Systemic Therapies for Moderate-to-Severe Atopic Dermatitis in Children and Adolescents: A Systematic Review (2025)

Type of publication:

Systematic Review

Author(s):

Hamza Osman, Sahar Khalil; *Mohamed Ahmed, Malaz Awad; Idrees, Hoda; Mohammad Ali, Aziza Mohammad Hassan; Ahmed Taha, Aisha Hassan; Musa Shaikhelsafi, Fatima Hassan; Mirghani Hamour, Afrah Mohamed.

Citation:

Cureus. 17(10):e94907, 2025 Oct.

Abstract:

Moderate-to-severe atopic dermatitis (AD) in children and adolescents imposes a significant burden, often requiring systemic therapy. With the recent development of targeted biologics and Janus kinase (JAK) inhibitors, the treatment landscape has evolved rapidly. This systematic review aims to critically evaluate the efficacy and safety of these advanced systemic therapies in the pediatric population. A systematic search of PubMed/MEDLINE, Scopus, Web of Science, and ClinicalTrials.gov was conducted for randomized controlled trials (RCTs) published from 2020 onwards, yielding 250 records. Thirteen studies involving over 3,500 pediatric patients were included. Studies evaluating biologics or JAK inhibitors in children and adolescents (0-18 years) with moderate-to-severe AD were included. The Cochrane Risk of Bias 2 (ROB 2) tool was used for quality assessment. A narrative synthesis was performed due to clinical heterogeneity. Dupilumab and tralokinumab (biologics) demonstrated significant efficacy, with Eczema Area and Severity Index 75 (EASI-75) response rates of 43.3% at week 16 and sustained improvements in disease severity (SCORAD, IGA) and pruritus. The JAK inhibitors, abrocitinib and upadacitinib, showed rapid and high-magnitude efficacy, with EASI-75 and Validated Investigator Global Assessment (vIGA-AD) response rates frequently exceeding 70-90% by weeks 12-16 and providing rapid itch relief. Dupilumab's safety profile was favorable, with mostly mild-to-moderate adverse events (e.g., conjunctivitis). JAK inhibitors were associated with acne, nausea (abrocitinib), and herpes infections, necessitating routine monitoring. The overall risk of bias was low across most studies. Advanced systemic therapies are highly effective for moderate-to-severe pediatric AD. Biologics offer a well-established safety profile, while JAK inhibitors provide superior and faster efficacy, particularly for itch, but require careful safety monitoring. Treatment choice should be individualized based on disease severity, preference, and risk profile.

DOI: 10.7759/cureus.94907

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Atezolizumab-Induced Thyroiditis and Immune Checkpoint Inhibitor (ICI)-Type 1 Diabetes Mellitus: Diagnostic and Therapeutic Challenges in ICI-Associated Endocrinopathies (2025)

Type of publication:

Journal article

Author(s):

*Al-Rubaye, Rafal; *Nadeem, Sabha; Soliman, Ahmed Abdulkader Zaki Ali.

Citation:

Cureus. 17(10):e94593, 2025 Oct.

Abstract:

The rapid evolution and increased application of immune checkpoint inhibitors (ICIs) in the oncology setting have introduced novel diagnostic and therapeutic challenges, particularly in the setting of endocrine immune-related adverse events (irAEs). Thyroid dysfunction is among the most common of these, while ICI-type 1 diabetes mellitus is uncommon and can present abruptly with life-threatening complications. A 76-year-old man with extensive-stage small-cell lung cancer being treated with atezolizumab developed new-onset diabetes, which manifested as diabetic ketoacidosis in connection with immunotherapy-induced thyroiditis. Metabolic instability in this patient was compounded by the inappropriate initiation of thyroid hormone replacement during the thyrotoxic phase of the thyroiditis as a result of misinterpretation of his thyroid function tests (TFTs). The unique clinical course of this patient illustrates the broad and unpredictable spectrum of ICI-related endocrinopathies and highlights the importance of prudent interpretation of TFTs, early recognition of ICI-induced type 1 diabetes mellitus, and close multidisciplinary management of irAEs.

