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Progression-free survival
EVENTS
PFS events occurred in 6/26 patients in Part A (median follow-up, 35.8 months; range, 3.5–46.1) and in 10/45 patients in Part B (median follow-up, 25.3 months; range, 1.5–32.8). Median PFS for the entire cohort was not reached (NR; interquartile range [IQR]: 28.6 months to NR) (Fig. 
PMC10264885
Overall survival
EVENTS
OS events (any cause) occurred in 2/26 patients in Part A (median follow-up, 37.9 months; range, 4.6–47.0) and in 7/45 patients in Part B (median follow-up, 26.2 months; range, 9.5–33.1). Median OS estimates for Parts A and B separately and for the entire cohort were NR (range, NR–NR) (Fig. 
PMC10264885
Pharmacokinetics
Based on results from the PK population for NCA on Cycle 1, when given in combination with VRd, isatuximab PK parameters (area under the plasma concentration versus time curve from 0 to 1 week [AUC
PMC10264885
Immunophenotyping
Flow cytometry experiments were conducted to explore changes in the immune microenvironment upon treatment. Data from Parts A and B were obtained from peripheral blood collected at Day 1 of Cycle 1 (27 and 43 patients, respectively), Day 1 of Cycle 3 (20 and 38 patients), and end of treatment (8 and 9 patients) for the...
PMC10264885
Discussion
This Phase 1b study was designed to investigate Isa-VRd for the first time in adult patients with NDMM who were ineligible/had no intent for immediate transplantation. Treatment with isatuximab plus triplet bortezomib-lenalidomide-dexamethasone resulted in deep and durable responses, with an MRD- rate of 51% at a sensi...
PMC10264885
Supplementary information
The online version contains supplementary material available at 10.1038/s41375-023-01936-7.
PMC10264885
Acknowledgements
The authors thank the participating patients and their families, and the study centers and investigators for their contributions to the study. Medical writing support was provided by Erin Burns-Tidmore, PhD, of Envision Pharma Group, contracted by Sanofi for publication support services.
PMC10264885
Author contributions
EMO, AP, PB, JFSM, IWB, LK, JM-L, WP, SB, MM, M-VM, PRO, and PM were investigators in the study and contributed to data acquisition. LD, SM, TF, NLR, and MG contributed to the analysis, verification, and interpretation of data for the work. All authors revised the work for important intellectual content and assume resp...
PMC10264885
Funding
This study was sponsored by Sanofi.
PMC10264885
Data availability
Qualified researchers can request access to patient-level data and related study documents including the clinical study report, study protocol with any amendments, blank case report forms, statistical analysis plan, and dataset specifications. Patient-level data will be anonymized, and study documents will be redacted ...
PMC10264885
Competing interests
MM
EMO: Honoraria – Amgen, BMS/Celgene, GSK, Janssen, MSD, Oncopeptides, Sanofi, Takeda. AP: Honoraria – AbbVie, Amgen, BMS/Celgene, GSK, Janssen, Sanofi, Takeda. PB: nothing to disclose. JFSM: Honoraria – AbbVie, Amgen, BMS, Celgene, GSK, Haemalogix, Janssen, Karyopharm, MSD, Novartis, Regeneron, Roche, Sanofi, SecuraBio...
PMC10264885
References
PMC10264885
Introduction
Symptoms reported following the administration of investigational drugs play an important role in decisions for registration and treatment guidelines. However, symptoms are subjective, and interview methods to quantify them are difficult to standardise. We explored differences in symptom reporting across study sites of...
PMC10476314
Methods
Data were derived from the IMPROV trial, a randomised, placebo-controlled double blinded trial of high dose primaquine to prevent
PMC10476314
Results
anorexia, fever
ANOREXIA
A total of 2,336 patients were included. The greatest variation between sites in the proportion of patients reporting symptoms was for anorexia between day 0 and day 13: 97.3% (361/371) of patients in Arba Minch, Ethiopia, reported the symptom compared with 4.7% (5/106) of patients in Krong Pa, Vietnam. Differences att...
