HIGH-RISK MDS AND CMML: THE GAP BETWEEN IDENTIFYING RISK AND ACCESSING DISEASE-MODIFYING THERAPY
- 1Hospital das Clínicas, Faculdade de Medicina, Universidade de São Paulo (HCFMUSP), São Paulo, SP, Brazil
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.
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BibTeX
@article{marques2026highrisk,
title = {{HIGH-RISK MDS AND CMML: THE GAP BETWEEN IDENTIFYING RISK AND ACCESSING DISEASE-MODIFYING THERAPY}},
author = {Maria Eduarda Lisboa MARQUES and André Góis ARRUDA and Ana Beatriz Araújo SOUZA and Balduíno BIASOLI and Joaquim Gasparini SANTOS and Thales Dalessandro Meneguin PEREIRA and Valéria BUCCHERI and Vanderson ROCHA and Elvira Deolinda Rodrigues Pereira VELLOSO},
journal = {Hematology, Transfusion and Cell Therapy},
year = {2026},
month = oct,
doi = {10.1016/j.htct.2026.107955},
url = {https://doi.org/10.1016/j.htct.2026.107955},
note = {CC BY},
}
RIS
TY - JOUR
TI - HIGH-RISK MDS AND CMML: THE GAP BETWEEN IDENTIFYING RISK AND ACCESSING DISEASE-MODIFYING THERAPY
AU - Maria Eduarda Lisboa MARQUES
AU - André Góis ARRUDA
AU - Ana Beatriz Araújo SOUZA
AU - Balduíno BIASOLI
AU - Joaquim Gasparini SANTOS
AU - Thales Dalessandro Meneguin PEREIRA
AU - Valéria BUCCHERI
AU - Vanderson ROCHA
AU - Elvira Deolinda Rodrigues Pereira VELLOSO
PY - 2026
DA - 2026/10/23
JO - Hematology, Transfusion and Cell Therapy
DO - 10.1016/j.htct.2026.107955
UR - https://doi.org/10.1016/j.htct.2026.107955
AB - 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.
SN - 2531-1379
SN - 2531-1387
ER -