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1、 Good morning, everyone.Im Chuck Peterson, Director of the division of blood diseases and resource force the national heart lung blood institute.and this is a somewhat historic moment I think and certainly an honor for me to be here to introduce the start-up here.its been about a hundred years since
2、 sickle cell disease was described clinically and about 35 years, a little plus since NHLBI was given the responsibility to the sickle cell disease act to try and devise approaches to sickle cell disease.and this is really the first time we have had a drug targeted for sickle cell disease that we ca
3、n look at in terms of an evidence-base in the context of this type of conference so its really cause for celebration.and my job I think in part is to acknowledge the giants in the field on whose shoulders we stand to get to this point.some of whom are here, some of whom are not here.but in fact, it
4、is been a community of dedicated investigators and a community of willing patients who has made these steps possible.so we have the privilege today of taking a look at the evidence base and putting hydroxyurea in perspective for the patient community and those who care for them.didnt mean to do that
5、.now, this semiquantitative plot looks at sickle cell disease basically from its clinical description up through the present time.and when I first got into sickle cell disease in the early 70s, late 60s while people did live to adulthood, the general life expectancy was perhaps in the late teens.and
6、 from that time on life expectancy has increased concomitant with or because of a number of factors some of which are highlighted here and one is the multi-center study of hydroxyurea.these sort of parallel graphs give you a sense that perhaps something happening.but it doesnt really help us quantif
7、y the evidence base and look at it in some detail.thats our challenge today.We have really got an all-star panel to help us do that.so the goals are what is the efficacy results from clinical studies of hydroxyurea treatment for patients who have sickle cell disease?In three groups, inpants, pre-ad
8、- infants, pre-adolescents and adolescent adults.What is effective of hydroxyurea for patient whose have it, the efficacy, toxicity ratio and what are barriers to hydroxyurea treatment for patient whose have the disease and what are potential solutions?I would like to place this in the context of wh
9、o makes the drug because sickle cell disease is perhaps the exemplar of orphan diseases.Its difficult to get industry involved in these projects.Hydroxyurea is an example of this.if our current trial baby hug proves a utility for the drug, who is going to make it?And ultimately who is going to pay f
10、or it?This is a societal problem but these are issues that those of us who care for sickle cell disease, certainly those of us who have relatives, friends, or happen to have sickle cell disease are intimately involved in these critical issues.What are the future resource needs?Of course, this is the
11、 raise on dent for the national institutes of health and we need the kind of guidance these types of panels can give us.so what were looking at now is I think a quantum step forward in making therapies for sickle cell disease and I use the plural because I dont think one will be sufficient given the
12、 protoian nature of this disease but we can look toward a day where sickle cell therapies are personalized, diagnosis is predictive in terms of complications and needed therapies, preemptive and ultimately looking for the empowerment of patients to take the best care of themselves, the best guidance
13、 that we can give them.so thank you for coming, I look forward to the results of this conference.and its my privilege to introduce Dr. Kramer from OMAR who will continue. Thank you, Dr. Peterson.good morning Im Barry Kramer Director of the NIH office of medical applications of research.Im pleased to
14、 welcome all of you to this NIH consensus development conference on hydroxyurea treatment for sickle cell disease.the conference is sponsored by the national heart lung and blood institute and the office of applications research.co-sponsored by the national human genome research institute, the natio
15、nal institute of child health and human development, the national institute of diabetes and digestive and kidney diseases, neurologic disorders and stroke and the NIH office of rare diseases.the centers for disease control and prevention and the health resource administration are also partners in th
16、is conference T. agency for healthcare research and quality has prepared a systematic literature review on several aspects of the conference.the contract for the preparation of this report was awarded to the Johns Hopkins university evidence based practice center.the draft of the systematic literature review as well as the draft panel report for this conference will be posted on Wednesday afternoon
