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Methods
PMC10504071
Study design
CRB-401 (
PMC10504071
Patients
DISEASE
Patients with RRMM who were ≥18 years of age and had ECOG PS 0-1 were eligible for the study. Patients were required to have measurable disease (serum M-protein ≥0.5 g dl
PMC10504071
Treatment
PMBCs obtained from leukapheresis were stimulated with αCD3 and αCD28 antibodies, transduced with the anti-BCMA CAR-containing lentiviral vector and expanded over 10 d as previously described
PMC10504071
Assessments
MULTIPLE MYELOMA
The IMWG Uniform Response Criteria for Multiple Myeloma were used to assess response and progression
PMC10504071
Translational
Cellular kinetics (PB and bone marrow) and sBCMA were evaluated as previously described
PMC10504071
PBMC and product characterization
PBMC used for ide-cel manufacturing and DP characteristics were evaluated from cryopreserved samples produced during the ide-cel clinical manufacturing process. PBMC or DP samples were thawed, washed and stained with cocktails of fluorescently labeled antibodies targeting T cell differentiation, exhaustion and senescen...
PMC10504071
Statistical analysis
The sample size planned for this study was based on clinical considerations and a standard dose-escalation design, as previously describedAnalyses were performed with SAS software, version 9.4.
PMC10504071
Reporting summary
Further information on research design is available in the
PMC10504071
Online content
Any methods, additional references, Nature Portfolio reporting summaries, source data, extended data, supplementary information, acknowledgements, peer review information; details of author contributions and competing interests; and statements of data and code availability are available at 10.1038/s41591-023-02496-0.
PMC10504071
Supplementary information
The online version contains supplementary material available at 10.1038/s41591-023-02496-0.
PMC10504071
Acknowledgements
Media
The study was supported by Celgene, Bristol Myers Squibb, and 2Seventy Bio (previously bluebird bio). The authors thank the study patients and their families and the clinical study teams. Writing and editorial assistance were provided by P.J. Simon of MediTech Media and A.L. Loeb and R. Beck Jr of SciMentum, both of wh...
PMC10504071
Author contributions
Y.L., N.S.R., M.V.M., O.F., N.M., J.F., T.B.C., K.H. conceived, designed or planned the study. Y.L., N.S.R., J.G.B., D.S.S., S.J., D.M., M.L., J.R., M.V.M., M.M., F.P., A.Y., N.C.M., J.N.K. acquired the data. A.Y., Z.Y., T.B.C., N.M., K.H., J.F. analyzed the data. Y.L., D.S.S., A.Y., O.F., T.B.C., N.M., A.C., Z.Y., K.H...
PMC10504071
Peer review
PMC10504071
Data availability
De-identified and anonymized data will be made available within a secured portal to qualified researchers who submit an in-scope proposal approved by the Independent Review Committee. Proposals will be reviewed to ensure that there is adequate scientific rationale and methodology, a robust statistical analysis plan and...
PMC10504071
Competing interests
NSR, Cancer
ONCOLOGY, CANCER, MYELOMA, EMD
Y.L. received institutional grants and consulting fees from Bristol Myers Squibb, Kite-Gilead and Janssen; institutional grants only from 2Seventy Bio, Merck and Takeda; institutional consulting fees only from Novartis, CellMedica and Legend Biotech; and participated in a data safety monitoring or advisory board for Pf...
PMC10504071
References
PMC10504071
Abstract
PMC10154863
Introduction
breast cancer
SECONDARY, BREAST CANCER
Understanding participants' concerns and information needs regarding broadened consent is crucial to ensure transparency and participant autonomy. Our study qualitatively examined these issues in women participating in the Personalized RISk‐based MAmmascreening study (PRISMA). The original PRISMA informed consent was p...
PMC10154863
Methods
Focus groups (FGs) were performed following a semistructured discussion guide. Two independent researchers analysed the data thematically using an inductive approach.
PMC10154863
Findings
breast cancer
BREAST CANCER
Twenty‐three asymptomatic women and 13 women diagnosed with breast cancer were randomly divided into six FGs. Four superordinate themes were identified: (1) Normalization, (2) Attitude towards the pharmaceutical industry, (3) Privacy and (4) Knowledge. Our participants viewed data sharing as an important conduit for ad...