PMC10476314
Conclusion
Despite standardised training, there was large variation in symptom reporting across trial sites. The reporting of severe symptoms was less skewed compared to mild and moderate symptoms, which are likely to be more subjective. Trialists should clearly distinguish between safety and tolerability outcomes. Differences be...
PMC10476314
Trial registration
Clinicaltrials.gov: NCT01814683; March 20
PMC10476314
Supplementary Information
The online version contains supplementary material available at 10.1186/s12874-023-02022-3.
PMC10476314
Keywords
PMC10476314
Background
vivax malaria
ADVERSE EVENTS, VIVAX MALARIA
Clinical trials are designed to evaluate drug safety and efficacy. The assessment of safety usually involves identifying adverse events (AEs), that can be detected either by laboratory testing, clinical assessment or patient questioning for symptoms. While laboratory tests can be standardised, patient questioning metho...
PMC10476314
Methods
PMC10476314
Study design
Data used in this analysis were derived from the IMPROV trial. The design of the trial [
PMC10476314
Study sites
This study included patients recruited in eight study sites across four countries: Afghanistan, Ethiopia, Indonesia and Vietnam [
PMC10476314
Data collection of symptoms
diarrhoea, itching, abdominal pain, pain, dizziness, skin rash, nausea,, headache, aches, shortness of breath
At each follow up visit a symptom questionnaire was completed. Patients were encouraged to report to the study centre if they became unwell between scheduled visits. The symptom questionnaire consisted of a 13-point symptom checklist for fever/hot body, headache, muscle/joint aches and pain, abdominal pain, poor appeti...
PMC10476314
Data analysis
diarrhoea, nausea, fever, abdominal pain, malaria, vomiting, dizziness, parasitaemia, anorexia
REGRESSION, MALARIA, ANOREXIA
The number and proportion of patients who had the most clinically relevant symptoms (vomiting, diarrhoea, anorexia, nausea, abdominal pain and dizziness) following the administration of primaquine or placebo are presented by study site and treatment. The confounder-adjusted proportion of the presence of each symptom at...
PMC10476314
Variation across study sites
anorexia, fever
REGRESSION, ANOREXIA
The widest range of the proportion of patients reporting a specific symptom across the sites was for anorexia. A total of 97.3% (361/371) of patients in Arba Minch, Ethiopia reported anorexia between day 0 and day 13 compared with 4.7% (5/106) of patients in Krong Pa, Vietnam. The proportion of patients at the remainin...
PMC10476314
Variation between treatment arms
parasitaemia, anorexia, fever
REGRESSION, ANOREXIA
At the study site in Arba Minch, Ethiopia, anorexia between day 3 and day 13 was reported in 87.3% (62/71) of patients receiving placebo, 76.2% (112/147) of patients receiving low dose primaquine, and 83.3% (120/144) patients receiving high dose primaquine. In contrast, in Krong Pa, Vietnam, none (0/21) of the patients...
PMC10476314
Variation in severity of symptoms across study sites
diarrhoea, nausea, abdominal pain, vomiting, dizziness, anorexia
ANOREXIA
Of 1,747 patients reporting vomiting, diarrhoea, anorexia, nausea, abdominal pain or dizziness, 1,033 (59.1%) reported only mild symptoms (grade 1), 682 (39.0%) reported their most severe symptom as moderate (grade 2) and 32 (1.8%) reported their most severe symptom as severe (grade 3). For all reported symptoms, less ...
PMC10476314
Discussion
diarrhoea, nausea, fever, abdominal pain, malaria, vomiting, pain, dizziness, anorexia
MALARIA, EVENTS, DISEASE, ANOREXIA
The overall number of symptoms reported in the IMPROV study varied significantly between sites, and this difference remained apparent after adjusting for demographics, disease severity estimated by presence of fever and parasite density at enrolment, and treatment arm. One site in Ethiopia reported almost 100% of study...
