effects of hydroxyurea

How soon does hydroxyurea start increasing hb. How soon can u see its effects ?

A base line hemoglobin electrophoresis should be done at the start to measure the HbF level, in addition to the Hb. Periodically, electrophoresis should be repeated to measure changes in the HbF. If the HbF level does not rise significantly over time, it shows the drug is not having the desired effect. You should see changes in the HbF level during the first 3-6 months.

Just got alyshah hb report after using 2 weeks of HU. What do u think of it.
Hb before 7.2       hb now 6.8
Reticulocyte before 10% now 2.5%
All other things have only changed minutely.

My doctor says its normal for the hb to drop and if comes
Stable in the range of 6 we don't need to be worried about transfusions. She
Is extremely active and looks better then before.

My question is that is there a chance that her hb can increase Significantly
With HU or is that my wishful thinking. Doctor also says she can less a normal life with this
Hb using hydroxyurea.
Her base line HbF is 30%
Thanks for your quick replies

With a baseline HbF of 30% there is a very good chance that hydroxyurea will work for her. A good sign already is the lowered retic count, as this indicates that the bone marrow is working less and producing fewer inefficient red blood cells. I suggest patience and that you give it at least one year to see the full effect of the drug.

Andy is it possible that with time HU increases her hb to around 8. Plz give me a realistic answer to this. I don't want to hope too much.

Yes. Patients who respond well to hydroxyurea will see a 1 to 2 point gain in Hb, but it does take time and the full effect may not be seen until the drug has been used for two years. You will know however, if it is beginning to work within 6 months.

Thanks for you informative answers. They always give me hope. Hope is what I live on these days!!!

Better drugs will come as your child grows. It is expected that the new HbF inducing drugs that are currently in development will raise Hb levels by 3 to 4 points. Stay hopeful.

Hi Andy

Can you please give more information about new drugs being created. Maybe if you can send a link to further research being done on HbF increasing drugs? How long will they take to come into the market.

Regards
Asma

This is the current status of the HQK-1001 trials, which are ongoing. They do not give any specific information as to gain in Hb, but they do use this language, "Based on favorable study results" so it does appear that things are progressing as they move deeper into trials.

http://www.hemaquest.com/technology/tech_HQK-1001.asp

HQK-1001
HQK-1001HQK-1001 is an orally administered short chain fatty acid (SCFA) derivative, which has a unique combination of biological effects - induction of fetal globin and stimulation of red blood cell production - that addresses the underlying pathological mechanisms in sickle cell disease and beta thalassemia. Extensive studies in both the laboratory and relevant animal models carried out in transgenic mice and non-human primates have demonstrated the potential therapeutic effects of this compound in treating these serious, chronic illnesses. HemaQuest has obtained an exclusive worldwide license for a series of patents and patent applications from Boston University for these technologies that give it a strong proprietary position.

In 2010, HemaQuest completed proof of concept trials of HQK-1001 in both beta thalassemia and sickle cell disease. These studies confirmed the safety of HQK-1001 in this patient population and provided evidence of pharmacodynamic effects, such as rises in fetal hemoglobin and total hemoglobin. In March 2012, HemaQuest completed an open-label, multi-dose Phase 2a study of HQK-1001 given for up to 6 months in 52 patients with sickle cell disease. Based on favorable study results, a multicenter, placebo-controlled Phase 2b study was initiated in 2012 to evaluate HQK-1001 given twice a day for 48 weeks in 74 patients with sickle cell disease.

Two-investigator-sponsored studies were initiated in 2012 to study the safety and pharmacodynamics of HQK-1001 given daily for 6 months in patients with beta thalassemia.

HemaQuest has been granted Orphan Drug Designation in the US and the EU for HQK-1001 in both sickle cell disease and beta thalassemia.

Thanks Andy your replies are always very informative. Can u tell / guess when this can be available for us.
Regards
Asma

I have no information about how long the trials will be and how long before approval is sought.

Dear Andy,

Pls let me know the side effects of hydroxyurea? Does it impacts women's fertility?