Abstract
Introduction: Despite recent treatment advances in Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ALL), survival outcomes are still dismal among underserved populations. Tyrosine kinase inhibitors (TKI) have globally improved survival, however, their access is still limited in resource-limited settings. In Peru, Ph+ALL represents a challenging entity with additional barriers, such as allogeneic stem-cell transplant access and geographical-economical disparities. This study aims to provide real-world clinical characteristics and outcomes for Ph+ ALL patients treated at a Latin American referential cancer center (Institute of Neoplastic Diseases, INEN, Lima-Peru).
Methods: A retrospective cohort study. Patients diagnosed with Ph+ ALL and treated under a BFM-like protocol (PETHEMA-09 or CALGB10403) were included between January 1st, 2010, and December 31st, 2018. Data was collected from electronic medical records. Vital status was confirmed using the national identification system (RENIEC). OS was defined as the time from diagnosis to death from any cause. Kaplan-Meier and Cox proportional hazards models were used for survival analysis.
Results: A total of 162 patients were included in the study. The median age was 33 years (range 14-68), with a female-to-male ratio of 1:1.3. Of the patients, 67% were under 40 years old, and 33% were 40 years or older. Hyperleukocytosis was present in 31% of patients. The p190 transcript was detected in 71.2% of patients, while p210 was found in 28.8%. The median follow-up was 3 years. OS at 7 years was 12% (95% CI: 3.69-25.68), with a median survival of 15 months (IQR: 8-41) and a 3-year survival of 31.17% (95% CI: 20.7-37.4). The median survival for patients aged ≥40 years was 5 months (IQR: 1-26; p < 0.005). Among 91 responders, 50 (55%) had minimal residual disease (MRD), with a median survival of 13 months (IQR: 7-33; p < 0.005).
Conclusion: This initial report remarks that Ph+ ALL still has a poor prognosis in Peru, with high mortality and limited response rates. Interventions such as improving access to allo-SCT, managing MRD positivity, and ensuring availability of second-generation TKIs are critical. We found that age and laboratory parameters significantly impact prognosis, underscoring the need for updated treatment regimens to reduce mortality. Future studies should explore the role of TKIs and analyze disease-specific mortality, particularly in adults and older adults, to improve outcomes.
