High-risk Mds and Cmml: the Gap Between Identifying Risk and Accessing Disease-modifying Therapy
Year: 2026
Type: article
Study design: Observational Study
Abstract: Background In patients with high-risk MDS and CMML, only allogeneic hematopoietic stem cell transplantation (allo-HCT) offers curative potential, whereas hypomethylating agent-based therapies may prolong survival. In public health systems, access to these therapies is constrained by clinical and structural barriers that are rarely quantified. Aim To describe access to azacitidine and allo-HCT among high-risk patients across three five-year periods and to assess whether this access changed over time. Materials and methods Retrospective, single-center study conducted at a Brazilian tertiary hospital, including patients with high-risk MDS and CMML diagnosed between 2010 and 2025, drawn from the institutional Myelodysplastic Syndromes Registry database in REDCap. High risk was defined as MDS with an IPSS-R greater than 3.5 and CMML with a CPSS of 2 or higher. The proportions of azacitidine and HSCT use were compared across the five-year periods (2010-2014, 2015-2019, and 2020-2025) using the chi-square test, in R software (version 4.5.3). The study was approved by the institutional ethics committee. Results Of the 427 patients in the cohort, 194 were classified as high risk (55 in 2010-2014, 74 in 2015-2019, and 65 in 2020-2025). Azacitidine use reached 35% of the high-risk group and increased from 22% in the first five-year period to 52% in the last (p=0.001). Allo-HCT was performed in 12% of high-risk patients and in 8.5% of the overall cohort, with no difference across the five-year periods (15%, 16%, and 6.3%; p=0.2). Among high-risk patients eligible for allo-HCT under the Brazilian public health system (SUS; those younger than 65 years), 19 of 70 (27%) underwent transplantation. The median age of transplanted patients was 49 years. Discussion and Conclusion We describe access to disease-modifying therapies (azacitidine and allo-HCT) in patients with high-risk MDS and CMML at a large Brazilian tertiary center over a 16-year period. Access to hypomethylating agents expanded markedly: in the South American series by Belli et al. (2015), 7.5% of Brazilian patients (all risk categories) received a hypomethylating agent, compared with 20% in our overall cohort and 35% in the high-risk subgroup. In contrast, allo-HCT - the only curative option - remained limited (12% of high-risk patients), with no increase over time; the 8.5% rate in the overall cohort is similar to the 6.4% reported by Belli et al. (2015) in Brazil. When restricted to high-risk patients aged 65 years or younger, eligible under the SUS, the rate rose to 27%, showing that age and clinical eligibility continue to limit the procedure. This contrast reveals a concrete care gap between identifying high risk and delivering the therapy capable of altering its course, and highlights the need for strategies that broaden eligibility for and referral to transplantation in our setting.
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: diamond
OpenAlex ID: W7223115862