2f:I[703058,["/_next/static/immutable/chunks/3bhq_5eh1d760.js","/_next/static/immutable/chunks/1855-ogyi8eh4.js","/_next/static/immutable/chunks/2pfsjpn6k6zlg.js","/_next/static/immutable/chunks/26c7v3_3uwnwu.js","/_next/static/immutable/chunks/25a3ozcrvi1ff.js","/_next/static/immutable/chunks/2r4wx50nvjh_4.js","/_next/static/immutable/chunks/23fsw06hsi5b1.js","/_next/static/immutable/chunks/06_yt270h41rg.js","/_next/static/immutable/chunks/3ezsiz-7shkna.js","/_next/static/immutable/chunks/20x2y96ldu5wx.js","/_next/static/immutable/chunks/2lxntcm3pd1_h.js","/_next/static/immutable/chunks/00dgco8irq3jw.js","/_next/static/immutable/chunks/00nwe-kq0rboc.js","/_next/static/immutable/chunks/2051ffx3xzio9.js"],"PaperListPageClient"] 30:T596,Of 24 included patients, 9 (38%) were female, 15 (63%) were male, and the median (range) age at diagnosis was 65 (32-85) years. A total of 19 were diagnosed with systemic LE and 5 with cutaneous LE. The median (range) follow-up was 4.5 (1-31) years. Cutaneous involvement was the most common manifestation of LE (17 [71%]). Chilblain lupus was the predominant subtype (6 [35%]). Compared with idiopathic systemic LE, patients with MDS/CMML-associated LE were older (median [range] age, 65 [32-85] years vs 23 [11-55] years; P < .001), more frequently male (10 [53%] vs 3 [8%]; P = .008), had less kidney involvement (2 [10%] vs 27 [71%]; P < .001), had less articular involvement (7 [36%] vs 37 [97%]; P < .001), and had reduced anti-double-stranded DNA positivity (6 [32%] vs 29 [76%]; P = .001). The underlying hematologic diseases included MDS (16 [66%]) and CMML (8 [34%]), with 22 (92%) classified as lower risk (Revised International Prognostic Scoring System score of 3.5 or less). Centralized histopathological review reclassified 6 skin biopsies (50%) as MDS/CMML cutis. Identical myeloid variants were detected in blood and skin in 6 of 8 patients, supporting a clonal inflammatory process. Standard LE therapies were often poorly effective, while clone-directed therapies (azacitidine or allogeneic hematopoietic stem cell transplant) led to parallel hematologic and LE responses in 5 of 7 patients.31:Tb2e,Immune-mediated inflammatory diseases are rare but increasingly reported among patients with myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML). Systemic lupus erythematosus (LE) and cutaneous LE associated with MDS/CMML have been rarely described, with atypical features and refractory disease. To provide a comprehensive description of the phenotype and therapeutic responses of LE associated with MDS/CMML and to compare them with idiopathic LE. This retrospective case-control study included nationwide, multicenter data from January 1975 to January 2023. Patients with MDS/CMML who either fulfilled classification criteria for systemic LE or had skin lesions diagnosed as cutaneous LE were included. For MDS/CMML systemic LE, a 2:1 case-control study was conducted with idiopathic systemic LE. Clinical features, centralized skin histopathology, and targeted next-generation sequencing were analyzed. Data were analyzed from May 2022 to June 2025. The clinical, pathological, and molecular features of LE occurring in the setting of MDS or CMML compared with idiopathic LE. Of 24 included patients, 9 (38%) were female, 15 (63%) were male, and the median (range) age at diagnosis was 65 (32-85) years. A total of 19 were diagnosed with systemic LE and 5 with cutaneous LE. The median (range) follow-up was 4.5 (1-31) years. Cutaneous involvement was the most common manifestation of LE (17 [71%]). Chilblain lupus was the predominant subtype (6 [35%]). Compared with idiopathic systemic LE, patients with MDS/CMML-associated LE were older (median [range] age, 65 [32-85] years vs 23 [11-55] years; P < .001), more frequently male (10 [53%] vs 3 [8%]; P = .008), had less kidney involvement (2 [10%] vs 27 [71%]; P < .001), had less articular involvement (7 [36%] vs 37 [97%]; P < .001), and had reduced anti-double-stranded DNA positivity (6 [32%] vs 29 [76%]; P = .001). The underlying hematologic diseases included MDS (16 [66%]) and CMML (8 [34%]), with 22 (92%) classified as lower risk (Revised International Prognostic Scoring System score of 3.5 or less). Centralized histopathological review reclassified 6 skin biopsies (50%) as MDS/CMML cutis. Identical myeloid variants were detected in blood and skin in 6 of 8 patients, supporting a clonal inflammatory process. Standard LE therapies were often poorly effective, while clone-directed therapies (azacitidine or allogeneic hematopoietic stem cell transplant) led to parallel hematologic and LE responses in 5 of 7 patients. In this study, MDS/CMML-associated lupuslike manifestations were a distinct entity mimicking systemic LE or cutaneous LE and characterized by clonal inflammation rather than classic autoimmunity in most cases. Early recognition is important, as treatment may require clone-targeting therapies rather than conventional LE therapy.32:T1152, Importance Immune-mediated inflammatory diseases are rare but increasingly reported among patients with myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML). Systemic lupus erythematosus (LE) and cutaneous LE associated with MDS/CMML have been rarely described, with atypical features and refractory disease. Objective To provide a comprehensive description of the phenotype and therapeutic responses of LE associated with MDS/CMML and to compare them with idiopathic LE. Design, Setting, and Participants This retrospective case-control study included nationwide, multicenter data from January 1975 to January 2023. Patients with MDS/CMML who either fulfilled classification criteria for systemic LE or had skin lesions diagnosed as cutaneous LE were included. For MDS/CMML systemic LE, a 2:1 case-control study was conducted with idiopathic systemic LE. Clinical features, centralized skin histopathology, and targeted next-generation sequencing were analyzed. Data were analyzed from May 2022 to June 2025. Main Outcomes and Measures The clinical, pathological, and molecular features of LE occurring in the setting of MDS or CMML compared with idiopathic LE. Results Of 24 included patients, 9 (38%) were female, 15 (63%) were male, and the median (range) age at diagnosis was 65 (32-85) years. A total of 19 were diagnosed with systemic LE and 5 with cutaneous LE. The median (range) follow-up was 4.5 (1-31) years. Cutaneous involvement was the most common manifestation of LE (17 [71%]). Chilblain lupus was the predominant subtype (6 [35%]). Compared with idiopathic systemic LE, patients with MDS/CMML–associated LE were older (median [range] age, 65 [32-85] years vs 23 [11-55] years; P &lt; .001), more frequently male (10 [53%] vs 3 [8%]; P = .008), had