PMC10476314
Conclusions
EVENTS
The findings from our analysis suggest three pragmatic approaches to how symptom reporting in antimalarial trials could be improved. Firstly, the reporting of more severe symptoms is less skewed between sites and more likely to be reproducible compared to mild and moderate events. Trialists should therefore distinguish...
PMC10476314
Acknowledgements
We wish to express our sincere gratitude to the patients for volunteering to join this study and to the nurses and laboratory staff.
PMC10476314
Authors’ contributions
MR, TTH
TSD
LvS, KT, RJC and MR conceived the idea for this study. LvS, JKB, NJW, NPD, and RNP conceived the IMPROV trial. TSD, KC, NHC, AA, MNN, APP, AGR, IS, NCT, TTH, AH, MAH, LLE, AW and TT were responsible for data collection. KT, RJC, MR and LvS did the data analyses and interpretation and wrote the first draft of the manusc...
PMC10476314
Funding
The IMPROV study was funded by the UK Department for International Development, UK Medical Research Council, UK National Institute for Health Research, and the Wellcome Trust through the Joint Global Health Trials Scheme (MR/K007424/1) and the Bill & Melinda Gates Foundation (OPP1054404). KT is funded through an CSL Ce...
PMC10476314
Availability of data and materials
The data are available for access via the WorldWide Antimalarial Resistance Network (WWARN.org). Requests for access will be reviewed by a Data Access Committee to ensure that use of data protects the interests of the participants and researchers according to the terms of ethics approval and principles of equitable dat...
PMC10476314
Declarations
PMC10476314
Ethics approval and consent to participate
The study received full ethical approval from the Human Research Ethics Committee of the Northern Territory Department of Health (HREC), the Oxford Tropical Research Ethics Committee (OxTREC), the Institutional Review Board at the Ministry of Public Health of the Islamic Republic of Afghanistan, the National Research E...
PMC10476314
Consent for publication
Not applicable.
PMC10476314
Competing interests
The authors declare no competing interests.
PMC10476314
References
PMC10476314
Background
tightness, knee osteoarthritis, KOA
KNEE OSTEOARTHRITIS
The association between hamstring tightness and knee osteoarthritis (KOA) is significant because tight hamstrings can put more strain on the knee joint, reduce its range of motion, and cause compensatory movements that worsen the KOA.
PMC10695107
Objective
proprioceptive neuromuscular, KOA
To compare the effects of instrument-assisted soft tissue mobilization (IASTM) and proprioceptive neuromuscular (PNF) on hamstring flexibility in patients with KOA.
PMC10695107
Methods
Data for the randomized controlled trial (NCT05110326) was collected from
PMC10695107
Results
The study found a significant interaction (
PMC10695107
Conclusions
pain
Both the IASTM technique and PNF stretching resulted in increased hamstring flexibility, decreased pain, and enhanced general health. The IASTM technique, however, showed potential benefits over PNF stretching in terms of flexibility, pain relief, and public health enhancement. Physical therapists and manual therapists...
PMC10695107
Introduction
injury or overuse damages the articular cartilage, osteoarthritis, muscle flexibility, musculoskeletal disease, knee osteoarthritis, KOA, overweight, articular cartilage loss, Knee osteoarthritis, disability, proprioceptive neuromuscular facilitation, trauma
DEGENERATION, CONTRACTIONS, OSTEOARTHRITIS, MUSCULOSKELETAL DISEASE, KNEE OSTEOARTHRITIS, KNEE OSTEOARTHRITIS
Knee osteoarthritis (KOA) is a painful musculoskeletal disease caused by degeneration and articular cartilage loss over time. KOA mostly affects the elderly population and commonly causes disability throughout the world (Age, congenital and acquired deformity, trauma and female gender are all risk factors that have bee...
