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Thalassaemia Patients

Volume 487: debated on Wednesday 4 February 2009

It is a pleasure to have secured the debate and to speak under your chairmanship, Mrs. Dean. I understand that this is the first time that a debate has taken place in the House on the important issue of the care of thalassaemia patients. Thalassaemia is the name of a group of serious genetic blood disorders that inhibit the normal production of haemoglobin—the part of red blood cells that supplies oxygen to the body.

In one sense, my interest in this issue is local, as the UK Thalassaemia Society is based in my constituency, in Southgate, but my interest goes wider than that, given that there are about 1,000 patients with thalassaemia in the UK requiring regular transfusion treatment for their condition and that more than 200,000 people in the UK carry the beta thalassaemia gene. The overwhelming majority of thalassaemia patients come from ethnic minority communities, particular those of Mediterranean and south Asian origin, and many of them already suffer significant health disadvantages. There are several patients in my constituency.

Thalassaemia remains a little-known condition, which is why I particularly welcome the opportunity to raise the issue in the House today. There are wide variations in treatment and care across primary care and hospital trusts, particularly in areas with low numbers of patients. I have secured the debate in order to highlight the variations in care and the health inequalities to which they contribute and to ask the Minister to consider what can be done to improve the lives of those with thalassaemia in the UK.

People with severe forms of thalassaemia, of which there are about 1,000, do not produce enough healthy, mature red blood cells, and if left untreated, the patient would be expected to die in the first 10 years of life. Thankfully, all pregnant women are now screened for the carrier state through the national screening programme. That is welcome because it makes the identification of patients and earlier medical intervention possible. Happily, the past few decades have seen welcome medical advances that mean that patients with thalassaemia can live long and productive lives if they receive the treatment and care they require.

Medical treatment takes the form of blood transfusions, which the patient typically receives every three to four weeks. One of the problems that thalassaemia patients face is a lack of flexibility from the NHS on receiving those life-saving blood transfusions. Patient choice is lauded loudly by the Government in their vision of the NHS, but the reality for patients with thalassaemia is that they have little or no choice of where or when they receive blood transfusions. That unnecessarily affects their education and employment prospects as they are forced to take frequent and often unpredictable leaves of absence from work or school to receive treatment.

Another issue in the treatment of thalassaemia patients, in which we see a wide postcode lottery of provision, is iron chelation therapy. As blood contains large amounts of iron that cannot be excreted from the body naturally, treatment is required to remove the excess iron that builds up as a result of regular blood transfusions. The process of removing excess iron is called iron chelation, and without it, iron deposits would eventually compromise the vital organs and lead to death, usually from cardiac complications.

The standard chelation therapy is for desferoxamine—Desferal—to be given as a subcutaneous infusion via a needle placed, often painfully, under the skin, usually on the abdomen. The treatment lasts for approximately 12 hours and is administered five to seven times a week. As is obvious from that description, the treatment can cause significant distress and impacts on aspects of daily life such as sleeping and the ability to socialise. I have spoken to several patients with thalassaemia within my constituency about the practical impact of that infusion. The drug is also a strong irritant and often causes pain and swelling at the injection sites. The parents of small children with thalassaemia have to administer those injections to their children, which as one can understand is very distressing to both parent and child. Although the treatment is life-saving, because of its painful and time-consuming nature, the rate of non-adherence to treatment is high. In fact, non-adherence to the treatment is the main cause of death among thalassaemic patients in the UK today.

Fortunately, there is hope of improvement in the administering of the treatment. Recent years have seen the introduction of oral chelating drugs that can transform a patient’s quality of life—the difference between taking the drug as a drink or tablets and having a needle under the skin for 12 hours out of every 24 need hardly be emphasised. The UK Thalassaemia Society wants those oral iron chelating drugs, when clinically indicated, to be available to all patients. Sadly, that is not the case due to the wider discrepancy of provision across the NHS. Therefore, many children and adults with thalassaemia must undergo hours of needless suffering every day.

The process of applying for oral iron chelating drugs in England is often through exceptional case procedures within individual primary care trusts. Those procedures are often time consuming and lead to unnecessary suffering for patients, many of whom are small children. The stress and trauma to parents and carers is immense, and the process requires additional time from doctors who often have other taxing requirements to make those detailed funding applications.

I appreciate that the Minister will say that she cannot direct that application process and that it is for individual PCTs to deal with them, but I hope that she will join me in encouraging PCTs in areas where the prevalence of thalassaemia is high, as in my own PCT in Enfield, to consider putting in place a standardised policy with regard to the provision of oral iron chelators to prevent such delays in funding and, crucially, to avoid the distress and frustration for patients and clinicians.

