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Blood Cancer: The Forgotten Fifth

Blood Cancer and MDS, the forgotten cancers

Blood cancer patients are less likely to see their needs fully met than patients with the four most common cancers - breast, colorectal, lung and prostate.

The blood cancer community, via the umbrella group Blood Cancer Alliance, has launched an important new campaign, called the Forgotten Fifth, pointing out that blood cancer is just as dangerous as the 4 main cancers, but often not given the same attention, nor allocated the same resources.

The Forgotten Fifth campaign aims to achieve equal status for blood cancer patients to the other four most common cancer patients, enabling better treatment and faster diagnosis. 

MDS, the most forgotten

In addition, MDS is ‘the forgotten cancer’, as it is often not labelled as a form of cancer, and therefore not captured in data collected by the health authorities. As a support group, we have seen hundreds of cases where diagnosis of MDS was delayed. Delays means impaired quality of life for most patients, frequent infections, or, for a few of them a death sentence.

This campaign reinforces the evidence shown in our national MDS survey.

We are calling for attention to blood cancers, and MDS specifically.

"Neil had a stem cell transplant scheduled for early March. Then UCLH checked his ferritin levels. If you’re having regular transfusions your iron levels should be monitored, Neil’s weren’t. After a year of iron-rich blood transfusions these were found to be stratospheric, so the transplant was put on the back burner in order for these levels to be brought under control. Sadly, this wasn’t a treatment routinely offered by our local NHS Trust due to funding issues."

Read Neil's story >

What are we asking?

  • The Alliance is now calling for the NHS to treat blood cancer patients as equal to those with the four most common cancers in NHS policy making and decision making.
  • As a basic measure, the Alliance is asking that NHS England add data on blood cancer patients to the Cancer Data Dashboard it uses as a key information source for cancer strategies.

Do please point your MP to this campaign, to ensure that blood cancer stops being forgotten. Help us get there, please.

  1. Share your story with your MP. You can find out their email here.
  2. Share the images below on social media and with friends and family.

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Deferasirox (Exjade) access in MDS – Myelodysplastic Syndromes Overview of a UK wide postcode lottery – 2015 to 2021

Deferasirox is only licensed second line for the treatment of chronic iron overload despite real-world experience showing that deferasirox is better tolerated and its compliance is far superior than first line treatment.

This has badly affected many MDS patients as some pockets of the UK still will not grant funding to this drug, effectively creating a postcode lottery.

We request an end to this postcode lottery. In the absence of a NICE submission/decision, access should be aligned to the positive Scottish Medicines Consortium (SMC) decision.

Below you'll find excerpts of the document and explanations for:

  1. Patients (and families) affected by MDS, Myelodysplastic Syndromes and on regular blood transfusions
  2. MPs representing these patients in parliament
  3. Clinicians needing support to obtain access to deferasirox (Exjade) for their MDS patients 
  • MDS patients on regular transfusions?
  • Ferritin levels increasing?
  • Discussions about iron chelation?
  • Difficulties accessing the oral version of the iron chelation treatment?

 If you, or a patient you know are living in a region which STILL denies access to deferasirox (Exjade), the oral iron chelation treatment, please read on.

In the absence of a NICE decision, some pockets of the UK still will not grant funding to this drug, effectively creating a postcode lottery.

We first reported on this anomaly in 2015: https://mdspatientsupport.org.uk/mds-patients-achieve-access-to-vital-new-treatment-option/

2021 - and we still have reports of patients denied access, despite special circumstances:

  • Bournemouth area, a half-blind elderly MDS patient who could not handle needles, was denied Exjade. He since passed away.
  • Exeter area, an MDS patient, AND main carer for his wife with a degenerative condition, has been forced to use the cumbersome pump option to reduce his ferritin levels.
  • Cornwall, further reports from consultants, who have given up on applying for IFR’s Individual Funding Requests, as all are rejected.

Below, you will find:

Part 1 - The current national BSH Guidelines for the use of iron chelation in MDS patients

Part 2 – A selection of regional decisions regarding the funding of deferasirox (Exjade), in chronological order

Part 3 – The latest clinical paper supporting the use of deferasirox as the most beneficial treatment option in this group of MDS patients.

Request: As the national patient support group for MDS, we request an end to this postcode lottery.

In the absence of a NICE submission/decision, access should be aligned to the positive SMC decision.

