{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,12,18]],"date-time":"2025-12-18T20:01:48Z","timestamp":1766088108171,"version":"build-2065373602"},"reference-count":24,"publisher":"MDPI AG","issue":"20","license":[{"start":{"date-parts":[[2023,10,17]],"date-time":"2023-10-17T00:00:00Z","timestamp":1697500800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Novartis"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JCM"],"abstract":"<jats:p>Myelodysplastic syndromes and myeloproliferative neoplasms both represent hematologic diseases associated with bone marrow failure often resulting in anemia. For those patients, transfusion of red blood cell (RBC) units is essential but results in iron overload (IOL) that may affect various organ functions. Therefore, iron chelation therapy plays a major role in anemic patients, not only because it reduces IOL, but also because it may improve hematopoietic function by increasing hemoglobin or diminishing the requirement for RBC transfusions. To assess the utility, efficacy, and safety of the different iron chelation medications approved in Germany, as well as to examine the effect of chelation on hematopoietic insufficiency, a prospective, multicenter, noninterventional study named EXCALIBUR was designed. In total, 502 patients from 106 German hospitals and medical practices were enrolled. A large proportion of patients switched from a deferasirox dispersible tablet to a deferasirox-film-coated tablet, mainly because of more convenient application, which was reflected in the treatment satisfaction questionnaire for medication scores. Iron chelation was effective in lowering serum ferritin levels, with the observed adverse drug reactions being in line with the known safety profile. Hematologic response occurred in a few patients, comparable to other studies that examined hematologic improvement in patients with MDS.<\/jats:p>","DOI":"10.3390\/jcm12206569","type":"journal-article","created":{"date-parts":[[2023,10,17]],"date-time":"2023-10-17T08:25:09Z","timestamp":1697531109000},"page":"6569","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Iron Chelation in Patients with Myelodysplastic Syndromes and Myeloproliferative Neoplasms\u2014Real-World Data from the German Noninterventional Study EXCALIBUR"],"prefix":"10.3390","volume":"12","author":[{"given":"Felicitas","family":"Schulz","sequence":"first","affiliation":[{"name":"Department for Hematology, Oncology and Clinical Immunology, University Hospital Duesseldorf, 40225 D\u00fcsseldorf, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ulrich","family":"Hauch","sequence":"additional","affiliation":[{"name":"Practice for Hematology and Oncology, 99084 Erfurt, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sandra","family":"Ketzler-Henkel","sequence":"additional","affiliation":[{"name":"Practice for Hematology and Oncology, 44309 Dortmund, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Eyck","family":"von der Heyde","sequence":"additional","affiliation":[{"name":"Oncologic Center at Raschplatz, 30161 Hannover, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michael","family":"Koenigsmann","sequence":"additional","affiliation":[{"name":"MediProjekt GbR, 30171 Hannover, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michael","family":"Lauseker","sequence":"additional","affiliation":[{"name":"Institut f\u00fcr Medizinische Informationsverarbeitung Biometrie und Epidemiologie (IBE), Fakult\u00e4t f\u00fcr Medizin, Ludwig-Maximilians Universit\u00e4t M\u00fcnchen, 81377 Munich, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nora","family":"Schulte","sequence":"additional","affiliation":[{"name":"Novartis Pharma GmbH, 90429 Nuremberg, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ulrich","family":"Germing","sequence":"additional","affiliation":[{"name":"Department for Hematology, Oncology and Clinical Immunology, University Hospital Duesseldorf, 40225 D\u00fcsseldorf, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2023,10,17]]},"reference":[{"key":"ref_1","first-page":"847","article-title":"The Evaluation of Iron Deficiency and Iron Overload","volume":"118","author":"Gattermann","year":"2021","journal-title":"Dtsch. \u00c4rzteblatt Int."