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Beyond Numbers: Uses and Value of In-Trial Interview Data for Regulatory and Health Technology Assessment (HTA) Decision Making.

This research explores the growing importance of qualitative in-trial research (ITR) in regulatory and health technology assessment (HTA) decision making. Since 2020, the Food and Drug Administration (FDA) and European Medicines Agency (EMA) have emphasized incorporating patient experience data into drug development and approval. We reviewed regulatory guidance documents, drug approval records, and HTA reports from January 2017 to March 2025. This included labels and reviews for new drug applications from the FDA and EMA, the Patient-Focused Drug Development guidance series, guidance on rare d

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… . ^^ EMA-reviewed drugs and indications with in-trial interview data: Kayfanda (cholestatic pruritus in Alagille Syndrome adenocarcinoma), Luxturna (Leber congenital amaurosis, retinitis pigmentosa), Opzelura (vitiligo), Paxneury (attention deficit disorder with hyperactivity), Ryeqo (leiomyoma), Vyloy … Area Scoring Index, TTCD time to first confirmed deterioration, UFS-QOL BPS Uterine Fibroid Symptom and Quality of Life Questionnaire Bleeding and Pelvic Discomfort Scale, VNS Vitiligo Noticeable Scale a Sample size is reported as the number of interviews or surveys out of the entire sample Table 3 …

Clinical characteristics and HLA alleles of a family with simultaneously occurring alopecia areata.

Alopecia areata (AA) is a T-cell-mediated autoimmune disease resulting in partial or total noncicatricial hair loss. HLA class II antigens are the most important markers that constitute genetic predisposition to AA. Various life events and intense psychological stress may play an important role in triggering AA attacks. We report an unusual case series of 4 family members who had simultaneously occurring active AA lesions. Our aim was to evaluate the clinical and psychiatric features of 4 cases of active AA lesions occurring simultaneously in a family and determine HLA alleles. The clinical an

Immunotherapy of melanoma: a dichotomy in the requirement for IFN-gamma in vaccine-induced antitumor immunity versus adoptive immunotherapy.

The mechanism by which tumors are rejected following the adoptive transfer of tumor-specific T cells is not well characterized. Recent work has challenged the requirement for cytotoxicity mediated by either the perforin/granzyme or Fas/Fas ligand pathway in T cell-mediated tumor regression. Many reports, including ours, suggest that tumor-specific production of IFN-gamma is critical for T cell-mediated tumor regression. However, in most of these studies the evidence to support the role for IFN-gamma is only indirect. We have directly examined the requirement for IFN-gamma using IFN-gamma knock

Hematopoietic stem cell transplantation: an Italian monocentric experience on the health assessment and eligibility of adult-related donors.

IntroductionIndications for HSCT are increasing worldwide, paralleled by a growing demand for donors of therapeutic cells.MethodsHerein, we report our real-world experience of adult HPC donor assessment during a 5-year study period (2018-2023): we have retrospectively revised data of 455 potential related stem cell donors, consecutively evaluated at our center. Donor medical history was assessed by a questionnaire and an interview with a trained physician experienced in donation procedures to evaluate donor fitness and medical history. Pre-existing health disorders were fully investigated. Beh

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… Among single-organ autoimmune diseases, Hashimoto thyroiditis, Graves’ disease, pernicious anemia, psoriasis, alopecia areata, and vitiligo are included ( 11 ). … They include thyroid diseases, type 1 diabetes, immune thrombocytopenia, vitiligo, and psoriasis ( 38 – 42 ). …

Аутоиммунные заболевания и риск неблагоприятных последствий беременности: five million Беременность в Великобритании.

СПРАВОЧНАЯ ИНФОРМАЦИЯ: С учетом усиливающейся тенденции распространения аутоиммунных заболеваний у матерей в период беременности необходимы доказательства связи между аутоиммунными заболеваниями и результатами беременности. МЕТОДЫ: В рамках данного исследования групп населения использовались данные о беременности в результате применения методов первичной медико-санитарной помощи, которые способствовали созданию в Соединенном Королевстве базы данных исследований в области клинической практики (CPRD) (Gold and Aurum) между 2000 и 2022, связанных со статистикой больничных эпизодов (HES). Модифици

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… Women with vitiligo were more likely to develop hyperemesis gravidarum aRR 1.15 (1.03–1.29), or miscarriage aRR 1.07 (1.00-1.14). … Increased proinflammatory cytokines in autoimmune conditions like psoriasis and vitiligo disrupt immune balance during pregnancy, leading to higher rates of miscarriage and lower live birth rates [ 60 , 61 ]. …

Development Trends of Janus Kinase Inhibitors (JAKi) Over Three Decades: From Common to Rare Diseases.