I have talked about treatment and would like to move on to the matter of curing thalassaemia, which I hope the Minster will agree should be a long-term aim of the health system. The most common method for curing thalassaemia is by bone marrow transplants. The success rate of that method varies considerably, depending on various factors. The alternative to bone marrow transplants is umbilical cord blood transfusion. The Minister will remember that the last time I secured a health-related debate I spoke about the merits of umbilical cord blood. I have insufficient time today to describe the details of umbilical cord blood and the hopes for it as a future treatment. Nevertheless, it is important to make the point that cord blood is a rich source of stem cells, which could be tapped into without the complications that accompany bone marrow transplants.

To date, the number of cord blood transfusions for thalassaemia has been sadly limited, but one small study placed the success rate at 79 per cent. Does the Minister agree that cord blood holds out the prospect of promising future treatments for not only thalassaemia, but other blood-related disorders? Will she ensure that the necessary cord blood banking is available to reach the ethnic communities who need it most? I look forward to her colleague, the Minister of State, Department of Health, the right hon. Member for Bristol, South (Dawn Primarolo), meeting with the all-party group on umbilical cord blood and adult stem cells, which was recently launched to discuss the outcome of the cord blood review.

So much for the future, but with regard to the care for thalassaemia patients now, access to psychological support and community and social care is also needed. There is a real need, and indeed call, for the Department of Health to work with local government, social services and education services to provide thalassaemia patients with a care package that addresses those holistically—I know that that term is often bandied about, but in this case it is truly needed—and deals with the social, psychological and educational needs of the patient alongside their medical treatment.

The issues that I have raised reflect a wider problem, as thalassaemia is in many respects a forgotten condition, and that is perhaps shown by the fact that this is the first time we have had a debate on thalassaemia. It is now forgotten within the health service, but hopefully not in the House. Although there are several committed specialists, to whom I pay tribute, dedicated to the treatment of thalassaemia and similar conditions that should be given a proper mention, such as sickle cell disease, there is a general lack of training and funding for posts in the haemoglobinopathies for both doctors and nurse specialists. Most haematologists deal mainly with white cell haematology, such as leukaemia and other malignancies, and red cell medicine is very much the poor relation when it comes to the research grants and training available.

Also reflecting the low profile of thalassaemia in the medical world is the fact that many GPs are not properly aware of the condition. More needs to be done to educate GPs on the causes and symptoms of thalassaemia to ensure that patients are passed on to appropriate specialists as soon as possible.

Perhaps one of the reasons why the condition has not received as much attention as other rare diseases is, sadly, the population it affects. As I have mentioned, thalassaemia overwhelmingly affects people of south Asian and Mediterranean origin.

Those communities often face social and economic disadvantages, and in the past many have struggled to find a voice within the health service. English is not the first language of many parents of children born with thalassaemia, which makes access difficult. Often families have to navigate the complications in the health and education systems. The lack of access to appropriate treatment and care for thalassaemia patients can contribute to the health and social inequalities that many patients and communities already face. This ethnic profile makes the spread of thalassaemia patients across the country uneven, which means that primary care trusts in certain areas, such as London, Birmingham and Manchester, face a disproportionate number of thalassaemia patients. That creates a number of problems with the burden on resources among PCTs with a high prevalence of thalassaemia. For low-prevalence PCTs, the cost is lessened, but patient access to specialist services is often patchy. They are required to travel long distances to access services.

To overcome problems of disparity in care in other conditions, particularly where patient numbers are relatively low and patients require a package of specialist care, the NHS has responded by developing managed clinical networks with the aim of improving cross-boundary working between health professionals and organisations in order to achieve the desired equitable provision of high-quality, clinically effective services. The UK Thalassaemia Society has produced guidelines on the standards of care for the treatment of thalassaemia patients, with the assistance of leading clinicians. Central to achieving those standards and improving outcomes is the establishment of clinical networks. Clinicians hope that if those clinical networks are established, the service for patients will improve. I understand that for the past two years the Government have been developing clinical networks for thalassaemia and sickle cell, but sadly there is no evidence that they are functioning and delivering the desired service. I would appreciate it, therefore, if the Minister outlined a time scale for the implementation of her Government’s plans for clinical networks.

Variations in treatment and care between PCTs and hospital trusts come particularly to the fore in the provision of certain drugs. This is a familiar theme that no doubt has been the subject of other Adjournment debates secured by other hon. Members. The case of thalassaemia is different and particularly pertinent because it affects the whole package of specialised services, the provision of which varies widely between different PCTs. There is not just a disparity in drug treatment; the whole service is often lacking. Patient numbers are low, but unlike many other conditions the ethnic profile of the people affected means that prevalence is not evenly spread through the population, and often patients must contend with the other inequalities that I have mentioned already.

