Front-Line Tyrosine Kinase Inhibitors in Pediatric Chronic Myeloid Leukemia: A Study on Efficacy and Safety
Abstract
:Simple Summary
Abstract
1. Introduction
2. Materials and Methods
2.1. Study Cohorts
2.2. TKI Treatments
2.3. Evaluation of Efficacy
2.4. Safety Analysis
2.5. Statistical Analysis
3. Results
3.1. Patient Characteristics
3.2. Efficacy
3.3. Efficacy of Dasatinib for Patients with Imatinib Resistance/Intolerance
3.4. Safety
3.5. Outcomes
4. Discussion
5. Conclusions
Supplementary Materials
Author Contributions
Funding
Institutional Review Board Statement
Informed Consent Statement
Data Availability Statement
Conflicts of Interest
References
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IMA Group (N = 33) | DSA Group (N = 18) | p-Value | |
---|---|---|---|
Age at diagnosis, median (range) | 12.1 (3.5–17.6) | 11.8 (2.8–17.5) | - |
Sex, N (%) Male Female | 23 (69.6) 10 (30.4) | 5 (27.7) 13 (72.3) | 0.004 |
CBC at diagnosis, median (range) WBC (109/L) Hemoglobin (g/dL) Platelet (109/L) | 293.7 (12.9–562.7) 9.2 (5.7–13.0) 562.0 (162.0–1767.7) | 265.7 (20.1–416.2) 8.5 (5.4–14.2) 550.0 (153.0–1558.0) | - |
Splenomegaly at diagnosis, N (%) Yes No Unknown | 23 (69.6) 6 (18.1) 4 (12.3) | 14 (77.8) 4 (22.2) 0 | - |
BCR-ABL1 transcript type, N (%) b3a2 b2a2 e14a2 Not applicable | 14 (42.4) 8 (24.2) 1 (3.0) 10 (30.4) | 12 (66.6) 2 (11.1) 1 (5.5) 3 (16.8) | - |
Switching or discontinuation of the initial TKI before 12 months, N (%) Intolerance Suboptimal response Progression | 6 (18.1) 7 (21.2) 2 (6.0) | 0 0 2 (11.1) | 0.034 |
Tanner stage, N (%) I II III IV V | 7 (21.2) 9 (27.2) 8 (24.2) 5 (15.1) 4 (12.1) | 4 (22.2) 5 (27.7) 3 (9.0) 2 (11.1) 4 (22.2) | - |
Height SDS TKI start FU 1 FU 2 | 0.14 ± 1.32 −0.27 ± 1.23 −0.44 ± 1.14 | −0.27 ± 0.81 0.05 ± 1.02 −0.48 ± 1.14 | - |
Weight SDS TKI start FU 1 FU 2 | −0.11 ± 1.41 −0.83 ± 2.24 0.03 ± 1.33 | −0.36 ± 0.73 −0.04 ± 0.63 −0.26 ± 0.89 | - |
BMI SDS TKI start FU 1 FU 2 | −0.23 ± 1.31 −0.57 ± 3.58 0.28 ± 1.27 | −0.68 ± 0.71 −0.11 ± 0.59 −0.04 ± 0.71 | - |
IMA Group (N = 33) | DSA Group (N = 18) | |||
---|---|---|---|---|
Adverse Events | Any Grade | Grade 3/4 | Any Grade | Grade 3/4 |
Hematologic toxicity | ||||
Neutropenia | 0 | 0 | 3 (16) | 3 (16) |
Thrombocytopenia | 5 (15) | 1 (3) | 2 (11) | 1 (5) |
Anemia | 1 (3) | 1 (3) | 1 (5) | 0 |
Extra-hematologic toxicity | ||||
Myalgia or arthralgia | 6 (18) | 0 | 4 (22) | 0 |
Nausea and/or vomiting | 5 (15) | 0 | 1 (5) | 0 |
Diarrhea | 0 | 0 | 1 (5) | 0 |
Abdominal pain | 2 (6) | 0 | 1 (5) | 0 |
Hematochezia | 0 | 0 | 1 (5) | 0 |
Superficial/generalized edema | 0 | 0 | 1 (5) | 0 |
Pleural or pericardial effusion | 0 | 0 | 1 (5) | 1 (5) |
Elevated AST or ALT | 2 (6) | 2 (6) | 0 | 0 |
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Yoo, J.W.; Jo, S.; Ahn, M.B.; Kim, S.; Lee, J.W.; Kim, M.; Cho, B.; Chung, N.-G. Front-Line Tyrosine Kinase Inhibitors in Pediatric Chronic Myeloid Leukemia: A Study on Efficacy and Safety. Cancers 2023, 15, 3862. https://doi.org/10.3390/cancers15153862
Yoo JW, Jo S, Ahn MB, Kim S, Lee JW, Kim M, Cho B, Chung N-G. Front-Line Tyrosine Kinase Inhibitors in Pediatric Chronic Myeloid Leukemia: A Study on Efficacy and Safety. Cancers. 2023; 15(15):3862. https://doi.org/10.3390/cancers15153862
Chicago/Turabian StyleYoo, Jae Won, Suejung Jo, Moon Bae Ahn, Seongkoo Kim, Jae Wook Lee, Myungshin Kim, Bin Cho, and Nack-Gyun Chung. 2023. "Front-Line Tyrosine Kinase Inhibitors in Pediatric Chronic Myeloid Leukemia: A Study on Efficacy and Safety" Cancers 15, no. 15: 3862. https://doi.org/10.3390/cancers15153862