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18th July 2023
A comprehensive Heart Cell Atlas developed using single cell and spatial genomics combined with computational techniques has uncovered cellular niches in eight regions of the human heart.
In a study published in Nature, researchers produced the most detailed and comprehensive human Heart Cell Atlas to date, including the specialised tissue of the cardiac conduction system, which has not previously been understood at such a detailed level in humans.
The study forms part of the international Human Cell Atlas (HCA) initiative, which is mapping every cell type in the human body, to transform understanding of health and disease. The assembly of an atlas for the heart cells and function is considered particularly essential given how cardiovascular diseases are the leading cause of global deaths and rising.
As part of the study, the researchers developed a new computational tool that is able to predict drug targets and side effects. This Drug2cell tool is able to leverage single-cell profiles and the 19 million drug-target interactions in the European Bioinformatics Institute’s ChEMBL database, which brings together chemical, bioactivity and genomic data to aid the translation of genomic information into effective new drugs.
Using Drug2cell, researchers identified that pacemaker cells serve as a target for specific medicines such as GLP1 drugs for diabetes and weight loss. While a recognised side effect from these drugs is an increased heart rate, exactly how this occurred was unclear. The Drug2cell tool suggested that GLP1 drugs had a direct action on pacemaker cells and this was confirmed using an experimental stem cell model.
Dr Michela Noseda, senior lecturer in cardiac molecular pathology at the National Heart and Lung Institute at Imperial College London, and a lead author on the current study, said: ‘We often don’t fully know what impact a new treatment will have on the heart and its electrical impulses – this can mean a drug is withdrawn or fails to make it to the market. Our team developed the Drug2cell platform to improve how we evaluate new treatments and how they can affect our hearts, and potentially other tissues too. This could provide us with an invaluable tool to identify new drugs which target specific cells, as well as help to predict any potential side effects early on in drug development‘.
A distinctive repertoire of ion channels, G-protein-coupled receptors, regulatory networks and implicated FOXP2 in the pacemaker phenotype was also observed in the study. The researchers identified how the sinoatrial node is compartmentalised, with a core of pacemaker cells, fibroblasts and glial cells supporting glutamatergic signalling.
Taken together, the study offers greater clarity to cardiac electro-anatomy and immunology. Moreover, the researchers believe their suite of computational approaches can be applied to other tissues and organs. The work in the current study, builds on earlier work in 2020, also published in Nature, in which the research team were able to characterise six anatomical adult heart regions using state-of-the-art analyses of large-scale single-cell and single-nucleus transcriptomes.
The HCA programme, founded in 2016, was designed to create comprehensive reference maps of all human cells as a basis for both understanding human health and diagnosing, monitoring, and treating disease. Since 2016, the HCA has grown to include more than 2,900 HCA members, from over 1,500 institutes and 94 countries around the world. It brings together an international community of biologists, clinicians, technologists, physicists, computational scientists, software engineers and mathematicians.
Using cutting-edge single-cell and spatial genomics combined with computational techniques, HCA researchers are able to determine which of the 20,000 genes in an individual cell are switched on, creating a unique ‘ID card‘ for each cell type. This allows the scientists to discover new cell types and functions.
With additional rapidly-evolving spatial analysis methods, HCA scientists map these individual cells into precise locations in organs and tissues, and understand the cell functions and relationships with their neighbours.
17th July 2023
JNJ-77242113 is the first oral peptide able to directly bind to the interleukin-23 (IL-23) receptor, and appears to be effective in moderate to severe plaque psoriasis.
Data presented at the recent 25th World Congress of Dermatology (WCD) from the FRONTIER 1 trial, suggests that JNJ-77242113 (also known as JNJ-2113), a first-in-class oral IL-23 inhibitor, provides a significant improvement in Psoriasis Area Severity Index (PASI) scores compared to a placebo in a dose ranging study.
In the phase 2, randomised, placebo-controlled trial presented at WCD, patients with moderate-to-severe plaque psoriasis were randomised to six different dosing regimens taken either daily (QD) or twice a day (BID) for a total of 16 weeks: 25 mg QD, 50 mg QD, 100 mg QD, or 25 mg BID, 100 mg BID, 100 mg BID or placebo.
The primary outcome was the proportion of patients achieving a 75% improvement in PASI scores at week 16 – known as PASI75. In addition, the team also considered the proportion achieving a PASI90.
