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Several speakers presented information about the continuing benefits of enzyme replacement therapy at the EWGGD meeting including reports from Belgium, the USA, Italy, Greece, Germany, Holland and Israel. In addition the practice of patients having their infusions of enzyme replacement therapy at home, was discussed by patients and doctors.
'The practice of patients receiving infusions of enzyme replacement therapy (Ceredase and Cerezyme) in their homes has been carried out successfully and safely for over 12 years,' explained Susan Lewis of the UK Gauchers Association and co-founder of the European Gaucher Alliance which includes 24 European patient groups.
'However the legal and/or medical authorities in some European countries disapprove of or explicitly forbid the practice.
'Home infusions, either self-administered, or infused with the help of a family member or friend, or assisted by a visiting nurse or doctor, has been possible in Holland, Israel and the United Kingdom since enzyme replacement therapy became available. Some patients have to travel long distances to receive their fortnightly or weekly infusions in hospital and would welcome the opportunity to incorporate their treatment into their weekly routine.'
'In the UK nearly 90% of patients with Gaucher disease are infused in their home. 60% infuse themselves or with help from a member of their family, 30% have a nurse coming to their home and 10% are infused in a hospital. Most children have a portacath fitted to enable easy access.
'Prescriptions for Cerezyme, water for infusion and saline bags are sent to a home care service which supplies the enzyme replacement therapy direct to the patient with the accompanying ancillaries such as infusion sets, syringes, needles etc. Refrigerated deliveries are usually made every two months. 'Training to mix the drug, set up the infusion and insert the needle is either provided by the patients' specialist hospital or by a nurse from the home care service.
'Patients must be happy with the procedure before considering infusing alone. A back-up service from the home care service, local doctor or specialist hospital is essential and mostly needed if a patient cannot site the needle. 'There is no law in the UK governing home infusion; it is the prescribing doctors responsibility. However the UK has 12 years experience of safe home infusion.
'A survey by one of the specialist centres in the UK at the Royal Free Hospital in London showed that nearly all their Gaucher patients preferred home-based therapy. Treatment in the home was reported as more comfortable, convenient, less stressful, more effective and had less impact on family life.'
A Personal Story
Ria Guijt, Vice-Chairman and a founder member of the Dutch Gaucher Association, spoke about the benefits of patients taking care of themselves. She described how she has carried out her infusions over the past 12 years and how she has coped with having a chronic disease. She said: 'It is important to have independence and self-infusions helps this.
This has improved my quality of life.' She described her relationship with her physicians as one of equality and said that knowledge and commun-ication led to patients continuing to carry out their infusions regularly.
Dr Carla Hollak, who runs the Gaucher Centre at the Academic Medical Center in Amsterdam, described a study on home treatment with her patients in Holland and those attending the Gaucher Centre at Addenbrooke's Hospital in the UK.
'Out of a total of 189 patients with Gaucher disease, 149 had received enzyme replacement therapy of whom 124 (83%) were on home therapy. Patients had been treated with Ceredase and now Cerezyme with doses ranging from 15 units per kilogram of body weight per month to 60 units kg/bw per month. Frequency of infusions ranged from three times a week to once every two weeks. Venous access was usually performed by a butterfly needle and in eight patients with a Portacath.
'An estimated total of 35,000 infusions was given over periods of 3 months to 13 years. The majority of patients was capable of performing the infusions without any help although ten patients in the UK had assistance with their infusions from a nurse.
'In the eight patients with a Portacath, related complications occurred six times (four infections and two position problems) in five patients.
'Patients using butterfly needles usually had no complaints but some reported skin rigidity and increased pain after several years at the place of insertion of the needle. This could sometimes be relieved by local application of an anaesthetic cream. No obliteration of veins ever occurred as a result of the frequent infusions. Only one patient had transient infusion related reactions.
'Patients were very satisfied with home therapy and only one patient decided to change from home therapy to a hospital setting for practical reasons.
'These results show that home therapy for Gaucher disease is very safe and feasible even after many years of treatment in adults as well as in children. Minimal requirements are professional instruction and easy access to support in case of problems with the infusion. The absence of adverse reactions further encourage the application of this method.'
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Source: Gauchers News May 2005.
Copyright © Gauchers Association 2005.