Our impact We are proud of our achievements In 1980 only 3 in 10 patients survived aplastic anaemia. Thanks in large part to the Aplastic Anaemia Trust, our generous supporters and collaboration with researchers, 8 in 10 patients will survive the devastating effects of aplastic anaemia today. To date, we have raised and invested over £3m in transformative research and excellent emotional and clinical support for patients and their loved ones. 2018/19 was an incredible year! Here are some highlights of our achievements across our main objectives - only made possible by the generous support of our donors, volunteers and grant giving organisations: 1. Patient support and information Having been awarded a Transform Foundation grant, in June 2018 we developed and launched a brand-new website. The website, being search engine optimised, has been game changing for our charity and attracting interest from across the UK and internationally. A series of downloadable information resources, co-developed with patients and experts at King’s College Hospital, London and St James’ Hospital, Leeds, have been made available online focused on the following areas: Treatment involving Anti-thymocyte Globulin (ATG) Information on Steroids used as part of ATG treatment Treatment with Ciclosporin Treatment with Eltrombopag Androgens Supportive therapy Practical advice on travel and insurance Cold season advice Advice on clean/ neutropenic diet Benefits advice. Information resources on treatments were distributed across major treatment centres in England and Wales. Our online patient community within the closed and moderated Facebook group grew by over 300 new members, demonstrating a significant rise of over 300% compared to previous years. We introduced a number of events, providing AA patients, their family members opportunities to get together for face to face emotional peer support and discuss with clinicians any aspects of their care and recovery. It is important that patients with AA take an active role in their care and have as much information about the disease, its treatment and advice on lifestyle. It’s great that the AAT provide this. 2. Research Despite the advances made over the past decades, stem cell transplant remains the only hope of permanent cure for the majority of patients. Most require lifelong care even when the marrow appears to have returned to normal blood production. The AAT aims to raise more funds and use its resources to support ground-breaking research and management methods in all types of bone marrow failure as well as its pre-eminence in acquired aplastic anaemia. Such a broadening of our remit will benefit the understanding of individual failure mechanisms and improve the lives of many. In May 2018 we unveiled our very first Research Strategy with its ambitious aims, to: Improve baseline understanding of rare bone marrow failures in child and adult populations. Improve the understanding of the diseases by molecular/ genetic research into the damaged cells and their environment – the pathogenesis of rare bone marrow failures Identify risk factors in AA patients that predispose to later MDS/AML Improve treatments, identifying alternative cures, enabling experimental therapies and ultimately eradicating the diseases, including the personalisation of discovery platforms and treatments for rare BMF syndromes and AA. Research focused highlights in 2019 include: The Research Advisory Panel was convened comprising of a member of our patient community and leading rare bone marrow failure experts from across the UK. They volunteer their time to provide independent scientific advice on research proposals thus helping us ensure we invest our funds as wisely as possible and in doing so get closer to achieving the charity’s mission and ambitions. Two medium-term research grants were awarded as follows, following a national call for proposals: Identification and relevance of novel somatic mutations in aplastic anaemia, led Dr Alex Smith and Prof. Judith Marsh, King’s College Hospital, London. This is a 2-year project with the value of £66,439 Proposal to establish a paediatric UK bone marrow failure registry and biobank, led by Dr Sujith Samarasinghe. The value of the funds awarded is £54,286 over 3 years. I am passionate about fundraising, as so much more research needs to be done into finding more treatments for AA. The AAT are driving this and working closely with the UK’s best clinical and research teams to develop relationships which means better care for patients. 3. Awareness, profile raising and advocacy A series of informative articles were published on Open Access Government platform – “The Importance of Research into Aplastic Anaemia”, “Paediatric Haematology Research in the UK – Why Starting at the Beginning is so Important” and “Raising Awareness of Aplastic Anaemia”. The combined impact of the articles was an audience reach of over 30,000. Two awareness raising campaigns were organised – in the run up to Christmas and around the International Rare Disease and Aplastic Anaemia Awareness Days in Spring 2018. 6 films were produced and publicised on our new You Tube channel and website highlighting key aspects of our work, focus on research and treatment landscape, a young and adult patients’ accounts of being diagnosed with a rare life-threatening illness and its impact on their lives. The focus of the Christmas campaign was on sharing 12 personal accounts of life with aplastic anaemia to draw attention to the illness, its impact and the work that we do. The personal blogs helped us reach an audience of over 20,000 on social media. The spring awareness drive drew attention to “what it’s really like…” to have a family after treatment for AA, save a sibling’s life, treat people as a clinician and other aspects of the disease, whilst celebrating hope, inspiration and people’s strength when faced with AA. Little known facts about the rare illness were also spread across all our social media outlets. Via Facebook alone we reached 24,500 people and significantly increased traffic to our website. We significantly boosted social media presence across all our social media outlets, including our new presence on Instagram. We published research findings and updates from the international community as research blogs on our website. We became members of Genetic Alliance UK – a national charity and alliance of over 200 organisations, working to improve the lives of patients and families affected by all types of genetic conditions. This provided us with an excellent platform for knowledge exchange and the sharing of learning and best practice. Excellent article highlighting the very rare nature of this devastating disease and the impact it has - life changing and life threatening. This hopefully will increase awareness and understanding across both the public and professional sectors and ultimately help all those affected by aplastic anaemia. Thank you the AAT! 4. Organisational development and growth An extensive programme of transformation was launched by the new CEO, with the aim of modernising the charity, exploiting digital opportunities, creating a positive profile and solid foundations for future growth and development. This is so that we become the Go-to organisation for research, patient and family-centric information and support across the UK. A vibrant and fresh new brand was developed and unveiled, with excellent expert pro bono support from Philosophy Design. This signalled the AAT’s transition to an open, proactive and dynamic small charity capable of expanding its reach, building progressive and productive partnerships across the UK and relentlessly focused on the achievement of its vision. A significant effort was put into building the foundations for diversifying our funding streams, to reduce over-reliance on individual donations and community fundraising and maximise opportunities for sustainable income. The effort will bear fruit next year. 2 grants were secured from grant giving Trusts and Foundations, to enable us to begin to broaden our Outreach activity - from Rowlands Trust (£2,000) and Barbour Foundation (£1,000). We were enormously grateful for this support as it enabled us to begin to expand our outreach activities in the Midlands and the North East. We registered with the Fundraising Regulator to ensure the AAT and everyone raising funds on our behalf comply fully with fundraising regulations and best practice. Over 40 committed volunteers have agreed to help us in our work by offering their time and skills – as part of our new website development project team, writing personal blogs, helping with our events, raising profile and awareness of the work that we do and much more. We whole-heartedly thank you for all your time and support. We began to build new valuable partnerships and strengthen existing ones across key haematology treatment and research centres in the UK as well as large medical research charities, such as LifeArc, and other partners – e.g. the British Society of Haematology (BSH), Findacure, Cambridge Rare Disease Network and others. Effort in this area is essential and will continue, to ensure we stay connected to developments in the current treatment and research pathways so that we can offer optimum value through our engagement and contributions. One of the first resources I read when diagnosed with aplastic anaemia came from the AAT. The fact sheets, the blogs, the comfort of the community - they've all supported me in different ways. I knew I wanted to offer whatever support I could, and I was pleased the AAT was happy for my help when at last I got in touch. There is so much more to be done to get us closer to a cure for aplastic anaemia. We need you to keep supporting us with enthusiasm and generosity, so that everyone affected by a rare bone marrow failure can get the best help and support there is.