At week 16, only 9.3% of participants achieved a PASI75. In contrast, a PASI75 was achieved by 37.2% of those given 25 mg and 58.1% of those with a 50 mg daily dose. The highest proportion of patients achieving a PASI75 was for the 100 mg twice daily dose (78.6%).
In addition, while only 2.3% of placebo patients achieved a PASI90, this occurred with 25.6% of those given the 25 mg dose and 59.5% of those given 100 mg twice daily.
For both PASI75 and PASI90 all comparisons with placebo were statistically significant (nominal p < 0.02).
The proportions of patients with adverse events were similar for the different doses of JNJ-77242113 and the placebo group, mainly Covid-19 and nasopharyngitis with no dose-dependent trends.
A recent study found that use of biologics in women with psoriasis who are either pregnant or planning to conceive is not associated with an increased risk of miscarriage, abortion or congenital malformations.
14th July 2023
A personalised oral cancer survival calculator has been found to provide a more accurate prediction of survival or mortality through the incorporation of a patient’s comorbidities.
The recently developed Oral Cancer Survival Calculator uses a novel modelling approach that gives an equal weight to the risk of death from other causes and gives a better estimate of survival in those with oral cancers.
Once an individual receives a cancer diagnosis, this becomes a focus as their main threat to survival. But the researchers sought to determine how the competing risk of death from noncancer causes affects the risk of death from oral cancer. In other words, do patients with oral cancer have a higher risk of death from other causes?
Oral cancer was selected to develop the model as the cancer develops over time and co-morbidities are an important feature.
In the study, published in the journal JAMA Otolaryngology Head and Neck Surgery, the researchers set out to design a statistical framework and accompanying Oral Cancer Survival Calculator, which factored in other pre-existing conditions, to provide personalised estimates of the probability of a patient surviving or dying from cancer or other causes.
The team used cancer data from the SEER database, SEER-Medicare linked files for co-morbidity information and the National Health Interview Survey (NHIS) – the principal source of information on the health of the civilian population in the US.
Using this data, researchers developed statistical methods to calculate natural life expectancy in the absence of the cancer, cancer-specific survival and other-cause survival. This was then applied to the oral cancer data. The main outcome of interest were the probabilities of surviving or dying from oral cancer or from other causes, and life expectancy in the absence of the cancer.
A total of 22,392 patients with oral squamous cell carcinoma (60.5% male) and 402,626 NHIS interviewees were included. The calculator was specifically designed for use in patients aged between 20 and 86 years, with newly diagnosed oral cancer.
Using the model, researchers estimated that conditional on having survived to age 50 year, a female and male patient diagnosed with stage III cancer would have a 60% and 44% chance, respectively, of being alive at age 70 years, in the absence of their cancer.
For comparative purposes, in the general US population, the corresponding estimates are 86% and 79%, respectively – an absolute difference of 26 and 35 percentage points, respectively.
Taken together, the overall findings suggested that patients with oral cancer have a greater risk of dying of other causes than a matched US population due to the number and type of comorbidities. In fact, even after adjustment for co-morbidities, their likelihood of dying of other causes increases as their cancer stage increases.
The researchers also highlighted that survival estimates that exclude the effects of coexisting conditions can lead to under- or overestimates of survival.
This new tool provides personalised data for discussions between clinicians and patients about the place of cancer treatment in the patient’s life as a whole. It will be broadly applicable for developing future prognostic models of cancer and noncancer aspects of a person’s health in other cancers, they concluded.
13th July 2023
Paroxetine is effective for erythematous rosacea which failed to respond to at least three months of tetracycline treatment or intense pulsed light therapy, according to a recent, randomised placebo-controlled trial.
In a previous study, the antidepressant paroxetine was shown to be effective for menopausal hot flushes, which suggests an anti-inflammatory effect. Here, Chinese researchers sought to evaluate the efficacy and safety of a 12-week course of paroxetine for moderate-to-severe erythema of rosacea.
The primary outcome was the proportion of participants achieving a Clinical Erythema Assessment (CEA) success, which was defined a CEA score of 0 (clear) or 1 (almost clear) or at least a two-grade or higher improvement in CEA score from baseline after 12-weeks treatment.
As rosacea is associated with facial flushing, the team also used a Flushing Assessment Tool, with success defined by a >2-point improvement and a self-reported overall flushing score (ranging from 0-10).
Additional secondary outcomes examined the level of skin burning, depression and the impact on a rosacea quality of life tool.
A total of 97 patients were enrolled and 49 were randomised to paroxetine 25 mg and the remainder to a placebo for a total of 12 weeks.
