{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T05:02:12Z","timestamp":1789016532248,"version":"build-2803163510"},"reference-count":29,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2022,4,19]],"date-time":"2022-04-19T00:00:00Z","timestamp":1650326400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"published-print":{"date-parts":[[2022,8]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>In order to improve the outcome observed with azacitidine (AZA) in higher\u2010risk Myelodysplastic syndrome (MDS), its combination with other drugs in MDS must be evaluated. So far, no combination has not been shown to be more effective than AZA alone. AZA\u2010PLUS was a phase II trial that, in a \u201cpick a winner\u201d approach, randomly assigned patients with higher\u2010risk MDS, CMML and low blast count AML to: AZA; AZA plus lenalidomide; AZA plus Valproic Acid or AZA plus Idarubicin. 322 patients were included. After six\u2009cycles, 69 (21.4%) CR\u2009+\u2009PR were observed with no benefit from any combination. Median EFS and OS were 17.2 and 19.7\u2009months in the whole cohort, respectively, with no difference across randomised arms. Infection and rates of hospitalisation during the first six\u2009cycles were higher in the AZA\u2010LEN And AZA\u2010IDA arm, related to increased myelosuppression. Factors associated with better response were IPSS, favourable or intermediate karyotype, haemoglobin, lower circulating blast count, fibrinogen level and lower LDH, while poorer survival was seen in therapy\u2010related MDS and, in the case of<jats:italic>TP53<\/jats:italic>,<jats:italic>PTPN11<\/jats:italic>or<jats:italic>CSF3R<\/jats:italic>mutation. The combinations used did not improve the outcome obtained with AZA alone. However, our \u201cpick a winner\u201d randomised strategy may remain useful with potentially more active drugs to be tested in combination with AZA.<\/jats:p>","DOI":"10.1111\/bjh.18193","type":"journal-article","created":{"date-parts":[[2022,4,19]],"date-time":"2022-04-19T18:27:29Z","timestamp":1650392849000},"page":"535-544","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":20,"title":["A randomised phase<scp>II<\/scp>study of azacitidine (<scp>AZA<\/scp>) alone or with Lenalidomide (<scp>LEN<\/scp>), Valproic acid (<scp>VPA<\/scp>) or Idarubicin (<scp>IDA<\/scp>) in<scp>higher\u2010Risk MDS<\/scp>or low blast<scp>AML<\/scp>:<scp>GFM<\/scp>'s \u201cpick a winner\u201d trial, with the impact of somatic 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