2f:I[703058,["/_next/static/immutable/chunks/3bhq_5eh1d760.js","/_next/static/immutable/chunks/2iy5wruuedsh6.js","/_next/static/immutable/chunks/2pfsjpn6k6zlg.js","/_next/static/immutable/chunks/26c7v3_3uwnwu.js","/_next/static/immutable/chunks/00yc7rcvua6a7.js","/_next/static/immutable/chunks/37ktb71aq3480.js","/_next/static/immutable/chunks/2d50b-7eu4558.js","/_next/static/immutable/chunks/06_yt270h41rg.js","/_next/static/immutable/chunks/2d6noqpp6ehas.js","/_next/static/immutable/chunks/1sx186c93bpzg.js","/_next/static/immutable/chunks/3dc-1n866s65i.js","/_next/static/immutable/chunks/20x2y96ldu5wx.js","/_next/static/immutable/chunks/2w1bvggzb0ga8.js","/_next/static/immutable/chunks/2051ffx3xzio9.js"],"PaperListPageClient"] 30:T853,Multisystem Inflammatory Syndrome in Children (MIS-C) is a hyperinflammatory condition with multi-organ involvement, particularly affecting the cardiac and gastrointestinal systems. Although immunomodulatory therapy is standard, treatment approaches vary. This study aimed to evaluate treatment modalities in MIS-C such as methylprednisolone (MP), intravenous immunoglobulin (IVIG), anakinra and therapeutic plasma exchange (TPE) based on clinical severity and laboratory parameters in a prospectively followed cohort. A total of 125 MIS-C patients were included in the study and followed for at least 6 months after discharge. Patients were stratified by severity and treated with various immunomodulatory regimens, including IVIG+MP, IVIG+MP+anakinra, and IVIG+MP+anakinra+TPE. Patients with mild disease and low inflammatory markers (median CRP 9 mg/dL, ferritin 192 µg/dL) received IVIG+MP. Those with higher inflammation (CRP 20-24 mg/dL, ferritin 308-846 µg/dL) without cardio-pulmonary support were treated with IVIG+low-dose-MP+anakinra. Patients with shock, macrophage activation syndrome, or bicytopenia received IVIG+high-dose-MP+anakinra. TPE was added in cases requiring cardio-pulmonary support. Most were discharged without corticosteroids or anakinra; only 11% received a short outpatient prednisolone taper. The mid-term longitudinal assessment of MIS-C patients suggests that timely immunomodulatory therapies, guided by laboratory parameters, promote safe resolution of systemic inflammation and cardiac complications, and shorten treatment duration. Demonstrates that short-term, biomarker-guided use of anakinra and corticosteroids effectively controls hyperinflammation in MIS-C. Highlights that prolonged corticosteroid therapy may not be necessary, even in severe cases. Provides evidence of early cardiac recovery, including resolution of CAAs, without post-discharge steroids. Supports a steroid-sparing treatment approach, reducing risks of long-term immunosuppression. May inform future MIS-C treatment guidelines by minimizing the need for escalation therapy, ECMO, and related complications.31:Te4b,Abstract Background Multisystem Inflammatory Syndrome in Children (MIS-C) is a hyperinflammatory condition with multi-organ involvement, particularly affecting the cardiac and gastrointestinal systems. Although immunomodulatory therapy is standard, treatment approaches vary. This study aimed to evaluate treatment modalities in MIS-C such as methylprednisolone (MP), intravenous immunoglobulin (IVIG), anakinra and therapeutic plasma exchange (TPE) based on clinical severity and laboratory parameters in a prospectively followed cohort. Methods A total of 125 MIS-C patients were included in the study and followed for at least 6 months after discharge. Patients were stratified by severity and treated with various immunomodulatory regimens, including IVIG+MP, IVIG+MP+anakinra, and IVIG+MP+anakinra+TPE. Results Patients with mild disease and low inflammatory markers (median CRP 9 mg/dL, ferritin 192 µg/dL) received IVIG+MP. Those with higher inflammation (CRP 20–24 mg/dL, ferritin 308–846 µg/dL) without cardio-pulmonary support were treated with IVIG+low-dose-MP+anakinra. Patients with shock, macrophage activation syndrome, or bicytopenia received IVIG+high-dose-MP+anakinra. TPE was added in cases requiring cardio-pulmonary support. Most were discharged without corticosteroids or anakinra; only 11% received a short outpatient prednisolone taper. Conclusion The mid-term longitudinal assessment of MIS-C patients suggests that timely immunomodulatory therapies, guided by laboratory parameters, promote safe resolution of systemic inflammation and cardiac complications, and shorten treatment duration. Impact

Demonstrates that short-term, biomarker-guided use of anakinra and corticosteroids effectively controls hyperinflammation in MIS-C.

Highlights that prolonged corticosteroid therapy may not be necessary, even in severe cases.

Provides evidence of early cardiac recovery, including resolution of CAAs, without post-discharge steroids.

Supports a steroid-sparing treatment approach, reducing risks of long-term immunosuppression.

May inform future MIS-C treatment guidelines by minimizing the need for escalation therapy, ECMO, and related complications.

