Real-World Evidence of Treatment-Free Remission Strategies and Outcomes in Chronic Myeloid Leukemia
Simple Summary
Abstract
1. Introduction
2. Methods
3. Results
4. Discussion
5. Conclusions
Author Contributions
Funding
Institutional Review Board Statement
Informed Consent Statement
Data Availability Statement
Conflicts of Interest
References
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Baseline Characteristics | TFR Cohort (N = 39) | Full Cohort (N = 233) | ||
---|---|---|---|---|
Age in Years (Median) | 47 | Range: 20 y–65 y | 54 | Range: 20 y–72 y |
Gender (%) | ||||
Female | 69 | 63 | ||
Race (%) | ||||
Caucasian | 76 | 72 | ||
African American | 20 | 26 | ||
Asian | 4 | 2 | ||
Vital Status (%) | ||||
Alive | 89 | 81 | ||
Deceased | 11 | 11 | ||
Lost to Follow-up | 0 | 8 |
Standard-Dose Abrupt Stop (N = 21) | Standard-Dose Tapering (N = 11) | Upfront Dose Reduction (N = 7) | ||||
---|---|---|---|---|---|---|
Age in Years (Median) | 47 | Range: 20–65 y | 58 | Range: 26–61 y | 46 | Range: 28–61 y |
Gender (%) | ||||||
Female | 65 | 73 | 71 | |||
Race (%) | ||||||
Caucasian | 73 | 71 | ||||
African | 65 | 9 | 29 | |||
American | 20 | |||||
Asian | 10 | |||||
TKI prior to TFR (%) | ||||||
Imatinib | 48 | 10 | 29 | |||
Dasatinib | 33 | 45 | 71 | |||
Nilotinib | 19 | 45 | ||||
CML Related Death (%) | ||||||
No | 95 | 91 | 100 | |||
Lost to Follow-up | 5 | 9 |
Total CML-CP Patients Receiving and/or Eligible for TFR (N = 56) | |
---|---|
Number Eligible (NCCN and/or ELN) | N = 46 |
NCCN Criteria (%) | 100 (N = 46) |
ELN Criteria (%) | 96 (N = 44) |
Unknown (%) | 4 (N = 2) |
TFR Eligible and Not Attempted (%) | 30 (N = 17) |
TFR Attempted | 70 (N = 39) |
Eligible and Attempted (%) | 74 (N = 29) |
Ineligible ant Attempted (%) | 21 (N = 8) |
Unknown Eligibility and Attempted (%) | 5 (N = 2) |
Time from Diagnosis to TFR Attempt (Months) | Median = 83 (8–257) |
Total Duration on TKI Prior to TFR (Months) | Median = 87 (23–220) |
Duration of Deep Response Prior to TFR (Months) | Median = 58 (0–153) |
MMR Before TFR (%) | |
MR4 | 16 |
MR4.5 | 84 |
Number of TKIs before TFR (%) | |
1 | 82 |
2 | 13 |
3 | 5 |
Prior Therapy | Median TFR Duration (m) | Median Time to Lose TFR (m) | TFR Lost 0–6 m | TFR Lost 6–12 m | TFR Lost 1–2 y | TFR Lost >2 y | MMR Reobtained | MR4.5 Reobtained | Median Time to Reobtain MMR (m) | Disease Progression/CML-Related Death |
---|---|---|---|---|---|---|---|---|---|---|
Initial TFR for All Participants (n = 39) | 14.6 (2.0–78.0) | 5.9 (2.0–50.7) | 55.00% | 16.00% | 8.00% | 21.00% | 100.00% | 100.00% | 3.1 (0.6–8.4) | 0.00% |
Standard-Dose Abrupt Cessation (n = 21) | 25.7 (2.9–78.0) | 8.1 (2.6–34.3) | 50.00% | 16.67% | 0.00% | 33.33% | 100.00% | 100.00% | 3.7 (0.8–8.4) | 0.00% |
Imatinib (n = 10) | 30.3 (3.2–78.0) | 25.1 (2.6–31.7) | 40% | 0.00% | 0.00% | 60% | 100.00% | 100.00% | 5.1 (0.8–6.6) | 0.00% |
Dasatinib (n = 7) | 9.8 (2.9–45.6) | 7.4 (4.3–13.6) | 100.00% | 0.00% | 0.00% | 0.00% | 100.00% | 100.00% | 3.40 (2.9–5.1) | 0.00% |
Nilotinib (n = 4) | 9.7 (3.0–39.1) | 5.5 (2.8–8.2) | 50.00% | 50.00% | 0.00% | 0.00% | 100.00% | 100.00% | 3.0 (2.1–4.9) | 0.00% |
Standard-Dose Taper (n = 11) | 12.9 (2.7–61.6) | 13.6 (2.7–50.7) | 25.00% | 25.00% | 25.00% | 25.00% | 100.00% | 100.00% | 1.2 (0.6–2.8) | 0.00% |
Imatinib (n = 1) | 11.3 (8.9–25.9) | N/A | N/A | N/A | N/A | N/A | N/A | N/A | N/A | 0.00% |
Dasatinib (n = 5) | 25.6 (11.2–54.0) | 10.6 (8.5–18.5) | N/A | 40% | 20% | 40% | 100.00% | 100.00% | 1.2 (0.8–4.1) | 0.00% |
Nilotinib (n = 5) | 13.8 (2.73–61.6) | 9.7 (2.7–16.7) | 60.00% | 0.00% | 40.00% | 0.00% | 100.00% | 100.00% | 1.7 (0.6–2.8) | 0.00% |
Upfront Dose Reduction (n = 7) | 7.3 (2.0–51.6) | 3.4 (2.0–5.7) | 100% | 0.00% | 0.00% | 0.00% | 100.00% | 100.00% | 4.3 (2.5–7.5) | 0.00% |
Imatinib (n = 2) | 4.0 (2.8–5.1) | 2.83 (2.5–3.3) | 100% | N/A | N/A | N/A | 100.00% | 100.00% | 4.7 (4.3–5.1) | 0.00% |
Dasatinib (n = 5) | 19.0 (2.0–51.6) | 5.11 (4.5–20.2) | 100% | 0.00% | 0.00% | 0.00% | 100.00% | 100.00% | 2.2 (1.5–6.9) | 0.00% |
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Bourne, G.; Diebold, K.; Bascug, G.; Knapp, J.; Espinoza-Gutarra, M.; Vachhani, P.; Bachiashvili, K.; Rangaraju, S.; Mohty, R.; Bhatia, R.; et al. Real-World Evidence of Treatment-Free Remission Strategies and Outcomes in Chronic Myeloid Leukemia. Cancers 2025, 17, 2148. https://doi.org/10.3390/cancers17132148
Bourne G, Diebold K, Bascug G, Knapp J, Espinoza-Gutarra M, Vachhani P, Bachiashvili K, Rangaraju S, Mohty R, Bhatia R, et al. Real-World Evidence of Treatment-Free Remission Strategies and Outcomes in Chronic Myeloid Leukemia. Cancers. 2025; 17(13):2148. https://doi.org/10.3390/cancers17132148
Chicago/Turabian StyleBourne, Garrett, Kendall Diebold, Greg Bascug, Joshua Knapp, Manuel Espinoza-Gutarra, Pankit Vachhani, Kimo Bachiashvili, Sravanti Rangaraju, Razan Mohty, Ravi Bhatia, and et al. 2025. "Real-World Evidence of Treatment-Free Remission Strategies and Outcomes in Chronic Myeloid Leukemia" Cancers 17, no. 13: 2148. https://doi.org/10.3390/cancers17132148
APA StyleBourne, G., Diebold, K., Bascug, G., Knapp, J., Espinoza-Gutarra, M., Vachhani, P., Bachiashvili, K., Rangaraju, S., Mohty, R., Bhatia, R., & Jamy, O. (2025). Real-World Evidence of Treatment-Free Remission Strategies and Outcomes in Chronic Myeloid Leukemia. Cancers, 17(13), 2148. https://doi.org/10.3390/cancers17132148