rdic questionnaire Adrenal insufficiency www.addison.dk www.hypofyse.dk
A THANK YOU TO OUR MEMBERS The rdic questionnaire that Addison Foreningen [The Danish Addison Association] and Hypofysenetværket [The Danish Pituitary Network] took part in is now officially concluded. We would like to take this opportunity to thank everybody who took the time to answer the questionnaire. It has been a supremely unique opportunity to collect information on adrenal insufficiency. All this data can provide us and others with a far better insight into the situation of patients with Addison disease and pituitary disease in the rdic countries. Addison Foreningen i Danmark Hypofysenetværket INTRODUCTION The survey was conducted in Denmark, rway and Sweden at the end of 2012. Everybody who had an email address registered with the five rdic patient support associations was sent a link to a page on the Internet. Morbus Addison Forening in rway, Addison Foreningen in Denmark, Hypofysenetværket in Denmark, Stödföreningen Hypofysis and Svenska Addisonföreningen in Sweden. The members were extremely interested in answering the survey questions, beating many records. One hour after the questionnaire had been uploaded on the Internet, almost 50% had responded. The survey was sent out to 1,268 members of which 106 were either incorrectly addressed or could not receive the questionnaire. A total of 711 members responded within a month, giving a response rate of 62%. Denmark came out on top with a response rate of 75%, followed by rway with 58% and Sweden with 53%. The response rates for all countries were higher than expected for comparable surveys. The patient support associations own the data which are stored on an external web server. All data are completely anonymous, and it is not possible to link individuals to the answers. Viropharma (a part of Shire) has assisted the associations not only in preparing the questionnaire but also with the analyses and setting up the material. 0
Adrenal insufficiency If untreated, adrenal insufficiency is a potentially fatal condition. Satisfactory treatment with medication is available; however, there is no cure. The disease is caused by either impaired or complete loss of adrenal cortex function or pituitary function. The most common cause of Addison's disease is the formation of antibodies against the adrenal glands, which is a so-called autoimmune disease. This autoimmune condition is the result of incorrect coding in the immune system that causes antibodies to attack the body's own cells. The antibodies affect production of cortisol, resulting in too little formation of this life-essential hormone. In primary adrenal insufficiency, another hormone is often also lacking. This is called aldosterone and is involved in the regulation of the salt and water balance. However, cortisol is the most important hormone that is replaced. It is essential for metabolism and replenishing our energy depots. rmally, cortisol is produced in the adrenal cortex in response to a signal from the pituitary. Therefore, disease not only in the adrenals but also in the pituitary can result in a lack of cortisol. In order to survive, life-long treatment with cortisol is necessary. The body cannot store cortisol, however, and the amount needed during the course of a day varies. Hydrocortisone, which is the synthetic form of cortisol, is normally used to replace the lost cortisol. The treatment objective in adrenal insufficiency is to provide the body with a sufficient dose of cortisone 1 to meet the body's needs but not so much that there are side effects. If the adrenal glands are the cause of the adrenal insufficiency this is called Addison's disease or Primary Addison's. Secondary adrenal insufficiency is due to a disease of the pituitary that results in loss of the signals from the pituitary that tell the adrenal cortex to produce cortisol; it is called secondary Addison's because it gives the same symptoms as primary Addison's. Conditions other than Addison's that are treated with large amounts of cortisone over a long period of time can cause drug-induced Addison's disease. This is because the cortisone stops the adrenals' production of cortisol. Usually, the adrenal glands return to normal, but sometimes it can take a long time. The body normally produces cortisol continuously. This production is increased in response to stress or infection, for example. An inability to increase this production or inadequate function of the adrenal cortex can result in an Addisonian crisis. An Addisonian crisis is a potentially life-threatening condition that occurs when the cortisol drops to a dangerous level. It requires the immediate administration of hydrocortisone, SoluCortef, which is often given intravenously. The treatment of adrenal insufficiency has not changed noticeably over the last 60 years. Reports have shown that patients with adrenal insufficiency have a reduced level of functionality and reduced quality of life. Moreover, they have an increased risk of developing cardiac disease and osteoporosis, and they have a decreased life expectancy. The results of this interesting study are presented on the following pages. 1 In this report, cortisone is used as a general term for all steroid-containing preparations. 1
The survey There was a quick and high response rate from all countries, but Denmark came out on top with regard to the number of responses. There was a large age difference among the responders, of which 58% had primary Addison s and 26% had pituitary insufficiency. More than twice as many women as men responded. Which country do you live in? Sweden rway Denmark I am... Woman Man 2
Which type of adrenal cortex insufficiency do you have? Primary (Addison s) Pituitary insufficiency CAH (AGS) Other Do you have any other autoimmune disease(s)? Hypothyroidism Diabetes Type 1 Vitamin B12 deficiency Vitiligo Autoimmune ovarian inflammation Other How old are you? Over 80 71-80 61-70 51-60 41-50 31-40 21-30 10-20 Below 10 3
