Following is an article by our friend and long-term MDS patient advocate from Germany, Bergit Kuhle.
Bergit, together with Anita Waldmann, runs an MDS Patient Group in Germany, and both are founding members of the international MDS Alliance – the umbrella organisation for all national patient groups in the world.

No chance to lay down my burden of iron overload somewhere

Our organism does not have any biochemical or physiological pathway to remove excess iron. Instead the excess iron accumulates mainly in the liver, joints, pancreas, bone marrow and at the worst in the heart, causing organ and tissue damage in the long run. We’re talking about transfusion-related iron overload.

I have been a Myelodysplastic Syndrome (MDS) patient for more than 12 years and I regularly need blood.

The main cause of iron overload is transfusion therapy. Each unit of blood transfused delivers 200–250mg of iron. After only 20 units of transfused blood, 4,000–5,000 mg of iron will have been delivered to the body. At this ironloading, the serum ferritin level rises to about 1,000μg/l. When this level is about to be reached, the doctor suggests iron chelation for transfusion-dependent patients like me.

Bergit Kuhle: MDS Patient on Iron Chelation Therapy

I have been a Myelodysplastic Syndrome (MDS) patient for more than 12 years and I regularly need blood.

Each unit of blood transfused delivers 200–250mg of iron. After only 20 units of transfused blood, 4,000–5,000 mg of iron will have been delivered to the body.

But our organism cannot remove the excess of iron so it accumulates mainly in the liver, joints, pancreas, bone marrow and at the worst in the heart, causing organ and tissue damage in the long run.

My experience with the iron chelator Deferasirox

I have never had a wealth of medical expertise on iron overload. I have accepted the most important fact: excess iron does harm to my vital organs, so chelation is necessary.

Everything went according to plan. I trusted the medication, not giving too much thought to the issue. I have been tolerating the iron chelator, Deferasirox, without significant side effects for 10 years now. I only had an uncomfortable rash in the beginning, which disappeared after two weeks and never ever came back. The ferritin level reliably has descended and always has
stayed below 1,000μg/l.

So, I felt safe from life-threatening organ iron deposition. I was as compliant as can be and always, always took my pills or my suspension even when travelling. I even prepared and swallowed the suspension in an airport’s ladies room. Finally, I have built confidence in my ferritin level, which was fluctuating a little from time to time. But ‘what the heck’, my haematologists have
been content with 400-700 μg/l and I have never been experiencing any discomfort.

I have been lucky with my iron-chelator Deferasirox, being free from side-effects. Whereas in MDS web forums and in my work with patients, I met with various people suffering from all kinds of bearable or unbearable side-effects, mainly such as all stages of gastrointestinal disorders.

A chemical compound binds with the excess of iron in the body to safely pass through the body

Whether you are offered iron chelation treatment or not will depend on the likely benefits versus the likely disadvantages in your
individual case. This will be discussed with you before you make a decision to start iron chelation.

Desferal is a drug to treat the build up of excess iron and is given as a continuous subcutaneous injection under your skin by a pump. There are special teams that can teach you how to administer the drug at home. Exjade is another iron chelator and comes in tablet form, making it easier for patients with poor sight, problems of finger dexterity or a fear of needles. However, in most cases this is only available for patients who cannot manage subcutaneous Desferal or who have serious side effects on Desferal.

Both treatments have certain side effects and often need to be continued for a long period of time to be effective. Your doctor can advise you which drug will be best in your situation.

From our booklet Understanding MDS: Patient Handbook. Click here to download

Devastating news

For me years of chronic transfusion dependence went by and I counted my blessings: more than 530 units of blood in total since 2008, with a stable transfusion interval of 10-18 days since 2010.

My treating professor initiated an invitation for me to a special clinic, where I underwent an MRI in autumn 2017. They scanned my heart, liver, pancreas and my bone marrow. The results were devastating. My view on iron overload broke with the medical report. I still have not fully recovered from what I was told. I would be very lucky, the radiologist said, if my heart was still free of iron. But my liver and my pancreas show a five-fold iron overload, also my bone marrow is full of iron. Another obstacle for
haematopoiesis in addition to my blood disorder.

Where do we go from here? Heading towards diabetes, liver cirrhosis or even hepatic cancer? My heart is iron-free, yes, but the pancreas is strongly loaded. No reason for long-lasting joy as the pancreas takes up similar iron species as the heart, only earlier. Iron overload of the pancreas serves as an early and robust marker of prospective cardiac risk.

It is neither an option for me to increase the dosage of the iron chelator – this could be toxic for my kidneys for instance - nor to reduce the number of transfusions – this would destroy my quality of life and would send me into severe anaemia. I’m stuck.

As so often in the course of my disease there is a diagnosis without any therapeutic consequences. That’s frustrating.

On the medical report I more recently read ‘severe hemosiderosis of the pancreas and the liver’ amongst my other serious health problems. However, my liver values (GPT, GOT) are not yet elevated. Not yet? I am anxious and confused: that’s the patient’s perspective.

Only now I am finding out that for any one patient, the predictive value of ferritin is quite poor. Two patients with identical total body iron burdens can have vastly different serum ferritin levels. Many factors affect the relationship between iron overload and serum ferritin levels. But still the ferritin level generally correlates with both, total body iron stores and clinical outcomes.

Serum ferritin therefore remains the most commonly used metric to monitor iron chelation therapy. Mainly because its measurements are inexpensive in comparison to MRI. However, the biology of circulating ferritin is poorly characterised and hardly comprehensible for non-physicians and non-scientists.

The way iron is regulated by the body relies on a regulated network of systemic and cellular mechanisms for the acquisition, transportation and cellular utilisation of the metal. I very well understand that these metabolisms are completely disturbed in case of strong iron overload.

Chelation Therapy: Good or Bad?

At the end of this article I want to make clear one thing: the outcomes of chelation therapy in Myelodysplastic Syndromes are not bad. There is a positive impact on survival, without doubt. I probably would not be alive any more without Deferasirox.

There are retrospective studies clearly indicating a significant improved survival in patients who received regular iron chelation. Transplant outcomes are also far better after iron chelation therapy. And in case there is a cure, high iron stores can be drained or at least reduced by a most effective phlebotomy therapy.

So yes, there are definitely some solutions for iron overload, if you are in the right cohort. But we urgently need more innovative iron chelation therapies.

Bergit writes a regular column in ‘MDS Europe’, the online home of the MDS-RIGHT project and the future hub for all European MDS information and guidance.

This article appeared first in MDS Patient Support Group Newsletter May 2018

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