DOI: 10.7759/cureus.94593

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Risk of major adverse cardiovascular events in Crohn's disease patients treated with biologic therapy: A meta-analysis and trial sequential analysis of randomised controlled trials (2025)

Type of publication:

Conference abstract

Author(s):

Bharadwaj H.; Perros I.; Biggs D.; *Butterworth J.; Gohar F.; Sokhal B.S.; Mallen C.

Citation:

United European Gastroenterology Journal. Conference: The 33rd United European Gastroenterology Week, UEGW 2025. Berlin Germany. 13(Supplement_8) (pp 628), 2025. Date of Publication: 01 Oct 2025.

Abstract:

Introduction: Biologic therapies are the preferred treatment for induction and maintenance of remission in Crohn's Disease, although their cardiovascular safety remains uncertain. This systematic review and meta-analysis aimed to estimate the risk of major adverse cerebrovascular and cardiovascular events (MACCEs) in adult CD patients receiving biologic or small molecule therapies in randomised controlled trials (RCTs). Aims & Methods: This study followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses. MEDLINE, EMBASE, and Cochrane were searched to identify RCTs that assessed the risk of MACCEs in CD induction and maintenance trials. Data were pooled and analysed using random effects modelling with 95% confidence intervals (CIs). Result(s): 40 RCTs were included, describing 20 induction and 20 maintenance trials. A total of 17,718 patients were included, with 11,148 (62.9%) receiving biologic agents or small molecules. The risk of MACCEs was lower in induction (OR=0.56, 95%CI: 0.24,1.34, P=0.19) and maintenance trials (OR=0.75, 95%CI: 0.35,1.57, P=0.44) compared to placebo or active comparators. MACCE risk remained unaffected by drug agent, drug class, and trial duration. Overall, there was no difference in MACCE risk based on receipt of biological therapy (OR=0.65, 95%CI: 0.37,1.15, P=0.14). Conclusion(s): Biologic agents and small molecules did not increase MACCE risk in CD induction and maintenance trials. A slight cardioprotective effect was observed. Longer follow-up studies with real-world data are required to confirm these findings outside the RCT setting.

DOI: 10.1002/ueg2.70032

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Impact of biological therapy on the risk of major adverse cerebrovascular and cardiovascular events in patients with ulcerative colitis: A systematic review, meta-analysis and trial sequential analysis of level 1 evidence (2025)

Type of publication:

Systematic Review

Author(s):

Bharadwaj H.; Perros I.; Biggs D.; *Butterworth J.; Gohar F.; Mallen C.; Sokhal B.S.;

Citation:

United European Gastroenterology Journal. Conference: The 33rd United European Gastroenterology Week, UEGW 2025. Berlin Germany. 13(Supplement_8) (pp 466), 2025. Date of Publication: 01 Oct 2025.

Abstract:

Introduction: Biological therapies have improved remission rates in Ulcerative colitis (UC) and are superior to standard treatment. This systematic review and meta-analysis of randomised controlled trials (RCTs) aimed to estimate the risk of major adverse cerebrovascular and cardiovascular events (MACCE) in adult UC patients receiving biologics. Aims & Methods: MEDLINE, EMBASE and Cochrane were searched to identify RCTs that investigated the risk of MACCE in UC induction and maintenance trials. Data were pooled and analysed using random effects modelling with 95% confidence intervals (CIs). This study followed the Preferred Reporting items for Systematic Reviews and Meta-Analyses. Result(s): 31 studies were retrieved from inception to November 2024. 54 RCTs were included, describing 29 induction and 25 maintenance phases. A total of 26,114 patients were included, with 17,271 (66.1%) receiving biologic agents or small molecules. The risk of MACCEs was not higher in induction (OR=0.62, 95%CI:0.32,1.18, P=0.14) or maintenance trials (OR=0.57, 95%CI:0.28,1.18, P=0.13) compared to placebo or active comparators. No drug agent, drug class or trial duration incurred a higher risk of MACCEs. Overall, those treated with biologic agents and small molecules had a lower MACCE risk (OR=0.60, 95%CI:0.37,0.97, P<0.05). Heterogeneity for all outcomes and subgroups was low (I2=0.00%, P=1.00). Conclusion(s): Biologics were not associated with risk of MACCE. Longer follow- up studies with real-world data are required to confirm these findings outside the RCT setting.

DOI: 10.1002/ueg2.70032

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Updates on trichoscopy in diagnosing scalp disorders: A systematic review of diagnostic accuracy, sensitivity and specificity (2025)

Type of publication:

Conference abstract

Author(s):

*Mehra S.

Citation:

British Journal of Dermatology. Conference: 105th Annual Meeting of the British Association of Dermatologists, BAD 2025. Glasgow United Kingdom. 193(Supplement 1) (pp i54), 2025. Date of Publication: 01 Jul 2025.

Abstract:

Trichoscopy has emerged as a pivotal diagnostic tool for evaluating scalp disorders. This systematic review assesses its diagnostic sensitivity, specificity and accuracy for various scalp conditions, proposing refinements to clinical protocols based on National Institute for Health and Care Excellence (NICE) and British Association of Dermatologists (BAD) guidelines. The aim is to provide recommendations for integrating trichoscopy into routine dermatology practice to optimize patient outcomes and diagnostic pathways. A systematic review was conducted following the PRISMA guidelines. The PubMed, MEDLINE and Embase databases were searched for studies published between 2010 and 2023. Keywords included 'trichoscopy', 'scalp disorders', 'diagnostic accuracy', 'sensitivity' and 'specificity'. Inclusion criteria encompassed studies evaluating the diagnostic performance of trichoscopy for common scalp conditions such as alopecia areata, androgenetic alopecia, telogen effluvium, scalp psoriasis, seborrhoeic dermatitis and tinea capitis. Only multicentre or single-centre studies with quantitative data were included. Exclusions included case reports and studies lacking statistical metrics. Data were analysed within the framework of the BAD and NICE recommendations to assess real-world applicability. Thirty-six studies involving 11 250 patients were included. Trichoscopy consistently demonstrated high diagnostic accuracy, surpassing traditional methods. (i) In alopecia areata, exclamation-mark hairs and black dots yielded 94% sensitivity and 92% specificity, aligning with BAD recommendations. (ii) In androgenetic alopecia, hair shaft diameter variability and perifollicular pigmentation showed 91% sensitivity and 89% specificity, supporting diagnostic integration. (iii) In telogen effluvium, empty follicles and short regrowth hairs demonstrated 86% sensitivity and 84% specificity, enabling earlier interventions. (iv) In tinea capitis, comma hairs and corkscrew hairs achieved 95% diagnostic accuracy, emphasizing the superiority of trichoscopy. (v) In scalp psoriasis and seborrhoeic dermatitis, differentiation was achieved using red dots, diffuse white scales and arborizing vessels, with 92% sensitivity. Reproducibility across centres was evident. Artificial intelligence (AI)-based algorithms were highlighted for enhancing diagnostic standardization, clinician training and accessibility. A national registry of trichoscopic images is proposed to improve data sharing and compliance with guidelines. In conclusion, trichoscopy is indispensable for diagnosing scalp disorders, offering a noninvasive, accurate alternative to biopsies. It enables earlier diagnosis, precise differentiation and improved outcomes, aligning with NICE and BAD guidelines. The results support the following recommendations. (i) Develop standardized trichoscopic criteria for scalp disorders. (ii) Include trichoscopic education in dermatology training. (iii) Invest in AI-based tools for image standardization. (iv) Establish a national trichoscopy registry for collaborative research. For application to clinical practice, this review provides actionable insights for enhancing diagnostic pathways and advancing the role in dermatology in trichoscopy, offering a foundation for improved patient care.

DOI: 10.1093/bjd/ljaf085.105

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Efficacy and safety of injectable bio-revitalizers and rejuvenate therapies, including platelet-rich plasma and exosome-based treatments: A systematic review of licensed products in the UK (2025)

Type of publication:

Conference abstract

Author(s):

*Mehra S.

Citation:

British Journal of Dermatology. Conference: 105th Annual Meeting of the British Association of Dermatologists, BAD 2025. Glasgow United Kingdom. 193(Supplement 1) (pp i34), 2025. Date of Publication: 01 Jul 2025.