Are you being treated at a University hospital or a Regional hospital? Regional hospital 10 University hospital Treatment of adrenal insufficiency The treatment involves replacing the hormones that the adrenal glands should normally have produced. Cortisol is replaced with hydrocortisone, which is a synthetic form of our natural cortisol. In rare cases, it is replaced with other corticosteroids. The most common treatment consists of 20-30 mg hydrocortisone 2 or 3 times a day. This dosing schedule is intended to replicate the pattern of cortisol excretion in a healthy person. Sometimes, if the patient does not produce enough aldosterone, it can also be necessary to give a mineral corticoid, Florinef (generally only in primary Addison s). The main contribution of this hormone is to normalise the salt-fluid balance and the blood pressure. Treatment of adrenal insufficiency aims at achieving a physiological cortisol level not only under normal conditions but also during stress when extra cortisol is needed. It is recommended to give the lowest dose of hydrocortisone that maintains the balance between the risk of developing an Addison crisis and the risks associated with too much cortisol. Both too much and too little cortisol is harmful to health. Forgotten doses, or too little cortisol during illness, can result in an Addison crisis with high fever, vomiting, diarrhoea, and a drop in blood pressure. Very high doses over a long period of time can result in obesity, high blood pressure, diabetes and osteoporosis. It is difficult to tell from blood tests, etc. whether the treatment is adequate/optimal. This means that the treating doctor must make a very careful clinical assessment. In this survey, almost everybody in Denmark and Sweden was treated with hydrocortisone. In contrast, almost everybody in rway was treated with cortisone acetate. A small majority, about 45%, take their medicine twice a day, and about 40% take their medicine 3 times a day. Almost 70% of the patients were satisfied or very satisfied with their treatment. 4
Which type of cortisone do you take? Dose Cortisone acetate 3 times a day Other Once a day Twice a day Sweden Hydrocortisone rway Denmark What is your normal daily dose of cortisone? >40 mg 40 mg 37.5 mg 35 mg 30 mg 25 mg 20 mg 15 mg 12.5 mg 10 mg 5 mg Other dose 5
Do you take any other medicines?* Diuretics Sex hormones Growth hormone Thyroxine Other * Only patients who answered that they had pituitary insufficiency Do you take Florinef? Yes 6
How satisfied are you with your cortisone treatment? Very dissatisfied Dissatisfied Neither nor Satisfied Very satisfied What would you like to change about your current treatment? thing/everything s fine Other Better follow-up/contact with hospitals/the same doctor Better information and advice That the hospital sees the whole patient That healthcare personnel listen/are more interested Tablets with lower strength/easier to divide Hydrocortisone t need medicine Avoid Addison crisis t awake at night Less tired/more energy Lower dose without tiredness Fewer side effects Better effect/dose without side effects Better adjusted to my needs Sustained release pill/once daily pill Pump/plaster/other than tablets Effect when you wake up 7
If you could change something in connection with your cortisone treatment, what would it be? 5 mg tablets. Difficult to divide 20 mg into many parts Auto-dose (surgically inserted) That I could take the morning dose without having to wake up :) That they develop instruments so that we get a more accurate dosage!!! The same doctor The tablets work well. Another medicine that does not give so many side effects Better contact with the endocrinologist. It would be really good if I could get down to 20-25 mg, and that I could have fun without getting ill afterwards - or not be able to meet up for that reason. It is very tiresome and very tedious. Reduce the side effects such as stomach ache, weight increase, hair loss, blisters and rash. 1 pill per day. I immediately feel unwell if I am an hour late. Get a more precise reaction. Dose as needed. One dose for the entire 24 hours. Have a tendency to forget the afternoon dose. Better follow-up from doctors and specialists I feel very much alone. Plasters instead of tablets or an injection once a week or once a month Tablet that you take at night and which doesn t begin to work before the morning and which then gives less and less effect as the day passes! Sustained release/long-acting/slow release/one dose instead of two/being able to take medicine just once a day. 8
If you could change something in connection with your cortisone treatment, what would it be? Difficult to answer, as I don t know what is the normal condition with this diagnosis. From next year, I want to switch to hydrocortisone. Cortisone pump. Have been given a cortisone pump - it is heaven!!!!!!! Impact on quality of life Several studies have shown that patients with adrenal insufficiency experience a reduced level of functionality and reduced quality of life. One German study shows that the quality of life is lower the higher the dose of hydrocortisone. Another study conducted in Germany with patients with adrenal insufficiency revealed that the most common symptoms are tiredness and lack of energy (84% of patients with primary adrenal insufficiency and 64% of those with secondary adrenal insufficiency). Several patients with adrenal insufficiency have reported a loss of appetite, nausea and vomiting. The non-specific symptoms associated with the disease means that it can take a long time before a diagnosis is made. Even though in our study more than 70% were satisfied with their treatment, more than 88% believe that the disease affects their quality of life, and 41% have problems with depression. Most people think that the most difficult aspect is that that it is an invisible disease; that you are not able to do what you did before; and that you could experience an Addison crisis. More than half are worried about long-term effects. 9
Does your disease (adrenal insufficiency) affect your quality of life? Yes, a lot Yes, quite a lot Yes, a little, not at all Do you have problems with depression? Yes 10
What is your general state of health? (1 = very poor, 10 = very good) Median 6.6 What do you think is the biggest problem with your disease? Other That I am not able to do what I did before That I can have an Addison crisis That it is invisible 11