After 12 weeks, significantly more patients given paroxetine achieved the primary outcome (42.9% vs 20.8%, p = 0.02). In addition, there was a significant improvement in flushing success (p = 0.04) and an improvement in overall flushing score (2.49 vs 1.68, p = 0.047).
Participants taking paroxetine also reported significant reductions in skin burning (p = 0.003) and depression (p = 0.041) compared to placebo. However, there was no significant differences in the extent to which telangiectasia improved between the two groups (p = 0.93) or in rosacea Quality of Life scores (p = 0.83).
During the study, treatment-emergent adverse events (TEAEs) occurred in 24.1% of paroxetine patients and in 11.1% of the placebo group. For the paroxetine group, these TEAEs included dizziness (10.3%), lethargy (10.3%), nausea (8.6%), dyspepsia (6.9%) and muscle tremors (5.2%).
Following cessation of therapy, the team also considered disease recurrence. Although only 21 patients were included in the 12-week follow-up, among the 16 who participated in a face-to-face interview, five (31.2%) reported disease recurrence and two relapsed to their initial CEA severity.
The researchers concluded that paroxetine is an effective and well-tolerated alternative treatment for moderate-to-severe erythema of rosacea.
Fish oil supplements have been shown to offer cardiovascular benefits in randomised trials, but is this advantage undermined by an increased risk of atrial fibrillation, and do these risks also apply to eating oily fish? Clinical writer Rod Tucker finds out more.
A study by Danish scientists in 1976 first observed how Greenland Eskimos had a fatty acid plasma profile that showed a higher content of long-chain polyunsaturated fatty acids – known as omega-3 fatty acids – derived from marine mammals.
Two key fatty acids perceived as physiologically more important were docosahexaenoic acid (DHA) and eicosapentaenoic acid (EPA). The researchers speculated that a higher intake of marine oils may account for the lower incidence of cardiovascular mortality seen among the indigenous people compared to the wider Danish population.
Other observational data also implies that eating fish may confer a cardiovascular benefit. For instance, one study from 1985 collected dietary information on fish intake from over 800 men without coronary heart disease and followed these individuals for the next 20 years. The results were surprising and impressive: coronary heart disease mortality was more than 50% lower among those who consumed at least 30 g of fish per day.
Later, in 1989, a randomised dietary trial of fatty fish intake in men who had sustained a myocardial infarction confirmed the benefits of eating fish. Among participants advised to eat fatty fish, there was a 29% reduction in two year all-cause mortality compared with those not advised to eat it. Moreover, eating fish also appeared to have a primary preventative role.
In 1995, a study of patients experiencing an out of hospital cardiac arrest observed that eating the equivalent of one fatty fish meal per week halved the risk of a primary cardiac arrest. As oily fish such as salmon and herring contains large amounts of both EPA and DHA, it was assumed that these oils were responsible for the cardioprotective effect.
In fact, a 2021 meta-analysis of 38 randomised controlled trials with nearly 150,000 participants confirmed that there were important cardiovascular benefits in patients receiving EPA and DHA.
Despite these positive findings, concerns began to emerge over the risks associated with fish oil supplementation, particularly when used at higher doses.
In a pairwise and network meta-analysis of the cardiovascular benefits of different doses of fish oil supplement intake, researchers concluded that higher doses appeared to significantly increase the risks of both bleeding events and atrial fibrillation (AF) compared to lower doses.
Subsequent analyses appeared to re-affirm these findings. For instance, a 2021 meta-analysis found a 37% higher risk of incident AF compared with placebo in randomised trials of fish oil supplementation in patients with cardiovascular disease.
Another study that specifically focused on the risk of AF from fish oil supplements found an elevated risk in trials testing a dose greater than 1 g per day. This heightened risk was more recently seen in an analysis of data from the UK Biobank, although the risk was only significant among those with pre-existing cardiovascular disease.
The association between fish oil intake and AF is far from clear, with some data suggesting it might be oil specific. For example, one case-cohort study with over 4,000 incident cases of AF found that those with the highest intake of EPA had a 45% lower risk of AF compared to those with the lowest intake. In contrast, while there was no overall association for DHA, there was a 30% lower risk of incident AF for the highest versus the lowest intake, but only for women.
Other studies reveal no effect of either type of fish oil on incident AF, whereas another analysis suggested that incident AF was significantly lower with higher levels of DHA but not for either EPA or a combination of the two oils.
