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[Treatment outcome for myelodysplastic syndromes (MDS) obtained by the Polish Children's Leukemia/Lymphoma Study
A Chybicka1, P Kołecki, W Pietras
1Klinika Hematologii i Chorób Rozrostowych Dzieci AM we Wrocławiu.
Insights
Bone marrow transplant (BMT) is the best therapy for children with myelodysplastic syndromes (MDS). For those without a donor, isotretinoin (Roacutan) therapy shows effectiveness.
Area of Science:
- Pediatric Hematology
- Oncology
- Hematologic Malignancies
Background:
- Myelodysplastic syndromes (MDS) are a group of clonal hematopoietic stem cell disorders.
- MDS in children is rare but can lead to acute myeloid leukemia.
- Treatment options and outcomes for pediatric MDS have evolved over time.
Purpose of the Study:
- To evaluate the treatment outcomes for children diagnosed with myelodysplastic syndromes.
- To compare the efficacy of different therapeutic approaches for pediatric MDS.
- To identify optimal treatment strategies for children with MDS, considering donor availability for bone marrow transplantation.
Main Methods:
- Retrospective analysis of 40 pediatric patients diagnosed with MDS.
- Patients were treated across seven centers of The Polish Children's Leukemia Lymphoma Study Group between 1975 and 1998.
- Diagnosis included Refractory Anemia with Excess Blasts in Transformation (RAEB-T), Chronic Myelomonocytic Leukemia (CMML), Refractory Anemia (RA), and Refractory Anemia with Excess Blasts (RAEB).
Main Results:
- Bone marrow transplantation (BMT) is identified as the most effective therapy for pediatric MDS.
- For patients lacking a suitable donor for BMT, isotretinoin (Roacutan) therapy demonstrated significant effectiveness.
- Specific diagnostic subtypes included RAEB-T (16 children), CMML (2 children), RA (10 children), and RAEB (12 children).
Conclusions:
- Bone marrow transplantation remains the gold standard treatment for pediatric myelodysplastic syndromes.
- Isotretinoin presents a viable and effective therapeutic alternative for pediatric MDS patients ineligible for BMT due to the absence of a donor.
- Further research into non-BMT therapeutic options for pediatric MDS is warranted.
Abstract:
Fourty children with MDS treated in seven centres of The Polish Children's Leukemia Lymphoma Study Group in period 1975-1998y were included to the study. In 16 children RAEB-T, in 2 CMML in 10 RA and in 12 RAEB were diagnosed. Our and literature data showed that BMT is the best therapy for children with MDS. For children, who don't have a donor for BMT. Roacutan therapy seems to be the most effective.