If asked to imagine something extremely rare and quite valuable, there would probably be a look of surprised skepticism if we replied, “That must be our 2024 aHUS Alliance Global Action Poll!”
In the years since the first official HUS diagnosed patient there have only been 3 surveys ever designed and conducted from ‘concept to analysis’ in conjunction with international atypical HUS patients and family caregivers.
That’s right, three. Surveys have attempted to capture what it’s like to live with aHUS, the first official patient only lived with it for 30 days, but many of those have been conducted by marketing professionals with little concept of what it’s like to live with an unpredictable yet life-threatening rare disease. Most often there’s no input from the people affected by aHUS prior to their polling initiatives, so vital questions remain unasked and few meaningful conclusions can be drawn by data sets that didn’t cover certain areas.
Most surveys for rare disease populations are created and conducted by third-party professionals, at the behest of businesses with a financial stake in discovering such information. A few surveys are conducted by research and medical teams, who genuinely have interest in patient care and/or wish to know more about the disease and its progression.
That’s why the aHUS Alliance Global Action team of aHUS patients and family caregivers have stepped up again in 2024 to request that other aHUS families worldwide participate in this important poll. Research teams, pharma, and medical professional need a clearer picture of what it’s like to live with a diagnosis of atypical HUS. Who better to tell the story of our challenges with this disease, and its impact on patients and their families, than aHUS families ourselves?
It’s ambitious to look back at the 2014 poll to see what has changed (and what has not) for adult patients and family caregivers of children diagnosed with atypical HUS. This ‘Ten Years On’ survey to compare situations and challenges from 2014 to 2024 will yield some expected changes, such as a ravulizumab’s approval in select nations as an treatment for aHUS. The 2014 survey provided information and insights from 214 respondents, replying in 6 different languages across 17 nations. Data analysis was provided in a single report, with additional details on a slidedeck of graphs provided by our 2014 survey partner RareConnect and its project lead Rob Pleticha.
In 2024 what is it like for aHUS patients and their families in terms of their experiences and effects on their lives? As the aHUS Global Action team has developed skills and the aHUS landscape has broadened, our ‘Ten Years On’ comparison of the aHUS community is faced with and challenged by, there is additional value behind this 2024 poll. It’s the first time in our survey history that we will be looking to publish in the results in a clinical journal, giving additional strength behind our initiative. Poll background and updates will appear HERE, with this sharable link https://bit.ly/2024aHUSglobalPoll
We invite ALL adult patients and family caregivers around the world to participate in the 2024 survey. The survey portals are open from 8th July to 18th August, and are available in 10 languages:
The following information is provided to inform consent at the beginning of the questionnaire.
aHUS Global Poll 2024 – Study of aHUS as a rare disease
QUESTIONNAIRE
Before volunteering to participate in this aHUS Global Study, please read the following information and guidance notes:
Information for consent to participate
- The Study is being carried out by aHUS alliance Global Action, an independent registered incorporated charity for Global aHUS patient advocacy.
- The Study is accessed only through its website, which has a Secure Socket Layer (SSL) site certificate.
- The Study Questionnaire is powered by Googe Forms, a well-established and reputable survey provider.
- Study questions cover characteristics of global aHUS patients as well as their relatives’ experience of rare disease issues’ themes of diagnosis, treatment and commissioning, research and registries, societal impact, access to a center of excellence, and information.
- The data being sought is needed to see if any progress has been made in the 10 years since a Global aHUS Poll was conducted in 2014 and elicit more understanding of the related rare disease issues.
- Participant’s response data will be kept anonymously, securely, and safely by the charity and not provided, nor sold for profit to any third party.
- All data will be kept confidentially for three years following the release of results.
- The results of this study may be published by aHUS alliance Global Action via newsletters, presentations, academic papers, reports, and visual graphics with multiple key stakeholders including researchers, multi-disciplinary healthcare workers, policymakers, and other stakeholders. It will not be possible to identify any individuals from reports or presentations.
- All participation is voluntary, and respondents must be willing and able to complete the questionnaire. Respondents can opt out during the questionnaire completion, and not answer some of the questions, but once the questionnaire is completed no changes can be made.
- No individual health care providers’ names, nor hospital or other care organizations’ names are required apart from aHUS expert centers, where only their roles are sought.
- Terms like aHUS (Atypical Hemolytic Uremic Syndrome), TMA (Thrombotic microangiopathy) TTP (Thrombotic Thrombocytopenic Purpura), HUS (Hemolytic Uremic Syndrome) will feature in some questions.
- If further information is required for your consent to participate, please contact info@ahusallianceaction.org for assistance.
Please confirm you have read the information and guidance and are giving your informed consent to participate in this poll and study by ticking this box.
| Read and confirmed |