PMC10695107
Methods
PMC10695107
Study design & setting
It was a single-blinded, randomized clinical trial (NCT05110326) conducted at the RHS Rehabilitation Centre (RHS/EC/28-06-2021-03), Islamabad, Pakistan. The study was completed within 1 year from July 2021-June 2022 and approval was taken from the research and ethical committee (REC) of the Faculty of Rehabilitation an...
PMC10695107
Participants
tightness, knee osteoarthritis, KOA
KNEE OSTEOARTHRITIS
The 35–50 years patient having grade 1&2 knee osteoarthritis (KOA), according to the Kellegren and Lawrence criteria, hamstring tightness of more than 20° from the active knee extension test (AKET) (
PMC10695107
Sample size
A total of
PMC10695107
CONSORT diagram.
PMC10695107
Randomization
The sealed enveloped method using a computerized random number generator was used for randomization. An individual who was not directly involved in the study did the random allocation. The random numbers were then written on the index cards and placed in a thick and opaque sealed envelope before the start of the study....
PMC10695107
Intervention
A 30-minute session was given to each patient 3 times per week and was given for 6 weeks. Baseline assessments were made prior to beginning the intervention and re-assessment was done after 6 weeks. The detail intervention protocol for group A (
PMC10695107
Intervention protocol.
PMC10695107
Assessments
Arthritis, knee osteoarthritis, pain
ARTHRITIS, KNEE OSTEOARTHRITIS
The researchers considered the ethical, legal and regulatory norms and standards for this research according to the Declaration of Helsinki as a statement of ethical principles for medical research involving human subjects, including research on identifiable human material and data.The visual analog scale (VAS) was use...
PMC10695107
Statistical methods
The descriptive statistics, such as frequency (n), percentage (%), mean, standard deviation (SD), and mean differences (MD), used to summarize the study’s findings and subsequently presented in tables and graphs. A two-way mixed ANOVA with partial eta squared (
PMC10695107
Results
The mean age and BMI of the study participants were 45.14 ± 4.67 years and 28.53 ± 5.65 kg/m
PMC10695107
Frequency distribution (BMI).
PMC10695107
Frequency distribution knee OA grades.
PMC10695107
Interaction effect.
To find the interaction effect between intervention and level of assessment, two-way mixed ANOVA was applied. As the sphericity was assumed, the results showed that there was a significant interaction effect between both interventions and time factor in all dependent variables with large effect size, including hamstrin...
PMC10695107
Discussion
tightness, reduced functional disability, knee osteoarthritis, pain
PATELLOFEMORAL PAIN SYNDROME, KNEE OSTEOARTHRITIS, ADHESIONS
This study was conducted to determine the effectiveness of the Instrument-Assisted Soft Tissue Mobilization (IASTM) technique and PNF stretching in improving hamstring flexibility, pain, and health status in knee osteoarthritis. In the present study within group analysis showed that participants in both groups had sign...
PMC10695107
Conclusion
knee osteoarthritis, pain
KNEE OSTEOARTHRITIS
This study indicated those with knee osteoarthritis benefited from both the IASTM technique and PNF stretching, resulting in increased hamstring flexibility, decreased pain, and enhanced general health. The IASTM technique, however, showed potential benefits over PNF stretching in terms of flexibility, pain relief, and...
PMC10695107
Supplemental Information
PMC10695107
CONSORT Checklist
Click here for additional data file.
PMC10695107
Trial Protocol
Click here for additional data file.
PMC10695107
Raw data
pain
SPSS or PSPP is required to view the datasetClick here for additional data file.Thanks to the participants of this study for sharing their personal experiences with pain.
PMC10695107
Additional Information and Declarations
PMC10695107
Competing Interests
The authors declare there are no competing interests.
PMC10695107
Human Ethics
The following information was supplied relating to ethical approvals (i.e., approving body and any reference numbers):The Research and ethical Committee (REC) of Faculty of Rehabilitation and Allied Health Sciences of RIphah International University approval to carry out the study (with Ref# Riphah/RCRS/REC-01055).
