The Vall d'Hebron Research Institute (VHIR) recently hosted a multidisciplinary meeting of the national INTEGRA programme, bringing together around twenty haematologists, paediatricians, nurses, researchers, psychologists, patients and caregivers from various hospitals and from the Spanish Sickle Cell Disease Association (ASAFE). The meeting aimed to advance the integration of Patient-Reported Outcome Measures (PROMs) into the research programme on sickle cell disease and other rare anaemias.

The INTEGRA programme, led by Dr María del Mar Mañú Pereira, Head of the Rare Anemic Disorders Laboratory in the Childhood Cancer and Hematological Diseases Group at VHIR, was launched five years ago with the creation of Spain's largest national registry of patients with sickle cell disease. The registry currently includes clinical, demographic, genomic and functional study data from 381 individuals, representing approximately 40% of patients with this disease in Spain. In recent years, the programme has expanded its scope by incorporating metabolomic studies, extending its work to other rare anaemias, and integrating PROMs to strengthen a patient-centred research model.

The meeting focused on one of the main challenges in rare disease research: ensuring that clinical studies assess not only medical parameters but also the impact of the disease on people's daily lives. Through the active participation of patients and caregivers in the development of these tools, the INTEGRA programme aims to provide instruments that better reflect the needs and experiences of those affected.

During the meeting, participants addressed topics including the selection of clinically relevant PROMs, the methodological challenges involved in their collection, their integration into multimodal research platforms, the influence of socioeconomic determinants, and the co-design of digital data collection tools with patients and caregivers.

The meeting also highlighted the ongoing collaboration with ASAFE, an organisation that has played a key role in promoting patient participation within the project. Within the framework of the INTEGRA and ERDERA projects, VHIR presented a €7,500 contribution to support the association's activities and strengthen its role in patient-centred research initiatives.

This meeting represents a further step in consolidating the INTEGRA programme as a national research platform for rare anaemias, promoting a collaborative model that brings together healthcare professionals, researchers, patients and caregivers with a shared goal: generating scientific evidence that better reflects the outcomes and experiences that truly matter to patients. Following the meeting, this work has already led to the launch of the digital tool, co-designed with patients and caregivers, for the collection of PROMs.

The INTEGRA program is partially funded by the Instituto de Salud Carlos III (ISCIII) through the REAL-RED project, PI24/00477, co-funded by the European Union, and by ERDERA under Grant Agreement Nº 101156595, also co-funded by the European Union.