{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,3]],"date-time":"2026-08-03T20:18:37Z","timestamp":1785788317396,"version":"3.56.0"},"reference-count":41,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2026,4,1]],"date-time":"2026-04-01T00:00:00Z","timestamp":1775001600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,4,1]],"date-time":"2026-04-01T00:00:00Z","timestamp":1775001600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Therapeutic Advances in Hematology"],"published-print":{"date-parts":[[2026,4]]},"abstract":"<jats:p>Thrombotic thrombocytopenic purpura (TTP) is a life-threatening thrombotic microangiopathy driven by severe ADAMTS13 deficiency with consequent ultra-large von Willebrand factor-mediated microthrombosis. Over the last two decades, understanding of the ADAMTS13\u2013vWF axis has transformed management and made TTP a model for precision medicine. This narrative review (adult focus) synthesizes contemporary evidence on diagnosis, treatment, genetics, and emerging therapies in immune-mediated TTP. We summarize a pragmatic, biomarker-guided algorithm integrating early therapeutic plasma exchange and corticosteroids, upfront rituximab to suppress anti-ADAMTS13 autoimmunity, and caplacizumab to block vWF\u2013platelet interactions; define escalation strategies for refractory disease (e.g., proteasome inhibition); and formalize treat-to-target de-escalation anchored to ADAMTS13 recovery. We emphasize the role of rapid ADAMTS13 assays for front-door triage, routine ADAMTS13 monitoring in remission, and preemptive rituximab at biochemical relapse to avert clinical recurrence. Precision diagnostics are extended by genetic markers (e.g., HLA associations) and emerging biomarkers that may refine relapse risk. Finally, we discuss near-term opportunities\u2014point-of-care ADAMTS13 assays, longer-acting vWF-pathway inhibitors, and data-driven risk tools\u2014that can further individualize timing and intensity of therapy. Collectively, a biomarker-first strategy offers a clear path to fewer exacerbations, fewer relapses, and more consistent outcomes.<\/jats:p>","DOI":"10.1177\/20406207261430423","type":"journal-article","created":{"date-parts":[[2026,4,10]],"date-time":"2026-04-10T00:26:36Z","timestamp":1775780796000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":3,"title":["Precision medicine in thrombotic thrombocytopenic purpura: a narrative review"],"prefix":"10.1177","volume":"17","author":[{"ORCID":"https:\/\/orcid.org\/0009-0001-1171-6034","authenticated-orcid":false,"given":"Rafal","family":"Al-Shibly","sequence":"first","affiliation":[{"name":"Internal Medicine Residency Program, Department of Medical Education, Hamad Medical Corporation, Doha 3050, Qatar"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1392-8831","authenticated-orcid":false,"given":"Rola","family":"Ghasoub","sequence":"additional","affiliation":[{"name":"Pharmacy Department, National Center for Cancer Care and Research, Hamad Medical Corporation, Doha, Qatar"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Muna","family":"AlRasheed","sequence":"additional","affiliation":[{"name":"Department of Hematology, Adan Hospital, Ministry of Health, Kuwait, Al Asimah Governorate, Kuwait"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lulwa","family":"AlTourah","sequence":"additional","affiliation":[{"name":"Department of Hematology, Adan Hospital, Ministry of Health, Kuwait, Al Asimah Governorate, Kuwait"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Taghreed","family":"Al-Eisa","sequence":"additional","affiliation":[{"name":"Al-Jahra Hospital, Ministry of Health, Kuwait, Al Jahra Governorate, Kuwait"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mohamed A.","family":"Yassin","sequence":"additional","affiliation":[{"name":"Department of Hematology, National Center for Cancer Care and Research, Hamad Medical Corporation, Doha, Qatar"},{"name":"College of Medicine, Qatar