PMC10695107
Clinical Trial Ethics
The following information was supplied relating to ethical approvals (i.e., approving body and any reference numbers):The Research and ethical Committee (REC) of Faculty of Rehabilitation and Allied Health Sciences of RIphah International University approval to carry out the study (with Ref# Riphah/RCRS/REC-01055).
PMC10695107
Data Availability
The following information was supplied regarding data availability:The raw data is available in the
PMC10695107
Clinical Trial Registration
The following information was supplied regarding Clinical Trial registration:NCT05110326
PMC10695107
References
PMC10695107
Abstract
The authors declare no competing financial interests.Author contributions: N.F. and C.J.P. designed research; N.A.S.F. and C.J.P. performed research; N.A.S.F. contributed unpublished reagents/analytic tools; N.A.S.F. and Z.Z. analyzed data; N.A.S.F., Z.Z., and C.J.P. wrote the paper.This work was supported by a Researc...
PMC10295813
Significance Statement
CORTEX
Interoception, the representation of the body’s internal state, is poorly understood compared with the external senses, with existing neuroimaging studies failing to match task difficulty between interoceptive and exteroceptive tasks. The present study used a novel fMRI task to compare interoceptive and exteroceptive a...
PMC10295813
Introduction
RECRUITMENT
Interoception, the sense of the body’s internal state, is central to human experience, providing homeostatic cues (Our understanding has been limited by a focus on interoceptive accuracy by the research community. For example, heartbeat detection paradigms model the ability to detect a liminal cardiac signal (Research ...
PMC10295813
Materials and Methods
PMC10295813
Experimental design
This fully within-participant study was conducted to validate a novel interoceptive attention task as part of an NIH-funded pilot study, a two-group randomized control trial to examine the neural correlates of interoceptive awareness in the context of mindful awareness in body-oriented therapy (MABT) training (
PMC10295813
Participants
Twenty-two right-handed study participants (11 male and 11 female), of adult age (mean: 36.1 years, range: 18–62) completed both baseline and postintervention assessments. Eleven participants (50% of the sample) were randomly allocated to receive eight MABT sessions, delivered individually once per week for eight weeks...
PMC10295813
Ethics statement
All participants provided informed consent. The study procedures were reviewed and approved by the institutional review board at the University of Washington in accordance with the World Medical Association Declaration of Helsinki.
PMC10295813
The Interoceptive/Exteroceptive Attention Task (IEAT)
The Interoceptive/Exteroceptive Attention Task (IEAT) is a novel paradigm for exploring the neural dynamics of respiratory attention and awareness. The IEAT consisted of five conditions: Passive Exteroception, Passive Interoception, Active Interoception, Active Exteroception, and Active Matching (a paced breathing cond...
PMC10295813
Passive conditions
During Passive Exteroception, participants were asked to visually monitor a circle as it expanded and contracted periodically on the MRI-compatible visual display without making any behavioral responses. The circle’s pulse frequency was set to match participants’ in-scanner breathing frequency, obtained from a respirat...
PMC10295813
Active conditions
CONTRACTION
During Active Exteroception, participants reported on the expansion and contraction of the circle on the screen, which again was set to pulse at participants’ in-scanner respiratory frequency. During Active Interoception, participants reported on inhalations and exhalations by making button box key presses with their r...
PMC10295813
Interoceptive attention tendency
Perhaps the most common dispositional index of subjective interoceptive engagement is the Multidimensional Assessment of Interoceptive Awareness (MAIA), which in validation has demonstrated strong associations to subjective wellbeing (Here, the MAIA was used to provide a subjective report of the ability to adaptively e...
PMC10295813
Procedures
Participants completed the IEAT during fMRI acquisition at two timepoints, baseline and three-month follow-up. The MAIA self-report questionnaire was administered to assess interoceptive awareness at both timepoints. Training effects are the subject of separate reports.
