News Archives | Page 16 Of 39 | MDS UK Patient Support Group

this must be archive.php

Call for review of COVID vaccine timings for blood cancer patients

Posted on 14/03/2021 at 10:17 am.

The Blood Cancer Alliance (BCA) is a group of fifteen UK charities working to tackle the issues blood cancer patients face, to improve the experience and outcomes of all those living with blood cancer.

The BCA has asked the Joint Committee on Vaccination and Immunisation to formally examine whether reducing the time between doses of the Pfizer vaccine for this patient group, to the 21 days that the vaccine manufacturer recommends, will give greater protection for this group of immunocompromised patients.

The BCA is also calling on the Government to establish investment in relevant clinical research to understand vaccine efficacy in blood cancer patients more fully, both after a single dose, and after full vaccination is complete.

The BCA is also asking that shielding guidance be extended while work is undertaken to understand how effective the vaccine is for the immunocompromised in the longer term. The clinically extremely vulnerable remain eligible for furlough for the duration of the scheme and we urge employers to continue furloughing staff who cannot work safely from home, even after the end of shielding guidance.

Zack Pemberton-Whiteley, Chair of the Blood Cancer Alliance, said:

"The emerging evidence published by Kings College and the Crick Institute today will be incredibly worrying for immunocompromised blood cancer patients, many of whom have already been shielding for 12 months now.

We strongly urge the JCVI to consider this evidence and undertake a formal review as to whether the time between doses for these vulnerable patients should be shortened.

The vaccines are safe, and we urge everyone to take up the offer of being vaccinated. However, in order to give adequate information to blood cancer patients, the Government must also invest in research to understand what level of protection the vaccines will afford the immunocompromised in the longer term in comparison to the general population.

Until we have this information, it is important that the Government extends its shielding guidance and support. We must also ensure blood cancer patients are not pressured to return to work before they can make an informed decision about the level of protection vaccination affords them.”

Coronavirus & MDS: Advice & implications for blood cancer patients – Updated on 25/02/2021

Posted on 26/02/2021 at 8:00 pm.

Written by Manuela Rotstein

Shielding guidance for those who are clinically extremely vulnerable from COVID-19 - from the Department of Health and Social Care.

See below for the latest advice to patients by the Government. Updated on 13/01/2021

This guidance is for everyone in England who has been identified as clinically extremely vulnerable. If you are in this group, you will previously have received a letter from the NHS or from your GP telling you this. You may have been advised to shield in the past.

This shielding guidance applies to clinically extremely vulnerable individuals only. Others living in a household with someone who is clinically extremely vulnerable are not advised to follow this guidance. They should instead follow the general advice and regulations set out in the national lockdown guidance that came into effect on 5 January 2021.

The clinically extremely vulnerable will get priority access to vaccination against COVID-19 before the general population and in line with the priority ordering set by the Joint Committee on Vaccination and Immunisation (JCVI). You will be contacted again by the NHS with more information on when and how you will be invited to get the vaccine.

The vaccine is likely to make an important contribution towards protecting you from COVID-19. Clinically extremely vulnerable people are expected to receive a vaccination against COVID-19 before the general population. Your local NHS will ensure that you can receive the vaccine as safely as possible, as well as any care and support needed. Even if you have had both doses of the vaccine, you should continue to follow this shielding advice, until further notice as we continue to assess the impact of vaccination among all groups. The people you live with should continue to follow the public health rules and guidance as long as they are in place, including if you have received the vaccine and also if they have received the vaccine.

LEARN MORE ABOUT THE COVID-19 VACCINE AND MDS

If you are particularly anxious – here are a few words from the counsellor at King’s College Hospital:

General guidance doesn’t replace your own assessment of your individual circumstances or risks.

Speak with your healthcare team to collaborate on a plan for what is reasonable for you.

If loved ones want you to socialise more, you may feel a sense of peer pressure, or even guilt, if you’re unable to or don’t feel ready to.

You are not alone in this. Such feelings are natural.

Try to actively adopt a compassionate view towards yourself by acknowledging that you are doing your best in a difficult situation outside of your control.

Unfortunately, being vulnerable sometimes leads to others forgetting your sense of agency & autonomy in managing your health condition. It may be helpful to reclaim this.

It’s okay to be assertive. Gently and lovingly remind others that while they should do what they think is right for them, you will do the same for yourself.

Anxiety about uncertainty is normal.

You may have thoughts about how long this will last.

Focus on the short term. Allow some flexibility in your mind that you’ll respond to future changes when they arise.

Surabhi Chaturvedi, Psychotherapist in Haemato-Oncology, King’s College Hospital

What has changed

COVID-19 case numbers are now very high and rising rapidly across the country, driven by the new variant of COVID-19.

Everyone in England, including those who are clinically extremely vulnerable, is required to follow the new national lockdown guidance, which has been set out by the government and applies to the whole population. These restrictions:

Require people to stay at home, except for specific purposes
Prevent people gathering with those they do not live with, except for specific purposes.
Close certain businesses and venues.
Support children and young people to learn remotely until March 8th, except for vulnerable children and the children of critical workers who may still attend school.

The new information below includes additional guidance for clinically extremely vulnerable people, to help protect you from COVID-19. We will also write to you with a version of this guidance.

These new formal shielding measures will apply across the whole of England until at least 31 March.

Socialising

The new national lockdown guidance, which applies to everyone, means that you must not leave or be outside of your home and garden, except for limited purposes which are set out in that guidance.

We are advising clinically extremely vulnerable people to stay at home as much as possible.

You can still go outside for exercise or to attend health appointments, but try to keep all contact with others outside of your household to a minimum, and avoid busy areas.

You can still meet with your support bubble, but you cannot meet others you do not live with unless they are part of your support bubble. Outdoors, you can meet one person from another household for exercise. This is part of the wider national regulations that apply to everyone.

Try to stay 2 metres away from other people within your household , especially if they display symptoms of the virus or have been advised to self-isolate.

