TIF MAGAZINE APRIL 2010 http://www.thalassaemia.org.cy/

Transition of patients with thalassaemia and sickle cell disease from paediatric to adult medicine

Based on a presentation given at the 2nd Pan-European Conference on Haemoglobinopathies

By Dr Holger Cario, Department.of Paediatrics and Adolescent Medicine, University Hospital Ulm, Germany

More than 30 years ago, the majority of chronically ill children did not survive into adulthood. With the advent of innovative technologies and medical advances, many chronically ill adolescents – including patients with haemoglobinopathies – cross the threshold into adulthood every year. Successful transition of chronically ill adolescents from paediatric to adult medical care continues to be a challenging process. Often, the transitioning experience consists of an abrupt transfer, which leaves the youth unprepared for the movement to adult health care. Because adolescents and young adults with haemoglobinopathy are at risk of developing severe medical complications, obstacles that impede transition to adult care must be identified to promote an uninterrupted transfer.

The transition of patients with haemoglobinopathies from paediatric to adult health care is a complex and multisided process. The consideration of the following “sides”, or aspects, of this process is of particular importance for its successful management and finally, for an optimal continuous medical care for patients with thalassaemia major (TM) or sickle cell disease (SCD).

1.Medical Treatment

This comprises all aspects of intrinsic medical treatment, i.e. treatment with regular medical drugs (for instance analgesics and hydroxycarbamide in SCD, chelation therapy and drugs for secondary complications in TM), emergency treatment, and transfusion in TM or exchange transfusion/erythrocytapheresis in SCD. It is of particular importance for the patients’ adherence and compliance, and thus for treatment success, that medical care for a specific disorder in both paediatric and adult health care is based on common standards. Depending on national or regional conditions, the degree of implementation of such common standards is currently varying.

2. Education and Training of medical staff

This aspect is very closely related to the first. Apart from the central medical care provider, the paediatric or adult haematologist, many other persons involved in the medical care need adequate training to meet the needs of these complex disorders. This group comprises subspecialists such as cardiologists and endocrinologists, nurses, educators, psychologists, social workers and others. A very close collaboration of these caregivers is essential for a successful treatment.

In countries where haemoglobinopathies affect primarily patients with a background of migration, and where migration occurred rather recently, patients only started to enter adulthood, and to look for treatment by adult health care services, during the last ten or fifteen years. Thus, adult health care providers inevitably had limited experience in the management of these rare disorders. In order to pass over knowledge and experience, not only is it important to have a close collaboration between different subspecialists and health care professionals within a medical care unit, but also a very close collaboration between paediatric and adult haematologists.

3. Health Care Setting

The health care setting varies from country to country, and sometimes even between regions within a country. For many patients, the transition from paediatric to adult care will be associated with transition from a rather familiar ambience within an in-patient clinic to an out-patient clinic focussed on the care for self-managing individuals. Even patients treated in paediatric out-patient clinics will feel such fundamental change, since they got used to the ambience and to individual health care providers, particularly nurses and other supporting staff, knowing them for their whole life.Therefore, transition to adult health care must include the careful preparation of patients to these changes to avoid frustration and retraction.

4. Adolescence and Puberty

At the time of transition, patients usually are at the end of their puberty. Classic adolescent rebellion can be an added problem for patients with chronic illness. Actually, puberty certainly represents one of the most vulnerable periods in the life of each patient with haemoglobinopathy. Adolescents with thalassaemia begin to challenge the need to continue chelation treatment, even after the introduction of alternative medical drugs without the physical burden of deferoxamine. They stop adhering. Patients become aware of physical problems caused by their disease or its treatment. And puberty itself can be disturbed by hypogonadism caused by iron overload representing a major burden for affected patients. All these problems have to be considered within the transition process, which should follow a standardised plan but has to be realised in an individualised way.

In addition to hypogonadism, other complications of iron overload, such as cardiac disease or diabetes mellitus, may occur in the same period. In these cases it has to be weighed very carefully where (in paediatric or adult medicine) treatment of such complications should be initiated. The ideal way, of course, would be the collaborative approach.

5. Social Activities

At the end of adolescence, there are other issues besides the transition from paediatric to adult medical care that predominate the life of young adult patients with TM and SCD. These include professional training, jobhunting, love, family planning and others. In some countries, personal responsibilities for health insurance and the payment for medical care are associated with additional problems for the patient. These aspects should be included in a transition programme. Both paediatric and adult health care workers have to develop possibilities to provide continuous support concerning these questions before, during, and after the transition.

6. The Families

The role of the family in the medical care of patients with haemoglobinopathies, and the transition process, varies depending on national, regional, and ethnic conditions, but everywhere it is of great importance. During infancy and childhood, the families and the parents in particular guarantee adequate medical treatment of their children. In adult medicine, the individual patient with self-care responsibility is the immediate partner of the physician or subspecialist. With regard to this aspect, an abrupt change from paediatric to adult care would certainly have an adverse effect on further treatment, adherence and compliance.