Janus kinase inhibitors (JAKi) have undergone three decades of research and development, demonstrating substantial therapeutic efficacy and broad clinical applicability in autoimmune diseases, inflammatory disorders, hematopoietic malignancies, and a spectrum of rare diseases. However, their research and development are constrained by three key challenges: the difficulty in target selection and safety concerns arising from their effects on cellular signaling pathways, as well as the challenges in formulating optimal dosing regimens due to interindividual variability in pharmacokinetics. In thi

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… To date, its highest development phase is the Phase III study in PsO, Phase II study in AA and Vitiligo. 56 Given their highly conserved ATP-binding sites, JAKi produce a wide range of effects and cause side effects. … As time passes, the indication scope in IMIDs experiences a significant change, from common IMIDs such RA, AA and vitiligo before 2010 to a surge exploration in rare IMIDs such as PsA, JIA, Giant Cell Arteritis (GCA), Behçet Syndrome (BS), from 2013 to 2025 ( Figure 4B ). …

Идентификация и каталог регуляторов вирусной транскрипции заболеваний человека.

Вирусные геномы кодируют регуляторы вирусной транскрипции (vTRs), которые манипулируют экспрессией гена хоста, чтобы облегчить воспроизведение и избежать иммунной обнаружения. Тем не менее их роль в нераковых болезнях по-прежнему в значительной степени недооценивается. Здесь мы представили 268 новых кандидатов в VTR из 14 из 20 вирусных семей, которые мы расследовали. Мы картировали связующие профили генома VTR и определили их потенциальные цели человека, которые были обогащены иммунно-посредованными путями, нейродегенеративными расстройствами и раком. На основе анализа предпочтений VTR, имеющ

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… Moreover, the terms in other immune-mediated diseases were largely enriched in T cell proliferation and activation, crucial for diseases such as asthma and vitiligo ( Figure S6 B). 41 , 42 Target genes related to neurodegenerative diseases, including AD and HD, were found to be enriched in neuronal … Consistent with the sLDSC analysis, AR, SLE, and vitiligo also showed substantial overlap across multiple vTR-binding sites. …

Drug Repurposing at the Interface of Melanoma Immunotherapy and Autoimmune Disease.

Cancer cells have a remarkable ability to evade recognition and destruction by the immune system. At the same time, cancer has been associated with chronic inflammation, while certain autoimmune diseases predispose to the development of neoplasia. Although cancer immunotherapy has revolutionized antitumor treatment, immune-related toxicities and adverse events detract from the clinical utility of even the most advanced drugs, especially in patients with both, metastatic cancer and pre-existing autoimmune diseases. Here, the combination of multi-omics, data-driven computational approaches with

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… In this context, antibodies against melanocytes provide natural protection against cancer and decreasing the risk of melanoma in vitiligo patients [ 59 ]. … In contrast, a similar antibody-based condition called melanoma-associated hypopigmentation (MAH) or vitiligo-like depigmentation develops in some melanoma patients. …

Skin manifestations associated with systemic diseases - Part I.

The skin demonstrates what is happening in the body in many diseases, as it reflects some internal processes on the surface. In this sense, skin as an organ, goes beyond its protective and barrier functions, as it provides clues for the identification of some systemic diseases. The dermatologist then raises diagnostic hypotheses for conditions related to all systems and refers them to the appropriate specialty. With easy access to examination by trained eyes and biopsies, the skin can present specific or non specific alterations on histopathology. In the first case this combination establishes

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… Specific lesions in mucous membranes: Lichen planus ACLE Neutrophilic dermatoses CCLE Erythema multiforme Seborrheic dermatitis Vitiligo Table 1 Specific and non specific lesions of lupus erythematosus. Specific manifestations of LE | Non specific manifestations of LE 1. … Pigment loss can be so intense as to show vitiligo characteristics ( Fig. 2 ). Scarring and atrophy are not seen. …

Coexistence of Anogenital Psoriasis and Genital Warts - Is There an Optimal Treatment?

The prevalence of psoriasis is 2% of the world's population (1). Inverse psoriasis is characterized by the development of erythematous shiny … er than in patients with psoriasis, the Koebner phenomenon can be found in other skin diseases like vitiligo, lichen planus, lichen nitidus, pityriasis rubra pilaris, flat warts, and keratosis follicularis (Darier disease) (5). According to Eyre at al., about 67% patients with psoriasis (4) present with clearing of psoriatic lesions following skin injury ( …

Incidence, prevalence, and co-occurrence of autoimmune disorders over time and by age, sex, and socioeconomic status: a population-based cohort study of 22 million individuals in the UK.