It is simply not ethical to deny access to a drug based on a post-code, for patients who are clearly struggling with quality of life issues, or elderly, or suffering with additional sight or manual dexterity issues.

NOTE: Clinical commissioning groups will be subsumed into integrated care systems by the end of 2021, and will be statutorily dissolved into ICS in April 2022 if the government’s planned health bill goes ahead. 

Read more:

https://www.england.nhs.uk/integratedcare/what-is-integrated-care/

https://www.pharmacymagazine.co.uk/analysis/ccgs-to-be-subsumed-into-ics-before-end-of-2021

Part 1 - BSH Guidelines for MDS – June 2021 revision

  • Expert opinion is that deferasirox is the drug of choice for transfusion-related iron overload in patients with MDS

Iron chelation in MDS

Patients with MDS are at risk of developing iron overload from transfusion of red cells where iron build-up is inevitable (one unit of red blood cells delivers 200–250 mg iron), and there is also increased intestinal absorption of iron driven by ineffective erythropoiesis,48 mostly relevant to MDS-RS. Excessive iron ultimately leads to secondary end organ damage and cardiac disease remains the main non-leukaemic cause of death in MDS.49, 50

Iron overload is associated with adverse outcome in MDS

Retrospective studies have shown that OS is significantly shorter in transfusion-dependent MDS patients either through cardiac deaths, hepatic cirrhosis50, 51 or increased leukaemic progression.50 The European LeukemiaNet MDS Registry showed that the risk of death in transfusion-dependent patients with detectable labile plasma iron levels is independent of risk of disease progression.52 Iron overload also increases transplant-related mortality in haematopoietic stem cell transplantation (HSCT) in MDS patients53 and total transfusion burden implied a worse prognosis in a European Society for Blood and Marrow Transplantation (EBMT) study.54

Measuring iron loading

Routine estimations of iron loading can be made by serial monitoring of ferritin and tracking of red cell units transfused. However, there is little correlation between units transfused, or serum ferritin, and the degree of organ iron deposition.

Magnetic resonance imaging (MRI) for R2 (liver proton relaxation rate),55 or cardiac and liver T2* assessments56 can be used to help quantify hepatic and cardiac iron loading and its impact on organ function. 

Iron chelation can improve natural history

Effective iron chelation may improve haemopoiesis. The EPIC study57 and the GIMEMA group58 showed an International Working Group (IWG) erythroid response in 15–25% of patients although median response duration was only eight weeks in the EPIC study. Platelet and neutrophil responses were also reported.

Desferrioxamine has been shown to lower cardiac iron assessed by MRI measurements59 and deferasirox has been shown to improve alanine transaminase (ALT) levels.60 A German registry study showed that chelation therapy improved survival in almost 200 transfused lower-risk MDS patients,61 supported by prospective data from the EUMDS registry.62 Furthermore, it is now accepted that iron chelation prior to HSCT in congenital anaemia can improve transplant-related mortality.53 Although this is not yet proven to be the case in haematological neoplasms including MDS, a recent EBMT joint expert panel recommended chelation in patients who have received more than 20 units of blood prior to HSCT.63

Choice of iron chelator

Desferrioxamine remains the most efficient iron chelator available and is given subcutaneously in overnight infusions, which may decrease the labile iron pool. However, many patients find it uncomfortable and cumbersome, reporting QoL issues. Deferasirox and deferiprone are given orally and are generally well tolerated, although deferiprone is associated with agranulocytosis in around 4% of patients. Deferiprone should not be used routinely in patients with MDS, and only after careful consideration with a haematologist experienced in treating MDS. It should be undertaken with very careful monitoring (weekly blood counts), and should not be used where the baseline neutrophils are <1·5 × 109/l. Deferasirox is the only iron chelator currently licensed for use in MDS patients with proven reduction in labile iron and improved haemopoiesis in some patients.57, 64

Discussion of recommendations

Iron chelation in lower-risk MDS patients

It is recommended that all suitable lower-risk patients (IPSS low and intermediate-1; IPSS-R low and very low) should be considered for iron chelation therapy around the time they have received 20 units of red cells, or when the ferritin is more than 1 000 μg/l. Patients should have ferritin levels measured every 12 weeks and have ophthalmological and auditory examinations before commencing therapy and annually while on treatment. Iron chelation with deferasirox should be stopped if the ferritin falls below 500 μg/l and desferrioxamine should be stopped if the ferritin falls below 1 000 μg/l.

