tend to have larger tumors, which tend to be more aggressive than small tumors, which are more difficult to biopsy so that this procedure is omitted.

Another important question that needs to be addressed is whether radiotherapy is as effective as enucleation in preventing metastatic disease. The Collaborative Ocular Melanoma Study (COMS) concluded that radiotherapy is as ‘safe’ as enucleation. This may be the case when the radiotherapy is successful at sterilizing the tumor, but what about radiotherapy failures? Several studies have shown that metastatic death is more common in patients whose tumor has recurred after radiotherapy. Is this because tumors that recur tend to be more aggressive and life threatening? Or is it the surviving tumor that metastasizes? A clinical trial addressing this question would need very large patient numbers to have enough ‘statistical power’ to prevent the wrong conclusions from arising by chance and these patients would need to be followed up for very many years.

What this all means is that we need a lot of high-quality data, that is, data that are complete and correct, from large numbers of patients, who must also be representative of patients who are not included in the study. This is difficult to achieve because ocular melanomas are so rare. Further, the data must be collected contemporaneously, that is, as things happen and not years later, and it must be verified to confirm that it is true and accurate. This requires efficient methods for collecting all data, without leaving any gaps, and for entering the data correctly into the registry, without making any errors. What this means is that patients and practitioners must be reminded and encouraged to complete forms and submit data in a timely manner. Special measures must be taken to collect data if the patient is no longer able to submit forms because of ill health. It is essential to have a fool proof system for documenting the date and cause of all treatment outcomes. The data must be saved in a form that is appropriate for statistical analysis. The registry itself must be safe, secure, and future-proof. All analyses must be performed by professional statisticians to avoid misconceptions from arising. Ethical standards must be adhered to, for example, ensuring that proper patient consent is obtained for the collection, storage and use of personal data, while preserving patient confidentiality and anonymity at all times. This means restricting access to the data to only those who need to use it for the research being undertaken.

All these requirements make research very labor intensive and expensive, so that it tends to be performed only in ‘centers of excellence,’ but the results achieved in such centers may not be representative of the rest of the country, where perhaps most patients are treated. The CURE OM registry is seeking to overcome this problem by reaching out to patients directly while also collaborating with healthcare providers. This initiative may therefore create research opportunities that are not otherwise possible so that patients with ocular melanoma can receive better care, with better outcomes and higher survival rates.

Through projects like the CURE OM Patient Reported Registry, the MRF’s CURE OM initiative is committed to driving progress towards better treatments, improved patient outcomes and, one day, a cure for ocular melanoma. Please consider supporting this life-saving work with a tax-deductible gift to CURE OM: