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Updated: Oct 4, 2025
Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
Published on: October 3, 2018
[Clinical Characteristics and Prognosis of Children with Myelodysplastic Syndrome]
Xia Zheng1, Ji-Xin Xu2, Wei-Qin Jiang3
1Department of Pediatrics, Huashan Hospital North Fudan University, Shanghai 201900, China.
Insights
Pediatric myelodysplastic syndrome (MDS) is often refractory cytopenia. Younger age, low platelets, genetic mutations, and no hematopoietic stem cell transplantation (HSCT) indicate poor prognosis, while HSCT offers the best cure.
Area of Science:
- Pediatric Hematology
- Oncology
- Stem Cell Transplantation
Background:
- Myelodysplastic syndromes (MDS) are a heterogeneous group of clonal hematopoietic stem cell disorders.
- MDS in children is rare, with refractory cytopenia being the most common subtype.
- Accurate risk stratification and effective treatment are crucial for improving outcomes in pediatric MDS.
Purpose of the Study:
- To analyze the clinical characteristics and prognosis of pediatric myelodysplastic syndrome (MDS).
- To identify high-risk factors influencing survival in children with MDS.
- To evaluate the effectiveness of different treatment modalities, including hematopoietic stem cell transplantation (HSCT).
Main Methods:
- Retrospective analysis of 40 pediatric MDS cases diagnosed between 2011 and 2017.
- Clinical data, risk stratification (IPSS, WPSS, IPSS-R), and treatment regimens were reviewed.
- Kaplan-Meier survival analysis was used to estimate overall survival (OS) and event-free survival (EFS) rates.
Main Results:
- Refractory cytopenia was the most common MDS subtype in children.
- Patients undergoing HSCT showed significantly better 3-year OS (72.2%) and EFS (65.0%) rates compared to those without HSCT (35.3% OS, 19.2% EFS).
- High-risk factors identified included age < 7 years, initial platelet count < 50×10^9/L, complex karyotypes/gene mutations, and lack of HSCT.
Conclusions:
- Pediatric MDS, particularly MDS-RCC, requires careful prognostic evaluation.
- Age, initial platelet count, genetic abnormalities, and HSCT status are critical prognostic indicators.
- HSCT is the most effective treatment for pediatric MDS, while current risk assessment tools have limitations in this population.
Objective:
To analyze the clinical characteristics and prognosis of 40 children with myelodysplastic syndrome (MDS), and provide ideas for clinical diagnosis and treatment.
Methods:
The clinical characteristics, risk stratification, and different treatment regimens of 40 cases with MDS admitted in Department of Hematology of Children's Hospital of Soochow University from January 1, 2011 to December 31, 2017 was retrospectively analyzed. Kaplan-Meier survival curve were used to estimate 3-year overall survival (OS) rate and event-free survival (EFS) rate.
Results:
In 40 cases, the ratio of male to female was 1.4∶1.0, male was more than female, and median age was 6.0 years old. Among them, refractory cytopenia (MDS-RCC) was the most common type, and 11 cases were chromosomal abnormalities, 21 cases genetic abnormalities. Fifteen cases accepted hematopoietic stem cell transplantation (HSCT) treatment, while 25 cases did not but drug therapy alone. The 3-year OS rate of the cases who accepted HSCT or not was (72.2±12.2)% and (35.3±10.2)% (P=0.039), 3-year EFS rate was (65.0±12.9)% and (19.2±8.4)% (P=0.012), respectively. Cox regression analysis showed that age < 7 years old (P=0.0333), initial diagnosed platelet < 50×109/L (P=0.007), presence of complex karyotypes and/or gene mutations (P=0.0002), and treatment without HSCT (P=0.016) were the high-risk factors of prognosis. All the children were classified according to IPSS, WPSS and IPSS-R, while analysis result showed that the above three risk assessment had limitations for risk assessment of MDS in children, they could not comprehensively assess the prognosis of children with MDS.
Conclusion:
MDS-RCC in children is more common. Cox multivariate analysis shows that age < 7 years old, initial diagnosed platelet < 50×109/L, presence of complex karyotypes and/or gene mutation, and treatment without HSCT are the high-risk factors of prognosis in children with MDS. HSCT is the most effective treatment to cure children with MDS at present. The current methods such as IPSS-R commonly used in assessment of prognosis in children with MDS show obvious limitation.
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