Iron chelation in higher-risk MDS patients

Patients who are considered suitable for HSCT should have iron levels monitored and iron chelation therapy given prior to transplant, if time allows. 

Drug recommendations

Deferasirox is only licensed second line (after desferrioxamine) for the treatment of chronic iron overload due to blood transfusions in patients with anaemia, such as MDS. However, real-world experience is that deferasirox is better tolerated, compliance is far superior and safety data are now mature. For these reasons, expert opinion is that deferasirox is the drug of choice for transfusion-related iron overload in patients with MDS. Desferrioxamine remains an option in those resistant to or intolerant of deferasirox. The two drugs may be combined in exceptional circumstances with heavy cardiac iron overload, but only under the supervision of a haematologist experienced in MDS treatment, although there are no data to support the combination.

There is no contraindication to the use of iron chelation in combination with other disease-modulating treatments such as lenalidomide or azacitidine.

Recommendations

  • All suitable lower-risk patients (IPSS low and intermediate-1; IPSS-R low and very low) should be considered for iron chelation therapy at the time they have received 20 units of red cells, or when the ferritin is more than 1 000 μg/l (1B).
  • Iron chelation therapy should be considered in patients prior to stem cell transplant, if time allows (2C).
  • Expert opinion is that deferasirox (although only licensed second line in MDS) is the drug of choice based on tolerability, compliance and mature safety data (2C).
  • Deferiprone is not routinely recommended in MDS (2C).
  • Iron chelation therapy with deferasirox should be stopped if the ferritin falls below 500 μg/l and desferrioxamine should be stopped if the ferritin falls below 1 000 μg/l (2C).

Part 2 - Selection of regional decisions regarding the funding of deferasirox (Exjade), in chronological order

SMC HTA – January 2017

  • Deferasirox accepted for use

“In the base case analysis, deferasirox was considered to dominate desferrioxamine ie was cheaper and more effective. Compared to desferrioxamine, deferasirox resulted in savings of -£6,783 and a QALY gain of 0.58.

Although deferasirox was associated with a relatively large incremental drug cost (£27,890) compared to desferrioxamine, this was offset by the lack of iron chelation therapy equipment costs; these were £28,847 for desferrioxamine. In addition, deferasirox resulted in fewer transfusion-related costs, treatment-related monitoring costs and complication costs.

At the PACE meeting, it was said that transfusion requirements appear to decrease in some MDS patient treated with deferasirox. Additionally, the use of an oral treatment may reduce risk of infection as patients no longer need to use needles.

Patient and clinician engagement (PACE)
A PACE meeting with patient group and clinical specialist representation was held to consider the added value of deferasirox, as an ultra-orphan medicine, in the context of treatments currently available in NHS Scotland.

The key points expressed by the group were:
· Around half of patients with MDS will develop severe anaemia and the resultant need for regular transfusions. The associated iron overload can cause iron accumulation in vital organs causing potentially life threatening problems such as liver, heart and renal dysfunction. Full compliance with chelation therapy is essential to minimise the impact of iron overload.
· Deferasirox is the only chelation treatment option available for those low to intermediate risk MDS patients who cannot tolerate administration of the desferrioxamine subcutaneous infusion or where this treatment is contra-indicated. It is of particular benefit for patients with sight or dexterity issues or those with a needle phobia.
· Existing chelation treatment involves subcutaneous infusion administered 5-7 days per week. Patients usually use the pump overnight or wear it underneath clothes for eight hours during the day, which may cause pain and discomfort. As such, current treatment is generally associated with an inferior quality of life for the patients.
· PACE participants highlighted their experience that compliance with the oral treatment is greater than that seen with the infusion and consequently deferasirox is a more effective chelation therapy option.

Deferasirox offers a vastly improved quality of life for patients with MDS and their families/carers by reducing the physical, psychological and emotional burden associated with desferrioxamine infusion therapy.”

https://www.scottishmedicines.org.uk/medicines-advice/deferasirox-exjade-resubmission-34707/

Thames Valley – July 2017

  • Subcut infusion not well tolerated, not advisable in MDS
  • Deferasirox ‘safe and effective

http://nssg.oxford-haematology.org.uk/myeloid/guidelines/ML-25-guidelines-for-oral-iron-chelation-in-mds.pdf

East of England data – June 2021

  • Routine funding not recommended
  • IFR’s or group approval via business case submission

https://medicines.blmkccg.nhs.uk/wp-content/uploads/2020/06/iron-chelators-for-iron-overload-in-myelodysplastic-syndromeMERGED.pdf

