Home Infusions Fact Sheet for Ceredase
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Forty six Gauchers sufferers are currently (November 1993) receiving Ceredase enzyme replacement therapy in the UK with infusions ranging from three times a week to once a fortnight. Their Ceredase must be mixed with saline solution in either a special bag or syringe and be infused into a vein in an arm, hand or foot, through a butterfly needle over a period of time ranging from two hours to 10 minutes. One young patient has a Port-a-Cath. This is a permanent device surgically implanted under the skin into a vein in the chest; the infusion needle is inserted into the Port-a-Cath each time.
All patients start infusions in hospital as an outpatient under a doctor's supervision. Later some receive infusions at their GP's surgery. Some patients have learned to administer the infusion themselves with the help of another person (not necessarily medically qualified) and parents have learned to infuse their children. This means the infusions can be done at home.
Dr David Evans, who retired last year as Consultant Paediatric Haematologist at the Royal Manchester Children's Hospital, said at the Association's Conference last November that he encouraged his patients to have the infusions at home and that almost everyone could master the technique.
Eleven year old Katie, one of Dr Evans' patients, started Ceredase two years ago. For the past year, she has been infused every two weeks by her mother Sharon. Initially Sharon found the procedure nerve-wrecking and only did it because she thought her daughter would not get the treatment if she did not learn how to do it. Now she says 'It is no problem but I still get a bit nervous.'
Doctors advocate caution because the procedure for mixing Ceredase with saline solution, setting up the apparatus and finding a suitable vein can be complicated and the utmost care must be taken not to introduce any infection.
A few other patients in the UK like Sharon have successfully learned how to carry out infusions at home. Gill infuses her 10 year old son Jonathan three times a week. Jon started Ceredase in January 1992 and the following June, Gill started infusions at home. She says 'We were very fearful at first that I should do it. Jon wanted the doctor or nothing; he did not trust me. Jon and I spoke about it for two weeks before he came to terms with it. We decided to work as a team.
We agreed that I would support him in what he could do and if he could not manage, he would support me in what I had to do. At the beginning it was traumatic but it soon got a lot better. Nowadays it's still teamwork and just part of our lives.'
Gill adds 'In order to maintain the veins in his hand, we are now finding veins in his feet and I am trying to use a different arm or leg on a rota basis so that I can give every vein three weeks rest/recovery.'
A Great Advantage
Gill and her family live about 18 miles from the local hospital and Gill does not have a car. She also has two other young children to look after so carrying out the infusions at home is a great advantage.
Some patients prefer to continue infusions at hospital or at their GP although a few have been put off by waiting around for the doctor or nurse to arrive. Also a few patients have been upset by negative comments from the medical staff on the lines of 'so you are the one who is having this expensive treatment; do you think you (or it) is worth it?'.
Dr Ari Zimram of the Shaare-Zedek Medical Centre in Jerusalem recently
reported1 on 33 patients receiving home treatment in Israel, Holland and
California. The patients ranged from age 4 to 53, 17 female and 16 male.
Twentyfour had had Port-a-Caths or a similar device implanted; nine
received their infusions through a vein. They had received a total of
approximately 4,500 infusions over a period of between 13 to 82 weeks per
patient. All had initially received treatment in hospital and were trained
before starting treatment at home.
The report says 'Low-dose enzyme replacement therapy for Gaucher disease seems to be an ideal candidate for intravenous home therapy. The treatment, administered over a long period of time with high frequency, is very safe and practially free of significant complications. Most of the patients are clinically stable and do not require frequent examinations by a physician. In some of the patients, especially children, contact with other patients (with other disorders) may create additional fears.'
Susan reports 'I wanted to have my infusions at home and the hospital staff were very supportive in teaching me how to do it. My husband was also taught because I did need help. I found it impossible to set up the equipment, insert the needle into a vein and connect everything on my own. You just don't have enough hands. Also it is reassuring to have someone there. 'I found it difficult at first to locate a vein and insert the needle correctly. It's easy to pierce the vein and go through the other side. If so, you must try another vein. It's not dangerous but causes bruising. I gave up at one point and went back to the hospital but the doctors and nurse continued to encourage and advise me and after 3 weeks I could do it again at home.'
Doctors are also cautious because the risk of an allergic reaction to Ceredase cannot be ruled out, especially during the first year of treatment. This happened to Ross, a 17 year old boy receiving the high dose regime of Ceredase every two weeks. About six months into treatment, during one infusion at his home, he felt itchy and a rash appeared on his face and body. He also felt slightly unwell. His mother Pam says 'We stopped the infusion and immediately called in the doctor who fortunately has his surgery opposite our home. He came within a few minutes. Ross was given an antihistamine and cortisone. It was decided to give him antihistamine and cortisone before each infusion and continue the infusions at the hospital. He was alright on the subsequent infusion but the one after that, Ross had a similar reaction. That might have been because although his dose had been decreased, it was given only a week after the last one and it may have been just too much for him. Subsequently he has been receiving infusions every week without difficulty. He has now stopped taking the antihistamine and cortisone and has had no reactions. He would like to have the infusions at home again when the doctors agree to it.'
Because safety is a major consideration, Dr Zimran reviewed 61 patients being treated with Ceredase ( in hospital and at home). The report (details below) says 'Combining the minimal adverse effects with the large number of infusions (about 9,000 infusions to date) we suggest that the safety profile of low-dose Ceredase compares very favourably with other medications that are administered at home. The absence of severe infectious complications is remarkable in this population of Gaucher patients, which includes some who are relatively immuno-compromised be- cause of splenectomy or defective neutrophil function. Thus, we find that home intravenous enzyme replacement therapy for Gaucher disease is safe, feasible and well accepted by the patients and their families.'
Dr Richard Moscicki, Medical Director of Genzyme Biotherapeutics, says 'We believe that the risk of allergic reaction is primarily during the first 9 to 12 months and it is important that a medical person capable of treating an allergic reaction be on hand during this initial period of therapy. Patients who develop antibodies to Ceredase or who exhibited symptoms of allergic reactions during that time, are probably not good candidates for home therapy as their risk of problems may be higher than the average patient.
'I believe each patient must be considered individually. In some patients, the risks may be outweighed by the benefit of home infusion This should be decided with the physician.
'In patients currently on home infusion therapy, if symptoms of a reaction should occur, the infusion should be stopped immediately and medical advice or treatment sought.'
Prof Timothy Cox of Addenbrooke's Hospital, Cambridge concludes 'The risk of antibodies developing represents a point against unsupervised therapy but in some patients, any risks are outweighed by the benefits of home infusion. With advice, diabetics treat diabetes; in future, patients with Gauchers disease may similarly take responsibility for their replacement therapy.'
A fact sheet giving Guidelines on Home Infusions has been produced by the Gauchers Association and is available on request.
Home Treatment With Intravenous Enzyme Replacement Therapy for Gaucher Disease: An International Collaborative Study of 33 Patients, A Zimran, C E M Hollak, A Abrahamov, M H J van Oers, M Kelly and E Beutler, Blood, Vol 82 No 4 (August 15) 1993.
Home Infusions Fact Sheet for Ceredase
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