less kidney involvement (2 [10%] vs 27 [71%]; P &lt; .001), had less articular involvement (7 [36%] vs 37 [97%]; P &lt; .001), and had reduced anti–double-stranded DNA positivity (6 [32%] vs 29 [76%]; P = .001). The underlying hematologic diseases included MDS (16 [66%]) and CMML (8 [34%]), with 22 (92%) classified as lower risk (Revised International Prognostic Scoring System score of 3.5 or less). Centralized histopathological review reclassified 6 skin biopsies (50%) as MDS/CMML cutis. Identical myeloid variants were detected in blood and skin in 6 of 8 patients, supporting a clonal inflammatory process. Standard LE therapies were often poorly effective, while clone-directed therapies (azacitidine or allogeneic hematopoietic stem cell transplant) led to parallel hematologic and LE responses in 5 of 7 patients. Conclusions and Relevance In this study, MDS/CMML–associated lupuslike manifestations were a distinct entity mimicking systemic LE or cutaneous LE and characterized by clonal inflammation rather than classic autoimmunity in most cases. Early recognition is important, as treatment may require clone-targeting therapies rather than conventional LE therapy. 25:[[["$","script","0",{"type":"application/ld+json","dangerouslySetInnerHTML":{"__html":"{\"@context\":\"https://schema.org\",\"@type\":\"CollectionPage\",\"name\":\"Myelodysplastic Syndrome: condition Research & Clinical Takeaways\",\"description\":\"Explore peer-reviewed papers on Myelodysplastic Syndrome (condition). Clinician-written summaries and actionable practice insights. Updated daily.\",\"url\":\"https://www.reachrx.ai/hub/tag/condition/Myelodysplastic%20Syndrome\",\"breadcrumb\":{\"@type\":\"BreadcrumbList\",\"itemListElement\":[{\"@type\":\"ListItem\",\"position\":1,\"name\":\"Home\",\"item\":\"https://www.reachrx.ai\"},{\"@type\":\"ListItem\",\"position\":2,\"name\":\"Research Hub\",\"item\":\"https://www.reachrx.ai/hub\"},{\"@type\":\"ListItem\",\"position\":3,\"name\":\"Conditions\",\"item\":\"https://www.reachrx.ai/hub/tag/condition\"},{\"@type\":\"ListItem\",\"position\":4,\"name\":\"Myelodysplastic Syndrome\",\"item\":\"https://www.reachrx.ai/hub/tag/condition/Myelodysplastic%20Syndrome\"}]}}"}}]],["$","$L2f",null,{"title":"Myelodysplastic Syndrome: Research & Clinical Takeaways","description":"Discover peer-reviewed research on Myelodysplastic Syndrome with concise clinician summaries and next-step guidance.","papers":[{"doi":"10.1016/j.chest.2026.02.012","pmid":"42575657","title":"Reversible Intrapulmonary Vascular Dilatation in a Patient Treated With Luspatercept for Myelodysplastic Syndrome","journal_title":"CHEST","journal_issn":"0012-3692","published_at":"2026-08-10T00:00:00.000Z","url":"https://doi.org/10.1016/j.chest.2026.02.012","source_score":3,"is_retracted":false,"is_open_access":true,"raw_metadata":{"pubmed":true,"crossref":true,"unpaywall":true},"created_at":"2026-08-17T13:33:10.108Z","updated_at":"2026-08-17T13:33:10.108Z","tags":[{"paper_doi":"10.1016/j.chest.2026.02.012","tag_type":"age_group","value":"older_adult","value_numeric":null},{"paper_doi":"10.1016/j.chest.2026.02.012","tag_type":"brand","value":"Reblozyl","value_numeric":null},{"paper_doi":"10.1016/j.chest.2026.02.012","tag_type":"category","value":"Respiratory System","value_numeric":null},{"paper_doi":"10.1016/j.chest.2026.02.012","tag_type":"condition","value":"Hypoxemia","value_numeric":null},{"paper_doi":"10.1016/j.chest.2026.02.012","tag_type":"condition","value":"Intrapulmonary Vascular Dilatation","value_numeric":null},{"paper_doi":"10.1016/j.chest.2026.02.012","tag_type":"condition","value":"Myelodysplastic Syndrome","value_numeric":null},{"paper_doi":"10.1016/j.chest.2026.02.012","tag_type":"generic","value":"luspatercept","value_numeric":null},{"paper_doi":"10.1016/j.chest.2026.02.012","tag_type":"impact_flag","value":"safety_signal","value_numeric":null},{"paper_doi":"10.1016/j.chest.2026.02.012","tag_type":"level_of_evidence","value":"5","value_numeric":null},{"paper_doi":"10.1016/j.chest.2026.02.012","tag_type":"modality","value":"Erythroid Maturation Agent","value_numeric":null},{"paper_doi":"10.1016/j.chest.2026.02.012","tag_type":"sample_size","value":"n=1","value_numeric":1},{"paper_doi":"10.1016/j.chest.2026.02.012","tag_type":"sex","value":"male","value_numeric":null},{"paper_doi":"10.1016/j.chest.2026.02.012","tag_type":"study_design","value":"Case Report","value_numeric":null},{"paper_doi":"10.1016/j.chest.2026.02.012","tag_type":"warning","value":"Causality between luspatercept and IPVD is inferred by temporal association and reversibility after cessation — no rechallenge was performed. The underlying probable usual interstitial pneumonia is a confounding comorbidity. This is a single case report; findings cannot be generalized.","value_numeric":null}],"summaries":{"what_studied":"A 70-year-old man with transfusion-dependent myelodysplastic syndrome (MDS) developed new hypoxemia 9 months into luspatercept treatment (2 months after a dose increase), prompting investigation into the cause and reversibility of his intrapulmonary vascular dilatation (IPVD).","key_result":"Hypoxemia (resting SpO₂ 88%, requiring up to 90% FiO₂) was attributed to IPVD with a 7.5% shunt on MAA scintigraphy. After luspatercept was stopped, supplemental oxygen was discontinued by 7 weeks and repeat MAA scintigraphy at 10 weeks confirmed shunt resolution (3.6%).","limitations":"Single case report limits causal certainty; underlying mild probable usual interstitial pneumonia may have contributed to hypoxemia; no rechallenge with luspatercept was performed to confirm causality.","practice_takeaway":"Clinicians prescribing luspatercept should monitor for new or unexplained hypoxemia, as IPVD may be a reversible drug-related complication. If suspected, cessation of luspatercept appears to lead to resolution within weeks."