Abstract:

The aim of this study was to evaluate the efficacy and safety of injectable bio-revitalizers and rejuvenative therapies licensed under Medicines and Healthcare products Regulatory Agency (MHRA) and National Institute for Health and Care Excellence (NICE) guidelines. These included platelet-rich plasma (PRP) and exosome-based treatments. This review synthesizes clinical outcomes, safety profiles and patient-reported satisfaction to provide evidence-based recommendations for advancing dermatological practice in the UK. A systematic review was conducted following the PRISMA guidelines. Databases including PubMed, MEDLINE, Embase and Cochrane Library were searched for studies published between 2010 and 2023. Keywords included 'injectable bio-revitalisers', 'hyaluronic acid', 'polynucleotides', 'platelet-rich plasma', 'exosome therapy', 'efficacy' and 'safety'. Inclusion criteria focused on licensed products approved in the UK under MHRA and NICE regulations, such as Profhilo, Restylane Skinboosters, Sunekos, Nucleofill, Juvelook, PRP and exosomebased therapies. Data extraction covered clinical efficacy (e.g. hydration, elasticity, wrinkle reduction), safety (e.g. adverse events, tolerability) and patient-reported outcomes. Twenty-six studies involving 2450 patients were included. Key findings highlighted consistent efficacy and safety across therapies. Hyaluronic acid-based bio-revitalizers (e.g. Profhilo, Restylane Skinboosters) improved hydration (20-25%), elasticity (15-18%) and wrinkle reduction, with patient satisfaction rates of 88-90%. Adverse events were limited to transient erythema and swelling. Polynucleotidebased therapies (e.g. Nucleofill, Juvelook) enhanced skin regeneration, provided antioxidant benefits and showed sustained results up to 6 months, with minimal adverse effects and improved firmness (18-22%). PRP showed moderate-to-significant improvements in skin texture and fine lines, with 76% of patients reporting enhanced skin quality. Adverse events were mild. Exosome-based therapies had emerging evidence indicating improvements in tone, texture and collagen stimulation, with high satisfaction rates (89%) and minimal adverse events. In conclusion, injectable bio-revitalizers, including PRP and exosome-based therapies, demonstrate high efficacy and safety profiles when adhering to MHRA and NICE guidelines. These therapies provide innovative, minimally invasive options for skin rejuvenation, with consistent patient satisfaction. Standardized treatment protocols, tailored patient selection criteria, and long-term studies are needed to optimize outcomes. PRP and exosome-based therapies expand regenerative dermatology and enhance patient care. Recommendations for practice are as follows: (i) incorporate PRP and exosome therapies into bio-revitalization strategies, emphasizing regenerative potential; (ii) develop national guidelines for standardized protocols; (iii) establish multicentre registries for tracking long-term outcomes and (iv) implement training programmes on appropriate use and safety considerations. This review offers a comprehensive evaluation of licensed bio-revitalizers and rejuvenate therapies, providing colleagues with evidence-based insights for improving patient care and advancing practice in aesthetic and therapeutic dermatology. The findings serve as a valuable teaching tool, fostering reflection on current practices and encouraging innovation within the field.

DOI: 10.1093/bjd/ljaf085.065

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Psychological impact of hair loss in women: A qualitative systematic review (2025)

Type of publication:

Conference abstract

Author(s):

*Mehra S.

Citation:

British Journal of Dermatology. Conference: 105th Annual Meeting of the British Association of Dermatologists, BAD 2025. Glasgow United Kingdom. 193(Supplement 1) (pp i85), 2025. Date of Publication: 01 Jul 2025.

Abstract:

Hair loss in women is associated with profound psychological distress, affecting mental health, self-esteem and social functioning. This systematic review consolidates findings from qualitative studies to explore the psychological burden of hair loss and evaluate the efficacy of supportive interventions. The aim is to inform best practices for holistic, patient-centred management in dermatology clinics, aligning with National Institute for Health and Care Excellence guidelines and international protocols. This review adhered to the PRISMA guidelines, with a comprehensive search of the PubMed, MEDLINE, PsycINFO and Embase databases for qualitative studies published between 2010 and 2023. Keywords included 'psychological impact', 'hair loss', 'women', 'qualitative studies' and 'supportive interventions'. Inclusion criteria encompassed studies on the emotional and psychological burden of hair loss in women, with or without supportive interventions. Mixed-methods studies were included if qualitative data could be extracted. Singlecase studies and those without a psychological focus were excluded. Data were thematically synthesized to identify psychological impacts and assess the effectiveness of interventions such as counselling, peer support and cosmetic solutions. Twenty-six studies involving 1450 participants met the inclusion criteria. The key findings are reported here. (i) Emotional distress: hair loss caused significant emotional distress, with 78% of women reporting feelings of shame, anxiety or depression. Younger women and those with more extensive hair loss experienced greater psychological burdens. (ii) Impact on self-image: self-esteem was negatively affected in 85% of participants, with themes of loss of femininity and perceived diminished attractiveness. (iii) Social withdrawal: over 60% of women avoided social interactions due to embarrassment, compounding isolation and low selfworth. (iv) Supportive interventions: psychosocial therapies such as cognitive behavioural therapy (CBT) and peer support groups reduced anxiety and improved coping in 68%, while cosmetic solutions such as wigs, scalp micropigmentation and hairpieces enhanced confidence and social reintegration for 72%. Emerging themes underscored the importance of empathic clinician-patient communication, with patients emphasizing the need for guidance and emotional support alongside clinical care. Hair loss in women exerts a profound psychological impact on mental health, self-esteem and social functioning. Supportive interventions, including CBT, peer support and cosmetic solutions, effectively alleviate distress and enhance quality of life. A multidisciplinary approach integrating psychological support into routine care pathways is essential. Recommendations for practice are (i) to implement psychological assessments to identify at-risk patients; (ii) to develop integrated care models combining dermatological and psychological support; (iii) to create age-specific, culturally sensitive interventions and (iv) to educate patients on supportive therapies, fostering proactive engagement. This review highlights the critical need for dermatologists to address both the physical and psychological dimensions of hair loss, offering practical insights for patient-centred care. By incorporating psychological screening, empathetic communication and evidence-based supportive interventions, dermatology clinics can significantly improve patient outcomes.

DOI: 10.1093/bjd/ljaf085.164

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Nail changes as indicators of systemic disease: A systematic review of correlations and diagnostic outcomes (2025)

Type of publication:

Conference abstract

Author(s):

*Mehra S.

Citation:

British Journal of Dermatology. Conference: 105th Annual Meeting of the British Association of Dermatologists, BAD 2025. Glasgow United Kingdom. 193(Supplement 1) (pp i119), 2025. Date of Publication: 01 Jul 2025.

Abstract:

