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:T6e0,Hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA) is a potentially fatal multisystem complication of hematopoietic cell transplantation for which there is no approved treatment. In a single-arm study (NCT02222545), narsoplimab treatment for TA-TMA demonstrated a median overall survival (OS) of 274 days from date of diagnosis. Here, we compare OS observed in 2 cohorts treated with narsoplimab to OS in a well-matched external control to test survival benefit in patients with high-risk TA-TMA. OS in patients (aged ≥16 years) with high-risk TA-TMA treated with narsoplimab in a single-arm, open-label study (NCT02222545) or in the narsoplimab expanded access program (EAP; NCT04247906) was compared with OS in a control group with high-risk TA-TMA from the Kyoto Stem Cell Transplantation Group (KSCTG) registry. Narsoplimab-treated patients in the single-arm study (N = 28) had a fourfold reduction in risk of mortality compared with patients from the KSCTG registry (N = 111; hazard ratio [HR], 0.25; 95% confidence interval [CI], 0.19, 0.34; P < .0001). Similarly, in high-risk patients treated with narsoplimab in the EAP (N = 49), mortality risk was significantly lower than among high-risk patients from the KSCTG registry (N = 121; HR 0.38; 95% CI 0.28, 0.51; P < .0001). When narsoplimab-treated patients from the single-arm study and the EAP (N = 77) were compared with KSCTG patients, the HR for mortality was 0.28 (95% CI, 0.22, 0.37; P < .0001). In conclusion, in patients with high-risk TA-TMA, narsoplimab treatment significantly reduced mortality relative to a well-matched external control group who did not receive narsoplimab. These results support narsoplimab as a potential therapeutic option for TA-TMA.31:T77b, Abstract Hematopoietic stem cell transplant–associated thrombotic microangiopathy (TA-TMA) is a potentially fatal multisystem complication of hematopoietic cell transplantation for which there is no approved treatment. In a single-arm study (NCT02222545), narsoplimab treatment for TA-TMA demonstrated a median overall survival (OS) of 274 days from date of diagnosis. Here, we compare OS observed in 2 cohorts treated with narsoplimab to OS in a well-matched external control to test survival benefit in patients with high-risk TA-TMA. OS in patients (aged ≥16 years) with high-risk TA-TMA treated with narsoplimab in a single-arm, open-label study (NCT02222545) or in the narsoplimab expanded access program (EAP; NCT04247906) was compared with OS in a control group with high-risk TA-TMA from the Kyoto Stem Cell Transplantation Group (KSCTG) registry. Narsoplimab-treated patients in the single-arm study (N = 28) had a fourfold reduction in risk of mortality compared with patients from the KSCTG registry (N = 111; hazard ratio [HR], 0.25; 95% confidence interval [CI], 0.19, 0.34; P < .0001). Similarly, in high-risk patients treated with narsoplimab in the EAP (N = 49), mortality risk was significantly lower than among high-risk patients from the KSCTG registry (N = 121; HR 0.38; 95% CI 0.28, 0.51; P < .0001). When narsoplimab-treated patients from the single-arm study and the EAP (N = 77) were compared with KSCTG patients, the HR for mortality was 0.28 (95% CI, 0.22, 0.37; P < .0001). In conclusion, in patients with high-risk TA-TMA, narsoplimab treatment significantly reduced mortality relative to a well-matched external control group who did not receive narsoplimab. 