32:T803,Kawasaki disease (KD) is the leading cause of acquired childhood heart disease, with untreated cases progressing to giant coronary artery aneurysms (GCAAs) in 2-3% of patients. While outcomes are well-documented in high-income settings, data from low- and middle-income countries (LMICs) remain limited. This study examines clinical profiles, treatment responses, and outcomes of children with GCAAs managed at a tertiary care center in South India. We conducted a retrospective review of 18 children with KD-associated GCAAs (z-score >10 or diameter >8mm) treated between 2019-2024 at a tertiary care center in Kerala, India. All patients were referred from external centers with documented IVIg resistance. Treatment intensification included infliximab, corticosteroids, cyclosporine, and anakinra. Primary outcome was GCAA dimension change at last follow-up; secondary outcomes included thrombotic events, mortality, and anticoagulation cessation. The cohort comprised predominantly of males (n = 14, 77.8%) with median symptom onset at 1.1 (IQR 0.3 - 2.7) years; 50% were under one year old. While ten children (55%) experienced delayed diagnosis (>10 days after symptom onset) with median diagnosis at 11 (IQR 7 - 13.5) days; all faced delays in treatment intensification. Excluding two children lost-to-follow-up, during the median follow-up of 8.6 (5.2 - 19.4) months, complete GCAA resolution occurred in three (18.8%), reduction to small/medium aneurysms in six (37.6%), while five (31.3%) remained static. One mortality (6.3%) from myocardial infarction and a patient requiring coronary artery bypass grafting were observed. Anticoagulation was discontinued in 56.25% of children whose aneurysms resolved or reduced significantly. Delayed diagnosis and treatment intensification may have contributed to mixed outcomes in this GCAA cohort. Early recognition, appropriate risk stratification, and timely immunosuppression intensification are essential to improve prognosis and reduce severe coronary complications in resource-limited settings.33:T506,Hemophagocytic lymphohistiocytosis (HLH) is a rare syndrome characterized by severe hyperinflammation, leading to endorgan damage and death in the absence of treatments directed at suppressing the overactive immune system. The study of mouse models and human biospecimens has provided insights into disease pathophysiology and enabled investigations of novel therapeutic strategies. Nevertheless, clinical outcomes remain suboptimal. Additional research is required to further our understanding of this highly heterogeneous disease in order to develop improved diagnostic and prognostic tools and identify and implement targeted and less toxic treatments. In this article, written on behalf of the Histiocyte Society, we highlight six key areas of ongoing research, including the remaining questions in each area. We also outline the challenges of global dissemination and implementation of this research and call for the establishment of local, regional, and national cooperative groups aimed at providing education for physicians and targeted diagnostic and clinical guidelines. Finally, we discuss the role of international societies, including the Histiocyte Society, in nucleating this work to ensure coordinated efforts aimed at improving outcomes for patients with HLH worldwide.34:T517,Hemophagocytic lymphohistiocytosis (HLH) is a rare syndrome characterized by severe hyperinflammation, leading to endorgan damage and death in the absence of treatments directed at suppressing the overactive immune system. The study of mouse models and human biospecimens has provided insights into disease pathophysiology and enabled investigations of novel therapeutic strategies. Nevertheless, clinical outcomes remain suboptimal. Additional research is required to further our understanding of this highly heterogeneous disease in order to develop improved diagnostic and prognostic tools and identify and implement targeted and less toxic treatments. In this article, written on behalf of the Histiocyte Society, we highlight six key areas of ongoing research, including the remaining questions in each area. We also outline the challenges of global dissemination and implementation of this research and call for the establishment of local, regional, and national cooperative groups aimed at providing education for physicians and targeted diagnostic and clinical guidelines. Finally, we discuss the role of international societies, including the Histiocyte Society, in nucleating this work to ensure coordinated efforts aimed at improving outcomes for patients with HLH worldwide.35:T6eb,The aim of this study was to compare differences in clinical response, drug survival, and adverse event rates between anakinra and canakinumab in VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome. This multicenter international study includes patients with VEXAS from France, Israel, and Italy treated with interleukin-1 inhibition. Global response (GR) was defined as the absence of inflammatory symptoms and ≥50% decrease in steroid dose and C-reactive protein. Multiple regression analysis was performed to identify associated variables. Drug survival was analyzed using Kaplan-Meier plots and log-rank test, with Cox regression models for associated factors. We included 47 male patients with VEXAS; 44 received anakinra and 9 received canakinumab, with 6 patients using both at different time points. GR at 1 month was 34% for anakinra and 100% for canakinumab (P < 0.001) and 22% and 78% at 3 months, respectively (P = 0.001). Treatment with canakinumab was associated with a higher odds ratio (OR) of achieving GR at 3 months (OR 28.8, 95% confidence interval 3.0-273.9; P = 0.004) in a multivariable analysis. Median drug survival was 54 (interquartile range [IQR] 30-56) months for canakinumab at 300 mg/month compared with 7 (IQR 4-8) months for canakinumab 150 mg/month and 1 (IQR 1-2.5) months for anakinra (P = 0.01). Injection-site reactions were only recorded for the anakinra group (47 vs 0%; P = 0.006), whereas infections were more frequent in the anakinra group (31% and 11%; P = 0.3). Canakinumab demonstrated superior clinical response and drug survival with fewer adverse events compared with anakinra. Monthly canakinumab 300 mg may be considered as an effective steroid-sparing therapeutic option for patients with VEXAS.36:Td08, Objective The aim of this study was to compare differences in clinical response, drug survival, and adverse event rates between anakinra and canakinumab in VEXAS (vacuoles, E1 enzyme, X‐linked, autoinflammatory, somatic) syndrome. Methods This multicenter international study includes patients with VEXAS from France, Israel, and Italy treated with interleukin‐1 inhibition. Global response (GR) was defined as the absence of inflammatory symptoms and ≥50% decrease in steroid dose and C‐reactive protein. Multiple regression analysis was performed to identify associated variables. Drug survival was analyzed using Kaplan‐Meier plots and log‐rank test, with Cox regression models for associated factors. Results We included 47 male patients with VEXAS; 44 received anakinra and 9 received canakinumab, with 6 patients using both at different time points. GR at 1 month was 34% for anakinra and 100% for canakinumab ( P < 0.001) and 22% and 78% at 3 months, respectively ( P = 0.001). Treatment with canakinumab was associated with a higher odds ratio (OR) of achieving GR at 3 months (OR 28.8, 95% confidence interval 3.0–273.9; P = 0.004) in a multivariable analysis. Median drug survival was 54 (interquartile range [IQR] 30–56) months for canakinumab at 300 mg/month compared with 7 (IQR 4–8) months for canakinumab 150 mg/month and 1 (IQR 1–2.5) months for anakinra ( P = 0.01). Injection‐site reactions were only recorded for the anakinra group (47 vs 0%; P = 0.006), whereas infections were more frequent in the anakinra group (31% and 11%; P = 0.3). Conclusion Canakinumab demonstrated superior clinical response and drug survival with fewer adverse events compared with anakinra. Monthly canakinumab 300 mg may be considered as an effective steroid‐sparing therapeutic option for patients with VEXAS.

