Efficacy of Allogeneic Hematopoietic Cell Transplantation in Patients with Chronic Phase CML Resistant or Intolerant to Tyrosine Kinase Inhibitors

Farah Yassine, Division of Hematology-Oncology and Blood and Marrow Transplantation and Cellular Therapies Program, Mayo Clinic Florida, Jacksonville, FL, USA
Tea Reljic, Research Methods and Biostatistics Core, Morsani College of Medicine, University of South Florida, Tampa, FL, USA
Muhamad Alhaj Moustafa, Division of Hematology-Oncology and Blood and Marrow Transplantation and Cellular Therapies Program, Mayo Clinic Florida, Jacksonville, FL, USA
Madiha Iqbal, Division of Hematology-Oncology and Blood and Marrow Transplantation and Cellular Therapies Program, Mayo Clinic Florida, Jacksonville, FL, USA
Hemant S. Murthy, Division of Hematology-Oncology and Blood and Marrow Transplantation and Cellular Therapies Program, Mayo Clinic Florida, Jacksonville, FL, USA
Ambuj Kumar, Research Methods and Biostatistics Core, Morsani College of Medicine, University of South Florida, Tampa, FL, USA
Mohamed A. Kharfan-Dabaja, Division of Hematology-Oncology and Blood and Marrow Transplantation and Cellular Therapies Program, Mayo Clinic Florida, Jacksonville, FL, USAFollow

Abstract

Approximately 15-20% of chronic myeloid leukemia (CML) patients fail tyrosine kinase inhibitor (TKI) therapy secondary to resistance or intolerance. In the pre-TKI era, front-line allogeneic hematopoietic cell transplantation (alloHCT) represented the standard approach for patients with chronic phase-CML (CP-CML) who were deemed fit to tolerate the procedure and had a human leukocyte antigen compatible donor available. Currently, CP-CML patients are eligible for allo-HCT only if they fail more than one TKI and/or are intolerant to the drug. We performed a systematic review/meta-analysis of the available literature to assess the evidence regarding allo-HCT efficacy in CP-CML patients. Data from eligible studies were extracted in relation to benefits (overall survival [OS], progression-free survival, disease free survival [DFS], complete remission [CR], and molecular response [MR]) and harms (nonrelapse mortality [NRM], relapse, and acute and chronic graft-versus-host disease), and stratified by age into adult and pediatric groups. For adult allo-HCT recipients, the pooled OS, DFS, CR and, MR were 84% [95% confidence interval (CI) 59e99%], 66% (95% CI 59e73%), 56% (95% CI 30e80%), and 88% (95% CI 62e98%), respectively. Pooled NRM and relapse were 20% (95% CI 15e26%) and 19% (95% CI 10e28%), respectively. For the pediatric group, the OS rate was reported in one study and was 91% (95% CI 72e99%). Our results suggest that allo-HCT is an effective treatment for TKI-resistant or TKI-intolerant CPCML. Post-transplant strategies are still needed to further mitigate the risk of relapse.

Recommended Citation

Yassine, Farah; Reljic, Tea; Moustafa, Muhamad Alhaj; Iqbal, Madiha; Murthy, Hemant S.; Kumar, Ambuj; and Kharfan-Dabaja, Mohamed A. (2022) "Efficacy of Allogeneic Hematopoietic Cell Transplantation in Patients with Chronic Phase CML Resistant or Intolerant to Tyrosine Kinase Inhibitors," Hematology/Oncology and Stem Cell Therapy: Vol. 15 : Iss. 1 , Article 6.
Available at: https://doi.org/10.1016/j.hemonc.2021.02.003