PMC10295813
Data analysis
PMC10295813
Respiration confounds
Changes in breathing depth and rate modulate CO
PMC10295813
Respiration frequency
Respiration data were acquired using a MR-compatible respiration belt sampling at 500 Hz (Philips model 452213117812). Respiration data were first smoothed using a 1 s zero phase low-pass filter window and then mean-corrected. Breath frequency was then estimated using a fast Fourier transform (FFT) of the respiration p...
PMC10295813
Respiration volume/time
RVT
The respiratory signal is influenced by changes in respiratory volume in addition to frequency, with respiratory volume/time (RVT) predicting widespread changes in BOLD activity (To correct for RVT influence, belt amplitude signal was used to calculate RVT as recently recommend (
PMC10295813
Stimulus and behavior timeseries
RVT
CONTRACTION
The three active localizer conditions, Active Exteroception, Active Interoception, and Active Matching required button-presses to track the sensory target, i.e., inhalation/exhalation during the respiratory cycle, or expansion/contraction during the visual circle cycle. Each active condition therefore produced three pe...
PMC10295813
Tracking accuracy
NCC
To calculate trial-specific tracking accuracy in the active tracking conditions (Active Interoception, Active Exteroception, and Active Matching), the appropriate sensory stimulus waveform (respiration in Active Interoception, the visual circle in Active Exteroception) was first phase-corrected to maximally align with ...
PMC10295813
Neuroimaging data acquisition
PAD
Neuroimaging was performed using a 3T Philips Achieva scanner (Philips Inc.) at the Diagnostic Imaging Sciences Center, University of Washington. Imaging began with the acquisition of a T1-weighted anatomic scan (MPRAGE) to guide normalization of functional images (∼6 min) with TR = 7.60 ms, TE = 3.52 ms, TI = 1100 ms,...
PMC10295813
Preprocessing
A set of preprocessing steps was performed using the consortium-developed fMRIprep robust preprocessing pipeline for fMRI data (
PMC10295813
First-level analysis
RVT
Within-participant statistical models were used to characterize the neural distinction between task conditions. Participant time series data from the IEAT was submitted to separate first-level general linear statistical models using Statistical Parametric Mapping software (v12). Task-specific boxcar stimulus functions ...
PMC10295813
Second-level analysis
Participant first-level maps for each experimental condition [Passive Interoception, Passive Exteroception, Active Interoception, Active Exteroception, Active Matching] were analyzed at the second level using a full-factorial mixed-model ANOVA in SPM12 (Familywise control for multiple comparisons (corrected
PMC10295813
Trial-level confounds
RVT
The current study comes from an exploratory clinical trial, the results of which are the subject of a separate report. We combined data across the trial to power the comparison of IEAT task conditions and modelled any effects of trial Group (MABT vs Control), Time (Baseline vs Postintervention) and their interactions a...
PMC10295813
Region of interest (ROI) analysis
For region of interest (ROI) analysis, all signal extractions were taken from models containing the nuisance covariates. Using the built-in SPM12 function, the median value of the raw, unwhitened signal was extracted from all voxels within the ROI, yielding one value per participant at each scanning session. These valu...
PMC10295813
Hypothesis testing
RVT
Hypothesis 1 aimed to compare interoceptive and exteroceptive attention. To this end we first evaluated a whole-brain interaction between reporting demand [active vs passive] and attentional target [interoception vs exteroception] to evaluate whether the effects of attentional target should be evaluated separately for ...
PMC10295813
Test-retest reliability
The fact that each participant was scanned twice offered a unique opportunity to test the reliability of study effects across two independent scanning sessions. A conjunction analysis for the four main contrasts reported in this paper was conducted. As the TFCE algorithm does not currently perform conjunction analysis,...
PMC10295813
Data and code availability
The full study protocol was preregistered with the Open Science Framework (
PMC10295813
Results
PMC10295813