Work

You are strongly advised to work from home because the risk of exposure to the virus in your area may currently be higher. If you cannot work from home, then you should not attend work.

You may want to speak to your employer about taking on an alternative role or change your working patterns temporarily to enable you to work from home where possible.

If you need support to work at home you can apply for Access to Work. Access to Work will provide support for the disability-related extra costs of working that are beyond standard reasonable adjustments an employer must provide.

If you cannot make alternative arrangements, your employer may be able to furlough you under the Coronavirus Job Retention Scheme, which has been extended until the end of April 2021. You should have a conversation with your employer about whether this is possible.

As you are being advised not to attend work, you may be eligible for Statutory Sick Pay (SSP) or Employment Support Allowance (ESA). The formal shielding letter you receive will act as evidence for your employer and the Department of Work and Pensions that you are advised to shield and may be eligible for SSP or ESA. Members of the household who are not clinically extremely vulnerable should continue to attend work if they are unable to work from home, in line with the wider rules set out in the national lockdown guidance.

Education settings

As our knowledge of COVID-19 has grown, we now know that very few children and young people are at highest risk of severe illness due to the virus. Doctors have therefore been reviewing all children and young people who were initially identified as clinically extremely vulnerable to confirm whether they are still thought to be at highest risk.

If you have already discussed this with your child’s doctors and they have confirmed your child is still considered clinically extremely vulnerable, your child should follow this shielding advice.

Under the current national lockdown, children will learn remotely until February half term, except for vulnerable children and the children of critical workers who may still attend school.

Clinically extremely vulnerable children and young people should not attend school or other educational settings, because the risk of exposure to the virus in the community is now very high. Your school or college will make appropriate arrangements for you to be able to continue your education at home.

Travel

You are advised to stay at home as much as possible and not to travel unless essential (for example, to attend health appointments).

Shopping

You are advised not to go to the shops. Use online shopping if you can, or ask others to collect and deliver shopping for you (friends and family, local volunteers or NHS Volunteer Responders).

You can register to request priority access to supermarket delivery slots, if you do not have someone you can rely on to go shopping for you. If you already have priority access to a supermarket delivery slot, that will continue – you do not need to do anything further. When registering you will be asked for your NHS number. You can find it on any letter the NHS has sent you, or on a prescription.

Registering on the site just gives you priority. It does not mean you’ll definitely get a delivery slot. If you want access to supermarket deliveries, you will also need to set up an account with at least one supermarket and book slots yourself.

If you need other forms of help, including support to register for priority access to supermarket delivery slots, you should contact your local council directly. Find out how your local council can help.

Medicines

You are strongly advised not to go to a pharmacy.

In the first instance, you should ask if any friends, family or volunteers can collect medicines for you.

If friends and family are not able to collect your medicines for you, and you and/or the pharmacy are unable to arrange for a volunteer, then you will be eligible for free medicines delivery. Please contact your pharmacy to inform them that you are clinically extremely vulnerable and need your medicines delivered, and they will arrange this free of charge.

Accessing care and support

You can still receive informal care at home from people within your support bubble.

You can still receive care at home from professional social care and medical professionals.

We urge you to continue using the NHS and other health providers for your existing health conditions and any new health concerns.

You can access a range of NHS services from home, including ordering repeat prescriptions or contacting your health professional through an online consultation. To find out more visit Health at home or download the NHS App. If you have an urgent medical need, call NHS 111 or, for a medical emergency, dial 999.

If you do need to receive care in person, you can. Your local NHS services are well prepared and will put in measures to keep you safe.

It is also really important to look after your mental health. Go to the Every Mind Matters website for advice and practical steps that you can take to support your wellbeing and manage your mental health during this pandemic. If you or someone you care for are experiencing a mental health crisis, we urge you to make contact with a local health professional immediately.

Any carers or visitors who support you with your everyday needs can continue to visit. This also applies for those of a child or young person in your care. They should follow social distancing guidance where close or personal contact is not required.

You should continue to access support from local charities and organisations, as well as NHS Volunteer Responders. As well as helping with shopping and medicines delivery, NHS Volunteer Responders can help with a regular, friendly phone call, and transport to and from health appointments. Call 0808 196 3646 between 8am and 8pm to arrange support or visit NHS Volunteer Responders website.

Registering for additional support

If you need additional help to follow this guidance, your local council may be able to help. If you are advised to shield you will be able to register yourself or someone else to:

request priority access to a supermarket delivery slot (if you have already got priority access that will continue – you do not need to do anything further)
tell your council if you need support to follow shielding guidance, especially if you are unable to arrange this yourself or with the help of friends, family or other support networks
make sure your details, such as your address, are up to date

When registering you will be asked for your NHS number. You can find it on any letter the NHS has sent you, or on a prescription. It is helpful if you register even if you do not have any support needs at this time. You can log in and update your needs if circumstances change at any time.

Part 2. Implications for blood cancer patients

Learn more on the NHS Website: Who's at higher risk from coronavirus?

If I have blood cancer, am I more at risk?

For many people, their body will be able to fight off coronavirus like other viruses and colds. However, coronavirus can have more serious effects on anyone who has a long-term health condition or a weakened immune system, including some people with cancer. This includes:

People having chemotherapy, or who’ve had chemotherapy in the last 3 months.
People having immunotherapy or other antibody treatments for cancer.
People having targeted cancer treatments that can affect the immune system, such as protein kinase inhibitors.
People who’ve had a bone marrow or stem cell transplant in the last 6 months, or who are still taking immunosuppression drugs.
People with some types of blood cancer which affect the immune system, such as MDS, chronic leukaemia, lymphoma or myeloma, even if no treatment is being given.

Part 3. Implications for our MDS UK local patient group meetings

It doesn't seem likely that we shall be able to resume face-to-face meetings for a while yet.

