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Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
Published on: September 18, 2013
Pediatric and adolescent chronic myeloid leukemia: A follow-up study in Western India
Sangita A Vanik1, Dhaval Jetly1, Biren Parikh1
1Department of Oncopathology, The Gujarat Cancer and Research Institute, Ahmedabad, Gujarat, India.
Insights
Pediatric and adolescent Chronic Myeloid Leukemia (CML) patients often present with high white blood cell (WBC) counts. Treatment response to tyrosine kinase inhibitors (TKI) showed no significant correlation with adult prognostic scores in this age group.
Area of Science:
- Hematology
- Pediatric Oncology
- Molecular Biology
Background:
- Chronic Myeloid Leukemia (CML) is a rare hematologic malignancy in pediatric and adolescent populations.
- Understanding CML in younger individuals requires specific clinical and hematopathological evaluation.
Purpose of the Study:
- To assess clinical, hematopathological, and biochemical parameters in pediatric/adolescent CML.
- To evaluate treatment response to first-line tyrosine kinase inhibitors (TKI).
- To correlate TKI response with adult prognostic scoring systems.
Main Methods:
- Retrospective analysis of 44 BCR-ABL1-positive pediatric/adolescent CML cases.
- Evaluation of clinical and laboratory data using hospital software.
- Monitoring treatment response and applying prognostic scoring systems.
Main Results:
- Patients presented with a mean age of 11.6 years and high WBC counts (63.6% >250,000/μL).
- High rates of complete hematological response (97.7%) and molecular response (78.1%) were observed.
- Most patients had low Sokal and EUTOS scores, with CHR achieved by 75% at 3 months.
Conclusions:
- Pediatric/adolescent CML cases typically present with elevated WBC counts.
- Prognostic scoring systems showed no statistically significant association with treatment response.
- Further large-scale studies are needed for TKI treatment response in this demographic.
Background:
Chronic myeloid leukemia (CML) is relatively rare in pediatric and adolescent age groups. The purpose of this study was to evaluate the clinical, hematopathological, and biochemical parameters of CML in pediatric and adolescent age groups, along with an assessment of the treatment response with first-line tyrosine kinase inhibitors (TKI) and its correlation with the prognostic scoring systems of adults.
Materials And Methods:
A retrospective study of 44 Breakpoint Cluster Region-Abelson leukemia virus (BCR-ABL1)-positive pediatric and adolescent CML cases registered at our hospital was done. The clinical and laboratory parameters were evaluated using hospital software. The treatment response was monitored and scoring was performed using mathematical calculations.
Results:
The mean age was 11.6 (±4.7) years. The median hemoglobin was 8.4 g/dL and 63.6% of the cases showed white blood cell (WBC) counts >250,000/μL. The average follow-up was 21 months. A total of 97.7 and 78.1% cases achieved complete hematological response (CHR) and molecular response, respectively, during the treatment course. The maximum number of patients had low Sokal and European treatment and Outcomes Study (EUTOS) scores. Seventy-five per cent of the cases achieved CHR at 3 months, while 73.6 and 78.6% CML-Chronic phase (CP) cases with low Sokal and EUTOS scores achieved CHR at 3 months, respectively.
Conclusion:
This study revealed that the CML cases in pediatric and adolescent age groups are normally present with higher WBC counts at the time of diagnosis. The association of the prognostic scoring system with treatment response was statistically insignificant. However, a larger cohort study is needed to determine the treatment response of TKI in children and adolescent CML and its correlation with the prognostic scoring systems.
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