PMC10154863
Conclusion
Our participants expressed clear information needs and a strong desire to be actively engaged in future data sharing decisions. Given that many researchers collaborate with commercial parties, building public confidence in these institutions would be beneficial. Illustrative examples addressing privacy concerns and cla...
PMC10154863
Patient and Public Contribution
breast cancer
BREAST CANCER
Women were recruited from the general breast cancer screening population. Their perceptions and information needs, as reported in this study, will not only inform broadened consent for PRISMA but ideally guide other consent templates and decisions regarding consent processes.
PMC10154863
INTRODUCTION
breast cancer
BREAST CANCER
Informed consent refers to the autonomous permission granted for an activity with the underlying premise that the individual fully comprehends what that activity entails.Disconcertingly, there is no globally accepted consensus on the definition of broad consent. Following guidance from the World Medical Association's 2...
PMC10154863
METHODS
PMC10154863
Design
Focus groups (FGs) were performed following a semistructured discussion guide to explore PRISMA participants' perceptions of expanding the boundaries of their previous consent. Per the Regional Ethics Committee CMO Arnhem‐Nijmegen Ethics Committee (the Netherlands), this study fell outside the remit of the Dutch Medica...
PMC10154863
Setting
Cancer, breast cancer
CANCER, BREAST CANCER
PRISMA is an observational prospective cohort study which was carried out in the Netherlands between 2014 and 2019. Since participants were recruited from the Dutch breast cancer screening programme, in line with screening eligibility criteria, all participants were asymptomatic females aged between 50 and 75 years. Fo...
PMC10154863
Original PRISMA consent protocol
breast cancer
BREAST CANCER
Participants in the PRISMA study were asked to consent to use their data and biomaterial for research into early detection, prognosis and treatment of breast cancer. They were further asked to consent to use their data and any residual biomaterial for ancillary studies into breast cancer; this included the possibility ...
PMC10154863
PRISMA Biobank
Biomaterials are stored at the Radboud Biobank, a hospital‐integrated central biobanking facility located within the Radboud University Medical Center (Radboudumc), Nijmegen, the Netherlands.
PMC10154863
Participants
breast cancer
BREAST CANCER
Women were randomly selected from the PRISMA database using a digital random number calculator, stratified across asymptomatic participants and incident breast cancer cases. This stratification aimed to explore whether breast cancer status affects women's opinions of data sharing. Inclusion criteria included: Consented...
PMC10154863
Procedures
Due to the Covid‐19 pandemic and subsequent government regulations, the FGs were performed digitally. We used Zoom,The discussion guide, available in Supporting Information: FGs were recorded and transcribed verbatim. FGs were organised until data saturation was achieved, that is, until no new themes were raised. Parti...
PMC10154863
Data analysis
Data were thematically analyzed by two independent researchers (L. R., M. d. J.) using an inductive approach.Final thematic map of perspectives on broadened consent: Themes and subthemes.
PMC10154863
FAIR data statement
The authors are highly committed to FAIR data to ensure that data are Findable, Accessible, Interoperable and Reusable.
PMC10154863
FINDINGS
breast cancer
BREAST CANCER
Twenty‐three asymptomatic women (response: 5.8%) and 13 women who had been diagnosed with breast cancer (response: 13.0%) participated in our study. We organized six FGs ranging in size from four to seven women; on average, there were six women per group. The average age of participants was 62 years (SD: 7; range: 52–7...
PMC10154863
Theme 1: Normalisation
Participants perceived data sharing as
PMC10154863
Theme 2: Attitude towards the pharmaceutical industry
EVENTS
Attitude towards the pharmaceutical industry was a major theme in the FGs and it was evident that topical events, namely the COVID‐19 pandemic, influenced this discussion. Participants expressed a great
PMC10154863
Theme 3: Privacy
Concerns about privacy were raised frequently. In general, participants agreed that data sharing is acceptable as long as their data are de‐identified and the key to their identifiable information is kept at the university, for example, ‘If anonymity can be guaranteed, I'll allow data sharing’ and ‘I assume that the li...