We still want to maintain our services to you, and provide you with all the regular news about MDS, as well as giving you an opportunity to connect to one another virtually. We know how important patient to patient contact is for everyone.

We've held many online/virtual Zoom meetings. Feedback has been very positive, and patients who had not been able to attend face-to-face meetings for various reasons were able to participate. Hence, we are now offering online meetings open to all members and carers.

Whatever happens during and after the COVID-19 pandemic, we aim to maintain some virtual meetings, as it has attracted new members, who had not attending face to face meetings previously.

We are also providing nationwide meetings with particular topics which you may find of interest. Please let us know if you have any topic in mind.

Many patients have already embraced the video meeting call technology Zoom (or something similar) in order to stay in touch with family members during the lockdown. If you haven't, please contact us.

If you are a first time user of video call – please don’t worry. The first 15 minutes of each meeting will be set aside to sort out any technical issues.

There might not be tests available at any given time.

The NHS is experiencing very high demand for testing at the moment.

More tests, both for drive-through sites and home delivery, might be made available later. Please visit this page often.

If your condition gets worse, or you do not get better after 7 days, use the NHS 111 online coronavirus service. Only call 111 if you cannot get help online. For a medical emergency, dial 999.

Symptoms of coronavirus

The symptoms of coronavirus are:

a cough
a high temperature
shortness of breath

These are also symptoms of other common conditions such as a regular cold, virus or flu. Having these symptoms does not necessarily mean you have coronavirus.

If you feel unwell

If you are getting a test because you have symptoms, you and anyone you live with must stay at home (self-isolate) until you get your result.

Anyone in your support bubble must also self-isolate until you get your result.

If you’ve been in contact with someone with coronavirus, or been to a high risk area in the last 14 days, call 111 or use 111 online (see the NHS list of high risk areas).

Getting help in Scotland, Wales or Northern Ireland

Scotland: call your GP surgery or call 111 if your surgery is not open
Wales: call 111
Northern Ireland: call 111

Check if you need medical help

NHS 111 has an online coronavirus service that can tell you if you need medical help and advise you what to do. Use this service if:

you think you might have coronavirus
in the last 14 days you've been to a country or area with a high risk of coronavirus
you've been in close contact with someone with coronavirus

Use the 111 coronavirus service

Learn more about the situation right now: GOV.UK: information on coronavirus and the situation in the UK

Immune response to COVID-19 reduced in patients with blood cancers

Posted on 09/02/2021 at 10:48 am.

Written by Manuela Rotstein

Patients with blood cancers such as leukaemia vary in their immune response to COVID-19 and can struggle to clear infection for very many weeks, according to new research published in Cancer Cell. By contrast, most patients with solid tumours, even those with advanced cancer, can mount an effective and lasting immune defence.

Researchers from King’s College London and the Francis Crick Institute have been studying the way SARS-CoV-2 impacts the immune system through the COVID-IP project and have already identified immune signatures that are associated with severe disease and hospitalisation. Their latest collaboration, funded by Cancer Research UK, called SOAP, specifically looks at people with cancer to see if their immune system responds differently to the coronavirus and if there are any long-term effects.

Until now, doctors have had very little information about how COVID-19 might impact patients with cancer and many have faced delays with treatment. So it’s vital to build a picture of cancer-specific infection and immunity that could help inform treatment strategies. This is all the more relevant given the UK government’s renewed advice on shielding.

The team led by Dr Sheeba Irshad, in collaboration with Professor Adrian Hayday, Dr Piers Patten, and staff at Guy’s and St Thomas’ and King’s College Hospitals, analysed the blood of 76 cancer patients - 41 who had COVID-19 and 35 who had not been exposed to the virus. 23 of the people with cancer had solid tumours, and 18 had blood cancers.

When they compared samples with the immune signatures from COVID-19 patients without cancer, they saw that patients with solid tumours had a similar immune response to those without cancer, regardless of cancer stage or if they were undergoing treatment. These patients were still able to mount an effective and lasting antibody response.

Sheeba Irshad, SOAP project lead at King’s College London, said:

“Whilst we need to maintain caution, our study provides some confidence and reassurance to care providers that many of our patients with solid cancers will mount a good immune response against the virus, develop antibodies that last and hopefully resume their cancer treatment as soon as possible. These conclusions imply that many patients despite being on immunosuppressive therapies will respond satisfactorily to COVID-19 vaccines.

For patients with blood cancers, especially those with B-cell malignancies, this may not hold true even in the era of COVID-19 vaccines. Our work suggests that they may be susceptible to persistent infection despite developing antibodies, so the next [and ongoing] stage of our study will focus on monitoring their response to the vaccines. At present the best way to protect them may be to vaccinate all the carers to achieve herd immunity in the clinic.”

COVID-IP project lead Adrian Hayday, who heads the Crick’s Immunosurveillance Laboratory and is Professor of Immunobiology at King’s College London, said: “This study is a prime example of a fast-tracked collaboration between basic scientists and clinical teams that in a short space of time revealed several unexpected findings that are highly informative and very helpful clinically.

“The COVID-IP study was made possible because of technologies originally used to track the immune response in patients receiving cancer immunotherapies and we’ve been able to adapt this work to further understand the impact of SARS-CoV-2.”

The next phase of the SOAP study will be monitoring the immune responses of cancer patients to the COVID-19 vaccine.

David P. Steensma, MD, looks for a novel therapy to treat TP53-Mutant MDS

Posted on 20/01/2021 at 7:05 am.

Written by Manuela Rotstein

TP53 gene mutations or chromosome 17 loss which results in TP53 deletion is present in up to 20% of MDS patients. Therapies for these patients have been relatively unsuccessful in delaying the progression of the disease.

Emerging treatments for patients with MDS are frequently based on cytogenetics, i.e. the study of the chromosomes. For this reason it's becoming more important for haematologists to check for mutations, and for patients to know those results.

Ask your haematologist for the results of your molecular test, to find out if you have any mutations, and which ones they are. This information also helps when reading news about future MDS treatments.