The influence of the parents may decrease during puberty. In the positive case, patients develop self-efficacy and self-care responsibility. In the negative case, the lack of compliance and adherence result in a long period of inadequate treatment, which can have even fatal consequences. Thus, patients’ education concerning their diagnosis, treatment, and preventive measures, but also psychological and social support, are of central importance within this period. Parents need encouragement to handle this situation, to acknowledge the increasing personal responsibility of their children, and to settle into their new role as accompanying partner and supporter. The transition of patients with haemoglobinopathies and other chronic disorders is not a one-off event, but a long-lasting process. Transition programmes must be developed in collaboration between paediatric and adult health care providers. They should consider the above-mentioned aspects, and therefore include the availability of common treatment standards, the collaboration within a multidisciplinary team and between paediatric and adult haematologists, the training of health care professionals, the education of the patient, the integration of the patient into decision-making, and the help for the families to strengthen their ability to support the patient in and after the transition process. The administrative handling of the transfer from paediatric to adult medical care should be planned in advance, including the transfer of relevant records. The timing of transfer should be flexible, depending on the patient’s developmental and social background. Thus, based on a standardised programme, the transition process should be tailored for each individual patient.

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Theme: “THE INFORMED PATIENT: KNOWLEDGE IS POWER”

“The improvement of understanding is for two ends: first, our own increase of knowledge; secondly, to enable us to deliver that knowledge to others.”
- John Locke (1632–1704)

This year’s theme for 8th May has two aspects. The first is the patient’s knowledge and information. At TIF we believe that an informed patient is an empowered patient: the more you know, the better you can manage your condition and your life. The more you know, the better you can interact with your doctors and other carers. And when a lot of knowledgeable, strong individuals come together and join efforts, their collective voice will be even stronger.

Knowledge is power
How often have doctors, nurses and other health professionals said about their patients, particularly those with chronic conditions: “My patients understand their disease better than I do!” The experience and knowledge held by the patient can greatly benefit patient care and quality of life, something which gradually is becoming more recognised by the medical world.

Today’s knowledgeable patients can make decisions regarding their treatment and work in partnership with their doctors, rather than simply being passive recipients of instructions. But in order to do this, the patient needs the power that comes through education and information. This has long been, and still is, one of TIF’s main educational aims.

Sharing the knowledge
The second aspect, as so wisely expressed by the great Enlightenment philosopher, is sharing the knowledge. Patients working together with other patients, parents, volunteers and health professionals – creating associations, getting involved in health policy formulation at a local, then national and even international level. We can really make a difference, not just for ourselves, but for others, too!

The idea of the 2010 slogan is to encourage every patient to become educated, but also to recognise her/his already-existing knowledge, to use it and share it.

© 2008 Thalassaemia International Federation

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D E F E R I P R O N E

How many times a day should Deferiprone be taken?
Deferiprone is taken in three divided doses, morning, midday and evening.

Is there an interaction with other medicines?
There have been no reports of drug interactions with other prescribed medicines. There is, however, a potential interaction with aluminium-based based antacids, since Deferiprone binds other metallic ions as well as iron. The concurrent use of Vitamin C has not been studied but it is potentially toxic and should be avoided.

Why is it necessary to do a weekly blood count?
In a proportion of patients there is fall in the number of polymorphonuclear cells, the white blood cells responsible for the defence of the body against bacterial infections. This may be severe fall to below 500/cmm known as agranulocytosis: this occurs in about 1.2% of patients taking Deferiprone and it may lead to a serious infection. A less severe fall in the neutrophil count may also occur, between 500–1500/cmm, known as neutropenia. This may be indicate that agranulocytosis is imminent and may also predispose to infection. It is important to detect this fall in white cell count early since interruption of the drug will restore the neutrophil count and avoid any complications. This is the reason for the weekly blood counts.

Is Deferiprone used in pregnancy and breastfeeding?
In animal studies it was demonstrated that this drug can cause damage to the early embryo (teratogenic). This has not been recorded in humans but because of the experience in animals it is unwise to use the drug in both pregnancy and breastfeeding. In fact it is strongly advised to avoid pregnancy while on this medication.

Does Deferiprone predispose to Yersinia enterocolitica infection?
Experimental evidence from the Pasteur Institute of Paris, both in vitro and in a mouse model, indicate that this drug does not promote Yersinia growth and virulence. However, in a Canadian review of clinical infections in thalassaemia patients over 15years, one patient was receiving Deferiprone, while 2 patients were not on any chelating agent and the rest of their cases (11) were taking Deferoxamine. For this reason it can be concluded that Deferiprone is less likely to predispose to Yersinia. In numerous studies on Deferiprone in thalassaemia, cases of Yersinia infection are not mentioned.

Does Deferiprone protect the heart more than other agents?
Comparisons have been made mainly with Deferoxamine. Recent publications confirm that Deferiprone is superior as a cardio-protective agent than Deferoxamine, probably because as a smaller molecule, it can remove intracellular iron from myocardial cells.

Can Deferiprone be used in children?
Formal trials of safety and efficacy have not been conducted in children under the age of 10 years.

© 2008 Thalassaemia International Federation

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