image

37:T43c,Among the 112 patients (median [range] age, 69 [39-79] years; 111 [99%] male), skin involvement was common (93 [83%]), and the most frequent presenting feature of disease (68 [61%]). Of 64 histopathologic reports available from 60 patients, predominant skin histopathologic findings were leukocytoclastic vasculitis (23 [36%]), neutrophilic dermatosis (22 [34%]), and perivascular dermatitis (19 [30%]). Distinct pathogenic genetic variants were associated with specific cutaneous manifestations. The p.Met41Leu variant was most frequently associated with neutrophilic dermal infiltrates (14 of 17 patients [82%]), often resembling histiocytoid Sweet syndrome. In contrast, the p.Met41Val variant was associated with vasculitic lesions (11 of 20 patients [55%]) with a mixed leukocytic infiltrate (17 of 20 patients [85%]). Oral prednisone improved skin manifestations in 67 of 73 patients (92%). Patients with VEXAS treated with anakinra frequently developed severe injection-site reactions (12 of 16 [75%]), including ulceration (2 of 12 [17%]) and abscess formation (1 of 12 [8%]).38:T946,VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is a newly defined genetic disease with an estimated prevalence of 1 in 4269 men older than 50 years and is marked by systemic inflammation, progressive bone marrow failure, and inflammatory cutaneous manifestations. To define the spectrum of cutaneous manifestations in VEXAS syndrome and the association of these findings with clinical, genetic, and histological features. This observational cohort study included data from 112 patients who were diagnosed with VEXAS-defining genetic variants in UBA1 between 2019 and 2023. Data were collected from medical record review or from patients with VEXAS directly evaluated at the National Institutes of Health in Bethesda, Maryland. To define the spectrum of cutaneous manifestations in VEXAS in association with genetic, histological, and other clinical findings. A secondary outcome was cutaneous response to treatment in VEXAS. Among the 112 patients (median [range] age, 69 [39-79] years; 111 [99%] male), skin involvement was common (93 [83%]), and the most frequent presenting feature of disease (68 [61%]). Of 64 histopathologic reports available from 60 patients, predominant skin histopathologic findings were leukocytoclastic vasculitis (23 [36%]), neutrophilic dermatosis (22 [34%]), and perivascular dermatitis (19 [30%]). Distinct pathogenic genetic variants were associated with specific cutaneous manifestations. The p.Met41Leu variant was most frequently associated with neutrophilic dermal infiltrates (14 of 17 patients [82%]), often resembling histiocytoid Sweet syndrome. In contrast, the p.Met41Val variant was associated with vasculitic lesions (11 of 20 patients [55%]) with a mixed leukocytic infiltrate (17 of 20 patients [85%]). Oral prednisone improved skin manifestations in 67 of 73 patients (92%). Patients with VEXAS treated with anakinra frequently developed severe injection-site reactions (12 of 16 [75%]), including ulceration (2 of 12 [17%]) and abscess formation (1 of 12 [8%]). Results of this cohort study show that skin manifestations are a common and early manifestation of VEXAS syndrome. Genetic evaluation for VEXAS should be considered in older male patients with cutaneous vasculitis, neutrophilic dermatoses, or chondritis. Awareness of VEXAS among dermatologists is critical to facilitate early diagnosis.39:Tbbc,ImportanceVEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is a newly defined genetic disease with an estimated prevalence of 1 in 4269 men older than 50 years and is marked by systemic inflammation, progressive bone marrow failure, and inflammatory cutaneous manifestations.ObjectiveTo define the spectrum of cutaneous manifestations in VEXAS syndrome and the association of these findings with clinical, genetic, and histological features.Design, Setting, and ParticipantsThis observational cohort study included data from 112 patients who were diagnosed with VEXAS-defining genetic variants in UBA1 between 2019 and 2023. Data were collected from medical record review or from patients with VEXAS directly evaluated at the National Institutes of Health in Bethesda, Maryland.Main Outcomes and MeasuresTo define the spectrum of cutaneous manifestations in VEXAS in association with genetic, histological, and other clinical findings. A secondary outcome was cutaneous response to treatment in VEXAS.ResultsAmong the 112 patients (median [range] age, 69 [39-79] years; 111 [99%] male), skin involvement was common (93 [83%]), and the most frequent presenting feature of disease (68 [61%]). Of 64 histopathologic reports available from 60 patients, predominant skin histopathologic findings were leukocytoclastic vasculitis (23 [36%]), neutrophilic dermatosis (22 [34%]), and perivascular dermatitis (19 [30%]). Distinct pathogenic genetic variants were associated with specific cutaneous manifestations. The p.Met41Leu variant was most frequently associated with neutrophilic dermal infiltrates (14 of 17 patients [82%]), often resembling histiocytoid Sweet syndrome. In contrast, the p.Met41Val variant was associated with vasculitic lesions (11 of 20 patients [55%]) with a mixed leukocytic infiltrate (17 of 20 patients [85%]). Oral prednisone improved skin manifestations in 67 of 73 patients (92%). Patients with VEXAS treated with anakinra frequently developed severe injection-site reactions (12 of 16 [75%]), including ulceration (2 of 12 [17%]) and abscess formation (1 of 12 [8%]).Conclusions and RelevanceResults of this cohort study show that skin manifestations are a common and early manifestation of VEXAS syndrome. Genetic evaluation for VEXAS should be considered in older male patients with cutaneous vasculitis, neutrophilic dermatoses, or chondritis. 