In this excellent, very clear interview with USA MDS expert Prof David Steensma, he lists some positive news for MDS patients with the TP53 mutation. It involves a combination of drugs, eprenetapopt (APR-246) and azacitidine. Combining treatments seems increasingly promising generally in MDS.

Click here to read the full article on Onclive

OncLive: Historically, how have TP53 mutations been targeted, and how effective have these therapeutic strategies been?

Steensma: TP53-mutant disease is one of the biggest unmet needs in MDS. When patients with TP53 mutations receive allogeneic hematopoietic cell transplantation, they tend to do more poorly than those without TP53 mutations, including frequent early relapses. We know that TP53 is enriched in therapy-related MDS, so people who have had treatment for some other type of non-myeloid cancer many years later may develop MDS as a result of the treatment, and when that happens, it is often TP53-mutant disease.

TP53 mutations are associated with short survival, especially when both copies of TP53 are lost. Clinically, the TP53-mutant population is a very high risk group that is often treated with one of the DNA hypomethylating agents, either azacitidine or decitabine, and those lead to a high rate of response. But the response duration is often pretty short, with a median of 8 to 9 months, so the overall survival for these patients tends to be less than 1 year.

How do recent data support the use of this agent in the treatment of TP53-mutant MDS?

One of the exciting things is that on phase 1 and 2 trial eprenetapopt (APR-246) was associated with quite a high overall and complete response rate, certainly higher than we would typically expect to see when looking at historical controls of azacitidine monotherapy. Anytime patients are enrolled in uncontrolled clinical trials, though, we wonder, “Are they just healthier than the general population? Were they destined to do well otherwise?” So the uncontrolled data are difficult to interpret but are encouraging. Still, in MDS, we’ve been burned many times by phase 2 trials in which interventions looked like they had a high response rate, were then taken into phase 3, and showed no improvement.

Some examples are the combination of azacitidine with histone deacetylase inhibitors, such as vorinostat [Zolinza] or entinostat. More than 4 randomized trials of azacitidine with deacetylase inhibitors showed no benefit compared to azacitidine monotherapy, just more adverse events with the combination. Azacitidine plus lenalidomide [Revlimid] also looked pretty good in a phase 2 study but didn’t pan out in the phase 3 investigation [NCT01522976]. The phase 3 trial that is testing APR-246 plus azacitidine versus azacitidine alone [NCT03745716] has now completed accrual and hopefully will report out by the end of the year. It is a really important study for trying to understand if this is a viable strategy.

UPDATE FROM MDS UK - 20-01-2021:

A second phase II study to evaluate if it is possible to improve outcomes for patients with TP53-mutant myelodysplastic syndromes (MDS) and oligoblastic acute myeloid leukemia, who had poor outcomes with standard-of-care azacitidine therapy, produced again encouraging results.

This second study was conducted by David A. Sallman, MD; Amy E. DeZern, MD; Guillermo Garcia-Manero, MD; David P. Steensma, MD and others.

Read the full study in the Journal of Clinical Oncology

The combination of azacitidine and eprenetapopt (APR-246) was well-tolerated in patients with TP53-mutant MDS or acute myeloid leukemia.

Azacitidine and eprenetapopt resulted in a 71% overall response rate and 44% complete remission rate in MDS patients, comparing favourably with a therapy of azacitidine alone.

These results support the ongoing pivotal phase III, multicenter, randomised study of eprenetapopt in combination with azacitidine versus azacitidine alone in patients with TP53-mutant MDS.

Please explain the rationale for combining eprenetapopt and azacitidine.

Both azacitidine and decitabine are important standards of care for patients with MDS. Those are the backbones of therapy, and azacitidine is the only agent that’s been shown to improve overall survival for higher-risk patients in a randomized trial—so patients deserve to have exposure to this drug.

The reason for combining azacitidine with APR-246 is that reactivating TP53 alone only may partially sensitize the cell to dying if it is an abnormal cell. Normally if there’s DNA injury detected in a cell, that cell would be programmed to die, and that program would be mediated by p53. If you restore a p53 function in an abnormal cell but there’s not enough DNA damage to the cell, then the cell may still survive. If you combine APR-246 with an agent that causes DNA damage or cellular stress like azacitidine, then that intact p53 may finally be able to sound the alarm and kill the cell, so to speak.

In MDS, we’ve seen a number of trials where there was no logic behind combining agents with azacitidine. Previously, it’s been, “Oh, our drug has a little bit of effectiveness, or even no effectiveness, but maybe it will be better if we combine it with azacitidine,” and that’s kind of a desperate move. Here, as with venetoclax—which also sensitizes cells to death but doesn’t have a lot of independent activity on its own in myeloid disorders—there’s a real rationale for combination.

What do we know about the safety profile of eprenetapopt (APR-246)?

From what we’ve seen so far, APR-246 has a favorable safety profile. The recurrent toxicity that we’ve seen is a peculiar form of neuropathy where patients may feel altered sensation, such as their skin feeling especially sensitive. They may feel like ants are crawling on their skin or other peculiar sensations, and this usually gets better with prochlorperazine, which is a widely used antinausea medicine. So, for the most part, that adverse event has been manageable.

I was impressed in the phase 1/2 experience by just how well patients did with the drug. Overall, APR-246 didn’t seem to cause a lot of trouble, other than the neuropathy. We will see in the randomized comparison how the toxicity patterns compare to azacitidine alone, and that will provide more information. My suspicion is that we’ll see more neuropathy with the combination group, but probably not a lot of other differences in terms of adverse events.

What challenges remain in other subtypes of MDS?

There are a number of unmet needs for patients with MDS outside of TP53-mutant disease. In the higher-risk population, for the majority of patients, hypomethylating agents will either stop working within 1 to 2 years or they weren’t working in the first place. We don’t have a second-line therapy that’s useful and has been shown to improve survival or delay disease progression, so that’s a big unmet need. In fact, the long-awaited, randomized trial [INSPIRE; NCT02562443] of rigosertib just reported out in that post hypomethylating agent setting, and data showed that rigosertib failed to improve survival compared with physician’s choice of alternate therapy.