}},{"doi":"10.1038/s41375-026-03061-7","pmid":"42562919","title":"SLF2 and SMC5 dysfunction drives HSC aging and predisposes to MDS, defining a new inherited bone marrow failure syndrome","journal_title":"Leukemia","journal_issn":"0887-6924","published_at":"2026-08-07T00:00:00.000Z","url":"https://doi.org/10.1038/s41375-026-03061-7","source_score":41,"is_retracted":false,"is_open_access":false,"raw_metadata":{"pubmed":true,"crossref":true,"unpaywall":true},"created_at":"2026-08-13T13:22:58.593Z","updated_at":"2026-08-13T13:22:58.593Z","tags":[{"paper_doi":"10.1038/s41375-026-03061-7","tag_type":"age_group","value":"pediatric","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03061-7","tag_type":"category","value":"Hematology","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03061-7","tag_type":"category","value":"Oncology","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03061-7","tag_type":"condition","value":"Atelis Syndrome","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03061-7","tag_type":"condition","value":"Inherited Bone Marrow Failure Syndrome","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03061-7","tag_type":"condition","value":"Myelodysplastic Syndrome","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03061-7","tag_type":"generic","value":"SLF2 (SLF2 gene)","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03061-7","tag_type":"generic","value":"SMC5 (SMC5 gene)","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03061-7","tag_type":"impact_flag","value":"practice_changing","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03061-7","tag_type":"level_of_evidence","value":"4","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03061-7","tag_type":"modality","value":"Gene Function / Genomic Integrity","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03061-7","tag_type":"sex","value":"mixed","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03061-7","tag_type":"study_design","value":"Translational/Basic Science Study with Patient-Derived iPSC Model","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03061-7","tag_type":"warning","value":"Study design is best classified as a translational/mechanistic study with in vitro and xenograft components rather than a standard clinical study design; level of evidence is approximate.","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03061-7","tag_type":"warning","value":"The impact_flag 'practice_changing' is applied because this study reclassifies Atelis Syndrome as a new IBMFS with MDS risk, which has direct clinical surveillance implications, though the evidence base remains early-stage.","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03061-7","tag_type":"warning","value":"The study is primarily mechanistic, using iPSC lines from a single patient; the clinical cohort size is very small (2 patients with lab data shown), limiting generalizability. No formal sample size is reported for the human clinical component.","value_numeric":null}],"summaries":{"what_studied":"This study investigated the hematopoietic consequences of pathogenic SLF2 and SMC5 variants (Atelis Syndrome) using iPSC-derived hematopoietic progenitor cells (HPCs) from a patient with compound heterozygous SLF2 mutations, alongside SMC5 knockdown in cord blood CD34+ cells, to define a new inherited bone marrow failure syndrome (IBMFS).","key_result":"Mutant HPCs showed impaired colony-forming capacity, defective erythroid differentiation with myeloid bias, and markedly reduced xenotransplantation engraftment. Mechanistically, SLF2-SMC5 disruption induced genomic instability, p53/p21 activation (CDKN1A log2FC +1.98, MDM2 log2FC +0.96), and a senescence-like phenotype. ATAC-seq revealed increased chromatin accessibility at PU.1 motifs (ΔZ-score +7.06, p=4.02×10⁻⁴) consistent with HSC aging and myeloid bias. Longitudinal follow-up confirmed MDS development in some patients at a young age.","limitations":"- iPSC model derived from a single patient (one male individual with 46,XY karyotype); findings may not generalize across all Atelis Syndrome genotypes.\n- Xenotransplantation and colony assays used iPSC-derived HPCs, which may not fully recapitulate adult bone marrow biology.\n- No randomization or blinding was performed in mouse experiments.","practice_takeaway":"Clinicians managing Atelis Syndrome (SLF2/SMC5 variants) should be aware that it constitutes a bona fide IBMFS with risk of early-onset MDS, warranting longitudinal hematologic surveillance. Recognition of this syndrome expands the differential for inherited bone marrow failure with neurodevelopmental features."}},{"doi":"10.1080/09273948.2026.2689365","pmid":"42417824","title":"Severe Paraneoplastic Anterior and Posterior Scleritis Associated with Myelodysplastic Syndrome","journal_title":"Ocular Immunology and Inflammation","journal_issn":"0927-3948","published_at":"2026-07-08T00:00:00.000Z","url":"https://doi.org/10.1080/09273948.2026.2689365","source_score":2,"is_retracted":false,"is_open_access":false,"raw_metadata":{"pubmed":true,"crossref":true,"unpaywall":true},"created_at":"2026-07-12T13:19:42.316Z","updated_at":"2026-07-12T13:19:42.316Z","tags":[{"paper_doi":"10.1080/09273948.2026.2689365","tag_type":"age_group","value":"older_adult","value_numeric":null},{"paper_doi":"10.1080/09273948.2026.2689365","tag_type":"brand","value":"Rituxan","value_numeric":null},{"paper_doi":"10.1080/09273948.2026.2689365","tag_type":"category","value":"Ophthalmology","value_numeric":null},{"paper_doi":"10.1080/09273948.2026.2689365","tag_type":"condition","value":"Myelodysplastic Syndrome","value_numeric":null},{"paper_doi":"10.1080/09273948.2026.2689365","tag_type":"condition","value":"Paraneoplastic Syndrome","value_numeric":null},{"paper_doi":"10.1080/09273948.2026.2689365","tag_type":"condition","value":"Scleritis","value_numeric":null},{"paper_doi":"10.1080/09273948.2026.2689365","tag_type":"generic","value":"prednisone","value_numeric":null},{"paper_doi":"10.1080/09273948.2026.2689365","tag_type":"generic","value":"rituximab","value_numeric":null},{"paper_doi":"10.1080/09273948.2026.2689365","tag_type":"impact_flag","value":"limited_evidence","value_numeric":null},{"paper_doi":"10.1080/09273948.2026.2689365","tag_type":"level_of_evidence","value":"5","value_numeric":null},{"paper_doi":"10.1080/09273948.2026.2689365","tag_type":"modality","value":"Anti-CD20 Monoclonal Antibody","value_numeric":null},{"paper_doi":"10.1080/09273948.2026.2689365","tag_type":"modality","value":"Corticosteroid","value_numeric":null},{"paper_doi":"10.1080/09273948.2026.2689365","tag_type":"sample_size","value":"n=1","value_numeric":1},{"paper_doi":"10.1080/09273948.2026.2689365","tag_type":"sex","value":"male","value_numeric":null},{"paper_doi":"10.1080/09273948.2026.2689365","tag_type":"study_design","value":"Case Report","value_numeric":null},{"paper_doi":"10.1080/09273948.2026.2689365","tag_type":"warning","value":"Brand name 'Rituxan' assigned for rituximab; confirm local formulary name as biosimilars may apply.","value_numeric":null},{"paper_doi":"10.1080/09273948.2026.2689365","tag_type":"warning","value":"The paraneoplastic nature of the scleritis is presumed based on temporal association with MDS — no mechanistic proof is provided.","value_numeric":null},{"paper_doi":"10.1080/09273948.2026.2689365","tag_type":"warning","value":"This is a single case report; conclusions about treatment efficacy and malignancy association are hypothesis-generating only.","value_numeric":null}],"summaries":{"what_studied":"A case report and literature review describing a 59-year-old male with severe unilateral anterior and posterior scleritis (with ciliochoroidal detachment and secondary angle closure) that led to the diagnosis of high-risk myelodysplastic syndrome (MDS), evaluating presentation, treatment response, and outcomes.","key_result":"Prednisone alone could not be tapered below 30 mg without recurrence; rituximab infusions achieved ocular symptom resolution and successful steroid taper, but the patient died while on chemotherapy before allogenic bone marrow transplantation.","limitations":"Single case report limits generalizability. No control or comparison group. Causal link between MDS and scleritis is presumed (paraneoplastic) but not mechanistically confirmed.","practice_takeaway":"Consider malignancy workup — including full blood counts and hematology referral — in patients with unexplained cytopenias, severe scleritis at presentation, or steroid-refractory scleritis. If an underlying cancer is found, definitive scleritis treatment requires treating the malignancy itself."