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Which patients need more blood transfusions to improve quality of life and can we identify the patients who need iron chelation more precisely?

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Professor David Bowen

18 July 2017

I tend to transfuse patients with more units of blood than our national guidelines would suggest are ‘necessary’. The motivation for this is my belief that patients will feel better if they can maintain a haemoglobin level above the critical value that we have identified together, and below which they develop symptoms that interfere substantially with their lifestyle.

We need to develop better tools for this rather simplistic and subjective approach to deciding on a patient’s symptom threshold.

How far has the haemoglobin level fallen from ‘normal’ for the individual patient?

I often delve back deep into the case notes to try to find a ‘normal’ haemoglobin level for that individual from many years before their diagnosis. I think that it helps to say to a patient for example ‘you are 30% down on your normal haemoglobin so it’s not surprising that you are feeling the effects’. This also helps us both to understand that the patient may have meaningful symptoms of anaemia despite their haemoglobin level being higher than that recommended for active treatment in the guidelines that we follow.

Should patients worry about the complications of iron overload (future) or take more blood transfusions if it helps to make daily life better (now)?

I often recommend that patients increase the frequency of their transfusion episodes or the number of units that are transfused at each visit given my comments in the first paragraph above. However, some patients are quite reluctant to do this because of a fear of iron overload, even though we both know that they will feel better day-in, day-out if they followed the advice to increase the transfusions. So is this fear of iron overload justified? We are all working hard to study iron overload in MDS patients.

In terms of the effectiveness of blood transfusions:

We believe that we know:

  • that blood transfusions lead to (transient) improvements in a patient’s quality of life; most patients tell us this in relation to their individual lives when we discuss it in the clinic.
  • that regular blood transfusions in severely chronically anaemic patients prolongs overall survival

Thus far we do know:

  • that we are measuring health-related quality of life (HRQoL) over time in almost all patients in the EU MDS registry

But we don’t know:

  • how much quality of life (measured as HRQoL) can be improved with transfusions - we have not yet worked out how to measure this appropriately.

With regard to the importance of iron overload and iron chelation:

Thus far we do know:

  • that iron overload is an inevitable consequence of many blood transfusions; all patients receiving many blood transfusions will get iron overload.
  • that we can routinely measure iron overload crudely in the blood (ferritin) and more precisely in the liver and heart (MRI scan).
  • that some patients will develop complications that could be related to iron overload such as heart failure, liver abnormalities and diabetes, but these complications have other causes in older age and it is always difficult to be certain how much iron overload is responsible.
  • that we have drugs that can effectively remove iron from the body.
  • that we can measure some of the toxic components of iron overload in blood samples and we are doing this as part of the EU MDS registry and MDS-RIGHT European research programmes.

But we don’t know:

  • how much quality of life (measured as HRQoL) may be improved with transfusions - we have not yet worked out how to measure this appropriately.
  • which patients with iron overload will develop complications that are definitely related to iron overload and have an impact on the quality of patients’ life or will shorten their life expectancy.
  • which test best measures the type of iron damage (or damaging iron component) that will help us predict which individual patient will be affected by the toxic iron overload.
  • there are side effects with all of the iron chelator drugs.
  • For sure that treatment with iron chelation will prevent the complications that matter to patients, namely damage to organs that can have an impact on the quality of patients’ life or will shorten their life expectancy. In other diseases like thalassemia this is definitely the case and you would expect it to be so in MDS. The key is therefore identifying the patients who benefit from iron chelation treatment and showing that it helps; this is a huge challenge.

In the EUMDS research programme we are hoping that by evaluating the health-related quality of life over time and by measuring the toxic iron species, particularly labile plasma iron (LPI), that we can find some of the answers to these conundrums.

In the meantime the various guidelines that we create as experts can only be informed by proxy evidence, as it has not been possible to do large scale clinical trials in the field and probably now never will be. It is reasonable to continue to recommend iron chelation therapy to a small group of MDS patients who will be transfused over long periods of time.

Please comment below. I will be interested in all of your thoughts.

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