Herts Valley – Sept 2017

  • Not routinely commissioned, despite SMC cost-effectiveness acknowledged

https://hertsvalleysccg.nhs.uk/application/files/5515/3633/5366/FINAL_Iron_chelators_in_MDS_PAC_decision_document_201709_HMMC.pdf

Thames Valley – Sept 2018

  • Deferasirox to be used when subcut ICT contraindicated or inadequate

http://fundingrequests.nhsdigital.org.uk/wp-content/uploads/2018/12/26-09-18-Minutes-TVPC-Final.pdf

London data – Jan 2020

  • Clinical judgement should define choice of chelator

https://www.kingshealthpartners.org/assets/000/003/347/Pan_London_MDS_Guideline_Jan_2020_original.pdf

Greater Manchester – May 2020 (revised)

  • Deferasirox mentioned on its own

Iron Chelation
General recommendations are primarily based on studies in thalassemia, in which there is strong evidence for iron chelation (15). There is no doubt that heavily transfused MDS patients accumulate iron to potentially harmful effect.
There is evidence that iron chelation reduces iron overload in MDS, and even data suggesting reduced risk of leukaemic transformation. Iron chelation may also improve outcome after transplantation. However, there are currently no good quality controlled studies proving a survival or other long-term outcome advantage for iron chelation in MDS (16).

On balance, it is recommended that patients who satisfy the following criteria be considered for iron chelation:
• Where long-term transfusion therapy is likely (eg MDS-SLD, MDS-RS, 5q- patients; unless very high age or severe concomitant disease)
• In more advanced MDS (MDS-MLD, MDS-EB), iron chelation should be considered if life expectancy exceeds 2 years from time of iron overload (ferritin >1500 μg/l, or after 24 units of RBC).
• Candidates for allogeneic transplantation

Deferasirox (Exjade®) is an oral agent with now many years’ experience that is broadly well tolerated. Iron excretion occurs almost entirely in the faeces and is dose dependent. Caution is advised in renal and liver impairment. A film-coated preparation (FCT) is now available with better side effect profile. It is licenced but not NICE assessed (recommended by Scottish Medicines Consortium in first-line for Low/INT-1 risk MDS). Recommended starting dose is 10-30 mg/kg; starting at lower doses may improve tolerance; for FCT recommended dose is 7-21mg/kg.

https://gmcancerorguk.files.wordpress.com/2019/08/mds-guidelines-july-2019.pdf 

Somerset – March 2021

  • Deferasirox recommended for specialist prescribing only

https://www.somersetccg.nhs.uk/wp-content/uploads/2021/03/Somerset-Traffic-Light-System-39th-Edition-March-2021.pdf

Part 3 – Latest clinical paper supporting the use of deferasirox as the most beneficial treatment option in this group of MDS patients

 From Biology to Clinical Practice: Iron Chelation Therapy With Deferasirox – October 2021

Conclusions

In patients with MF and MDS, but also with AA and hemolytic anemias, the prognosis is not merely associated with individual factors such as age, comorbidities, or underlying condition, but also with complications associated with the degree of cytopenia present (severity of anemia, hemorrhages, and infections) and the treatment that they receive (blood transfusions, allogeneic transplants, etc.). In these patients, any therapeutic interventions should also face not only the management of comorbidities but also complications caused by the evolution of disease and adverse effects of primary treatments.

 The excess iron, associated with direct and indirect toxicity on the various tissues, leads to a worsening in age-related comorbidities, ultimately resulting in cumulative organ damage. Iron-induced toxicity also affects the evolution of the MDS or MF, by increasing cellular genomic instability and altering the bone marrow stroma through the oxidative stress, thus favoring progression to acute leukemia (15, 65).

The performance of a multidisciplinary evaluation of patients treated with deferasirox, at baseline and during follow-up, makes it possible to improve the therapeutic approach and optimally manage elderly patients with MF and MDS, consequently avoiding the need to discontinue specific therapy and reducing the risk of developing organ damage.”
https://www.frontiersin.org/articles/10.3389/fonc.2021.752192/full


Ask your MP to do more to support blood cancer patients

COVID-19: Requests to the government on behalf of the blood cancer community

The UK’s blood cancer patient community are clinically extremely vulnerable to COVID-19. Several studies showed that about 60% of blood cancer patients failed to develop effective antibodies for an immune response to the virus after their second jab.

