{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,3]],"date-time":"2026-11-03T15:30:33Z","timestamp":1793719833310,"version":"4.6.1"},"reference-count":24,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2026,8,25]],"date-time":"2026-08-25T00:00:00Z","timestamp":1787616000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,8,25]],"date-time":"2026-08-25T00:00:00Z","timestamp":1787616000000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/501100001411","name":"Indian Council of Medical Research","doi-asserted-by":"publisher","award":["211807"],"award-info":[{"award-number":["211807"]}],"id":[{"id":"10.13039\/501100001411","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["European J of Haematology"],"published-print":{"date-parts":[[2026,12]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>\n                      Sickle cell disease (SCD) is an inherited hemoglobin disorder caused by a point mutation in the\n                      <jats:italic>\u03b2<\/jats:italic>\n                      \u2010globin gene, producing hemoglobin S (HbS). Under hypoxic conditions, HbS polymerizes, leading to rigid, sickled erythrocytes with reduced deformability and increased endothelial adhesion, resulting in vaso\u2010occlusion and impaired tissue perfusion. Desidustat, a hypoxia\u2010inducible factor prolyl hydroxylase inhibitor, stabilizes HIF and enhances endogenous erythropoietin production. This Phase IIa study evaluated its efficacy and safety in SCD.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Twenty\u2010four Indian adults with confirmed SCD (Hb 7\u201311\u2009g\/dL for males, 6.5\u201311\u2009g\/dL for females) were enrolled. Patients were randomized (3:1) across three cohorts receiving 50\u2009mg, 100\u2009mg, or 150\u2009mg desidustat, or placebo, thrice weekly for 8\u2009weeks. The primary endpoint was the proportion achieving \u2265\u20091\u2009g\/dL hemoglobin increase from baseline to Week 8. Secondary endpoints included changes in hemoglobin, hemolysis biomarkers, HbS percentage, transfusion requirement, and vaso\u2010occlusive events. Safety was assessed via adverse events and clinical parameters.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Among 24 patients (10 males, 14 females; age 25\u201338\u2009years), Hb response was observed in 50% of patients in the 150\u2009mg cohort versus 16.7% in placebo. Hemoglobin increased dose\u2010dependently, with reduced LDH levels. No significant changes were noted in HbS or most hemolysis markers. No transfusions or vaso\u2010occlusive crises occurred. Reported adverse events were mild (nasopharyngitis, polyarthritis, headache), non\u2010serious, and unrelated to treatment.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>Desidustat was well tolerated, with the 150\u2009mg dose showing promising improvements in hemoglobin levels and response rates in SCD patients.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Trial Registration<\/jats:title>\n                    <jats:p>CTRI\/2024\/06\/068363<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/ejh.70296","type":"journal-article","created":{"date-parts":[[2026,8,25]],"date-time":"2026-08-25T11:06:44Z","timestamp":1787656004000},"page":"1593-1600","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["A Phase\n                    <scp>IIa<\/scp>\n                    , Double Blind, Randomized, Placebo Controlled, Parallel, Multicentre, Proof\u2010of\u2010Concept Study to Evaluate the Efficacy and Safety of Desidustat Oral Tablet for Treatment of Sickle Cell Disease"],"prefix":"10.1111","volume":"117","author":[{"given":"Mohit","family":"Desai","sequence":"first","affiliation":[{"name":"Zydus Medical College and Hospital  Dahod Gujarat India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bhargav","family":"Solanki","sequence":"additional","affiliation":[{"name":"Silver Touch Hospital  Ahmedabad Gujarat India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manish","family":"Hathila","sequence":"additional","affiliation":[{"name":"Sai Krupa Research Centre  Ahmedabad Gujarat India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2652-610X","authenticated-orcid":false,"given":"Vikas","family":"Asati","sequence":"additional","affiliation":[{"name":"Sri Aurobindo Institute of Medical Sciences  Indore India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suchi","family":"Shah","sequence":"additional","affiliation":[{"name":"Zydus Research Centre Clinical R&amp;D  Ahmedabad Gujarat India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Deven","family":"Parmar","sequence":"additional","affiliation":[{"name":"Zydus Research Centre Clinical R&amp;D  Ahmedabad Gujarat India"},{"name":"Zydus Therapeutics Inc. 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