{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,15]],"date-time":"2026-10-15T03:07:49Z","timestamp":1792033669986,"version":"4.4.0"},"reference-count":36,"publisher":"BMJ","issue":"8","license":[{"start":{"date-parts":[[2026,8,28]],"date-time":"2026-08-28T00:00:00Z","timestamp":1787875200000},"content-version":"unspecified","delay-in-days":27,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"name":"Faron Pharmaceuticals Ltd","award":["N\/A"],"award-info":[{"award-number":["N\/A"]}]}],"content-domain":{"domain":["bmj.com"],"crossmark-restriction":true},"short-container-title":["BMJ Open"],"accepted":{"date-parts":[[2026,8,12]]},"published-print":{"date-parts":[[2026,8]]},"abstract":"<jats:sec>\n                    <jats:title>Objectives<\/jats:title>\n                    <jats:p>To quantify the per-patient-per-month (PPPM) cost for each phase of care in higher-risk myelodysplastic syndromes (HR-MDS) and the overall cost of a base-case (illustrative) non-curative management pathway followed by a patient with HR-MDS in England.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design<\/jats:title>\n                    <jats:p>We conducted a retrospective, literature-based, micro-costing analysis from the National Health Service (NHS) England provider perspective using published sources and publicly available price lists. No individual-patient data were used. Based on literature, a base-case management pathway followed by a patient with HR-MDS was defined as a diagnostic work-up at month 0, 12 cycles of azacitidine (given for 7\u2009days in each 28-day cycle), 5 months of post-hypomethylating agent (HMA) failure supportive care and 1\u2009month of terminal care.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Setting<\/jats:title>\n                    <jats:p>Healthcare resource utilisation was analysed for adults with HR-MDS who received first-line azacitidine in routine practice if cycle-level or phase-level transfusion and admission rates were reported in the literature. Patients who required allogeneic haematopoietic stem cell transplantation or whose HR-MDS transformed to acute myeloid leukaemia were excluded because the diagnostic and\/or therapeutic pathways differ. Unit costs for 2024\/2025 were taken from published English national sources and an English trust tariff.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>PPPM costs were \u00a38721.58 during active azacitidine therapy, \u00a35399.58 after HMA failure and \u00a312 554.58 for hospital-dominant terminal care; a one-off diagnostic work-up with genomics cost \u00a32710 to \u00a32810. The total cost for the base-case management pathway was \u00a3146 921 per patient. An alternative pathway excluding genomic testing and assuming hospice-dominant terminal care reduced the total cost to approximately \u00a3136 840 to \u00a3140 990, depending on whether the lower or upper bound of hospice bed-day costs is applied.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>The direct NHS-provider cost burden of this non-curative HR-MDS management pathway is concentrated in azacitidine acquisition and administration during active treatment, transfusions and admissions after HMA failure and setting-dependent costs at the end of life. The total cost for the illustrative management pathway followed by a patient with HR-MDS (\u00a3146 921) is of a similar order to the estimate in the National Institute for Health and Care Excellence technology appraisal for azacitidine uprated to \u00a3124 848 for 2024\/2025. This comparison is provided for context only and should not be interpreted as validation of the present model, given differences in population, model structure, treatment duration and price year. The phase-specific PPPM estimates can inform, subject to local validation and scenario testing, UK budget-impact analyses, service planning and future economic models.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1136\/bmjopen-2026-119858","type":"journal-article","created":{"date-parts":[[2026,8,28]],"date-time":"2026-08-28T11:05:49Z","timestamp":1787915149000},"page":"e119858","update-policy":"https:\/\/doi.org\/10.1136\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":1,"title":["Health-system burden of higher-risk myelodysplastic syndromes in England: a literature-based micro-cost analysis"],"prefix":"10.1136","volume":"16","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8306-1559","authenticated-orcid":false,"given":"Joab","family":"Williamson","sequence":"first","affiliation":[{"name":"Faron Pharmaceuticals Oy, Turku, Southwest Finland, Finland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emma J","family":"Searle","sequence":"additional","affiliation":[{"name":"The Christie NHS Foundation Trust, Manchester, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elina","family":"Louramo","sequence":"additional","affiliation":[{"name":"Faron Pharmaceuticals Oy, Turku, Southwest Finland, Finland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ralph","family":"Hughes","sequence":"additional","affiliation":[{"name":"Faron Pharmaceuticals Oy, Turku, Southwest Finland, Finland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3963-0416","authenticated-orcid":false,"given":"Petri","family":"Bono","sequence":"additional","affiliation":[{"name":"Faron Pharmaceuticals Oy, Turku, Southwest Finland, Finland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0365-2605","authenticated-orcid":false,"given":"Vijay S","family":"Gc","sequence":"additional","affiliation":[{"name":"Centre for Health Economics, University of York, York, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"239","published-online":{"date-parts":[[2026,8,28]]},"reference":[{"key":"2026082804051576000_16.8.e119858.1","article-title":"Association between transfusion status and clinical and economic outcomes in patients with myelodysplastic syndromes from the physicians\u2019 perspective","volume":"6","author":"Gupta","year":"2023","journal-title":"Cancer Rep (Hoboken)"},{"key":"2026082804051576000_16.8.e119858.2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2012-03-420489"},{"key":"2026082804051576000_16.8.e119858.3","doi-asserted-by":"crossref","first-page":"4017","DOI":"10.2217\/fon-2022-0334","article-title":"Clinical impact of transformation to acute myeloid leukemia in patients with higher-risk myelodysplastic syndromes","volume":"18","author":"Kota","year":"2022","journal-title":"Future Oncol"},{"key":"2026082804051576000_16.8.e119858.4","unstructured":"NICE . Azacitidine for the treatment of myelodysplastic syndromes, chronic-myelomonocytic leukaemia and acute myeloidleukaemia. 2011. 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