University, Doha, Qatar"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2026,4,9]]},"reference":[{"key":"e_1_3_4_2_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2016-10-709857"},{"key":"e_1_3_4_3_2","doi-asserted-by":"publisher","DOI":"10.1016\/S2352-3026(16)30018-7"},{"key":"e_1_3_4_4_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199811263392202"},{"key":"e_1_3_4_5_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199811263392203"},{"key":"e_1_3_4_6_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0899-3467(07)60142-6"},{"key":"e_1_3_4_7_2","doi-asserted-by":"publisher","DOI":"10.1111\/jth.15006"},{"key":"e_1_3_4_8_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jtha.2025.06.002"},{"key":"e_1_3_4_9_2","doi-asserted-by":"publisher","DOI":"10.1111\/jth.15009"},{"key":"e_1_3_4_10_2","doi-asserted-by":"publisher","DOI":"10.1016\/S2352-3026(17)30026-1"},{"key":"e_1_3_4_11_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0010208"},{"key":"e_1_3_4_12_2","doi-asserted-by":"publisher","DOI":"10.1055\/s-0039-1696718"},{"key":"e_1_3_4_13_2","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.26241"},{"key":"e_1_3_4_14_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2020005395"},{"key":"e_1_3_4_15_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2020009150"},{"key":"e_1_3_4_16_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199108083250604"},{"issue":"3","key":"e_1_3_4_17_2","first-page":"245","article-title":"Immune thrombotic thrombocytopenic purpura management without therapeutic plasma exchange","volume":"144","author":"K\u00fchne L","year":"2024","unstructured":"K\u00fchne L, Ebert N, T\u00f6lle M, et al. Immune thrombotic thrombocytopenic purpura management without therapeutic plasma exchange. Blood 2024; 144(3): 245\u2013255.","journal-title":"Blood"},{"key":"e_1_3_4_18_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.blre.2025.101326"},{"key":"e_1_3_4_19_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1505533"},{"key":"e_1_3_4_20_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1806311"},{"key":"e_1_3_4_21_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2402567"},{"key":"e_1_3_4_22_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2014-01-550244"},{"key":"e_1_3_4_23_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2018-04-840090"},{"key":"e_1_3_4_24_2","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.13993"},{"issue":"1","key":"e_1_3_4_25_2","first-page":"22","article-title":"Bortezomib in the management of immune-mediated TTP: a systematic review","volume":"202","author":"Lee NCJ","year":"2023","unstructured":"Lee NCJ, Yates S, Rambally S, et al. Bortezomib in the management of immune-mediated TTP: a systematic review. Br J Haematol 2023; 202(1): 22\u201331.","journal-title":"Br J Haematol"},{"key":"e_1_3_4_26_2","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.27550"},{"issue":"4","key":"e_1_3_4_27_2","first-page":"617","article-title":"Daratumumab for refractory immune thrombotic thrombocytopenic purpura","volume":"203","author":"Aggarwal A","year":"2023","unstructured":"Aggarwal A, Balasubramanian SK, George JN, et al. Daratumumab for refractory immune thrombotic thrombocytopenic purpura. Br J Haematol 2023; 203(4): 617\u2013620.","journal-title":"Br J Haematol"},{"key":"e_1_3_4_28_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2014-11-551580"},{"issue":"13","key":"e_1_3_4_29_2","first-page":"3088","article-title":"ADAMTS13-guided duration of caplacizumab therapy in immune-mediated thrombotic thrombocytopenic purpura","volume":"4","author":"V\u00f6lker LA","year":"2020","unstructured":"V\u00f6lker LA, Kaufeld J, Miesbach W, et al. ADAMTS13-guided duration of caplacizumab therapy in immune-mediated thrombotic thrombocytopenic purpura. Blood Adv 2020; 4(13): 3088\u20133094.","journal-title":"Blood Adv"},{"key":"e_1_3_4_30_2","unstructured":"Scully M Peyvandi F Kremer Hovinga JA et al. Caplacizumab plus immunosuppression without plasma exchange for immune thrombotic thrombocytopenic purpura (MAYARI study). Presented at: International Society on Thrombosis and Haemostasis (ISTH) Congress; 2025."