BACKGROUND: A rise in the incidence of some autoimmune disorders has been described. However, contemporary estimates of the overall incidenc … ariations were observed for childhood-onset type 1 diabetes (more commonly diagnosed in winter) and vitiligo (more commonly diagnosed in summer), and regional variations were observed for a range of conditions. Autoimmune disorders were commonly associated with each other, particularly Sjögren's syndrome, systemic lupus erythematosus, and systemic sclerosi …

Evaluation of the efficacy, safety, and satisfaction rate of topical latanoprost in patients with hypopigmented burn scars treated with fractional CO2 laser: a double-blind randomized controlled clinical trial.

Burn scars present psychological and social challenges for patients, classified into atrophic and hypertrophic types. Treatments like cortic … , latanoprost and bimatoprost, analogs of prostaglandin PGF2a, showed effectiveness in repigmenting vitiligo lesions. However, no study has examined the role of these topical drugs in treating hypopigmented and depigmented burn scars. WHAT DOES THIS STUDY ADD? : The fractional CO2 laser caused significant repigmentation in hypopigmented lesions of burn sca …

Lichen Sclerosus on the Sites of Striae Distensae and a Surgical Scar in a Patient with Coexistent Morphea.

Lichen sclerosus (LS), also known as lichen sclerosus et atrophicus, is a chronic inflammatory mucocutaneous disease affecting the genital a … uring the development of striae has been suggested to cause the Koebner phenomenon in patients with vitiligo, psoriasis, and lichen planus (7), but it has been suggested that the occurrence of leukemia cutis on SD in a patient reflects Wolf's isotopic response (8). Although chronic graft-versus-host disease, urticarial vasculitis, keloid, lupus erythematos …

The role of necroptosis in disease and treatment.

Necroptosis, a distinctive type of programmed cell death different from apoptosis or necrosis, triggered by a series of death receptors such as tumor necrosis factor receptor 1 (TNFR1), TNFR2, and Fas. In case that apoptosis process is blocked, necroptosis pathway is initiated with the activation of three key downstream mediators which are receptor-interacting serine/threonine protein kinase 1 (RIPK1), RIPK3, and mixed lineage kinase domain-like protein (MLKL). The whole process eventually leads to destruction of the cell membrane integrity, swelling of organelles, and severe inflammation. Ove

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… Skin diseases Vitiligo Vitiligo is a common autoimmune depigmenting skin disorder histologically characterized by the extensive destruction of epidermal melanocytes. … Li et al. found that necroptosis was involved in the death of melanocytes through the RIPK1‐RIPK3‐MLKL pathway under oxidative stress in vitiligo. …

Impact of Vitamin D Supplementation on Multiple Sclerosis.

Multiple sclerosis (MS) is an autoimmune disease affecting a large number of people every year. The exact causal factor for this disease is unclear, but it commonly affects middle-aged women, with known triggers like stress, childbirth, infections, poor diet, lack of sleep, etc. Many epidemiological studies have indicated that various genetic abnormalities are also critical drivers of the onset of MS. The major risk factors of MS identified include hypovitaminosis D while environmental protective factors include allele HLA DRB1 1501, obesity, Epstein-Barr virus infection, sexual hormones, and

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… Brazil's clinical research program has been conducting studies with very high dosages of vitamin D3 in order to cure a variety of autoimmune illnesses, including psoriasis, vitiligo, and multiple sclerosis [ 50 ]. … A pilot study assessing the effect of prolonged administration of high daily doses of vitamin D on the clinical course of vitiligo and psoriasis. Finamor DC, Sinigaglia-Coimbra R, Neves LC, et al. Dermatoendocrinol. 2013;5:222–234. doi: 10.4161/derm.24808. 51. …

Association of stiff-person syndrome with autoimmune endocrine diseases.

BackgroundStiff-person syndrome (SPS) and its subtype, stiff limb syndrome (SLS), are rare neurological disorders characterized by progressive muscular rigidity and spasms. Glutamic acid decarboxylase (GAD) is the enzyme that catalyzes the production of γ-aminobutyric acid (GABA), a major inhibitory neurotransmitter of the central nervous system. SPS is an autoimmune disease triggered by anti-glutamic acid decarboxylase antibody (anti-GAD Ab). Clinically, anti-GAD Ab is associated with SPS, type 1 diabetes mellitus (T1DM), and other autoimmune diseases.AimTo investigate the link of autoimmune

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… SPS is strongly associated with other autoimmune diseases, such as type 1 diabetes mellitus (T1DM), autoimmune thyroid disease (AITD), pernicious anemia, vitiligo, and Sjögren syndrome[ 6 ], with symptoms ranging from mild to severe and the potential of developing into a significant disability[ 2 ]. … In addition to T1DM, about 5%–10% of SPS patients also have AITD, Graves’ disease, pernicious anemia, or vitiligo[ 25 ]. Moreover, SPS and a spectrum of anti-GAD ab positive neurologic disorders are also associated with autoimmune polyglandular syndrome type 1 (APS1) and type 2 (APS2). …

The Pharmacology of T Cell Therapies.

