Você pode desempenhar um papel importante no combate ao ECD.
Se você é um paciente ou cuidador direto, participe desta pesquisa até 30 de outubro de 2021.
When the ECD Global Alliance (ECDGA) was formed research was needed to help improve the diagnosis and treatment of Erdheim-Chester Disease (ECD). Little was known about the disease and research was difficult because patient access was limited, and funding was almost non-existent. Only a little over a year after the formation of the ECDGA, the organization began to fund ECD-focused research.
The ECDGA has historically funded nine (9) research projects worth over $700,000 proposed by the medical community. This has worked well to push ECD research forward. In the past decade, much has been learned about ECD and there have been substantial breakthroughs in the treatment of ECD, including an FDA-approved treatment. Although not FDA-approved, other targeted treatments have been found as well. With better treatments, patients are now living more normal lives.
Até o momento, as pesquisas realizadas têm se concentrado em (1) ciência básica, (2) pesquisa de transição e (3) pesquisa clínica.
1) Pesquisa em Ciências Básicas allows scientists to do lab investigations to increase knowledge and understanding of ECD. The ECD-funded research to better understand the various mutations seen in ECD patients was funded in part as a basic science research project. The findings eventually allowed more targeted treatments to be discovered for many histiocytic patients.
2) Pesquisa translacional takes knowledge learned in basic research to determine how that knowledge might be “translated” into clinical care. For instance, when different ECD mutations were found, the translational research projects were able to discover the possible treatments that could be used to target those mutations.
3) Pesquisa Clínica looks at increasing the understanding of what works best for patients regarding diagnosis, treatment, and care management. Diagnostic research is important to help patients arrive at a diagnosis early in disease progression when less irreversible damage may have occurred to organs. Treatment and care management research allows an increased understanding of what treatments and care bring about the best outcomes for patients.
Algumas das pesquisas clínicas sobre a ECD realizadas até o momento têm se concentrado não apenas em melhores opções de diagnóstico e tratamento em geral, mas também em compreender melhor como a ECD afeta diferentes órgãos, principalmente o cérebro e o sistema neurológico.
A ECDGA também financia, em parte, um Registro de Pacientes com ECD which is a database to collect patients’ medical histories in an organized format. This allows information about patients who are seen at many different institutions to be analyzed together in a more holistic way. By analyzing the ECD population, scientists can begin to see patterns and clues that will help understand ECD and hopefully find the most effective treatments in a given circumstance. The ECDGA has also partnered with the National Institute of Health for a Natural History Study of ECD. The collected data continues to be used to further research.
Para garantir que os avanços futuros no conhecimento sobre a ECD estejam alinhados com as questões mais relevantes para os pacientes e suas famílias, o Conselho Administrativo da ECDGA está atualmente avaliando a possibilidade de financiar projetos de pesquisa baseados nas prioridades dos pacientes e cuidadores, além das pesquisas propostas de acordo com as necessidades da comunidade médica.
Para compreender as prioridades e necessidades dos pacientes e de seus familiares, foi elaborado um questionário com o objetivo de coletar opiniões da comunidade. Convidamos todos os pacientes com ECD e seus familiares a preencher e enviar este questionário antes de 30 de outubro de 2021.
As informações coletadas na pesquisa serão analisadas e discutidas com a comunidade médica especializada em desenvolvimento na primeira infância (ECD) para identificar possíveis projetos de pesquisa que possam gerar conhecimentos que sejam mais significativos para a comunidade.
Caso ainda não tenha feito isso, pedimos que preencha a pesquisa clicando no botão abaixo.
Saiba mais:
Estudos e ensaios clínicos em andamento
Financiamento por meio de subsídios do ECD