Why Bother Measuring Outcomes in Rheumatology?

You can’t bank on the outcome

Daniel Berriman

For a many years we Rheumatologists relied on clinical judgement to decide how our patients were doing. We tended to assume that this would result in the best outcome for our patients. Actually, we weren’t doing nearly as well as we thought. Most of us were aware of disease scoring methods, but we regarded these scores as mainly for use in research. Things are changing, and it is high time both patients and doctors had another think about outcomes and how to measure them.

When we talk about ‘outcome measures’ in arthritis, I would guess that some people might wonder what we’re on about. What is an outcome when there’s no income, I hear you ask! Some people may be more familiar with the terms ‘bottom line’ or ‘end product’. Just as in business, if we want to improve our effectiveness or efficiency at treating people with arthritis, we know that we need to not only ‘measure’ what we’re doing but also set targets. There is now a mounting body of evidence to support a ‘Treat to Target‘  approach to RA treatment. This strategy involves changing treatments every time Disease Activity Scores go above a certain level. This ‘aggressive’ approach to treatment is more likely to achieve ‘remission’ or a ‘low disease activity state’ than the traditional approach. It is reliant on the regular use of outcome measures which determine the target to aim at. But what exactly should we be measuring and how should we do it?

Most people who attend Rheumatology clinics have arthritic conditions where there is joint inflammation and/or damage. We can speak about ‘pathology’ meaning that there is something abnormal going on in the tissues in and around the joints. So one of the things we need to measure is how active the disease is and how well we are doing at reducing the disease activity. In the early stages of inflammatory arthritis, there is still some hope of a full remission if the inflammation can be controlled. However, we also know that prolonged periods of inflammation in a joint lead to damage, so we also need to measure the severity of joint damage. Once a joint has been permanently damaged, completely controlling disease activity will not be able to restore the joint to normal and the chance of a full recovery is lost. We need drugs that not only reduce disease activity but also reduce damage. We must not assume that drugs that reduce inflammation will also be effective at stopping joint damage. At some point we will also need to target ‘prevention’ or ‘cure‘ as outcomes. At the moment we can’t claim to have cured any type of arthritis with the possible exception of gout. Of course we mustn’t forget that RA is a systemic disease, so there are other aspects of disease activity we may not be measuring (such as anaemia).

Diseases are only important to people if they have an impact on how they function, their ability to work or do normal daily activities, their quality of life, or if they increase the chances of dying prematurely. Treatments are of little use unless they result in an improvement in some of these outcomes. Twenty years ago the available treatments for arthritis had – at best – a roughly 50-50 chance of making the patient feel better but also a one in three chance of causing unpleasant side effects. In recent years, there has been a shift away from over emphasis on disease related outcomes toward ‘patient related outcome measures’ (PROMS). Many of these outcomes are common to most diseases, so we can compare how the quality of life of a patient with arthritis compares with that of a patient with another condition such as chronic lung disease or chronic Heart Failure. It has to be remembered that many patients suffer from several conditions, so these outcome measures may reflect the influence of other diseases as well as arthritis. I don’t think there would be many doctors who would suggest that we should measure PROMs without any reference to disease related outcome measures because we do need to keep our focus on tackling the underlying disease.

Finally, we shouldn’t forget the importance of ‘patient satisfaction‘ as an outcome and (on a population scale) the ‘health economics‘ of the intervention.

Measuring outcomes takes time. Outpatient clinics are busy. It is easy to recommend that everyone should have three or four outcome measures checked every time they come to clinic, but it is not so easy to do this when the clinic is running late or when the patient comes in with a long list of questions. Rheumatologists are used to seeing outcomes reported in the medical research literature reporting whether or not an intervention is effective. It is only very recently that it has become generally accepted that recording a disease activity score such as the DAS28 should be routinely recorded for RA patients at every visit to clinic. A recent Spanish survey reported at EULAR 2012 suggested that the DAS28 score was being recorded about half of the time in clinic.

This is the first in a series of articles about how patients and health professionals can make sense out of the existing disease outcome measures and how things are likely to change for the better in the near future…


Sydenham: Granddaddy of Rheumatology?

Here’s a little quiz for all you medical historians:

  • Which physician when asked to recommend a medical textbook famously advised the enquirer to buy ‘Don Quixote’?
  • Which physician developed (and personally tested) the liquid preparation of opium better known as Laudanum?
  • Which physician was known as ‘the English Hippocrates’?
  • Which physician wrote the greatest description of a gouty attack in English Literature?

The answer to all of these questions is of course Thomas Sydenham, an English Physician who lived in the seventeenth century in an era where only the rich and famous were privileged enough to be afflicted by gout. He is my pick as the granddaddy of Rheumatology, although I suppose his own hero Hippocrates himself is the great-granddaddy (more of that for another blog). In 1683 he published one of the first texts on gout ‘Tractatus de podagra et hydrope (The Management of [Arthritis] and [Dropsy])’ and in it he shared not only the insights he had gleaned from treating patients but also those learned the painful way. For Sydenham himself had been afflicted with gout since the age of 30, and his constant pain led him to personally try (and reject) most of the proposed ‘cures’ of the time. He eventually relied upon taking Laudanum during acute attacks and barley water as prophylactic. He is quoted as saying “Of all the remedies it has pleased almighty God to give man to relieve his suffering, none is so universal and so efficacious as opium”. It is clear that Sydenham was not a great fan of rational medicine and taught others to learn by careful observation and experience.

If Sydenham were around today, I believe he would be a vigorous proponent of ‘narrative medicine’. His classic description of a gouty attack is for me unparalleled in medical literature and speaks directly from the voice of personal experience.