}},{"doi":"10.1038/s41375-026-03015-z","pmid":"42386909","title":"Single-cell DNA methylation analysis uncovers epigenetic pathways in the transformation of MDS to AML","journal_title":"Leukemia","journal_issn":"0887-6924","published_at":"2026-07-01T00:00:00.000Z","url":"https://doi.org/10.1038/s41375-026-03015-z","source_score":11,"is_retracted":false,"is_open_access":true,"raw_metadata":{"pubmed":true,"crossref":true,"unpaywall":true},"created_at":"2026-07-09T13:35:57.636Z","updated_at":"2026-07-09T13:35:57.636Z","tags":[{"paper_doi":"10.1038/s41375-026-03015-z","tag_type":"age_group","value":"adult","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03015-z","tag_type":"category","value":"Hematology","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03015-z","tag_type":"condition","value":"Acute Myeloid Leukemia","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03015-z","tag_type":"condition","value":"Myelodysplastic Syndrome","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03015-z","tag_type":"modality","value":"Epigenetic Analysis","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03015-z","tag_type":"study_design","value":"Translational/Epigenomic Study","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03015-z","tag_type":"warning","value":"Only the paper title, DOI, journal, and publication date were provided — no abstract, methods, results, or sample data were included. All structured fields reflect metadata only; key result, sample size, sex, level of evidence, and practice takeaway cannot be determined.","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03015-z","tag_type":"warning","value":"Publication date is July 2026, which is very recent — full text may not yet be widely available for independent verification.","value_numeric":null},{"paper_doi":"10.1038/s41375-026-03015-z","tag_type":"warning","value":"Study design label is inferred from the title only; actual design (e.g., cohort, case-control, in vitro) is unknown.","value_numeric":null}],"summaries":{"what_studied":"This study used single-cell DNA methylation analysis to investigate epigenetic changes during the transformation from myelodysplastic syndrome (MDS) to acute myeloid leukemia (AML), aiming to uncover cell-level methylation pathways driving disease progression.","limitations":"The paper metadata provided does not include results, methods detail, or sample size, making it impossible to assess specific findings, statistical power, or generalizability."}},{"doi":"10.1001/jamaoncol.2026.1924","pmid":"42348196","title":"Long-Term Outcomes in Patients With Recurrent Ovarian Cancer and Exceptional Response to PARP Inhibitors","journal_title":"JAMA Oncology","journal_issn":"2374-2437","published_at":"2026-06-25T00:00:00.000Z","url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC13306488/","source_score":17,"is_retracted":false,"is_open_access":true,"raw_metadata":{"pubmed":true,"crossref":true,"unpaywall":true},"created_at":"2026-07-04T13:26:19.768Z","updated_at":"2026-07-04T13:26:19.768Z","tags":[{"paper_doi":"10.1001/jamaoncol.2026.1924","tag_type":"age_group","value":"adult","value_numeric":null},{"paper_doi":"10.1001/jamaoncol.2026.1924","tag_type":"category","value":"Oncology","value_numeric":null},{"paper_doi":"10.1001/jamaoncol.2026.1924","tag_type":"condition","value":"Acute Myeloid Leukemia","value_numeric":null},{"paper_doi":"10.1001/jamaoncol.2026.1924","tag_type":"condition","value":"Myelodysplastic Syndrome","value_numeric":null},{"paper_doi":"10.1001/jamaoncol.2026.1924","tag_type":"condition","value":"Ovarian Cancer","value_numeric":null},{"paper_doi":"10.1001/jamaoncol.2026.1924","tag_type":"impact_flag","value":"practice_changing","value_numeric":null},{"paper_doi":"10.1001/jamaoncol.2026.1924","tag_type":"impact_flag","value":"safety_signal","value_numeric":null},{"paper_doi":"10.1001/jamaoncol.2026.1924","tag_type":"level_of_evidence","value":"3","value_numeric":null},{"paper_doi":"10.1001/jamaoncol.2026.1924","tag_type":"modality","value":"PARP Inhibitor","value_numeric":null},{"paper_doi":"10.1001/jamaoncol.2026.1924","tag_type":"sample_size","value":"n=320","value_numeric":320},{"paper_doi":"10.1001/jamaoncol.2026.1924","tag_type":"sex","value":"female","value_numeric":null},{"paper_doi":"10.1001/jamaoncol.2026.1924","tag_type":"study_design","value":"Retrospective Cohort Study","value_numeric":null},{"paper_doi":"10.1001/jamaoncol.2026.1924","tag_type":"warning","value":"Brand/generic tags for specific PARP inhibitors (e.g., olaparib, niraparib, rucaparib) were not reported in the available text and could not be tagged.","value_numeric":null},{"paper_doi":"10.1001/jamaoncol.2026.1924","tag_type":"warning","value":"Full text was inaccessible; all data extracted from the abstract and results section only — OS data, dose-reduction rates, and full subgroup analyses could not be independently verified.","value_numeric":null},{"paper_doi":"10.1001/jamaoncol.2026.1924","tag_type":"warning","value":"The '2 (7.5%)' figure for disease progression beyond 5 years appears inconsistent with the surrounding percentages (likely a typo for '2.5%' or similar); clinicians should verify against the published manuscript.","value_numeric":null},{"paper_doi":"10.1001/jamaoncol.2026.1924","tag_type":"warning","value":"The discontinuation comparison (stopped vs. continued PARPi) is not randomized; patients who stopped may have been selected for better prognosis, which could confound the PFS difference.","value_numeric":null}],"summaries":{"what_studied":"This international, multicenter, retrospective cohort study (41 sites, 14 countries) evaluated long-term outcomes — including PFS, overall survival, toxic effects, and MDS/AML risk — in 320 patients with platinum-sensitive recurrent ovarian cancer (PS-ROC) who had an exceptional response (PFS ≥5 years) to maintenance PARP inhibitors, with a median follow-up of 6.8 years.","key_result":"At 10 years, PFS was 78.7% overall; patients who stopped PARP inhibitors without progression (n=85) had a 10-year PFS of 90.1% vs 72.5% for those who continued. Late-onset MDS/AML occurred in only 5 patients (1.6%). Exceptional responders were enriched for BRCA1 RING domain and BRCA2 DNA-binding domain variants.","limitations":"- Retrospective design introduces selection bias; patients who discontinued may differ systematically from those who continued.