Hematopoietic cell transplantation for myelodysplastic syndromes
DOI:
https://doi.org/10.46765/2675-374X.2025v7n1e350Keywords:
Myelodysplastic syndromes, Hematopoietic stem cell transplantation, Hematology, Diagnosis, Classification, TherapeuticsAbstract
For patients with myelodysplastic syndromes (MDS), allogeneic hematopoietic cell transplantation (HCT) is considered the only treatment with curative potential. However, its use is restricted to a selected group of patients, and transplant outcomes are influenced by a complex interaction of disease related characteristics, patient-specific factors, and the HCT procedure. Since the publication of the previous Brazilian consensus, important advances have been made in the classification and risk stratification of MDS. In 2022, two new classification systems have been updated: the World Health Organization classification and the International Consensus Classification. In addition, the incorporation of molecular information through the Molecular International Prognostic Scoring System has further refined prognostic assessment and therapeutic decision making in patients with MDS. This article updates the Brazilian consensus published in 2021 for Brazilian Society of Cellular Therapy and Bone Marrow Transplantation, incorporating recent developments in the management of patients undergoing HCT. It aims to guide hematologists in transplant indication.
References
1. Gurnari C, Robin M, Adès L, Aljurf M, Almeida A, Duarte FB, Bernard E, Cutler C, Porta MGD, Witte T, DeZern A, Drozd-Sokolowska J, Duncavage E, Fenaux P, Gagelmann N, Garcia-Manero G, Haferlach C, Haferlach T, Hasserjian R, Hellström-Lindberg E, Jacoby M, Kulasekararaj A, Lindsley RC, Maciejewski JP, Makishima H, Malcovati L, Mittelman M, Myhre AE, Ogawa S, Onida F, Papaemmanuil E, Passweg J, Platzbecker U, Pleyer L, Raj K, Santini V, Sureda A, Tobiasson M, Voso MT, Yakoub-Agha I, Zeidan A, Walter M, Kröger N, McLornan DP, Cazzola M. Clinical-genomic profiling of MDS to inform allo-HCT: recommendations from an international panel on behalf of the EBMT. Blood. 2025;145(18):1987-2001. https://doi.org/10.1182/blood.2024025131 DOI: https://doi.org/10.1182/blood.2024025131
2. Stempel JM, Kewan T, Zeidan AM. Advances and challenges in the management of myelodysplastic syndromes. Cancers. 2025;17(15):2469. https://doi.org/10.3390/cancers17152469 DOI: https://doi.org/10.3390/cancers17152469
3. Bazinet A, Bataller A, Chien K, Sasaki K, Montalban-Bravo G, Hammond D, Bouligny I, Swaminathan M, Jen WY, Kugler E, Kanagal-Shamanna R, Kadia T, Borthakur G, DiNardo C, Short N, Pemmaraju N, Jabbour E, Daver N, Ravandi F, Urrutia S, Gener-Ricos G, Braish J, Briski R, Rodriguez-Sevilla JJ, Li Z, Champlin RE, Shpall EJ, Alousi A, Oran B, Hosing C, Ramdial JL, Olson A, Pierce S, Chen J, Kantarjian H, Garcia-Manero G, Popat U. Impact of allogeneic stem cell transplantation in patients with higher risk myelodysplastic syndromes. Blood Cancer J. 2026;16(1):48. https://doi.org/10.1038/s41408-026-01479-x DOI: https://doi.org/10.1038/s41408-026-01479-x