The second big unmet need is for the lower-risk patients, whose main problem is low blood counts that could cause them to be transfusion dependent on a regular basis, which is quite inconvenient for them. We had luspatercept [Reblozyl] approved for MDS with ring sideroblasts and SF3B1 mutations in April, which was the first new drug approved for an MDS indication in 14 years. However, luspatercept only helps a subset of patients, and the approval was only for those with ring sideroblasts, which is 15% to 20% of patients at most. There are a lot of other patients with low blood counts that either aren’t good candidates for luspatercept or have been failed by luspatercept, and the question becomes, “How can we improve counts for them?”

These are probably the 2 biggest needs, but there are others. For the transplant population, how to prevent relapse after transplant is also a key question.

References:

Aprea Therapeutics receives FDA breakthrough therapy designation for APR-246 in combination with azacitidine for the treatment of myelodysplastic syndromes (MDS) with a TP53 mutation. News release. Aprea Therapeutics January 30, 2020. Accessed August 25, 2020. https://bit.ly/3hsVKFR
Sallman DA, DeZern AE, Garcia-Manero G, et al. Phase 2 results of APR-246 and azacitidine (AZA) in patients with TP53 mutant myelodysplastic syndromes (MDS) and oligoblastic acute myeloid leukemia (AML). Blood. 2019;134(suppl 1):676. doi:10.1182/blood-2019-131055

MDS UK wishes for a healthy, safe Christmas and a much improved 2021!

Posted on 24/12/2020 at 9:03 am.

Written by Manuela Rotstein

Dear MDS UK members,

We hope you have been coping as best as possible during this very difficult year for everyone.

From the many phone calls and emails we have received since March 2020, we know how worried you have been about Covid-19. At the height of the pandemic, our phone line was open 24/7 and the phone was indeed ringing all day. We hope we have been able to assist you in some way.

Many of you have been feeling even more isolated than ever before. You have had issues about access to your GP. Thankfully, our Covid-19 MDS survey shows that most haematology departments managed to maintain good care services, thanks to amazing staff.

As a small charity, we have tried to do our best, in very difficult circumstances, with all 4 staff members working from home, in the midst of growing financial concerns, due to the cancellation of all of our fundraising events.

We had some fantastic achievements despite these difficulties, and have experienced true silver linings, thanks to all of you, the NHS staff, our committee members, our coordinators and our own staff.

For instance, we have been amazed at the creativity of many of you, our members, taking part in the 20for20 fundraising campaign – and the incredible generosity of donors.

This campaign managed to raise just over £40,000 for the charity. These funds have been an absolute lifesaver, as our financial reserves have dangerously dwindled this year, causing us serious concerns for the future.

Despite these hurdles, we have managed to keep going.

Information to members

We have worked closely with all MDS experts, to provide up to date information regarding NHS haematology services, treatment recommendations, MDS clinical trial news and now guidance about the upcoming Covid-19 vaccine.

We have responded to a record number of new membership requests and phone calls. I had great phone calls with so many of you. Another silver lining, as we got to know each other even better.

We know we sometimes struggled to respond to the many phone calls and emails.

So, thank you for your patience in those circumstances.

Together with so many of you, we embraced online technologies, and set up virtual zoom meetings, enabling patients to speak and see one another, as well as get access to Q&A sessions with experts.

That has been the other significant silver lining! So many of you reported how enjoyable and welcome these meetings have been. We have seen so many new faces too – a real joy and relief from this COVID-19 isolation.

Research and access to treatment

We co-worked on two MDS clinical trials. One (Repair-MDS) will start recruitment in early 2021.

It is a particularly exciting trial, as it uses ‘repurposed drugs’, meaning it will use existing drugs with a different purpose (MDS), and will therefore be much cheaper to use, if the trial is successful of course.

For the other trial – we are waiting to heart whether it will be approved. That trial aims to improve the diagnosis and care of fungal infections in haematology patients.

Re access to treatment, we have campaigned tirelessly with our international colleagues, to gain early access to the new MDS drug Luspatercept, designed to help transfusion dependent patients diagnosed with the RARS MDS sub-type. Unfortunately, the pharmaceutical company has still not given any indication of when they may submit their approval request to NICE, nor have they agreed to any compassionate use of the drug. We have been promised more information on this topic in January 2021.

Let’s hope this promise is kept!

The PhD project at Belfast University Hospital, which we are funding, thanks to 3 major legacies, is progressing nicely. We will provide an update in January, which will mark the end of the 1st year of work. Three more years to go till results!

For awareness of MDS

We will share our new MDS awareness cartoon ‘Myelo’s Hope’ more widely and ask more hospitals to share it on TV’s in their waiting rooms. If you have the opportunity, be an MDS Ambassador and please speak to your haematology staff about it. Share how much MDS UK is helping you, so that future MDS patients don’t have to trawl the internet looking for information.

We worked lots more on the international scene too, as part of the MDS Alliance, contributing to European projects like Harmony and EuroBloodNet, which aim to improve treatment and care for several blood diseases, MDS included.

In 2021

We have prepared more online meetings, with more people, more locations, more speakers, due to popular demand.

We also aim to restore the paper newsletter, which has much suffered during this Covid year.

We’ll report on the first results of the MDS – Covid-19 survey, which over 300 of you completed in the last few months. Thank you so much!!!!

We’ll keep reporting on and assisting with information on the new vaccines which may get approved by early 2021. We also hope to provide more news on efficacy in blood cancer patients, thanks to the wonderful team of MDS experts we work with closely.

I take this opportunity to thank all of them. From our own official scientific advisors, Dr Dominic Culligan, Prof Bowen and Dr Austin.