The growing spread of the virus is causing concern and fear within blood cancer patients and it is imperative the Government does more to support them.

The Blood Cancer Alliance Policy Group, which includes ACLT, Anthony Nolan, Blood Cancer UK, CLL Support, CML Support, DKMS, Leukaemia Care, Leukaemia and Lymphoma Northern Ireland, Leukaemia Cancer Society, Leukaemia UK, Lymphoma Action, MDS UK, Myeloma UK, Race Against Blood Cancer and WMUK, have recently sent two open letters to Sajid Javid, Health Secretary, requesting more support for blood cancer patients.

Read the open letter here >

"For my family, the fact that I live with the uncertainty of how well vaccines work in patients like me, post stem cell transplant, is a great strain. Not being able to do more, go to more places and socialise more, for me this gives the feeling of guilt and many still don't understand why we have to be extra careful."

Read Rebecca's story >

What are we asking from government and MPs?

The Blood Cancer Alliance group (to which we belong), has sent a letter with five specific requests:

  1. Commitment to ensure the continuation of universal access to free lateral flow tests
  2. Communicating the need for immunocompromised patients to receive a third dose
  3. Reintroduction of the mandatory wearing of face masks
  4. Reintroduction of social distancing measures
  5. Communication on employment rights of the immunocompromised

YOU CAN REINFORCE THIS MESSAGE

  1. Read the full letter and forward it to your MP to inform them.

  2. Feel free to add details about your personal circumstances, specifically as a patient or family affected by MDS, Myelodysplastic Syndromes.

Letter sent by Blood Cancer Alliance to the Health Secretary Sajid Javid about the impact of coronavirus on the blood cancer community:

Click here to read the letter sent by Blood Cancer Alliance to the Health Secretary Sajid Javid about the impact of coronavirus on the blood cancer community

"I am being pressed by the DWP to make myself available for work again immediately or lose my benefit. They have said that the only thing that can take the pressure off is to be declared unfit for work by my GP, which is difficult as I am a watch and wait patient who is currently not physically unwell with my condition. But myself and my father are still extremely vulnerable to Covid."

Read Daniel" story >

We have also co-signed a second letter - sent by the Anthony Nolan organisation, requesting Sajid Javid to appoint a government lead for immunocompromised patients.

Click here to read and download the letter sent by Anthony Nolan, requesting the appointment of a government lead 

You can also forward this document to your MP, explaining in a few personal words why you believe that such a new role would be beneficial.

If you are writing to your MP:

Are you on regular transfusions? Have you been told your ferritin (iron) levels are increasing?
Have you had difficulties accessing the oral version of the iron chelation treatment (called Exjade)?
Some pockets of the UK still will not grant funding to this drug, effectively creating a postcode lottery.

If you are living in a region which STILL denies access to Exjade, please take this opportunity to explain your situation to your MP. Find all the information here >


What needs to be done for rare and less common cancers? Join our Do you C us campaign!

Rare and less common cancers account for nearly half of new cancer diagnoses

They affect people of all ages, genders, ethnicities and locations. However, the nature of these conditions means that people can often face a range of issues, such as recognising symptoms, obtaining a diagnosis, receiving the most suitable treatment or getting appropriate levels of support.

‘Do You C Us?’ aims to unite PfizerCancer52 and its member charities to raise awareness of the challenges facing people living with rare and less common cancers and highlight the need for change to address these.

Kes' Story: Watch the video

"I first had strange blood test results in my early twenties back in the 1980s but it wasn’t until many years later that my GP started to investigate my continuing strange results. It took them two years of prodding and poking me before they diagnosed my condition. MDS, a form of blood cancer, is usually a condition older men get and so at first they thought there was something wrong with my pituitary gland and I was given a brain scan. Finally, in 2000, I was sent to Kings, a centre of excellence, and they confirmed I had MDS.

I was put on ‘watch and wait’ where the doctors monitored my condition rather than taking any action at that point. It is a horrible term because it’s like you are waiting for something to happen. It’s like the sword of Damocles hanging over your head and it can fall at any time. Active monitoring is a much more positive way of putting it."

"Unfortunately, my condition progressed and I have suffered several bouts of sepsis, infection and flu. When all this started, I was a young fit football player. I never walked anywhere, instead I used to run. I rode a big motorbike. I worked full time, sang in a choir, volunteered with St John Ambulance, was a Venture Scout Leader as well as playing and training hard with my football. I played for Millwall Lionesses and went on to play for Charlton Ladies. After all these years of cancer being my constant companion, I now live a very different life.