4. Rathje K, Kröger N. Optimal timing of allogeneic hematopoietic stem cell transplant in MDS. Leuk Lymphoma. 2025;66(10):1788-800. https://doi.org/10.1080/10428194.2025.2514662 DOI: https://doi.org/10.1080/10428194.2025.2514662
5. Simione AJ, Silva CC, Vigorito AC, Macedo AV, Neves HRA, Sabaini PMS, Ammi M, Costa FF, Cavilha AMQ, Concilio RR, Colturato VAR, Scheinberg P, Nabhan SK, Lerner D, Hamerschlak N, Colella MP, Barros GMN, Silvério A, Seber A, Ferreira SO, Novis YAS, Gasparini J, Rocha VG, Moreira MCR, Astigarraga CC, Daudt LE, Macedo MCMA, Chiattone R, Fernandes JF, Vilela VAL, Soares RDA, Teixeira GM, Arrais Rodrigues C, Silva RL, Funke VAM, Arcuri LJ, Schmidt Filho J, Molla VC, Farias JSH, Pasquini R, Bonfim CMS, Hallack Neto AE, Calixto RF, Feliciano JVP, Gaiolla RD, Capra M, Atalla A, Aranha MAF, Schaffel R, Veloso GDC, Bettarello G, Melo AL, Franco SCR, Aduan MA, Flowers ME, Pasquini MC, Duarte FB. Current use and outcomes of hematopoietic cell transplantation: Brazilian summary slides – 2025. J Bone Marrow Transplant. 2025;6(1):259. https://doi.org/10.46765/2675-374X.2025v6n1p259 DOI: https://doi.org/10.46765/2675-374X.2025v6n1p270
6. Khoury JD, Solary E, Abla O, Akkari Y, Alaggio R, Apperley JF, Bejar R, Berti E, Busque L, Chan JKC, Chen W, Chen X, Chng WJ, Choi JK, Colmenero I, Coupland SE, Cross NCP, De Jong D, Elghetany MT, Takahashi E, Emile JF, Ferry J, Fogelstrand L, Fontenay M, Germing U, Gujral S, Haferlach T, Harrison C, Hodge JC, Hu S, Jansen JH, Kanagal-Shamanna R, Kantarjian HM, Kratz CP, Li XQ, Lim MS, Loeb K, Loghavi S, Marcogliese A, Meshinchi S, Michaels P, Naresh KN, Natkunam Y, Nejati R, Ott G, Padron E, Patel KP, Patkar N, Picarsic J, Platzbecker U, Roberts I, Schuh A, Sewell W, Siebert R, Tembhare P, Tyner J, Verstovsek S, Wang W, Wood B, Xiao W, Yeung C, Hochhaus A. The 5th edition of the World Health Organization Classification of Haematolymphoid Tumours: Myeloid and Histiocytic/Dendritic Neoplasms. Leukemia. 2022;36(7):1703-19. https://doi.org/10.1038/s41375-022-01613-1 DOI: https://doi.org/10.1038/s41375-022-01613-1
7. Arber DA, Orazi A, Hasserjian RP, Borowitz MJ, Calvo KR, Kvasnicka HM, Wang SA, Bagg A, Barbui T, Branford S, Bueso-Ramos CE, Cortes JE, Dal Cin P, DiNardo CD, Dombret H, Duncavage EJ, Ebert BL, Estey EH, Facchetti F, Foucar K, Gangat N, Gianelli U, Godley LA, Gökbuget N, Gotlib J, Hellström-Lindberg E, Hobbs GS, Hoffman R, Jabbour EJ, Kiladjian JJ, Larson RA, Le Beau MM, Loh ML, Löwenberg B, Macintyre E, Malcovati L, Mullighan CG, Niemeyer C, Odenike OM, Ogawa S, Orfao A, Papaemmanuil E, Passamonti F, Porkka K, Pui CH, Radich JP, Reiter A, Rozman M, Rudelius M, Savona MR, Schiffer CA, Schmitt-Graeff A, Shimamura A, Sierra J, Stock WA, Stone RM, Tallman MS, Thiele J, Tien HF, Tzankov A, Vannucchi AM, Vyas P, Wei AH, Weinberg OK, Wierzbowska A, Cazzola M, Döhner H, Tefferi A. International Consensus Classification of myeloid neoplasms and acute leukemias: integrating morphologic, clinical, and genomic data. Blood. 2022;140(11):1200-28. https://doi.org/10.1182/blood.2022015850 DOI: https://doi.org/10.1182/blood.2022015850