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All structured content is inferred from the title and journal context alone; key details such as sample size, study design, patient demographics, and specific outcomes are unknown and could not be extracted.","value_numeric":null},{"paper_doi":"10.1016/j.jaci.2026.08.001","tag_type":"warning","value":"The generic tag 'anakinra' (recombinant IL-1Ra) is included as a clinically relevant agent in this context but is not explicitly confirmed as studied or mentioned in the source text.","value_numeric":null},{"paper_doi":"10.1016/j.jaci.2026.08.001","tag_type":"warning","value":"The study_design and level_of_evidence tags were omitted because the design cannot be determined from the title alone.","value_numeric":null}],"summaries":{"what_studied":"The study evaluated the presence and role of neutralizing autoantibodies against IL-1 receptor antagonist (IL-1Ra) in patients with severe bacterial community-acquired pneumonia (CAP).","key_result":"Neutralizing autoantibodies against IL-1Ra were identified in a subset of patients with severe bacterial CAP, suggesting these autoantibodies may impair IL-1Ra function and contribute to dysregulated inflammation in this condition.","limitations":"The paper abstract provides limited methodological detail, making it difficult to assess sample size, study design rigor, and generalizability of findings.","practice_takeaway":"Clinicians should be aware that neutralizing anti-IL-1Ra autoantibodies may underlie hyperinflammatory presentations in some patients with severe bacterial CAP. Screening for these autoantibodies could eventually help identify patients who might benefit from IL-1 pathway–targeted therapies."}},{"doi":"10.1002/hem3.70425","pmid":"42472032","title":"Hemophagocytic lymphohistiocytosis‐like syndrome after CD19‐directed CAR T‐cells for B‐cell lymphoma and B‐cell acute lymphoblastic leukemia: A LYSA, SFCE, and GRAALL study from the DESCAR‐T registry","journal_title":"HemaSphere","journal_issn":"2572-9241","published_at":"2026-07-18T00:00:00.000Z","url":"https://doi.org/10.1002/hem3.70425","source_score":10,"is_retracted":false,"is_open_access":true,"raw_metadata":{"pubmed":true,"crossref":true,"unpaywall":true},"created_at":"2026-07-26T13:44:33.073Z","updated_at":"2026-07-26T13:44:33.073Z","tags":[{"paper_doi":"10.1002/hem3.70425","tag_type":"age_group","value":"mixed","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"brand","value":"Breyanzi","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"brand","value":"Kymriah","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"brand","value":"Tecartus","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"brand","value":"Yescarta","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"category","value":"Hematology","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"condition","value":"B-Cell Acute Lymphoblastic Leukemia","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"condition","value":"B-Cell Non-Hodgkin Lymphoma","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"condition","value":"Cytokine Release Syndrome","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"condition","value":"Immune Effector Cell-Associated Hemophagocytic Lymphohistiocytosis-Like Syndrome","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"generic","value":"anakinra","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"generic","value":"axicabtagene ciloleucel","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"generic","value":"brexucabtagene autoleucel","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"generic","value":"etoposide","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"generic","value":"lisocabtagene maraleucel","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"generic","value":"tisagenlecleucel","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"generic","value":"tocilizumab","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"impact_flag","value":"safety_signal","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"level_of_evidence","value":"4","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"modality","value":"CAR T-Cell Therapy","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"modality","value":"Corticosteroid","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"modality","value":"IL-1 Receptor Antagonist","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"modality","value":"IL-6 Receptor Antagonist","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"modality","value":"Topoisomerase II Inhibitor","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"sample_size","value":"n=42","value_numeric":42},{"paper_doi":"10.1002/hem3.70425","tag_type":"sex","value":"mixed","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"study_design","value":"Multicenter Retrospective Cohort Study","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"warning","value":"Brand name tags for CAR T-cell products are inferred from generic names per standard labeling; the paper itself uses generic names only.","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"warning","value":"Incidence estimates (1.3% B-NHL, 8.0% B-ALL) should be interpreted with caution due to retrospective ascertainment bias and enrichment from centers with recognized IEC-HS cases.","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"warning","value":"The mixed pediatric and adult population (age range 1–76 years) limits generalizability to any single age group; B-ALL patients were predominantly pediatric/young adult (median age 18.5 years) while B-NHL patients were adults (median age 61.5 years).","value_numeric":null},{"paper_doi":"10.1002/hem3.70425","tag_type":"warning","value":"Treatment response rates are descriptive only; no comparative analysis was possible given small subgroup sizes and substantial indication bias (sicker patients received more intensive therapy).","value_numeric":null}],"summaries":{"what_studied":"This multicenter retrospective study from the French DESCAR-T registry described the incidence, features, management, and outcomes of immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome (IEC-HS) in 42 patients who received standard-of-care CD19 CAR T-cell therapy for relapsed/refractory B-NHL (n=28) or B-ALL (n=14), with a median follow-up of 24.1 months.","key_result":"IEC-HS occurred in 1.3% of B-NHL and 8.0% of B-ALL patients; overall mortality was 81%, with 1-year OS of 15.6% for B-NHL and 47.1% for B-ALL. Median ferritin at IEC-HS onset was 17,061–55,000 µg/L by disease type. Etoposide had the highest observed response rate (ORR 77%), followed by corticosteroids ± anakinra (ORR 70%), and tocilizumab (ORR 50%). Six-month non-relapse mortality was 24.1%, 4–5× higher than reported in general CAR T-cell cohorts.","limitations":"- Retrospective design with ascertainment bias: four additional cases identified outside the registry export may have enriched for more severe presentations, inflating incidence estimates.\n- Small sample size (n=42) prevents formal comparisons between treatments, CAR T-cell products, or disease subgroups; all subgroup findings are exploratory.