Adoptive cellular therapy using T cells with tumor specificity derived from either natural T cell receptors (TCRs) or an artificial chimeric antigen receptor (CAR) has reached late phase clinical testing, with two CAR T cell therapies achieving regulatory approval within the United States in 2017. The effective use of these therapies depends upon an understanding of their pharmacology, which is quite divergent from traditional small molecule or biologic drugs. We review the different types of T cell therapy under clinical development, the factors affecting cellular kinetics following infusion,

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… Off-tumor targeting of immunotherapy antigen has also been associated with T cell immunotherapies for melanoma, in which some responding patients develop vitiligo, immune-mediated destruction of skin melanocytes. … In some cases, melanocytic cells within the eye can also be targeted, leading to ophthalmic complications. 75 Patients who develop vitiligo after melanoma therapy, including adoptive T cell therapy, demonstrate increased survival compared to those who do not develop vitiligo. 76 Severe toxicity due to …

Cancer regression and autoimmunity induced by cytotoxic T lymphocyte-associated antigen 4 blockade in patients with metastatic melanoma.

Cytotoxic T lymphocyte-associated antigen 4 (CTLA-4) is a critical immunoregulatory molecule (expressed on activated T cells and a subset of regulatory T cells) capable of down-regulating T cell activation. Blockade of CTLA-4 has been shown in animal models to improve the effectiveness of cancer immunotherapy. We thus treated 14 patients with metastatic melanoma by using serial i.v. administration of a fully human anti-CTLA-4 antibody (MDX-010) in conjunction with s.c. vaccination with two modified HLA-A*0201-restricted peptides from the gp100 melanoma-associated antigen, gp100:209-217(210M) a

Association of type 1 stiff-person syndrome and insulinoma: a case report and narrative review.

INTRODUCTION: Stiff-person syndrome (SPS) is a rare neurological disorder that causes progressive muscle rigidity, gait disturbances, and functional impairment; type 1 is autoimmune, with positive anti-GAD antibodies (Ab), while type 2 is paraneoplastic and associated with antiamphiphysin Ab. CASE PRESENTATION: A 41-year-old man with a silent medical history presented with stiffness and functional impairment; after numerous rheumatological and neurological investigations, he was diagnosed with SPS, with evidence of high titer anti-GAD Ab. After treatment with benzodiazepines was started, the

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… As already mentioned, SPS may be associated with several autoimmune disorders (e.g., pernicious anemia, vitiligo, celiac disease, and myasthenia gravis), including endocrine diseases, such as type 1 diabetes mellitus (T1DM), Hashimoto’s thyroiditis, and Graves’ disease [ 1 , 7 ]. … In addition to T1DM, LADA, and autoimmune thyroiditis, 5–10% of patients with SPS also have Graves’ disease, pernicious anemia, or vitiligo [ 6 ]. Moreover, SPS is also associated with autoimmune polyglandular syndrome type 1 (APS1) and type 2 (APS2). …

The role of alemtuzumab in the development of secondary autoimmunity in multiple Sclerosis: a systematic review.

BackgroundSecondary autoimmune disease (SAID) in the context of alemtuzumab treatment is one of the main safety concerns that may arise following administration in people with multiple sclerosis (pwMS). Contributing factors underlying this adverse event are not well understood. The purpose of this systematic review was to appraise the literature investigating the role of alemtuzumab in the development of SAID in pwMS following treatment and identify potential biomarkers/ risk factors that may be predictive of onset of this manifestation.MethodsRelevant publications were retrieved from PubMed,

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… Despite B-cell driven autoimmune conditions being most frequently described in the literature, the development of T-cell-mediated autoimmune diseases such as vitiligo and alopecia have also been reported following alemtuzumab treatment in pwMS [ 17 – 19 ]. … Ruck T Vitiligo after alemtuzumab treatment: secondary autoimmunity is not all about B cells Neurology 2018 91 24 e2233 e2237 10.1212/WNL.0000000000006648 30404783 PMC6329330 Ruck T, et al. Vitiligo after alemtuzumab treatment: secondary autoimmunity is not all about B cells. …