\n- Full text was inaccessible, so granular subgroup data, OS figures, and dose-reduction details could not be verified beyond the abstract.\n- Genotype-phenotype associations are exploratory and require prospective validation.","practice_takeaway":"For ovarian cancer patients with ≥5 years of disease control on a PARP inhibitor, discontinuation does not appear to compromise long-term outcomes and may be a reasonable option to discuss — particularly given the low but real MDS/AML risk (1.6%) with prolonged use. BRCA variant location (RING or DNA-binding domains) may help identify patients most likely to achieve functional cure."}},{"doi":"10.1002/hem3.70406","pmid":"42339339","title":"Real‐world, multi‐omics validation of the clinical relevance of molecular taxonomy for myelodysplastic syndromes (MDS)","journal_title":"HemaSphere","journal_issn":"2572-9241","published_at":"2026-06-22T00:00:00.000Z","url":"https://doi.org/10.1002/hem3.70406","source_score":9,"is_retracted":false,"is_open_access":true,"raw_metadata":{"pubmed":true,"crossref":true,"unpaywall":true},"created_at":"2026-06-28T13:27:01.038Z","updated_at":"2026-06-28T13:27:01.038Z","tags":[{"paper_doi":"10.1002/hem3.70406","tag_type":"age_group","value":"adult","value_numeric":null},{"paper_doi":"10.1002/hem3.70406","tag_type":"age_group","value":"older_adult","value_numeric":null},{"paper_doi":"10.1002/hem3.70406","tag_type":"category","value":"Hematology","value_numeric":null},{"paper_doi":"10.1002/hem3.70406","tag_type":"condition","value":"Myelodysplastic Syndrome","value_numeric":null},{"paper_doi":"10.1002/hem3.70406","tag_type":"generic","value":"azacitidine","value_numeric":null},{"paper_doi":"10.1002/hem3.70406","tag_type":"impact_flag","value":"confirms_prior","value_numeric":null},{"paper_doi":"10.1002/hem3.70406","tag_type":"level_of_evidence","value":"3","value_numeric":null},{"paper_doi":"10.1002/hem3.70406","tag_type":"modality","value":"Allogeneic Hematopoietic Stem Cell Transplantation","value_numeric":null},{"paper_doi":"10.1002/hem3.70406","tag_type":"modality","value":"Hypomethylating Agent","value_numeric":null},{"paper_doi":"10.1002/hem3.70406","tag_type":"sample_size","value":"n=5583","value_numeric":5583},{"paper_doi":"10.1002/hem3.70406","tag_type":"sex","value":"mixed","value_numeric":null},{"paper_doi":"10.1002/hem3.70406","tag_type":"study_design","value":"Retrospective Cohort Study with Prospective Transcriptomic Validation","value_numeric":null},{"paper_doi":"10.1002/hem3.70406","tag_type":"warning","value":"cnLOH was not directly measured; biallelic TP53/TET2 inactivation was inferred, which may introduce misclassification within taxonomy subgroups.","value_numeric":null},{"paper_doi":"10.1002/hem3.70406","tag_type":"warning","value":"No specific HMA drug breakdown (azacitidine vs. decitabine) was reported; 'HMA' is used as a class throughout.","value_numeric":null},{"paper_doi":"10.1002/hem3.70406","tag_type":"warning","value":"The retrospective cohort spans diagnoses from 1990 to 2021, meaning treatment practices and genomic testing methods changed substantially over this period, potentially confounding treatment-outcome analyses.","value_numeric":null},{"paper_doi":"10.1002/hem3.70406","tag_type":"warning","value":"The two cohorts (retrospective and prospective) differ substantially in disease stage distribution — the prospective cohort has a much higher proportion of AML-evolving cases — limiting direct comparisons between them.","value_numeric":null}],"summaries":{"what_studied":"This international multi-cohort study validated the IWG-PM MDS molecular taxonomy (16 subgroups based on 21 genes, 6 cytogenetic events, and TP53/TET2 LOH) in a retrospective cohort of 5,136 MDS patients and a prospective transcriptomic cohort of 447 patients, evaluating its prognostic value, predictive value for HMA response and HSCT outcomes, and biological distinctiveness via RNA-seq of CD34+ bone marrow progenitors.","key_result":"The MDS taxonomy identified subgroups with distinct OS (median 1.3–10.9 years, P<0.001) and AML transformation risk (0–19.1%, P<0.001), but did not outperform IPSS-M (C-index for OS: taxonomy 0.70 vs. IPSS-M 0.78). Taxonomy did not predict HMA response (ORR 30.7%, no significant variation across groups, P=0.85) or HSCT outcomes in multivariable analysis, except the TP53-complex subgroup, which had significantly higher post-HSCT relapse (36.1%, HR 2.39, P=0.001) and worse post-HMA OS (median 5.7 months, HR 1.78, P<0.001). RNA-seq confirmed distinct transcriptomic signatures for AML-like (recall 94%), TP53-complex (88%), SF3B1 (75%), and biTET2 (54%) subgroups.","limitations":"- cnLOH data were unavailable; biallelic TP53/TET2 inactivation was inferred indirectly from VAF and karyotype, which may misclassify some patients.\n- Retrospective treatment data introduce selection bias; therapeutic decision-making varied across centers and time (diagnoses 1990–2021).\n- Several taxonomy subgroups (e.g., DDX41, EZH2-ASXL1, ZRSR2) were underrepresented in the transcriptomic cohort, limiting RNA-seq conclusions for those groups.","practice_takeaway":"IPSS-M remains the stronger prognostic tool for MDS; the molecular taxonomy adds biological context but should not replace IPSS-M for individual risk stratification or HMA treatment decisions. Clinicians should be aware that TP53-complex MDS carries exceptionally high post-HSCT relapse risk (36.1%) and very short post-HMA survival (median 5.7 months), warranting early consideration of novel or investigational approaches for this subgroup."