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Case Report Open Access

Decitabine monotherapy for pediatric myelodysplastic syndrome: A case report

  • Authors:
    • Zhimin Fan
    • Nengwen Xu
    • Alicia Senin
    • Junyu Zhang
  • View Affiliations / Copyright

    Affiliations: Department of Hematology, Lishui Central Hospital, Lishui, Zhejiang 323400, P.R. China, Hospital del Mar Medical Research Institute, Hospital del Mar, Barcelona 08003, Spain
    Copyright: © Fan et al. This is an open access article distributed under the terms of Creative Commons Attribution License.
  • Article Number: 172
    |
    Published online on: April 23, 2026
       https://doi.org/10.3892/etm.2026.13167
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Abstract

The occurrence of myelodysplastic syndrome (MDS) in children is infrequently documented and most commonly manifests in the form of refractory cytopenia of childhood (RCC). The management of pediatric MDS remains controversial and involves primarily chemotherapy, immunosuppressive therapy (IST) and allogeneic hematopoietic stem cell transplantation (HSCT). However, therapeutic data for patients with RCC who are ineligible for either HSCT or IST are scarce. In the present case, the 8‑year‑old patient achieved disease stabilization following decitabine monotherapy, thereby suggesting a potential novel therapeutic avenue for children with low‑risk chronic MDS or RCC.
View Figures

Figure 1

(A) BM aspirate demonstrating small
megakaryocytes (Wright-Giemsa stain; x400 magnification). (B and C)
Dysplastic neutrophils exhibiting nuclear hypolobation and abnormal
chromatin condensation (Wright-Giemsa stain; x400 magnification).
(D) BM biopsy showing dysplastic features in the neutrophil lineage
(red arrow) and megakaryocytes with nuclear hyperchromasia and
irregular nuclear contours (green arrows) (H&E stain; x400
magnification). (E) BM biopsy revealing hypocellularity with ~50%
cellularity (H&E stain; x200 magnification). For this pediatric
patient, the normal range of the proportion of BM hematopoietic
tissue was 80-90%. Therefore, hypocellularity was considered
present. (F) High-power view demonstrating dysplastic granulocytes
(red arrows) and hypolobated megakaryocytes (green arrows); no
definitive morphological features diagnostic of refractory
cytopenia of childhood were observed (H&E staining; x400
magnification). BM, bone marrow; H&E, hematoxylin and
eosin.

Figure 2

(A and B) Normal mature neutrophils
and erythroblasts were observed, with complete resolution of
dysplastic features in both granulocytic and erythroid lineages.
(C) Morphologically normal granular megakaryocytes with adequate
lobulation were also identified (Wright-Giemsa stain; x400
magnification). (D) Following decitabine treatment, bone marrow
aspirate demonstrated active marrow hyperplasia (Wright-Giemsa
stain; x100 magnification).
View References

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Copy and paste a formatted citation
Spandidos Publications style
Fan Z, Xu N, Senin A and Zhang J: Decitabine monotherapy for pediatric myelodysplastic syndrome: A case report. Exp Ther Med 31: 172, 2026.
APA
Fan, Z., Xu, N., Senin, A., & Zhang, J. (2026). Decitabine monotherapy for pediatric myelodysplastic syndrome: A case report. Experimental and Therapeutic Medicine, 31, 172. https://doi.org/10.3892/etm.2026.13167
MLA
Fan, Z., Xu, N., Senin, A., Zhang, J."Decitabine monotherapy for pediatric myelodysplastic syndrome: A case report". Experimental and Therapeutic Medicine 31.6 (2026): 172.
Chicago
Fan, Z., Xu, N., Senin, A., Zhang, J."Decitabine monotherapy for pediatric myelodysplastic syndrome: A case report". Experimental and Therapeutic Medicine 31, no. 6 (2026): 172. https://doi.org/10.3892/etm.2026.13167
Copy and paste a formatted citation
x
Spandidos Publications style
Fan Z, Xu N, Senin A and Zhang J: Decitabine monotherapy for pediatric myelodysplastic syndrome: A case report. Exp Ther Med 31: 172, 2026.
APA
Fan, Z., Xu, N., Senin, A., & Zhang, J. (2026). Decitabine monotherapy for pediatric myelodysplastic syndrome: A case report. Experimental and Therapeutic Medicine, 31, 172. https://doi.org/10.3892/etm.2026.13167
MLA
Fan, Z., Xu, N., Senin, A., Zhang, J."Decitabine monotherapy for pediatric myelodysplastic syndrome: A case report". Experimental and Therapeutic Medicine 31.6 (2026): 172.
Chicago
Fan, Z., Xu, N., Senin, A., Zhang, J."Decitabine monotherapy for pediatric myelodysplastic syndrome: A case report". Experimental and Therapeutic Medicine 31, no. 6 (2026): 172. https://doi.org/10.3892/etm.2026.13167
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