Furthermore, mechanisms such as clinical networks and specialised commissioning, which exist in the NHS to deal with other conditions, as I have pointed out, requiring similar levels of specialised services for smaller numbers of patients, are undeveloped for thalassaemia and other haemoglobinopathies. Will the Minister explain why thalassaemia remains a poor relation to other genetic conditions, such as cystic fibrosis, in terms of funding for specialist drugs, the provision of services and general recognition? Will she explain when the Government will implement the clinical networks for thalassaemia to improve care and reduce variation in provision? What will the Government do to ensure that oral iron chelation treatment is made available to all patients?

The Minister will be aware of the forthcoming prescription charges review for patients with long-term conditions being undertaken by Professor Ian Gilmore, the president of the Royal College of Physicians. Thalassaemia patients commonly have as many as eight prescriptions per month. Patients with other chronic conditions, such as diabetes, are exempt from paying prescription charges. However, thalassaemia patients bear the financial burden of repeat prescriptions. Given the manifold challenges faced by thalassaemia patients, which I have only just touched on, does the Minister agree that it is unfair and inequitable that they have to pay these charges when patients with other chronic conditions are exempt?

I look forward to hearing the Minister’s response, either today or in correspondence, if further details arise. I pay tribute to the UK Thalassaemia Society not only for its assistance in the debate, but for the work that it does to assist patients who often fall beneath the radar of proper clinical provision and support across the realms of health, education and social services. I hope that I receive her support today.

It is a pleasure to serve under your chairmanship, Mrs. Dean. I thank the hon. Member for Enfield, Southgate (Mr. Burrowes) for raising this important debate. He takes a strong interest in the treatment and care of patients with haemoglobin disorders, such as thalassaemia major and sickle cell disease. As he will know, the prevalence of adult thalassaemia in his constituency is among the highest in the country. I also know that he has strong links with the UK Thalassaemia Society, which has its base in his constituency and provides such a positive voice for its members. It is doing sterling work in helping to drive up standards of care.

I welcome the opportunity to raise awareness of these long-term, chronic, debilitating conditions, the impact of which can be devastating not only for those affected but for their families and carers. An estimated 850 patients with thalassaemia major and about 12,500 to 15,000 patients with sickle cell disease live in England today. I would like to talk about the progress we have made in developing policy in this area and to attempt to address the questions raised and comments made. Should I fail to do so because of the time constraint, I would welcome the opportunity to meet the hon. Gentleman outside the Chamber at an appropriate time.

Our work builds on the success of the national programme for antenatal and newborn screening for sickle cell and thalassaemia, directed by Dr Allison Streetly. This programme is the first worldwide linked screening initiative. It is identifying some 350 affected babies a year and is estimated to save the lives of some 15 infants a year through early intervention. The progress has been achieved in partnership with key stakeholders, such as the UK Thalassaemia Society, the Sickle Cell Society, the UK Forum on Haemoglobin Disorders and other committed clinicians in the field. The Archbishop of York chairs the steering group, and it is a privilege to have such a champion supporting this area. I feel that his chairmanship will move us all faster in a very positive direction.

Key to the development of high-quality services for haemoglobin disorders is the formal designation of clinical networks for specialist care across the country. The hon. Gentleman asked what progress had been made; I can let him know very soon after the debate. The concept behind this is that every patient should have access to optimal specialist management and care, co-ordinated with routine care and provided conveniently close to home. We are working with specialised commissioning groups across the country to support the development of such networks, which will ensure that complications requiring specialist care are managed in the right place with the appropriate resources.

For example, strokes in children with sickle cell disease are common, and early diagnosis and immediate exchange blood transfusion can make the difference between a return to near normal and a long-term serious disability. Such early intervention is critical. Other clinical problems require highly specialist care, such as the management of potentially dangerous chest crises. The assessment and management of iron overload in those with thalassaemia is a rapidly changing area that, again, requires specialist interpretation and treatment guidance.

Our aim is to allow equitable access to comprehensive care across the country that is of a consistently high standard, and that addresses the concerns of patients and their families. This is a relevant time to mention the recently launched NHS constitution, which sets out our intention to ensure transparency and much greater consistency across all NHS commissioners in the way in which decisions are made about treatment and drug therapy. Such development underlines our commitment to addressing public concerns about the inequalities and unfairness in access to drugs.

As a first step to ensuring consistency of care in specialist provision and specialist commissioning engagement, we have succeeded in getting a national agreement—it has support from the Royal College of Pathology and the Royal College of Paediatrics and Child Health—on a specialist service definition for haemoglobin disorders, which covers thalassaemia major and sickle cell. That will help to ensure a consistent approach to service development across England, to encourage service planning and to underline the role of a specialised commissioner.