}},{"doi":"10.1002/ajh.70391","pmid":"42240074","title":"Therapeutic Silencing of Tmprss6 Reduces Iron‐Induced Inflammation and Prolongs Survival in\n MDS\n Mice","journal_title":"American Journal of Hematology","journal_issn":"0361-8609","published_at":"2026-06-04T00:00:00.000Z","url":"https://doi.org/10.1002/ajh.70391","source_score":17,"is_retracted":false,"is_open_access":true,"raw_metadata":{"pubmed":true,"crossref":true,"unpaywall":true},"created_at":"2026-06-19T13:23:13.721Z","updated_at":"2026-06-19T13:23:13.721Z","tags":[{"paper_doi":"10.1002/ajh.70391","tag_type":"age_group","value":"adult","value_numeric":null},{"paper_doi":"10.1002/ajh.70391","tag_type":"category","value":"Hematology","value_numeric":null},{"paper_doi":"10.1002/ajh.70391","tag_type":"condition","value":"Iron Overload","value_numeric":null},{"paper_doi":"10.1002/ajh.70391","tag_type":"condition","value":"Myelodysplastic Syndrome","value_numeric":null},{"paper_doi":"10.1002/ajh.70391","tag_type":"generic","value":"deferiprone","value_numeric":null},{"paper_doi":"10.1002/ajh.70391","tag_type":"generic","value":"SLN124","value_numeric":null},{"paper_doi":"10.1002/ajh.70391","tag_type":"impact_flag","value":"practice_changing","value_numeric":null},{"paper_doi":"10.1002/ajh.70391","tag_type":"level_of_evidence","value":"6","value_numeric":null},{"paper_doi":"10.1002/ajh.70391","tag_type":"modality","value":"Iron Chelation","value_numeric":null},{"paper_doi":"10.1002/ajh.70391","tag_type":"modality","value":"RNA Interference (siRNA)","value_numeric":null},{"paper_doi":"10.1002/ajh.70391","tag_type":"study_design","value":"Preclinical Animal Study","value_numeric":null},{"paper_doi":"10.1002/ajh.70391","tag_type":"warning","value":"Sample sizes for the mouse cohorts are not reported in the abstract; statistical robustness cannot be fully assessed from available information.","value_numeric":null},{"paper_doi":"10.1002/ajh.70391","tag_type":"warning","value":"SLN124 is identified as a GalNAc-conjugated siRNA targeting Tmprss6 but does not yet have a widely recognized nonproprietary (INN) drug name; tagged under its study designation.","value_numeric":null},{"paper_doi":"10.1002/ajh.70391","tag_type":"warning","value":"Study is entirely preclinical; level of evidence is set to 6 (animal/laboratory study).","value_numeric":null},{"paper_doi":"10.1002/ajh.70391","tag_type":"warning","value":"The 'practice_changing' impact flag reflects the novelty and strength of the preclinical survival data, but no human efficacy data exist yet — clinicians should interpret with caution.","value_numeric":null}],"summaries":{"what_studied":"This study evaluated hepatocyte-targeted silencing of *Tmprss6* using a GalNAc-conjugated siRNA (SLN124, 3 mg/kg monthly subcutaneous) versus oral deferiprone (1.25 mg/mL) in the NUP98-HOXD13 (NHD13) mouse model of MDS, focusing on iron overload, inflammasome activation, disease progression, and survival.","key_result":"SLN124 produced the most pronounced reduction in tissue iron deposition and ASC-speck (inflammasome) abundance. Long-term SLN124 treatment significantly prolonged survival: 30% of treated mice survived beyond 450 days, versus complete mortality by Day 420 in both controls and deferiprone-treated mice.","limitations":"Results are from a single preclinical mouse model (NHD13); translation to human MDS is unconfirmed. No human or clinical trial data are presented. Deferiprone dose/route may not reflect optimal human-equivalent chelation therapy.","practice_takeaway":"TMPRSS6 silencing via SLN124 is not yet in clinical use, but these preclinical data suggest it may offer disease-modifying benefit beyond conventional iron chelation in MDS. Clinicians managing iron overload in MDS should watch for clinical trials of SLN124 or similar hepcidin-upregulating agents."}},{"doi":"10.1182/blood.2025030554","pmid":"42148659","title":"Stag2 dependent chromatin remodeling enforces the erythroid-specific Gata1 cistrome","journal_title":"Blood Journal","journal_issn":"0006-4971","published_at":"2026-05-18T00:00:00.000Z","url":"https://doi.org/10.1182/blood.2025030554","source_score":13,"is_retracted":false,"is_open_access":null,"raw_metadata":{"pubmed":true,"crossref":true,"unpaywall":false},"created_at":"2026-05-26T13:35:15.077Z","updated_at":"2026-05-26T13:35:15.077Z","tags":[{"paper_doi":"10.1182/blood.2025030554","tag_type":"age_group","value":"mixed","value_numeric":null},{"paper_doi":"10.1182/blood.2025030554","tag_type":"category","value":"Hematology","value_numeric":null},{"paper_doi":"10.1182/blood.2025030554","tag_type":"condition","value":"Dyserythropoiesis","value_numeric":null},{"paper_doi":"10.1182/blood.2025030554","tag_type":"condition","value":"Myelodysplastic Syndrome","value_numeric":null},{"paper_doi":"10.1182/blood.2025030554","tag_type":"condition","value":"Myeloid Leukemia of Down Syndrome","value_numeric":null},{"paper_doi":"10.1182/blood.2025030554","tag_type":"generic","value":"STAG2","value_numeric":null},{"paper_doi":"10.1182/blood.2025030554","tag_type":"impact_flag","value":"practice_changing","value_numeric":null},{"paper_doi":"10.1182/blood.2025030554","tag_type":"level_of_evidence","value":"3","value_numeric":null},{"paper_doi":"10.1182/blood.2025030554","tag_type":"modality","value":"Chromatin Remodeling / Cohesin Complex","value_numeric":null},{"paper_doi":"10.1182/blood.2025030554","tag_type":"sex","value":"mixed","value_numeric":null},{"paper_doi":"10.1182/blood.2025030554","tag_type":"study_design","value":"Mechanistic Animal and Translational Study","value_numeric":null},{"paper_doi":"10.1182/blood.2025030554","tag_type":"warning","value":"No specific sample size (N) is reported in the abstract for either murine cohorts or human MDS patient samples; sample_size tag was omitted.","value_numeric":null},{"paper_doi":"10.1182/blood.2025030554","tag_type":"warning","value":"'Published: 2026-05-18' is slightly in the future relative to today's date (May 26, 2026); this appears to be the formal publication date as listed.","value_numeric":null},{"paper_doi":"10.1182/blood.2025030554","tag_type":"warning","value":"Study design is a hybrid basic science/translational investigation — no standard clinical trial design label fully applies; 'Mechanistic Animal and Translational Study' is used as the closest descriptor.","value_numeric":null},{"paper_doi":"10.1182/blood.2025030554","tag_type":"warning","value":"The 'practice_changing' impact flag is applied cautiously: while the mechanistic finding is novel and clinically relevant, direct therapeutic applications have not yet been tested in clinical trials.","value_numeric":null}],"summaries":{"what_studied":"This study examined how loss of *Stag2* (a cohesin complex member) alters chromatin accessibility and GATA1 binding specificity in erythroid progenitors (EryPs), using *Stag2*-deficient mice, human cell models, and primary MDS patient samples to understand dyserythropoiesis in MDS and myeloid leukemia of Down Syndrome.","key_result":"*Stag2* loss reprogrammed GATA1 occupancy away from erythroid targets toward megakaryocyte targets — enriched with FLI1 motifs — without changing *Gata1* expression levels; this was associated with reduced erythroid progenitor numbers, impaired terminal erythroid differentiation, expanded megakaryocyte progenitors, and augmented megakaryocyte output. *Fli1* knockdown partially reversed the erythroid-to-megakaryocyte skewing.","limitations":"The primary model is murine (*Stag2*∆ mice); human validation relies on cell line models and primary MDS patient samples without a dedicated clinical cohort. Causal directionality between chromatin remodeling and GATA1 retargeting is inferred from correlative genomic data rather than direct rescue experiments restoring full chromatin accessibility.","practice_takeaway":"In MDS patients with *STAG2* mutations, dyserythropoiesis may be driven by chromatin-mediated retargeting of GATA1 toward megakaryocyte programs rather than loss of GATA1 itself — making chromatin accessibility and FLI1 activity potential therapeutic targets. Clinicians should be aware that *STAG2*-mutant MDS may present with a distinct erythroid-to-megakaryocyte lineage skewing phenotype."