While there is a lack of clarity with the available data, to date, only one randomised trial has directly examined whether fish oil supplements actually reduces the incidence of AF. The trial included patients scheduled for cardiac surgery and provided perioperative fish oil and the primary outcome was set as postoperative AF. The results showed that perioperative fish oil supplements did not significantly reduce postoperative AF compared to placebo, but also that there was no increased risk of arrhythmia.
Questions remain whether the same lack of clarity exists for the link between eating fish and AF. In a prospective analysis published in June 2023, researchers assessed whether dietary marine omega-3 fatty acid intake was associated with the risk of AF. Data were obtained from 301,294 individuals who had a median intake of omega-3 fatty acids of 219 mg per day.
In fully adjusted regression models, there was a nonlinear and inverse relationship with incident AF. There was an initial 11% lower risk when eating 750 mg per day, which then plateaued. In other words, there was no additional benefit from consuming more fish and, as the authors stated, ‘consumption of marine omega-3 fatty acids was not associated with a higher incidence of AF’.
There was also no detectable difference in risk between EPA and DHA. Even when looking at the highest quintile of fatty acid intake (708-6,085 mg per day), there was still a 7% reduced risk of incident AF (hazard ratio, HR = 0.93, 95% CI 0.88 – 0.98).
While these findings were encouraging, insofar as they suggest that eating fish does not pose a higher risk of AF, a limitation of the study was that it was undertaken in predominately white, older males which means that the findings might not be generalisable. However, earlier work in both genders does appear to support the notion that fish intake does not increase the risk of AF.
There’s good news, too, for vegetarians. Evidence suggests that intake of the plant-based omega-3 fatty acid alpha-linolenic acid (ALA) found, for example, in flaxseed and rapeseed oils, is associated with cardiovascular benefits. In fact, the Cardiovascular Health Study, which was a community-based longitudinal cohort of adults aged 65 or older, found no association of plasma phospholipid or dietary ALA and incident AF.
An important caveat attached to the findings from randomised trials of fish oil supplements is that in many cases AF was not always considered as a pre-specified outcome and, in some cases, not reported in exploratory analysis. Consequently, the trials were not powered for detecting AF as an endpoint so that the findings should be interpreted with caution.
On the basis of the available evidence, it seems plausible that consumption of fish oil supplements, especially at higher doses, could increase the risk of AF. Clinicians and patients therefore need to weigh up the observed cardiovascular benefits against the potential risk of not only AF but increased bleeding.
Given the strength of the observational evidence, it is reasonable to suggest that eating fish, or increasing intake of ALA for those who do not eat fish, offers cardiovascular health benefits that are not eroded by an associated greater risk of atrial fibrillation.
12th July 2023
A high pre-treatment tumour monocyte content prior to immunochemotherapy in patients with inoperable oesophageal cancer is associated with greater overall survival, according to a recent study.
Writing in the journal Cell Cancer, the researchers from the Ludwig Institute for Cancer Research, University of Oxford together with international colleagues, sought to identify which patients would best respond to an initial four week course of immune checkpoint inhibitors followed by immunochemotherapy.
As part of their analysis, the researchers performed comprehensive biomarker profiling, as well as multi-timepoint transcriptomic profiling during the initial four-week course of an immune checkpoint inhibitor. The analysis revealed how a novel T-cell inflammation signature, which they termed INCITE, was up-regulated and correlated with immune checkpoint inhibitor induced tumour shrinkage.
As such, they determined that a high tumour monocyte content in patients receiving immunochemotherapy, was predictive of greater overall survival.
The study included 35 patients with inoperable oesophageal cancer. While four weeks of immune checkpoint inhibitors was enough to induce tumour shrinkage in some patients, overall, around 40% achieved a clinical benefit (defined as 12 months of progression-free survival) from the immunochemotherapy. The factors responsible for this clinical benefit were unclear.
Using deconvolution of pre-treatment gastroesophageal cancer transcriptomes, it was found that a cluster of patients had tumours with a high monocyte content pre-treatment. In Cox regression on the pre-treatment level of each cell type, the tumour monocyte count was significantly associated with improved overall survival (hazard ratio, HR = 0.38, 95% CI 0.22 – 0.67, p = 0.0008).
When researchers stratified the monocyte content as either high or low, the median overall survival was 24.3 months for the high content group but only 8.6 months for the lower level group.
Commenting on these findings, the researchers suggested that the pre-treatment tumour monocyte count could be a particularly useful marker of durable benefit for those prescribed immunochemotherapy.
The researchers observed a drop in monocyte levels following immune checkpoint inhibitor therapy, yet patients still went on to achieve a clinical benefit.