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Advances"],"published-other":{"date-parts":[[2026,1,13]]},"published-print":{"date-parts":[[2026,1,13]]},"reference-count":44,"published-online":{"date-time":null,"timestamp":null,"date-parts":[[2025,12,31]]},"references-count":44,"short-container-title":[],"is-referenced-by-count":1},"unpaywall":{"doi":"10.1182/bloodadvances.2025017540","is_oa":true,"title":"Survival in adults with high-risk TA-TMA: a comparative analysis of narsoplimab vs supportive care","updated":"2026-01-18T01:04:10+00:00","published_date":"2025-10-10","best_oa_location":{"url":"https://ashpublications.org/bloodadvances/article-pdf/doi/10.1182/bloodadvances.2025017540/2416073/bloodadvances.2025017540.pdf","pmh_id":null,"url_for_pdf":"https://ashpublications.org/bloodadvances/article-pdf/doi/10.1182/bloodadvances.2025017540/2416073/bloodadvances.2025017540.pdf","url_for_landing_page":"https://doi.org/10.1182/bloodadvances.2025017540"}}},"created_at":"2026-01-08T08:12:02.165Z","updated_at":"2026-01-21T12:05:26.220Z","tags":[{"paper_doi":"10.1182/bloodadvances.2025017540","tag_type":"age_group","value":"mixed","value_numeric":null},{"paper_doi":"10.1182/bloodadvances.2025017540","tag_type":"category","value":"Hematology","value_numeric":null},{"paper_doi":"10.1182/bloodadvances.2025017540","tag_type":"condition","value":"Transplant-Associated Thrombotic Microangiopathy","value_numeric":null},{"paper_doi":"10.1182/bloodadvances.2025017540","tag_type":"generic","value":"narsoplimab","value_numeric":null},{"paper_doi":"10.1182/bloodadvances.2025017540","tag_type":"impact_flag","value":"limited_evidence","value_numeric":null},{"paper_doi":"10.1182/bloodadvances.2025017540","tag_type":"level_of_evidence","value":"3","value_numeric":null},{"paper_doi":"10.1182/bloodadvances.2025017540","tag_type":"modality","value":"Complement Inhibitor","value_numeric":null},{"paper_doi":"10.1182/bloodadvances.2025017540","tag_type":"study_design","value":"Cohort Study","value_numeric":null},{"paper_doi":"10.1182/bloodadvances.2025017540","tag_type":"warning","value":"Age criterion includes patients ≥16 years despite “adults” in the title.","value_numeric":null},{"paper_doi":"10.1182/bloodadvances.2025017540","tag_type":"warning","value":"Comparator details beyond lack of narsoplimab are not provided in the abstract.","value_numeric":null},{"paper_doi":"10.1182/bloodadvances.2025017540","tag_type":"warning","value":"Nonrandomized comparison using matched external registry controls; design classification is approximate.","value_numeric":null},{"paper_doi":"10.1182/bloodadvances.2025017540","tag_type":"warning","value":"The 274‑day median OS cited refers to a prior single‑arm study, not the comparative analyses.","value_numeric":null}],"summaries":{"what_studied":"Compared overall survival in patients aged ≥16 years with high‑risk transplant‑associated thrombotic microangiopathy treated with narsoplimab in a single‑arm study (N=28) or an expanded access program (N=49) versus well‑matched external controls from the KSCTG registry (N=111 and N=121).","key_result":"Narsoplimab was associated with substantially lower mortality than registry controls: HR 0.25 (95% CI 0.19–0.34; P<.0001) in the single‑arm cohort (N=28 vs 111) and HR 0.38 (95% CI 0.28–0.51; P<.0001) in the expanded access cohort (N=49 vs 121). Pooled analysis of narsoplimab‑treated patients (N=77) vs KSCTG yielded HR 0.28 (95% CI 0.22–0.37; P<.0001).","practice_takeaway":"In high‑risk TA‑TMA, narsoplimab was linked with a marked survival benefit over matched external controls. These data support narsoplimab as a potential treatment option where available."}}],"initialOffset":1,"fetchConfig":{"kind":"tags","tags":[{"tag_type":"condition","value":"Transplant-Associated Thrombotic Microangiopathy"}]},"breadcrumbItems":[{"name":"Home","href":"/"},{"name":"Research Hub","href":"/hub"},{"name":"Conditions","href":"/hub/tag/condition"},{"name":"Transplant-Associated Thrombotic Microangiopathy"}],"hrefConfig":{"basePath":"/hub/papers","webViewQueryParams":{"hub_ctx":"tag","hub_tag_type":"condition","hub_tag_value":"Transplant-Associated Thrombotic Microangiopathy"}}}]]

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