8. Bernard E, Tuechler H, Greenberg PL, Hasserjian RP, Arango Ossa JE, Nannya Y, Devlin SM, Creignou M, Pinel P, Monnier L, Gundem G, Medina-Martinez JS, Domenico D, Jädersten M, Germing U, Sanz G, van de Loosdrecht AA, Kosmider O, Follo MY, Thol F, Zamora L, Pinheiro RF, Pellagatti A, Elias HK, Haase D, Ganster C, Ades L, Tobiasson M, Palomo L, Della Porta MG, Takaori-Kondo A, Ishikawa T, Chiba S, Kasahara S, Miyazaki Y, Viale A, Huberman K, Fenaux P, Belickova M, Savona MR, Klimek VM, Santos FPS, Boultwood J, Kotsianidis I, Santini V, Solé F, Platzbecker U, Heuser M, Valent P, Ohyashiki K, Finelli C, Voso MT, Shih LY, Fontenay M, Jansen JH, Cervera J, Gattermann N, Ebert BL, Bejar R, Malcovati L, Cazzola M, Ogawa S, Hellström-Lindberg E, Papaemmanuil E. Molecular international prognostic scoring system for myelodysplastic syndromes. NEJM Evidence. 2022;1(7):EVIDoa2200008. https://doi.org/10.1056/evidoa2200008 DOI: https://doi.org/10.1056/EVIDoa2200008
9. Duarte FB, Barroso KSN, Silva RL, Gurgel LA, Leitão JPV, Pitombeira BSGS, Soares RDA, Pádua L, Betarello G. HCT for myelodysplastic syndromes (MDS). J Bone Marrow Transplant Cell Ther. 2021;2(1):120-6. https://doi.org/10.46765/2675-374X.2021v4n1p120-126 DOI: https://doi.org/10.46765/2675-374X.2021v4n1p120-126
10. Hasserjian RP, Germing U, Malcovati L. Diagnóstico e classificação das síndromes mielodisplásicas. Blood. 2023;142(26):2247-57. https://doi.org/10.1182/blood.2023020078 DOI: https://doi.org/10.1182/blood.2023020078
11. Duarte F, Gurnari C, McLornan D. Myelodysplastic syndromes versus acute myeloid leukaemia: biology or blasts—what truly defines the disease and does it matter? Lancet Haematol. 2026;13(3):e181-5. https://doi.org/10.1016/s2352-3026(25)00359-x DOI: https://doi.org/10.1016/S2352-3026(25)00359-X
12. Kröger N. Treatment of high risk myelodysplastic syndrome. Haematologica. 2024;110(2):339-49. https://doi.org/10.3324/haematol.2023.284946 DOI: https://doi.org/10.3324/haematol.2023.284946
13. Cazzola M, Malcovati L. Genome sequencing in the management of myelodysplastic syndromes and related disorders. Haematologica. 2025;110(2):312-29. https://doi.org/10.3324/haematol.2023.284947 DOI: https://doi.org/10.3324/haematol.2023.284947
14. Sauta E, Robin M, Bersanelli M, Travaglino E, Meggendorfer M, Zhao LP, Caballero Berrocal JC, Sala C, Maggioni G, Bernardi M, Di Grazia C, Vago L, Rivoli G, Borin L, D’Amico S, Tentori CA, Ubezio M, Campagna A, Russo A, Mannina D, Lanino L, Chiusolo P, Giaccone L, Voso MT, Riva M, Oliva EN, Zampini M, Riva E, Nibourel O, Bicchieri M, Bolli N, Rambaldi A, Passamonti F, Savevski V, Santoro A, Germing U, Kordasti S, Santini V, Diez-Campelo M, Sanz G, Sole F, Kern W, Platzbecker U, Ades L, Fenaux P, Haferlach T, Castellani G, Della Porta MG. Real-world validation of molecular international prognostic scoring system for myelodysplastic syndromes. J Clin Oncol. 2023;41(15):2827-42. https://doi.org/10.1200/jco.22.01784 DOI: https://doi.org/10.1200/JCO.22.01784
15. Garcia-Manero G. Myelodysplastic syndromes: 2023 update on diagnosis, risk-stratification, and management. Am J Hematol. 2023;98(8):1307-25. https://doi.org/10.1002/ajh.26984 DOI: https://doi.org/10.1002/ajh.26984