PMC10154863
Theme 4: Knowledge
Choosing whether or not to be informed about future findings, particularly from genetic research, was critical for participants. Some felt that it is their
PMC10154863
Procedural preferences and information needs
RISk‐based, death, ’
Participants drew on both their professional and personal experiences when stating their procedural preferences and information needs. Although broadly posed, this question elicited quite specific responses. One participant who worked in the area of knowledge transfer, for example, mentioned the availability of a ‘soci...
PMC10154863
DISCUSSION
cancer, confusion, fatigue, breast cancer
CANCER, MINOR, LENS, BREAST CANCER
In our study, participants could reflect on their previous project‐specific consent when discussing under what conditions they would be willing to broaden their consent. Since this was not solely a hypothetical exercise, it was evident that their receptiveness to broadened consent was largely rooted in their underlying...
PMC10154863
CONCLUSION
Our participants expressed very clear information needs and a strong desire to be actively engaged in future data sharing decisions. Given that many researchers collaborate with commercial parties and/or pharmaceutical companies, building public confidence in these institutions may be of benefit. Illustrative examples ...
PMC10154863
AUTHOR CONTRIBUTIONS
PMC10154863
CONFLICT OF INTEREST STATEMENT
The authors declare no conflict of interest.
PMC10154863
Supporting information
Supplementary information.Click here for additional data file.Supplementary information.Click here for additional data file.
PMC10154863
ACKNOWLEDGEMENTS
Cancer
CANCER
The authors want to acknowledge the considerable contribution and time investment of all the participating women, without whom this research would not have been possible. This study was funded by the Dutch Cancer Society (KWF) under Grant 12522. The funder had no role in the study design; in the collection, analysis an...
PMC10154863
DATA AVAILABILITY STATEMENT
Supporting Information: 
PMC10154863
REFERENCES
PMC10154863
1. Introduction
First-Episode Psychosis, AQ-27, insidious, psychosis, mental disorders
SECONDARY
Background: Mental-health-related stigma prevents active help seeking and therefore early therapeutic approaches and the recovery of functionality. National and international agencies recommend the implementation of prevention and mental health promotion programs that support the elimination of stigma in the classroom,...
PMC10671828
2. Material and Methods
PMC10671828
2.1. Design
SECONDARY
This was a quasi-experimental (pre and post-test) clustered pilot study carried out in a secondary school located in Spain. For this reason, the TREND checklist (Transparent Reporting of Evaluations with Nonrandomized Designs) was used [
PMC10671828
2.2. Participants and Recruitment
SECONDARY, RECRUITMENT
The sample was composed of teachers and counsellors of a secondary school located in Spain. It was selected out of a simple random sampling between the secondary schools to which we had access that belong to the department in Spain. The software programme ExcelThis type of population was chosen since they are considere...
PMC10671828
2.3. Inclusion and Exclusion Criteria
SECONDARY
Inclusion criteria: To work as a teacher and/or counsellor at the moment of the programme presentation, to have Spanish as mother tongue, to develop the teaching activity full-time, to voluntarily participate, and to be public, semi-private, and private secondary schools.Exclusion criteria: Not having signed the inform...
PMC10671828
2.4. Assessment Instruments
PMC10671828
2.4.1. Stigma
psychosis, AQ-27, Anger
DISORDER
The tool used for the assessment of stigma was the Attribution Questionnaire (AQ-27) (created by Weiner and collaborators) “(1988)” in its Spanish version, which is composed of 27 items (AQ-27-E). The evaluation was carried out by Muñoz and collaborators “(2015)”, obtaining a reliability of 0.855 (Cronbach’s alpha) for...
PMC10671828
2.4.2. Satisfaction Survey
The satisfaction survey adapted from Feixas and Vilaplana [
PMC10671828
2.5. Intervention
schizoaffective disorder, psychosis
DISORDERS
A training workshop on psychosis and general mental health was carried out in order to raise the awareness of the teaching staff and counselling department. It was a psychoeducational-type intervention that was fundamentally inspired by the content of other guides, recommendations, and renowned entities in the field of...
