Life without bone marrow transplant

When successful, bone marrow transplant can bring an end to the constant need for blood transfusions and over time, reduce the need for iron chelation therapy. Sadly, bone marrow transplant is not a viable option for many. And for many others [including some known to me personally] it not an option they would even consider.
My mentor, from whom I learnt much about the Thalassaemias, would often ask why bone marrow transplant is such an attractive option to some families. Why do we as people with Thalassaemia [their families and medical staff involved in their care] accept the risks involved in the procedure and go through the difficult treatment protocols?
Would we be as willing for bone marrow transplant if our Thalassaemia was better managed?
I recall the many conversations I had with a number of people with Thalassaemia in Melbourne. I saw in them individuals empowered to take hold of their lives, setting goals and carrying on with life with confidence and pride. Never considering themselves as anything less than a most capable and normal person. They were normal people living normal lives, their medical condition being just a part of who they were.
It wasn't denial; they accepted the medical condition they had and put in an effort to make sure that it wouldn't hold them back in life. With this attitude and the amazing support structure they built around themselves they were able to achieve a healthy state of living from where the risks associated with bone marrow transplant were not worth the risk.
Sadly, the same is not true in many other places. Accessibility to care, the immense hardship individual people and their families have to go through to get much needed precious blood, the lack of a decent support structure to assist in the sometimes difficult chelation therapy and the overall poor results of the largely inadequate therapy makes these differences all too obvious.
Optimised blood transfusions beginning early in life has in those centers reduced the bony changes that are still so visible on our faces. "They look like normal people" said this friend of mine with Thalassaemia upon meeting a middle aged woman with Thalassaemia from another country. And how true his statement was! The disappointment was not hidden in his voice when he later said "My face tells others that I have Thalassaemia". True again. His under-transfused body was trying to make more blood by expanding his facial bones causing tell tale facial features to appear. If we were to offer optimized (and personalized) blood transfusion regimes to cases with Thalassaemia in Maldives, we will be able to minimize the physical deformity associated with the condition. Accepting the fact that our blood donor pool is small and finding others ways of getting blood {say for example getting Red Cross in other countries to donate blood to us} would make it possible to provide adequate transfusions.
But perhaps the most significant difference that I noticed was that the chelation therapy was very well accepted as a part of the treatment in Melbourne. People used their pumps [yes they use pump and needles] diligently. One young girl from Melbourne said, "This is what will keep me well till you can find a cure". Another said, "I have blood to keep me going just the same way as my car needs petrol to run and it is Desferal that keeps my body’s engines in tip-top shape to work".
They keep themselves well by pumping iron out. Chelation is such an essential part of management that much of the treating physician’s time and effort is spent on that aspect of care. Again, we are failing very badly indeed in this aspect. Compliance with Desferal therapy is so very poor in a large number of people that accumulating iron slowly but surely poisons the body organs.
This is in my opinion why bone marrow transplant is such an attractive treatment option for our cases. Not requiring difficult and time consuming treatment on a regular basis is a blessing for anyone. What we at times fail to bring into the equation are the risks associated.
For those who do not have the bone marrow transplant option on the table; there is still life. These people from Melbourne with the medical condition have shown me this.