}},{"doi":"10.1182/blood.2025032031","pmid":"41940951","title":"The ASH HematOmics Program supports integrative analysis of genomic and clinical data in hematologic diseases","journal_title":"Blood Journal","journal_issn":"0006-4971","published_at":"2026-04-06T00:00:00.000Z","url":"https://doi.org/10.1182/blood.2025032031","source_score":45,"is_retracted":false,"is_open_access":false,"raw_metadata":{"pubmed":true,"crossref":true,"unpaywall":true},"created_at":"2026-05-07T22:51:13.921Z","updated_at":"2026-05-07T22:51:13.921Z","tags":[{"paper_doi":"10.1182/blood.2025032031","tag_type":"age_group","value":"mixed","value_numeric":null},{"paper_doi":"10.1182/blood.2025032031","tag_type":"category","value":"Hematology","value_numeric":null},{"paper_doi":"10.1182/blood.2025032031","tag_type":"condition","value":"Acute Myeloid Leukemia","value_numeric":null},{"paper_doi":"10.1182/blood.2025032031","tag_type":"condition","value":"B-Cell Precursor Acute Lymphoblastic Leukemia","value_numeric":null},{"paper_doi":"10.1182/blood.2025032031","tag_type":"condition","value":"Chronic Lymphocytic Leukemia","value_numeric":null},{"paper_doi":"10.1182/blood.2025032031","tag_type":"condition","value":"Myelodysplastic Syndrome","value_numeric":null},{"paper_doi":"10.1182/blood.2025032031","tag_type":"condition","value":"T-Cell Acute Lymphoblastic Leukemia","value_numeric":null},{"paper_doi":"10.1182/blood.2025032031","tag_type":"impact_flag","value":"practice_changing","value_numeric":null},{"paper_doi":"10.1182/blood.2025032031","tag_type":"level_of_evidence","value":"5","value_numeric":null},{"paper_doi":"10.1182/blood.2025032031","tag_type":"sample_size","value":"n=5960","value_numeric":5960},{"paper_doi":"10.1182/blood.2025032031","tag_type":"sex","value":"mixed","value_numeric":null},{"paper_doi":"10.1182/blood.2025032031","tag_type":"study_design","value":"Database / Resource Description","value_numeric":null},{"paper_doi":"10.1182/blood.2025032031","tag_type":"warning","value":"No prospective or randomized data are reported; clinical outcomes cited are illustrative use cases drawn from existing datasets within the platform.","value_numeric":null},{"paper_doi":"10.1182/blood.2025032031","tag_type":"warning","value":"The 'practice_changing' impact flag is applied in the context of research and genomic data interpretation workflows, not direct patient management changes.","value_numeric":null},{"paper_doi":"10.1182/blood.2025032031","tag_type":"warning","value":"This paper describes a bioinformatics platform and resource, not a clinical study — 'level of evidence' and 'impact_flag' reflect its utility as a discovery/reference tool rather than a clinical intervention.","value_numeric":null}],"summaries":{"what_studied":"The ASH HematOmics Program (ASHOP) is an open-access web platform integrating genomic, transcriptomic, and clinical data from 5,960 patients across B-cell precursor ALL, T-cell ALL, AML, MDS, and CLL, enabling user-driven exploratory and correlative analyses.","key_result":"ASHOP supports four illustrated use cases: (1) stratification of DUX4-rearranged B-ALL into Early/Multipotent vs. Committed subgroups with distinct outcomes, (2) HOXA/HOXB expression patterns in AML, (3) linking mutational burden to mismatch repair deficiency and mutational signatures, and (4) mapping the TP53 alteration landscape across cohorts.","limitations":"This is a platform/resource description rather than a clinical trial, so no comparative efficacy or patient-outcome data are directly tested; the current dataset is limited to four disease types and will require ongoing expansion to broaden applicability.","practice_takeaway":"Clinicians and researchers can use ASHOP (ashop.hematology.org) as a free, interactive tool to explore somatic mutations, gene fusions, transcriptomics, and clinical outcomes across major hematologic malignancies. It is particularly useful for defining and analyzing genomic sub-cohorts to contextualize findings from individual patients or studies."}},{"doi":"10.1182/blood.2025031858","pmid":"41785374","title":"Nonmyeloablative conditioning combined with the anti-CD117 antibody briquilimab in older adults with high-risk AML and MDS","journal_title":"Blood","journal_issn":"0006-4971","published_at":"2026-03-05T00:00:00.000Z","url":"https://doi.org/10.1182/blood.2025031858","source_score":6,"is_retracted":false,"is_open_access":false,"raw_metadata":{"pubmed":true,"crossref":true,"unpaywall":true},"created_at":"2026-06-02T13:27:32.032Z","updated_at":"2026-06-02T13:27:32.032Z","tags":[{"paper_doi":"10.1182/blood.2025031858","tag_type":"age_group","value":"older_adult","value_numeric":null},{"paper_doi":"10.1182/blood.2025031858","tag_type":"brand","value":"Briquilimab","value_numeric":null},{"paper_doi":"10.1182/blood.2025031858","tag_type":"category","value":"Hematology","value_numeric":null},{"paper_doi":"10.1182/blood.2025031858","tag_type":"condition","value":"Acute Myeloid Leukemia","value_numeric":null},{"paper_doi":"10.1182/blood.2025031858","tag_type":"condition","value":"Myelodysplastic Syndrome","value_numeric":null},{"paper_doi":"10.1182/blood.2025031858","tag_type":"generic","value":"fludarabine","value_numeric":null},{"paper_doi":"10.1182/blood.2025031858","tag_type":"impact_flag","value":"limited_evidence","value_numeric":null},{"paper_doi":"10.1182/blood.2025031858","tag_type":"level_of_evidence","value":"3","value_numeric":null},{"paper_doi":"10.1182/blood.2025031858","tag_type":"modality","value":"Allogeneic Hematopoietic Cell Transplantation","value_numeric":null},{"paper_doi":"10.1182/blood.2025031858","tag_type":"modality","value":"Monoclonal Antibody","value_numeric":null},{"paper_doi":"10.1182/blood.2025031858","tag_type":"sample_size","value":"n=32","value_numeric":32},{"paper_doi":"10.1182/blood.2025031858","tag_type":"sex","value":"mixed","value_numeric":null},{"paper_doi":"10.1182/blood.2025031858","tag_type":"study_design","value":"Phase 