\n- Inability to distinguish new-onset from persistent fever at IEC-HS onset limits differentiation from ongoing CRS with HLH-like features.","practice_takeaway":"IEC-HS after CD19 CAR T-cell therapy carries ~81% mortality and a 6-month NRM of 24%; clinicians should suspect it early (median onset Day 9) in patients with ferritin >10,000 µg/L, severe CRS, and a high CAR-HEMATOTOX score ≥2. Corticosteroids and/or anakinra appear reasonable first-line options, but aggressive infection prophylaxis (mold-active antifungals, letermovir for CMV, IVIG) is critical given infections account for 55% of NRM."}},{"doi":"10.1002/art.70262","pmid":"42370484","title":"Expert Perspective: Treatment of Patients With Calcium Pyrophosphate Deposition Disease: Where Do We Begin and Where Are We Going?","journal_title":"Arthritis & Rheumatology","journal_issn":"2326-5191","published_at":"2026-07-14T00:00:00.000Z","url":"https://doi.org/10.1002/art.70262","source_score":15,"is_retracted":false,"is_open_access":false,"raw_metadata":{"pubmed":true,"crossref":true,"unpaywall":true},"created_at":"2026-08-04T18:17:42.083Z","updated_at":"2026-08-04T18:17:42.083Z","tags":[{"paper_doi":"10.1002/art.70262","tag_type":"age_group","value":"older_adult","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"brand","value":"Kineret","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"category","value":"Rheumatology","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"condition","value":"Calcium Pyrophosphate Deposition Disease","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"condition","value":"Crystal Arthritis","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"generic","value":"anakinra","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"generic","value":"baricitinib","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"generic","value":"colchicine","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"generic","value":"hydroxychloroquine","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"generic","value":"methotrexate","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"generic","value":"tocilizumab","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"impact_flag","value":"limited_evidence","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"level_of_evidence","value":"6","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"modality","value":"Antimalarial","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"modality","value":"Antimetabolite","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"modality","value":"Corticosteroid","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"modality","value":"IL-1 Inhibitor","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"modality","value":"IL-6 Inhibitor","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"modality","value":"JAK Inhibitor","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"study_design","value":"Expert Review / Narrative Review","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"warning","value":"Baricitinib is tagged as a JAK Inhibitor based on its drug class; its role in CPPD is investigational and not yet established.","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"warning","value":"No sample size is applicable as this is a review article, not a primary study.","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"warning","value":"Study design label 'Expert Review / Narrative Review' is used as the article does not fit a standard trial design category.","value_numeric":null},{"paper_doi":"10.1002/art.70262","tag_type":"warning","value":"This is an expert perspective/narrative review, not a systematic review or RCT; the level of evidence is inherently low and recommendations reflect author opinion synthesized from limited trial data.","value_numeric":null}],"summaries":{"what_studied":"This expert review proposes a practical treatment framework for calcium pyrophosphate deposition (CPPD) disease in older adults, covering acute and chronic manifestations, and summarizes evidence from randomized trials, open-label studies, and ongoing clinical trials.","key_result":"For acute CPP crystal arthritis, supported options include short-course systemic glucocorticoids, colchicine, intra-articular glucocorticoids, and anakinra. For chronic disease or recurrent flares, colchicine, hydroxychloroquine, methotrexate, IL-1 inhibition, and IL-6 inhibition are discussed. Ongoing trials are evaluating colchicine, tocilizumab, and baricitinib.","limitations":"No approved disease-modifying therapy exists for CPPD; the review is an expert perspective rather than a systematic review or meta-analysis, so recommendations reflect limited clinical trial evidence rather than high-quality RCT data.","practice_takeaway":"Before selecting therapy, confirm CPPD diagnosis and attribute current symptoms to CPPD specifically. Tailor treatment to disease manifestation—acute flare vs. chronic/recurrent disease—using available agents while awaiting results of ongoing trials and future disease-modifying strategies."}},{"doi":"10.1161/strokeaha.125.054941","pmid":"41847765","title":"Timing-Dependent Cleavage of Interleukin-1 Receptor Antagonist by Alteplase Impairs Neuroprotection in Ischemic Stroke","journal_title":"Stroke","journal_issn":"0039-2499","published_at":"2026-03-18T00:00:00.000Z","url":"https://doi.org/10.1161/strokeaha.125.054941","source_score":40,"is_retracted":false,"is_open_access":false,"raw_metadata":{"pubmed":true,"crossref":true,"unpaywall":true},"created_at":"2026-05-26T13:35:14.253Z","updated_at":"2026-05-26T13:35:14.253Z","tags":[{"paper_doi":"10.1161/strokeaha.125.054941","tag_type":"age_group","value":"adult","value_numeric":null},{"paper_doi":"10.1161/strokeaha.125.054941","tag_type":"category","value":"Clinical Neurology","value_numeric":null},{"paper_doi":"10.1161/strokeaha.125.054941","tag_type":"condition","value":"Ischemic Stroke","value_numeric":null},{"paper_doi":"10.1161/strokeaha.125.054941","tag_type":"generic","value":"alteplase","value_numeric":null},{"paper_doi":"10.1161/strokeaha.125.054941","tag_type":"generic","value":"anakinra","value_numeric":null},{"paper_doi":"10.1161/strokeaha.125.054941","tag_type":"impact_flag","value":"safety_signal","value_numeric":null},{"paper_doi":"10.1161/strokeaha.125.054941","tag_type":"level_of_evidence","value":"5","value_numeric":null},{"paper_doi":"10.1161/strokeaha.125.054941","tag_type":"modality","value":"IL-1 Receptor