16. Galanopoulos AG, Papi C. Novel therapies in high-risk myelodysplastic syndromes. Eur J Haematol. 2026;116(6):744-58. https://doi.org/10.1111/ejh.70146 DOI: https://doi.org/10.1111/ejh.70146
17. Gagelmann N, Kröger N. Allogeneic stem cell transplant for myelofibrosis and myelodysplastic syndromes: a contemporary review. Am J Hematol. 2025;100(Suppl. 4):16-29. https://doi.org/10.1002/ajh.27660 DOI: https://doi.org/10.1002/ajh.27660
18. Duarte FB, Garcia YDO, Hamerschlak N, Funke VAM, Moreira MCR, Paz AA, Filho JS, Astigarraga CC, da Silva RL, de Molla VC, Silvério A, Rocha VG, Feliciano JVP, Barros GMN, Colturato VAR, Nabhan SK, Farias JSH, Maia ACA, Atalla Â, Chiattone R, Macedo MCMA, Aranha MAF, Zogbi YAN, Lener D, Soares RDA, Scheinberg P, Calixto RF, Teixeira GM, Colella MP, Rodrigues CA, Simione AJ, da Silva CC, Martin PJ, Flowers ME. Allogeneic hematopoietic cell transplantation in elderly patients in a Latin American country: analysis of 11 years of data from the Brazilian Registry SBTMO/CIBMTR. Transplant Cell Ther. 2025;31(2):79.e1-e9. https://doi.org/10.1016/j.jtct.2024.12.003 DOI: https://doi.org/10.1016/j.jtct.2024.12.003
19. Kanate AS, Perales MA, Hamadani M. Eligibility criteria for patients undergoing allogeneic hematopoietic cell transplantation. J Natl Compr Canc Netw. 2020;18(5):635-43. https://doi.org/10.6004/jnccn.2020.7559 DOI: https://doi.org/10.6004/jnccn.2020.7559
20. Duarte FB, Garcia YDO, Funke VAM, João A, Hamerschlak N, Villela NC, Macedo MCMA, Vigorito AC, Soares RDA, Paz AA, Stevenazzi M, Diaz L, Hallack Neto AE, Bettarello G, Gusmão BM, Salvino MA, Calixto RF, Moreira MCR, Teixeira GM, Silva CC, Paton EJA, Rocha VG, Enrico A, Bonfim CMS, Chiattone R, Arrais-Rodrigues C, Coelho EOM, Iastrebner M, Duarte BA, Colturato VAR. Outcomes after allogeneic hematopoietic cell transplantation in adults with myelodysplastic syndrome with 65 years or older compared to youngers. A retrospective analysis of the Latin America Registry. Eur J Haematol. 2025;115(4):349-57. https://doi.org/10.1111/ejh.70001 DOI: https://doi.org/10.1111/ejh.70001
21. Artz AS, Logan B, Saber W, Geller N, Bellach A, Kou J, Wood WA, McCarty JM, Knight TG, Runaas L, Johnston L, Walston J, Nakamura R, Mishra A, Uberti J, Dahi PB, Saultz JN, McCurdy SR, Morris L, Imus P, Hogan WJ, Nadiminti K, Bhatt VR, Olin R, Maakaron J, Sobecks R, Wall S, Mattila D, Protz B, Devine SM, Horowitz MM, Sorror ML. CHARM is prognostic of geriatric morbidity and toxicity after allogeneic transplant for older adults: BMT CTN 1704 study. Blood Adv. 2026;10(5):1657-69. https://doi.org/10.1182/bloodadvances.2025018340 DOI: https://doi.org/10.1182/bloodadvances.2025018340
22. Kanate AS, Majhail NS, Savani BN, Bredeson C, Champlin RE, Crawford S, Giralt SA, LeMaistre CF, Marks DI, Omel JL, Orchard PJ, Palmer J, Saber W, Veys PA, Carpenter PA, Hamadani M. Indications for hematopoietic cell transplantation and immune effector cell therapy: guidelines from the American Society for Transplantation and Cellular Therapy. Biol Blood Marrow Transplant. 2020;26(7):1247-56. https://doi.org/10.1016/j.bbmt.2020.03.002 DOI: https://doi.org/10.1016/j.bbmt.2020.03.002