1 Clinical Trial","value_numeric":null},{"paper_doi":"10.1182/blood.2025031858","tag_type":"warning","value":"Briquilimab does not yet have a nonproprietary INN-style generic name confirmed in RxNorm; listed under brand name only.","value_numeric":null},{"paper_doi":"10.1182/blood.2025031858","tag_type":"warning","value":"Level of evidence assigned as 3 (non-randomized phase 1 trial with proof-of-concept design); no randomized comparator exists in this study.","value_numeric":null},{"paper_doi":"10.1182/blood.2025031858","tag_type":"warning","value":"The AML-in-relapse subgroup (n=3) is too small to interpret efficacy results and should be treated as anecdotal.","value_numeric":null}],"summaries":{"what_studied":"Phase 1 trial evaluating briquilimab (anti-CD117 monoclonal antibody) added to nonmyeloablative fludarabine/TBI conditioning in adults ≥median age 70 years with high-risk AML or MDS undergoing matched donor allogeneic HCT (NCT04429191).","key_result":"No infusion reactions, dose-limiting toxicities, or primary graft failures were observed (n=32). In AML-in-CR patients (n=13), 1-year EFS was 69.2% and OS was 75%; in MDS patients (n=16), 1-year EFS was 53.8% and OS was 76.4%. Briquilimab depleted AML/MDS hematopoietic stem and progenitor cells by a mean of 62.4% ± 22.7%, with a resultant ~3-fold rise in serum SCF.","limitations":"- Small phase 1 sample (n=32), limiting power to draw efficacy conclusions.\n- No control arm; comparisons to standard conditioning are not possible.\n- Only 3 patients had active AML in relapse, too few for meaningful efficacy assessment in that subgroup.","practice_takeaway":"Briquilimab combined with nonmyeloablative Flu/TBI appears safe and feasible as HCT conditioning in older adults with AML/MDS, with no graft failures and manageable toxicity. 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dose and duration data are absent.","value_numeric":null},{"paper_doi":"10.1182/bloodadvances.2025019262","tag_type":"warning","value":"Only 6.1% of the cohort reported AO exposure (n=130), limiting statistical power for subgroup analyses.","value_numeric":null},{"paper_doi":"10.1182/bloodadvances.2025019262","tag_type":"warning","value":"Study design is labeled prospective cohort (enrollment), but the Agent Orange exposure analysis is retrospective/observational — causal inference is limited.","value_numeric":null},{"paper_doi":"10.1182/bloodadvances.2025019262","tag_type":"warning","value":"The cohort was enriched for patients referred for bone marrow biopsy (all had cytopenias), which may overestimate AO–MDS association relative to the general veteran population.","value_numeric":null}],"summaries":{"what_studied":"This retrospective cohort study used data from the National MDS Natural History Study (n=2,115 patients with ≥1 cytopenia enrolled at 161 US sites, 2016–2024) to assess whether Agent Orange (AO) exposure is associated with MDS diagnosis, molecular features, cytogenetics, and clinical outcomes, comparing 130 AO-exposed vs. 1,985 non-exposed patients.","key_result":"AO-exposed patients were significantly more likely to be diagnosed with MDS or CCUS (54% vs. 37%, P=.022). On multivariable analysis, AO exposure was associated with more molecular mutations (OR 1.10; 95% CI 1.0–1.2; P=.047), poor/very poor cytogenetics (OR 2.39; 95% CI 1.04–5.14; P=.03), younger age at MDS diagnosis (OR 0.96/year; P=.008), TET2 mutations (26% vs. 17%; P=.008), RNA splicing pathway alterations (35% vs. 23%; P=.004), and a higher 2-year risk of disease progression (HR 1.8; 95% CI 1.2–2.8; P=.004); overall survival did not differ (HR 1.1; P=.72).","limitations":"- No data on AO exposure dose or duration, limiting dose-response analysis.\n- Unable to control for subsequent occupational exposures linked to MDS.\n- AO exposure was self-reported, introducing potential recall or ascertainment bias.","practice_takeaway":"Clinicians evaluating unexplained cytopenias in Vietnam-era veterans should inquire about AO exposure, as it is independently linked to MDS diagnosis and faster disease progression within 2 years. Black veterans appear disproportionately represented among AO-exposed MDS patients and may warrant heightened vigilance."}},{"doi":"10.1001/jamadermatol.2025.4586","pmid":"41259063","title":"Lupuslike Manifestations in Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia","journal_title":"JAMA Dermatology","journal_issn":"2168-6068","published_at":"2025-11-19T00:00:00.000Z","url":"https://doi.org/10.1001/jamadermatol.2025.4586","source_score":21,"is_retracted":false,"is_open_access":false,"raw_metadata":{"ooir":{"day":"2025-11-21","doi":"10.1001/jamadermatol.2025.4586","days":"14","issn":"2168-6068","rank":"10","field":"Clinical Medicine","score":"21","category":"Dermatology","categories":["Dermatology"],"_categories":["Dermatology"]},"pubmed":{"doi":"10.1001/jamadermatol.2025.4586","title":"Lupuslike Manifestations in Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia.","journal":"JAMA 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Centralized skin histopathology and targeted next-generation sequencing were performed; 24 patients were included.","key_result":"Compared with idiopathic systemic lupus, MDS/CMML-associated cases were older (median 65 vs 23 years), more often male (53% vs 8%), and had less kidney (10% vs 71%) and articular involvement (36% vs 97%), with fewer anti–double-stranded DNA positives (32% vs 76%). Central review reclassified 6 skin biopsies (50%) as MDS/CMML cutis; identical myeloid variants were found in blood and skin in 6/8 patients, and clone-directed therapy (azacitidine or allogeneic hematopoietic stem cell transplant) produced parallel hematologic and lupus responses in 5/7.","practice_takeaway":"In MDS/CMML patients with lupuslike disease, standard lupus therapies were often ineffective, while clone-targeted approaches achieved coupled hematologic and lupus responses in 5 of 7 cases; consider hematology-directed treatment when features and testing suggest clonal inflammation. Early dermatopathology review and targeted sequencing can help separate MDS/CMML cutis from lupus and guide therapy."}}],"initialOffset":12,"fetchConfig":{"kind":"tags","tags":[{"tag_type":"condition","value":"Myelodysplastic Syndrome"}]},"breadcrumbItems":[{"name":"Home","href":"/"},{"name":"Research Hub","href":"/hub"},{"name":"Conditions","href":"/hub/tag/condition"},{"name":"Myelodysplastic Syndrome"}],"hrefConfig":{"basePath":"/hub/papers","webViewQueryParams":{"hub_ctx":"tag","hub_tag_type":"condition","hub_tag_value":"Myelodysplastic Syndrome"}}}]]

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