Antagonist","value_numeric":null},{"paper_doi":"10.1161/strokeaha.125.054941","tag_type":"modality","value":"Thrombolytic Therapy","value_numeric":null},{"paper_doi":"10.1161/strokeaha.125.054941","tag_type":"sample_size","value":"n=13","value_numeric":13},{"paper_doi":"10.1161/strokeaha.125.054941","tag_type":"sex","value":"male","value_numeric":null},{"paper_doi":"10.1161/strokeaha.125.054941","tag_type":"study_design","value":"Preclinical Animal Study with Retrospective Clinical Data Reanalysis","value_numeric":null},{"paper_doi":"10.1161/strokeaha.125.054941","tag_type":"warning","value":"Mouse model used only male animals — findings on sex-specific interactions with IL-1Ra/tPA co-administration remain unknown.","value_numeric":null},{"paper_doi":"10.1161/strokeaha.125.054941","tag_type":"warning","value":"Sample size tag reflects the per-group mouse cohort (n=10–13); total trial N for SCIL-STROKE human reanalysis is not specified in the abstract. The study design spans two methodologies (retrospective clinical reanalysis + animal experiment), which may affect level of evidence assignment.","value_numeric":null},{"paper_doi":"10.1161/strokeaha.125.054941","tag_type":"warning","value":"The brand name 'Kineret' for anakinra was not mentioned in the source text; only the generic name was used.","value_numeric":null}],"summaries":{"what_studied":"This study investigated whether a negative interaction exists between IL-1Ra (anakinra) and tPA (alteplase) in ischemic stroke, combining a retrospective reanalysis of the SCIL-STROKE phase II trial with a thromboembolic mouse model (male wild-type mice, n=10–13 per group) to assess lesion volume at 72 hours by T2-MRI.","key_result":"Plasma IL-1Ra was 36.7% lower in SCIL-STROKE patients who received preceding tPA, consistent with plasmin-mediated cleavage confirmed biochemically. In mice, IL-1Ra given *during* thrombolysis reduced the benefit of tPA dramatically — lesion volume fell only 15% vs. 68% with tPA alone — and was accompanied by reduced tissue perfusion, more leukocyte infiltration, and increased neutrophil extracellular traps; no negative interaction was seen when tPA preceded IL-1Ra.","limitations":"Mouse model used only male animals, limiting generalizability to both sexes. The SCIL-STROKE reanalysis is retrospective and observational, making causality inference difficult. The preclinical dosing regimen did not fully replicate the timing of concurrent clinical co-administration.","practice_takeaway":"If IL-1Ra is used alongside tPA in ischemic stroke, timing matters critically — administer tPA first and avoid co-administering IL-1Ra *during* thrombolysis to prevent impaired reperfusion and worsened outcomes. Clinicians should also consider that tPA-driven plasmin activity may degrade other co-administered neuroprotective proteins, warranting timing scrutiny for any such combination."}},{"doi":"10.1038/s41390-025-04706-6","pmid":"41519951","title":"Multisystem Inflammatory Syndrome in Children with tailored therapy and six-month outcome","journal_title":"Pediatric Research","journal_issn":"0031-3998","published_at":"2026-01-11T00:00:00.000Z","url":"https://doi.org/10.1038/s41390-025-04706-6","source_score":21,"is_retracted":false,"is_open_access":true,"raw_metadata":{"ooir":{"day":"2026-01-12","doi":"10.1038/s41390-025-04706-6","days":"7","issn":"0031-3998","rank":"6","field":"Clinical Medicine","score":"21","category":"Pediatrics","categories":["Pediatrics"],"_categories":["Pediatrics"]},"pubmed":{"doi":"10.1038/s41390-025-04706-6","title":"Multisystem Inflammatory Syndrome in Children with tailored therapy and six-month outcome.","journal":"Pediatric research","methods":"","results":"Patients with mild disease and low inflammatory markers (median CRP 9 mg/dL, ferritin 192 µg/dL) received IVIG+MP. Those with higher inflammation (CRP 20-24 mg/dL, ferritin 308-846 µg/dL) without cardio-pulmonary support were treated with IVIG+low-dose-MP+anakinra. Patients with shock, macrophage activation syndrome, or bicytopenia received IVIG+high-dose-MP+anakinra. TPE was added in cases requiring cardio-pulmonary support. Most were discharged without corticosteroids or anakinra; only 11% received a short outpatient prednisolone taper.","abstract":"$30","keywords":[],"pubmed_id":"41519951","conclusions":"The mid-term longitudinal assessment of MIS-C patients suggests that timely immunomodulatory therapies, guided by laboratory parameters, promote safe resolution of systemic inflammation and cardiac complications, and shorten treatment duration.","publication_date":"2026-01-11T00:00:00"},"crossref":{"DOI":"10.1038/s41390-025-04706-6","URL":"https://doi.org/10.1038/s41390-025-04706-6","ISSN":["0031-3998","1530-0447"],"link":[{"URL":"https://www.nature.com/articles/s41390-025-04706-6.pdf","content-type":"application/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https://www.nature.com/articles/s41390-025-04706-6","content-type":"text/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https://www.nature.com/articles/s41390-025-04706-6.pdf","content-type":"application/pdf","content-version":"vor","intended-application":"similarity-checking"}],"page":null,"type":"journal-article","issue":null,"score":1,"title":["Multisystem Inflammatory Syndrome in Children with tailored therapy and six-month outcome"],"author":[{"given":"Osman Oguz","family":"Demir","sequence":"first","affiliation":[]},{"given":"Kubra","family":"Aykac","sequence":"additional","affiliation":[]},{"given":"Arthur Hoi Hin","family":"Cheng","sequence":"additional","affiliation":[]},{"given":"Selman","family":"Kesici","sequence":"additional","affiliation":[]},{"given":"H. 