},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"431","DOI":"10.1016\/j.beha.2013.09.009","article-title":"When is iron overload deleterious, and when and how should iron chelation therapy be administered in myelodysplastic syndromes?","volume":"26","author":"Steensma","year":"2013","journal-title":"Best Pract. Res. Clin. Haematol."},{"key":"ref_3","doi-asserted-by":"crossref","unstructured":"Santini, V., Girelli, D., Sanna, A., Martinelli, N., Duca, L., Campostrini, N., Cortelezzi, A., Corbella, M., Bosi, A., and Reda, G. (2011). Hepcidin Levels and Their Determinants in Different Types of Myelodysplastic Syndromes. PLoS ONE, 6.","DOI":"10.1371\/journal.pone.0023109"},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"1617","DOI":"10.1007\/s00277-013-1839-5","article-title":"Serum hepcidin measured with an improved ELISA correlates with parameters of iron metabolism in patients with myelodysplastic syndrome","volume":"92","author":"Zipperer","year":"2013","journal-title":"Ann. Hematol."},{"key":"ref_5","doi-asserted-by":"crossref","first-page":"55","DOI":"10.1007\/s12185-017-2367-1","article-title":"Iron overload in myelodysplastic syndromes (MDS)","volume":"107","author":"Gattermann","year":"2017","journal-title":"Int. J. Hematol."},{"key":"ref_6","doi-asserted-by":"crossref","first-page":"420","DOI":"10.1002\/ajh.24668","article-title":"New film-coated tablet formulation of deferasirox is well tolerated in patients with thalassemia or lower-risk MDS: Results of the randomized, phase II ECLIPSE study","volume":"92","author":"Taher","year":"2017","journal-title":"Am. J. Hematol."},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"1433","DOI":"10.3324\/haematol.2011.044602","article-title":"Impact of the degree of anemia on the outcome of patients with myelodysplastic syndrome and its integration into the WHO classification-based Prognostic Scoring System (WPSS)","volume":"96","author":"Malcovati","year":"2011","journal-title":"Haematologica"},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"S7","DOI":"10.1016\/S0145-2126(07)70460-5","article-title":"Improving clinical outcome in patients with myelodysplastic syndrome and iron overload using iron chelation therapy","volume":"31","author":"Leitch","year":"2007","journal-title":"Leuk. Res."},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"419","DOI":"10.1182\/blood-2005-10-4149","article-title":"Clinical application and proposal for modification of the International Working Group (IWG) response criteria in myelodysplasia","volume":"108","author":"Cheson","year":"2006","journal-title":"Blood"},{"key":"ref_10","doi-asserted-by":"crossref","first-page":"1531","DOI":"10.1007\/s00432-018-2665-x","article-title":"Tolerability and efficacy of deferasirox in patients with transfusional iron overload: Results from a German 2-year non-interventional study","volume":"144","author":"Nolte","year":"2018","journal-title":"J. Cancer Res. Clin. Oncol."},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"1143","DOI":"10.1016\/j.leukres.2010.03.009","article-title":"Deferasirox in iron-overloaded patients with transfusion-dependent myelodysplastic syndromes: Results from the large 1-year EPIC study","volume":"34","author":"Gattermann","year":"2010","journal-title":"Leuk. Res."},{"key":"ref_12","doi-asserted-by":"crossref","first-page":"1131","DOI":"10.1016\/j.leukres.2011.06.013","article-title":"Clinical management of gastrointestinal disturbances in patients with myelodysplastic syndromes receiving iron chelation treatment with deferasirox","volume":"35","author":"Nolte","year":"2011","journal-title":"Leuk. Res."},{"key":"ref_13","unstructured":"(2023, September 15). Available online: https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/exjade."},{"key":"ref_14","unstructured":"(2023, September 15). Available online: https:\/\/klinischeforschung.novartis.de\/dokumente\/fachinformationen-desferal\/."},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"513","DOI":"10.7326\/M19-0916","article-title":"Iron Chelation in Transfusion-Dependent Patients With Low- to Intermediate-1\u2013Risk Myelodysplastic Syndromes","volume":"172","author":"Angelucci","year":"2020","journal-title":"Ann. Intern. Med."},{"key":"ref_16","doi-asserted-by":"crossref","first-page":"2134","DOI":"10.1200\/JCO.2010.34.1222","article-title":"Deferasirox Reduces Serum Ferritin and Labile Plasma Iron in RBC Transfusion\u2013Dependent Patients with Myelodysplastic Syndrome","volume":"30","author":"List","year":"2012","journal-title":"J. Clin. Oncol."},{"key":"ref_17","doi-asserted-by":"crossref","first-page":"858","DOI":"10.1002\/ajh.21269","article-title":"Consensus statement on iron overload in myelodysplastic syndromes","volume":"83","author":"Bennett","year":"2008","journal-title":"Am. J. Hematol."},{"key":"ref_18","doi-asserted-by":"crossref","first-page":"e2017021","DOI":"10.4084\/mjhid.2017.021","article-title":"Myelodysplastic syndromes and iron chelation therapy","volume":"9","author":"Angelucci","year":"2016","journal-title":"Mediterr. J. Hematol. Infect. Dis."},{"key":"ref_19","doi-asserted-by":"crossref","first-page":"267","DOI":"10.1111\/bjh.17612","article-title":"British Society for Haematology guidelines for the management of adult myelodysplastic syndromes","volume":"194","author":"Killick","year":"2021","journal-title":"Br. J. Haematol."},{"key":"ref_20","doi-asserted-by":"crossref","first-page":"54","DOI":"10.1016\/j.critrevonc.2019.06.002","article-title":"Hematologic improvement with iron chelation therapy in myelodysplastic syndromes: Clinical data, potential mechanisms, and outstanding questions","volume":"141","author":"Leitch","year":"2019","journal-title":"Crit. Rev. Oncol."},{"key":"ref_21","doi-asserted-by":"crossref","first-page":"1364","DOI":"10.3324\/haematol.2011.048546","article-title":"Hematologic responses to deferasirox therapy in transfusion-dependent patients with myelodysplastic syndromes","volume":"97","author":"Gattermann","year":"2012","journal-title":"Haematologica"},{"key":"ref_22","doi-asserted-by":"crossref","first-page":"527","DOI":"10.1111\/ejh.12300","article-title":"Deferasirox for transfusion-dependent patients with myelodysplastic syndromes: Safety, efficacy, and beyond (GIMEMA MDS0306 Trial)","volume":"92","author":"Angelucci","year":"2014","journal-title":"Eur. J. Haematol."},{"key":"ref_23","doi-asserted-by":"crossref","first-page":"52","DOI":"10.1111\/ejh.12476","article-title":"Deferasirox chelation therapy in patients with transfusion-dependent MDS: A \u2018real-world\u2019 report from two regional Italian registries: Gruppo Romano Mielodisplasie and Registro Basilicata","volume":"95","author":"Maurillo","year":"2015","journal-title":"Eur. J. Haematol."},{"key":"ref_24","doi-asserted-by":"crossref","first-page":"1067","DOI":"10.1016\/j.leukres.2012.04.006","article-title":"Improved survival in MDS patients receiving iron chelation therapy\u2014A matched pair analysis of 188 patients from the D\u00fcsseldorf MDS registry","volume":"36","author":"Neukirchen","year":"2012","journal-title":"Leuk. Res."}],"container-title":["Journal of Clinical Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/2077-0383\/12\/20\/6569\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,10]],"date-time":"2025-10-10T21:08:08Z","timestamp":1760130488000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/2077-0383\/12\/20\/6569"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,10,17]]},"references-count":24,"journal-issue":{"issue":"20","published-online":{"date-parts":[[2023,10]]}},"alternative-id":["jcm12206569"],"URL":"https:\/\/doi.org\/10.3390\/jcm12206569","relation":{},"ISSN":["2077-0383"],"issn-type":[{"type":"electronic","value":"2077-0383"}],"subject":[],"published":{"date-parts":[[2023,10,17]]}}}