},{"issue":"2","key":"e_1_3_4_31_2","first-page":"297","article-title":"Caplacizumab and other novel approaches in thrombotic thrombocytopenic purpura","volume":"109","author":"Capecchi M","year":"2024","unstructured":"Capecchi M, Peyvandi F. Caplacizumab and other novel approaches in thrombotic thrombocytopenic purpura. Haematologica 2024; 109(2): 297\u2013310.","journal-title":"Haematologica"},{"key":"e_1_3_4_32_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2016-06-724161"},{"issue":"18","key":"e_1_3_4_33_2","first-page":"2797","article-title":"Biomarkers for relapse in immune-mediated thrombotic thrombocytopenic purpura","volume":"3","author":"Sui J","year":"2019","unstructured":"Sui J, Zhang C, Li Y, et al. Biomarkers for relapse in immune-mediated thrombotic thrombocytopenic purpura. Blood Adv 2019; 3(18): 2797\u20132806.","journal-title":"Blood Adv"},{"key":"e_1_3_4_34_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2016-12-758656"},{"issue":"12","key":"e_1_3_4_35_2","first-page":"2396","article-title":"Markers of residual disease in immune thrombotic thrombocytopenic purpura","volume":"104","author":"Staley EM","year":"2019","unstructured":"Staley EM, Terrell DR, Vesely SK, et al. Markers of residual disease in immune thrombotic thrombocytopenic purpura. Haematologica 2019; 104(12): 2396\u20132404.","journal-title":"Haematologica"},{"key":"e_1_3_4_36_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jtha.2023.10.014"},{"issue":"15","key":"e_1_3_4_37_2","first-page":"1373","article-title":"Open ADAMTS13 conformation is a biomarker for relapse risk in immune-mediated thrombotic thrombocytopenic purpura","volume":"135","author":"Roose E","year":"2020","unstructured":"Roose E, Vidarsson G, Kangro K, et al. Open ADAMTS13 conformation is a biomarker for relapse risk in immune-mediated thrombotic thrombocytopenic purpura. Blood 2020; 135(15): 1373\u20131383.","journal-title":"Blood"},{"key":"e_1_3_4_38_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2017-06-788026"},{"key":"e_1_3_4_39_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jtha.2023.11.030"},{"key":"e_1_3_4_40_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ebiom.2015.06.007"},{"issue":"4","key":"e_1_3_4_41_2","first-page":"341","article-title":"Caplacizumab without plasma exchange in acquired thrombotic thrombocytopenic purpura","volume":"380","author":"Chander DP","year":"2019","unstructured":"Chander DP, Cataland SR, Coppo P, et al. Caplacizumab without plasma exchange in acquired thrombotic thrombocytopenic purpura. N Engl J Med 2019; 380(4): 341\u2013343.","journal-title":"N Engl J Med"},{"issue":"2","key":"e_1_3_4_42_2","article-title":"Successful caplacizumab-only therapy for immune thrombotic thrombocytopenic purpura: case report","volume":"30","author":"Spencer BT","year":"2023","unstructured":"Spencer BT, George JN, Terrell DR, et al. Successful caplacizumab-only therapy for immune thrombotic thrombocytopenic purpura: case report. Am J Ther 2023; 30(2): e156\u2013e160.","journal-title":"Am J Ther"}],"container-title":["Therapeutic Advances in Hematology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/20406207261430423","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/20406207261430423","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/20406207261430423","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,30]],"date-time":"2026-04-30T23:18:19Z","timestamp":1777591099000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/20406207261430423"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,4]]},"references-count":41,"alternative-id":["10.1177\/20406207261430423"],"URL":"https:\/\/doi.org\/10.1177\/20406207261430423","relation":{},"ISSN":["2040-6207","2040-6215"],"issn-type":[{"value":"2040-6207","type":"print"},{"value":"2040-6215","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,4]]},"article-number":"20406207261430423"}}