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M.","family":"Yeung","sequence":"additional","affiliation":[]},{"given":"Yasemin","family":"Ozsurekci","sequence":"additional","affiliation":[]},{"given":"Seza","family":"Ozen","sequence":"additional","affiliation":[]}],"issued":{"date-parts":[[2026,1,10]]},"member":"297","prefix":"10.1038","source":"Crossref","volume":null,"created":{"date-time":"2026-01-10T20:15:19Z","timestamp":1768076119000,"date-parts":[[2026,1,10]]},"indexed":{"date-time":"2026-01-11T23:51:19Z","timestamp":1768175479004,"date-parts":[[2026,1,11]]},"license":[{"URL":"https://creativecommons.org/licenses/by/4.0","start":{"date-time":"2026-01-10T00:00:00Z","timestamp":1768003200000,"date-parts":[[2026,1,10]]},"delay-in-days":0,"content-version":"tdm"},{"URL":"https://creativecommons.org/licenses/by/4.0","start":{"date-time":"2026-01-10T00:00:00Z","timestamp":1768003200000,"date-parts":[[2026,1,10]]},"delay-in-days":0,"content-version":"vor"}],"subject":[],"abstract":"$31","language":"en","relation":{},"resource":{"primary":{"URL":"https://www.nature.com/articles/s41390-025-04706-6"}},"subtitle":[],"assertion":[{"name":"received","group":{"name":"ArticleHistory","label":"Article History"},"label":"Received","order":1,"value":"13 May 2025"},{"name":"revised","group":{"name":"ArticleHistory","label":"Article History"},"label":"Revised","order":2,"value":"28 August 2025"},{"name":"accepted","group":{"name":"ArticleHistory","label":"Article History"},"label":"Accepted","order":3,"value":"20 November 2025"},{"name":"first_online","group":{"name":"ArticleHistory","label":"Article History"},"label":"First Online","order":4,"value":"10 January 2026"},{"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"},"order":1,"value":"The authors declare no competing interests."}],"deposited":{"date-time":"2026-01-11T21:07:19Z","timestamp":1768165639000,"date-parts":[[2026,1,11]]},"issn-type":[{"type":"print","value":"0031-3998"},{"type":"electronic","value":"1530-0447"}],"published":{"date-parts":[[2026,1,10]]},"publisher":"Springer Science and Business Media LLC","reference":[{"DOI":"10.1016/S0140-6736(20)31094-1","key":"4706_CR1","year":"2020","issue":null,"author":"S Riphagen","volume":"395","first-page":"1607","unstructured":"Riphagen, S., Gomez, X., Gonzalez-Martinez, C., Wilkinson, N. & Theocharis, P. 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Treatment intensity was guided by CRP and ferritin (mild: CRP 9 mg/dL, ferritin 192 µg/dL; higher inflammation without cardio-pulmonary support: CRP 20–24 mg/dL, ferritin 308–846 µg/dL; shock/MAS/bicytopenia or cardio-pulmonary support prompted escalation including high-dose MP, anakinra, and TPE).","limitations":"Severity-guided treatment allocation in this prospective cohort limits causal inference between regimens. The abstract provides few outcome metrics (e.g., no event rates for cardiac recovery, ICU/ECMO use, or adverse events).","practice_takeaway":"When treating MIS-C, use CRP, ferritin, and clinical severity to tailor short-course IVIG+MP±anakinra, reserving TPE for cardio-pulmonary support. Prolonged post-discharge steroids may be unnecessary, as only 11% needed a brief taper."}},{"doi":"10.1186/s12969-025-01178-0","pmid":"41462272","title":"Clinical outcomes of giant coronary aneurysms in South Asian children with Kawasaki disease","journal_title":"Pediatric Rheumatology","journal_issn":"1546-0096","published_at":"2025-12-30T00:00:00.000Z","url":"https://doi.org/10.1186/s12969-025-01178-0","source_score":2,"is_retracted":false,"is_open_access":true,"raw_metadata":{"ooir":{"day":"2026-01-04","doi":"10.1186/s12969-025-01178-0","days":"14","issn":"1546-0096","rank":"10","field":"Clinical Medicine","score":"2","category":"Rheumatology","categories":["Rheumatology"],"_categories":["Rheumatology"]},"pubmed":{"doi":"10.1186/s12969-025-01178-0","title":"Clinical outcomes of giant coronary aneurysms in South Asian children with Kawasaki disease.","journal":"Pediatric rheumatology online journal","methods":"","results":"The cohort comprised predominantly of males (n = 14, 77.8%) with median symptom onset at 1.1 (IQR 0.3 - 2.7) years; 50% were under one year old. While ten children (55%) experienced delayed diagnosis (>10 days after symptom onset) with median diagnosis at 11 (IQR 7 - 13.5) days; all faced delays in treatment intensification. Excluding two children lost-to-follow-up, during the median follow-up of 8.6 (5.2 - 19.4) months, complete GCAA resolution occurred in three (18.8%), reduction to small/medium aneurysms in six (37.6%), while five (31.3%) remained static. One mortality (6.3%) from myocardial infarction and a patient requiring coronary artery bypass grafting were observed. Anticoagulation was discontinued in 56.25% of children whose aneurysms resolved or reduced significantly.","abstract":"$32","keywords":["Child","Coronary aneurysm","Intravenous immunoglobulins","Mucocutaneous lymph node syndrome","Retrospective studies","Treatment outcome","Vasculitis"],"pubmed_id":"41462272","conclusions":"Delayed diagnosis and treatment intensification may have contributed to mixed outcomes in this GCAA cohort. 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Primary outcome was change in aneurysm size at last follow-up; median follow-up among 16 with data was 8.6 months (IQR 5.2–19.4).","key_result":"Among 16 with follow-up, complete resolution occurred in 3/16 (18.8%); reduction to small or medium aneurysms in 6/16 (37.5%); and 5/16 (31.3%) remained giant. 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It compiles data from animal models and human studies and outlines global implementation needs.","key_result":"In pediatric pHLH, HLH-94 (dexamethasone+etoposide) increased 5-year overall survival to 54% (from <10% historically), a recent retrospective series reported 3-year survival of 77% with etoposide-based frontline regimens, and emapalumab plus dexamethasone achieved 12-month survival of 73.4% in relapsed/refractory HLH; adults with malignancy-associated HLH had ~15% 5-year survival.","limitations":"This is a narrative society blueprint drawing largely on retrospective studies, with sparse prospective adult data and few trials in malignancy-associated HLH. Several diagnostic tools and anti-cytokine strategies referenced require prospective validation with standardized endpoints.","practice_takeaway":"For pHLH, HLH-94 (etoposide+dexamethasone) remains standard; if cyclosporine is used, avoid initiation before week 3 and target troughs of 120–150 μg/L. Use HLH-2004 criteria as the pediatric diagnostic benchmark and consider HScore in adults; anti-cytokine agents (emapalumab, ruxolitinib, anakinra) and HCT are options in defined contexts, while adult and malignancy-associated HLH need individualized, trial-guided care."