In October 2016, I had a stem cell transplant. My little sister was my donor. I am a priest in the Church of England, but I haven’t been able to work since the transplant and am living with many ongoing consequences of my treatment and cancer, including needing to use a walking stick, mobility scooter and hearing aids.

I count my blessings that I am still breathing. I am thankful to have seen my daughter and grandchildren grow up. I take things on the chin and get on with it. And I try to help and support others with the condition. I’m not just the sum of my blood results."

What are we asking from government and MPs?

What needs to be done for rare and less common cancers?

  1. In the NHS Long Term Plan, the NHS committed to diagnosing 75% of all cancers at stage 1 or 2 by 2028. In order to meet this target, improvements are required across all cancers – including rare and less common cancers.
  2. Work should be undertaken to identify people 'missing' from the system, including people with rare and less common cancers. People must be reassured that it is safe to come forward for treatment.
  3. Cancer must remain a Government priority. Cancer services and the cancer workforce, including those for people with rare and less common cancers, should receive sufficient investment and funding for the future. This must be set out in the next Comprehensive Spending Review.

2nd Stem Cell Transplants Funding Re-instated Thanks to Your Support!

NHS England will fund 2nd stem cell transplants for patients whose blood cancer relapses

Since NHS England initially announced in December 2016 that it wouldn’t fund second transplants, more than 25,000 people joined our campaign to reverse the decision.

Thanks to every single person that signed the petitions, and wrote to their MPs. We all did it together.

Thanks to colleagues at Anthony Nolan, all other blood cancer charities, Emma Paine, Mark Tami MP, the APPG on Stem Cell Transplant, many MPs and clinicians, Sasha Jones and friends.

An extra-special thanks to the Hepburn family, who showed so much courage and campaigned selflessly after Gavin's death. We owe them a huge debt of gratitude.

Second Stem Cells Transplant Campaign

NHS Announcement in detail: routine funding for 2nd stem cell transplants for patients who relapse more than a year after their 1st transplant

On Friday 24 February NHS England announced that they will routinely fund second stem cell transplants for patients who relapse more than one year after their first transplant. This replaces the announcement in December 2016 that second stem cell transplants were ‘not currently affordable’.

Every year, a small number of patients with a blood cancer or blood disorder who have already received one stem cell transplant from a donor will unfortunately relapse (their disease will come back). For some of these patients, their doctor might recommend a second donor stem cell transplant. It is estimated that 16 to 20 people every year in England will need a second transplant because their blood cancer or blood disorder has relapsed.

This decision affects a small number of patients in England who:
• have received a first donor (allogenic) stem cell transplant;
• were in complete remission;
• subsequently relapsed more than 12 months after their first transplant; and
• their doctor now recommends a second stem cell transplant.

This decision does not affect patients who suffer graft failure (their first transplant fails) or patients who have received a first transplant using their own cells (autologous). This decision only affects patients in England; patients in other parts of the UK are not affected.

Why does this decision only apply to patients who relapsed more than 12 months after their first transplant?

The recommendation of clinical experts is that patients who relapse more than 12 months after their first transplant have the best chance of a successful second transplant. Unfortunately, there is weak evidence for the effectiveness of second transplants for patients who relapse within a year of their first donor transplant.

Read More: Blood and Marrow Transplantation

Join the Register!


MDS patients and carers attend parliamentary reception for Cancer52

On 20th April a group of seven MDS (Myelodysplastic Syndromes, a rare blood cancer) patients and carers went to Westminster and attended a parliamentary reception organised by Cancer52, a coalition of more than 80 charities representing rare and less common cancers. The reception was hosted and addressed by Chairman of the All-Party Parliamentary Group on Cancer (APPGC), John Baron MP.

The purpose of the reception was to raise awareness of these cancers and highlight the need for earlier diagnosis.

Rare and less common cancers together account for more than half of all UK cancer deaths – more than the ‘big four’ (breast, prostate, lung and bowel cancer together) – and on average have poorer earlier diagnosis rates.

