The use of nonrandomized evidence to estimate treatment effects in health technology assessment.

Health technology assessment (HTA) is increasingly informed by nonrandomized studies, but there is limited guidance from HTA bodies on expectations around evidence quality and study conduct. We developed recommendations to support the appropriate use of such evidence based on a pragmatic literature review and a workshop involving 16 experts from eight countries as part of the EU's Horizon-2020 IMPACT-HTA program (work package six). To ensure HTA processes remain rigorous and robust, HTA bodies should demand clear, extensive and structured reporting of nonrandomized studies, including an in-depth assessment of the risk of bias. In recognition of the additional uncertainty imparted by nonrandomized designs in estimates of treatment effects, HTA bodies should strengthen early scientific advice and engage in collaborative efforts to improve use of real-world data.

Optimal time-points for minimal residual disease monitoring change on the basis of the method used in patients with acute myeloid leukemia who underwent allogeneic stem cell transplantation: a comparison between multiparameter flow cytometry and Wilms' tumor 1 expression.

Minimal residual disease (MRD) of 30 adult AML patients was monitored by multiparameter flow cytometry (MFC) and WT1 expression before and after allogeneic stem cell transplantation (allo-SCT). Diagnostic performance of pre-transplant MRD measured by MFC was higher than that obtained by WT1 expression. Comparable results were displayed at day +30 post-transplant, while better values by WT1 compared to MFC were found at day +90. Positive MRD by MFC predicted a shorter disease free survival (DFS) before and 1 month after transplant (p=0.006 and p=0.005), while only high WT1 levels at 1 month from the transplant significantly impacted on DFS (p=0.010). Our results support the idea that MRD monitoring by MFC should be suggested before and 30 days after the transplant, while WT1 expression should be preferred after this procedure. The assessment of MRD at day +30 from allo-SCT is recommended as post transplant check-point for the predictive role displayed, independently of the method used.

Cognitive decline in Dravet syndrome: is there a cerebellar role?

PURPOSE: The aim of the study was to perform a detailed assessment of cognitive abilities and behaviour in a series of epileptic patients with Dravet syndrome (DS) in order to establish a possible cerebellar-like pattern. METHODS: Nine children with DS without major behavioural disturbances and with cognitive abilities compatible with the assessment of specific cognitive skills (IQ>45) were enrolled in the study, in parallel with another group of nine epileptic patients (cryptogenic or symptomatic with minor brain injuries) consecutively admitted into the hospital matched for chronological age and IQ. All cases underwent neurological examination, long term EEG monitoring, neuroimaging and genetic analysis as well as a neuropsychological assessment including specific cognitive skills. RESULTS: On neurological examination 8 of the 9 DS patients had cerebellar signs, which were mild in six and more severe in the other two cases. DS patients had a constant discrepancy between verbal and performance items scales (verbal better than visual-spatial) that was not found in the control group. As to specific cognitive competence, the DS patients differ from the control group in the pattern of cognitive defects involving four main areas of cognitive abilities (a) expressive language with relatively spared comprehension, (b) visual-spatial organization, (c) executive function defects, (d) behavioural disorders. CUNCLUSIONS: The pattern of cognitive difficulties found in DS patients is consistent with what is reported in literature as cerebellar cognitive syndrome and may account for a possible cerebellar origin (at least as co-factor) of the cognitive decline observed in DS patients, as suggested by other clinical and experimental studies.

A review of evidence on equitable delivery, access and utilization of immunization services for migrants and refugees in the WHO European Region

This review focuses on existing immunization policies and practices for migrants and refugees and provides an overview of barriers and facilitators for access to and utilization of immunization services. Evidence was obtained by a scoping review of academic and grey literature in English and a further 11 languages and included official documents available from the websites of ministries of health and national health institutes of the WHO European Region Member States. The review highlights that vaccination policies tailored to migrants and refugees are very heterogeneous among WHO European Region Member States. By comparison, common barriers for the implementation and utilization of immunization services can be identified across countries. Outlined policy options are intended to strengthen information about immunization for migrants and refugees, support future evidence-informed policy-making, enable the achievement of national vaccination coverage goals and improve the eligibility of migrants and refugees to access culturally competent immunization services.

Public health aspects of migrant health: a review of the evidence on health status for undocumented migrants in the European Region

Undocumented migrants are people within a country without the necessary documents and permits. They are considered at higher risk for health problems because of their irregular status and the consequences of economic and social marginalization. A systematic review found 122 documents that suggested policies and interventions to improve health care access and delivery for undocumented migrants. Undocumented migrants mostly have only access to emergency care across Europe, and even in the countries where they are fully entitled to health care, formal and informal barriers hinder their access. This raises concerns for both public health and migrant care. On the basis of findings, policy options are suggested regarding data collection, research, entitlement to health care, information and communication, training and intersectoral approaches.