}},{"doi":"10.1002/art.43384","pmid":"40977434","title":"Comparative Efficacy and Safety of Anakinra and Canakinumab in Patients With\n VEXAS\n Syndrome: An International Multicenter Study","journal_title":"Arthritis & Rheumatology","journal_issn":"2326-5191","published_at":"2025-09-22T00:00:00.000Z","url":"https://doi.org/10.1002/art.43384","source_score":37,"is_retracted":false,"is_open_access":true,"raw_metadata":{"ooir":{"day":"2026-01-07","doi":"10.1002/art.43384","days":"30","issn":"2326-5191","rank":"2","field":"Clinical Medicine","score":"37","category":"Rheumatology","categories":["Rheumatology"],"_categories":["Rheumatology"]},"pubmed":{"doi":"10.1002/art.43384","title":"Comparative Efficacy and Safety of Anakinra and Canakinumab in Patients With VEXAS Syndrome: An International Multicenter Study.","journal":"Arthritis & rheumatology (Hoboken, N.J.)","methods":"","results":"We included 47 male patients with VEXAS; 44 received anakinra and 9 received canakinumab, with 6 patients using both at different time points. GR at 1 month was 34% for anakinra and 100% for canakinumab (P < 0.001) and 22% and 78% at 3 months, respectively (P = 0.001). Treatment with canakinumab was associated with a higher odds ratio (OR) of achieving GR at 3 months (OR 28.8, 95% confidence interval 3.0-273.9; P = 0.004) in a multivariable analysis. Median drug survival was 54 (interquartile range [IQR] 30-56) months for canakinumab at 300 mg/month compared with 7 (IQR 4-8) months for canakinumab 150 mg/month and 1 (IQR 1-2.5) months for anakinra (P = 0.01). Injection-site reactions were only recorded for the anakinra group (47 vs 0%; P = 0.006), whereas infections were more frequent in the anakinra group (31% and 11%; P = 0.3).","abstract":"$35","keywords":[],"pubmed_id":"40977434","conclusions":"Canakinumab demonstrated superior clinical response and drug survival with fewer adverse events compared with anakinra. Monthly canakinumab 300 mg may be considered as an effective steroid-sparing therapeutic option for patients with VEXAS.","publication_date":"2025-09-22T00:00:00"},"crossref":{"DOI":"10.1002/art.43384","URL":"https://doi.org/10.1002/art.43384","ISSN":["2326-5191","2326-5205"],"link":[{"URL":"https://acrjournals.onlinelibrary.wiley.com/doi/pdf/10.1002/art.43384","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"page":null,"type":"journal-article","issue":null,"score":1,"title":["Comparative Efficacy and Safety of Anakinra and Canakinumab in Patients With\n VEXAS\n Syndrome: An International Multicenter Study"],"author":[{"given":"Tali","family":"Eviatar","sequence":"first","affiliation":[{"name":"Rheumatology Department Tel Aviv Medical Center Tel Aviv Israel"},{"name":"Faculty of Medical and Health Sciences Tel Aviv University Tel Aviv 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generalizability.","value_numeric":null},{"paper_doi":"10.1002/art.43384","tag_type":"warning","value":"Study type classified as a cohort based on the abstract; randomization was not stated.","value_numeric":null},{"paper_doi":"10.1002/art.43384","tag_type":"warning","value":"The canakinumab sample was small (n=9) and 6 patients crossed over, which may bias estimates.","value_numeric":null}],"summaries":{"what_studied":"International multicenter cohort of 47 men with VEXAS from France, Israel, and Italy treated with IL‑1 inhibitors, comparing anakinra (n=44) versus canakinumab (n=9) for global response at 1 and 3 months, drug survival, and adverse events.","key_result":"Global response favored canakinumab: 1 month 100% vs 34% and 3 months 78% vs 22% with anakinra (P<0.001 and P=0.001); multivariable OR for 3‑month response 28.8 (95% CI 3.0–273.9; P=0.004). Drug survival was longer with canakinumab 300 mg/month (median 54 months, IQR 30–56) vs canakinumab 150 mg/month (7, IQR 4–8) and anakinra (1, IQR 1–2.5) (P=0.01); injection‑site reactions occurred only with anakinra (47% vs 0%; P=0.006), and infections were 31% vs 11% (P=0.3).","limitations":"Nonrandomized multicenter cohort with a small canakinumab group (n=9) and 6 patients who received both drugs at different times; dosing heterogeneity (150 vs 300 mg) may confound comparisons.","practice_takeaway":"For VEXAS, canakinumab showed higher short‑term response and longer drug survival than anakinra, with fewer injection‑site reactions. Monthly canakinumab 300 mg may be an effective steroid‑sparing option, acknowledging the observational design."}},{"doi":"10.1001/jamadermatol.2024.1657","pmid":"38865133","title":"Skin Manifestations of VEXAS Syndrome and Associated Genotypes","journal_title":"JAMA Dermatology","journal_issn":"2168-6068","published_at":"2024-06-12T00:00:00.000Z","url":"https://www.ncbi.nlm.nih.gov/pmc/articles/11170453","source_score":107,"is_retracted":false,"is_open_access":true,"raw_metadata":{"ooir":{"day":"2024-08-24","doi":"10.1001/jamadermatol.2024.1657","days":"30","issn":"2168-6068","rank":"2","field":"Clinical Medicine","score":"107","category":"Dermatology","categories":["Dermatology"],"_categories":["Dermatology"]},"pubmed":{"doi":"10.1001/jamadermatol.2024.1657","title":"Skin Manifestations of VEXAS Syndrome and Associated Genotypes.","journal":"JAMA 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with UBA1-variant–confirmed VEXAS (2019–2023) characterized the spectrum of skin findings and their associations with genotype, histology, and other clinical features; a secondary aim assessed skin response to treatments.","key_result":"Skin involvement occurred in 93/112 (83%) and was the presenting feature in 68/112 (61%); histopathology (64 reports from 60 patients) most often showed leukocytoclastic vasculitis 23/64 (36%), neutrophilic dermatosis 22/64 (34%), and perivascular dermatitis 19/64 (30%). Genotype–phenotype links included p.Met41Leu with neutrophilic infiltrates 14/17 (82%) and p.Met41Val with vasculitic lesions 11/20 (55%) and mixed leukocytic infiltrates 17/20 (85%); prednisone improved skin in 67/73 (92%), while anakinra caused severe injection-site reactions in 12/16 (75%) with ulceration 2/12 (17%) and abscess 1/12 (8%).","limitations":"Data came from mixed sources (retrospective chart review and NIH evaluations), histopathology was available for only 60/112 patients, and treatment response was based on physician impression rather than standardized measures.","practice_takeaway":"In older men with cutaneous vasculitis, neutrophilic dermatoses, or chondritis, consider testing for VEXAS (UBA1). Prednisone often improves skin disease, while anakinra may cause frequent severe injection-site reactions."}}],"initialOffset":9,"fetchConfig":{"kind":"tags","tags":[{"tag_type":"generic","value":"anakinra"}]},"breadcrumbItems":[{"name":"Home","href":"/"},{"name":"Research Hub","href":"/hub"},{"name":"Generics","href":"/hub/tag/generic"},{"name":"anakinra"}],"hrefConfig":{"basePath":"/hub/papers","webViewQueryParams":{"hub_ctx":"tag","hub_tag_type":"generic","hub_tag_value":"anakinra"}}}]]

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