All of the UK MDS Forum team of experts are phenomenal in their willingness to support us and their readiness to supply advice and information.

A very special thank you to Dr Simone Green, who works on the MDS clinical trials news page – so essential to patients and general haematologists alike.

We leave you with a few words from some of our MDS UK committee members and staff, whom you had the chance to meet during some zoom meetings this year.

Greetings Everyone in our MDS Community!

This last year has certainly tested the resolve and strength of all those suffering with MDS and their carers. Despite the new Covid vaccine, it would seem that the pandemic is going to remain with us for some time to come. MDS-UK staff, our Central Committee and our many supporters have responded magnificently to the issue of support and fundraising during these troubled times. I want to say thank you to all those involved in enabling us to continue our support and service provision to our MDS Family, as well as our clinical research. Our targeted Zoom meetings have been one positive outcome, proved popular and have helped to reach some of those who would not normally have attended traditional meetings. Obviously, as the pandemic evolves we must continue to think of new, innovative ways to fund our Charity and I would ask everyone to consider possible alternative future fundraising ideas.

I know many of you have been experiencing difficult journeys with your respective MDS and, sadly, some have departed this life during the year. To those grieving, we remain here for you and to those who have recently started their MDS journeys, I wish you every success with your various treatments. I have undergone a stem cell transplant during lock-down and I am on a journey of recovery and look forward to once again fully participating in the management of MDS-UK in 2021.

2020 has been a difficult year for all, however, I wish you the best Christmas possible and a better and safer 2021.

Best Wishes

Ted Peel, MDS UK Chair

2020 has been a very difficult year for us all, but particularly for extremely vulnerable MDS patients who have had to deal with threat of COVID and the disruption to lifestyles resulting from the response to this threat.
However, as in all bad times, there are lots of good things ongoing in the background. The COVID vaccine is top of the list and will be a crucial part of the way forward for most MDS patients. The superb work of MDSUK in supporting patients through these times has been inspirational and we must thank Sophie and all her colleagues for this invaluable work.
From the medical point of view research has continued to take small steps forward in improving the treatment and outlook for patients with MDS. Sophie rightly highlights luspatercept, which is an important new therapy for some anaemic patients with MDS with ring sideroblasts.
I would also like to highlight the funding of the REPAIR MDS trial, which hopefully will start recruiting patients during 2021. Having been involved with the NCRI MDS Clinical Trials Group since its inception many years ago it is heartening to see a major randomised trial, which will be accessible across much of the UK, come close to fruition after more than four years of planning. Hats off especially to the team in Birmingham and also to colleagues on the NCRI MDS Clinical Trials Group for this sterling effort.
I would like to wish you all the very best for Christmas and for a better 2021.

Dominic Culligan
Scientific Advisor, MDS UK

2020 has been a watershed year for our charity. Despite the challenges set before us we have managed, somehow, almost miraculously, to ensure that we still have some level of fundraising. I would like to take the opportunity to thank all of our supporters, our patients, and the families and carers of those patients for their most tremendous endeavours this year. We can't have done it without you. Next year brings new challenges and this Christmas will not be without some concerns, so my New Year wish would be that the charity becomes ever more active and encourages even more people to become involved with us.
Merry Christmas and a happy New Year

Russell, Head of Fundraising and Deputy Chair

"During my lifetime I’ve had a few dodgy years. Never in my wildest dreams did I imagine collectively we would all go through so much in 2020. I was diagnosed with MDS 20 years ago. I’ve looked after my health and protected myself ever since. What’s hard about the pandemic is that those of us with wonky immune systems are now reliant on everyone else doing the right thing.

I have been impressed by all the changes that haematology departments have made to keep patients safe. I’ve represented patients in discussions about these changes and will continue to be a strong advocate for our MDS community."

Hope this is ok. Keep safe everyone and I hope you all have a happy and healthy Christmas.

Take care, Kes

Love is what’s with you in the room at Christmas if you stop opening your presents and listen.

Our online Support Group Meetings have been a great way for everyone to meet this year. We have been lucky enough to have wonderful Doctors, Consultants and CNS Nurses join many of these meetings. For anyone who has never joined a zoom call or a local meeting in previous years you really must join in 2021. I have had the joy of laughing with so many of you, over this very strange, and isolating year. I would like to say a huge Thank You

Wishing everyone a Merry Christmas and a healthy and happy 2021.

Caroline, Office & Database Manager

I'd like to say a huge thank you to everyone at MDS UK for making me feel so welcome in my first 9 months here! It's been a challenging year to say the least but it's been great getting to know many of you so far and your inspirational stories spur me on!
There's too many great individuals to mention so another big thank you to everyone who has fundraised / donated to MDS UK this year.
With many of our biggest events cancelled or postponed, the determination of you all to help keep our work going is remarkable!

Wishing you all a merry Christmas and (hopefully) a better and less stressful new year! Looking forward to speaking to more of you in the months to come!

Jan Edwards, Fundraising Officer

My A-Z of 2020!

Absence -it really does make the heart grow fonder when you cannot see family and friends

Berries -of all kinds from our allotment

Coronavirus -the frightening cause of it all

Depression -finding strategies to avoid feeling low and Deaths -too many unnecessary ones.

Energy -newfound in the Spring, sowing seeds for garden and allotment

Fundraising -and Focusing on something positive

Grandchildren -oh, how we’ve missed them.....

Holding their hands, hugging and just hanging out with them

Information - so much to absorb

Judging friends on their COVID behaviour and attitudes (!)

Kicking a ball with grandchildren in the garden when allowed in the summer

Loving my husband so much for shielding and protecting me by being Head Shopper and Errand Boy!

My Mum who is 92 but has embraced Face Time!

Nice meals with fresh produce from our allotment

Opportunities to meet new people from MDS community

Power walks for my 20 for 20 project

Questions Questions Questions. (Aren’t scientists amazing?!)

