{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,29]],"date-time":"2026-08-29T22:38:07Z","timestamp":1788043087444,"version":"build-2784847793"},"reference-count":0,"publisher":"American Society of Hematology","license":[{"start":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T00:00:00Z","timestamp":1787011200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"abstract":"<jats:p>Polycythemia vera is characterized by erythrocytosis and increased risk of thrombosis. Sapablursen is an antisense oligonucleotide that suppresses transmembrane serine protease 6 (TMPRSS6), increases hepcidin production, and reduces iron availability for erythropoiesis. IMPRSSION; an ongoing phase 2a, randomized, open-label study includes patients meeting 2016 WHO criteria for polycythemia vera, requiring \u22653 phlebotomies within 6 months of screening and \u22651 phlebotomy within the last 12 weeks. Cohort A (120 or 80 mg) and Cohort B (40 mg) received sapablursen subcutaneously once every 4 weeks (Q4W). The primary efficacy endpoint was the reduction in phlebotomy frequency from baseline to weeks 17-37 in patients who received \u22651 dose. Forty-nine patients received \u22651 sapablursen dose (median age 61 years; 82% male). The baseline mean\u00b1SD weekly phlebotomy rate was 0.15\u00b10.07 in Cohort A; 0.17\u00b10.07 in Cohort B and was reduced by -0.10 (95%CI, -0.13, -0.07; n=32;p&amp;lt;0.0001) in Cohort A; -0.10 (95%CI, -0.14, -0.06; n=17;p=0.0001) in Cohort B. Sapablursen reduced the median (IQR) number of phlebotomies from 5.0 (3.0-6.0,Cohort A; 3.5-6.5,Cohort B) at baseline (26-weeks) to 0 (0-1.0) in the efficacy evaluation window (20-weeks) in Cohort A and 1.5 (0-2.5) in Cohort B. Most adverse events were mild or moderate; anemia and fatigue were most common. The mean change in MPN-SAF-TSS at week 37 was -6.21 points (95%CI: -10.56, -1.85, p=0.0052) in Cohort A; -2.71 points in Cohort B (95% CI: -8.33, 2.91, p=0.34). Sapablursen reduced phlebotomy need, improved symptoms, and was safe and well-tolerated in patients with polycythemia vera. (Funded by Ionis Pharmaceuticals; ClinicalTrials.gov: NCT05143957)<\/jats:p>","DOI":"10.1182\/blood.2026033792","type":"journal-article","created":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T17:06:29Z","timestamp":1787072789000},"update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":1,"title":["Sapablursen for Erythrocytosis Control in Patients with Polycythemia Vera"],"prefix":"10.1182","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2185-1504","authenticated-orcid":false,"given":"Jeanne M","family":"Palmer","sequence":"first","affiliation":[{"name":"Mayo Clinic College of Medicine and Science, Division of Hematology\/Oncology, United States"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jonathan","family":"How","sequence":"additional","affiliation":[{"name":"Division of Hematology, Royal Victoria Hospital, McGill University, Montreal, Quebec, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4343-5712","authenticated-orcid":false,"given":"Prithviraj","family":"Bose","sequence":"additional","affiliation":[{"name":"University of Texas MD Anderson Cancer Center, Houston, Texas, United States"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9779-6217","authenticated-orcid":false,"given":"Pankit","family":"Vachhani","sequence":"additional","affiliation":[{"name":"4. University of Alabama at Birmingham, Birmingham, AL, USA, United States"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8522-1002","authenticated-orcid":false,"given":"Adam J.","family":"Mead","sequence":"additional","affiliation":[{"name":"5. Medical Research Council Weatherall Institute of Molecular Medicine (MRC WIMM) and NIHR Biomedical Research Centre Hematology Theme, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jonathan","family":"Sillar","sequence":"additional","affiliation":[{"name":"Calvary Mater Hospital, Waratah, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3422-1309","authenticated-orcid":false,"given":"Aaron T","family":"Gerds","sequence":"additional","affiliation":[{"name":"Cleveland Clinic Taussig Cancer Institute, Cleveland, Ohio, United States"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3701-6079","authenticated-orcid":false,"given":"Angela G","family":"Fleischman","sequence":"additional","affiliation":[{"name":"University of California, Irvine, Irvine, California, United States"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9227-5789","authenticated-orcid":false,"given":"Caitlin M","family":"O'Neill","sequence":"additional","affiliation":[{"name":"Keck School of Medicine, University of Southern California, Los Angeles, California, United States"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2728-6788","authenticated-orcid":false,"given":"Ross Ian","family":"Baker","sequence":"additional","affiliation":[{"name":"Perth Blood Institute, PERTH, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ronald","family":"Hoffman","sequence":"additional","affiliation":[{"name":"Mount Sinai Medical Center, New York, New York, United States"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shiangtung","family":"Jung","sequence":"additional","affiliation":[{"name":"Ionis Pharmaceuticals, Carlsbad, California, United States"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jon L","family":"Ruckle","sequence":"additional","affiliation":[{"name":"Pacific Pharma Group, LLC, Tacoma, Washington, United States"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Keyvan","family":"Yousefi","sequence":"additional","affiliation":[{"name":"Ionis Pharmaceuticals, Carlsbad, California, United States"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sanjay","family":"Bhanot","sequence":"additional","affiliation":[{"name":"Ionis Pharmaceuticals, Carlsbad, California, United States"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0850-8365","authenticated-orcid":false,"given":"Terrance D.","family":"Barrett","sequence":"additional","affiliation":[{"name":"Ionis Pharmaceuticals, Carlsbad, California, United States"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ruben A","family":"Mesa","sequence":"additional","affiliation":[{"name":"Atrium Health Wake Forest Baptist Comprehensive Cancer Center, Winston-Salem, North Carolina, United States"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"234","published-online":{"date-parts":[[2026,8,18]]},"container-title":["Blood Journal"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/ashpublications.org\/blood\/article-pdf\/doi\/10.1182\/blood.2026033792\/2538982\/blood.2026033792.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/ashpublications.org\/blood\/article-pdf\/doi\/10.1182\/blood.2026033792\/2538982\/blood.2026033792.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T17:06:29Z","timestamp":1787072789000},"score":1,"resource":{"primary":{"URL":"https:\/\/ashpublications.org\/blood\/article\/doi\/10.1182\/blood.2026033792\/570260\/Sapablursen-for-Erythrocytosis-Control-in-Patients"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,8,18]]},"references-count":0,"URL":"https:\/\/doi.org\/10.1182\/blood.2026033792","relation":{},"ISSN":["0006-4971","1528-0020"],"issn-type":[{"value":"0006-4971","type":"print"},{"value":"1528-0020","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,8,18]]},"article-number":"blood.2026033792"}}