PMC10671828
2.6. Data Analysis
All the statistical analyses were performed with IBM SPSS Statistics version 23. The level of significance was established with a Univariate analysis. The baseline sociodemographic and clinical features of the group were described using measures of central tendency for quantitative variables, and total frequency table ...
PMC10671828
3. Results
PMC10671828
3.1. Characteristics of Patients
A total of 22 participants (18% men and 82% women) were recruited for this study and completed the pre- and post-intervention questionnaires. The sample profile was mostly women aged 47.96 ± 10.12 years, with undergraduate studies and a professional experience of 18.64 ± 10.34 years. The rest of the demographic charact...
PMC10671828
3.3. Correlations “Age”–“AQ-27 Pre-Intervention” and “Experience”–“AQ-27 Pre-Intervention”
These correlations can be seen in
PMC10671828
3.4. Correlations of “Psychosis Test”–“Age” and “Psychosis Test”–“Professional Experience”
As seen in
PMC10671828
3.5. Psychosis Test
The mean score obtained before the intervention was 4.89 out of 10.
PMC10671828
3.6. Course Rating
Points are shown in
PMC10671828
3.7. Semi-Structured Interview
Since the end of the program, counsellors reported 11 cases referred to the health system, 9 of which required assessment and follow-up by the Child and Adolescent Mental Health Unit: 5 boys and 4 girls between 13 and 16 years due to behavioural alterations and anxious symptoms.
PMC10671828
4. Discussion
substance-induced psychosis, illness, Anger
SECONDARY, SAID, DISORDERS
From what is known, this is the first study that has explored the effect of a psychoeducational programme on mental health and FEP aimed at teachers and counsellors in a secondary school in Spain. The outcomes showed that the intervention was useful for increasing knowledge on mental health and reducing the stigma asso...
PMC10671828
4.1. Strengths and Limitations
psychosis
Since the study was carried out voluntarily in real life in La Ribera health department, it was not possible to randomise the participants, there could not be a control group, the sample could not be randomised given the prior need of planning lessons, and the intervention content had to be adapted to the available tim...
PMC10671828
4.2. Future Lines
psychosis
Given the promising data of this Mental Health Nursing psychoeducational-type intervention, its performance in other educational centres is suggested, taking into account that the sample should be more homogeneous and representative, preferably scheduled within work hours and with the possibility of obtaining further o...
PMC10671828
5. Conclusions
First-Episode Psychosis, mental disorder, psychosis
SECONDARY
The impact of a nursing intervention on mental health stigma in teachers and counsellors of a secondary school was positive, given the statistically significant differences observed in the change of average scores on stigmatising attitudes of the AQ-27 scale pre- and post-intervention. The stigmatising attitude that pr...
PMC10671828
Author Contributions
Conceptualization, L.S.A., L.A.P. and J.V.C.-S.; methodology, L.S.A., L.A.P. and J.V.C.-S.; validation, L.S.A., L.A.P., A.R.-H. and J.J.G.C.; formal analysis, L.S.A. and J.V.C.-S.; investigation, L.S.A. and J.V.C.-S.; resources, C.M.M., E.G.P. and S.C.P.; data curation, J.V.C.-S.; writing—original draft preparation, L....
PMC10671828
Institutional Review Board Statement
Ethical approval was granted from the Ethics Committee-Research Commission of the pertinent Health Department [PI06072022]. All participants gave written permission for their anonymized data to be used for academic purpose.
PMC10671828
Informed Consent Statement
All participants gave written permission for their anonymized data to be used for academic purpose.
PMC10671828
Data Availability Statement
Data available on request due to privacy and ethical restrictions. Data presented in this study are available upon request from the corresponding author.
PMC10671828
Conflicts of Interest
There were no conflicts of interest with any person, company and/or institution that could affect the conduct of this research.
PMC10671828
Clinical Relevance
There are adolescents who have suffered stigma from their teachers, and consequently have minimized the symptoms and have not asked for help. For this reason, we have implemented a nursing intervention, based on education and promotion of mental health with the aim of expanding knowledge and reducing stigma. In fact, t...