Re-assessing priorities in life

Surprised by support and kindness of neighbours and friends in my fundraising efforts

Twenty for Twenty and meeting such a great group of people in the fundraising group

Unbelievable ignorance from a politician from across the pond and elsewhere with hoax stories and fake news

Vaccines, vials, validation, value - remains to be verified in long -term trials

Wintrich -Sophie - holding it all together, making massive changes to how MDS patients are supported

X -Xmas -not what we were promised but now safer for all

Year 2021 -hopefully an improvement for us all

Zoom -what else?! The great enabler of 2020! It helped us keep in touch, attend NHS,MHRA,EMA meetings, meet people with MDS from around the UK. My choir met every week so we got to sing some wonderful choruses - all muted!

Take care all

Have a safe, enjoyable Christmas and a wonderful New Year

Chris Dugmore Xx

I’d like to personally thank all of you for your collaboration, presence, engagement, support, feedback, generosity and willingness to share your experiences and emotions.

Your input is unique and irreplaceable.

Many of you have been a tremendous inspiration to newly diagnosed families, helping them through the first few difficult months after diagnosis, or during some tough treatment times.

Or, sadly after the loss of some of our members.

Thanking our great local group coordinators, who make everyone feel welcome and engaged in these new zoom meetings. An essential group of people, without whom the charity could not function.

Thanking our entire Committee for their volunteer work throughout the year. A hard working bunch, without whom this charity would not exist.

And our staff, especially the newcomers, Chaudian and Jan, who joined just as Covid started!

A challenging beginning for them.

And thanking our website editor Manuela for her brilliant work and advice.

This MDS community is stronger, louder, able to contribute to research and raise awareness of this rarer blood cancer.

A few practical details now:

Our office will be closed from:

Thursday 24th December at 6pm

to Monday 4th January 2021 at 9.30am

Hope to see you all for a return to work, bit refreshed, hopefully virus free.

If you do require medical advice or assistance, as ever, please call your nurse specialist at your hospital.

From all of us at MDS UK, staff, committee members and volunteers, we wish you a healthy, safe Christmas, and a much improved 2021.

Stay well, stay safe and hope to connect again in January 2021.

Sophie Wintrich, CEO MDS UK

Tel – 02077337558

Christmas Poem 2020 – by Kate D.

Posted on 20/12/2020 at 5:20 pm.

Written by Manuela Rotstein

Christmas Poem 2020 - by Kate D.

A tradition of traditions
that once a year
consumes us with
Nutcracker images,
ginger and spice,
stockings and F.C.,
a decorated tree.
But to me,
I look around and
see the secret of Christmas…
Beauty.

Lights twinkling,
a sparkle of
glitter and idealised
childhood Christmases.
I could go on, but I
stop as
Beauty is everywhere
if you look beyond the obvious.

Look at loved ones;
their smiles, their eyes-
gestures and laughter
from deep within.
Explore expressions of
excitement and pleasure,
surprise and comfort
when disappointment
and loneliness threaten to
takeover.

20for20 “Charity Shop”

Posted on 09/12/2020 at 9:25 am.

Written by Manuela Rotstein

Welcome to our "#20for20 Charity Shop". This is the place where our amazing fundraisers are swapping things for donations! What an excellent way to support MDS UK!

How does it work?

To Donate:

Take a look at the available items below and decide what you'd like to swap. More items will be added in the coming days.
Send an email to the address provided. Please put the name of the item in the subject
The fundraiser or Jan -our Fundraising Officer- will be in touch shortly thereafter
The fundraiser sends the item to your address!

To Fundraise using our "20for20 Charity Shop":

Look around your house or make something to swap!
Contact Jan at fundraising@mdspatientsupport.org.uk
Start fundraising!

Imerge (Imetelstat) – Clinical Trial Open to Recruitment

Posted on 08/12/2020 at 3:09 pm.

Written by Manuela Rotstein

SUB-TYPE OF MDS: Low risk MDS patients
SEVERITY OF MDS: IPSS Low or Intermediate-1 risk MDS
NAME OF DRUG: Imetelstat
Aims and benefits: This phase 2/3 study aims to determine the effectiveness of Imetelstat in transfusion dependent low risk MDS patients who have either lost their response or not responded to erythropoiesis-stimulating agent (ESA) treatment. The study has 2 parts. Part 1 seeks to determine the effectiveness and safety of Imetelstat, looking more closely at improvement in blood counts and reduction in transfusion requirements. Part 2 is double-blind and randomised to compare the effectiveness of Imetelstat with placebo.
Primary outcome:To determine the percentage of patients who have not required a red cell transfusion in an 8 week period.
Secondary outcome:To determine adverse effects, red cell transfusion independence, improvement in blood counts, progression to acute myeloid leukaemia and overall survival.

Myelo’s Hope Christmas Animation

Posted on 08/12/2020 at 2:03 pm.

Written by Manuela Rotstein

Christmas is just around the corner and it will look drastically different for millions of people across the globe this year. Hope and encouragement will be needed to find safe ways of enjoying the season.

Many of you will have seen Myelo's Hope the animation we released on MDS World Awareness Day which explains MDS in an accessible way and signposts patients and their loved ones to support.

Buoyed by the warm response, the animators have now released a fantastic festive version, further highlighting the issues in the original while calling for much-needed support!

A novel treatment for MDS patients in need of frequent blood transfusions

Posted on 09/11/2020 at 3:12 pm.

Written by Manuela Rotstein

The innovative drug Imetelstat has been trialed on patients with lower-risk MDS who are RBC (Red Blood Cells) transfusion dependent and have experienced relapse or not been helped by erythropoiesis-stimulating agent (ESA).

This group of patients does not have many treatment options at the moment.

In this press briefing, recorded at the 24th Congress of the European Hematology Association (EHA) 2019, held in Amsterdam, Netherlands, Pierre Fenaux of St Louis Hospital, Paris, France, presents data on the use of Imetelstat in lower risk myelodysplastic syndrome (MDS) patients.

