Contributions
Abstract: PB1927
Type: Publication Only
Background
Juvenile myelomonocytic leukemia (JMML) is a chronic malignant myeloproliferative disease of early childhood
Aims
To define the status of juvenile myelomonocytic leukemia (JMML) patients in Turkey, in terms of time of diagnosis, clinical characteristics, mutational studies, clinical course and treatment strategies.
Methods
Data including clinical and labarotory characteristics and treatment strategies of JMML patients were collected retrospectively from pediatric hematology-oncology centers in Turkey.
Results
Sixty-five children with JMML diagnosed between 2002 and 2016 in 18 institutions throughout Turkey were enrolled into the study. The median age at diagnosis was 17 months (range, 2-117 months). Splenomegaly was present in 92 % of patients at the time of diagnosis. The median WBC, monocyte and platelet counts were, 32.9x109/L, 5.4x109/L and 58.3x109/L, respectively. Monosomy 7 was present in 18% of patients. JMML mutational analysis was performed in 32 out of 65 patients (49%), PTPN11 was the most common mutation. Hematopoetic stem cell transplantation (HSCT) could only be performed in 28 (44%) patients, majority of being after the year 2012. The most frequent reason for not performing HSCT was the inability to find a suitable donor. The median time from diagnosis to HSCT was 9 months (range,2-63 months). The 5-year cumulative survival rate was 33% and median estimated survival time was 30±17.4 months (95% CI:0-64.1) for all patients. Survival time was significantly better in the HSCT group (log-rank p=0.019). Older age at diagnosis (>2 years), platelets less than 40x109/L and PTPN11 mutation were the factors significantly associated with shorter survival time.
Conclusion
Although there has recently been improvement in terms of definitive diagnosis and HSCT in JMML patients, the overall results are not satisfactory and it is necessary to put more effort into this issue in our country.
Session topic: 10. Myelodysplastic syndromes - Clinical
Keyword(s): JMML, Hematopoietic cell transplantation
Abstract: PB1927
Type: Publication Only
Background
Juvenile myelomonocytic leukemia (JMML) is a chronic malignant myeloproliferative disease of early childhood
Aims
To define the status of juvenile myelomonocytic leukemia (JMML) patients in Turkey, in terms of time of diagnosis, clinical characteristics, mutational studies, clinical course and treatment strategies.
Methods
Data including clinical and labarotory characteristics and treatment strategies of JMML patients were collected retrospectively from pediatric hematology-oncology centers in Turkey.
Results
Sixty-five children with JMML diagnosed between 2002 and 2016 in 18 institutions throughout Turkey were enrolled into the study. The median age at diagnosis was 17 months (range, 2-117 months). Splenomegaly was present in 92 % of patients at the time of diagnosis. The median WBC, monocyte and platelet counts were, 32.9x109/L, 5.4x109/L and 58.3x109/L, respectively. Monosomy 7 was present in 18% of patients. JMML mutational analysis was performed in 32 out of 65 patients (49%), PTPN11 was the most common mutation. Hematopoetic stem cell transplantation (HSCT) could only be performed in 28 (44%) patients, majority of being after the year 2012. The most frequent reason for not performing HSCT was the inability to find a suitable donor. The median time from diagnosis to HSCT was 9 months (range,2-63 months). The 5-year cumulative survival rate was 33% and median estimated survival time was 30±17.4 months (95% CI:0-64.1) for all patients. Survival time was significantly better in the HSCT group (log-rank p=0.019). Older age at diagnosis (>2 years), platelets less than 40x109/L and PTPN11 mutation were the factors significantly associated with shorter survival time.
Conclusion
Although there has recently been improvement in terms of definitive diagnosis and HSCT in JMML patients, the overall results are not satisfactory and it is necessary to put more effort into this issue in our country.
Session topic: 10. Myelodysplastic syndromes - Clinical
Keyword(s): JMML, Hematopoietic cell transplantation