Background Metachromatic Leukodystrophy (MLD) is a rare lysosomal disorder. Patients suffer from relentless neurological deterioration leading to premature death. Recently, new treatment modalities, including gene therapy and enzyme replacement therapy, have been developed. Those advances increase the need for high-quality research infrastructure to adequately compare treatments, execute post-marketing surveillance, and perform health technology assessments (HTA). To facilitate this, a group of MLD experts started the MLD initiative (MLDi) and initiated an academia-led European MLD registry: the MLDi. An expert-based consensus procedure, namely a modified Delphi procedure, was used to determine the data elements required to answer academic, regulatory, and HTA research questions. Results Three distinct sets of data elements were defined by the 13-member expert panel. The minimal set (n = 13) contained demographics and basic disease characteristics. The core set (n = 55) included functional status scores in terms of motor, manual, speech and eating abilities, and causal and supportive treatment characteristics. Health-related quality of life scores were included that were also deemed necessary for HTA. The optional set (n = 31) contained additional clinical aspects, such as findings at neurological examination, detailed motor function, presence of peripheral neuropathy, gall bladder involvement and micturition. Conclusion Using a modified Delphi procedure with physicians from the main expert centers, consensus was reached on a core set of data that can be collected retrospectively and prospectively. With this consensus-based approach, an important step towards harmonization was made. This unique dataset will support knowledge about the disease and facilitate regulatory requirements related to the launch of new treatments.

Background: Despite high prevalence of mental problems among elderly migrants in The Netherlands, the use of psychosocial care services by this group is low. Scientific evidence points at the crucial role of social support for mental health and the use of psychosocial services. We therefore explored the role of social networks in the access to psychosocial care among elderly migrants in The Netherlands. Methods: A qualitative study was conducted using semi-structured group interviews and individual interviews. The eight group and eleven individual interviews (respectively n = 58 and n = 11) were conducted in The Netherlands with Turkish, Moroccan, Surinamese, and Dutch elderly. The data were analysed through coding and comparing fragments and recognizing patterns. Results: Support of the social network is important to navigate to psychosocial care and is most frequently provided by children. However, the social network of elderly migrants is generally not able to meet the needs of the elderly. This is mostly due to poor mental health literacy of the social network, taboo, and stigma around mental illness and the busy lives of the social network members. Conclusions: Strategies to address help-seeking barriers should consider mental health literacy in elderly migrants as well as their social networks, and counteract taboos and stigma of mental health problems.