Currently, eight informal clinical networks are operating outside London and six within London, covering adjacent counties. Network meetings have identified some gaps in local services and those will be addressed via discussions with the local specialised commissioners. We have provided funding for the training of 13 additional staff—three doctors, six nurses and four clinical scientists. Once trained, the personnel will act as a focus for further educational development within the clinical networks. We are also working with the royal colleges to increase and enhance training in haemoglobin disorders. The hon. Gentleman specifically raised the issue of GP training and education in that role.

With the support of the UK Thalassaemia Society, the Sickle Cell Society, health professionals and other stakeholders, the National Haemoglobinopathy Registry was launched last October. It was supported by a report of the National Confidential Enquiry into Patient Outcome and Death on sickle cell and thalassaemia. The registry will allow us to collect details on patients with thalassaemia and sickle cell, and provide valuable information on patient numbers, complication rates and outcomes, which is a key factor underpinning the quality care agenda. We acknowledge that there is still a way to go in the development of service provision.

The hon. Gentleman raised the issue of cord blood. The NHS Cord Blood Bank has more than 12,000 units stored, some 40 per cent. of which is from the black and minority ethnic community. There are plans to increase the units to 20,000 by 2013. We have also asked NHS Blood and Transplant and the Anthony Nolan Trust to work together to develop plans on how we can meet the target more quickly.

On the question of bone marrow, it is important to put on the record that there are more than 100,000 bone marrow donors registered in Cyprus. That is 10 per cent. of the island’s population aged between 18 and 45, which makes it the fifth biggest registry in Europe and the 12th biggest in the world. The Cyprus registry is a member of the World Marrow Donor Association and is linked with more than 67 registers worldwide, including the UK. If a unit is needed, it is usually possible to source it from one of the network banks. The UK has access to such a network through the World Marrow Donor Association.

We acknowledge that there is more that we can do on service provision and the holistic approach that the hon. Gentleman outlined so well in his contribution. We have set the policy framework. With our support, the NHS can continue to build on the momentum for improvement. Our aim is to support high-quality, patient-centred clinical services for haemoglobin disorder. Key to that is the development of clinical networks of specialist care, and the concept that every patient should have access to optimal specialist management and care, as well as routine care provided conveniently close to home.

The treatment of thalassaemia is about the whole patient experience, which means annual reviews by specialists, routine—often monthly—blood transfusion and multiple prescriptions, including a requirement for oral iron chelation. We have recently carried out an informal review of services for haemoglobin disorders through which we hope to gain a better understanding of what is happening in clinical practice. That includes how the national clinical standards for care are impacting on services, and how current networks and commissioning arrangements are evolving. So far, reports have been very encouraging, with many examples of good and innovative practice and effective networking being noted. If any variations or issues arise, we will consider facilitating discussions between health professionals and specialist commissioners.

The Government are committed to phasing out prescription charges over the next few years in England for those with long-term conditions. Professor Ian Gilmore, president of the Royal College of Physicians, is undertaking a review to consider how the exemption should be phased in. The views of the public, clinicians and patient representative bodies, such as the UK Thalassaemia Society, have been sought.

A web survey hosted on the Department’s website gives anyone an opportunity to contribute their views until 27 February 2009. In line with Lord Darzi’s report, “High Quality Care for All”, our aim is to improve management of long-term conditions by offering more personalised packages of care closer to home, in order to allow people more choice and active involvement in decision making.

In conclusion, I thank the hon. Gentleman for initiating this debate and for identifying this important issue. I hope that he and other hon. Members will agree that considerable progress has been made in raising the standard of care for haemoglobin disorders throughout the country and ensuring more equitable access to excellent care. I accept that there is room for improvement, and remain committed to supporting the NHS in achieving that improvement for patients. To do that, we will be working in partnership with key voluntary organisations such as the UK Thalassaemia Society and the Sickle Cell Society.

One issue that goes wider than the medical and clinical perspective is the concern that care crosses into other areas, such as psychological and social care support. That is very important, given that the communities suffering from thalassaemia are often disadvantaged. Is there a contact or a programme in which there is cross-departmental consideration of the need to provide particular support? Although such support needs to take place at a local level, it may need some national direction, as well.

I appreciate the hon. Gentleman reminding me that he raised the point about the need for psychological and social support for the very young through to the troubled teenager and the entire family. As a former nurse who had patients with thalassaemia in my care, I know only too well how necessary that approach is. In line with Lord Darzi’s review on quality of care, personalised care is paramount to me, my Department and all Ministers. I look forward to working with all the societies to achieve that.