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. 2025 Aug 1;110(8):1798-1807.
doi: 10.3324/haematol.2024.287177. Epub 2025 Feb 27.

Maintenance therapy with oral decitabine plus cedazuridine after allogeneic stem cell transplantation for myelodysplastic syndrome

Portia Smallbone  1 , Terri Lynn Shigle  2 , Oren Paslovsky  3 , Chitra Hosing  1 , Amin Alousi  1 , Qaiser Bashir  1 , Yosra Aljawai  1 , Jeremy Ramdial  1 , Uday Popat  1 , Richard Champlin  1 , Elizabeth J Shpall  4 , Betül Oran  5
Affiliations

Maintenance therapy with oral decitabine plus cedazuridine after allogeneic stem cell transplantation for myelodysplastic syndrome

Portia Smallbone et al. Haematologica. .

Abstract

Disease relapse remains the primary challenge for patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT) for myelodysplastic syndromes. Maintenance therapies with hypomethylating agents are under investigation for use in mitigating relapse in high-risk patients. In this retrospective study, we assessed the safety and efficacy of oral decitabine- cedazuridine maintenance in 18 high-risk myelodysplastic syndromes patients post-HSCT. A total of 66.7% (N=12) received decitabine/cedazuridine (35/100 mg) on days 1 and 3, while 33.3% (N=6) received therapy on days 1-3. Patients completed a median of six treatment cycles (range, 1-20), with one third of patients completing all planned cycles. No unexpected adverse events were observed, with the primary toxicity being myelosuppression. Grade 1-2 upper respiratory tract infections occurred in four patients, and fungal pneumonia in one patient. Overall, patients achieved a median 2-year relapse- free survival of 66.7% (95% confidence interval [CI]: 40.4-83.4) and 2-year overall survival of 72.2% (95% CI: 45.6-87.4), with relapses occurring predominantly in TP53-mutated cases. Prospective clinical trials are essential to confirm the best tolerated dose with potential to improve transplant outcomes.

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Figures

Figure 1.
Figure 1.
Peripheral blood counts for patients on cycle 1 of decitabine/cedazuridine maintenance. Counts on days 1, 8, 15, 22, 29, 36 of cycle 1 have been presented. (A) Median absolute neutrophil count (K/µL). (B) Median platelet counts (K/µL). The bars in all plots represent median values with error bars representing the interquartile range between the 25% and 75% quartiles. D: day.
Figure 2.
Figure 2.
Swimmer plot for the cohort (N=18), including time to death or last known alive. Each row identifies a unique patient received maintenance with decatibine and cedazudirine. ▲: initiation of maintenance treatment; •: disease relapse after transplant; ■ : death; →: continues to be alive.
Figure 3.
Figure 3.
Kaplan-Meier estimate of progression-free survival and overall survival. (A) Progression-free survival. (B) Overall survival.
See this image and copyright information in PMC

References

    1. Lim Z, Brand R, Martino R, et al. Allogeneic hematopoietic stem-cell transplantation for patients 50 years or older with myelodysplastic syndromes or secondary acute myeloid leukemia. J Clin Oncol. 2010;28(3):405-411. - PubMed
    1. McClune BL, Weisdorf DJ, Pedersen TL, et al. Effect of age on outcome of reduced-intensity hematopoietic cell transplantation for older patients with acute myeloid leukemia in first complete remission or with myelodysplastic syndrome. J Clin Oncol. 2010;28(11):1878-1887. - PMC - PubMed
    1. Mina A, Greenberg PL, Deeg HJ. How I reduce and treat posttransplant relapse of MDS. Blood. 2024;143(14):1344-1354. - PMC - PubMed
    1. Bewersdorf JP, Tallman MS, Cho C, Zeidan AM, Stahl M. Safety and efficacy of maintenance treatment following allogeneic hematopoietic cell transplant in acute myeloid leukemia and myelodysplastic syndrome - a systematic review and meta-analysis. Blood. 2020;136(Suppl 1):34-35.
    1. Kungwankiattichai S, Ponvilawan B, Roy C, Tunsing P, Kuchenbauer F, Owattanapanich W. Maintenance with hypomethylating agents after allogeneic stem cell transplantation in acute myeloid leukemia and myelodysplastic syndrome: a systematic review and meta-analysis. Front Med (Lausanne). 2022:9:801632. - PMC - PubMed

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