PMC10671828
References
Psychosis, AQ-27
Sociodemographic data of the sample.AF: Absolute Frequency.Correlations of the AQ-27 variable pre- and post-intervention. Wilcoxon Test.* Correlations between the variables “Age” and “AQ-27 pre-intervention”.* Correlations between the variable “Professional experience” and “AQ-27 pre-intervention”.* Correlations of “Ps...
PMC10671828
Keywords
PMC10699085
Trial structure
In broad terms, the SNAP trial model is designed around several ‘structural’ trial elements: silos, domains, subdomains, subgroups, covariates, regions and countries, and epochs. In a slight abuse of notation, for simplicity, we will often use
PMC10699085
Domains
A Domains are denoted by
PMC10699085
Subdomain
For participants within a specific silo, it is possible for the set of allocated interventions within each domain to be a subset of the full set of interventions available for that domain. We define the set of these silo-specific domain interventions by:This silo-specific set of domain interventions leads us to the def...
PMC10699085
Subgroups
A
PMC10699085
Covariates
We define Values of a particular covariate are labelled by the lower-case letter associated with that covariate indexed by covariate specific subscript
PMC10699085
Regions and countries
A We define
PMC10699085
Epochs
The concept of an
PMC10699085
Statistical modelling
The SNAP trial data will be analysed using Bayesian statistical methods, which combine probability distributions that summarise the state of knowledge independent of the observed data (a prior probability distribution) with the observed data model (through a likelihood function) to produce probability distributions tha...
PMC10699085
Models
PMC10699085
Primary endpoint model
The SNAP trial primary endpoint is binary, denoted We interpret the
PMC10699085
Prior distributions and model hierarchy
The following subsections outline the prior distribution structure for the parameters of the model using a Bernoulli distributed endpoint and logistic link function, including the primary model. The model for alternatively distributed endpoints will require an alternative prior structure, which will be described in det...
PMC10699085
Reference log-odds
The log-odds for domain-specific interventions
PMC10699085
Effects of interventions
The log-odds Where a silo has a unique subdomain (i.e. it exists only for a single silo), the log-odds ratios where the intervention is silo-specific are modelled as normally distributed such that all subgroups have the same silo-specific mean and variance: Where two or more silos share a subdomain, and the log-odds ra...
PMC10699085
Effects of domain eligibility
The log-odds ratios for domain ineligibility are assigned independent normal prior distributions such that:
PMC10699085
Effects of two-way interactions
The log-odds ratios for two-way interactions are assigned independent normal prior distributions such that:
PMC10699085
Effects of regions and countries
The log-odds ratios for regions are assigned normal distributions such that:
PMC10699085
Effects of covariates
The log-odds ratios for covariate factors are assigned independent normal prior distributions such that:
PMC10699085
Effects of epoch
Temporal trends will be accounted for by splitting the trial sample into separate cohorts defined by contiguous 26 week epochs and using first-order dynamic normal linear model within (Furthermore, an additional sensitivity analysis may be performed to evaluate all outcomes of interest using cohorts that are restricted...
PMC10699085
Exploratory analyses
Additional analyses are described in the SNAP statistical appendix and relevant domain-specific appendices that include parameters for the interactions between interventions and other covariates to enable the comparative effectiveness by covariates as a pre-planned exploratory analysis.
PMC10699085
Computational methods
The joint posterior probability distributions of the model parameters described in the preceding sections are analytically intractable, and therefore computational Bayesian methods will be used for the data analyses. We will use Markov-chain Monte Carlo (MCMC) methods implemented in stan, a probabilistic programming la...
PMC10699085
Missing data
Death
Death from
PMC10699085
Concurrently randomised cohorts
By design, the way that participants are assigned to treatments in adaptive randomised trials changes over time, which can lead to treatment-outcome confounding in certain cases. For when that confounder is based on calendar time, we have included epoch modelling as one countermeasure to confounding (see the ‘As a furt...
PMC10699085
Randomisation
PMC10699085
General principles
Consented participants will be randomly allocated among interventions in subdomains for which they are eligible. Participants will be randomised to one intervention from each domain that their enrolling site is participating in, according to allocation probabilities detailed in each domain-specific appendix and stratif...
PMC10699085