Do you have problems with your weight?, not at all Yes, a little Yes, rather much Yes, a lot Do you have problems with your teeth?, no problems Yes, a little Yes, a lot Are you concerned about long-term effects of your treatment? Yes, a little Yes, a lot 12
Other offical studies have shown that many patients with adrenal insufficiency have had to make changes to their life. Many also experience that the disease affects their capacity to work or go to school. This study has also shown similar results. 35% of the respondents consider that they are healthy enough to work full time. Many have had to cut back on their level of activity, both at work and in sports and social activities. Do you feel well enough to work? Yes, full-time Yes, part-time, I am on sick leave, I have taken early retirement I am unemployed Which activities have you had to change because of your Addison s/pituitary insufficiency Work Social activities Physical activities Family life ne Other 13
In the last 3 months, have you been absent from school/work because of something related to your illness (adrenal insufficiency)? Yes If yes, how many days have you been absent? More than 16 days 11-15 6-10 1-5 14
An Addison crisis is very serious and requires immediate treatment at a hospital s emergency department with administration of cortisol directly into the blood stream. The condition can occur in various phases of the disease. It can be the first symptom of Addison s disease, but an Addison crisis can also occur in connection with an accident or an infection. Diarrhoea and vomiting can also trigger an Addison crisis. It is important that all patients with Addison s disease carry an information card in their wallet which states that immediate adminstration of cortisone is life-essential in cases of accident or serious disease. In order to avoid a life-threatening lack of cortisol it is important that the treating doctor provides clear information to the patient on appropriate measures in case of fever or other illness. Verbal and written information should be given at the onset of the disease and repeated at routine follow-up appointments. Most people who answered the questionnaire carry either the cortisol card or a medallion. About 40% were not satisfied with the information they received when they were diagnosed, but 70% were satisfied with the information they are given when visiting the doctor and when they contact the hospital or doctor between appointments. About half of the patients had been prescribed Solu- Cortef. A quarter of those who responded have had to go to the Emergency department at least once during the last year because of an Addison crisis. Of those who had an Addison crisis, about 7% received a cortisone injection in the ambulance, and 53% had to wait longer than 1 hour before being given the cortisone injection at the hospital. 15% had to wait more than 4 hours before they received an injection. 57% believe that nurses do not have adequate knowledge of Addison s disease. Which symptoms do you experience when your cortisol level is low? Headache Feeling unwell Pain in the joints Stomach ache Dizziness Other 15
Do you yourself notice when your cortisol level is low? Always Sometimes/Occass ionally Rarely Never What do you do when you discover your cortisol level is low? Increase the dose Wait and rest thing Other 16
In which of the following situations do you increase your cortisone dose? Fever In hot weather Mental stress Physical stress Infection Other Do you carry a medallion/medical alert or emergency card? Yes, the card Yes, the medallion Yes, both the card and medallion Did you receive adequate information when you were diagnosed? Yes 17
Do you feel you are given adequate information when you ask questions during or in between appointments? Yes Has your doctor told you about Solu-Cortef? Yes Have you been given a prescription for Solu-Cortef? Yes 18
Have you been denied a prescription for Solu-Cortef? Yes Have you received training in how to give an injection? Yes If you have Solu-Cortef: Have you or your close family given you an injection? Yourself, yes Yourself, no Close family, yes Close family, no 19
How many times within the last year have you been to the Emergency department with an Addison crisis? ne Once Less than 5 times 5 or more times What was the most frequent cause of your Addison crisis? Gastroenteritis Influenza Other infection Other If you have been transported by ambulance in an Addison crisis, were you given a cortisone injection in the ambulance? Yes 20
How quickly were you given a cortisone injection at the hospital? Within 0-1 hour Within 1-2 hours Within 2-4 hours It took more than 4 hours Do you think that the nursing staff know a lot about Addison s disease? Yes Which information/service would you like your patient association to provide? Information about the disease Information about the treatment Medical alert/crisis card Opportunity to meet others with the same disease Telephone service with the opportunity to ask questions Access to a mentor/helper Other I do not need more information 21
Which information/service would you like your patient association to provide? Online chat Positive stories Emergency card in English Opportunity to buy a medallion That one should not only focus on the disease itself, but what we can do to improve things, e.g. sport. Feel that there is a lot of focus on everything being negative; it doesn t have to be. Dietary advice Telephone service A FACEBOOK page International research and groups; opportunity for follow-up abroad, not only in little Denmark Am satisfied with the info we are given. It is up to oneself how you use it. Thanks. Would you like to have received/receive further information about your disease or your medicine? Better information on hydrocortisone That I should increase the dose if I have a fever Better information about the medicine and its effects and side effects; information on where I should go if I have an illness and need help. Information on what I can do if I my cortisol gets too low, and information on the possibility of getting emergency medicine and if I can keep it at home. Information on precautions when travelling, etc. 22