These seven patients and carers were invited because they are members of MDS UK Patient Support Group, one of the member charities of Cancer52:

Prof Rodney Taylor is Chairman of MDS UK, an MDS patient himself, has experienced a number of MDS treatments (supportive care, transfusions, an MDS specific drug) over the course of 5-6 years. A year ago, when he stopped responding to a life extending drug azacitidine, he had to resort to a stem cell transplant at age 71. His wife and carer, Dr Janet Taylor accompanied him. They met with their MP Dr Tania Mathias.

Reverend Kes Grant, was diagnosed with MDS 16 years ago, at age 35. Following a gradual but serious deterioration of her health and quality of life, she too is now facing the prospect of a stem cell transplant. A treatment she is not looking forward to, as outcomes vary greatly and carry a good deal of uncertainty. Kes recently had to give up her work as a school chaplain, due to MDS, despite trying very hard to still work part-time. This has been devastating for her. Kes attended with her partner and carer Maggie Buckley. They met with their MP Matthew Pennycock.

Leanne Tilbrook, a very young MDS patients (typical age at diagnosis is 71yrs old), needs a transplant soon. She attended with her father Steve Tilbrook – who has been supporting MDS UK for a long time.
Neil Harrap very sadly lost his wife Julie to MDS two years ago, following complications of her stem cell transplant. He has now vowed to help raise awareness of the disease with MDS UK.

MDS UK members and their MP’s were briefed on the number of people diagnosed with rare and less common cancers in their area.

Jane Lyons, CEO of Cancer52, said, “It was fantastic to see so many people affected by rare and less common cancers at the reception raising awareness of these diseases with their MPs. For many the speed of diagnosis is critical as rare and less common cancers are more likely than the common cancers to be diagnosed late, often in A&E, and this has an impact on one year survival rates."

Rodney Taylor said, “It was a pleasure to join Cancer52 at their reception and discuss such an important issue. We need to be doing far better at diagnosing, referring and treating cancers earlier, particularly rare and less common cancers. Access to innovative treatments need to be much improved too.”

Dr Tania Mathias MP said “A privilege to meet patient-expert and constituent Prof Taylor: excellent action points concerning rare cancers.”

Useful links:
For more information about MDS UK please visit www.mdspatientsupport.org.uk
For more information about Cancer52 please visit: http://www.cancer52.org.uk/.

The All-Party Parliamentary Group on Cancer is the voice of the cancer community in Parliament, raising cancer at every opportunity, including on the Floor of the House and in meetings with Ministers and others.
The Cancer Strategy (Achieving world-class cancer outcomes: a strategy for England 2015-2020) was published in July 2015


MDS patients achieve access to vital new treatment option

January 2015 update
Equal and easy access to MDS drugs is crucial for all MDS patients. Here is an update on the MDS specific treatments available currently :

  • Azacitidine (Vidaza) – NICE and SMC recommended for routine NHS use – available nationwide in the UK
    Licensed for IPSS risk groups Intermediate 2 and high-risk MDS, AML and CMML.
    Must be given for at least 6 cycles – followed by a biopsy to check response to the drug.

  • Lenalidomide (Revlimid) – NICE and SMC recommended for routine use in NHS:
    Licensed for isolated del 5q MDS patients who are transfusion dependent.For patients with del 5q MDS plus 1 other chromosomal abnormality – lenalidomide is available through the Cancer Drug Fund.

    Lenalidomide is sometimes also used for MDS patients without del 5q – either as part of a trial, or via IFRs (Individual Funding Requests), and has shown good results for many patients.
    Please speak to your haematologist to find out if you may benefit from trying this drug out.

  • Deferasirox (Exjade) – this oral iron chelation should be available anywhere in the UK, but patients may need to try the traditional pump treatment deferoxamine first – to evaluate its ease of use.
    Let us know if you need/wish to be on this oral chelation drug but experience funding problems
    For MDS patients on regular blood transfusion and with a high ferritin level.

Always contact us should you have problems accessing any drugs recommended by your haematologist.  Local commissioning can vary unfortunately.

Always also involve your MP if you have any difficulties accessing medication to help with your MDS.

March 2013:
Azacitidine now seems to be easily available throughout the country – which is wonderful news.

Should anyone encounter any issues however – please contact us.
As a reminder – azacitidine must be given and funded for at least 4 cycles to establish whether the patient is responding to the drug (unless of course clinicians decide to stop the treatment for clinical reasons).
Some patients may not respond until 6 cycles – so discuss this with your physician.

