research study for children with salt wasting congenital adrenal hyperplasia

Conditions: Adrenal Hyperplasia, Congenital

Summary: the purpose of this study is to develop a more physiological approach to the management of children and adolescents with salt wasting congenital adrenal hyperplasia. we will administer the glucocorticosteroid via insulin infusion pump to see whether this treatment will improve the serum hormone concentrations.

the adrenal gland is a small organ of the body. it produces very important chemicals called hormones. one of these hormones, cortisol (the stress hormone) helps the body fight diseases. the other hormone is the aldosterone helps to maintain the normal amount of salt and water in the body. the third type of hormones are the androgens or male hormones, which cause some of the changes during puberty, like the growth of body hair and pimples. the salt wasting congenital adrenal hyperplasia or cah disease is a disease of the adrenal gland. patients with this disease cannot make cortisol or the aldosterone. as a result, their body cannot fight diseases and cannot keep normal amounts of salt and water in the body. at the same time, the gland makes too much of the male hormones, which is bad for the body because too much male hormone slows down growth, increases the growth of body hair, and causes pimples and abnormal period in girls. patients with this disease have to take medications every day. however, the treatment does not work very well, because usually the patients do not have the right amount of hormone in their body. usually the body gets too much hormone right after taking the pills. a couple of hours later the body has too little of the hormones, because in the meantime the body gets rid of the medication.the healthy adrenal gland makes the hormones throughout the day in different amounts. the patients with this disease take the medication only a couple of times a day. they take the florinef tablet once a day and the cortisol tablet two or three times a day. the treatment that we use today by mouth cannot copy the natural hormone productions of the adrenal gland. because of this it does not make much of a difference in the patient's life. we would like to improve the treatment and find out the effect of a new treatment. in this study we will try to imitate the body's normal hormone production and will give the medication via an insulin pump to see if this treatment method will decrease the male hormones in the blood. this study will help us to develop a new and better treatment for children and adolescents.

Eligibility criteria:
inclusion criteria: * children with salt wasting cah otherwise healthy without other chronic disease * age: between 3 and 18 years of age * body weight 23 kg (50 lbs) or above * hemoglobin equal to or higher than 12 g/dl before the study * supportive family environment exclusion criteria: * age less than 3 or older than 18 years at the time of study * other chronic disease * hemoglobin less than 12 g/dl * non-supportive family * allergy to local anesthetics criteria for study termination: if the subject's parents are unable to manage/operate the pump, the subject will be withdrawn from the study.

Keywords: salt wasting congenital adrenal hyperplasia subcutaneous hydrocortisone