This systematic review evaluates the correlation between nail changes and systemic diseases, assessing their diagnostic utility, sensitivity and specificity. The aim is to highlight the role of nail examination as a noninvasive diagnostic tool, enhancing dermatology practice and interdisciplinary care in line with National Institute for Health and Care Excellence (NICE) guidelines and clinical protocols. This practice is particularly relevant in resource-limited settings, where access to advanced diagnostics may be restricted. A systematic review was conducted following the PRISMA guidelines. The PubMed, MEDLINE and Embase databases were searched for studies published between 2010 and 2023 using keywords such as 'nail changes', 'systemic disease' and 'diagnostic indicators'. Studies were included that analysed correlations between nail abnormalities (e.g. clubbing, Beau's lines, splinter haemorrhages, leuconychia, onycholysis) and systemic diseases like cardiovascular, pulmonary, autoimmune or metabolic conditions. Case reports and studies without statistical analysis were excluded. Data extraction focused on nail abnormalities, associated systemic diseases, and diagnostic metrics such as sensitivity and specificity. Thirty-two studies involving 7250 patients were included. Key findings demonstrated significant correlations between nail changes and systemic diseases. (i) Clubbing was associated with pulmonary and cardiovascular conditions, such as bronchiectasis and congenital heart defects, with a specificity of 92%. (ii) Beau's lines were linked to systemic stressors like severe infections, chemotherapy and malnutrition, with a sensitivity of 81%. (iii) Splinter haemorrhages were observed in vasculitis, infective endocarditis and connective tissue disorders, with a sensitivity of 78%. (iv) Leukonychia was linked to hypoalbuminaemia and liver cirrhosis, with a specificity of 85%. (v) Onycholysis was indicative of thyroid dysfunction, particularly hyperthyroidism, with a diagnostic accuracy of 80%. In 30% of cases, nail changes preceded other clinical manifestations, offering opportunities for early diagnosis and intervention. While nail findings alone lacked specificity in some cases, combining them with clinical history and investigations enhanced utility. These findings are impactful in resource-limited settings, where noninvasive tools can guide early management without expensive testing. This review underscores the value of nail examination as a noninvasive tool for identifying systemic diseases, often serving as an early indicator of pathology. Nail changes such as clubbing, Beau's lines and leuconychia provide clinically significant insights, particularly when integrated with broader assessments. Increased awareness, standardized documentation and incorporation into practice can improve diagnostic accuracy and patient outcomes, especially in settings with limited access to advanced diagnostics. Nail examination is an underutilized diagnostic tool. It bridges dermatology and systemic medicine, prompting timely referrals and collaborative management. In resource-limited settings, it offers an accessible alternative, reducing reliance on costly tests. Standardized protocols should be integrated into NICE guidelines globally.

DOI: 10.1093/bjd/ljaf085.237

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Efficacy and safety of hair gloss treatments: A systematic review of licensed products in the UK (2025)

Type of publication:

Conference abstract

Author(s):

*Mehra S.

Citation:

British Journal of Dermatology. Conference: 105th Annual Meeting of the British Association of Dermatologists, BAD 2025. Glasgow United Kingdom. 193(Supplement 1) (pp i117-i118), 2025. Date of Publication: 01 Jul 2025.

Abstract:

Hair gloss treatments have gained popularity for enhancing shine, hydration and overall hair health. However, their safety profiles, formulation integrity and clinical efficacy remain under scrutiny within dermatology. This systematic review evaluates the efficacy, safety and formulation of licensed hair gloss products in the UK, examining compliance with Medicines and Healthcare products Regulatory Agency (MHRA) and National Institute for Health and Care Excellence guidelines. The review bridges academic findings with practical application, offering insights for patient care and regulatory considerations. A systematic review was conducted following PRISMA guidelines, covering studies published between 2010 and 2023. Databases searched included PubMed, MEDLINE, Embase and Cochrane Library, focusing on licensed UK products. Keywords included 'hair gloss', 'safety', 'efficacy', 'formulations' and 'adverse reactions'. Products analysed were Olaplex No. 6 Bond Smoother, Redken Shades EQ Gloss, Wella Professionals Shinefinity, and L'Oreal Professional DIA Light Gloss. Data extracted included efficacy metrics (e.g. shine, hydration and smoothness), safety outcomes (e.g. adverse reactions and ingredient profiles) and adherence to regulatory standards. Twenty-two studies and regulatory reports involving 1000 participants and 150 licensed products were analysed. Key findings include the following. (i) Efficacy. Shine and smoothness improved in 85% of users, with 72% noting enhanced hydration and reduced frizz. Benefits lasted 4-6 weeks. Products with panthenol and plant-based proteins showed superior efficacy, particularly for dryness and breakage. (ii) Safety profiles. While 82% of products adhered to MHRA standards, 18% contained harmful ingredients (e.g. formaldehyde derivatives, parabens and sulfates) linked to mild scalp irritation (9%), allergic responses (3%) and transient dryness (2%). Licensed products like Wella Professionals Shinefinity and L'Oreal DIA Light Gloss exhibited excellent safety profiles due to ammonia-free, conditioning-focused formulations. (iii) Formulation integrity. 'Ammonia-free' and plant-derived hydrating products had fewer adverse reactions and higher satisfaction. Non-clinical-grade products lacked standardized labelling and ingredient reporting, challenging safety and consistency. In conclusion, licensed hair gloss treatments offer significant cosmetic benefits with acceptable safety profiles. However, harmful additives in some formulations emphasize the need for stricter regulatory oversight and clinician guidance. Dermatologists play a key role in addressing patient concerns and optimizing outcomes. Recommendations for practice are as follows. (i) Dermatologists must educate patients on choosing licensed products and highlight risks of unregulated formulations. (ii) Stricter labelling standards and transparency are essential. (iii) Hair gloss treatments should be viewed as adjuncts to address hair health concerns. This review highlights the growing relevance of product safety and efficacy in patient consultations. With academic findings and real-world application, practitioners can better navigate the intersection of cosmetic science and clinical dermatology, ensuring that patient outcomes are both aesthetically and medically optimized. The reflections herein encourage dermatologists to critically assess the safety and utility of cosmetic products in practice, fostering an evidence-based approach.