Other issues our patients encounter:
Oral iron chelation drug: Some patients have reported access/funding problems for the drug Exjade (deferasirox).
Generally speaking, patients who need iron chelation – due to numerous blood transfusions – are first asked to use the Desferal (deferoxamine) pump or injections to reduce their ferritin levels.
If this method proves unsuitable for patients – or when patients do not tolerated the desferal pump, the oral chelator Exjade (deferasirox) can be prescribed and must be funded by the PCT (Primary Care Trust).
Each PCT will have issues their own funding guidelines about this – so it is worth checking those and discussing this with your haematologist or specialist nurse should you encounter problems.

Also – always inform your MP about any treatment access issues you encounter.  This is of special importance with the new commissioning methods in place.

And of course – send us details of the problems – so we can help – and highlight the issues.

August 2012:
Access to MDS treatment has improved immensely since the NICE approval of MDS drug azacitidine in February 2011.

See all details in article below.  Azacitidine is now available on the NHS in all hospitals – and funding is now no longer an issue.

Should you encounter any difficulties – please contact us.

We will shortly also include more news about treatment access.

London, 17 February 2011

The National Institute for Health and Clinical Excellence (NICE) announced today that Vidaza (azacitidine), the only licensed drug available specifically to treat myelodysplastic syndromes (MDS) – a range of life-threatening bone marrow disorders – will be available through the NHS. The MDS UK Patient Support Group, which has been in consultation with NICE during the approval process and campaigned on behalf and in conjunction with MDS patients across the UK, welcomes this announcement.

There are nearly 3,000 new cases of MDS in the UK each year and many people newly diagnosed with MDS have not heard of this disorder before. A person with MDS will suffer from chronic tiredness and weakness due to the often extremely low levels of haemoglobin, owing to a malfunction in the bone marrow in producing the correct quantity and quality of blood cells. This is debilitating in itself and often requires regular blood transfusions.

Azacitidine is an anticancer drug that is thought to work by re-establishing cells’ natural mechanisms to control abnormal growth. The final appraisal determination by NICE recommends the use of azacitidine for the treatment of MDS, chronic myelomonocytic leukaemia (CML) and acute myeloid leukaemia (AML) following a revision to the patient access scheme provided by Celgene (the manufacturers of azacitidine). Rodney Taylor, Deputy Chairman of MDS UK Patient Support Group said, “I am delighted to hear of NICE’s decision to recommend azacitidine for these patient groups which can benefit from this form of treatment. Having been on azacitidine myself, I know how effective it can be in promoting a good quality of life, giving independence from blood transfusions and allowing patients to lead a normal family life. Azacitidine is the only specific treatment for MDS that improves quality of life, prolongs survival and delays disease progression. It is great news that many more patients will now be able to benefit from it.”

While the announcement is good news for MDS patients in England and Wales, MDS UK Patient Support Group is concerned that access to this vital new treatment is still denied to MDS patients in Scotland, who are still waiting for a decision from The Scottish Medicines Consortium (SMC). We urge all concerned to apply both maximum effort and the highest priority to bring azacitidine to Scotland, in line with England and Wales. “Clearly I am delighted that NICE has approved azacitidine for use in England and Wales in conjunction with the associated patient access scheme,” said Dominic Culligan, Consultant Haematologist, Aberdeen Royal Infirmary, Scotland. “I hope that Celgene will re-submit azacitidine to the SMC as a matter of urgency, so that further consideration can be given to making this important treatment for high risk MDS and some patients with AML available in Scotland.”

It is a distressing reality that, during the protracted evaluation process attendant to the successful outcome of this appraisal, some MDS patients have progressed to AML, and some did not survive, in the absence of azacitidine. MDS UK Patient Support Group are aware of the complex financial constraints and cost effectiveness criteria attendant to the adoption of new drugs, and ask only that even more effort is applied in reaching speedier positive conclusions in critical, end of life situations such as MDS.

This recommendation for use of azacitidine was obtained after the manufacturer of the drug agreed a revised patient access scheme with the Department of Health in which azacitidine for the treatment of myelodysplastic syndromes, chronic myelomonocytic leukaemia and acute myeloid leukaemia would be available with a discount. This discount is commercial-in-confidence.

Implementation of this new NICE guideline:

When a NICE technology appraisal recommends use of a drug or treatment, or other technology, the NHS must usually provide funding and resources for it within 3 months of the guidance being published. Please do let us know if you experience any problems accessing azacitidine during this time, as the funding for the drug moves from the Cancer Drug Fund to a standard NHS drug recommended by NICE.


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