Hi Zahra,
In two months the Hb jumped two points while on hydroxyurea. The HbF also rose. I would say you should start again. Hydroxurea reduces ineffective erythropoiesis, which means there are fewer bad red blood cells floating around in the blood, which in turn leads to less destruction of the good blood cells, and then the good red cells live longer, causing an increase in Hb, along with the increased HbF. I would really go six months more and then evaluate. I know parents worry about the side effects of the drug, but there is a silent danger within the blood of thalassemics, and it is caused by a combination of bad red blood cells and unmatched alpha globin chains, that are in excess due to the deficient beta globin production. We should not underestimate the damage that this does to thals. Even thals with no iron load suffer from a shrinking pituitary gland that eventually disappears altogether. It is not just iron that is the danger. The same thing goes for the heart and pulmonary artery. The damage is not solely iron related. When you consider this, hydroxyurea becomes a much safer option in relative terms because of this ability to reduce the ineffective erythropoiesis. Results are also seen in reduced spleen size, lower bone marrow activity, which means less stress on the bones from expansion, and more normal growth. I have yet to see a study that did not show a positive effect on the majority of thals who used hydroxyurea. In fact, I have often found a tone of surprise in some reports, because they really did not expect it to work so well. Even with a low increase in Hb, these other things are taking place. As I have advised with Manal's son and found that Dr Marwaha concurred, we have to look at the child. If growth is in the normal range and the child is healthy and has normal activity, then we have to keep doing what we are doing. If you see noticeable changes in activity level, increased lethargy and worsened health, then transfusion may be necessary, but as long as a child is healthy, transfusion isn't an ideal choice.
Doctors are really pushing BMT these days because of increased success rates. You may want to investigate if a mother to child transplant is possible. I don't think there's a good way to screen. Only siblings would have a good chance of being a perfect match. I doubt there is any better chance within a set of relatives than there is in the general public. I would suggest contacting Eileen at CAF to see if she can give you information on the bone marrow registry.
Patient Services Manager
Eileen Scott
eileen.s@cooleysanemia.org