Scientists have observed that patients with MDS have a high telomerase activity in their stem cells. Telomerase is a naturally occurring enzyme, and its activity prevents cells from dying. Telomerase is repressed in most normal cells, preventing uncontrolled proliferation. In cancer cells, however, this enzyme is active.

Imetelstat, is a "telomerase inhibitor" and targets cells with active telomerase.

The study has reported that Imetelstat shows efficacy to control the enzyme and it's safe to use. It also reports biomarker data for patients with low-risk MDS who are red blood cells transfusion dependent and who were relapsed/refractory to erythropoiesis-stimulating agents.

About the study

The study is a two-part phase II/III study.

The primary endpoint (the main result that is measured at the end of a study to see if a given treatment worked) was 8-week red blood cells transfusion independence rate.

Key secondary end points were:

24-week red blood cells transfusion independence rate
transfusion independence duration
haematologic improvement-erythroid

Of 57 patients enrolled and treated (overall population), 38 were non-del(5q) and hypomethylating agent and lenalidomide naïve (subset population).

Encouraging results from phase II of the study

A Phase II trial is still early days in terms of clinical trials – to read more about how this trial progresses – see this page at ClinicalTrials.gov

The 8- and 24-week red blood cells transfusion independence rate in the overall population were 37% and 23%, respectively, with a median transfusion independence duration of 65 weeks.
In the subset population, 8- and 24-week red blood cells transfusion independence rates were 42% and 29%, respectively, with a median transfusion independence duration of 86 weeks.
Eight-week transfusion independence rate was observed across all subgroups evaluated.
Cytogenetic and mutational data revealed a reduction of the malignant clones, suggesting disease modification activity.
The most common adverse events were cytopenias, typically reversible within 4 weeks.

In conclusion, Imetelstat treatment results in a meaningful, durable transfusion independence rate across a broad range of heavily transfused patients with Low-Risk MDS who are ineligible for or relapsed/refractory to erythropoiesis-stimulating agents. Biomarker analyses indicated effects on the mutant malignant clone.

Authors

David P. Steensma, MD¹; Pierre Fenaux, MD, PhD²; Koen Van Eygen, MD³; Azra Raza, MD⁴; Valeria Santini, MD⁵; Ulrich Germing, MD, PhD⁶; Patricia Font, MD⁷; Maria Diez-Campelo, MD, PhD⁸; Sylvain Thepot, MD⁹; Edo Vellenga, MD, PhD¹⁰; Mrinal M. Patnaik, MBBS¹¹; Jun Ho Jang, MD¹²; Helen Varsos, MS, RPh¹³; Jacqueline Bussolari, PhD¹³; Esther Rose, MD¹³; Laurie Sherman, RN¹⁴; Libo Sun, PhD¹⁴; Ying Wan, MD, PhD¹⁴; Souria Dougherty, BS, MBA¹⁴; Fei Huang, PhD¹⁴; Faye Feller, MD¹⁴; Aleksandra Rizo, MD, PhD¹⁴; and Uwe Platzbecker, MD¹⁵

¹Dana-Farber Cancer Institute, Boston, MA
²Hôpital Saint-Louis, Université Paris Diderot, Paris, France
³Algemeen Ziekenhuis Groeninge, Kortrijk, Belgium
⁴Columbia University Medical Center, New York, NY
⁵MDS Unit, AOU Careggi-University of Florence, Florence, Italy
⁶Klinik für Hämatologie, Onkologie and Klinische lmmunologie, Universitätsklinik Düsseldorf, Heinrich-Heine-Universität, Düsseldorf, Germany
⁷Department of Hematology, Hospital General Universitario Gregorio Marañon, Madrid, Spain
⁸Hematology Department, The University Hospital of Salamanca, Salamanca, Spain
⁹CHU Angers, Angers, France
¹⁰Department of Hematology, University Medical Center Groningen, University of Groningen, Groningen, the Netherlands
¹¹Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN
¹²Department of Hematology, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea
¹³Janssen Research & Development, Raritan, NJ
¹⁴Geron Corporation, Menlo Park, CA
¹⁵Department of Hematology and Cell Therapy, University Clinic Leipzig, Leipzig, Germany

Zack Pemberton-Whiteley, Chair of the Blood Cancer Alliance:

BCA letter to Matt Hancock, Secretary of State for Health

BCA letter to the JCVI, NHS England

Latest News

Shielding guidance: What do you need to do if you are extremely vulnerable?

Part 1. Advice regarding the Coronavirus outbreak in UK

What has changed

Socialising

Work

Education settings

Travel

Shopping

Medicines

Accessing care and support

Registering for additional support

Coronavirus Structure

Part 2. Implications for blood cancer patients

If I have blood cancer, am I more at risk?

Would like a specialist MDS consultation? You can talk online, from the comfort of your home, with Prof. Bowen

Part 3. Implications for our MDS UK local patient group meetings

Part 4: What to do if you suspect you have Coronavirus

There might not be tests available at any given time.

Symptoms of coronavirus

If you feel unwell

Getting help in Scotland, Wales or Northern Ireland

Check if you need medical help

Learn more about the situation right now: GOV.UK: information on coronavirus and the situation in the UK

Latest News

Building a picture of cancer-specific infection and immunity could help inform treatment strategies

Lymphocyte B cell

Learn more about this clinical trial

Latest News

Latest News

Latest News

Latest News

How does it work?

Items: Jean's Cross Stitches

Items: Molly Viner's "MDS UK by Autumn"

Items: Rebecca Bennett's Animal Paintings

Item: Christine Peel's MDS UK Christmas cards

Imerge (Imetelstat)

Myelo's Hope: Christmas Animation

Latest News

Imetelstat has achieved meaningful and durable transfusion independence in clinical trials

How does Imetelstat work? The science behind it

About the study

Encouraging results from phase II of the study

Authors

Latest News