They therefore suggested that the use of immune checkpoint inhibitors may drive differentiation of intra-tumoural monocytes into pro-inflammatory myeloid effectors and that this was a more likely explanation for the improved outcomes.
A minimally invasive procedure to detect epileptic foci in the brain without the need for surgery has been successfully conducted on a child in the Middle East for the first time.
The cutting-edge stereoelectroencephalography (SEEG) technique was performed on an 11-year-old boy with drug-resistant – or intractable – epilepsy, with the aim of locating the epileptic foci in the brain to pave the way for their future removal.
Carried out at the Neuroscience Centre of King Faisal Specialist Hospital and Research Centre (KFSH&RC), the procedure involves creating 2mm holes in the skull to implant electrical monitoring electrodes directly into the brain.
These electrodes enable precise measurement and mapping of electrical activity, allowing specialists to closely monitor the patient and identify the specific regions where epileptic seizures originate.
They can then determine the specific tissues that need to be removed to eliminate epilepsy seizures effectively, ultimately improving the patients’ quality of life and that of their families.
Discussing the technique, Dr Ibrahim Althubaiti, a consultant at the Epilepsy Integrated Practice Unit at KFSH&RC, highlighted the challenges faced when locating epileptic foci in children as opposed to adults. ‘Unlike adults who can easily comply with instructions, children’s movement can be challenging to control for prolonged periods, posing a greater difficulty in executing the medical procedure,’ he said. ‘Nevertheless, the successful implementation of the SEEG technique allowed us to overcome this challenge.’
In practice, SEEG takes 45 minutes to complete, requires no more than 48 hours of recovery and is 60% less expensive when compared to previously used procedures.
Prior to its introduction, procedures took nine hours to complete and involved the temporary removal of a substantial part of the skull to access the brain for the electrode placement. This carried the risks of pain, infections and potential complications from anaesthesia, as well as considerable recovery time.
11th July 2023
A faecal microbiota transplantation (FMT) from healthy donors prior to PD-1 inhibitor immunotherapy could represent a novel and effective approach to the management of advanced melanoma, according to a recent phase 1 trial.
Published in the journal Nature Medicine, researchers combined healthy donor FMT with the PD-1 inhibitors nivolumab or pembrolizumab in previously untreated patients with advanced melanoma.
The primary outcome of interest was safety, with key secondary endpoints of the objective response rate, changes in gut microbiome composition and systemic immune and metabolomics analyses.
A total of 20 patients with a confirmed diagnosis of unresectable or metastatic cutaneous melanoma and with no previous anti-PD-1 treatment were enrolled.
Patients received a single FMT via capsules containing 80-100 mg of faeces from heavily screened healthy donors. Individuals were required to consume 36-40 capsules under supervision followed by a 30-minute period of observation.
When considering the primary outcome, eight patients experienced grade 1-2 FMT-related toxicities, mainly diarrhoea, flatulence and abdominal discomfort. However, there were no grade 3 or higher adverse events before receipt of the first dose of anti-PD-1 therapy.
The objective response rate was 65% and this included four patients who experienced a complete response. Microbiome profiling revealed that all of the patients engrafted strains from their respective donors although the acquired similarity between microbiomes increased over time in responders.
The researchers concluded that FMT from healthy donors represents a safe new therapeutic tool that has clinical potential and should be explored further in randomised trials.
Additional approaches are needed in advanced melanoma given that five-year survival even with combination immunotherapy is just over 50%. Previous work suggested that FMT and anti-PD-1 therapy, could overcome resistance to anti-PD-1.
7th July 2023
The presence of persistent CD19 CAR T-cells may account for why some children with relapsed-refractory acute lymphoblastic leukaemia have longer remission, according to a study by a team of UK researchers.
The use of CAR T-cells that target CD19 is an effective therapy for relapsed and refractory acute lymphoblastic leukaemia in children. In fact, the therapy is now a widely used therapeutic approach for relapsed or refractory lymphomas.
The effectiveness of CAR T-cell therapy requires that these modified T-cells persist within the body. But what enables these cells to persist was the question posed in a recent study published in the journal Nature Medicine.
The team of UK researchers systematically analysed CD19 CAR T-cells of 10 children who had either relapsed or refractory acute lymphoblastic leukaemia (B-ALL) enrolled in the CARPALL study. The researchers then studied molecular features and clonal dynamics of CAR T-cells in this CARPALL study up to five years after infusion.