DOI: 10.1093/bjd/ljaf085.233

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Cure vs. toxicity: quantifying preferences for non-surgical management of rectal cancer using a prospective discrete choice experiment study (2025)

Type of publication:

Conference abstract

Author(s):

Webb E.J.; Twiddy M.; Noutch S.; Adapala R.; Bach S.P.; Brown S.; Burnett C.; Burrage A.; Gilbert A.; Hawkins M.; Howard D.; Hudson E.; Jefford M.; Kochhar R.; Saunders M.; Seligmann J.; Smith A.; Teo M.; West N.; Sebag-Montefiore D.; *Gollins S.; Appelt A.L.

Citation:

Radiotherapy and Oncology. Conference: ESTRO 2025. Vienna Austria. 206(Supplement 1) (pp S1253-S1255), 2025. Date of Publication: 01 May 2025.

Abstract:

Purpose/Objective: Dose-escalation may increase the chance of successful non-surgical rectal cancer management, but requires an understanding of acceptable trade-offs between chance of cure and toxicity risks. This study is the first to measure patient preferences for non-surgical management (NOM) of rectal cancer using a discrete choice experiment (DCE). Material/Methods: A prospective, multicentre study conducted in seven UK radiotherapy centres. Patients consented to participation prior to initiation of radiotherapy-based NOM for rectal cancer (any stage), and completed the survey pre-treatment and 6 months post-treatment. The DCE was developed with qualitative patient input and had a Bayesian D-efficient design. Patients made repeated choices between hypothetical NOM treatments, described using six attributes: treatment length; chance of being cancer-free two years post-treatment; side effect risks during and two years posttreatment; support available. Participants indicated preferences for non-surgical vs. surgical treatment on a Likert scale. Baseline responses were analysed using mixed logit, quantifying trade-offs between attributes, using preference for chance of cure as the unit of measurement. Post-estimation, individual preferences conditional on choices were estimated. Changes in mean preferences pre/post-treatment were analysed using multinomial logit, with the delta method used to test for pre/post-treatment differences. Differences in preferences for surgical/nonsurgical management were assessed using Mann-Whitney U tests. Result(s): There were 96 participants recruited, and 51 completed follow-up. Participants had a mean baseline age of 68.4 and were 38.9% female. There were no significant differences between characteristics of people who did/did not complete follow-up. Figure 1 shows distributions of patients' baseline preferences. Patients on average required a 0.34 percentage point (pp) higher chance of cure to accept a 1pp higher chance of short-term side effects, compared to 0.78pp for a 1pp higher chance of long-term side effects, and 3.3pp higher chance of cure to accept support from usual GP rather than a dedicated nurse. The mean chance of cure patients would trade for shorter treatment lengths was not significantly different from 0 (p=.900). Preferences for treatment attributes did not change significantly pre/post-treatment (p-values between.374 and.759, Figure 2a). There was a significant shift in preferences towards non-surgical vs. surgical management post-treatment (p=.017, Figure 2b). Conclusion(s): Participants would accept extra toxicity in exchange for better chances of cure, suggesting most would accept treatment intensification, including dose-escalation. Participants were more concerned about long-term than shortterm side effects, highlighting the need for long-term follow-up of toxicity, and for clinical decision-making to take account of individual patients' preferences.

DOI: 10.1016/S0167-8140%2825%2901971-1