23. Hellström-Lindberg E, Kröger N. Clinical decision-making and treatment of myelodysplastic syndromes. Blood. 2023;142(26):2268-81. https://doi.org/10.1182/blood.2023020079 DOI: https://doi.org/10.1182/blood.2023020079
24. Jimenez Jimenez AM, Spellman SR, Politikos I, McCurdy SR, Devine SM, Malki MMA, Bolon YT, Lee SJ, Dehn J, Pidala J, Maiers M, Askar M, Malmberg C, Auletta JJ, Stefanski H, Broglie L, Qayed M, Horwitz M, Wilder JS, Gooptu M, Mehta RS, Fernandez-Viña M, Shaw BE, Shaffer BC. Allogeneic hematopoietic cell donor selection: contemporary guidelines from the NMDP/CIBMTR. Transplant Cell Ther. 2025;31(12):973-88. https://doi.org/10.1016/j.jtct.2025.07.004 DOI: https://doi.org/10.1016/j.jtct.2025.07.004
25. Mina A, Greenberg PL, Deeg HJ. How I reduce and treat posttransplant relapse of MDS. Blood. 2024;143(14):1344-54. https://doi.org/10.1182/blood.2023023005 DOI: https://doi.org/10.1182/blood.2023023005
26. Stahl M, Zeidan AM. The conundrum of drug development in higher-risk MDS: lessons learned from recently failed phase 3 clinical trials. Blood. 2026;147(8):811-20. https://doi.org/10.1182/blood.2025029727 DOI: https://doi.org/10.1182/blood.2025029727
27. Garcia-Manero G, Platzbecker U, Fenaux P, Roboz GJ, Fong CY, Hus M, Delage R, Brunner A, Lee JH, Turgut M, Mazure D, Feng YH, Miyazaki Y, Korístek Z, Dimicoli-Salazar S, Campelo MD, Lavie D, Culligan D, Ku G, Xiao Z, Fang F, Headley M, Potluri J, Zeidan A, Garcia J. MDS-1497: primary analysis of the randomized, phase 3 VERONA Study of venetoclax with azacitidine versus placebo with azacitidine in patients with treatment-naïve, intermediate and higher-risk myelodysplastic syndromes. Clin Lymphoma Myeloma Leuk. 2025;25(Suppl. 1):S644-5. https://doi.org/10.1016/S2152-2650(25)02083-X DOI: https://doi.org/10.1016/S2152-2650(25)02083-X
28. Robin M, D’Aveni M, Stamatoullas A, Raffoux E, Chevallier P, Garnier A, Mediavilla C, Carre M, Himberlin C, Sébert M, Ravinet A, Desseaux K, Labussière H, Alani M, Rubio MT, Huynh A, Adès L, de Latour RP, Paul F, Chermat F, Petit R, Mokeddem C, Charbonnier A, Thépot S, Chevret S, Fenaux P; Société Francophone de Greffe de Moelle et de Thérapie Cellulaire (SFGM-TC) and the Groupe Francophone des Myélodysplasies (GFM). Oral decitabine and cedazuridine maintenance after haematopoietic stem-cell transplantation in very high-risk acute myeloid leukaemia or myelodysplastic syndrome (GFM-DACORAL-DLI): a multicentre, singlearm, phase 2 trial. Lancet Haematol. 2025;12(9):e705-16. https://doi.org/10.1016/s2352-3026(25)00172-3 DOI: https://doi.org/10.1016/S2352-3026(25)00172-3
29. Zawacka JE. Biologia e reativação do p53 para terapia aprimorada em SMD e LMA. Biomark Res. 2024;12:34. https://doi.org/10.1186/s40364-024-00579-9 DOI: https://doi.org/10.1186/s40364-024-00579-9
Downloads
Published
How to Cite
Issue
Section
License
Copyright (c) 2026 Fernando Barroso Barroso, Yhasmine Delles Oliveira Garcia, Karine Sampaio Nunes Barroso, Roberto Luiz da Silva, Lívia Andrade Gurgel, João Paulo de Vasconcelos Leitão, Beatriz Stela Gomes de Souza Pitombeira, Rodolfo Daniel de Almeida Soares, Gustavo Bettarello
This work is licensed under a Creative Commons Attribution 4.0 International License.
