Challenges in the management of patients with chronic myelomonocytic leukemia in the Brazilian public healthcare system: from diagnosis to transplantation
DOI:
https://doi.org/10.46765/2675-374X.2025v7n1e318Keywords:
Chronic myelomonocytic leukemia, Bone marrow transplantation, Hypomethylating agentsAbstract
Chronic myelomonocytic leukemia (CMML) represents a diagnostic and therapeutic challenge in the Brazilian public healthcare system, particularly due to limited access to advanced diagnostic tools and disease-modifying therapies. A retrospective chart review was conducted, including patients diagnosed with CMML from 2015 to 2025 at a university hospital in northeastern Brazil. Fourteen patients were identified, with a mean age of 58.7 years, and 50% were male. At diagnosis, 57.1% were in the chronic phase and 42.9% in the acute phase. Only two patients (14.3%) received hypomethylating agents, and four (28.6%) underwent allogeneic hematopoietic stem-cell transplantation. The median overall survival for the cohort was 25 months. Patients diagnosed in the acute phase had a shorter median overall survival compared to those diagnosed in the chronic phase (8 vs. 25 months). A trend toward improved survival was observed among transplanted patients (25 vs. 8 months). Compared with contemporary data, patients in this cohort had lower exposure to hypomethylating therapy, although transplant-related characteristics and early engraftment outcomes were similar. Structural and therapeutic limitations within the public healthcare system may impact disease management and outcomes, reinforcing the importance of improving access to diagnostic resources, disease-modifying therapies, and timely referral for transplantation.
References
1. Castaño-Díez S, López-Guerra M, Bosch-Castañeda C, Bataller A, Charry P, Esteban D, et al. Real-world data on chronic myelomonocytic leukemia: clinical and molecular characteristics, treatment, emerging drugs, and patient outcomes. Cancers (Basel). 2022;14(17):4107. https://doi.org/10.3390/cancers14174107 DOI: https://doi.org/10.3390/cancers14174107
2. Patnaik MM, Tefferi A. Chronic myelomonocytic leukemia: 2024 update on diagnosis, risk stratification, and management. Am J Hematol. 2024;99(6):1142-65. https://doi.org/10.1002/ajh.27271 DOI: https://doi.org/10.1002/ajh.27271
3. Marando L, Csizmar CM, Patnaik MM. Chronic myelomonocytic leukemia: molecular pathogenesis and therapeutic innovations. Haematologica. 2025;110(1):22-36. https://doi.org/10.3324/haematol.2024.286061 DOI: https://doi.org/10.3324/haematol.2024.286061
4. Patnaik MM. How I diagnose and treat chronic myelomonocytic leukemia. Haematologica. 2022; 107(7): 1503-17. https://doi.org/10.3324/haematol.2021.279500 DOI: https://doi.org/10.3324/haematol.2021.279500
5. Kwon J. Diagnosis and treatment of chronic myelomonocytic leukemia. Blood Res. 2021;56(S1):S5-S16. https://doi.org/10.5045/br.2021.2020321 DOI: https://doi.org/10.5045/br.2021.2020321
6. Elena C, Galli A, Such E, Germing U, Rissling O, Schuler E, et al. Integrating clinical features and genetic lesions in the risk assessment of patients with chronic myelomonocytic leukemia. Blood. 2016;128(10):1408-1417. https://doi.org/10.1182/blood-2016-05-714030 DOI: https://doi.org/10.1182/blood-2016-05-714030
7. Selimoglu-Buet D, Wagner-Ballon O, Saada V, Bardet V, Itzykson R, Bencheikh L, et al. Characteristic repartition of monocyte subsets as a diagnostic signature of chronic myelomonocytic leukemia. Blood. 2015;125(23):3618-26. https://doi.org/10.1182/blood-2015-01-620781 DOI: https://doi.org/10.1182/blood-2015-01-620781
8. Alfonso A, Montalban-Bravo G, Takahashi K, Patel K, Sasaki K, Bueso-Ramos C, et al. Natural history of chronic myelomonocytic leukemia treated with hypomethylating agents. Am J Hematol. 2017;92(7): 599-606. https://doi.org/10.1002/ajh.24735 DOI: https://doi.org/10.1002/ajh.24735
9. lsugair A, Gauto Mariotti E, Alhousani MM, Patnaik MM, Lasho TL, Finke CM, et al. Allogeneic stem cell transplantation in chronic myelomonocytic leukemia: analysis of post-transplant survival and risk factors in 138 Mayo Clinic patients. Blood Cancer J. 2025;15(1):157. https://doi.org/10.1038/s41408-025-01359-w DOI: https://doi.org/10.1038/s41408-025-01359-w
10. Duarte FB, Moura ATG, Funke VAM, Setubal DC, Bonfim CMS, Vigorito AC, et al. Impact of treatment prior to allogeneic transplantation of hematopoietic stem cells in patients with myelodysplastic syndrome: results of the Latin American Bone Marrow Transplant Registry. Biol Blood Marrow Transplant. 2020;26(5):1021-4. https://doi.org/10.1016/j.bbmt.2020.01.030 DOI: https://doi.org/10.1016/j.bbmt.2020.01.030
11. Pophali P, Matin A, Mangaonkar AA, Lasho TL, Finke CM, Gangat N, et al. Prognostic impact and timing considerations for allogeneic hematopoietic stem cell transplantation in chronic myelomonocytic leukemia. Blood Cancer J. 2020;10(11):121. https://doi.org/10.1038/s41408-020-00387-y DOI: https://doi.org/10.1038/s41408-020-00387-y
12. Wedge E, Hansen JW, Dybedal I, Antunovic P, Brinch L, Gedde-Dahl T, et al. Allogeneic hematopoietic stem cell transplantation for chronic myelomonocytic leukemia: clinical and molecular genetic prognostic factors in a Nordic population. Transplant Cell Ther. 2021;27(12):991.e1-991.e9. https://doi.org/10.1016/j.jtct.2021.08.028 DOI: https://doi.org/10.1016/j.jtct.2021.08.028
13. Mei M, Pillai R, Kim S, Lasho TL, Finke CM, Patnaik MM, et al. The mutational landscape in chronic myelomonocytic leukemia and its impact on allogeneic hematopoietic cell transplantation outcomes: a Center for Blood and Marrow Transplantation Research (CIBMTR) analysis. Haematologica. 2023;108(1): 150-60. https://doi.org/10.3324/haematol.2021.280203 DOI: https://doi.org/10.3324/haematol.2021.280203
14. Robin M, de Wreede LC, Padron E, Itzykson R, Fenaux P, Platzbecker U, et al. Role of allogeneic transplantation in chronic myelomonocytic leukemia: an international collaborative analysis. Blood. 2022;140(12): 1408-18. https://doi.org/10.1182/blood.2021015173 DOI: https://doi.org/10.1182/blood.2021015173
15. Onida F, Gagelmann N, Chalandon Y, Kobbe G, Robin M, Symeonidis A, et al. Management of adult patients with CMML undergoing allo-HCT: recommendations from the EBMT PH&G Committee. Blood. 2024;143(22):2227-44. https://doi.org/10.1182/blood.2023023476 DOI: https://doi.org/10.1182/blood.2023023476
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Copyright (c) 2026 Emanuelly Thays Muniz Figueiredo Silva Vasconcelos, Felipe de Menezes Cunha, Gustavo Mesquita de Oliveira, Maria Laura Fontoura Chagas Rocha, Hércules Amorim Mota Segundo, Fernando Barroso Duarte
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