The team studied 15 consecutive patients with high-risk or relapsed CD19 positive B-ALL treated with CAR T-cell therapy using cells isolated from cryopreserved samples of blood or bone marrow. From this cohort, 13 had achieved complete remission and six had subsequently relapsed. However, the remaining seven achieved long-lived remissions maintained by detectable CAR T-cells and concomitant B cell aplasia.
The researchers analysed a total of 264,827 single cells, approximately 50,000 of which were CAR T-cells. The long-lived CAR T-cells developed a CD4/CD8 double-negative phenotype with an exhausted-like memory state and distinct transcriptional signature. In addition, the signature was dominant among circulating CAR T-cells in those children with a long-lived treatment response.
Investigating further, the researchers found that this same signature was present across T-cell subsets and clonotypes, indicating that persisting CAR T-cells converge transcriptionally. They also found the signature in two adult patients who had previously been given a different CD19 CAR T-cell product for chronic lymphocytic leukaemia and had a decade-long remission.
An important finding from the study was how this persistent transcriptional signature was reproducible across thousands of cells in every patient with long-lived CAR T-cells and durable anti-B-ALL responses. These findings suggest that this persistence signature might be specific to long-lived CAR T-cells and raises the possibility of a universal transcriptional signature indicative of clinically effective, persistent CD19 CAR T-cells.
Topical ruxolitinib has been approved by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of non-segmental vitiligo in adults and adolescents with facial involvement.
The MHRA has granted marketing authorisation for ruxolitinib cream 15mg/g (brand name Opzelura) for treating patients from 12 years of age who have non-segmental vitiligo affecting facial areas.
The approval was based on the findings from two identical phase 3 trials TRuE-V1 and TRuE-V2, which evaluated the efficacy and safety of ruxolitinib cream compared to placebo in over 600 patients with non-segmental vitiligo.
Manufactured by Incyte, Opzelura is now the first and only approved treatment in the UK to offer eligible patients with non-segmental vitiligo support for repigmentation. The MHRA decision follows the European Commission approval in April 2023 and FDA approval in July 2022.
Dr Viktoria Eleftheriadou, consultant dermatologist and lead for vitiligo clinic and research, Walsall Healthcare NHS Trust and The Royal Wolverhampton NHS Trust, said: ‘The MHRA approval is welcome news for dermatologists and people with vitiligo seeking treatment who until now have had limited options. The data supporting this approval demonstrate the potential for ruxolitinib cream to make a difference in the lives of people living with this condition.’
Founder and chief executive officer of the charity Vitiligo Support UK Emma Rush added: ‘While more and more people are proud of their vitiligo, there are still so many people who don’t feel comfortable in their skin. This new treatment option provides a choice for those who wish to treat their condition.’
Voicing the manufacturer’s delight at the approval, Peter Williams, general manager at Incyte UK and Ireland, said: ‘We are now working in partnership with the NHS to ensure that eligible patients seeking to treat their vitiligo are able to access this innovative medicine.’
The combined results of the two randomised, double-blind, vehicle-controlled trials were published as a single paper in the New England Journal of Medicine in October 2022. Both included patients aged 12 years or older who had non-segmental vitiligo with depigmentation covering 10% or less of total body-surface area.
Patients were randomly assigned in a 2:1 ratio to apply 1.5% ruxolitinib cream or vehicle control twice daily for 24 weeks to all vitiligo areas on the face and body, after which all patients could apply 1.5% ruxolitinib cream through to week 52.
The primary end point was an improvement of at least 75% from baseline in the facial Vitiligo Area Scoring Index (F-VASI75), which ranges from 0 to 3, with higher scores reflecting a greater area of facial depigmentation.
A combined total of 674 patients, 330 in TRuE-V1 and 344 in TRuE-V2, were randomised to ruxolitinib. After 24 weeks of therapy, a F-VASI75 response occurred in 29.8% of those taking ruxolitinib in TRuE-V1 compared to 7.4% of those assigned the vehicle control (p < 0.001). Similarly, in TRuE-V2, 0.9% of those receiving ruxolitinib cream achieved a F-VASI75 response (p < 0.001) compared to 7.4% in the vehicle group.
Around one in 100 people in the UK develop vitiligo, with eight in 10 suffering from the non-segmental vitiligo, where both sides of the body are affected by symmetrical white patches. The treatment is approved for twice-daily topical use to the depigmented skin areas up to a maximum of 10% body surface area. Satisfactory re-pigmentation may require treatment with ruxolitinib cream for more than 24 weeks.
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